Material events

Full Phase 3 ALKIVIA data in autoimmune myositis, to be presented as a late-breaking oral presentation at ACR Convergence 2026 (argenx, GlobeNewswire, November 7, 2026, 10:00 AM ET): the study met its primary endpoint in the combined immune-mediated necrotizing myopathy (IMNM) and dermatomyositis (DM) population, with a statistically significant and clinically meaningful 15.4-point greater improvement in mean Total Improvement Score (TIS) at Week 52 versus placebo (p=0.0011; 48.0 vs 32.6), and met key secondary endpoints in the combined population including response rate, time to response, muscle strength, and disease activity. IMNM patients showed a 14.8-point greater improvement in mean TIS versus placebo at Week 52 (p=0.0048; 45.1 vs 30.2), greater improvement in Manual Muscle Testing-8 (5.4 points versus placebo), and a 43.5 percentage-point greater reduction in creatine kinase versus placebo. DM patients showed a clinically meaningful 14.5-point greater improvement in mean TIS versus placebo (p=0.1093; 51.5 vs 37.0, not statistically significant in the smaller cohort) and a 5.6-point greater improvement in skin disease (CDASI) at Week 52. Efgartigimod was generally well tolerated, with a safety profile consistent with prior studies. Based on the ALKIVIA results, argenx plans to submit a Supplemental Biologics License Application (sBLA) to the FDA for efgartigimod in IMNM by the end of 2026, supported by a Breakthrough Therapy Designation. First Phase 3 study to demonstrate statistically significant and clinically meaningful benefit in IMNM, an autoimmune myositis subtype with no approved therapies.

Positive Phase 3 ICONIC-PsA 1 data for ICOTYDE (icotrokinra, targeted oral IL-23 receptor peptide) in biologic-naive adults with active psoriatic arthritis (Johnson & Johnson, November 7, 2026), presented as a late-breaking poster at ACR Convergence 2026: the study (NCT06878404) met its primary endpoint, with 58% of patients achieving an American College of Rheumatology 20 (ACR20) response compared with 41% receiving placebo at Week 16. ACR50 was 29% versus 16% and ACR70 was 13% versus 5% at Week 16. Among patients with qualifying skin involvement, 57% achieved clear or almost clear skin (IGA 0/1) compared with 14% on placebo (nominal p<0.001), and 37% achieved PASI 90 compared with 6% on placebo (nominal p<0.001). Response rates continued to increase numerically through Week 24 (ACR20 69%, ACR50 40%, ACR70 18%; IGA 0/1 64% and PASI 90 56% among patients with qualifying baseline skin involvement). Safety data through Week 24 were consistent with the established safety profile of ICOTYDE, with no new safety signals identified. ICOTYDE is not approved for the treatment of active psoriatic arthritis.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 3.02: financing-related)

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01, 2.03, 3.02: financing-related)

U.K. MHRA approved an extension of the Joenja (leniolisib) marketing authorization to include children aged 4 to 11 years weighing at least 27 kg with activated PI3K delta syndrome (APDS), with 40 mg and 50 mg twice-daily dosing, making Joenja the first treatment approved in the U.K. specifically for children with APDS. The approval expands the existing U.K. indication, previously authorized for adults and adolescents aged 12 years and older; Joenja is expected to become available for the expanded indication in February 2027. The MHRA decision was supported by data from Pharming's pediatric clinical program under the MHRA-agreed Pediatric Investigation Plan, in which leniolisib was generally well tolerated in patients aged 4 years and above.

8-K filed (items 1.01: financing-related)

Updated efficacy and pooled safety data for amsoki-cel (amsokigene autoleucel, formerly OBX-115) in immune-checkpoint inhibitor-resistant advanced melanoma, to be presented at SITC 2026 (Obsidian Therapeutics, November 6, 2026): in the Phase 1/2 Agni-01 study (NCT06060613), among 15 patients treated at the recommended Phase 2 dose (September 25, 2026 data cutoff), objective response rate was 67% (10 of 15), including 2 confirmed complete responses and 8 confirmed partial responses; median progression-free survival was 8.3 months and median duration of response was not reached at a median follow-up of 14.3 months, with 33% of patients (5 of 15) remaining in ongoing response at 12 months post-infusion. In the pooled safety set of 37 patients across Agni-01 and the Phase 1 first-in-human study (NCT05470283), no dose-limiting toxicities, treatment-related mortality, ICANS or ICU transfers were reported. Topline data from the registration-enabling Agni-01 cohort are expected by year-end 2027.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus supplement filed

Following FDA feedback, Satellos changed the primary efficacy endpoint in the BASECAMP Phase 2 trial of forazapadin in Duchenne muscular dystrophy (ambulatory boys ages 7, 8 or 9) to change in vastus lateralis fat fraction measured by MRI, replacing dynamometry, and submitted a protocol amendment to implement the new endpoint. The company said fat fraction, together with clinical endpoints, may support a potential accelerated approval pathway for forazapadin. Topline data from the 12-week placebo-controlled period are expected in Q1 2027. The release also reiterated open-label Phase 2 TRAILHEAD data in adults with DMD: a mean 3.7 percentage-point reduction in MRI-measured muscle fat fraction of the biceps brachii, from 49.7% at baseline to 46.0% at Day 140.

Offering prospectus supplement filed

Positive Phase 3 ICONIC-ASCEND data for ICOTYDE (icotrokinra, targeted oral IL-23 receptor peptide) in moderate-to-severe plaque psoriasis (adults and pediatric patients 12 years and older; Johnson & Johnson, November 6, 2026): the study (NCT06934226) met both co-primary endpoints at Week 16 versus placebo, PASI 90 55.2% versus 3.6% and IGA 0/1 70.4% versus 9.5%; across all multiplicity-controlled key secondary endpoints icotrokinra achieved non-inferiority to ustekinumab at Week 28 with numerically higher skin-clearance rates (IGA 0, IGA 0/1, PASI 90, PASI 100) in both systemic-naive and biologic-experienced patients, and a prespecified subgroup analysis showed complete skin clearance (PASI 100) of 48.1% versus 31.2% versus ustekinumab (nominal p=0.028) at Week 28; safety profile consistent with previous studies, no new safety signals. First head-to-head study of an oral pill versus an injectable biologic in plaque psoriasis.

Offering prospectus filed (possible follow-on or ATM takedown)

New translational data from the randomized Phase 2 OVATION 2 study of IMNN-001 in newly diagnosed advanced ovarian cancer, presented at SITC 2026 (IMUNON, November 6, 2026): analysis of paired patient biopsies showed IMNN-001 increased CD8+/Treg, CD8+/CD4+ and CD8+/IDO+ ratios in both tumor and stroma, which the company said provides an immunological mechanism for the 14.7-month improvement in final median overall survival reported in OVATION 2 (45.1 months versus 30.4 months with standard-of-care chemotherapy alone) and a three-month improvement in median progression-free survival. The pivotal Phase 3 OVATION 3 trial is enrolling. The release gives no new safety summary.

Phase 1/2 data for brenetafusp plus KIMMTRAK (tebentafusp) in HLA-A*02:01-positive metastatic uveal melanoma, presented at SITC 2026 (Immunocore, November 6, 2026): in 17 evaluable first-line patients on the combination, overall response rate was 29%, median duration of response 24.1 months, disease control rate 71%, median progression-free survival 8.1 months, and median overall survival was not reached at a median follow-up of 29 months, with overall survival rates of 88% at one year and 70% at two years and 10 patients remaining on study. In 16 evaluable patients on brenetafusp monotherapy in the second-line or later setting, overall response rate was 19%, median duration of response 14.5 months, disease control rate 63%, median progression-free survival 4.2 months and median overall survival 20.3 months. Safety was generally well tolerated with no new signals; cytokine release syndrome occurred in 77% of combination patients and 73% of monotherapy patients, mostly Grade 1 or 2, and no treatment-related adverse event led to discontinuation or death. The company is planning DUET-UVEAL, a randomized Phase 2/3 trial of the combination.

Peer-reviewed publication of first-in-human Phase 1 data for izicopan (formerly INF904), an oral C5a receptor inhibitor, in Clinical Pharmacology in Drug Development (InflaRx, November 6, 2026): in the randomized, placebo-controlled single- and multiple-ascending-dose study in 86 healthy participants, izicopan was well tolerated at single doses from 3 mg to 240 mg and at multiple doses up to 90 mg twice daily for 14 days, with adverse events mild or moderate and no serious adverse events. Single doses of at least 10 mg generally produced greater than 90% blockade of C5a-induced CD11b upregulation within two to four hours. The company noted the results were previously announced.

8-K filed (items 2.03: financing-related)

Registration statement filed (primary offering)

New biomarker data from the Phase 2 trial of personalized cancer vaccine EVX-01 in advanced melanoma (Evaxion, November 6, 2026): EVX-01-induced neoantigen-specific T cells were detected directly ex vivo, which the company said indicates strong EVX-01-induced immune responses; ex vivo responses were dominated by CD4+ T-cells, while CD8+ T-cell responses were observed in half of patients. The data also showed a significant association (p<0.001) between predictions by the company's AI-Immunology platform and actual T-cell responses. The biomarker data supplement the recently published three-year data showing 89% vaccine target precision and sustained T-cell and clinical responses (two-year objective response rate 75%, 12 of 16 patients, with four complete responses). The release gives no safety summary. Data presented in oral and poster sessions at SITC 2026 (abstract 502). Announced November 6, 2026 (GlobeNewswire, 11:00 ET).

Offering prospectus filed (possible follow-on or ATM takedown)

Updated interim monotherapy data from the ongoing Phase 1/2a trial of [212Pb]lapemelanotide zapixetar (VMT01) in heavily pretreated advanced melanoma, presented at SITC 2026 (Perspective Therapeutics, November 6, 2026): among 20 patients treated across three monotherapy dose cohorts as of the September 18, 2026 data cutoff (NCT05655312), at the focus dose of 3.0 mCi two of the eight patients in the monotherapy cohort had objective partial responses, one remaining in partial response approximately 40 weeks after first treatment, with a disease control rate of 75% including two patients with long-lasting disease stabilization. Across all 20 monotherapy patients, no dose-limiting toxicities, treatment-related serious adverse events, or discontinuations due to adverse events were reported; Grade 3 or higher treatment-emergent adverse events occurred in five patients (25%), none considered treatment-related.

Clinical and biomarker data from three SITC 2026 poster presentations (Candel Therapeutics, November 6, 2026): in the Phase 2 PrTK05 trial of aglatimagene plus standard-of-care radiation in intermediate-risk localized prostate cancer, CD8+ cytotoxic T cells increased at 2-6 weeks after the first injection (p<0.05), supporting the planned BLA submission. In the Phase 2a trial of aglatimagene in progressive non-squamous NSCLC, median overall survival was 25.4 months, 23 of 46 patients (50%) were alive at 24 months, and biomarker features including increases in CD40L, NCR1, CXCL5 and granzyme B were associated with survival beyond 24 months. In the Phase 1b trial of linoserpaturev in recurrent glioblastoma, median overall survival was 12.0 months in Arms A+B (50 patients) and 14.3 months in Arm C (12 patients receiving up to six administrations).

52-week open-label extension data from the Phase 2 study of HRS/BHB-1893, a next-generation cardiac myosin inhibitor, in obstructive hypertrophic cardiomyopathy, presented at AHA Scientific Sessions 2026 (Hengrui Pharma and Braveheart Bio, November 6, 2026): after the 12-week core period and a washout period, all 42 patients (NCT06516068) restarted HRS/BHB-1893 for an additional 52 weeks in the OLE. At Week 52, peak oxygen uptake (pVO2) increased by a mean of 2.7 mL/kg/min from OLE baseline, with 70% of patients achieving a ≥1.5 mL/kg/min improvement and 50% achieving a ≥3.0 mL/kg/min improvement; Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-CSS) increased by a mean of 17.3 points, with 45% achieving a ≥20-point improvement; 60% of patients achieved NYHA Class I. At Week 52, 79% of patients (33/42) achieved a Valsalva LVOT gradient below 30 mmHg, with 90% achieving a Valsalva gradient <30 mmHg at one or more visits during the treatment period; 57% of patients (24/42) were maintained on the 40 mg twice-daily (BID) starting dose. HRS/BHB-1893 was generally well tolerated across the 52-week OLE period, with no on-treatment severe or serious adverse events, all on-treatment treatment-emergent adverse events mild or moderate in severity, and no adverse events leading to permanent study drug discontinuation; two patients had transient reductions in LVEF below 50% (48% and 49% site-measured) and both resumed treatment at a lower dose. In a separate presentation, post hoc analyses of the completed Phase 2 study in non-obstructive HCM (NCT06816251, 84 patients) showed HRS/BHB-1893 improved indices of diastolic function while preserving systolic function. A planned fixed-dose Phase 2b study in oHCM (TRUEHEART-HCM) was discussed.

8-K filed (items 1.01, 3.02: financing-related)

BioCardia completed the clinical consultation process with Japan's Pharmaceuticals and Medical Devices Agency (PMDA) concerning the application materials required to obtain regulatory approval in Japan for its CardiAMP Cell Therapy System for ischemic heart failure, advancing toward submission of a marketing approval application (BioCardia, November 6, 2026). The company said completion of the consultation process does not constitute regulatory approval, and that it is targeting treatment of its first patients in Japan and commercial revenues in 2028.

Offering prospectus filed (possible follow-on or ATM takedown)

The European Medicines Agency's Committee for Orphan Medicinal Products adopted a positive opinion recommending maintenance of orphan medicinal product designation for once-weekly TransCon CNP (navepegritide) for achondroplasia in children, which would provide 10 years of orphan market exclusivity upon final adoption by the European Commission (Ascendis Pharma, November 6, 2026). The opinion follows the CHMP positive opinion recommending EU marketing authorization; the final European Commission decision on the marketing authorisation application is expected during the fourth quarter of 2026.

Offering prospectus supplement filed

Shelf registration filed (enables future raises incl. ATM)

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Positive interim data from Part 3B of the Phase 1 trial of DA-1726 (novel oxyntomodulin analog, GLP-1/glucagon dual agonist, 64 mg target dose once weekly) in otherwise healthy adults with obesity (16-week, randomized, placebo-controlled; 15 DA-1726 vs 4 placebo; presented at the Precision Liver Symposium, November 4, 2026): at Day 109, a 68% placebo-adjusted reduction in liver fat by MRI-PDFF (GMR 0.32, p<0.001), a 13.5% placebo-adjusted body weight reduction (-12.19 kg from baseline, p<0.001), and HbA1c down from 5.79% to 5.33% (-0.24 percentage points placebo-adjusted, p=0.006) in participants without diabetes; 10 of 12 DA-1726-treated participants (83%) in the MRI substudy achieved a relative liver fat reduction of at least 30% vs none on placebo, and all 5 participants with baseline steatosis achieved MRI-PDFF below 5%. Safety: gastrointestinal AEs in 9 of 15 treated participants (vomiting in 7), one serious AE of severe vomiting considered possibly related (participant recovered); no new gastrointestinal AEs after Week 9 through Day 109 and no gastrointestinal AE led to study discontinuation. Full Part 3A and Part 3B data expected in Q1 2027; results support a 48-week, non-biopsy Phase 2 study in MetALD and ALD in up to 150 patients, targeted to begin in 2H 2027. (PR Newswire, November 5, 2026)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.02: financing-related)

FDA granted accelerated approval to RYBREVANT FASPRO (amivantamab and hyaluronidase-lpuj) as a single agent for adults with recurrent or metastatic head and neck squamous cell carcinoma (HNSCC) whose disease has progressed on or after platinum-based chemotherapy and an anti-PD-(L)1 antibody (excluding HPV-associated oropharyngeal squamous cell carcinoma) (Johnson & Johnson, November 5, 2026): based on Phase 1b/2 OrigAMI-4 Cohort 1 (n=102), confirmed overall response rate 42% (95% CI 32-52), including complete responses in 15% and partial responses in 28%; 56% of responders maintained a response for more than six months. First approval of a dual EGFR/MET-targeted therapy in this setting and the first RYBREVANT FASPRO indication beyond non-small cell lung cancer; continued approval is contingent on the confirmatory Phase 3 OrigAMI-5 study.

8-K filed (items 1.01, 3.02: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01, 1.02: financing-related)

Preliminary results from a randomized, double-blind, placebo-controlled pilot study of ET-700 (extended-release zinc acetate) in healthy volunteers (36 safety, 29 per-protocol): hepatic copper uptake decreased 12% with ET-700 vs 46% with GALZIN and an 11% increase with placebo by copper-64 PET/CT (primary endpoint, post-treatment-to-baseline ratio); treatment-related adverse events in 1 of 12 ET-700 participants vs 8 of 12 on GALZIN, no serious adverse events. Eton plans additional study arms in 2027 evaluating faster-release formulations to improve copper absorption inhibition while preserving the favorable tolerability.

8-K filed (items 1.01: financing-related)

New and updated Phase 1 ELIMINATE-B data for PBGENE-HBV in chronic hepatitis B, announced ahead of a late-breaking oral presentation at AASLD The Liver Meeting 2026 (Precision BioSciences, November 5, 2026): as of the September 28, 2026 data cutoff, 25 patients had received 59 administrations across four dose levels (NCT06680232). Among 12 evaluable patients with detectable pgRNA at baseline, 11 (92%) achieved pgRNA loss to undetectable, including all six evaluable patients at the go-forward dose of 0.4 mg/kg, and 100% of patients with at least six months of follow-up maintained undetectable pgRNA. Paired liver biopsies from two patients at 0.4 mg/kg showed approximately 1-log (10-fold) and 1.5-log (35-fold) reductions in cccDNA. All 24 evaluable patients had HBsAg reductions, with maximal reductions approaching 94% at 0.4 mg/kg. No dose-limiting toxicities were observed and no serious adverse events occurred at 0.4 mg/kg; the most common adverse events were infusion-related reactions. Precision selected 0.4 mg/kg as the go-forward dose and is initiating the global ELIMINATE-B2 Phase 2 study, with dosing expected in the first quarter of 2027.

Incremental data and analyses from the ongoing Phase 1/2a trial of bamzireotide ([212Pb]VMT-alpha-NET) in unresectable or metastatic SSTR2-expressing neuroendocrine tumors, presented at NANETS 2026 (Perspective Therapeutics, November 5, 2026): among 57 gastroenteropancreatic NET patients whose target lesions all express SSTR2 in Cohorts 1-3, progression-free survival was 75% at 18 months, disease control was observed in 35 of 38 Cohort 2 patients (92%) and all 17 Cohort 3 patients (100%), and responses were seen in 14 of 38 Cohort 2 patients (37%) and 8 of 17 Cohort 3 patients (47%). Across all 68 treated patients there were no dose-limiting toxicities, no treatment-related discontinuations and no serious adverse events related to study medication; Grade 3 treatment-emergent adverse events occurred in 20 of 68 patients (29%), with no Grade 4 or 5 events. The company is targeting an End-of-Phase 2 meeting in the first quarter of 2027.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 1.02: financing-related)

Positive topline 12-week results from the Phase 1b study of ATTO-1310 (anti-IL-31 ATTOBODY, single subcutaneous dose) in chronic pruritus (CP) and high-itch atopic dermatitis (AD) (NCT06787586): in CP, PP-NRS4 response (≥4-point itch reduction) at Week 12 was 82% pooled ATTO-1310 vs 22% pooled placebo (p < 0.01), with 47% itch-free or nearly itch-free (PP-NRS<2) vs 0% placebo; approximately 95% suppression of IL-31 through Week 12 supports potential quarterly dosing. Generally well tolerated: no TEAEs led to discontinuation in CP/AD patients, one serious TEAE (worsening of pre-existing hiatal hernia) deemed unrelated, anti-drug antibodies in 7.8% with no observed impact. Global Phase 2 trials in chronic pruritus of unknown origin and high-itch AD expected to initiate in 1H 2027.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 2.03: financing-related)

Positive topline results from VOCAL, an open-label trial of three times per week (TIW) dosing of vadadustat (Vafseo) vs standard-of-care ESA (Mircera) in 353 patients with anemia due to CKD receiving in-center hemodialysis: vadadustat met the primary endpoint of non-inferiority to ESA in maintaining hemoglobin, with a mean hemoglobin difference of -0.45 g/dL against a non-inferiority margin of -0.75 g/dL; serious adverse event rates were similar (159.7 vs 183.9 events per 100 patient-years). TIW dosing is investigational; Vafseo is approved for once-daily dosing.

8-K filed (items 1.02: financing-related)

Offering prospectus supplement filed

Offering prospectus supplement filed

Selling-shareholder prospectus filed

Positive Phase 2 data for SLS009 (tambiciclib, CDK9 inhibitor) combined with azacitidine and venetoclax in relapsed/refractory AML after prior venetoclax-based therapy (GlobeNewswire, November 4, 2026): 62 heavily pretreated patients (median age 70, median 2 prior lines); across all doses (n=59) ORR 34% (12 CR/CRi, 8 MLFS), with ORR 38% in AML-MR, 44% in monocytic AML and 36% in TP53-mutated AML; at the selected dose (30 mg twice weekly), monocytic AML ORR 54% with median overall survival 5.8 months and TP53-mutated AML ORR 57% with median overall survival 8.8 months, versus roughly 2.5 months historically in these adverse-risk settings. Combination well tolerated; non-hematologic treatment-related adverse events predominantly Grade 1/2 nausea and diarrhea. Data to be presented at ASH 2026.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

New translational data from the ongoing Phase 1/2 study of pelareorep plus a checkpoint inhibitor in second-line or later unresectable squamous cell anal cancer (SITC 2026 abstract; GlobeNewswire, November 4, 2026): immune-activation biomarkers CXCL10, IFN-gamma and soluble PD-L1 rose more than two-fold after one treatment cycle in 7 of 8 evaluable patients; after three cycles, all 4 evaluable patients showed expansion of tumor-infiltrating lymphocyte clones in peripheral blood, which was associated with tumor shrinkage (2 complete responses and 2 partial responses in those four patients) and expansion of at least one HPV-16-specific T cell clone; among 14 patients evaluated for HPV-16-specific immune responses, 6 showed clonal expansion and 4 of those 6 had tumor shrinkage.

Updated ronde-cel (rondecabtagene autoleucel, CD19/CD20 CAR T) data in relapsed/refractory large B-cell lymphoma (data cutoff June 18, 2026; GlobeNewswire, November 4, 2026): PiNACLE pivotal trial in 3L+ - 58 patients treated; in 44 response-evaluable at Day 84, overall response rate 91% (40/44) and complete response rate 77% (34/44); median duration of complete response not reached (92% of complete responders still in CR at 6 months, 75% at 12 months); median PFS not reached (estimated PFS 76%/63%/54% at 6/12/24 months); no Grade 3 or higher CRS, Grade 3 or higher ICANS 9%. Phase 1/2 cohort in 2L - 43 treated; in 37 response-evaluable, ORR 95% (35/37) and CR rate 76% (28/37); median PFS not reached (75% at 6 months, 55% at 12 months); no Grade 3 or higher CRS, Grade 3 or higher ICANS 9%. Further updated results expected at ASH 2026.

8-K filed (items 3.02: financing-related)

Initial global clinical experience with gamgertamig (CM336/OM336, BCMAxCD3 T-cell engager) in relapsed/refractory autoimmune cytopenias from two parallel ongoing Phase 1b studies (NCT07083960, NCT07175493; GlobeNewswire, November 4, 2026, ASH 2026 abstract): 33 patients received short-course subcutaneous gamgertamig over a maximum of three weeks (22 immune thrombocytopenia [ITP], 11 autoimmune hemolytic anemia [AIHA]). ITP: 18/20 response-evaluable patients (90%) achieved response (platelet count ≥30 × 10^9/L and at least double baseline), with platelet normalization (≥150 × 10^9/L) in 17/18 responders (94%); median time to response 11 days; 15/18 responders (83%) had an ongoing response at median follow-up of 141 days. AIHA: all 9 response-evaluable patients achieved response (≥2 g/dL increase in hemoglobin from baseline; 6/6 mid-dose and 3/3 high-dose), median time to response 22 days, 8/9 responders (89%) ongoing at median follow-up of 85 days. Safety: Grade 1 cytokine release syndrome in 6/33 patients (18%), no Grade ≥2 CRS, no ICANS or other neurotoxicity; infections in 10/33 patients (30%), mostly Grade 1 or 2, all managed successfully. Registrational studies expected in 2027.

8-K filed (items 3.03: financing-related)

Phase 1 multiple-ascending-dose (MAD) data for BP-205 (orexin-2 receptor agonist), reported with Q3 results: 14-day double-blind placebo-controlled study in 94 healthy volunteers, once-daily 0.1 mg, 0.6 mg, 1 mg and 3 mg; mean Tmax ~60 minutes and mean effective half-life ~29 hours after 14 days, supporting once-daily dosing; generally well tolerated, headache the most common adverse event (~25% of participants), insomnia in <20% at all doses and urinary frequency/urgency in <20% at 1 mg and 3 mg (all mild, no discontinuations), no serious or severe adverse events and no liver or visual adverse events. Preliminary data; full MAD results to be presented at an upcoming medical meeting; Phase 1b study in sleep-deprived healthy volunteers data expected Q1 2027.

Registration statement filed (primary offering)

Offering prospectus supplement filed

Offering prospectus supplement filed

8-K filed (items 1.01, 3.02: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Shelf registration filed (enables future raises incl. ATM)

Initial Phase 2 data for soquelitinib (ITK inhibitor) in autoimmune lymphoproliferative syndrome (ALPS) (GlobeNewswire, November 4, 2026; NIAID-led trial, data to be presented at ASH 2026): as of August 3, 2026, three ALPS patients were enrolled and completed one year of treatment with soquelitinib 200 mg twice per day; treatment was well tolerated, with no grade 3 or 4 adverse events and no interruptions in therapy; all three patients were able to discontinue background medications including steroids and reported improvement in ALPS-related systemic symptoms; Corvus plans to explore longer treatment durations and enroll younger pediatric patients.

First clinical pharmacokinetic/pharmacodynamic data for sutacimig in congenital Factor VII deficiency (FVIID) from the single-ascending-dose cohort of the Phase 2b study HMB-001-201 (NCT07347249; GlobeNewswire, November 4, 2026, ASH 2026 abstract): as of July 24, 2026, two participants with severe congenital FVIID had received a single subcutaneous dose of sutacimig; both had a baseline prothrombin time of ≥180 seconds, which shortened by more than 50% following dosing; in the participant with PK/PD data available to date, sutacimig produced sustained accumulation (~5x) of endogenous total FVII(a), with exposure maintained through Day 22. Adverse events were low grade and assessed as not related to treatment. Updated data for all dosed participants will be presented at ASH 2026.

Offering prospectus supplement filed

8-K filed (items 3.03: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 2.03: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

New RISE UP Phase 3 data for mitapivat in sickle cell disease and first Phase 1 data for AG-236 (GlobeNewswire, November 4, 2026; to be presented at ASH 2026): in the open-label extension, of 176 patients who completed the double-blind period, 174 (118 from the mitapivat arm and 56 from placebo) enrolled; among all 118 patients continuing on mitapivat, 62.7% (n=74) achieved a hemoglobin improvement (defined as ≥1.0 g/dL increase from baseline in average hemoglobin during any two consecutive study visits) during the OLE period (treatment up to 115 weeks); of 63 double-blind non-responders, 39.7% (n=25) achieved a hemoglobin improvement with continued treatment; of 56 placebo-crossover patients, 51.8% (n=29) achieved a hemoglobin improvement; OLE safety was consistent with the double-blind period. Post hoc kidney analysis in 132 patients with hyperfiltration (eGFR >135 mL/min/1.73 m²) at baseline: mitapivat (n=92) showed a 14.7 mL/min/1.73 m² decrease in eGFR at Week 52 vs a 4.9 decrease with placebo (n=40). Post hoc cardiac analysis at Week 52: left ventricular mass index decreased 5.9 g/m² in mitapivat hemoglobin responders (n=40) vs increases of 5.8 in non-responders (n=45) and 4.6 in placebo (n=27). AG-236 (TMPRSS6 siRNA) first-in-human Phase 1 single-ascending-dose data in 24 healthy volunteers: sustained hepcidin increases and decreases in serum iron and transferrin saturation through Month 6 post-dose at 1.0, 3.0 and 9.0 mg/kg; well tolerated with no serious adverse events and no dose-limiting toxicities, supporting every-six-month dosing in polycythemia vera.

8-K filed (items 1.01, 2.03, 3.02: financing-related)

Phase 2 SKYWAY basket trial: the psoriatic arthritis (PsA) and axial spondyloarthritis (axSpA) sub-studies of SPY072 both missed their primary endpoints (ACR20 response in PsA; change from baseline in ASDAS in axSpA). SPY072 was well tolerated, and Spyre does not plan to advance SPY072 in rheumatic diseases (SPY072 development continues in hidradenitis suppurativa). Announced in the company's Q3 2026 update; PsA/axSpA topline had been guided to Q4 2026.

8-K filed (items 1.01, 3.02: financing-related)

Hoth Therapeutics, Rocket One's biotechnology subsidiary, is ending the Phase 2a trial of HT-001 (topical aprepitant for EGFR inhibitor-associated skin toxicity) early, citing strong interim efficacy results; no numeric results were disclosed in the release. Hoth plans to seek FDA feedback on advancing HT-001 into a potential Phase 3 program.

Ultragenyx will terminate development of UX701 (rivunatpagene miziparvovec, AAV9 gene therapy) for Wilson disease and complete the wind-down of all ongoing GTX-102 studies for Angelman syndrome (GlobeNewswire, November 3, 2026). UX701 Cyprus2+ dose-finding data, including the fourth dosing cohort, showed clinical activity in some patients but did not meet the criteria (majority of patients achieving biochemical response and elimination of standard of care) to advance to the pivotal Phase 3 stage; safety was favorable across all cohorts. GTX-102 wind-down follows the Phase 3 Aspire failure reported September 2, 2026. Announced with a ~20% headcount reduction as the company targets a >50% cut in 2027 R&D expense vs 2025.

Pfizer terminated the Phase 2 open-label extension study of PF-07868489, a BMP9 inhibitor in pulmonary arterial hypertension, on business grounds rather than safety or efficacy concerns, disclosed in its third-quarter 2026 update and reported by Fierce Biotech (November 3, 2026). The Phase 1/2 trial began in 2023 and the extension in 2025, and had been due to end in the second half of 2028. The same quarterly clearout confirmed the end of work on PF-06835375 (whose Phase 2 ITP termination was logged on October 20, 2026) and ended four other Phase 1 programs, including the obesity candidate PF-07999415, the Phase 1 combination study of the GIPR agonist PF-08654696, and the KRAS inhibitors PF-07985045 and PF-07934040.

Offering prospectus filed (possible follow-on or ATM takedown)

Sanofi discontinued the Phase 1 first-in-human clinical trial of SAR448272, an oral STAT6 degrader licensed from Nurix, and terminated the SAR448272 program (GlobeNewswire, November 3, 2026). Nurix does not anticipate receiving any future development, regulatory or commercial milestone payments or royalties for the program; its other licensed programs under the Sanofi collaboration are unaffected, and cash runway guidance (into H2 2029) is unchanged.

Offering prospectus filed (possible follow-on or ATM takedown)

Phase 1b results for SER-155 published in Nature Medicine: in the randomized, placebo-controlled Cohort 2 in adults undergoing allogeneic hematopoietic cell transplantation (outcomes through day 100), bloodstream infections occurred in 10% of SER-155 patients vs 42.9% on placebo, a 77% relative risk reduction (p=0.0423); mean systemic antibiotic exposure was 9.2 days vs 21.1 days (p=0.0494); febrile neutropenia 65% vs 78.6%. SER-155 was generally well tolerated with no treatment-related serious adverse events. SER-155 holds FDA Breakthrough Therapy designation for reducing bloodstream infections in allo-HCT and is Phase 2-ready pending funding.

8-K filed (items 3.02: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01, 2.03: financing-related)

8-K filed (items 1.01: financing-related)

Shelf registration amended

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus filed (possible follow-on or ATM takedown)

Shelf registration filed (enables future raises incl. ATM)

Offering prospectus supplement filed

8-K filed (items 1.01: financing-related)

AbbVie discontinued clinical development of livmoniplimab (ABBV-151), an anti-GARP-TGF-beta monoclonal antibody, after deciding its potential benefit appeared limited relative to current standards of care in solid tumors such as colorectal cancer and lung cancer; an AbbVie spokesperson confirmed the decision to Fierce Biotech (November 3, 2026). The program included a Phase 2/3 trial, begun in 2024, of livmoniplimab plus budigalimab (ABBV-181) against pembrolizumab in non-squamous non-small cell lung cancer. A Phase 1 study in hepatocellular carcinoma had reported a 42% overall response rate for the combination in 12 patients in 2024.

8-K filed (items 1.01: financing-related)

Positive topline results from the Phase 2a INFORM-MS trial of intranasal foralumab in non-active secondary progressive multiple sclerosis (GlobeNewswire, November 2, 2026): the trial met its dual primary endpoints, with a statistically significant reduction in microglial activation on TSPO PET imaging at the 50 ug dose versus placebo (p=0.0168) and safety/tolerability met at both doses; Modified Fatigue Impact Scale scores improved -6.17 points at 50 ug (p=0.035) and -7.18 at 100 ug (p=0.040) versus baseline, with no change on EDSS over 12 weeks and no deaths, drug-related serious adverse events, or discontinuations due to adverse events.

Teva will discontinue the Phase 2 TOPAS-MSA study (NCT06568237) of emrusolmin (TEV-'286) in adults with multiple system atrophy, following an independent data monitoring committee recommendation based on a prespecified interim futility analysis (GlobeNewswire, November 2, 2026): the analysis met the predefined futility criterion, indicating a low probability of the study meeting its primary efficacy endpoint if continued; no safety concerns were identified.

8-K filed (items 1.01, 2.03, 3.03: financing-related)

New Phase 2 data from the ongoing TUPELO trial of REC-4881 (oral MEK1/2 inhibitor) in familial adenomatous polyposis (FAP), presented at CGA-IGC and reported with Q3 2026 results (GlobeNewswire, November 2, 2026): after 12 weeks of REC-4881 4 mg QD, 87% of patients (13 of 15) had a reduction in total polyp burden with a 47% median reduction; median reductions were 55% in the upper GI and 50% in the lower GI; 85% of evaluable patients remained below baseline 12 weeks after stopping treatment; no new safety signals (highest-grade treatment-related adverse events Grade 1/2 in 21 of 26 patients, Grade 3 in four, no Grade 4/5). The release also gave a REC-1245 Phase 1 DAHLIA update: 2 confirmed partial responses in endometrial cancer and 1 unconfirmed partial response in liver cancer, with a 91.7% disease control rate (11/12) at doses >=45 mg, and 45 mg selected for exploratory expansion cohorts.

FDA has accepted for review the Biologics License Application (BLA) for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington's disease submitted under the Accelerated Approval pathway, granted Priority Review, and set a PDUFA target action date of May 1, 2027 (GlobeNewswire, November 2, 2026).

8-K filed (items 1.01, 2.03: financing-related)

Favorable 12-month topline data from Phase I/IIa trial ENX-CL-05-001 of Allocetra in moderate to severe knee osteoarthritis (GlobeNewswire, November 2, 2026): in patients aged 64+, 79.2% achieved a clinically meaningful response (reduced pain and improved knee function) vs 47.6% placebo at 12 months (p=0.027); benefit consistent with 3- and 6-month response rates; WOMAC pain difference vs placebo -12.2 points at 12 months (p=0.113) and function -13.1 (p=0.067); no treatment-related serious adverse events, adverse events predominantly transient mild to moderate local knee reactions; Phase IIb topline in the same 64+ population expected by end of Q2 2027.

8-K filed (items 1.01: financing-related)

FDA has placed a partial clinical hold on the IND for Developmental and Epileptic Encephalopathies (DEEs) and related Protocol BMB-101-105, the planned study of BMB-101, following the FDA's review; the company was notified by the FDA via email (GlobeNewswire, November 2, 2026).

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 1.02, 2.03, 3.02: financing-related)

Regulatory filings for ORPATHYS (savolitinib) in combination with TAGRISSO (osimertinib) have been accepted for review in Europe and Japan (GlobeNewswire, November 1, 2026). In Europe, the EMA has validated and accepted for regulatory review the Marketing Authorization Application (MAA) submitted by AstraZeneca, seeking approval for patients with locally advanced or metastatic NSCLC whose tumors have MET overexpression or amplification and who had disease progression on or after EGFR-TKI therapy. In Japan, AstraZeneca has submitted an NDA to the MHLW, also accepted for review, for unresectable, advanced or recurrent NSCLC with MET overexpression or amplification after EGFR-TKI progression. The filings follow the US FDA NDA submission in September 2026 and are supported by the positive SAFFRON global Phase III trial, which showed a statistically significant improvement in PFS (HR 0.55; 95% CI 0.42-0.72; p<0.001; median PFS 7.6 vs 5.4 months) and OS (HR 0.68; 95% CI 0.50-0.91; p=0.010; median OS 17.8 vs 12.9 months) vs doublet platinum-based chemotherapy, with no new safety concerns.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 3.02: financing-related)

Interim results from the ongoing Phase 1/2 first-in-human trial of Regeneron's RNAi therapy targeting CIDEB in adults with MASLD, reported in its Q3 2026 results (GlobeNewswire, October 30, 2026): a single dose was generally well tolerated and reduced liver fat by approximately 70% on average from baseline at 12 weeks, with effects sustained through 24 weeks of follow-up; interim results to be presented at the AASLD Meeting in November 2026, and a Phase 3 study in MASH/MASLD is planned to initiate in Q4 2026.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 2.03, 3.02: financing-related)

8-K filed (items 1.01, 2.03, 3.02, 3.03: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

FDA approved NRx's Abbreviated New Drug Application (ANDA) for Preservative-Free Ketamine, ahead of the FDA-announced November 10, 2026 approval goal date (GDUFA, FDA Office of Generic Drugs). The product contains no benzethonium chloride (BZT), is manufactured in the US with Nephron Pharmaceuticals (West Columbia, SC) using blow-fill-seal technology, and commercial supply is on hand; ketamine injection has been on the FDA drug shortage list since 2018 and is used primarily for anesthesia and pain control, including in labor and delivery. Separate NRX-100 program (IV ketamine for depression/suicidality) still seeks NDA approval.

FDA approved INO-3107, branded Voceryq, a DNA-based immunotherapy targeting HPV types 6 and 11, for recurrent respiratory papillomatosis (RRP) (Reuters, October 30, 2026 — the PDUFA target action date; Inovio's first approved product). The application was supported by an early- to mid-stage study in adults who had undergone at least two or more surgeries in the previous year. The most commonly reported adverse reactions were injection site reactions, headache, fatigue, hypertension, nausea, and dizziness, per the FDA. Inovio plans to commercialize the therapy on its own in the United States, supported by Syneos Health.

EMA validated the Marketing Authorization Application (MAA) for varegacestat, an investigational oral once-daily gamma secretase inhibitor (GSI), for the treatment of adults with desmoid tumors (BusinessWire, October 30, 2026); validation confirms the application is complete and the EMA will initiate review under the centralized marketing authorization procedure. The MAA is supported by the global Phase 3 RINGSIDE trial (NCT04871282), which met its primary endpoint of progression-free survival with an 84% reduction in the risk of disease progression or death (hazard ratio = 0.16, p<0.0001) and an objective response rate of 56% vs 9% with placebo (p<0.0001). MAA validation follows FDA acceptance of the varegacestat NDA in July 2026, with a PDUFA target action date of April 28, 2027.

FDA-aligned partial clinical hold on the two KITE-753 (anti-CD19/CD20 CAR-T) DLBCL trials, Phase 2 PALISADES-1 (second-line) and Phase 3 PALISADES-2 (third-line plus relapsed/refractory), announced on Gilead's Q3 earnings call October 29, 2026 (Fierce Biotech, October 30, 2026): after an initial voluntary pause, Gilead aligned with the FDA on a partial clinical hold to investigate safety differences noted between Phase 1 and Phase 2, specifically grade 3 and grade 4 ICANS in a small number of patients; no deaths or grade 5 adverse events.

Shelf registration filed (enables future raises incl. ATM)

Registration statement filed (primary offering)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.02: financing-related)

FDA extended the PDUFA goal date for the mitapivat sNDA in sickle cell disease by three months to February 1, 2027, after classifying Agios's response as a major amendment (GlobeNewswire, October 30, 2026). The sNDA was submitted under the accelerated approval pathway with Priority Review and an original PDUFA date of November 1, 2026, based on the RISE UP Phase 2 and Phase 3 trials in patients aged 16 years and older. On October 9, 2026 the FDA requested additional information to evaluate whether a REMS is necessary; Agios responded October 19 with approximately nine months of updated safety data through October 9, 2026 from the RISE UP open-label extension periods, with no cases of hepatocellular injury showing a pattern and severity similar to those seen in thalassemia trials and the benefit-risk profile unchanged. The FDA informed Agios of the major-amendment classification on October 28, 2026. Labeling discussions, including elements of a potential REMS, continue.

Phase 2 ALTITUDE-AD trial of sabirnetug (ACU193) in early Alzheimer's disease did not meet its primary endpoint (GlobeNewswire, October 30, 2026): topline data showed no meeting of the primary endpoint of change from baseline in cognitive and functional decline as measured by iADRS at 18 months versus placebo at either the 35 mg/kg or 50 mg/kg dose; key secondary endpoints including CDR-SB also did not achieve statistical significance, with no meaningful difference versus placebo. Sabirnetug was generally well tolerated, with ARIA-E in ~10% of treated participants, and showed statistically significant amyloid plaque reduction by PET SUVr at week 36 and week 76. Acumen plans to cease clinical development of sabirnetug, including the open-label extension, preserve cash and explore strategic options. Trial NCT06335173 enrolled 542 participants with early AD.

Positive Phase 1 results for XEN1701 (NaV1.7 inhibitor) and XEN1120 (KV7 opener) in healthy participants, presented in two posters at IASP 2026 (GlobeNewswire, October 29, 2026): both Phase 1 SAD/MAD studies achieved their goals, supporting advancement to Phase 2 proof-of-concept studies in acute pain expected to initiate in H1 2027. XEN1701 (SAD/MAD/food-effect/CSF cohorts, n=76 on drug and n=22 placebo): proposed Phase 2 dose levels achieved and maintained more than 75% NaV1.7 receptor occupancy in both the peripheral and central nervous system with twice-daily dosing, the level human genetics associates with a pain-free phenotype; no treatment-related dose-limiting, serious, or discontinuation treatment-emergent adverse events, and no notable autonomic cardiovascular pattern. XEN1120 (SAD/MAD/food-effect cohorts, n=54 on drug and n=18 placebo): plasma levels exceeded predicted efficacious exposures from preclinical models; no treatment-related dose-limiting, serious/severe, or discontinuation TEAEs, most TEAEs mild (in MAD cohorts tachycardia 33.2% vs 12.5% placebo, dizziness 29.2% vs 0%, somnolence 25% vs 0%). Preclinical cynomolgus data for compounds comparable to XEN1701/XEN1120 showed significant reductions in capsaicin-evoked heat pain (about 85-100% at the two highest XEN1701-comparable doses at >75% occupancy).

Submitted a New Drug Application (NDA) to the FDA for remibetadex (Trappsol Cyclo, intravenous hydroxypropyl-beta-cyclodextrin) for the treatment of the neurovisceral manifestations of Niemann-Pick Disease Type C (NPC) in adult and pediatric patients from birth (GlobeNewswire, October 29, 2026), via subsidiary Cyclo Therapeutics, LLC. The NDA is supported by the Phase 3 TransportNPC study, which did not achieve statistical significance on its primary endpoint (64% relative slowing of disease progression vs placebo, p=0.19) but showed 71% relative slowing in a prespecified analysis of patients receiving background therapy (approximately 83% of the study population, p=0.046), plus earlier clinical studies and an overall survival analysis showing an 85% reduction in mortality risk in infantile-onset NPC vs matched registry controls (p=0.044). Generally well tolerated with no new safety signals; serious adverse events 35.9% vs 16.7% on placebo. Remibetadex holds Orphan Drug, Fast Track and Rare Pediatric Disease designations; FDA will determine within 60 days whether the application is filed and whether Priority Review is granted, and if filed will assign a PDUFA target action date. The USAN Council assigned the nonproprietary name remibetadex.

8-K filed (items 1.02: financing-related)

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01: financing-related)

FDA cleared the Investigational New Drug (IND) application for VT-7208 for a Phase 2 study in chronic spontaneous urticaria (CSU) (GlobeNewswire, October 29, 2026). VT-7208 is a next-generation, CNS-penetrant, once-daily oral Bruton's tyrosine kinase (BTK) inhibitor acquired with Vidya Therapeutics. The clearance enables a planned multicenter, randomized, double-blind, placebo-controlled Phase 2 study of approximately 120 adults with moderate-to-severe CSU not adequately controlled with H1 antihistamines, evaluating VT-7208 versus placebo over 12 weeks for efficacy, safety, tolerability and pharmacologic activity. Additional Phase 2 proof-of-concept programs are planned in food allergy and relapsing multiple sclerosis. The Vidya acquisition was accompanied by an approximately $200 million private placement, and the company expects current cash resources to fund operations into the second half of 2029.

8-K filed (items 1.01, 1.02: financing-related)

Shelf registration filed (enables future raises incl. ATM)

Eli Lilly discontinued the Phase 2 program for mevidalen (LY3154207) in symptomatic Alzheimer's disease, disclosed in its third-quarter 2026 earnings update, which listed mevidalen among the assets removed from the pipeline (Fierce Biotech, October 29, 2026). A Lilly spokesperson told Fierce Biotech the program "did not meet our high internal bar for success"; Lilly has not disclosed detailed results from the Alzheimer's trial and has not clarified whether the decision affects mevidalen's development in other indications. The Phase 2 trial, begun in August 2024, evaluated mevidalen in 304 patients with mild-to-moderate Alzheimer's disease dementia, assessing safety and effects on cognition, daily activities and other symptoms, and completed about six months ago. Mevidalen is a dopamine D1 receptor positive allosteric modulator; in 2021 it failed to meet primary and secondary cognitive endpoints in a separate Phase 2 trial in Lewy body dementia.

Positive results from the completed first-in-human Phase 1 healthy volunteer trial of KT-579 (oral IRF5 degrader), reported with third-quarter 2026 financial results (GlobeNewswire, October 29, 2026): the trial met or exceeded its objectives and established proof of mechanism. In the multiple ascending dose portion, KT-579 showed deep, sustained IRF5 degradation in blood, with ≥90% median degradation across multiple dose levels and saturation of degradation by dose level 3; similar reductions in IRF5 levels were observed in skin. Ex vivo-stimulated IRF5-dependent biomarkers at Day 14 (median of all MAD dose cohorts) showed inhibition of 88% of TNFα, 87% of IL-12/23p40, 94% of IL-1β, and 98% of IFNβ, with broad downregulation of inflammatory cytokine, interferon and B-cell associated gene transcripts. Safety was comparable to placebo: no serious or severe adverse events, no AEs leading to discontinuation, very few mild treatment-related AEs, and no dose-dependent pattern in treatment-emergent AEs. The LUMORA Phase 1b trial of KT-579 in approximately 30 adults with systemic lupus erythematosus (16 weeks of dosing) is planned to start by early 2027. The company also reported the last patient completed the week 16 visit in the KT-621 (STAT6) BROADEN2 Phase 2b atopic dermatitis trial, with topline data to be reported by year-end 2026, and $1.6 billion in cash, cash equivalents and investments as of September 30, 2026, with runway into 2029.

8-K filed (items 1.01: financing-related)

Updated results from Cohorts 1 and 2 of the Phase 2 IOV-LUN-202 trial of lifileucel (TIL cell therapy) in previously treated non-squamous metastatic NSCLC (GlobeNewswire, October 29, 2026; data cutoff October 14, 2026): objective response rate 24.6% (17 of 69 patients; 15 confirmed responses and 2 unconfirmed partial responses), consistent with the 25.6% ORR previously reported in 39 patients. KRAS wild-type subgroup ORR was 43.3% (13 of 30) versus 11.4% (4 of 35) in KRAS-mutant disease, with both complete responses in KRAS wild-type tumors; a biomarker-enriched subgroup (KRAS wild-type, HER2 wild-type, current or former smoker) achieved a 59.1% ORR (13 of 22). Response durability and safety remained consistent with previous reports, with an updated reduced-dose lymphodepletion regimen improving the overall safety profile. Cohorts 1 and 2 are completing enrollment (approximately 80 patients expected in the final dataset) and support a planned sBLA for lifileucel in non-squamous mNSCLC in 2027; POSITILITY, a prospective biomarker-enriched cohort with registrational intent, begins enrollment in November 2026. In frontline Cohort 3A of IOV-COM-202, 6 of 7 biomarker-matched patients responded to lifileucel plus pembrolizumab (ORR 85.7%).

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 3.02: financing-related)

FDA cleared the Investigational New Drug (IND) application for exaluren for the treatment of nonsense-mutation autosomal dominant polycystic kidney disease (ADPKD) (PR Newswire, October 29, 2026). The clearance enables Eloxx to open U.S. clinical sites for EXPRESS, its global Phase 2 study of exaluren in nonsense-mutation ADPKD. EXPRESS will randomize approximately 60 adults with genetically confirmed PKD1 or PKD2 nonsense mutations 1:1 to exaluren or placebo for 52 weeks, with the annualized rate of change in height-adjusted total kidney volume (htTKV, by MRI) as the primary endpoint; first patient dosing is expected in 3Q 2027 and topline 52-week data in 1H 2029. In written pre-IND feedback, FDA indicated that a substantial and durable htTKV effect, with supporting eGFR and safety data, could support Breakthrough Therapy Designation, and FDA lists change in htTKV as a potential surrogate endpoint for accelerated approval in ADPKD. Exaluren, a small-molecule ribosomal modulator designed to promote readthrough of premature stop codons, holds orphan drug designation for ADPKD in the United States and Europe.

Bristol Myers Squibb discontinued development of BMS-986495 (PRX019), an investigational monoclonal antibody for neurodegenerative diseases licensed from Prothena in May 2024 for $80 million upfront and up to $617 million in milestones plus royalties (BioSpace, October 29, 2026). Prothena completed the Phase 1 trial of PRX019 in healthy adults. A BMS spokesperson told BioSpace: 'Following review of Phase 1 data, we have decided not to advance this program.' BMS did not disclose the Phase 1 findings or what in the data prompted the decision. The broader neuroscience collaboration with Prothena continues, including moponetug (PRX005), a tau-targeting antibody in a Phase 2 trial in Alzheimer's disease.

8-K filed (items 2.03: financing-related)

Shelf registration filed (enables future raises incl. ATM)

Shelf registration filed (enables future raises incl. ATM)

FDA accepted for filing and granted Priority Review to the New Drug Application (NDA) for QTORIN 3.9% rapamycin anhydrous gel (QTORIN rapamycin) for the treatment of microcystic lymphatic malformations (microcystic LMs); PDUFA target action date February 28, 2027 (GlobeNewswire, October 28, 2026). QTORIN rapamycin could become the first FDA-approved therapy for microcystic LMs, a rare disease affecting an estimated more than 30,000 diagnosed children and adults in the U.S. The NDA is supported by the Phase 3 SELVA study, which achieved statistical significance across all six prespecified efficacy endpoints (all p<0.001), with 95% of participants aged 6 years and older who completed the efficacy evaluation period demonstrating improvement on the mLM-IGA primary endpoint, and Phase 2 results that supported Breakthrough Therapy designation. QTORIN rapamycin was well tolerated in SELVA, with no drug-related serious adverse events reported and systemic rapamycin concentrations below 2 ng/mL at all measured timepoints for all participants. Palvella is preparing for a potential standalone U.S. commercial launch in the first half of 2027, subject to FDA approval.

Positive topline results from TRANSCEND-T2D-2 (NCT06260722), a Phase 3 head-to-head trial of retatrutide vs injectable Ozempic 2 mg in adults with type 2 diabetes inadequately controlled with metformin, with or without an SGLT2 inhibitor (PR Newswire, October 28, 2026): 1,298 participants randomized 2:2:1 to retatrutide 9 mg, retatrutide 12 mg or Ozempic 2 mg. At 80 weeks retatrutide met the primary and all key secondary endpoints. Change in A1C from an average baseline of 8.3%: -2.2% (9 mg) and -2.2% (12 mg) vs -1.8% with Ozempic. Percent change in body weight from an average baseline of 102.4 kg (225.8 lbs; BMI 36.8 kg/m2): -19.5% (-20.1 kg; -44.3 lbs) at 9 mg and -20.9% (-21.5 kg; -47.4 lbs) at 12 mg vs -9.4% (-9.5 kg; -20.9 lbs) with Ozempic (p<0.001 vs Ozempic for the family-wise controlled comparisons). Retatrutide also delivered greater improvements than Ozempic across certain cardiovascular risk factors, including non-HDL cholesterol, triglycerides and systolic blood pressure. Most common adverse events with retatrutide (9 mg, 12 mg vs Ozempic) were diarrhea (28.3%, 32.7% vs 26.4%), nausea (21.2%, 23.8% vs 21.3%), constipation (14.2%, 13.1% vs 19.8%), vomiting (10.2%, 15.0% vs 13.2%) and dyspepsia (10.4%, 8.5% vs 11.6%); dysesthesia incidence was 4.8%, 6.5% vs 2.7% and urinary tract infections 5.8%, 4.2% vs 5.8%. Most events were mild to moderate. Treatment discontinuation rates due to adverse events were 4.8% (9 mg) and 7.9% (12 mg) vs 2.7% with Ozempic. TRANSCEND-T2D-2 is the sixth Phase 3 trial in which retatrutide has delivered efficacy in Lilly's program; detailed results will be presented at a future medical meeting.

Offering prospectus supplement filed

Registration statement amended

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01: financing-related)

Positive topline results from the global Phase 3 LiGeR-HN1 trial (NCT06525220, Business Wire, October 28, 2026) of petosemtamab (EGFR x LGR5 bispecific antibody) in combination with pembrolizumab versus pembrolizumab alone in first-line PD-L1 positive recurrent/metastatic head and neck squamous cell carcinoma (HNSCC): at the first interim analysis assessed by an independent data monitoring committee (IDMC), the combination met both dual primary endpoints, demonstrating a statistically significant and clinically meaningful improvement in overall survival (OS: HR 0.50 (95% CI, 0.37, 0.67); p<0.0001, a 50% reduction in the risk of death) and objective response rate (ORR: odds ratio 3.17 (95% CI, 2.06, 4.89); p<0.0001) versus pembrolizumab, regardless of HPV status. The safety profile of petosemtamab in combination with pembrolizumab was generally well tolerated and consistent with the previously reported safety profiles of the individual agents, with no new safety signals identified. Genmab plans to engage global regulatory authorities and will submit the full results for presentation at a future medical meeting.

Discontinued the Phase 3 IMpactMF trial of imetelstat in JAK inhibitor relapsed/refractory myelofibrosis (GlobeNewswire, October 28, 2026): Geron announced the decision to discontinue IMpactMF based on a recommendation from the independent Data Monitoring Committee (DMC). The independent DMC advised Geron that the trial was unlikely to meet its primary endpoint of overall survival. There were no new safety signals observed in the trial that change the known safety profile of imetelstat or require additional safety monitoring. IMpactMF evaluated 327 patients with intermediate-2 or high-risk myelofibrosis who were relapsed or refractory to a JAK inhibitor, randomized 2:1 in an open-label, controlled trial designed to evaluate a potential improvement in overall survival compared to best available therapy; patients in the imetelstat arm received 9.4 mg/kg IV every three weeks. A detailed analysis of the data will be conducted and study findings will be shared with the scientific community at a future date. Imetelstat is marketed as RYTELO, approved in the U.S. and EU for certain patients with lower-risk myelodysplastic syndromes (LR-MDS).

Interim clinical data from the TREGCHECK program evaluating tagmokitug, its investigational cytolytic CCR8 antibody, in combination with toripalimab, with or without chemotherapy (GlobeNewswire, October 28, 2026): First-line ESCC (Phase 1b TREGCHECK 102 Cohort C; tagmokitug 700 mg plus toripalimab, 5-fluorouracil and cisplatin; as of October 14, 2026, 10 patients safety evaluable and 8 efficacy evaluable): confirmed ORR 75% (6/8) irrespective of PD-L1 status and 100% in PD-L1 positive patients; subsequent to the data cutoff, two additional partial responses were confirmed, increasing the confirmed ORR to 89% (8/9); 1 confirmed complete response. Second-line ESCC (Cohort B; randomized tagmokitug 300 mg or 700 mg plus toripalimab; 17 safety evaluable, 8 efficacy evaluable): in the 700 mg arm, clinical benefit rate 33.3% (2/6), confirmed ORR 16.7% (1/6) and median PFS 5.5 months. Other cohorts: 2L HNSCC HPV-positive confirmed ORR 13.0% (3/23) and CBR 26.1% (6/23); 2L upper GI (GC/GEJ/EAC) at 700 mg confirmed ORR 23.5% (4/17); 4L+ non-liver-metastatic MSS CRC confirmed ORR 5.9% and CBR 11.8%. Safety: in more than 120 patients treated with tagmokitug in combination with toripalimab, no new safety signals beyond those associated with PD-1/immune checkpoint inhibitor therapy and no Grade 5 treatment-related adverse events; overall immune-related adverse events 40% (all grades) and 20% Grade 3-5; most common treatment-related adverse events were infusion-related reactions (25.0%), fatigue (19.2%) and rash (15.8%); monotherapy in 33 patients showed no dose-limiting toxicities through 1200 mg every three weeks. Coherus plans to expand enrollment in first-line and second-line ESCC cohorts. Tagmokitug is being evaluated in Phase 1b/2a trials TREGCHECK 101 (NCT05635643) and TREGCHECK 102 (NCT06657144).

Positive interim clinical results from the ongoing BB-301 Phase 1b/2a first-in-human study (NCT06185673) in dysphagic patients with oculopharyngeal muscular dystrophy (OPMD) (GlobeNewswire, October 28, 2026): updated analyses include 12-month post-treatment follow-up for all six patients in Cohort 1 (low dose) and available follow-up for all three patients in Cohort 2 (high dose; 9 months for the first patient, 3 months for the second and third). 100% of patients treated with BB-301 across both doses are formal Responders to therapy (all six Cohort 1 and all three Cohort 2 patients). At the 3-month post-treatment time-point, 67% of Cohort 1 and 100% of Cohort 2 patients met the statistical criteria for response for improved throat emptying ability (videofluoroscopic evaluation of post-swallow food and liquid residue), and 17% of Cohort 1 and 67% of Cohort 2 patients met the criteria for improved functional swallowing capacity (Cold-Water Timed Drinking Test); 100% of patients in both cohorts met the criteria for improved total Sydney Swallow Questionnaire score and 67% in both cohorts for improved throat closing ability. There have been no Serious Adverse Events in any patient treated with BB-301 to date. Benitec is advancing toward a pivotal study in mid-2027.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01: financing-related)

Positive topline results from the Phase 3 Study 3601 (VP-VLY-686-3601) of NEREUS (tradipitant), an oral neurokinin-1 (NK-1) receptor antagonist, for prevention of vomiting associated with GLP-1 receptor agonist therapy (PR Newswire, October 27, 2026): randomized, placebo-controlled study using a single 1 mg semaglutide dose without titration (n=142 tradipitant, 164 placebo). The study met its primary endpoint (any vomiting): 43.0% (61/142) vs 58.5% (96/164) on placebo (p=0.006, adjusted for site), a 27% relative reduction. Key secondary (any vomiting with worst nausea >=3): 33.1% (47/142) vs 50.6% (83/164) (p=0.002), a 35% relative reduction. Frequent multi-day vomiting (>2 episodes on more than one day): 6.3% vs 15.9% (p=0.008), 60% relative reduction; extreme vomiting (>10 total episodes): 2.8% vs 14.6% (p=0.0003), 81% relative reduction (higher-burden analyses post hoc/supportive). Generally well tolerated, safety consistent with prior studies, no new safety signals. Vanda plans to discuss the results with the FDA and is planning a supplemental NDA (sNDA) for prevention of vomiting associated with GLP-1 receptor agonist therapy. Announced October 27, 2026 (06:50 ET, PR Newswire).

Shelf registration filed (enables future raises incl. ATM)

Positive clinical and molecular results from the first five patients to complete treatment in the expanded access study (NCT06478641) of tafenoquine-containing combination regimens (tafenoquine plus atovaquone and other antimalarial or antibiotic medications) in relapsing babesiosis in immunosuppressed patients (GlobeNewswire, October 27, 2026): all five completed patients (100%) reported being much improved or very much improved on study-completion forms and had negative conventional PCR test results for Babesia documented during treatment or subsequent follow-up; four of the five (80%) were also nonreactive on a highly sensitive blood-donor nucleic acid amplification test (NAT) for Babesia performed on blood collected 66-120 days after completion of tafenoquine treatment, while the remaining patient had a reactive blood-donor NAT despite a negative follow-up conventional PCR result from Mayo Clinic. Two additional patients remain on treatment, bringing total enrollment to seven, and the study remains open pending potential discussions with the FDA. The Company plans to pursue accelerated approval if the efficacy section of its registration dossier is based primarily on data from immunosuppressed patients with relapsing babesiosis, proposing post-treatment blood-donor NAT nonreactivity as a surrogate endpoint; the separate randomized, placebo-controlled study of tafenoquine added to standard of care in patients hospitalized for babesiosis (NCT06207370) is expected to unblind in January 2027, after which the Company intends to request a pre-sNDA meeting. Tafenoquine is FDA-approved for malaria prophylaxis as ARAKODA but is not FDA-approved for babesiosis.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

Positive topline results from the Phase 3 MagnetisMM-32 study of ELREXFIO (elranatamab) monotherapy in relapsed/refractory multiple myeloma (Business Wire, October 27, 2026): at the pre-specified interim analysis for efficacy, the trial met its primary endpoint of progression-free survival (PFS) by blinded independent central review, demonstrating a statistically significant and clinically meaningful reduction in the risk of disease progression or death versus investigator's choice of elotuzumab, pomalidomide and dexamethasone (EloPd), pomalidomide, bortezomib and dexamethasone (PVd), or carfilzomib and dexamethasone (Kd). The study enrolled 523 adults across 26 countries who had received at least one prior line of treatment with anti-CD38 therapy (daratumumab or isatuximab) and lenalidomide. Median PFS was not yet reached; more than 75% of patients receiving ELREXFIO had not experienced disease progression or death at the time of the analysis, with an estimated 68% PFS at 18 months. Overall survival, a key secondary endpoint, was immature, with follow-up continuing to the next pre-specified OS analysis. Safety was consistent with the known profile of ELREXFIO and no new safety signals were identified; Grade 3 or higher infections occurred in 27.5% of patients receiving ELREXFIO vs 18.1% on investigator's choice, and Grade 5 infections in 0.8% vs 1.6%. Pfizer plans to submit these data to global health authorities.

Updated data from Cohort 4 of the Phase 1/2 GOBLET study of pelareorep in combination with atezolizumab (Tecentriq) in second-line or later squamous cell anal cancer (SCAC) (GlobeNewswire, October 27, 2026): median overall survival had not yet been reached, and median duration of response was approximately 15 months, versus a historical 9.5 months for FDA-approved retifanlimab monotherapy in a similar population; all patients who responded survived beyond 12 months, including multiple patients surviving over 24 months. The release reports no objective response rate, patient count, or safety figures for the cohort. More than 90% of SCAC cases are associated with human papillomavirus (HPV). Following recent FDA interactions and receipt of Fast Track designation, the Company plans to explore additional clinical studies with a potential checkpoint inhibitor partner to support registrational development in SCAC, believes the durable responses may support an accelerated approval pathway with overall survival supporting full approval (subject to FDA agreement), and sees potential expansion into HPV-associated head and neck squamous cell carcinoma, where HPV accounts for approximately 70% of oropharyngeal cancers in the United States and pelareorep has not yet been evaluated in combination with a checkpoint inhibitor.

Novartis discontinued the Phase III study of remibrutinib (oral BTK inhibitor, marketed as Rhapsido) in generalized myasthenia gravis (gMG) following a futility analysis which indicated the treatment was unlikely to meet its efficacy objectives, disclosed in the R&D update of its third-quarter 2026 results release (GlobeNewswire, October 27, 2026). No safety concerns were identified, and Novartis said there is no impact on remibrutinib studies in other indications. The gMG study is the Phase 3 RELIEVE trial (NCT06744920), a randomized, double-blind, placebo-controlled study of remibrutinib versus placebo on stable standard of care, with change in MG-ADL total score at Month 6 as the primary measure. Remibrutinib remains in development in relapsing multiple sclerosis after the positive Phase III REMODEL-1 and REMODEL-2 results and is FDA-approved in chronic spontaneous urticaria and symptomatic dermographism.

Registration statement filed (primary offering)

8-K filed (items 1.01, 2.03, 3.03: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

Updated Phase 1/2 data for BEAM-301, an in vivo base editor correcting the R83C mutation in glycogen storage disease type Ia (GSDIa), presented at ESGCT 2026 (data cutoff August 17, 2026; updates the October 13, 2026 release, data cutoff June 26, 2026): 6 patients received a single infusion (3 at 20 mg, 3 at 40 mg), median follow-up 7.6 months (range: 1.9–16.6 months). Dose-dependent improvements in time to fasting hypoglycemia: median increased from 2.9 hours at baseline to 4.3 hours at Day 36 in the 20 mg cohort and from 2.5 hours to 15.0 hours in the 40 mg cohort; 2 of 3 patients in the 40 mg cohort achieved the protocol-defined maximum controlled fasting duration of 15 hours and achieved a 100% reduction in daily starch requirements, no longer requiring starch supplementation. Safety consistent with LNP-based therapies: no dose-limiting toxicities, Grade 4 or 5 treatment-emergent adverse events, or treatment discontinuations; 4 of 6 patients experienced serious TEAEs considered related to BEAM-301, including 3 Grade 3 infusion-related reactions; other serious TEAEs of diarrhea and rhabdomyolysis resolved; transaminase elevations were mostly Grade 1 (one Grade 2 AST) and resolved. Beam is advancing into the 60 mg dose cohort (enrollment underway) and plans to engage the FDA on a potential path to approval. Announced October 27, 2026 (07:00 ET, GlobeNewswire).

Artiva discontinues development of AlloNK (AB-101) and evaluates strategic alternatives (GlobeNewswire, October 27, 2026): updated clinical data continued to show deep B-cell depletion and clinical activity consistent with previously reported findings, but longer-term follow-up showed durability of clinical response did not meet the threshold the Company required to support continued development of AlloNK plus rituximab in rheumatoid arthritis and other B-cell-driven autoimmune diseases. Two recent Grade 5 events were reported: a Phase 2a basket-trial participant experienced fatal leukoencephalopathy following study treatment (lymphodepletion with cyclophosphamide and fludarabine), assessed by the investigator and an independent safety review as likely related to fludarabine-associated neurotoxicity; Artiva reported it to the FDA, investigators and the DSMB, after which the FDA asked Artiva to pause enrollment and dosing in the Phase 2a study, and the Company also paused planned Phase 3 enrollment and dosing. A second participant experienced sudden death approximately two months after initiating study treatment, assessed as unrelated to AlloNK or any other component of the study treatment, with the cause of death not determined. No additional patients will be dosed in any ongoing AlloNK clinical study and the planned Phase 3 pivotal trial will not be initiated; the clinical development program will be closed out in coordination with the FDA, investigators and IRBs. Artiva is reducing its workforce by approximately 94% and has engaged Leerink Partners as financial advisor for an evaluation of strategic alternatives, which may include a merger, business combination, sale or licensing of assets, or dissolution and liquidation. Cash, cash equivalents and investments were approximately $319.1 million as of September 30, 2026 (preliminary and unaudited). The previously announced ACR Convergence 2026 data presentation will not proceed and the investor webcast scheduled for November 9, 2026 is cancelled.

Positive primary analysis results from the Phase 1b proof-of-concept (POC) trials of ATI-052 (anti-TSLP/IL-4Ralpha bispecific antibody) in atopic dermatitis (AD) and asthma (GlobeNewswire, October 27, 2026; also furnished as EX-99.1 to the October 27, 2026 8-K, accession 0001104659-26-118952). Both trials achieved the primary objective (safety and tolerability). AD trial: randomized (3:1), double-blind, placebo-controlled, 14 patients with severe AD given five weekly 480 mg doses of ATI-052 or placebo (weeks 0-4), primary analysis at day 57 (4 patients excluded from the prespecified per-protocol analysis). EASI: 45% placebo-adjusted mean change from baseline at day 57 (absolute change 58% in the active arm vs 13% on placebo); 78% placebo-adjusted EASI-50 at day 57. Itch (PP-NRS): 43% placebo-adjusted mean improvement at day 57 (38% absolute improvement in the active arm vs a 5% worsening on placebo), with relief emerging in the first week. Pharmacodynamics: 95% normalization in the lesional skin proteome at week 8. Asthma trial: randomized (3:1), double-blind, placebo-controlled, 16 patients with mild-to-moderate asthma and baseline FeNO of at least 35 ppb given a single 480 mg dose, endpoints through day 29. FeNO: 59% mean absolute reduction (45 ppb) at day 15 vs 15% on placebo, and a 40% reduction at day 29; 58% of ATI-052 patients achieved FeNO normalization (<=25 ppb) at day 15. FEV1: placebo-adjusted and absolute mean improvement of 200 ml at day 15; in a post hoc subgroup with baseline FEV1 <80% and reversibility, absolute mean FEV1 improvement was 350 ml at day 15 vs a worsening of -140 ml on placebo. Safety: treatment-emergent adverse events were predominantly Grade 1, there were no Grade 3 or 4 TEAEs and no adverse events led to study discontinuation; injection site reactions were transient, almost exclusively Grade 1 and at low frequency; no conjunctivitis was observed. Aclaris intends to prioritize Phase 2b development of ATI-052 initially in eosinophilic esophagitis (EoE), with a Phase 2b trial planned to initiate early next year.

Announced pricing of an underwritten public offering of 7,785,714 shares of common stock at $3.50 per share and, in lieu of shares of common stock to certain investors, pre-funded warrants to purchase 6,500,000 shares of common stock at a purchase price of $3.49999 per share, for expected gross proceeds of approximately $50.0 million, before deducting underwriting discounts and commissions and other offering expenses (GlobeNewswire, October 27, 2026, 06:43 ET). The offering includes participation from TCGX, RA Capital Management, Invus and a large mutual fund. The offering is expected to close on October 28, 2026. Leerink Partners, Piper Sandler, Cantor and LifeSci Capital are acting as joint bookrunning managers.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

AbbVie submitted a Biologics License Application (BLA) to the U.S. FDA for etentamig, a second-generation BCMA x CD3 bispecific antibody T-cell engager administered intravenously, for the treatment of adults with relapsed or refractory multiple myeloma (PR Newswire, October 27, 2026, 08:00 ET). The submission is supported by the global Phase 3 CERVINO study (NCT06158841) in triple-class exposed relapsed or refractory multiple myeloma after at least two prior lines of therapy (393 patients randomized 1:1 vs investigator's choice of standard available therapies): etentamig met both dual primary endpoints, with overall response rate 74.0% vs 45.7% and a 60% reduction in the risk of progression or death versus standard available therapies. The CERVINO topline results were previously announced September 3, 2026. Most common treatment-emergent adverse events in the etentamig arm (at least 25%) were cytokine release syndrome, hypogammaglobulinemia, neutropenia and anemia; among patients receiving a single step-up dose, CRS was 28.3%, all grade 1 (23.9%) or grade 2 (4.4%), with no grade 3 or higher CRS and one grade 1 ICANS event; grade 3/4 infections 27.7% vs 19.2% and grade 5 infections 1.5% vs 3.1% versus standard therapy; TEAE discontinuations 3.6% vs 14%. Etentamig is dosed monthly (Q4W) after a single step-up dose.

PRESENTATION DATED OCTOBER 26, 2026 Exhibit 99.1 ® Designing Proteins Delivering Medicines Updated Results from Dose Expansion Cohorts in the Phase 1 Study of XmAb819 October 26, 2026 2 Today's Speakers Bassil Dahiyat, Ph.D. President & Chief Executive Officer Xencor Management Guest Speaker...

Registration statement filed (primary offering)

Registration statement filed (primary offering)

8-K filed (items 1.02: financing-related)

8-K filed (items 1.01, 1.02, 3.02: financing-related)

Shelf registration amended

Offering prospectus supplement filed

FDA expanded approval of TALZENNA (talazoparib) plus XTANDI (enzalutamide) to men with metastatic androgen pathway modulation-naive or -sensitive (mAPMN/S, also known as metastatic hormone-sensitive) prostate cancer with homologous recombination repair (HRR) gene mutations, including both BRCA and non-BRCA mutations (Business Wire, October 26, 2026), based on the pivotal Phase 3 TALAPRO-3 study (NCT04821622, 599 patients): the combination reduced the risk of radiographic progression or death by 52% versus placebo plus XTANDI (HR 0.48; 95% CI 0.36-0.65; p<0.0001), with an estimated 77% of patients remaining progression-free at three years and consistent benefit across BRCA and non-BRCA subgroups. Overall survival data were immature at the rPFS analysis. The most common grade 3 or higher treatment-emergent adverse event was anemia, reported by 51% in the TALZENNA plus XTANDI group and 3% in the control group; 5% of patients discontinued TALZENNA due to anemia. The application was reviewed under FDA Priority Review and Project Orbis; an application is also under review by the EMA.

, DATED OCTOBER 26, 2026 Exhibit 99.1 Oruka Therapeutics Announces Positive Week 16 Results for ORKA-001 from the Ongoing EVERLAST-B Trial in Moderate-to-Severe Psoriasis 61% PASI 100 and 86% PASI 90 in the ORKA-001 high-dose (600 mg) group, highly similar to EVERLAST-A Continued favorable...

, DATED OCTOBER 26, 2026 Exhibit 99.1 Onconetix Announces Reverse Stock Split CINCINNATI, Ohio, Oct. 26, 2026 (GLOBE NEWSWIRE) -- Onconetix, Inc. (Nasdaq: ONCO) (the “Company”), today announced that its Board of Directors has approved a one-for-ten (1-for-10) reverse stock split of the Company’s...

NVCT: Positive expanded Phase 1b data for NXP200 (second-generation paradox-breaker BRAF inhibitor, study run by partner Haisco in China) in BRAF V600-mutated recurrent or progressive glioma: ORR by RANO 2.0 of 40.0% (8/20, including 3 complete responses) in high-grade glioma and 42.9% (3/7, including 1 CR) in low-grade glioma; median OS 17.15 months in high-grade glioma; US IND submission planned by end of 2026; presented at ESMO 2026.

MRK: Phase 3 KEYNOTE-C93 first results: KEYTRUDA monotherapy in first-line dMMR advanced or recurrent endometrial cancer produced a statistically significant 43% reduction in the risk of disease progression or death vs platinum-doublet chemotherapy (PFS HR 0.57; median PFS 38.2 vs 8.4 months); OS trend HR 0.77 (immature); presented at ESMO 2026 Presidential Symposium and published in The Lancet (NCT05173987).

MRK: Phase 3 TroFuse-005 first results: sacituzumab tirumotecan (TROP2 ADC, with Kelun-Biotech) in advanced/recurrent endometrial cancer after platinum chemotherapy and anti-PD-1/L1 therapy met both primary endpoints with statistically significant improvements: risk of death reduced 32% vs physician's-choice chemotherapy (OS HR 0.68; median OS 19.0 vs 12.1 months) and risk of progression or death reduced 41% (PFS HR 0.59; median 7.2 vs 3.9 months); ORR 35.1% vs 17.3%; FDA accepted the BLA for priority review with a PDUFA date of June 15, 2027; presented at ESMO 2026 and published in The Lancet (NCT06132958).

Positive results from Cohort 2 of the pivotal PROGRESS study (Business Wire, October 26, 2026) of ATEBRIOZ (zilurgisertib), an oral activin receptor-like kinase 2 (ALK2) inhibitor, in pediatric patients aged 6 to <12 years with fibrodysplasia ossificans progressiva (FOP): Cohort 2 enrolled 25 patients randomized 1:1 to once-daily oral zilurgisertib 75 mg (n=12) or placebo (n=13) during a 24-week double-blind period. At Week 24, mean total heterotopic ossification (HO) lesion volume decreased by 8.09 cm3 in patients receiving zilurgisertib compared with an increase of 9.14 cm3 in patients receiving placebo (treatment difference of -14.0 cm3, nominal p=0.003), and no zilurgisertib-treated patients developed new HO lesions (vs 1 (7.7%) on placebo). Clinician-assessed joint mobility (CAJIS) improved by a mean of 0.80 points on zilurgisertib compared with 0.09 points on placebo. Zilurgisertib was generally well tolerated with no new safety signals and no growth plate closures in zilurgisertib-treated patients. Mirum plans a supplemental New Drug Application (sNDA) submission in H1 2027 to expand the indication to pediatric patients aged 6 to <12 years.

Positive interim data from the Phase 2 LINNET study (NCT06730347, GlobeNewswire, October 26, 2026) of single-agent lorigerlimab (PD-1 x CTLA-4 bispecific DART molecule) in advanced gynecologic cancers, presented at the ESMO Congress 2026: among 16 response-evaluable patients with clear cell gynecologic cancer (CCGC), the confirmed objective response rate (ORR) was 25% (4/16) and the disease control rate was 44% (7/16), with all responses ongoing at the September 1, 2026 data cutoff; median PFS in the 17 safety-evaluable CCGC patients was 2.9 months (95% CI, 1.9-6.2) and the PFS rate at 6 months was 33%. All 41 treated patients (platinum-resistant ovarian cancer n=24, CCGC n=17) were evaluable for safety: Grade >=3 treatment-related adverse events (TRAEs) occurred in 49%, there was one Grade 5 TRAE in the PROC cohort, and TRAEs led to discontinuation in 24% of all patients (18% in CCGC patients). MacroGenics is enrolling 20 additional CCGC patients at 3 mg/kg Q3W and anticipates updated results in the first half of 2027.

Offering prospectus supplement filed

OF LONGEVERON INC., DATED OCTOBER 26, 2026 Exhibit 99.1 Longeveron Announces Updated Results from ELPIS II Phase 2b Clinical Trial Evaluating Laromestrocel as a Potential Treatment for Hypoplastic Left Heart Syndrome (HLHS) Important Secondary Clinical Outcome Endpoint MACE Achieved Statistical...

IOVA: Lifileucel pilot trial data in advanced soft tissue sarcomas (metastatic undifferentiated pleomorphic sarcoma and dedifferentiated liposarcoma): confirmed ORR of 50% among the first 8 evaluable patients (4 partial responses, all ongoing); registrational SARATOGA Phase 2 enrolling (NCT07741877); presented at ESMO 2026.

Positive Phase 3 SANOVO full results presented at ESMO 2026 (Proffered Paper session, abstract LBA69): ORPATHYS (savolitinib) + TAGRISSO (osimertinib) showed a statistically significant and highly clinically meaningful improvement in progression-free survival vs TAGRISSO alone in treatment-naive EGFR-mutated locally advanced or metastatic NSCLC with MET overexpression in China (n=326), with median PFS 24.9 vs 13.8 months in the intention-to-treat population (HR 0.56, p<0.0001) and 24.8 vs 11.1 months in the high-MET (IHC 3+) population (HR 0.45), and median OS 44.5 vs 34.9 months (HR 0.48). No new safety concerns were reported, and a supplemental NDA in China is planned for the first-line setting.

Positive results from the pivotal Phase 3 ARTEMIS-011 trial of risvutatug rezetecan (Ris-Rez), a B7-H3-targeted antibody-drug conjugate licensed from Hansoh Pharma, in patients in China with osteosarcoma that had progressed or relapsed after at least two prior lines of systemic therapy (GSK, October 26, 2026), presented as a late-breaking abstract in a Presidential Symposium session at ESMO Congress 2026: the trial met its primary endpoint of Independent Review Committee-assessed progression-free survival (PFS) versus chemotherapy (gemcitabine plus docetaxel), reducing the risk of disease progression or death by 67% after a median follow-up of 7.1 months (HR 0.33; 95% CI 0.20-0.55; p<0.0001), with median PFS of 5.5 months on Ris-Rez versus 1.4 months on chemotherapy. Patients were randomised 1:1 to Ris-Rez 12.0 mg/kg every 3 weeks or gemcitabine 1,000 mg/m2 on days 1 and 8 plus docetaxel 75 mg/m2 on day 8 every 3 weeks. Safety was consistent with prior findings in this tumour type: Grade 3 or higher treatment-related adverse events occurred in 93% of patients receiving Ris-Rez and 83% receiving chemotherapy, most commonly haematologic events, and treatment-related adverse events led to discontinuation in 4% versus 2%. The results will support a regulatory submission by Hansoh in China. GSK holds exclusive rights to Ris-Rez outside mainland China, Hong Kong, Macau and Taiwan, and its global Phase 2 trial in advanced osteosarcoma and soft tissue sarcoma is ongoing. This is Hansoh's second positive Phase 3 study for Ris-Rez in China.

Registration statement filed (primary offering)

Updated results from the Phase 2 trial (NCT03526835, GlobeNewswire, October 26, 2026) of petosemtamab (EGFR x LGR5 bispecific antibody) plus pembrolizumab in first-line recurrent/metastatic PD-L1+ head and neck squamous cell carcinoma (r/m HNSCC), presented at ESMO Congress 2026: in the 1L combination arm, median overall survival was 28.2 months, overall response rate was 63% and median progression-free survival was 9.0 months in efficacy-evaluable patients (n=43), including a complete response rate of 19%. As of May 15, 2026, median follow-up was 25.5 months; 12-month OS rate was 79%, median duration of response was not reached, with an estimated 55% of responders maintaining a response at 24 months, and median PFS was 9.0 months (95% CI, 5.2-12.9; 12-month PFS rate 40%). Responses were observed across HPV-positive and HPV-negative disease. Safety: most common treatment-emergent adverse events (all grade/Grade 3-4) were asthenia (51%/7%), acneiform dermatitis (51%/7%) and rash (49%/2%); infusion-related reactions occurred in 38% (7% Grade 3-4) and all resolved; no Grade 5 events or treatment-related deaths were reported. The Phase 3 program in 1L HNSCC is ongoing, with topline data anticipated later this year.

Equillium Announces First Subject Dosed in Phase 1 Clinical Trial of EQ504 Topline Phase 1 proof-of-mechanism data anticipated in Q2 2027 Randomized, double-blind, placebo-controlled study will evaluate safety, tolerability, PK and PD of EQ504 Triggers Milestone Closing of $20 million in...

New positive patient-reported outcomes data from the Phase 2 BESTOW clinical trial of tegoprubart (anti-CD40L antibody) versus standard of care tacrolimus following kidney transplantation (GlobeNewswire, October 26, 2026), featured in an oral presentation at the American Society of Nephrology (ASN) Kidney Week 2026 Annual Meeting: tegoprubart-treated patients reported continued reductions in total MTSOSD-59R symptom occurrence from baseline at Months 6 and 12, whereas symptom occurrence in the tacrolimus arm returned to slightly above baseline at Month 12, and tegoprubart demonstrated greater symptom improvement than tacrolimus in 22 of 23 domains where differential improvement was observed. On the KDQOL-SF Symptoms and Problems subscale, mean change from baseline at Month 12 was 9.0 points on tegoprubart versus 3.3 points on tacrolimus (p<0.05); the SF-12 mental component summary score increased by 1.5 points in the tegoprubart arm compared with a decline of 1.6 points in the tacrolimus arm, a 3.1-point between-group difference.

Updated data from TeLuRide-005 (NCT06246110), an open-label Phase 2 trial of EIK1001 (TLR7/8 dual agonist) plus pembrolizumab and histology-appropriate chemotherapy in previously untreated stage 4 non-small cell lung cancer, presented at the ESMO Congress 2026 on October 26, 2026 (GlobeNewswire): in the efficacy-evaluable population (n=65; median follow-up 17.2 months, efficacy cutoff October 1, 2026) the objective response rate was 63.1% and disease control rate 89.2%, with the squamous cohort (n=29) at 72.4% ORR, 100% DCR and median duration of response 12.9 months and the non-squamous cohort (n=36) at 55.6% ORR, 80.6% DCR and median duration of response 11.2 months, and responses across all PD-L1 tumor proportion score subgroups. In the safety-evaluable population (n=72), Grade 3 or higher treatment-related adverse events occurred in 48.6%, treatment-related adverse events led to discontinuation in 6.9%, cytokine release syndrome was all low-grade, and no treatment-related adverse events led to death.

CORT: Positive results from Phase 2 BELLA Part A (single-arm, n=95): Lifyorli (relacorilant) + nab-paclitaxel + bevacizumab in platinum-resistant ovarian cancer showed median PFS of 7.2 months and ORR of 59% by investigator assessment, median OS not reached, 88% of patients alive at 6 months; in bevacizumab-naive patients median PFS was 8.1 months and ORR 66%; no new safety signals.

Climb Bio Presents Initial PrisMN Phase 2 Data from Budoprutug in pMN at an Oral Session at ASN Kidney Week 2026 Low dose (200 mg) budoprutug demonstrated B-cell depletion, PLA2R Ab reduction, and immunologic remission at 12 weeks, consistent with Phase 1b data; additional data from the PrisMN...

Final positive data from the Phase IIa clinical study of namodenoson (selective A3 adenosine receptor agonist) in advanced pancreatic ductal adenocarcinoma (GlobeNewswire, October 26, 2026), presented at the European Society for Medical Oncology (ESMO) Congress 2026: the single-center, open-label study enrolled 20 patients who received namodenoson 25 mg twice daily (14 third-line, three second-line, three fourth-line) and achieved its primary safety endpoint, with only five adverse events in three patients considered related or possibly related to namodenoson, all mild to moderate (Grade 1-2), no Grade >=3 treatment-related adverse events and no treatment-related serious adverse events. In a post hoc exploratory analysis of eight third-line patients who survived beyond two months, median overall survival was 6.28 months and median progression-free survival was 2.27 months; one second-line patient achieved progression-free survival of 16.39 months and overall survival exceeding 20.7 months as of the September 23, 2026 data cutoff. The company noted the subgroup was selected based on survival with no randomized comparator, so the findings are hypothesis-generating.

Topline results from the DYNASTY-Breast02 global Phase 3 trial (NCT06018337, GlobeNewswire, October 26, 2026): trastuzumab pamirtecan met its primary endpoint with a statistically significant, yet modest improvement in progression-free survival (PFS) versus investigator's choice of chemotherapy in chemotherapy-naive patients with HR+/HER2-low metastatic breast cancer who progressed on endocrine therapy (541 patients randomized 1:1). At the interim analysis, overall survival data were not mature. A higher rate of interstitial lung disease (ILD) was reported versus prior trials, mostly Grade 1 and 2, with severe and fatal events (Grade 3-5) in a small proportion of patients; otherwise the safety profile was consistent with prior trials and no new safety signals were observed. The companies said the available data suggest the potential benefit to patients would be limited in the current HR+/HER2-low metastatic breast cancer treatment landscape and that they will discuss the data and determine next steps.

POSITIVE Phase 3 PANKU-Lung01 (NCT06382116) prespecified interim analysis (SystImmune/Biokin with Bristol Myers Squibb): iza-bren, a first-in-class EGFRxHER3 bispecific ADC, vs platinum-based chemotherapy in EGFR-mutated non-squamous NSCLC previously treated with an EGFR TKI (n=216 vs 206) MET the primary endpoint: median PFS by blinded independent central review 11.1 vs 5.7 months (HR 0.40; 95% CI 0.31-0.51; p<0.0001) at 14.6 months median follow-up; confirmed ORR 60.2% vs 34.5%; median duration of response 10.4 vs 5.7 months; overall survival immature with a favorable trend. Safety: grade >=3 treatment-related adverse events predominantly hematologic, 1.9% discontinued due to TRAEs, adjudicated ILD in 2 patients (0.9%). Presented as a late-breaking oral presentation at ESMO 2026 with simultaneous publication in The Lancet (joint release published Oct 26, 2026).

ArriVent Announces Initial Phase 1 Data for CDH17-targeted ADC ARR-217 in Patients with Gastrointestinal Cancers at ESMO Congress 2026 · 44.8% ORR at 6mg/kg dose in late-line CRC patients · ARR-217 was generally well-tolerated · ArriVent to host virtual webinar on Monday, October 26, 2026, at 8...

Aptevo Reports Mipletamig Combination Therapy Enabled 21% of Frontline AML Patients to Advance to Potentially Curative Stem Cell Transplant Ten patients initially deemed unfit for intensive chemotherapy proceeded to stem cell transplant Transplant rate compares favorably with historical...

Shelf registration filed (enables future raises incl. ATM)

REGN: Longer-term Phase 3 C-POST data (median follow-up 44 months) for adjuvant Libtayo (cemiplimab) in high-risk cutaneous squamous cell carcinoma after surgery and radiation: the overall survival benefit deepened to a 37% reduction in the risk of death (HR 0.63; 95% CI 0.36-1.11) despite 88% placebo crossover after recurrence; DFS benefit sustained, including in MRD-negative patients (DFS HR 0.45); presented at ESMO 2026.

New first-line data on olomorasib, Lilly's investigational next-generation KRAS G12C inhibitor, at ESMO Congress 2026 (PR Newswire, October 25, 2026): in a poster presentation of updated results from the Phase 1/2 LOXO-RAS-20001 trial and the safety lead-in portion of the Phase 3 SUNRAY-01 trial, olomorasib plus pembrolizumab without chemotherapy in 85 first-line KRAS G12C-mutant NSCLC patients showed median progression-free survival and median duration of response not yet reached after a median follow-up of 20.7 months, with durable activity across PD-L1 levels and an 18-month PFS rate of 55% overall and 65% in patients with PD-L1 expression of 50% or higher; the most common Grade >=3 treatment-related adverse events were increased ALT (15.3%), increased AST (12.9%) and diarrhea (8.2%), with dose reductions in 31.8%, discontinuation of the regimen in 11.8% and no Grade 5 events. In a separate rapid oral presentation, olomorasib plus chemoimmunotherapy (pembrolizumab, pemetrexed and platinum chemotherapy) in 77 first-line patients with a median follow-up of 18.7 months achieved an investigator-assessed confirmed objective response rate of 62% (48/77), a median duration of response of 14.5 months and a median PFS of 11.6 months. Lilly expects the first randomized Phase 3 data from SUNRAY-01 (NCT06119581) in first-line KRAS G12C-mutant NSCLC in the first half of 2027.

Positive Phase 3 SAFFRON (NCT05009836) full results presented at the ESMO 2026 Presidential Symposium and simultaneously published in the New England Journal of Medicine: ORPATHYS (savolitinib) + TAGRISSO (osimertinib) showed a statistically significant and clinically meaningful improvement over doublet platinum-based chemotherapy in EGFR-mutated NSCLC with high MET overexpression/amplification after progression on TAGRISSO (n=338), with median PFS 7.6 vs 5.4 months (HR 0.55, p<0.001) and median OS 17.8 vs 12.9 months (HR 0.68, p=0.010). Grade 3 or higher treatment-emergent adverse events occurred in 64% of patients on the combination vs 63% on chemotherapy, with no new safety concerns.

Updated follow-up analyses from the Phase 2 trial (NCT03526835, BusinessWire, October 25, 2026) of petosemtamab (EGFR x LGR5 bispecific antibody) plus FOLFOX or FOLFIRI in metastatic colorectal cancer (mCRC), presented at ESMO Congress 2026: among 79 treated patients (45 first-line, 34 second-line; data cutoff May 15, 2026), confirmed and unconfirmed objective response rate was 75% in 1L and 67% in 2L, with an ORR of 81% in 1L patients with left-sided tumors; disease control rate was 95% in 1L and 93% in 2L; median follow-up was 5.9 months (1L) and 7.1 months (2L), with 76% (1L) and 74% (2L) of patients remaining on treatment at cutoff. Responses were also seen in patients with liver metastases (ORR 76% 1L, 67% 2L). Safety: petosemtamab-related TEAEs led to discontinuation in 2 of 42 patients (5%) on petosemtamab + mFOLFOX6 and 1 of 37 (3%) on petosemtamab + FOLFIRI; no TEAEs leading to death; infusion-related reactions occurred in 48% (FOLFOX) and 43% (FOLFIRI), grade 3 in 7% and 3% respectively.

BNTX: First data from the Phase 2 ROSETTA Lung-107 trial (with Bristol Myers Squibb): pumitamig (PD-L1xVEGF-A bispecific) + docetaxel in metastatic NSCLC progressing after first-line immunotherapy plus chemotherapy showed a confirmed ORR of 36.7% and DCR of 90.0% in 30 efficacy-evaluable patients; median PFS 6.9 months in 50 treated patients; presented at ESMO 2026 (NCT06841055).

ZYME: Positive Phase 1 data for ZW191 (FR-alpha ADC) from the randomized dose-optimization portion in heavily pretreated platinum-resistant ovarian cancer: confirmed ORR 64.5% at 6.4 mg/kg in high-grade serous PROC (55.3% at 9.6 mg/kg), disease control rate 100%/92.1%, median duration of response not reached, 6-month PFS rate 61%; presented at ESMO 2026.

PBLS: Updated Phase 1/2 data for zolucatetide (beta-catenin:TCF inhibitor) in desmoid tumors: ORR 72.2% (13/18) at the 32-week landmark among patients starting at >=450 mg, disease control rate 100% across 46 response-evaluable patients, no discontinuations due to TRAEs; registrational PEDESTAL Phase 3 planned to start 1H 2027 (Phase 1/2 NCT05919264); presented at ESMO 2026.

First Phase 1 clinical data for sofetabart mipitecan (Sofe-M), Lilly's investigational FRalpha-targeting ADC, plus bevacizumab in platinum-sensitive ovarian cancer (PSOC), presented in an oral presentation at ESMO Congress 2026 (PR Newswire, October 24, 2026): the cohort enrolled 33 women with PSOC (median three prior systemic regimens; prior PARPi 67%, prior bevacizumab 70%) treated with Sofe-M 4 mg/kg plus bevacizumab 15 mg/kg. As of the July 6, 2026 data cutoff, the confirmed objective response rate among the 29 patients with measurable disease was 79.3% (23/29; 6 complete responses [20.7%] and 17 partial responses [58.6%]) and the disease control rate was 100%, with responses consistent across FRalpha expression levels and prior PARPi exposure and a confirmed response rate of 79% or higher in both PARPi-treated and PARPi-naive patients. Median duration of response and median progression-free survival were not reached at median follow-ups of 6.2 and 10.2 months, respectively. Safety was consistent with the known profiles of each medicine with no new safety signals: the most common treatment-emergent adverse events were epistaxis (73%), nausea (73%), anemia (58%), neutropenia (55%) and fatigue (52%); the most common Grade >=3 events were neutropenia (42%) and anemia (24%); dose reductions occurred in 42% of patients and 6% discontinued due to an adverse event. Sofe-M is being studied in the FRAmework-01 (NCT07213804) global Phase 3 program in platinum-resistant and platinum-sensitive ovarian cancer.

Updated Phase 1b data for IMA402 (PRAME TCR bispecific, NCT05958121; data cutoff June 1, 2026; 126 heavily pretreated patients treated) and Phase 1 data for IMA203CD8 (PRAME TCR T-cell therapy, NCT03686124; data cutoff August 7, 2026; 98 enrolled, 90 efficacy-evaluable), presented in two oral presentations at ESMO Congress 2026 (GlobeNewswire, October 24, 2026). IMA402 at the recommended Phase 2 dose range (10-45 mg): confirmed ORR 25% (10/40) and disease control rate 63%; in PRAME-high tumors confirmed ORR was 32% (10/31) overall and 32% (7/22) in melanoma, with median duration of response 12.5 months, median overall survival not reached and a 1-year overall survival rate of 91% in PRAME-high melanoma; most frequent treatment-related adverse events were transient lymphopenia and low-grade cytokine release syndrome, with no ICANS and no IMA402-related Grade 5 events. IMA203CD8 one-time infusion: ORR 51% (46/90), confirmed ORR 41% (36/87) and disease control rate 82% across tumor types; in gynecologic cancers at clinically relevant dose levels (n=20) confirmed ORR was 55% (11/20) with median duration of response 8.4 months; safety was manageable with transient cytopenias and low-grade cytokine release syndrome and no IMA203CD8-related Grade 5 events.

Positive interim results from the Phase II AZUR-1 trial of Jemperli (dostarlimab) monotherapy in adults with stage II and III MSI-H/dMMR locally advanced rectal cancer (GSK, October 24, 2026), to be presented in a late-breaking oral session at ESMO Congress 2026: in an exploratory analysis, 76% of evaluable patients (110/144; 95% CI: 69%, 83%) achieved a complete response to dostarlimab alone, including 68% (98/144; 95% CI: 60%, 76%) who achieved a sustained clinical complete response for at least 12 months (cCR12) without surgery as assessed by investigators, plus 12 additional patients whose complete response was confirmed pathologically after surgery. AZUR-1 met its primary objective; on the primary endpoint of cCR12 by independent central review, 47% of evaluable patients (54/116; 95% CI: 37%, 56%) had achieved cCR12 at the time of analysis, with the data still maturing and 38 additional participants already at clinical complete response but not yet at the 12-month assessment. No disease recurrences were reported among responders after a median follow-up of more than two years. Safety was consistent with the established profile: serious adverse reactions in 20% of patients, permanent discontinuation due to adverse reactions in 3%, and the most common adverse reactions were fatigue (32%), rash (22%), diarrhoea (18%) and pruritus (17%). The data supported the recent US approval of Jemperli in this indication, and regulatory reviews are ongoing through Project Orbis.

AMGN: Phase 3 DeLLphi-305 hit its primary endpoint: IMDELLTRA (tarlatamab) + IMFINZI (durvalumab) as first-line maintenance in extensive-stage SCLC reduced the risk of death by 58% vs durvalumab alone (OS HR 0.42; median OS 21.1 vs 12.7 months); PFS HR 0.34 (median 7.4 vs 1.9 months); presented at ESMO 2026 Presidential Symposium and published in NEJM.

8-K filed (items 1.01: financing-related)

First data from the global Phase 1b trial (ZL-1310-001, NCT06179069) of zocilurtatug pelitecan (zoci, formerly ZL-1310), a DLL3-targeted antibody-drug conjugate, in combination with atezolizumab with and without carboplatin as first-line treatment for extensive-stage small cell lung cancer, to be presented at ESMO Congress 2026 (Business Wire, October 23, 2026; September 2026 data cutoff). At the recommended Phase 3 dose of 1.6 mg/kg, zoci plus atezolizumab (n=33) achieved a confirmed ORR of 81.8% (27/33) and a 6-month progression-free survival rate of 81.9%; among 15 patients with at least 6 months of follow-up, median PFS and median duration of response were not reached after a median follow-up of 10 months. Grade 3 or higher adverse events related to zoci occurred in 9.1% (3/33), with no treatment-related discontinuations and one Grade 1 case of interstitial lung disease/pneumonitis. The triplet with carboplatin (n=13) showed an ORR of 92.3%, median PFS of 8.4 months and median duration of response of 7.7 months. A global randomized Phase 3 study in first-line ES-SCLC is expected to initiate by year-end 2026.

TRAW: Entered a Common Stock Purchase Agreement with Roth Principal Investments for an equity line of up to $50 million of newly issued common stock (8-K Items 1.01/3.02, filed Oct 23, 2026); resale registration statement on Form S-1 filed Oct 26, 2026.

Updated Phase 1/2 data for vopimetostat in combination with Revolution Medicines' RASONQUE (daraxonrasib) in MTAP-deleted RAS-mutant metastatic pancreatic ductal adenocarcinoma, reported October 23, 2026 by STAT from clinical trial results reported Friday at ESMO Congress 2026: the combination led to tumor responses in 82% of patients, and responses were durable, with 88% of patients still alive without tumor progression when assessed at nine months. STAT noted the regimen is chemotherapy-free. This is an updated, larger data cut versus the June 2026 initial readout (92% objective response rate in 11 of 12 response-evaluable patients). The remainder of the STAT article is paywalled, so no patient count, breakdown by dose or line of therapy, or safety data were disclosed in the accessible report, and Tango had issued no wire release with these updated numbers as of this poll (its October 16, 2026 release only announced the ESMO presentation and a PDAC investor webcast on October 23, 2026 at 7:30 am ET).

60 DEGREES PHARMACEUTICALS, INC. INVESTOR PRESENTATION, DATED OCTOBER 23, 2026 Exhibit 99.1 Tafenoquine for Babesiosis Progress across acute, relapsing and chronic disease Geoffrey S. Dow, PhD Chief Executive Officer 2026 ILADS Annual Scientific Conference October 22–25, 2026 | Herndon, Virginia...

Phase 1b data for enozertinib in first-line NSCLC with EGFR atypical mutations, presented at ESMO Congress 2026 (GlobeNewswire, October 23, 2026; data cutoff September 4, 2026; 26 patients treated with 80 mg once-daily oral enozertinib, including 23 with PACC mutations and 3 with L861Q mutations; brain metastases present in 65% of patients at baseline): 84% (21 of 25) confirmed systemic ORR by investigator and 80% (20 of 25) confirmed systemic ORR by BICR; 70% confirmed intracranial ORR by investigator and 67% (10 of 15) intracranial ORR by BICR RANO-BM, with a 40% (6 of 15) intracranial complete response rate; Kaplan-Meier 6-month landmark PFS was 88%, with median PFS and median duration of response immature. Safety: predominantly Grade 1 or 2 treatment-related adverse events, six patients (23%) experienced Grade 3 TRAEs (all resolved with dose modifications), no Grade 4 or 5 TRAEs and no treatment-related discontinuations. ORIC plans to initiate Redwood-1, a global Phase 3 registrational trial in EGFR atypical NSCLC enrolling approximately 360 patients, in 1H 2027.

Positive results from the pivotal Phase III REMODEL-1 (NCT05147220) and REMODEL-2 (NCT05156281) trials of remibrutinib (oral BTK inhibitor, 100 mg) vs teriflunomide in relapsing multiple sclerosis (RMS; ~2,000 patients), presented as a late-breaking session at MSToronto2026 and simultaneously published in The Lancet (Novartis, October 23, 2026): both trials met the primary endpoint of annualized relapse rate (ARR) - REMODEL-1 ARR 0.073 vs. 0.112 (35.2% relative reduction) and REMODEL-2 ARR 0.080 vs. 0.116 (31.2% relative reduction), translating to approximately one relapse every 13 years. Remibrutinib reduced the risk of 6-month confirmed disability progression (6mCDP) by 29.2% (nominal significance), with a 20.5% reduction in 3-month CDP; more than 92% of remibrutinib patients remained free from disease progression over 24 months as assessed by 6mCDP. MRI: annualized T2 lesion activity 0.94 vs. 2.71 (REMODEL-1) and 0.82 vs. 2.77 (REMODEL-2); Gd+ T1 lesions per scan 0.08 vs. 0.24 and 0.07 vs. 0.21. Pooled NEDA-3 55.0% vs. 32.1%. Safety: no liver safety signal and no Hy's Law cases; ALT >3xULN in 2.0% vs. 7.4% with teriflunomide; AEs 79.5% vs. 81.5%, serious AEs 6.0% vs. 7.0%, discontinuations due to AEs 3.1% vs. 5.8%; two deaths in the remibrutinib group, both assessed as unrelated to study treatment. Novartis plans to seek regulatory approval for remibrutinib in RMS globally.

Six-year follow-up results from the Phase III NATALEE trial (NCT03701334, Novartis media release, October 23, 2026) of Kisqali (ribociclib) plus endocrine therapy (ET) versus ET alone in stage II and III HR+/HER2- early breast cancer at high risk of recurrence (5,101 patients): Kisqali plus ET reduced the risk of death by 19.1% (overall survival HR 0.809; 95% CI 0.661-0.989), with benefit consistent across key subgroups and the absolute OS difference widening from 1.2% at five years to 2.1% at six years; invasive disease-free survival HR 0.741 (absolute benefit 5.3% at six years) and breast-cancer-specific survival HR 0.709. Patients had been off Kisqali for a median of 38 months. Novartis described the OS result as clinically meaningful (no p-value printed in the release). Safety was consistent with prior analyses with no new signals; secondary primary malignancies were 3.0% versus 3.3%. Late-breaking results presented at ESMO Congress 2026.

8-K filed (items 1.01, 3.03: financing-related)

Positive interim Phase 1 data for MGC026, a B7-H3-directed antibody-drug conjugate, in advanced solid tumors (NCT06242470; data cutoff September 1, 2026), presented at ESMO Congress 2026 (GlobeNewswire, October 23, 2026). In dose escalation, among 17 response-evaluable patients treated at doses of at least 6 mg/kg, confirmed ORR was 29% (5/17) and disease control rate 65% (11/17), with median duration of response 10.2 months. In the recurrent/metastatic squamous cell carcinoma of the head and neck expansion cohort at 7.5 mg/kg (22 response-evaluable, heavily pretreated), best ORR was 27% (6/22; 4 confirmed and 2 unconfirmed partial responses) and disease control rate 64% (14/22). Across 85 treated patients, Grade 3 or higher treatment-related adverse events occurred in 51% (neutropenia 27%, anemia 25%), treatment-related discontinuations were 5%, and there were no Grade 5 treatment-related events and no cases of interstitial lung disease, pneumonitis or ocular toxicity. MacroGenics plans a front-line SCCHN cohort of MGC026 plus pembrolizumab in early 2027.

Final Phase 2 analysis of Cohort 1A of the IOV-COM-202 trial (NCT03645928) of one-time lifileucel (TIL cell therapy) plus pembrolizumab in frontline advanced melanoma, presented in a Proffered Paper oral session at ESMO Congress 2026 (GlobeNewswire, October 23, 2026; 24 patients treated with lifileucel meeting pre-determined manufacturing specifications plus pembrolizumab for up to 24 months): physician-assessed confirmed ORR by RECIST v1.1 was 62.5% (15/24; 95% CI: 40.6, 81.2), including a 29.2% complete response rate; disease control rate 83.3% (20/24). Median duration of response was not reached (range: 4.3 to 57.7+ months) at a median follow-up of 26.9 months for DOR; median PFS was 19.8 months; median OS was not reached at a median study follow-up of 50.9 months. Safety was consistent with the underlying disease and the known safety profiles of lifileucel and pembrolizumab, with late adverse events consistent with continuous pembrolizumab monotherapy. The data support the ongoing Phase 3 TILVANCE-301 trial (NCT05727904) of lifileucel plus pembrolizumab in frontline advanced melanoma.

Ionis partner Roche announces new Phase 3 data showing sefaxersen significantly reduced proteinuria in people with IgA nephropathy (IgAN) – Sefaxersen achieved a statistically significant 43.1% reduction in proteinuria, a key indicator of kidney damage, relative to placebo at 37 weeks with...

First data from Cohort A of the Phase 2 INVINCIBLE-4 study (SAKK 66/22; NCT06358573) of INT230-6 plus standard-of-care neoadjuvant immuno-chemotherapy in early-stage, operable triple-negative breast cancer, to be presented as an ePoster at ESMO 2026 (PR Newswire, October 23, 2026): in the first seven Cohort A patients, pathological complete response in the breast (ypT0/Tis) and affected lymph nodes (ypN0) was achieved in 5 of 7 patients (71%; 95% CI 29-96%); no invasive breast cancer cells were detected in the axillary lymph nodes in 6 of 7 patients after INT230-6 treatment, despite 5 (71%) being initially biopsy-proven node-positive; tumor necrosis was seen after 1 or 2 injections in all 7 patients, and pre-surgery MRI showed RECIST 1.1 radiological complete response in 4 patients (57%) and partial response in 3 (43%); all 7 patients met the criteria for breast-conserving surgery. The INT230-6 dose was associated with dose-limiting local skin reactions, with all other adverse events in Cohort A low grade (<2); the protocol was amended to one injection at a lower drug volume to tumor volume ratio.

Offering prospectus filed (possible follow-on or ATM takedown)

Updated Phase 1 results for IDE849 (SHR-4849), a DLL3-targeting TOP1 ADC, presented at ESMO Congress 2026 (PR Newswire, October 23, 2026): Hengrui's Phase 1 study in China in relapsed SCLC and other neuroendocrine carcinomas (NECs) (n=122 enrolled at doses >=1.8 mg/kg IV Q3W, data cutoff August 21, 2026, median follow-up 12.1 months): 2L SCLC (n=51) ORR 72.5% (37/51), confirmed ORR 60.8% (31/51), median PFS 6.9 months, median OS 17.4 months; 2L + >=3L SCLC (n=101) ORR 68.3% (69/101), cORR 54.5% (55/101), median PFS 6.8 months, median OS 14.7 months; SCLC with brain metastases ORR 76.9% (20/26), cORR 65.4% (17/26); NECs (n=19) ORR 52.6% (10/19), cORR 36.8% (7/19), median PFS 7.0 months, nine-month OS rate 82.4%. Safety: Grade >=3 TRAEs 33.3% at the 2.4 mg/kg expansion dose; across the study (n=122) treatment-related discontinuation 2.5% and Grade >=3 ILD 0.8%. Following successful FDA Type C meetings, IDEAYA is targeting initiation of two Phase 3 registrational trials of IDE849 in ES-SCLC and NEC.

Positive prespecified interim results from the Phase 3 V012 trial (NCT05908084) of the acellular tissue engineered vessel (ATEV) vs arteriovenous (AV) fistula for hemodialysis access in female patients with end-stage kidney disease, presented as a late-breaking oral at ASN Kidney Week 2026 (GlobeNewswire, October 23, 2026): interim analysis of the first 80 patients (trial designed to enroll approximately 150, randomized 1:1) met the primary endpoint of catheter-free days from access creation through 365 days, with women implanted with the ATEV averaging 220.4 catheter-free days vs 129.3 days with AV fistula (+91 days; p<0.001, superiority); because the trial met its primary endpoint, enrollment was stopped. Median time to functional access was 1.7 months with the ATEV vs 5.3 months with fistula, and only approximately 65% of AV fistulas achieved functional maturation by Month 12. Safety was comparable to fistula: dialysis access infections of approximately 6 per 100 patient-years with the ATEV vs 23 per 100 patient-years with fistula, none of the ATEV infections related to the vessel itself, and no spontaneous ruptures in either group. Humacyte targets submission of a supplemental BLA to expand the Symvess (ATEV) label to AV access for hemodialysis this quarter.

8-K filed (items 3.02: financing-related)

Updated data from the global Phase I BEHOLD-1 trial (NCT06431594) of mocertatug rezetecan (Mo-Rez), an investigational B7-H4-targeted antibody-drug conjugate, in platinum-resistant ovarian cancer (GSK, October 23, 2026), to be presented in a late-breaking oral session at ESMO Congress 2026: at more than 13 months of follow-up, building on initial data announced earlier this year, Mo-Rez at the selected Phase III dose of 5.8 mg/kg achieved a confirmed objective response rate of 62% (28/45; 95% CI: 46.5-76.2%), a median duration of response of 12.2 months (95% CI: 7.0-12.7) and a median progression-free survival of 11.1 months (95% CI: 8.3-13.9). Safety remained consistent with previously published data: Grade >=3 treatment-related adverse events occurred in 71% (32/45) of patients and were predominantly haematologic; treatment-related events led to discontinuation in 2% (1/45) and interstitial lung disease/pneumonitis occurred in 2% (1/45). GSK has accelerated the Phase III development programme, with five pivotal Phase III studies now underway across gynaecological cancers.

Positive topline results from the global Phase 3 RAINFOL-02 trial (NCT06619236) of rinatabart sesutecan (Rina-S), an FRα-targeted TOPO1-inhibitor ADC, versus investigator's choice chemotherapy in platinum-resistant ovarian cancer (PROC), regardless of FRα expression level or prior treatment with mirvetuximab (GlobeNewswire, October 23, 2026): the trial met its primary endpoint of progression-free survival (PFS) and its key secondary endpoint of overall survival (OS). In the intent-to-treat (ITT) population (N=584), Rina-S demonstrated a 71% reduction in the risk of disease progression or death (HR 0.29; 95% CI, 0.23-0.37; p<0.0001); in the primary analysis set (PAS) population (N=492), PFS HR was 0.33 (95% CI, 0.26–0.42; p<0.0001). For OS, HR was 0.43 (95% CI, 0.31–0.59; p<0.0001) in the ITT population and 0.44 (95% CI, 0.31–0.63; p<0.0001) in the PAS population. Safety: the observed safety profile was consistent with the known profile of Rina-S, with no new safety signals or unexpected safety concerns identified. Data will be submitted for presentation at a future medical meeting and discussed with regulatory authorities worldwide; Genmab plans to engage with regulatory authorities regarding potential next steps.

Shelf registration filed (enables future raises incl. ATM)

Randomized Phase 2 AMPLIFY-7P results for camupepimut (ELI-002 7P) vs observation in mutant KRAS-driven pancreatic ductal adenocarcinoma following completion of standard locoregional therapy (144 patients enrolled across 24 U.S. sites), presented as a late-breaking oral at ESMO Congress 2026 and simultaneously published in Nature Medicine (GlobeNewswire, October 23, 2026): the study did not meet its pre-specified primary disease-free survival (DFS) endpoint in the intent-to-treat population (HR 0.85), in the context of a baseline imbalance in ctDNA positivity, a strong predictor of recurrence, which was more prevalent in the camupepimut arm (20.8% vs. 12.5%). In the 93 patients with baseline ctDNA-negative status, median DFS was not reached in the camupepimut arm vs. 21.2 months in the observation arm (HR 0.51; nominal p=0.0280), with recurrence or death in 27.4% (17/62) vs. 48.4% (15/31); in the ctDNA-positive subgroup (n=26), median DFS was 4.3 months vs. 1.9 months (HR 0.57; nominal p=0.0489); in patients without prior radiation (n=106), median DFS was 23.8 months vs. 10.4 months (HR 0.59; nominal p=0.0221). A multivariable model adjusting for baseline factors estimated adjusted HR 0.47 (p=0.0086). Safety: generally well tolerated, no new safety signals; Grade 3 or higher adverse events in 10.4% of camupepimut patients vs. 14.6% under observation, serious adverse events in 6.3% and 8.3% respectively, and no adverse events led to withdrawal of study medication. Elicio plans to discuss the findings with the FDA to inform a potential Phase 3 study in adjuvant pancreatic cancer.

Clinical data from Eikon's PARP1-selective and WRN helicase programs presented at the ESMO Congress 2026 in Madrid (GlobeNewswire, October 23, 2026). EIK1003 (Phase 1/2 trial EIK1003-001, NCT06253130, in advanced solid tumors with HRR gene mutations) monotherapy Cohort 1A ORR was 14.3% (7/49; 31.3% in PARP-naive participants) with disease control rate 38.8% (19/49) and median duration of response 6.3 months in confirmed responders; EIK1003 combinations showed ORR 20.8% (5/24) with abiraterone in metastatic prostate cancer (PSA response 30.8%, 12/39) and ORR 26.4% (14/53) with weekly paclitaxel in platinum-resistant ovarian or HER2-negative breast cancer (DCR 79.2%). Initial EIK1004 monotherapy data (Phase 1/2 trial EIK1004-001, NCT06907043, in ovarian, breast, prostate and pancreatic cancers) showed ORR 12.1% (7/58) and DCR 62.1% (36/58), generally well tolerated with Grade 3 or higher TEAEs in 17.6% (12/68), no dose-limiting toxicities and minimal hematologic toxicity. Preliminary EIK1005 data (Phase 1/2 trial EIK1005-002, NCT07262619, WRN helicase inhibitor in advanced solid tumors including MSI-H/dMMR) showed no dose-limiting toxicities, stable disease in 6 of 8 evaluable monotherapy participants (all MSI-H/dMMR) and a partial response in the single pembrolizumab-combination participant, with a half-life of about 9.4 days supporting weekly dosing.

Eikon Therapeutics Presents Data from PARP1-Selective and WRN Helicase Programs at the European Society for Medical Oncology Congress • Initial and updated cohort data from ongoing Phase 1/2 study of highly selective non-brain-penetrant PARP1 inhibitor EIK1003 continue to support potentially...

Top-line results from the Phase 2a ASSIST-SSc trial (NCT02682511, PR Newswire, October 23, 2026, 16:05 ET) of oral ifetroban, a thromboxane prostanoid receptor antagonist, in systemic sclerosis (SSc). In the 12-month pilot trial, 31 subjects received ifetroban (22 with diffuse cutaneous SSc and 9 with SSc-associated pulmonary arterial hypertension) and 10 received placebo (9 dcSSc and 1 SSc-PAH); the trial's purpose was to evaluate safety. Ifetroban was generally well tolerated, with a safety profile consistent with prior experience: gastrointestinal adverse events were more frequent than with placebo and were mostly mild and resolved, bleeding-related adverse events showed a modest increase and were predominantly mild, and no serious adverse event during the 12-month treatment period was assessed by the medical monitor as related to study drug. The trial was not designed or powered to evaluate efficacy, and limited disease progression in both groups reduced the ability to detect a treatment difference, so no efficacy conclusions were drawn. Next steps are submission of the clinical study report to the FDA, correlative biomarker studies, and evaluation of cardiac imaging from patients continuing on the open-label extension, which together will inform further development of ifetroban in SSc.

Climb Bio and Mabworks Announce CLYM116/MIL116 Data Demonstrating Deep, Durable APRIL Suppression and ~70% Gd-IgA1 Reduction at ASN Kidney Week 2026 >90% free APRIL suppression sustained through 12 weeks, supporting Q12W dosing interval ~70% reduction in Gd-IgA1 at 12 weeks ~33-day half-life...

Updated data from the Phase 1b/2 CELC-G-201 study of gedatolisib plus darolutamide in metastatic castration-resistant prostate cancer (mCRPC), presented as a poster (2237P) at ESMO Congress 2026 (GlobeNewswire, October 23, 2026; data cutoff August 14, 2026; 45 patients randomly assigned to 600 mg darolutamide twice daily combined with 120 mg gedatolisib in Arm 1 (n=19), 180 mg in Arm 2 (n=19) or 240 mg in Arm 3 (n=7), administered intravenously on days 1, 8, and 15 of a 28-day cycle, then two weeks off; over 50% had bone disease and 33% had PTEN loss): median radiographic progression-free survival (rPFS) was 15.7 months with a six-month rPFS rate (rPFS6) of 82.2% in patients with prior abiraterone exposure (n=22), and median rPFS was 9.1 months with an rPFS6 of 62.5% in patients with prior enzalutamide exposure (n=18). PSA >=50% decreases from baseline (PSA50) response rates were 36%, 47% and 67% at the 120-, 180-, and 240-mg gedatolisib dose levels, respectively, in patients with prior exposure to either abiraterone or enzalutamide, and exploratory analyses suggest activity is independent of PTEN status. Safety: manageable profile with one dose limiting toxicity (Grade 3 rash), no Grade 4/5 treatment-related adverse events (TRAEs), and no TRAEs leading to treatment discontinuation or death; Grade 3 TRAEs occurred in 9 (20%) patients, with only rash (n=4) and pruritus (n=2) occurring in more than one patient. The Phase 2 portion of the study is expected to begin in the first half of 2027.

Perspective’s [212Pb]Bamzireotide Navoxetan (VMT-α-NET) Demonstrates Encouraging Safety, Durable Disease Control, and Robust Tumor Response Across Multiple Dose Cohorts in Phase 1/2a Trial in Advanced SSTR2-Expressing NETs • Results presented at ESMO show a promising therapeutic index for [ 212...

8-K filed (items 1.01, 1.02, 3.02: financing-related)

Interim safety and efficacy results from the Phase 1 Breakfree-2 study (NCT06220201, Business Wire, October 23, 2026) of zolacabtagene autoleucel (zola-cel; BMS-986353), an investigational autologous CD19-directed CAR T cell therapy, in highly active relapsing MS (haRMS, n=30) and progressive MS (PMS, n=36), presented at MSToronto 2026: primary endpoint was safety. Cytokine release syndrome occurred in 42.4% (28/66, mostly Grade 1) and ICANS in 7.6% (5/66, including one Grade 4 event that resolved). After the data cutoff, one haRMS patient died of septic pneumonia 44 days after infusion, following high-dose cyclophosphamide administered for late-onset non-ICANS motor symptoms; no other Grade 5 events across 190 zola-cel patients in autoimmune Phase 1 programs. Efficacy among dose-level-2 patients with at least Week 26 follow-up (n=32): 6 of 7 haRMS patients met no-evidence-of-disease-activity criteria, all 8 haRMS patients evaluable for EDSS were stable or improved, 22 of 24 (92%) PMS patients were stable or improved on disability progression and all 24 PMS patients had no new MRI activity; all 60 patients treated at dose level 2 remained off disease-modifying therapies at last follow-up.

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01, 3.03: financing-related)

Positive initial Phase 1b data for brezosiran (ALN-HTT02) in Huntington's disease (Business Wire, October 23, 2026): single ascending intrathecal doses across the first five cohorts showed dose-dependent reductions of mutant huntingtin protein (mHTT) in cerebrospinal fluid, with a mean reduction from baseline of 60-64% at month 3 in the highest dose group evaluated to date and a maximum individual reduction of 83%; data include individuals followed for up to 12 months, with an encouraging safety and tolerability profile and no dose-dependent increases in CSF inflammatory markers, CSF neurofilament light chain (NfL) or brain ventricular volume. Data were presented at the European Huntington's Disease Network (EHDN) Clinical Research Congress 2026. Alnylam plans to initiate a placebo-controlled pivotal study in 2027, pending regulatory alignment.

FDA approved a subcutaneous (SC) induction regimen for SKYRIZI (risankizumab-rzaa) for adults with moderately to severely active Crohn's disease (PR Newswire, October 23, 2026, 15:33 ET), based on the Phase 3 AFFIRM trial (NCT06063967). SC induction is 720 mg delivered by on-body injector (OBI) at weeks 0, 2, 4 and 8, followed by maintenance OBI dosing every eight weeks from week 12; intravenous induction (600 mg at weeks 0, 4 and 8) remains an option. At week 12 in the total population, SC SKYRIZI met both co-primary endpoints versus placebo: clinical remission (CDAI <150) 56% vs 30% (p<0.001) and endoscopic response 43% vs 15% (p<0.001); endoscopic remission was 26% vs 11% (p<0.001). In patients without prior advanced therapy failure, clinical remission was 74% vs 27% and endoscopic response 60% vs 15%; in patients with prior advanced therapy failure, clinical remission was 45% vs 31% and endoscopic response 33% vs 15%. Safety for SC induction was generally consistent with the known SKYRIZI profile in Crohn's disease, with no new safety risks identified. The FDA also approved updated tuberculosis warning language in the U.S. Prescribing Information across all SKYRIZI indications, allowing clinicians to use clinical judgment on whether TB testing is needed before treatment.

Aardvark Therapeutics Reports Exploratory Data from the Terminated HERO and Open-Label Extension Trials • HERO trial terminated early (67 of 90 planned participants randomized); all efficacy data are considered exploratory • HQ-CT scores fell by Week 2 of dose escalation on ARD-101 and returned...

ZLAB: Entered a securities purchase agreement (dated Oct 22, 2026) with certain accredited investors for a private placement (PIPE) of 11,340,000 American depositary shares (each ADS representing ten ordinary shares) at an offering price of $22.04 per ADS, for expected gross proceeds of approximately $250 million before deducting placement agent fees and other expenses, to fund development of pipeline assets and for working capital and other general corporate purposes (8-K Items 1.01/3.02, filed Oct 23, 2026); closing expected on or about Oct 27, 2026.

Final two-year results from the Phase 3 TELIGAN trial of telitacicept in adults with IgA nephropathy (IgAN), conducted in China by collaborator RemeGen and presented in an oral presentation (TH-OR111) at ASN Kidney Week 2026 (GlobeNewswire, October 22, 2026): TELIGAN met the Stage B primary endpoint of annualized eGFR slope through 104 weeks, -0.6 mL/min/1.73 m²/year (95% CI: -1.7 to 0.4) for telitacicept versus -5.7 mL/min/1.73 m²/year (95% CI: -7.0 to -4.5) for placebo, a between-group difference of 5.1 mL/min/1.73 m²/year (95% CI: 3.5 to 6.7; p<0.001). Telitacicept was associated with an 89% lower risk of confirmed ≥30% eGFR decline (HR 0.11) and an 81% lower risk of confirmed ≥40% eGFR decline (HR 0.19) relative to placebo; no patient in the telitacicept group experienced a prespecified composite kidney failure endpoint event, compared with five in the placebo group, including two who required dialysis. At Week 104, 24h-UPCR decreased 60.3% from baseline in the telitacicept group compared with a 0.9% increase in the placebo group (placebo-adjusted reduction of 60.7%); 44.0% of telitacicept-treated patients achieved 24h-UPCR <0.5 g/g versus 8.2% in the placebo group. Serum galactose-deficient IgA1 decreased 47.79% from baseline with telitacicept, compared with a 19.06% increase with placebo. Safety: treatment-emergent adverse events occurred in 95.0% of telitacicept-treated patients and 89.3% of placebo-treated patients; serious adverse events occurred in 8.2% versus 18.2%; overall infections were reported in 67.3% of each group, and serious infections in 1.9% versus 4.4%; injection-site reactions were more frequent with telitacicept (56.0% versus 18.9%), with most mild to moderate in severity. TELIGAN enrolled 318 adults randomized 1:1 to telitacicept 240 mg or placebo, administered subcutaneously once weekly through Week 39, followed by every-other-week dosing from Week 40 through Week 104. Telitacicept received conditional approval in China in June 2026 for the reduction of proteinuria in adults with primary IgAN at risk of disease progression; Vor Bio holds exclusive rights outside Greater China.

Positive final efficacy analysis of the Phase 3 ORIGIN 3 trial of TRUTAKNA (atacicept-vymj) in adults with primary IgA nephropathy (IgAN) at risk for disease progression, presented as a featured late-breaking oral presentation at ASN Kidney Week 2026 and simultaneously published in The New England Journal of Medicine (GlobeNewswire, October 22, 2026): ORIGIN 3 met the primary endpoint of statistically significant proteinuria reduction at 36 weeks, and TRUTAKNA achieved statistical significance in all prespecified endpoints, including eGFR change from baseline and annualized slope, and risk reduction in composite kidney disease progression events; also statistically significant reductions in urine protein-to-creatinine ratio, galactose-deficient IgA1, and hematuria. Safety generally comparable to placebo: adverse events in 160/214 patients (75%) on TRUTAKNA vs 167/214 (78%) on placebo; serious adverse events 7% vs 15%; infection rates 51.4% vs 52.3%; no clinically relevant hypogammaglobulinemia or opportunistic infections; 1 death in the placebo group and no deaths in the TRUTAKNA group. Company plans to submit a supplemental BLA to the FDA in Q4 2026 for full approval expected in 2027.

Topline results from the Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) trial of intravenous SCY-247, its second-generation triterpenoid (fungerp) antifungal candidate for the treatment and prevention of invasive fungal infections (GlobeNewswire, October 22, 2026, 08:00 ET): 56 healthy participants enrolled across seven cohorts (42 received SCY-247 by peripheral intravenous infusion, 14 placebo); single ascending doses up to 300 mg and multiple ascending doses once daily for seven days up to 200 mg. IV SCY-247 was generally well tolerated, with the most commonly reported adverse reactions mild to moderate erythema and pain at the infusion site; there were no drug-related serious adverse events or discontinuations due to a related adverse event. Once-daily intravenous doses of 50 mg or higher achieved exposures at or above the efficacy target identified in murine models of invasive candidiasis (AUC0-24 of 20 µg·h/mL). The company expects to initiate Phase 2 development of SCY-247 in oral and IV formulations in 2027, subject to regulatory agreement; SCY-247 development is supported by a BARDA contract providing up to approximately $214 million in non-dilutive funding if milestones are achieved, and SCY-247 has Orphan Drug, QIDP and Fast Track designations.

Offering prospectus filed (possible follow-on or ATM takedown)

New Phase 2 ACUITY biomarker data for Privosegtor in optic neuritis (GlobeNewswire, October 22, 2026): post-onset increases in plasma neurofilament light chain (NfL) and glial fibrillary acidic protein (GFAP) were reduced with Privosegtor vs placebo, particularly at early timepoints close to acute optic neuritis onset; NfL levels remained stable in the Privosegtor group through Month 6, whereas placebo levels remained elevated above baseline at Month 1 before returning to baseline by Month 6, and GFAP levels remained below baseline in the Privosegtor group from Month 1 through Month 6 while the placebo group remained elevated above baseline. The biomarker results support previously reported functional low-contrast visual acuity +18-letter gains and structural retinal preservation at Month 3. Late-breaking data presented by Celine Louapre, M.D., Ph.D. at the 10th Joint ACTRIMS-ECTRIMS Meeting (MSToronto 2026). Privosegtor has FDA Breakthrough Therapy designation for optic neuritis and is being evaluated in the PIONEER registrational program.

Detailed positive results from the Phase 3 INTerpath-001 trial (NCT05933577, n=1,137) of intismeran autogene + KEYTRUDA vs KEYTRUDA alone as adjuvant treatment in completely resected stage IIB-IV melanoma (Business Wire, Oct 22, 2026; Merck and Moderna; to be presented at ESMO 2026 Presidential Symposium, accepted for publication in NEJM): at median follow-up 24.6 months, reduced risk of recurrence or death by 42% (RFS HR=0.58 [95% CI, 0.45-0.75]; p<0.001) and risk of distant metastasis or death by 38% (DMFS HR=0.62 [95% CI, 0.45-0.85]; p=0.002); 2-year RFS 82.2% vs 70.0%, 2-year DMFS 88.5% vs 80.2%; overall survival not yet mature with a favorable trend; safety consistent with prior studies, no new signals (TRAEs 95.8% vs 90.0%, Grade 3-5 TRAEs 14.2% vs 13.4%). First detailed dataset following the Aug 19, 2026 topline.

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01: financing-related)

Offering prospectus supplement filed

FDA alignment on the design of a pivotal Phase 3 trial of zervimesine (CT1812) in dementia with Lewy bodies (DLB): mild-to-moderate DLB participants randomized to 100 mg once-daily oral zervimesine or placebo for nine months; primary endpoint NPI-2 (neuropsychiatric inventory measuring hallucinations and delusions), key secondary ADCS-ADL (daily function). DLB has no approved treatments. (GlobeNewswire, October 22, 2026)

FDA accepted for review the resubmitted New Drug Application (NDA) for Vyglxia (troriluzole) for the treatment of spinocerebellar ataxia (SCA), disclosed by Biohaven in an Item 7.01 Form 8-K furnished October 22, 2026 (accession 0001935979-26-000083). The resubmission included new positive supportive analyses. The FDA communicated that it considers the resubmission a complete response to the November 4, 2025 action letter (the Complete Response Letter for the original NDA), and has assigned a user fee (PDUFA) goal date in the first quarter of 2027. The FDA plans to convene a meeting of the Peripheral and Central Nervous System Advisory Committee to obtain input from external stakeholders; the anticipated date of the advisory committee meeting will be forthcoming in the near term. Vyglxia would be the first and only FDA-approved treatment for SCA if approved; troriluzole is investigational and has not been approved for any indication in the United States.

FDA accepted for priority review the supplemental Biologics License Application (sBLA) for VYVGART (efgartigimod alfa-fcab, IV) and VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc, SC) for pediatric generalized myasthenia gravis (gMG), with a PDUFA target action date in February 2027, disclosed in argenx's third quarter 2026 financial results and business update (GlobeNewswire, October 22, 2026). The same release reported Q3 2026 VYVGART global product net sales of $1.7 billion and submission of a separate sBLA for VYVGART in ocular myasthenia gravis (oMG).

FDA lifted the clinical hold on the IND application for AB-101, Arbutus's oral PD-L1 inhibitor for chronic hepatitis B: the FDA notified the company that the hold has been removed and it may engage in additional clinical development for AB-101 in the United States; Arbutus is evaluating next steps for continued development. AB-101 has completed a Phase 1a/1b trial (AB-101-001) in healthy subjects and chronic HBV patients, where it was generally well tolerated, with high receptor occupancy in seven cHBV patients and hepatitis B surface antigen decline in two cHBV patients at the 30 mg dose. (GlobeNewswire, October 22, 2026; also furnished as Exhibit 99.1 to an Item 8.01 Form 8-K, accession 0001171843-26-006753.)

Positive topline results from the Phase 3 INTREPID study (NCT06468228) of lutikizumab, an investigational dual-variable-domain IL-1α/1β antagonist, in adults and adolescents with moderate to severe hidradenitis suppurativa (HS) (PR Newswire, October 22, 2026, 09:00 ET): 1,400 patients randomized 1:1 to lutikizumab SC or placebo. The study met its primary endpoint of HiSCR75 at Week 16 (31.4% vs 23.4% placebo; p≤0.001) and 8 ranked secondary endpoints, including NRS3 HS-related skin pain response at Week 8 (26.2% vs 15.0%; p<0.0001), NRS30 at Week 8 (36.0% vs 22.8%; p<0.0001), and fewer HS flares over the first 16 weeks (18.4% vs 28.1% of patients; p<0.0001). Lutikizumab was generally well tolerated with no new safety concerns: any AE 53.4% vs 53.7%, serious AE 3.0% vs 2.9%, AEs leading to discontinuation 1.7% vs 1.9%; no deaths in the lutikizumab group (one death on placebo). Additional data including the 36-week blinded extension are expected in 2027 and will support regulatory submissions for lutikizumab in HS in the second half of 2027.

FDA lifted the clinical hold on the IND for ARD-101 for hyperphagia in Prader-Willi syndrome (PWS): clinical development may resume. Aardvark voluntarily paused dosing in the Phase 3 HERO trial and its open-label extension (OLE) on February 27, 2026 after reversible ECG changes in a separate healthy-volunteer study; FDA placed the hold in May 2026. The company then ran a thorough ECG study in additional healthy volunteers (no cardiac events at 200 mg and 400 mg twice daily) and an in vitro cardiac sodium channel (NaV1.5) study in alignment with FDA. Aardvark plans to resume development in PWS after further FDA alignment on the Phase 3 design, and will host a webcast on Friday, October 23, 2026 at 8:00 a.m. ET to review the available dataset from the terminated HERO and OLE trials. (GlobeNewswire, October 22, 2026; also furnished as Exhibit 99.1 to an Item 8.01 Form 8-K, accession 0001193125-26-428943.)

FDA accepted the resubmission of the New Drug Application (NDA) for oxylanthanum carbonate (OLC), the Company's investigational oral phosphate binder for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis (GlobeNewswire, October 21, 2026). The NDA was resubmitted following a Complete Response Letter (CRL) received in June 2026. The FDA has deemed the OLC resubmission a Class II with a six-month review period from the resubmission date, and set a Prescription Drug User Fee Act (PDUFA) target action date of March 25, 2027. This resubmission includes Chemistry, Manufacturing, and Controls (CMC) data from a new drug product (DP) manufacturing vendor; the new vendor's facility was last inspected by the FDA in March 2024 and received 'No Action Indicated' status. The Company does not know whether the FDA will decide to conduct a pre-approval inspection of the new third-party DP vendor; additionally, if the original third-party manufacturing vendor is inspected in the near term and finds that its facility complies with current Good Manufacturing Practice (cGMP), the Company plans to seek FDA alignment on a shorter approval timeline.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Positive Phase 3 CLARITY-1 and CLARITY-2 results for brepocitinib (45 mg once daily, dual TYK2/JAK1 inhibitor) in non-infectious uveitis (NIU): two identical Phase 3 trials (CLARITY-1 N=166; CLARITY-2 N=204) both met the primary endpoint of time to treatment failure - hazard ratio vs placebo 0.27 (95% CI 0.17-0.43) in CLARITY-1 and 0.22 (0.14-0.34) in CLARITY-2, both P<0.0001; cumulative incidence of treatment failure at Week 48 35.4% vs 78.7% placebo (CLARITY-1) and 32.4% vs 77.8% (CLARITY-2); median time to treatment failure >48 weeks vs ~12 weeks on placebo in both studies. All key secondary endpoints met: steroid-free quiescence in both eyes at Week 48 58.8% vs 19.8% and 64.4% vs 19.4%; FA response (>=30% reduction among eyes with FA score >5 at baseline) 39.4% vs 8.6% and 54.9% vs 7.2%; resolution of macular edema 40.4% vs 2.1% (P=0.0041) and 42.6% vs 7.0% (P=0.0002); >=5-point NEI VFQ-25 improvement 36.4% vs 13.7% and 40.4% vs 14.1%. Generally well tolerated: most events mild or moderate, 3% discontinued due to an AE, no new safety signals. Priovant intends to file an sNDA for brepocitinib in NIU in the first half of calendar year 2027 (GlobeNewswire, October 21, 2026, 07:00 ET).

Offering prospectus filed (possible follow-on or ATM takedown)

European Commission (EC) approved Cosentyx (secukinumab) for the treatment of adults with polymyalgia rheumatica (PMR) who have had an inadequate response to glucocorticoids or who experience relapse during glucocorticoid taper (GlobeNewswire, October 21, 2026, 11:00 ET). Cosentyx becomes the first and only interleukin-17A (IL-17A) inhibitor approved in Europe for adults with PMR. The approval is based on the Phase III REPLENISH trial, which showed Cosentyx doubled sustained remission rates vs placebo at Week 52; safety results were consistent with the established Cosentyx profile. The recommended dose for adults with PMR is 150 mg by subcutaneous injection with initial dosing at Weeks 0, 1, 2, 3 and 4, followed by monthly maintenance dosing, in combination with a tapering course of glucocorticoids; based on clinical response the maintenance dose can be increased to 300 mg. Additional regulatory reviews of Cosentyx in PMR are ongoing globally, including in the US and Japan.

Offering prospectus filed (possible follow-on or ATM takedown)

Full one-year data set from KYSA-8, the registrational Phase 2 trial of miv-cel (mivocabtagene autoleucel, KYV-101) in stiff person syndrome (SPS), presented at MS Toronto 2026 (GlobeNewswire, October 21, 2026, 07:15 ET). The primary endpoint and all key secondary endpoints were previously reported achieved at AAN 2026; at one year (n=26), median improvement in the timed 25-foot walk (T25FW) was 49% (vs 46% at the Week 16 primary endpoint measurement; p<0.0001), with 77% (20/26) achieving a clinically meaningful >=20% T25FW improvement (81%, 21/26, at Week 16). Of the 12 patients requiring a walking aid before treatment, 67% (8/12) no longer required assistance; 92% (24/26) remained free of chronic immunotherapies for SPS. All secondary endpoints continued to be met, with significant mean improvements in Modified Rankin Scale (-1.0, p=.0003), Hauser Ambulation Index (-1.7, p<0.0001), Distribution-of-Stiffness (-1.6, p<0.0001) and Heightened Sensitivity Scale (-2.8, p<0.0001); median 6-minute walk improvement deepened from 89 meters at Week 16 to 119 meters at one year. Safety was described as well tolerated.

Offering prospectus filed (possible follow-on or ATM takedown)

FDA cleared the Investigational New Drug (IND) application for OPGx-RDH12, an investigational gene therapy for the treatment of RDH12-associated Leber congenital amaurosis (RDH12-LCA), a rare inherited retinal disease that causes progressive vision loss and blindness beginning in early childhood (GlobeNewswire, October 21, 2026, 07:00 ET). The IND clearance enables Opus Genetics to advance OPGx-RDH12 into clinical development; the Company plans to initiate a Phase 1/2 trial this quarter. OPGx-RDH12 is the Company's third gene therapy pipeline program to advance into clinical development, developed in collaboration with the Global RDH12 Alliance (patient advocacy organizations RDH12 Fund for Sight in the United States and Eyes on the Future in the United Kingdom; partnership established in July 2025).

8-K filed (items 1.01: financing-related)

Offering prospectus supplement filed

FDA granted Orphan Drug Designation to onvansertib, an oral PLK1 inhibitor, for the treatment of chronic myelomonocytic leukemia (CMML) (GlobeNewswire, October 21, 2026, 16:05 ET). CMML is difficult to treat with limited options; 40-50% of patients with myeloproliferative CMML have RAS-pathway mutations linked to more aggressive disease. A Phase 1 investigator-initiated study of onvansertib in relapsed/refractory CMML reported preliminary results at the 2025 ASH Annual Meeting, including tolerability as monotherapy and preliminary efficacy in about 40% of myeloproliferative CMML patients. The designation covers a rare condition affecting fewer than 200,000 people in the U.S. and can provide clinical-trial tax credits, exemption from certain FDA user fees, and up to seven years of market exclusivity upon approval.

FDA approved the supplemental New Drug Application (sNDA) for the oral pellet formulation of once-daily ORLADEYO (berotralstat) for prophylactic therapy in adults and adolescents aged 12 years and older (GlobeNewswire, October 21, 2026, 07:00 ET). The new 150 mg ORLADEYO pellet formulation provides an alternative administration option for adults and adolescents who may have difficulty swallowing, prefer not to take a capsule, or choose to remain on a pellet formulation beyond age 12; the sprinkle-like pellets can be poured directly into the mouth and swallowed immediately with water or milk, or sprinkled over a spoonful of soft, non-acidic food. ORLADEYO is the first and only targeted oral prophylactic therapy for patients with hereditary angioedema (HAE) aged 2 and above; the capsule formulation was approved in December 2020 for patients 12 years and older.

FDA granted accelerated approval of ULTOMIRIS (ravulizumab), Alexion/AstraZeneca Rare Disease's long-acting C5 complement inhibitor, to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression (AstraZeneca press release, October 21, 2026) - the first and only C5 inhibitor approved for IgAN and the fifth ULTOMIRIS indication; the approval followed FDA Priority Review. The indication is approved under accelerated approval based on reduction of proteinuria; it has not been established whether ULTOMIRIS slows kidney function decline in IgAN, and continued approval may be contingent on the ongoing I CAN Phase III trial (NCT06291376). In the prespecified interim analysis of I CAN, ULTOMIRIS reduced 24-hour urine protein creatinine ratio (UPCR) from baseline by 46.6% (95% CI: 39.0%, 53.2%) at week 34 versus 5.6% (95% CI: -4.9%, 15.0%) on placebo, a placebo-adjusted treatment effect of 43.4% (95% CI: 33.5%, 51.8%; p<0.0001); the proteinuria reduction was observed as early as week 10 (36.7% vs 8.5% for placebo) and sustained through week 34, consistent across subgroups. The trial continues to week 106, where the primary endpoint of change from baseline in estimated glomerular filtration rate (eGFR) will support traditional approval; late-breaking I CAN results will be presented at the American Society of Nephrology Kidney Week on October 23, 2026. ULTOMIRIS is given intravenously every eight weeks after a loading dose. Safety was consistent with the known profile, with no new safety concerns; the most common adverse events on ULTOMIRIS were upper respiratory tract infection (11.3%), nasopharyngitis (9.2%) and infusion-related reactions (8.4%), versus upper respiratory tract infection (10.2%), nasopharyngitis (9.0%) and hypertension (6.6%) on placebo. IgAN affects more than 200,000 people in the US, and regulatory submissions based on the interim analysis are also under review in Japan and additional countries.

Shelf registration amended

Positive 52-week efficacy results from ORCA-OL, a long-term open-label trial of cytisinicline 3 mg three times daily for cigarette smoking and e-cigarette (vaping) cessation, presented at CHEST 2026 (GlobeNewswire, October 21, 2026, 08:30 ET). ORCA-OL enrolled 479 adults (475 dosed; 84.6% cigarette smokers, 12.8% exclusively vaped, 2.5% both), all of whom had already made a counseling-supported quit attempt in a prior ORCA trial and were still smoking or vaping. Four-week sustained abstinence rates increased from 25.5% at Weeks 9-12 to 30.3% at Weeks 21-24 to 31.2% during Weeks 49-52 in the total population; among participants who had received placebo in their prior trial, abstinence was 26.3% at Weeks 9-12 and 39.4% at Weeks 49-52, and among those previously treated with cytisinicline it was 27.8% at Weeks 49-52. At the end of the year, 32.8% of participants who exclusively smoked and 24.6% of those who exclusively vaped had quit. Median cytisinicline exposure was 361 days and 69.7% (334/479) completed the 52-week study.

8-K filed (items 1.01: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

New Phase 2b long-term extension data for duvakitug (anti-TL1A monoclonal antibody, co-developed with Sanofi) in moderately-to-severely active ulcerative colitis (UC) and Crohn's disease (CD), presented at UEG Week 2026 (GlobeNewswire, October 20, 2026, 08:00 ET). RELIEVE UCCD LTE (NCT05668013): patients who responded to the 14-week induction study (n=130; 72 UC, 58 CD) were re-randomized to duvakitug 450 mg or 900 mg subcutaneously every four weeks for 44 weeks of maintenance. UC at Week 44 - 450 mg (n=34): clinical remission (modified Mayo Score) 47%, clinical response 82%, endoscopic improvement 59%; 900 mg (n=38): clinical remission 58%, clinical response 87%, endoscopic improvement 63%. CD at Week 44 - 450 mg (n=27): endoscopic response (50% or greater decrease in SES-CD) 41%, clinical response (CDAI) 81%, clinical remission 81%; 900 mg (n=31): endoscopic response 55%, clinical response 87%, clinical remission 84%. Efficacy was numerically higher at 900 mg in both diseases, consistent with a dose response. Duvakitug was generally well tolerated with a safety profile consistent with the induction phase and no new safety concerns; discontinuations due to adverse events were UC 4% (450 mg) and 2% (900 mg), and CD 6% in each arm. Biomarker analyses from the RELIEVE UCCD induction study (NCT05499130) were also presented. Phase 3 programs SUNSCAPE-1/-2 (UC) and STARSCAPE-1/-2 (CD) are ongoing.

8-K filed (items 3.02: financing-related)

New data from the ARC-20 platform study of casdatifan (next-generation HIF-2a inhibitor) in clear cell renal cell carcinoma (ccRCC), presented at an investor event in New York City (Business Wire, October 20, 2026; data cut-off September 4, 2026). First-line casdatifan + zimberelimab (anti-PD-1), n=30, 11.5 months median follow-up: landmark PFS 86% at 6 months and 69% at 12 months, confirmed objective response rate (cORR) 50%, primary progression 6.7% (n=2). First-line casdatifan + ipilimumab + zimberelimab, 3 months median follow-up: only one patient with primary progression (4%, n=24 with at least one scan; n=31 dosed). Second-line casdatifan + cabozantinib, n=43, 21.3 months median follow-up: median PFS 14.5 months, 18-month landmark overall survival 77%, cORR 44%, primary progression 5%; efficacy similar in patients who had progressed on a prior TKI (~60% of 2L patients). Late-line casdatifan monotherapy, four pooled cohorts (50 mg QD, 50 mg BID, 100 mg QD tablet, 150 mg QD), n=121 heavily pre-treated patients (52% with at least three prior lines), 28 months median follow-up: median overall survival 35 months, median PFS 13 months, cORR 36%. No unexpected safety signals in any cohort; monotherapy adverse events described as acceptable and manageable across doses. Phase 3 PEAK-1 (2L casdatifan + cabozantinib) is enrolling; the first-line Phase 3 PEAK-20 study (casdatifan + ipilimumab + nivolumab) is on track to start by year-end 2026, and Arcus plans a registrational trial of casdatifan + tivozanib in the late-line setting.

Positive Phase 2 topline results for RAP-219 (TARPγ8-selective AMPA receptor negative allosteric modulator, 0.75 mg once daily) in acute bipolar mania (NCT07046494; randomized, double-blind, placebo-controlled, n=253 adults with bipolar I disorder and YMRS>=25 at baseline, 3 weeks inpatient): met primary endpoint, YMRS total score LS mean reduction 15.4 points vs 12.3 points placebo at Week 3 (baseline 36.9 vs 35.8), a 3.1-point placebo-adjusted improvement (p=0.045); progressive placebo-adjusted YMRS improvement 0.9 at Week 1, 2.2 at Week 2, 3.1 at Week 3; all 11 YMRS items improved numerically vs placebo. Met pre-specified secondary endpoint, CGI-BP Severity of Mania placebo-adjusted improvement 0.4 points (p=0.024). Well tolerated: TEAEs 51.6% vs 52.8% placebo, all mild or moderate, no severe TEAEs and no SAEs; discontinuations due to TEAE 3.2% vs 0.8%; no orthostatic hypotension, akathisia or extrapyramidal symptoms reported; somnolence 0.8% vs 3.1%. Company called it clinical proof-of-concept; FDA end-of-Phase 2 meeting planned Q1 2027, two Phase 3 trials expected to begin 2H 2027 (GlobeNewswire, October 20, 2026, 06:00 ET).

8-K filed (items 1.01, 3.02: financing-related)

Termination of the Phase 2 trial of PF-06835375 (anti-CXCR5 antibody) in primary immune thrombocytopenia (NCT05070845; open-label, began 2022, final enrollment 61 participants vs 91 planned), reported by Fierce Biotech on October 20, 2026: Pfizer confirmed to Fierce on October 19 that the study was terminated due to a business decision and that the decision was not based on any safety concerns; the ClinicalTrials.gov record now lists the trial as Terminated with the same reason. Pfizer would not confirm whether all work on PF-06835375 has been halted; at publication the asset remained listed on Pfizer's pipeline site for ITP and systemic lupus erythematosus (a Phase 1 lupus study wrapped up in 2022). No efficacy data were disclosed.

Completed Phase 2a induction results for OD-001 (oral, selective RIPK2 scaffolding inhibitor) in moderate-to-severe ulcerative colitis (NCT06850727; open-label, dose-ranging, no concurrent placebo control, 56 efficacy-evaluable patients, 12-week induction), presented at UEG Week 2026 (GlobeNewswire, October 20, 2026, 07:00 ET): met primary endpoint, change from baseline in three-component modified Mayo score at Week 12 (mean reduction of 2.4 points; p<0.001). At Week 12, clinical remission, clinical response, and symptomatic remission were 27%, 61%, and 49%, respectively, in the pooled 20 mg BID group (n=41) and 20%, 40%, and 27%, respectively, at 10 mg BID (n=15). Clinical remission at 20 mg BID was 29% in advanced therapy-naive patients (n=28) and 23% in advanced therapy-experienced patients (n=13), and 16% in patients with a Mayo Endoscopic Score of 3 (n=19). Symptomatic remission at 20 mg BID reached 21% at Week 2 and increased through Week 12 with no plateau observed. Well tolerated: no serious adverse events, serious infections, treatment-related discontinuations, or treatment-emergent liver function abnormalities; three adverse events in the 20 mg BID group were considered drug-related (two fevers and one headache); one Grade 3 anemia event in the 10 mg BID group was considered unrelated to treatment. Company supports 20 mg BID as the projected lowest maximally efficacious dose and plans a randomized, placebo-controlled Phase 2b monotherapy trial and a Phase 2a combination trial (OD-001 plus vedolizumab), with topline induction results expected in the second half of 2027.

8-K filed (items 1.01: financing-related)

8-K filed (items 3.03: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Ionis Pharmaceuticals announced that partner GSK received FDA approval of HIBSAGO (bepirovirsen) for the treatment of chronic hepatitis B (CHB) virus infection in combination with nucleos(t)ide analogue reverse transcriptase inhibitor (NrtI) therapy in adult patients without cirrhosis, receiving NrtI therapy for at least 6 months, who have suppressed HBV DNA, and with HBsAg levels <=3000 IU/ml as determined by an FDA-authorized test (Business Wire, October 20, 2026). Bepirovirsen is a first-in-class RNA-targeted medicine discovered by Ionis and licensed to GSK, and the first FDA-approved antisense oligonucleotide for CHB; the approval was granted under FDA Priority Review and Breakthrough Therapy Designation, ahead of the October 26, 2026 PDUFA date. It was supported by the B-Well 1 and B-Well 2 Phase 3 trials (1,834 patients combined), which showed a functional cure rate of 19% with bepirovirsen versus less than 1% with current standard of care alone, and 26% in patients with HBsAg <=1000 IU/ml. Treatment is a six-month course with the option of at-home administration after the first dose. Ionis is eligible to receive milestone payments upon approval and other regulatory benchmarks.

Offering prospectus supplement filed

FDA approved HIBSAGO (bepirovirsen), a first-in-class antisense oligonucleotide (ASO), for the treatment of chronic hepatitis B (CHB) virus infection in combination with nucleos(t)ide analogue reverse transcriptase inhibitor (NrtI) therapy in adult patients without cirrhosis, receiving NrtI therapy for at least 6 months, who have suppressed HBV DNA, and with HBsAg levels <=3000 IU/ml as determined by an FDA-authorised test (GSK press release, October 20, 2026) - the first FDA-approved ASO for CHB, granted under FDA Priority Review and Breakthrough Therapy Designation, ahead of the October 26, 2026 PDUFA date. The approval was supported by the pooled B-Well 1 and B-Well 2 Phase 3 trials (1,834 patients combined): functional cure (HBV DNA and HBsAg undetectable for at least 24 weeks after stopping all treatment) was achieved by 19% of bepirovirsen patients (233 of 1,220) versus 0 of 614 on placebo; in the key secondary subgroup with HBsAg <=1000 IU/ml the functional cure rate was 26% (200 of 768) versus 0 of 393 on placebo. Bepirovirsen also reduced HBsAg to <=100 IU/ml in 49% of participants (598 of 1,220) overall and 62% (476 of 768) in the <=1000 IU/ml subgroup as of the week 72 visit. Treatment is a six-month once-weekly subcutaneous course with the option of self-administration after the first dose. The FDA also authorised Roche's Elecsys HBsAg II quant II RxDx assay as a companion diagnostic. The most frequently observed adverse events were injection site erythema, local pain and a temporary rise in a liver enzyme. Bepirovirsen was licensed from Ionis Pharmaceuticals; it is approved in Japan and under regulatory review in China and Europe, and GSK expects peak annual sales of more than GBP 2 billion.

8-K filed (items 1.01: financing-related)

Phase 2 SUNRISE-PD results for bezisterim (NE3107) in early Parkinson's disease (NCT06757010; randomized, double-blind, placebo-controlled, n=57 patients naive to carbidopa/levodopa, 20 mg twice daily vs placebo for 12 weeks), presented at the 151st Annual Meeting of the American Neurological Association (GlobeNewswire, October 20, 2026, 08:00 ET). Primary pharmacodynamic endpoint was the effect on hematologic inflammatory biomarker indices (MLR, SIRI, NLR, SII, PLR, AISI); clinical endpoints were secondary/exploratory and prespecified in the SAP. In the prespecified subgroup with higher baseline systemic inflammation (above-median platelet counts above 230 x 10^9/L, approximately half the trial population), bezisterim improved MDS-UPDRS Part I (non-motor) scores -1.62 vs +1.86 for placebo (Cohen's d = -1.33; p=0.0124), Part III (motor) scores -1.32 vs +1.26 (Cohen's d = -0.92; p=0.0265), and Total Scores -1.23 vs +1.21 (Cohen's d = -1.45; p=0.0006). In the overall modified intent-to-treat population, the Composite Benefit Score favored bezisterim (Cohen's d = -1.61; p=0.0004); patients receiving bezisterim were 6.25-fold more likely to achieve clinically meaningful improvement on the CBS than placebo (25% vs 4%), and the risk of clinically meaningful worsening was approximately 70% lower with bezisterim (14% vs 46%; relative risk 0.30). Well tolerated: TEAEs 39.3% on bezisterim vs 51.7% on placebo, no severe or serious adverse events in either group, and only one potentially treatment-related adverse event in each treatment arm.

Full results from the Phase 3 CROSSING trial of TEZSPIRE (tezepelumab, anti-TSLP monoclonal antibody, co-developed with Amgen) in eosinophilic esophagitis (EoE), presented as a late-breaking oral presentation at United European Gastroenterology Week 2026 (Business Wire, October 20, 2026). CROSSING (NCT05583227): randomized, double-blind, placebo-controlled, 368 patients aged 12-80 with symptomatic and histologically active EoE randomized 1:1:1 to tezepelumab 210 mg, 420 mg, or placebo subcutaneously every four weeks, on stable background medications. Both co-primary endpoints met at Week 24: histologic remission (peak eosinophil count <=6 per high power field) 64.8% (210 mg) and 68.9% (420 mg) vs 3.5% placebo (p<0.001 for both); LS mean change in Dysphagia Symptom Questionnaire (DSQ) score -18.8 points (210 mg; p=0.049) and -22.0 points (420 mg; p<0.001) vs -14.7 placebo. Efficacy sustained through Week 52: histologic remission 67.8% (210 mg) and 63.3% (420 mg) vs 5.4% placebo (p<0.001); DSQ change -22.1 and -24.0 vs -13.2 placebo (p<0.001); total endoscopic remission (EREFS=0) at Week 52 in 34.4% (210 mg) and 29.5% (420 mg) vs 4.1% placebo (p<0.001). Safety profile consistent with approved indications; most frequent adverse events were upper respiratory infection (nasopharyngitis), headache and diarrhea. The positive CROSSING topline was first announced qualitatively on August 27, 2026 (no numeric rates); these are the first published numeric results. AstraZeneca plans to share the data with regulatory authorities.

8-K filed (items 1.01: financing-related)

FDA approved a new manufacturing process (Process 2.0) for SIMLANDI (adalimumab-ryvk), Alvotech's biosimilar to Humira (GlobeNewswire, October 20, 2026, 04:30 ET; dateline Reykjavik, Iceland and London, UK). The approval allows Alvotech to introduce Process 2.0 for manufacture of adalimumab drug substance in its DSM2 manufacturing suite in Reykjavik, Iceland. The new process more than doubles drug substance output per batch without changing the upstream manufacturing scale, with demonstrated comparability to the previously approved process while maintaining product quality, safety and efficacy. Alvotech said producing the same quantity in fewer batches frees capacity in Reykjavik for other products in its biosimilars portfolio. This is distinct from the October 5, 2026 FDA approval of additional U.S. manufacturing capacity for SIMLANDI, which enabled use of the DSM2 suite itself.

Positive Phase 1 multiple ascending dose (MAD) topline results for ZP9830, a novel Kv1.3 ion channel blocker (first-in-human, single-center, randomized, double-blind, placebo-controlled Phase 1 trial, NCT06682975): the MAD part evaluated safety, tolerability and pharmacokinetics in 32 healthy male and female participants over a 4-week treatment duration with safety follow-up until day 41. Multiple doses were safe and well tolerated, with no serious or severe adverse events and no dose-limiting safety findings; all treatment-emergent adverse events in participants receiving ZP9830 were non-serious and mild in severity, and no clinically relevant safety findings were observed in vital signs, 12-lead ECG recordings, physical examinations or safety laboratory parameters. Pharmacokinetic parameters support continued development in trials of longer duration. A Phase 1b trial in people with plaque psoriasis has recently been initiated, and a Phase 2 trial in an additional autoimmune disease is planned for initiation in H1 2027. Announced October 19, 2026 (GlobeNewswire, 01:00 ET).

Positive Phase 2 final efficacy results from the investigator-sponsored TASKIN trial (NCT05201352) of vilamakitug (Natrunix, XB2001; anti-IL-1alpha monoclonal antibody) plus trifluridine/tipiracil with or without bevacizumab in refractory metastatic colorectal cancer (GlobeNewswire, October 19, 2026): the study met its pre-specified primary efficacy success criterion, with 61.3% of evaluable patients in the vilamakitug arm alive at 12 months (49/80) versus 55.7% in the control arm (44/79), above the protocol threshold of 42 survivors. Median PFS was 5.9 vs 5.6 months (12-month PFS rate 25.0% vs 21.0%, log-rank p=0.42). In a prospective exploratory subgroup of right-sided primary tumors (n=37), vilamakitug reduced the risk of death by 62% versus control (HR 0.38, 95% CI 0.14-0.99, log-rank p=0.038).

FDA granted clearance under the IND application to proceed with the SUMMIT clinical trial of treprostinil SMI (soft mist inhaler) in pulmonary hypertension associated with COPD (PH-COPD) (Business Wire, October 19, 2026). SUMMIT is a Phase 2, 16-week, double-blind study evaluating safety and tolerability in approximately 40 patients with PH-COPD, with secondary endpoints evaluating efficacy on exercise capacity and NT-proBNP; eligible patients may enroll in an open-label extension. Assuming a successful SUMMIT outcome, United Therapeutics anticipates commencing a Phase 3 registration study for treprostinil SMI in PH-COPD in 2028. Treprostinil SMI has the potential to be the first FDA-approved therapy specifically indicated for PH-COPD.

FDA accepted the New Drug Application (NDA) for UGN-103 (mitomycin) for intravesical solution for the treatment of adults with recurrent low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC) and assigned a PDUFA target action date of June 14, 2027 (GlobeNewswire, October 19, 2026). The NDA is supported by the Phase 3 UTOPIA trial (NCT06331299), in which UGN-103 showed a 77.8% complete response rate at three months, with a 94.5% Kaplan-Meier estimated probability of remaining event-free at six months among responders.

Phase 4 VERDICT (NCT04259138) primary results for ENTYVIO (vedolizumab) in moderate to severe ulcerative colitis (randomized treat-to-target trial, n=672 adults, conducted by Alimentiv in collaboration with Takeda; three corticosteroid-free (CSF) treatment-target groups: Group 1 symptomatic remission, Group 2 symptomatic + endoscopic remission, Group 3 symptomatic + endoscopic + histologic remission (disease clearance); treatment escalated with ENTYVIO-based algorithms): the primary endpoint was met, with the disease-clearance group (Group 3) experiencing a significantly longer time to first UC-related complication (hospitalization, colectomy, use of rescue therapy, treatment-related and other UC-related complications) than the symptomatic-remission group (Group 1) up to 80 weeks after target achievement (hazard ratio 0.33 [95% confidence interval 0.17-0.67]; P=0.0014), reducing UC complication risk by nearly 70 percent. Nearly 60% (400/672) of randomized patients achieved their assigned CSF target by Week 48 (Group 1: 137 [74.1%]; Group 2: 125 [56.3%]; Group 3: 138 [52.1%]); among those who achieved their treatment target, disease clearance was reached by more than half (57%) of ENTYVIO-treated patients at Week 16; time to UC-related complication was similar between Groups 2 and 3 (1.10 [0.45-2.69] P=0.8023); 51 UC-related complications occurred, with use of rescue therapy the most frequent. Adverse event rates were similar across groups; the safety profile through 96 weeks was generally consistent with previously reported data, with no new safety signals identified; most common adverse events (>=5%) were ulcerative colitis, anaemia, haematochezia, COVID-19 infection, influenza, nasopharyngitis and arthralgia. Presented during late-breaker and poster sessions at UEG Week 2026 and published in the United European Gastroenterology Journal (Business Wire, October 19, 2026).

Merck KGaA, Darmstadt, Germany agreed to acquire Silence Therapeutics for $20.00 in cash per ADS (each ADS representing three ordinary shares, or $6.6667 per ordinary share; approximately $1.3 billion equity value and $1.1 billion enterprise value; 130% premium to the October 16, 2026 closing ADS price and 82% premium to the 30-day volume-weighted average price of $10.99), definitive agreement announced October 19, 2026 (Business Wire). The transaction will be effected by a UK scheme of arrangement and is expected to close in the first quarter of 2027, subject to Silence shareholder approval, sanction by the High Court of Justice of England and Wales, and other customary closing conditions including regulatory approvals; the Silence board unanimously approved the deal and securityholders holding approximately 14% of voting power committed to vote in favor. Lead asset divesiran is a Phase 3-ready siRNA targeting TMPRSS6 for polycythemia vera, with FDA Fast Track and Orphan Drug designations.

8-K filed (items 1.01: financing-related)

New GAVRETO (pralsetinib) data across RET fusion-positive cancers, disclosed October 19, 2026 ahead of oral and poster presentations at ESMO Congress 2026 (PR Newswire, October 19, 2026, 08:05 ET). Pooled analysis of ARROW (NCT03037385) and TAPISTRY (NCT04589845) in RET fusion-positive solid tumors excluding NSCLC and thyroid tumors (49 patients): overall response rate 53% and median duration of response 12.2 months; previously treated pancreatic tumors ORR 88% (7/8). TAPISTRY RET fusion-positive thyroid cancer cohort (16 response-evaluable patients): ORR 81.3% with manageable safety, consistent with ARROW. ARROW RET fusion-positive NSCLC: first-line pralsetinib investigator-assessed ORR 75% vs 26% for patients who had received standard-of-care first-line therapies before ARROW enrollment, with longer median times to progression, treatment discontinuation, and next treatment or death; at least 75% of evaluable NSCLC patients had improved or stable health-related quality-of-life scores across all assessed domains at Cycle 12 Day 1. ESMO presentations scheduled October 25-26, 2026.

8-K filed (items 1.01, 3.02: financing-related)

Completed randomization in PREDICT-1, the genotype-based registrational trial of licaminlimab in TNFR1-mediated dry eye disease (GlobeNewswire, October 19, 2026, 02:00 ET): 176 patients enrolled in the randomized, multicenter, double-masked, vehicle-controlled study, two-thirds of whom carry the specified TNFR1 genetic variant previously linked to enhanced clinical response in Phase 2 trials. The primary endpoint is the change from baseline at Day 29 in global ocular discomfort severity score in patients with the specified TNFR1 genotype, with the same outcome measure in the overall study population as a key secondary endpoint. Oculis expects to report topline results around year-end 2026.

Positive topline results from Part A of the registration-directed Phase 3 Study '304' of VDPHL01 (extended-release oral minoxidil 8.5 mg QD or BID) in males with mild-to-moderate pattern hair loss (n=536, NCT06972264): met all primary and secondary endpoints with high statistical significance. Co-primary endpoints at Month 6: mean increase in non-vellus target area hair count of 28.6 hairs/cm2 (QD, p<0.0001) and 36.4 hairs/cm2 (BID, p<0.0001) vs 6.6 hairs/cm2 on placebo, and patient-reported improved/much improved hair coverage on the AAIRS. AAIRS improved/much improved at Month 6: 55.7% (QD) and 68.7% (BID) vs 15.0% placebo (both p<0.0001); any AAIRS improvement 78.8% (QD) and 85.0% (BID) vs 35.3% placebo; Investigator Global Assessment improved 78.4% (QD) and 82.7% (BID) (both p<0.0001). Statistically significant separation from placebo as early as Month 2. Generally well tolerated: no treatment-related serious adverse events and no adverse events of special interest of cardiac origin; peripheral edema 2.8% (QD) / 4.5% (BID) / 0.6% (placebo), hypertrichosis 2.2% / 9.0% / 0%.

Positive one-year Phase 3 SHINE-2 results for Omvoh (mirikizumab-mrkz) in pediatric patients ages 6 to 17 with moderately to severely active ulcerative colitis, presented as a late-breaking oral presentation at UEG Week 2026 (PR Newswire, October 19, 2026). Single-arm, open-label study (63 participants enrolled, ages 4 to 17; 52 Week 12 responders entered maintenance and form the Week 52 efficacy population, ages 6 to 17): 81% achieved clinical response to Omvoh induction at Week 12; among responders at Week 52, 50% achieved clinical remission, meeting the study's primary endpoint (evaluated against a pre-specified benchmark derived from the adult placebo remission rate in LUCENT-2), 50% achieved corticosteroid-free clinical remission without surgery, 73.1% achieved clinical response, 71.2% achieved symptomatic remission, 53.8% achieved endoscopic improvement and 44.2% achieved endoscopic remission. 54% of enrolled patients had previously experienced biologic treatment failure and half entered with severe disease activity. Safety through Week 52 was consistent with the established adult profile, with no new safety signals; the most common treatment-emergent adverse events were upper respiratory tract infection, injection site reaction, ulcerative colitis, headache, nasopharyngitis and anemia, most mild to moderate; serious adverse events occurred in 4 patients (6.3%), none considered related to study treatment, and 2 patients (3.2%) discontinued because of ulcerative colitis flare; no deaths, malignancies, opportunistic infections or growth-related safety concerns were reported. Omvoh is approved for adults with ulcerative colitis and Crohn's disease; Lilly plans to submit these data to global regulators to support a potential label expansion for pediatric ulcerative colitis, which would make Omvoh the first IL-23p19 inhibitor indicated for pediatric ulcerative colitis.

New TREMFYA (guselkumab) Crohn's disease data presented at UEG Week 2026 (GlobeNewswire, October 19, 2026, 08:23 ET). Phase 3 GALAXI long-term extension, Week 144 as-observed results in moderately to severely active Crohn's disease: clinical remission 94% (172/183) on guselkumab 100 mg q8w and 91% (185/204) on 200 mg q4w; 90-day corticosteroid-free clinical remission 89% (q8w) and 88% (q4w); endoscopic remission 52% (89/172) and 61% (119/195); 74% (432/582) of guselkumab-treated patients remained on treatment through three years. Phase 3 FUZION (Crohn's disease with perianal fistula), Week 48: combined fistula remission increased from 28% at Week 24 to 35% at Week 48 in the 100 mg q8w group (n=113) and was 26% at Week 48 (27% at Week 24) in the 200 mg q4w group (n=115); 90% of randomised patients completed treatment through Week 48. Safety findings through Week 144 (GALAXI) and Week 48 (FUZION) were consistent with the known safety profile of guselkumab in Crohn's disease.

Positive Phase 3 APOLLO (NCT06910358) topline for bitopertin (60 mg oral QD, n=183, adults and adolescents aged 12+ with EPP/XLP, 6 months): met both co-primary endpoints vs placebo - reduction in whole-blood metal-free PPIX -41.4% (p<0.0001; -45.8% vs -4.4% per the EX-99.2 deck) and increase in average monthly total time in light without pain during month 6 of +12.2 hours (p=0.0010; 41.1 hr vs 28.9 hr). All secondary endpoints statistically significant: reduced incidence rate of phototoxic reactions (p<0.0001; IRR 0.29, 71 vs 231 total reactions per deck), increased cumulative total time in light without pain (p<0.0001; 233.2 vs 174.6 hr per deck), increased average daily sunlight exposure time to prodrome (p=0.0077, adults only), and improved PGIC and PGIS scores (both p<0.0001). Generally well tolerated: dizziness the most common TEAE (50%, mild or moderate and generally transient), no TEAEs above Grade 3, no new safety signals. Disc plans to resubmit an NDA to FDA by end of 2026, with an FDA decision anticipated by mid-2027. Press release furnished as EX-99.1 to an 8-K filed October 19, 2026 (accession 0001193125-26-424649).

Announced commencement of an underwritten public offering of $350 million of common stock (and, in lieu of common stock to certain investors who so choose, pre-funded warrants to purchase common stock), following the positive Phase 3 APOLLO topline for bitopertin (GlobeNewswire, October 19, 2026, 16:01 ET); Disc intends to grant the underwriters a 30-day option to purchase up to an additional $52.5 million of common stock at the public offering price, less underwriting discounts and commissions. All shares and pre-funded warrants to be sold by Disc. Net proceeds, together with existing cash, cash equivalents and marketable securities, to fund research and clinical development of current or additional product candidates, support the potential commercialization of bitopertin for erythropoietic protoporphyria (EPP) and X-linked protoporphyria (XLP) and pre-commercialization activities for other candidates, with the remainder for working capital and general corporate purposes. Jefferies, Leerink Partners, Morgan Stanley and Cantor are joint book-running managers. The offering is subject to market and other conditions, with no assurance as to whether or when it may be completed or as to its actual size or terms.

Updated clinical data from three darovasertib trials in uveal melanoma, to be presented at ESMO Congress 2026 (PR Newswire, October 19, 2026, 06:00 ET). OptimUM-01 (Phase 1/2, darovasertib + crizotinib in HLA-A*02:01-positive metastatic uveal melanoma (mUM), Phase 2 expansion, data cutoff March 23, 2026, N=101 evaluated, median follow-up 10.4 months, ~66% with >=2 prior lines): treatment-naive (N=13) ORR 36.4% (4/11), DCR 90.9%, median DOR 10.2 months, median PFS 7.4 months, median OS 27.1 months; prior tebentafusp as only prior systemic therapy (N=15) ORR 35.7% (5/14), DCR 85.7%, median DOR 5.6 months, median PFS 5.7 months, median OS 12.1 months; overall (N=101) ORR 22.1% (19/86), DCR 80.2%, median DOR 7.4 months, median PFS 5.4 months, median OS 13.4 months; most common treatment-related adverse events diarrhea (82%), nausea (76%), peripheral edema (70%) and vomiting (57%), with related adverse events leading to discontinuation in 9 (8.9%) participants. OptimUM-02 (registrational Phase 2/3, 1L HLA-A*02:01-negative mUM): subgroup analyses showed consistent improvements in ORR and PFS for darovasertib + crizotinib versus investigator's choice of therapy across baseline LDH, ECOG performance status, largest target lesion size and choice of control therapy, building on the June 2026 topline (median PFS 6.9 months by blinded independent central review and 6.7 months by investigator versus 3.1 and 2.7 months for investigator's choice). OptimUM-09 (Phase 2, neoadjuvant single-agent darovasertib in primary uveal melanoma, abstract data on 116 patients: 66 enucleation Cohort 1, 50 plaque brachytherapy Cohort 2; updated ESMO data on 119 patients, cutoff June 15, 2026): robust tumor shrinkage in approximately 80% of patients, >=20% tumor shrinkage in 57.8% (67/116) (Cohort 1: 51.5% [34/66]; Cohort 2: 66% [33/50]), a 57% eye preservation rate in the enucleation cohort, 66% of plaque brachytherapy patients achieving a reduced projected radiation dose to critical eye structures, and 86.3% of patients free from metastasis at 15 months (Cohort 1: 79.7%; Cohort 2: 94.4%); most common treatment-related adverse events diarrhea (64%), nausea (58%), fatigue (33%) and vomiting (28%), treatment-related serious adverse events in 6.0% of patients. IDEAYA is submitting an NDA for darovasertib + crizotinib in 1L mUM under the FDA real-time oncology review program, expecting to complete the submission in 2H 2026.

8-K filed (items 3.02: financing-related)

FDA accelerated approval of Jemperli as first immunotherapy for adults with stage II/III MSI-H/dMMR locally advanced rectal cancer under accelerated approval (Oct 19, 2026, GSK press release). AZUR-1 Phase II (n=154) primary ICR cCR12 47% (54/116; 95% CI 37,56); investigator cCR12 68% (98/144); exploratory 76% (110/144) no cancer >=1yr (incl 12 pCR); no recurrences >2yr FU; serious AR 20%, fatigue 32%, rash 22%, diarrhea 18%, pruritus 17%, discon 3%. Ahead of Feb 2027 PDUFA via Priority Review + National Priority Voucher; Fast Track + BTD.

Offering prospectus supplement filed

Positive Phase 2 results for emvistegrast (GS-1427), an oral therapy in moderate to severe ulcerative colitis (NCT06290934), reported October 19, 2026 by STAT from data reported that Monday: a 200 mg daily dose taken for 12 weeks produced a 23% placebo-adjusted clinical remission rate, a statistically significant improvement that STAT reported exceeded Wall Street analyst expectations. Gilead's senior vice president for inflammation clinical development, Swati Tole, told STAT emvistegrast has the potential to be a best-in-class oral for ulcerative colitis. No patient numbers, absolute remission rates, or safety data were disclosed in the accessible STAT report, and Gilead had issued no wire release on the data as of this poll.

Negative topline results from LUCIA (NCT06683742), the second pivotal Phase 3 trial of DURAVYU (vorolanib intravitreal insert) in wet age-related macular degeneration (wet AMD) (GlobeNewswire, October 19, 2026, 07:00 ET): randomized, double-masked trial enrolling 475 patients, 1:1 DURAVYU 2.7 mg every 24 weeks versus on-label aflibercept 2 mg every 8 weeks (supplemental aflibercept as needed per pre-specified criteria, after 3 monthly loading doses). DURAVYU did not achieve the primary endpoint of statistical non-inferiority in the average change from baseline in best corrected visual acuity (BCVA) at blended Weeks 52 and 56 versus on-label aflibercept, despite demonstrating visual improvement; the release reported no numeric BCVA change values for either arm. Key secondary endpoints were met, including a superior 38% reduction in treatment burden (nominal p-value <0.0001), with 52% of eyes supplement-free and 71% receiving zero or one supplement up to Week 56, as well as strong anatomic control at Week 56, consistent with the first Phase 3 LUGANO trial (which also missed non-inferiority in August 2026). Safety: continued favorable safety profile for DURAVYU with redosing. EyePoint will complete additional analyses of the LUCIA and pooled Phase 3 datasets to assess next steps for DURAVYU in wet AMD.

Strategic prioritization following the LUCIA Phase 3 wet AMD topline miss (GlobeNewswire, October 19, 2026, 07:15 ET): EyePoint will conclude the pivotal Phase 3 wet AMD trials early, without full completion of the second year of each trial, and reduce its workforce by approximately 53% (substantial majority in Q4 2026, completed H1 2027), to focus resources on DURAVYU in diabetic macular edema (DME). Estimated annualized savings approximately $30 million; estimated one-time separation charges approximately $9 million; cash, cash equivalents and short-term investments approximately $118 million as of September 30, 2026. The DME Phase 3 program (COMO, NCT07449936; CAPRI, NCT07449923; 551 patients total) is expected to read out topline data in Q4 2027.

New three-year follow-up data from the Phase 2 trial of personalized cancer vaccine EVX-01 in advanced melanoma (NCT03220477, 16 patients enrolled; EVX-01 plus pembrolizumab in years 1-2, with 6 of 12 responders continuing into a one-year extension on EVX-01 stand-alone therapy in year 3): all patients at three-year follow-up maintained the clinical responses observed at two-year follow-up, with no tumor progression observed. The release states no overall survival or progression-free survival figures and no safety summary. The data build on the two-year results presented at ESMO 2025: 75% objective response rate with 25% of patients obtaining a complete response, 92% of patients still responding at two years with no relapses observed, 54% of patients with a deepened response, and tumor reduction in 15 of 16 patients; at two years EVX-01 had induced an immune response in all patients, with 86% of vaccine neoantigens generating potent specific T-cell responses. The three-year clinical and new immune data will be presented at ESMO Congress 2026 on October 24, 2026 (poster 2006P), followed by a company webinar on October 29, 2026. Announced October 19, 2026 (GlobeNewswire, 03:00 ET).

8-K filed (items 1.01, 3.02: financing-related)

Health Canada issued a Notice of Compliance authorizing MYQORZO (aficamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) of New York Heart Association (NYHA) class II-III in adult patients (GlobeNewswire, October 19, 2026, 07:30 ET) — the fifth regulatory approval for MYQORZO in less than a year, following approvals by the U.S. FDA (December 2025), China NMPA, the European Commission and the UK MHRA. The approval is supported by the pivotal Phase 3 SEQUOIA-HCM trial, in which treatment with aficamten for 24 weeks increased peak oxygen uptake (pVO2) by 1.76 mL/kg/min compared to baseline versus 0.0 mL/kg/min with placebo (least squares mean difference 1.74 mL/kg/min [95% CI 1.04 - 2.44]; p=0.000002). MYQORZO was well tolerated, with no instances of worsening heart failure or treatment interruptions due to low LVEF; treatment-emergent serious adverse events occurred in 5.6% of patients on MYQORZO and 9.3% on placebo; core laboratory echocardiographic LVEF was <50% in 5 patients (3.5%) on MYQORZO compared to 1 patient (0.7%) on placebo; hypertension (8% vs 2%) was the only adverse reaction occurring in >5% of patients and more commonly on MYQORZO than placebo. The Canadian Product Monograph includes a Boxed WARNING for the risk of heart failure due to systolic dysfunction.

FDA accepted the supplemental New Drug Application (sNDA) for REZZAYO (rezafungin for injection) for prophylaxis of invasive fungal disease and granted Priority Review, with a PDUFA target action date in Q1 2027 (GlobeNewswire, October 19, 2026, via TradingView). The sNDA is supported by the Phase 3 ReSPECT trial in adult patients undergoing allogeneic hematopoietic stem cell transplantation; management targets a potential commercial launch in H2 2027 if approved.

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.02: financing-related)

Submitted a New Drug Application (NDA) to the FDA earlier in October 2026 seeking accelerated approval of CNM-Au8 for the treatment of amyotrophic lateral sclerosis (ALS), based on neurofilament light chain (NfL) biomarker data and clinical outcomes (GlobeNewswire, October 19, 2026). Clene is continuing preparations for the confirmatory Phase 3 RESTORE-ALS study. No FDA filing-acceptance decision or PDUFA target action date was announced. Press release furnished as EX-99.1 to an 8-K filed October 19, 2026 (accession 0001437749-26-032882).

Cohort C Phase 2 REZILIENT2 results in NSCLC with EGFR ex20ins/uncommon mutations and active CNS metastases (data cutoff n=59, median FU 9.2mo): intracranial ORR 30.2% (13/43; 95% CI 17.2-46.1), intracranial DCR 72.1%, median iDoR 9.7mo; in TKI-naive iORR 37.5%. To be presented at ESMO 2026.

Phase 2 ADDRESS-LC results in Long COVID (NCT06847191), presented at the American Neurological Association annual meeting (GlobeNewswire, October 19, 2026, 08:00 ET): randomized, double-blind, placebo-controlled signal-finding trial, n=203, bezisterim 20 mg twice daily vs placebo for 12 weeks, 22 clinical outcome measures with no single endpoint designated as primary. The trial did not achieve statistical significance on any endpoint in the overall intent-to-treat population (trend toward improvement across most measures). In pre-specified subgroups with higher baseline symptom burden (about 78% of participants in at least one domain), results favored bezisterim: among the most fatigued half at baseline (PROMIS Fatigue T-score >=65), mean improvement in PROMIS Fatigue at Week 12 was 8.93 points with bezisterim vs 5.30 points with placebo (p=0.02), with statistically significant differences also on PGI-Severity Fatigue, PGI-Change Fatigue, PGI-Change Overall and CGI-Change Overall; in the high post-exertional malaise subgroup (DSQ-PEM >=67.5), statistically significant differences in objective global cognition, processing speed and identification/attention; in the greater objective cognitive impairment subgroup (Cogstate Composite Global z-score <=0.130), statistically significant differences in objective global cognition, verbal learning, verbal memory and processing speed. Safety comparable to placebo: treatment-emergent adverse events 41.6% bezisterim vs 55.9% placebo, drug-related TEAEs 18.8% vs 25.5%, no serious TEAEs with bezisterim vs one with placebo. Biomarker data pending. Company says findings inform design of a confirmatory Phase 3 study.

FDA accepted BioCardia's revised De Novo pre-submission package for marketing clearance of the Helix transendocardial delivery catheter (GlobeNewswire, October 19, 2026, 09:15 ET). At a May 2026 meeting, FDA agreed there are two pathways for Helix marketing clearance and raised no concerns regarding the product's safety data, performance, or compatibility with general classes of agents; the revised pre-submission incorporating CDRH advice was re-submitted in September 2026 and has now been accepted. BioCardia expects FDA substantive review with written feedback, followed by a meeting to discuss approval via the De Novo pathway, this quarter. Helix is supported by clinical data from fifteen clinical trials including nearly five hundred patients.

Phase 1b FURTHER (FURMO-002, NCT05364073) results for firmonertinib in treatment-naive advanced EGFR PACC-mutant non-small cell lung cancer (NSCLC), published in Cancer Cell (GlobeNewswire, October 19, 2026, 08:01 ET): confirmed overall response rate (ORR) of 43% at 160 mg and 68% at 240 mg, with median progression-free survival (PFS) of 11.1 and 16.0 months respectively, both by blinded independent central review (BICR). Confirmed intracranial ORR was 47% (8/17), including 41% complete responses among the 17 patients with baseline brain metastases. 80% of evaluable patients achieved a complete molecular (ctDNA) response by cycle 3, which was associated with a higher ORR (70% vs. 20%) and longer median PFS (15.1 vs. 3.0 months) than in patients without clearance. The safety profile was manageable and consistent with prior firmonertinib studies. ArriVent is enrolling the global Phase 3 ALPACCA trial (FURMO-006, NCT07185997) of firmonertinib versus investigator's choice of osimertinib or afatinib in first-line EGFR PACC-mutant NSCLC.

ANAB announced that partner GSK received FDA accelerated approval for Jemperli in MSI-H/dMMR locally advanced rectal cancer (Oct 19, 2026, GlobeNewswire; AZUR-1 data 47% ICR cCR12 etc). Royalty-bearing collaboration via TESARO/GSK license.

Registration statement amended

Phase 2a TARGET-CD (NCT06548542) primary results for ABBV-466, a dual-targeting therapy combining risankizumab (anti-IL-23 p19) with trosunilimab (anti-α4β7), in moderate to severe Crohn's disease (open-label, treat-through platform study, n=300: 106 ABBV-466, 94 risankizumab monotherapy, 100 trosunilimab monotherapy; 75% had failed at least one advanced therapy): ABBV-466 met the primary endpoint with higher endoscopic remission (SES-CD ≤4 and no subscore >1) at Week 12 at 30.2% versus 21.3% for risankizumab monotherapy and 18.0% for trosunilimab monotherapy; the difference increased at Week 24 (40.5% versus 22.4% and 20.0%). Gene expression analysis showed intestinal tissue repair and barrier restoration by Week 12, with tissue remodeling and metabolic changes at Week 24. Serious adverse events for ABBV-466 were 10.4%, comparable to risankizumab (9.6%) and trosunilimab (9.0%); most common adverse events (≥5%) in the ABBV-466 and trosunilimab arms were Crohn's disease and nausea, and in the risankizumab arm Crohn's disease and arthralgia. Presented as a late-breaker at UEG Week 2026 on October 19, 2026 (PR Newswire).

First overall survival data for azenosertib (first-in-class WEE1 inhibitor) monotherapy from an interim analysis of DENALI Part 1b (GOG-3066) in platinum-resistant ovarian cancer, published in the ESMO Congress 2026 abstract (data cutoff September 2, 2025): median OS, a secondary endpoint, was 15.8 months (95% CI, 8.7-17.8) in the Cyclin E1-positive subgroup (n=48) versus 7.5 months (95% CI, 5.8-12.3) in the Cyclin E1-negative subgroup (n=46). Data to be presented as a rapid oral presentation at ESMO Congress 2026 on October 23, 2026. Announced October 18, 2026 (GlobeNewswire).

Veradermics to share topline results on October 19, 2026 (investor call and live webcast 8:00 a.m. ET) from Part A of its randomized, double-blind, placebo-controlled registration-directed Phase 3 Study '304' trial of VDPHL01 in males with mild-to-moderate pattern hair loss. Announced October 18, 2026 (Business Wire): the company said it will host a call that morning to review the results; no topline numbers were disclosed in the announcement. VDPHL01 is a proprietary extended-release oral minoxidil formulation; Study '304' (NCT06972264) enrolled 536 men evaluating VDPHL01 8.5 mg once and twice daily, with Month 6 co-primary endpoints of change from baseline in non-vellus target area hair count (TAHC) and the proportion reporting improved or much improved hair coverage on the Androgenetic Alopecia Impact Rating Scale (AAIRS).

Disc Medicine to share topline results on October 19, 2026 (corporate webcast 8:00 a.m. ET) from the APOLLO Phase 3 trial of bitopertin in erythropoietic protoporphyria (EPP). Announced October 18, 2026 (GlobeNewswire): the company said it will host a webcast to present and discuss the results that morning; no topline numbers were disclosed in the announcement. Bitopertin is an oral glycine transporter 1 (GlyT1) inhibitor designed to modulate heme biosynthesis; APOLLO is the double-blind, placebo-controlled Phase 3 study of bitopertin in EPP.

First global data for risvutatug rezetecan (Ris-Rez), an investigational B7-H3-targeted antibody-drug conjugate licensed from Hansoh Pharma, in relapsed extensive-stage small cell lung cancer (ES-SCLC), from the Phase 1b dose-optimisation portion of EMBOLD PanTumour-101 (NCT06551142) (GSK press release, London, October 18, 2026). The global, open-label Phase 1a/b analysis included 50 ES-SCLC patients who had received a median of two prior therapies, randomised to Ris-Rez 6 mg/kg or 8 mg/kg intravenously every three weeks, at a median follow-up of 100.5 days: confirmed objective response rate was 61% at 8 mg/kg (11/18; 95% CI 35.7-82.7) versus 32% at 6 mg/kg (7/22; 95% CI 13.9-54.9). The safety profile was manageable across both dose levels: grade 3 or higher treatment-related adverse events in 42% of patients at 8 mg/kg and 15% at 6 mg/kg, most commonly haematological, and interstitial lung disease in 1 of 50 patients (2%, grade 2). Data will be presented in a poster session at ESMO Congress 2026; together with data from Hansoh's Phase 3 ARTEMIS-008 study in SCLC in China, the results supported selection of the 8 mg/kg dose now being evaluated in GSK's global Phase 3 EMBOLD SCLC-301 study.

20 IBD abstracts presented at UEG Week 2026 (Barcelona, Oct 17-20, 2026; PR Newswire Oct 17, 2026), covering long-term and real-world data for established therapies. SKYRIZI ulcerative colitis (COMMAND): sustained clinical and endoscopic outcomes up to three years of maintenance treatment; as-observed clinical remission rates of 54.0%-73.1% and endoscopic remission rates of 38.8%-53.5% were achieved at week 96 with 180 mg across advanced therapy-naive and advanced therapy-experienced patients. RINVOQ Crohn's disease (U-ENDURE long-term extension): as-observed analyses showed outcomes maintained through approximately four years; at week 144 clinical remission was achieved by 78.9% of patients receiving 15 mg and 86.6% receiving 30 mg, and endoscopic remission by 47.1% and 64.2% respectively. RINVOQ real world: interim UPlift results showed 70.2% of evaluable patients (N=497) with moderate to severe Crohn's disease achieved clinical response within 12 weeks, with a median time to first response of 13 days; a claims analysis (N=514, UC or CD) showed patients who switched from a TNFi to upadacitinib had lower rates of IBD-related hospitalizations (9.5% vs 16.3%) and surgery (6.9% vs 10.6%) over 12 months than patients who cycled to another TNFi. Also presented: additional Phase 3 AFFIRM analyses of subcutaneous SKYRIZI induction in Crohn's disease and Phase 2a TARGET-CD data on investigational dual-targeting ABBV-466 (no efficacy percentages disclosed in the release for either).

FDA approved RYZUMVI (phentolamine ophthalmic solution 0.75%) for the treatment of presbyopia in adults (Oct 16, 2026, PR Newswire), expanding its existing U.S. approval for reversal of pharmacologically induced mydriasis. Viatris holds exclusive U.S. commercialization rights under its global licensing agreement with Opus Genetics and said it is exploring a U.S. partner to commercialize RYZUMVI for presbyopia. The most common ocular adverse reactions in presbyopia (>5% of subjects) were conjunctival hyperemia (21%) and instillation site irritation (16%).

8-K filed (items 1.01: financing-related)

EMA's CHMP issued a negative opinion on the marketing authorization application for YARTEMLEA (narsoplimab) in hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA), confirming on re-examination its original negative opinion from June 2026. An eight-member ad hoc expert group convened for the re-examination recommended a review of individual patient-level data by external experts; Omeros said the substantial patient-level data it submitted to CHMP in August was not provided to that group. Narsoplimab was FDA-approved in December 2025 as the first and only approved therapy for TA-TMA (adults and children two years and older), and there is no approved TA-TMA therapy in Europe. Omeros also told CHMP it will terminate the YARTEMLEA compassionate-use program due to rising U.S. demand, with current patients supplied to complete their courses. Announced October 16, 2026 (Business Wire).

Positive interim Phase 3 results for MIUDELLA (hormone-free copper IUD, licensed globally from Sebela Women's Health in June 2026) through 6 years of use, to be presented at the Society of Family Planning Annual Meeting Oct 17-19, 2026: prospective single-arm study at 42 US sites; 6-year outcomes in 465 of 1397 evaluable-for-pregnancy and 592 of 1601 safety participants; cumulative 6-year Pearl Index 0.93 (95% CI, 0.62 to 1.34) and cumulative Kaplan-Meier pregnancy rate 3.9% (95% CI, 2.7% to 5.8%) at 6 years; no new safety signals during the additional three years of extended use; treatment-emergent adverse events included heavy menstrual bleeding, dysmenorrhea and pelvic discomfort, with bleeding/pain discontinuations in 139 participants (8.7%) in year 1 and 11 (1.9%) in year 6; follow-up ongoing. FDA has accepted an sNDA for extended use up to 6 years, PDUFA action expected Q2 2027.

Novartis terminated the Phase 2 study of Fabhalta (iptacopan) added to standard of care in lupus nephritis after completing the study's first part, citing strategic considerations, according to a ClinicalTrials.gov registry update reported by Reuters and Endpoints News on October 16, 2026. Novartis said the decision was based on strategic considerations and was not driven by any interim efficacy or safety findings, that other Fabhalta studies will continue as planned, and that investigators have been notified and are engaging participants on next steps. Fabhalta is FDA-approved for paroxysmal nocturnal hemoglobinuria, IgA nephropathy, and C3 glomerulopathy.

Reported early clinical improvement in the first patient treated under its Compassionate Care program in Abu Dhabi, UAE (Oct 16, 2026, GlobeNewswire): a critically ill three-year-old boy with an aggressive brain tumor who received investigational intranasal NEO100 (highly purified perillyl alcohol) outside the NEO100-03 pediatric trial (he did not meet trial eligibility) is, per his treating physicians, now breathing independently without ventilator support and beginning to move his legs after starting treatment. Single-patient compassionate-use observation only: the company states it does not establish that NEO100 caused the improvement, does not establish safety or efficacy, and may not predict outcomes in other patients.

Offering prospectus supplement filed

8-K filed (items 3.03: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

EMA's CHMP recommended approval of an indication extension for TALVEY (talquetamab) plus daratumumab, with or without pomalidomide, for adults with relapsed or refractory multiple myeloma who have received at least one prior therapy. The recommendation rests on the Phase 3 MonumenTAL-3 study (NCT05455320, N=864, all previously exposed to lenalidomide and a proteasome inhibitor): at a median follow-up of 24.6 months, progression-free survival favored the talquetamab combinations over daratumumab plus pomalidomide and dexamethasone (HR 0.28 for talquetamab-daratumumab-pomalidomide and HR 0.33 for talquetamab-daratumumab, both p<0.0001), with 24-month PFS rates of 81.3% and 77.6% respectively; overall survival also favored the combinations (HR 0.47 and HR 0.51), with 24-month OS rates of 89.2% and 87.9%. Overall response rates were 88.2% and 88.5% versus 77.6% for the control arm. Safety was consistent with the known profiles of each monotherapy. If the European Commission approves, this would be the only GPRC5D-targeting bispecific antibody combination available as early as second line. Announced October 16, 2026 (GlobeNewswire).

FDA approved the supplemental New Drug Application (sNDA) for phentolamine ophthalmic solution 0.75% (RYZUMVI) for the treatment of presbyopia in adults (Oct 16, 2026, GlobeNewswire), expanding its existing U.S. approval for reversal of pharmacologically induced mydriasis, one day ahead of the Oct 17, 2026 PDUFA goal date. The approval was supported by the pivotal Phase 3 VEGA-2 and VEGA-3 trials, which met their primary and all key secondary efficacy endpoints, with no treatment-related serious adverse events reported. The most common ocular adverse reactions in presbyopia (>5% of subjects) were conjunctival hyperemia (21%) and instillation site irritation (16%). Viatris holds exclusive U.S. commercialization rights under a global licensing agreement, and the approval triggers a milestone payment to Opus Genetics.

EMA's CHMP adopted a positive opinion recommending an indication extension for TRYNGOLZA (olezarsen), as an adjunct to diet in combination with other lipid-lowering therapies, for adults with severe hypertriglyceridemia (fasting triglycerides >=880 mg/dL). The opinion is based on the Phase 3 CORE (NCT05079919) and CORE2 (NCT05552326) studies, which met their primary endpoint with a placebo-adjusted mean reduction in fasting triglycerides of up to 72% at six months, sustained through 12 months, and an 85% reduction in adjudicated acute pancreatitis events over 12 months in a pooled analysis. The opinion came ahead of the previously anticipated timeline; a European Commission decision is expected in Q4 2026. If confirmed, TRYNGOLZA would be the first RNA-targeted therapy authorised in the EU for this population. Sobi holds ex-US commercialisation rights (excluding Canada and China). Announced October 16, 2026 (BusinessWire, Ionis).

8-K filed (items 2.03: financing-related)

Offering prospectus supplement filed

EMA's CHMP adopted a positive opinion recommending EU marketing authorization for once-weekly TransCon CNP (navepegritide), under the brand name YUVIWEL, for achondroplasia in children two years of age and older whose epiphyses are not closed. The opinion followed review of a marketing authorisation application that included data from three randomized, double-blind, placebo-controlled trials and up to three years of open-label extension data, including the pivotal ApproaCH trial. TransCon CNP was FDA-approved in February 2026 under the same brand name. A European Commission decision is expected in Q4 2026; if authorized, Ascendis expects a first EU launch in Germany in Q1 2027. Announced October 16, 2026 (GlobeNewswire, Ascendis Pharma).

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

Two topline clinical data updates. REC-4881 (MEK1/2 inhibitor, 4 mg QD) Phase 1b/2 TUPELO study in familial adenomatous polyposis, expanded adult population, Sep 10, 2026 cutoff: at 12 weeks about 87% of patients had reduced total polyp burden (median reduction 47%) and 92% had reduced upper-GI polyp burden (median 55%), with most patients still below baseline at Week 25 after 12 weeks off treatment; no treatment-related adverse events above Grade 3 (safety n=26); registrational trial planned 2H 2027 pending FDA discussions, full data at CGA-IGC on Nov 2. REC-1245 (RBM39 degrader) Phase 1 DAHLIA dose escalation in heavily pretreated solid tumors, Aug 21, 2026 cutoff: 3 partial responses at 45 mg QD or higher (2 confirmed, both endometrial cancer; 1 unconfirmed, hepatocellular), disease control rate 91.7% (11/12) at 45 mg or higher, no dose-limiting toxicities or treatment-related serious adverse events at the 45 mg expansion dose. Release also announced Sanofi's option exercise on a first development candidate from their collaboration ($10M milestone) and a $200M oversubscribed follow-on offering.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

EMA's CHMP adopted a positive opinion recommending EU marketing authorization for RASONQUE (daraxonrasib) once-daily oral tablets as monotherapy for adults with metastatic pancreatic adenocarcinoma who have received at least one prior systemic therapy, after an expedited phased review under EMA's Cancer Medicines Pathfinder project; a European Commission decision is now pending. The opinion rests on the Phase 3 RASolute 302 trial versus investigator's-choice chemotherapy, which met all primary and key secondary endpoints in both the RAS G12-mutant and overall intent-to-treat populations: in the ITT population, overall survival HR 0.40 (95% CI 0.30-0.53; p<0.0001) with median OS 13.2 months versus 6.7 months, and progression-free survival HR 0.49 (95% CI 0.38-0.64; p<0.0001) with median PFS 7.2 versus 3.6 months; a later ad hoc OS analysis showed median OS 12.4 versus 6.7 months (HR 0.49). Most common adverse reactions were diarrhea, dermatitis acneiform, stomatitis, nausea, vomiting and fatigue. RASONQUE was FDA-approved on August 26, 2026. Announced October 15, 2026 (GlobeNewswire).

Offering prospectus supplement filed

FDA approved Enspryng (satralizumab-mwge) for the treatment of adults with thyroid eye disease (TED), its second US indication after NMOSD (2020) and the first and only at-home subcutaneous treatment option for TED, following priority review of the sBLA. Approval was based on the Phase III SatraGO program (SatraGO-1 NCT05987423 and SatraGO-2 NCT06106828, 258 patients total): SatraGO-1 did not meet its pre-specified primary efficacy endpoint at week 24, while SatraGO-2 showed a proptosis response of 52.9% on Enspryng vs 23.4% on placebo and diplopia reduction of 60.7% vs 25.8% in the active TED population. No new safety signals were identified. Genentech announced October 15, 2026, on the PDUFA date tracked for this program.

EMA's CHMP recommended an extension of indication for Tukysa (tucatinib) to include use in combination with trastuzumab and pertuzumab for first-line maintenance treatment of adults with unresectable locally advanced or metastatic HER2-positive breast cancer following induction treatment. The recommendation mirrors the FDA approval of the same regimen on October 7, 2026, which rested on the Phase 3 HER2CLIMB-05 trial (NCT05132582, n=654): median progression-free survival 24.9 months on the tucatinib combination versus 16.3 months on placebo plus trastuzumab and pertuzumab (HR 0.64, 95% CI 0.51-0.80, p<0.0001), with overall survival not yet mature. A European Commission decision is now pending. Announced October 16, 2026 (EMA CHMP meeting highlights, opinion adopted October 15, 2026).

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01: financing-related)

FDA sent a Discipline Review Letter on the ANDA for NRX-100, its preservative-free ketamine product, requesting only minor labeling changes and sample labeling using the proposed proprietary name, which remains under FDA review, ahead of the November 10, 2026 GDUFA goal date. The company is completing final manufacturing process validation runs required for launch.

EMA's CHMP adopted a positive opinion recommending EU marketing authorization for Foundayo (orforglipron), a once-daily oral GLP-1 receptor agonist, for weight management and for the treatment of type 2 diabetes mellitus in adults. For weight management the indication is as an adjunct to healthy diet and physical activity in adults with BMI >=30 kg/m2, or >=27 to <30 kg/m2 with at least one weight-related comorbidity. For type 2 diabetes it covers adults with insufficiently controlled disease, as monotherapy when metformin is inappropriate or in combination with other diabetes medicines. Applicant is Eli Lilly Nederland B.V.; a European Commission decision is now pending. Foundayo was FDA-approved for obesity in April 2026. Announced October 16, 2026 (EMA CHMP meeting highlights, opinion adopted October 15, 2026).

2026 Animal Study #1 (GLP-1-A26-1) results in Sprague-Dawley rats (11 arms, DehydraTECH-CBD and DehydraTECH-semaglutide formulation enhancements vs historical reference formulations): DHT-CBD ALT2 showed a 566% improvement in brain absorption at 8 hours (62.8 ng/g vs REF 9.42 ng/g), and DHT-CBD ALT arms and DHT-SEMA ALT2 blood plasma absorption were not statistically significantly different from REF, while DHT-SEMA brain levels were below the assay quantitation limit. Lexaria filed 3 new patent applications related to the formulations evaluated.

FDA cleared the IND application for LB2501, an off-the-shelf in vivo CD19/CD20 dual-targeting CAR-T therapy given as a single infusion without lymphodepletion, for relapsed or refractory B-cell malignancies including B-cell non-Hodgkin lymphoma. A planned U.S. Phase 1/2 study will assess safety, tolerability, pharmacokinetics and preliminary efficacy. LB2501 is described as the first dual-targeting in vivo CAR-T to receive FDA IND clearance.

Positive clinical data for KB113 (low-dose intratumoral KB707, an HSV-1 therapy expressing IL-2 and IL-12) in a Gorlin syndrome pilot study (3 patients, 6 doses over 14 weeks): 69 of 83 assessed basal cell carcinoma landmark lesions regressed (83.1%), 42 of 83 (50.6%) regressed more than 50%, and responses occurred in both injected lesions (29/36, 80.6%) and non-injected lesions (40/47, 85.1%), consistent with an abscopal effect; one biopsied lesion per patient showed histologically confirmed complete clearance. All adverse events were mild or moderate, transient and resolved. FDA discussions on advanced development have begun; data presented at PeDRA, Oct 15-17, 2026.

Positive clinical data presented at Retina in Rio 2026 (Oct 15, 2026) for two gene therapy programs in inherited retinal diseases. OPGx-BEST1 Phase 1/2 BIRD-1 low-dose Cohort 1 (5 participants treated, follow-up 3 to 6 months): 3 of 4 evaluable participants (75%) showed improved retinal sensitivity in the treated transitional zone on microperimetry, and responders also had reductions in intraretinal fluid; new analyses linked retinal structure (OCT/imaging) to visual function and treatment response. High-dose Cohort 2 enrollment is complete (11 participants). OPGx-LCA5 Phase 1/2 study (6 participants at the low dose of 1E10 vector genomes per eye, 3 adults and 3 adolescents): adults showed sustained improvements in visual acuity and light sensitivity (full-field stimulus testing vs untreated eyes) through 24 months, and pediatric participants showed improvements through 6 months, including restoration of cone-mediated vision with sensitivity reaching normal ranges; microperimetry showed increased retinal sensitivity. The OPGx-LCA5 Phase 3 trial's natural history and treatment cohorts are fully enrolled, with dosing initiation expected in Q4 2026. No safety summary was disclosed in the release.

First patient dosed in the Phase 1 trial (NCT07493122) of IMC-S118AI in HLA-A*02:01-positive patients with type 1 diabetes. IMC-S118AI is the company's first tissue-specific autoimmune candidate: a bispecific PD-1 agonist designed to protect pancreatic beta cells from T-cell attack and preserve insulin production. The trial assesses safety and clinical activity.

FDA cleared the IND for EB06, a fully human anti-CXCL10 monoclonal antibody, allowing the already-enrolling Phase 2 study in moderate-to-severe nonsegmental vitiligo to expand to U.S. sites. The randomized, double-blind, placebo-controlled study (up to 80 adults, randomized 1:1 to EB06 or placebo every two weeks through Week 22, primary endpoint mean percent change in Facial VASI at Week 28) continues enrolling in Canada and Poland, with first U.S. dosing expected in Q1 2027 and topline results expected in Q4 2027.

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01: financing-related)

FDA cleared the IND application for blarcamesine (ANAVEX2-73) in Fragile X syndrome (FXS), so Anavex may proceed with its planned U.S. clinical study. The planned ANAVEX2-73-FXS-001 Phase 2a study is randomized, double-blind and placebo-controlled, evaluating safety, tolerability and pharmacokinetics of blarcamesine oral solution in approximately 60 adult males with FXS, with behavioral (ABC-FX) and cognitive (NIH Toolbox) measures as secondary endpoints. Anavex now has open INDs in all three core indications (Alzheimer's disease, Rett syndrome and Fragile X); blarcamesine previously received FDA Orphan Drug Designation for FXS, for which there is no FDA-approved treatment.

FDA accepted the resubmitted Anaphylm (dibutepinephrine) sublingual film NDA for Type I allergic reactions, including anaphylaxis, as complete, classified it as a Class 2 response, and assigned a PDUFA goal date of March 16, 2027. The Sep 16, 2026 resubmission followed the Jan 30, 2026 CRL and the Aug 2026 positive human-factors and PK study results. A Canadian NDS is planned for Q4 2026.

8-K filed (items 1.01, 3.02: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

Positive initial efficacy and updated safety data from the Phase 1/2 TARGET-D 101 trial of VS-7375, an oral KRAS G12D (ON/OFF) inhibitor, in advanced KRAS G12D-mutated solid tumors (data cutoff Oct 7, 2026). In metastatic pancreatic ductal adenocarcinoma on 900 mg QD monotherapy (mITT n=22, median follow-up 6.4 months): confirmed ORR 50% (4/8) in 2L, 41% (9/22) in 2L+, and 36% (5/14) in 3L/4L, including two complete responses (one confirmed); disease control rate 86% in 2L+, 6-month landmark PFS rate 67% and OS rate 80%, with 16/22 still on treatment. In 2L+ NSCLC on 600 mg QD monotherapy, confirmed ORR was 43% (10/23). In 3L+ metastatic colorectal cancer, 900 mg QD plus anti-EGFR therapy gave a 33% ORR (2/6, one confirmed). Safety at 900 mg monotherapy: discontinuation due to adverse events 2%, Grade 3 treatment-related adverse events 10%. Verastem also reached FDA alignment on the design of a Phase 3 trial of VS-7375 in first-line metastatic PDAC and plans to open it by year-end 2026.

Phase 3 Study 3201 (NCT07221578, n=546) of VQW-765, an alpha7 nicotinic acetylcholine receptor partial agonist, for on-demand treatment of acute anxiety in adults with social anxiety disorder did NOT meet its primary endpoint: SUDS distress during the performance phase of the Trier Social Stress Test in females, LS mean difference -0.4 vs placebo (95% CI -5.32 to 4.62, p=0.8899); key secondary in the full population also missed (LS mean difference 0.9, p=0.6605). Single-dose study. VQW-765 was safe and well tolerated, consistent with prior studies. The Phase 3 results did not confirm the earlier Phase 2 findings, and the VQW-765 program is on hold pending further analysis of the full dataset.

8-K filed (items 3.02: financing-related)

Positive topline from Cohort 1 of the Phase 1/2 ELEVATE-45-201 MAD study in ambulatory DMD amenable to exon 45 skipping (n=8, randomized 3:1; three doses of 5 mg/kg Q6W or placebo): primary safety/tolerability objectives met; mean dystrophin increase 0.10% from baseline in treated participants (n=6, nominal p=0.03) vs a 0.02% mean decrease on placebo; mean exon skipping increase 0.15% (n=5) vs a 0.04% mean decrease on placebo; no functional benefit expected or observed at this dose/duration. Safety: headache the most common TEAE, one participant had two serious TEAEs (vomiting and pyrexia) that resolved, no discontinuations, no hypomagnesemia or renal concerns; all Cohort 1 participants progressed to the open-label Phase 2 portion. Cohort 2 (10 mg/kg) enrollment complete, data expected 1H 2027; Cohort 3 up to 15 mg/kg to follow. A DMC recommendation to proceed to 18 mg/kg in Cohort 3 of ELEVATE-44-201 was also noted.

Detailed results from the pivotal Phase 2/3 IgGNITE trial (TAK-881-3001, NCT05755035) of TAK-881 (facilitated subcutaneous immunoglobulin 20% with recombinant human hyaluronidase) in primary immunodeficiency diseases, presented as late-breaking posters at ESID 2026 (participants aged 16 and older, randomized crossover vs HYQVIA): the trial had previously met its primary endpoint of pharmacokinetic comparability, with steady-state IgG trough geometric means of 11.7 vs 11.9 g/L, above protective thresholds. New detailed data: the same immunoglobulin dose delivered in 50% less volume, with median infusion times of 75.8 vs 132 minutes using one infusion site (58.3 vs 86 minutes using two sites); annualized infection rates of 2.62 vs 2.73; an annualized serious bacterial infection rate of 0.0558 on TAK-881, with no acute serious bacterial infections reported on HYQVIA; treatment-emergent adverse events in 56.1% (23/41) on TAK-881 vs 50.0% (19/38) on HYQVIA, mild to moderate, with no TAK-881-related Grade 3 or higher events, no TAK-881-related serious adverse events, and no discontinuations due to TAK-881-related adverse events (9 of 43 randomized participants discontinued across both treatment periods). Takeda plans regulatory submissions in the US, EU and Japan in fiscal 2026; the long-term extension study TAK-881-3002 (NCT06076642) is ongoing.

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01: financing-related)

EMA granted Orphan Drug Designation to QRX003 for the treatment of Peeling Skin Syndrome, the eighth regulatory designation for QRX003 across Peeling Skin Syndrome and Netherton Syndrome (it already holds FDA Fast Track and Rare Pediatric Disease designations for Peeling Skin Syndrome). The EU designation provides scientific advice on study protocols, fee reductions and grant access and, if QRX003 is approved in the EU, 10 years of market exclusivity for the indication. Quoin remains on track to initiate the first formal clinical trial in Peeling Skin Syndrome before year-end 2026.

Full Phase 2 data presented at ESID 2026 (Oct 14-17, Maastricht) for leniolisib in genetically identifiable PIDs with immune dysregulation linked to PI3Kδ signaling (single-arm, open-label, intra-patient dose-escalation; n=13 with CTLA4, SOCS1, NFKB1 or FAS variants, 9 also with CVID; leniolisib BID for 20 weeks, 10 mg for 4 weeks then 30 mg for 4 weeks then 70 mg for 12 weeks): mean spleen volume reduction from baseline 26.4% (n=11, reductions in 10 of 11 evaluable); mean index lymph-node lesion size reduction 33.7% (n=4, reductions in all 4); percent-predicted DLCO increased by a mean of 8.0% among participants with impaired gas exchange at baseline (n=7); CT-assessed interstitial lung disease scores decreased in 4 of 5 evaluable; liver stiffness improved in 2 of 3 with heightened baseline stiffness. Safety: generally well tolerated, infections the most common AEs, all treatment-related AEs Grade 1, two unrelated SAEs, one early discontinuation for acute hepatitis in a participant with a history of portal hypertension, no new safety signals. All 12 participants who completed the study enrolled in the three-year extension study (NCT06990529). Full dataset builds on the September 22, 2026 topline announcement. Separate Phase 2 leniolisib topline in CVID now expected November 2026.

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01, 1.02: financing-related)

Shelf registration filed (enables future raises incl. ATM)

FDA approved an expanded indication for Anktiva (nogapendekin alfa inbakicept-pmln, Altor BioScience, an indirect wholly-owned subsidiary of ImmunityBio) in combination with BCG for adults with BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The approval revises the prior indication (BCG-unresponsive NMIBC with carcinoma in situ, with or without papillary tumors) to include patients with high-grade papillary-only tumors.

8-K filed (items 1.01: financing-related)

Submitted Marketing Authorization Applications in Singapore, the United Kingdom and Switzerland via the Access Consortium pathway for oral tinlarebant in Stargardt disease type 1 (STGD1), where it would be the first approved treatment if cleared. The filings add to the accepted U.S. NDA (Priority Review, PDUFA February 12, 2027) and the Japanese submission under the Sakigake designation.

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

FDA accepted the resubmitted BLA for tabelecleucel (partner Pierre Fabre Pharmaceuticals) as monotherapy for adult and pediatric patients aged 2 years and older with EBV-positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. The Class 2 resubmission carries a PDUFA target action date of March 31, 2027. The accepted resubmission includes updated data with additional patients and longer follow-up from the pivotal Phase 3 ALLELE study, plus supplemental expanded-access and European commercial experience data. If approved, Tab-cel would be the first therapy available in the U.S. for EBV+ PTLD, and Atara would be eligible for a $31 million milestone payment from Pierre Fabre plus double-digit tiered royalties.

Interim Phase 1/2a data (AROMAPT-SC-1001, NCT07221344) in healthy volunteers: subcutaneous ARO-MAPT delivered siRNA across the blood-brain barrier and reduced CSF total tau at Day 105 by a mean of 40% at 2.1 mg/kg and 69% at 6.4 mg/kg (dosed Days 1, 8 and 15), and by a mean of 73% (maximum individual reduction 85%) at 12.8 mg/kg (dosed Days 1 and 15), sustained 90 days after the final dose, supporting potential quarterly or less-frequent dosing. First clinical validation of the TRiM platform for CNS delivery. Safety: generally well tolerated, most TEAEs Grade 1/mild, no serious TEAEs, deaths or discontinuations; most common TEAEs were injection-site reactions, vessel puncture-site bruising, upper respiratory tract infection, back pain and headache. Early Alzheimer's disease cohorts are enrolling, with further data updates expected in 2027.

8-K filed (items 1.01: financing-related)

Registration statement amended

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 2.03: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus supplement filed

8-K filed (items 3.02: financing-related)

Post-hoc Phase 2 data at ASBMR 2026 (Oct 9-12) for EB613 in postmenopausal women: in the 2.5 mg subgroup with baseline T-score at or below -2.5, placebo-adjusted BMD gains at 6 months were 4.23% at the total hip (n=6) and 4.22% at the lumbar spine (n=9), vs 1.02% and 1.59% respectively in women with T-score above -2.5; the pooled 2.5 mg group (n=21) gained 1.84% (total hip) and 2.72% (lumbar spine). Bone turnover marker changes were also larger in the lower T-score group. Exploratory, hypothesis-generating analysis; supports the planned Phase 3 population (about 900 patients, primary endpoint Month 12 total hip BMD change). Announced October 13, 2026 (GlobeNewswire).

Offering prospectus supplement filed

8-K filed (items 1.01, 2.03, 3.02: financing-related)

Offering prospectus supplement filed

First topline from Phase 2/3 AHEAD-MERIT (NCT04534205) in 1L unresectable recurrent/metastatic HPV16+ HNSCC expressing PD-L1: BNT113 + pembrolizumab did NOT demonstrate a statistically significant improvement in progression-free survival vs pembrolizumab alone; PFS is one of two dual primary endpoints (with overall survival). IDMC recommended the trial continue as planned to evaluate OS; under the pre-specified design a statistically significant OS improvement would establish a positive trial outcome despite the PFS miss. BioNTech remains blinded. Announced October 13, 2026 (06:45 ET, GlobeNewswire).

Phase 1 GLP-131 results (NCT07223216) for BMF-650, an oral small-molecule GLP-1 receptor agonist, in overweight/obese healthy adults: mean Day 29 weight reduction 1.5% to 3.2% across outpatient MAD Cohorts 1-4 vs 0.1% for pooled placebo; a fifth confined cohort with one-week titration to 400 mg QD showed 6.1% mean reduction, which the company said should be read separately because confined placebo participants also lost similar weight on reduced food intake. Across the five MAD cohorts (n=40) all TEAEs were mild or moderate, with no severe or serious TEAEs and no TEAE-related discontinuations. Company said further development needs longer toxicology work and it is positioning the program for a strategic partnership, with primary focus remaining on icovamenib. Announced October 13, 2026 (16:08 ET, GlobeNewswire).

Initial Phase 1/2 data for BEAM-301, an in vivo base editor correcting the R83C mutation in glycogen storage disease type Ia (GSDIa), to be presented at ESGCT Oct 27-30, 2026 (data cutoff June 26, 2026): 6 patients received a single infusion (3 at 20 mg, 3 at 40 mg). By Day 36 both evaluable 40 mg patients reached the protocol maximum of at least 15 hours of controlled fasting, consistent with restored endogenous glucose production (baseline median time to hypoglycemia 2.5 hours at 40 mg and 2.9 hours at 20 mg; 20 mg median rose to 4.3 hours by Day 36). Prescribed daily starch intake fell 71% and 92% in the two evaluable 40 mg patients. Safety consistent with LNP therapies: 5 serious adverse events resolved in 2 to 10 days including 3 Grade 3 infusion-related reactions, transient transaminase elevations mostly Grade 1, no dose-limiting toxicities, discontinuations or deaths. Announced October 13, 2026 (07:00 ET, GlobeNewswire).

8-K filed (items 1.01, 3.02: financing-related)

New Phase 3 AFFIRM (NCT06063967, n=289) endoscopic data at ACG 2026 in moderately to severely active Crohn's disease: Week 24 endoscopic remission 35.8% on SKYRIZI SC vs 9.1% placebo (advanced therapy-naive 45.1%, inadequate responders 30.1%); Week 24 ulcer-free endoscopy 36.6% vs 9.1% (naive 49.0%, inadequate responders 28.9%); Week 12 endoscopic remission 27.7% vs 10.2%. Builds on previously reported Week 12 co-primary superiority (clinical remission and endoscopic response). Safety through Week 24 consistent with the known profile, no new signals. Data support the FDA application for subcutaneous induction, currently under FDA and EMA review. Announced October 13, 2026 (PR Newswire).

New Phase 3 INDIGO open-label extension data in IgG4-related disease presented at the 6th International Symposium on IgG4-RD: 12-month flare-free probability 0.93; obexelimab significantly reduced cumulative glucocorticoid toxicity vs placebo at week 52 (p=0.021); vaccine sub-study showed preserved immune responses.

POSITIVE top-line results from Cohort 1 (gluten challenge, n=30) of the Phase 1b ANB033 trial in celiac disease. Statistically significant difference vs placebo across key histologic endpoints: LS mean change in villus height-to-crypt depth (Vh:Cd) ratio, intraepithelial lymphocyte (IEL) density, and VCIEL composite scale, plus symptom endpoints (CDSD). After 4 weeks of treatment followed by a 14-day gluten challenge (6g/day), ANB033 prevented IEL density increase and kept Vh:Cd changes below the clinically meaningful threshold. Well tolerated with no safety signals observed to date. Company to advance ANB033 into late-stage development in 2027. Cohort 2 (mucosal healing, up to 40 pts) topline expected Q1 2027. Conference call held Oct 12, 2026 (8:30am ET).

Publication of a 5-year matching-adjusted indirect comparison (MAIC) of BRIUMVI vs ocrelizumab and ofatumumab in relapsing MS (Multiple Sclerosis and Related Disorders): 24-week confirmed disability progression hazard was 44% lower vs ocrelizumab (HR 0.560) and 49% lower vs ofatumumab (HR 0.509); estimated CDP24 at 240 weeks 9.3% vs 16.1% (ocrelizumab) and 9.4% vs 17.9% (ofatumumab). Exploratory unanchored analysis of ULTIMATE I/II patient data against published OPERA and ASCLEPIOS data; requires head-to-head confirmation.

Phase 2 MAXPIRe topline in idiopathic pulmonary fibrosis (IPF): trial did not meet its primary endpoint (annualized rate of FVC decline). Axatilimab was well tolerated with safety comparable to placebo and no new safety signals. Syndax will not move forward with further development of axatilimab in IPF; ongoing cGVHD trials are unaffected, with results expected later this year and early 2028.

Positive 84-week extension results from Phase 3 OPTIMA in adults with fibrodysplasia ossificans progressiva (FOP) (63 randomized; 58 completed the double-blind extension to at least 84 weeks): from week 56 to week 84, no new heterotopic ossification lesions on Pasatru vs 9 new lesions on placebo by CT; new clinician-assessed flare-ups 1 on 10 mg/kg, 14 on 3 mg/kg, 17 on placebo. Safety generally consistent with the 56-week profile; skin and soft tissue infections were dose-dependent (33% placebo, 53% 3 mg/kg, 78% 10 mg/kg), all resolved, with no discontinuations for infection and no deaths. Data presented as a late-breaking oral session at ASBMR on Oct 12, 2026. Pasatru was FDA-approved in Aug 2026 for FOP; a pediatric Phase 3 (OPTIMA 2) is planned to start later this year.

Five ACG 2026 posters report Phase 1a/b clinical, PK, PD and mechanistic data strengthening Phase 2 rationale for oral PDE4 prodrug PALI-2108 in ulcerative colitis and Crohn's disease: all 5 UC patients responded with 2 in remission; 47.5% mean SES-CD reduction in Crohn's patients. Phase 2 ongoing.

Nurix discontinued further clinical development of NX-2127 (zelebrudomide), its oral BTK degrader, citing strategic prioritization of its wholly owned oncology portfolio. Patients already on NX-2127 may stay on treatment with continued safety monitoring. Announced October 12, 2026 in the Q3 2026 update (GlobeNewswire).

First participant dosed in RESTORE, an FDA-aligned Phase 3 registrational study of NVG-291 in chronic spinal cord injury (primary endpoint: hand function). Enrollment completion expected H2 2027; topline data expected H1 2028.

Phase 3 EXPAND study MET primary endpoint plus all 5 prespecified secondary endpoints: maralixibat 285 ug/kg BID vs placebo in 52 pts (46 pediatric, 6 adult) with ultra-rare cholestatic liver diseases incl. biliary atresia, 20-wk double-blind. Pediatric pruritus: mean 1.81-pt ItchRO(Obs) improvement vs 0.83 placebo (placebo-adjusted diff 0.98, p=0.0004); serum bile acids -70.1% vs -3.5% (69.0% treatment effect, p<0.0001). No new safety signals. sNDA planned H1 2027. Call held Oct 12, 2026 (8:30am ET).

Phase 3 GALAXI 2 and 3 (NCT03466411) Week 144 long-term extension data at ACG 2026 in moderately to severely active Crohn's disease: clinical remission 94% on TREMFYA 100 mg every 8 weeks and 91% on 200 mg every 4 weeks; 90-day corticosteroid-free clinical remission 88% on both regimens; endoscopic remission 52% (q8w) and 61% (q4w); 74% of treated patients remained on treatment through three years. Safety consistent with the known profile. Announced October 12, 2026 (PR Newswire).

Phase 3 FUZION (NCT05347095) prespecified subgroup analyses at ACG 2026 in Crohn's disease with perianal fistula: Week 24 combined fistula remission consistent across subgroups on TREMFYA (guselkumab) vs placebo, including prior biologic exposure (21% on 100 mg every 8 weeks and 29% on 200 mg every 4 weeks vs 5% placebo), baseline CDAI at or above 220 (30% and 21% vs 0%), and proctitis at baseline (26% and 27% vs 12%); overall population 28% (100 mg q8w) and 27% (200 mg q4w) vs 10% placebo. Adverse events consistent with the known profile. Announced October 12, 2026 (PR Newswire).

Additional ACACIA-HCM analyses presented at HFSA 2026 in non-obstructive HCM: prespecified comprehensive exercise (CPET/BEAP-RS) score improved vs placebo at Week 36 (p<0.001); global responder analysis showed 53% of aficamten patients responded in 3 or more of 5 domains vs 13% on placebo (p<0.001). Safety: LVEF <50% in 10.5% on aficamten vs 0.8% on placebo; heart-failure SAEs 4.7% vs 1.2%. Supplemental NDA submission planned this quarter.

Initial results of Phase 2a trial of neflamapimod in nonfluent variant primary progressive aphasia (nfvPPA): positive effects on key neurodegeneration biomarker - mean plasma NfL reduced ~3.1 pg/mL at 12 weeks with 72% of elevated-baseline participants declining. 24-week biomarker data expected at CTAD Nov 2026; clinical data expected Q1 2027.

POSITIVE topline from planned interim analysis of Phase 3 ACTION-1 (NCT05477576) of RYZ101 (actinium-225 DOTATATE) vs investigator's choice SOC in SSTR+ GEP-NETs progressed after Lu-177: met primary endpoint with statistically significant and clinically meaningful PFS improvement by blinded independent central review; safety consistent with known profile. First Phase 3 data for an alpha-based targeted RPT. FDA granted Orphan Drug Designation Aug 2026; company to engage FDA on regulatory submission. Detailed results at future medical meeting.

Biomea to announce Phase I results on October 13, 2026 (conference call and webcast 4:30 p.m. ET) from the GLP-131 study of BMF-650, an investigational next-generation oral small-molecule GLP-1 receptor agonist for diabetes and obesity. Announced October 12, 2026 (GlobeNewswire).

ACG 2026: real-world study (470 IBS-C pts, median 23.6-mo follow-up) showed reduced healthcare resource utilization after IBSRELA initiation; separate poster presented design of an ongoing Phase 3 study of tenapanor in cholestatic pruritus.

Positive Phase 4 open-label single-arm SYNCHRONICITY data (NCT05322070, n=108) in chronic non-infectious uveitis of the posterior segment, presented Oct 9, 2026 at AAO 2026 Uveitis Subspecialty Day: met both co-primary endpoints at 6 months - mean BCVA improved +3.6 letters (p=0.0077), mean central subfield thickness decreased 157.5 microns (p<0.0001); at 24 months BCVA +5.5 ETDRS letters, CST -171.2 microns, 79.6% of eyes with no inflammation recurrence. Safety consistent with prior fluocinolone implant trials (ocular hypertension 6%, cataract 5%). Announced October 12, 2026 (6:55am ET).

First Tracks to announce top-line data on October 12, 2026 (investor call 8:30 AM ET) from Cohort 1 of the global Phase 1b trial of ANB033, a CD122 antagonist, in celiac disease: gluten-challenge study assessing prevention of mucosal damage. Context: argenx's FB102 (same CD122 mechanism) hit its Phase 2 primary endpoint in celiac disease on Oct 8, 2026.

Mirum to share topline results on October 12, 2026 (investor call 8:30 AM ET) from the Phase 3 EXPAND study of LIVMARLI (maralixibat) for cholestatic pruritus in patients 6 months and older with ultra-rare cholestatic liver diseases, including biliary atresia.

Pivotal Phase 3 ALOFT-IPF trial (n~1,255, admilparant 60mg/120mg BID vs placebo for >=52 weeks) MET primary endpoint at the 60mg dose: statistically significant improvement vs placebo in absolute change in FVC from baseline to Week 52. The 120mg dose slowed FVC decline but was not statistically significant. Favorable safety and tolerability profile across both doses; discontinuations due to adverse events low and similar to placebo. Supports first-in-class LPA1 pathway in IPF. ALOFT-PPF (progressive pulmonary fibrosis) topline expected early 2027. Detailed results to be presented at a future medical meeting.

Phase 3 CALIBRATE 24-week data in ADH1 presented at ASBMR 2026: encaleret restored endogenous PTH to at/above the lower limit of normal in 91% of participants vs 0% on standard of care, with increased bone turnover markers; 100% reported improvement in at least one ADH1 symptom vs 46% on SoC. FDA accepted the NDA for encaleret in ADH1 with Priority Review, PDUFA target action date May 8, 2027; MAA submitted to EMA. First participant dosed in RECLAIM-HP, a global randomized, double-blind, placebo-controlled registrational Phase 3 study of oral encaleret in ~160 adults and adolescents with chronic hypoparathyroidism. BridgeBio announced October 11, 2026.

Independent real-world study of U.S. electronic health records: acoramidis associated with significantly improved clinical outcomes versus tafamidis in ATTR-CM. This is the second independent RWE data release BridgeBio has telegraphed for 2026 (following the September 23 claims-data study published in Cardiology and Therapy). BridgeBio announced October 11, 2026.

HICKORY Phase 3 missed its primary endpoint: efzimfotase alfa showed only a numerical improvement in Six-Minute Walk Test vs placebo (+17.05 m LS mean difference, p=0.1675) at week 25 in treatment-naive adolescents/adults (>=12 yrs) with hypophosphatasia, blamed on better-than-expected placebo performance in the adult-onset subgroup; post-hoc MCID analysis favored drug (OR 2.50, nominal p=0.0363). Nominally significant FACIT-Fatigue benefit (p=0.0016); other secondaries numerically favored drug. Announced Oct 11, 2026; presented same day at ASBMR 2026. Priority Review for HPP (ages >=2) still pending after Sept 18 grant.

New 1-year (52-week) data from Phase III MEERKAT and SANDCAT studies of vamikibart in uveitic macular edema (UME): sustained improvements in vision and reductions in macular thickness vs sham procedure; data presented at AAO 2026. FDA accepted the BLA (approval decision expected by July 2027); if approved, first non-steroid targeted treatment for UME. Filings also accepted in EU, China and Japan.

First 1-year results from the pivotal Phase 2b/3 BRUNELLO trial in diabetic macular edema presented at AAO 2026: both dose arms met the primary endpoint with non-inferior BCVA gains vs monthly ranibizumab (+9.1/+8.7 vs +11.8 letters); no secondary endpoint demonstrated superiority; higher proliferative diabetic retinopathy AEs (6.7%/6.1% vs 0.9%) and higher AE discontinuations (4.9%/4.5% vs 0.9%).

New interim data from the Phase 3 open-label extension in children ages 2-5 presented as a late-breaker at NACFC 2026: recovery of exocrine pancreatic function (mean FE-1 239.7 ug/g, +110.2 from baseline; 48.6% reached the pancreatic-sufficiency threshold of >=200 ug/g); 18 of 48 PERT-substudy children discontinued pancreatic enzyme replacement therapy.

FDA approved WELTRUZA (olanzapine) for extended-release injectable suspension — first and only once-monthly subcutaneous long-acting injectable olanzapine for adults with schizophrenia (Oct 9, 7:00am ET GNW). No loading doses, no oral supplementation, no post-injection monitoring. Based on pivotal Phase 3 SOLARIS (675 patients, 8 weeks): symptom (PANSS), illness severity (CGI-S), and personal/social functioning (PSP) improvement; safety consistent with oral olanzapine. Launching in coming weeks in 318/425/531 mg strengths. EMA accepted MAA (TEV-749) May 2026; not yet EMA-approved.

Offering prospectus filed (possible follow-on or ATM takedown)

First scientific presentation of Phase 1 PK data at ECNP Congress (poster PS02-2029, Oct 11): detailed analyses of the completed multi-part proof-of-concept trial (174 healthy volunteers) show dose-dependent increases in agomelatine exposure, no food effect, no accumulation over 7 days of MAD dosing, and 10-fold lower PK variability vs unmodified agomelatine; supports the 6.8-fold bioavailability increase in previously reported topline. Phase 2a in GAD + sleep disturbance initiated (topline early 2028); Phase 2/3 in GAD planned for 1H 2027.

Positive full 52-week results from license partner Ascletis' Phase 3 open-label extension trial of denifanstat in moderate-to-severe acne: continued improvement and sustained efficacy across all efficacy endpoints through 52 weeks; U.S. AURORA Phase 3 trial now screening patients. Data to be presented at 2026 FALC meeting.

Offering prospectus filed

8-K filed (items 1.01: financing-related)

Endpoints (Oct 9) reported new Rasonque data: patients with the most common KRAS mutation (G12D) in pancreatic cancer showed a lower response rate than the overall patient population; RVMD shares fell 6%+ on the report, which framed it as leaving an opening for rival treatments. Underlying dataset/venue not disclosed in the accessible article text; no company press release on GlobeNewswire dated Oct 9.

FDA approved Tecentriq (atezolizumab) and Tecentriq Hybreza (atezolizumab and hyaluronidase-tqjs), each in combination with a fluoropyrimidine and oxaliplatin, for the adjuvant treatment of stage III dMMR colon cancer — 12th US indication and first approved immunotherapy-based adjuvant regimen in this setting. Phase 3 ATOMIC (NCT02912559, n=712): atezolizumab + mFOLFOX6 reduced risk of recurrence or death by 50% (HR 0.50, p<0.001); 36-mo DFS 86% vs 76% (NEJM Mar 2026). Priority-review sBLA decided on the October 9, 2026 PDUFA date. Roche announced October 9, 2026.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 2.03: financing-related)

Shelf registration filed (enables future raises incl. ATM)

Offering prospectus supplement filed

New PK/PD analyses of Furoscix ReadyFlow autoinjector presented at HFSA ASM 2026 (Oct 9-12, Phoenix): comparable urine output to IV furosemide; consistent bioavailability, diuresis and natriuresis across injection sites; cross-study analysis showed comparable diuretic/natriuretic responses vs Furoscix On-body Infusor, supporting seamless transition to ReadyFlow. Builds on July 2026 FDA approval and U.S. launch for heart failure/CKD-related edema.

New lorundrostat data at AHA Hypertension Scientific Sessions 2026 (Oct 9, 9:00am ET GNW): from pivotal Phase 3 Launch-HTN (uncontrolled/treatment-resistant hypertension on 2-5 meds), 46% of lorundrostat 50 mg participants achieved AHA/ACC SBP target <130 mmHg at Week 12 and 70% achieved >=10 mmHg SBP reduction; final Phase 2 Explore-OSA results (overweight/obese adults with moderate-to-severe OSA + hypertension): evening 50 mg reduced morning SBP by 11.1 mmHg at Week 4 (p<0.0001) vs 1.0 mmHg placebo (p=NS), no change in apnea-hypopnea indices, no significant hyperkalemia. FDA NDA under review (per forward-looking statements).

First European study participant enrolled in the Phase 3 ReVeRA-301 pivotal trial of etripamil nasal spray for atrial fibrillation with rapid ventricular rate (AFib-RVR), at a Dutch Cardiovascular Investigator Network (WCN) site in the Netherlands; WCN leading European enrollment. ReVeRA-301: multinational, randomized, double-blind, placebo-controlled trial of the same 70 mg repeat-dose regimen FDA-approved for PSVT (marketed as CARDAMYST), self-administered at home prompted by symptoms; primary endpoint is reduction in ventricular rate within 30 minutes, key secondary is symptom improvement; target ~150 AFib-RVR patient events; Milestone pursuing a single-study sNDA registration pathway. U.S. enrollment began August 2026, since expanded to Canada. No readout timing given. Announced October 9, 2026 (8:00am ET).

Exploratory biomarker analysis from the Phase 3b TOGETHER-PsO trial (NCT06588283), presented at Fall Clinical Dermatology 2026: concomitant Taltz (ixekizumab) + Zepbound (tirzepatide) vs Taltz alone in adults with moderate-to-severe plaque psoriasis and obesity showed broader biomarker changes as early as Week 12 (482 vs 140 differentially expressed proteins and 467 vs 16 differentially expressed genes by Week 36); neutrophil-associated marker changes mediated part of the additional PASI response with the combination. NOT a new primary readout — the Week 36 primary endpoint results (superior skin clearance + clinically meaningful weight reduction with the combo) were previously reported.

Phase 3 ICONIC-LEAD (NCT06095115) Week 112 (2-year) late-breaking data at Fall Clinical Dermatology 2026 for icotrokinra (ICOTYDE, first targeted oral IL-23R peptide) in moderate-to-severe plaque psoriasis (adults + adolescents 12+): PASI 90 in >=72% and IGA 0/1 in >=70% sustained Week 64 through Week 112; complete clearance (PASI 100 >=44%, IGA 0 >=46%); clinically meaningful itch improvement sustained in 78%; 85% of PASI 90 responders regained response within 24 weeks of retreatment after Week 24 withdrawal; no new safety signals. ICOTYDE already approved in US, Europe, Japan, China.

FDA Type B meeting provided clear guidance for IND prep for lead candidate HCW11-018b (second-gen non-Fc-based T-cell engager, anti-CD3 x Tissue Factor BiTE + IL-15/IL-15Ra + TGFbRII trap) in solid tumors; cGMP manufacturing underway; IND application remains on track for 1H 2027.

Registration statement filed (primary offering)

Positive Phase 4 topline from randomized controlled trial GLK-101-04 of iDose TR administered with cataract surgery vs cataract surgery alone in OAG/OHT: met primary endpoint, mean diurnal IOP reduction 11.1 mmHg vs 7.4 mmHg (3.6 mmHg incremental, p<0.0001); superiority across all pre-specified secondary responder analyses; favorable safety, no treatment-related corneal endothelial cell loss or CME events.

Shelf registration filed (enables future raises incl. ATM)

Offering prospectus supplement filed

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 3.02: financing-related)

Offering prospectus supplement filed

8-K filed (items 1.01, 3.03: financing-related)

ZORYVE data at EADV + Fall Clinical 2026 (Oct 9, 8:00am ET GNW): Phase 2 INTEGUMENT-INFANT (101 infants ages 3 to <24 months with atopic dermatitis, ZORYVE cream 0.05% once daily): 34.4% achieved vIGA-AD Success at 4 weeks (n=96), 67.5% IGA-Scalp Success, 46.6% had >=25% itch improvement within 10 minutes of application; well tolerated, no serious AEs. Also new analyses from Phase 3 ARRECTOR and DERMIS-1/2 (scalp/body psoriasis clearance, sexual function), seborrheic dermatitis proteomics, and a real-world topical-corticosteroid survey. ZORYVE cream 0.05% for ages 3 to <24 months under FDA review with PDUFA date February 23, 2027.

New Phase 3 data: envudeucitinib rapidly and durably improved psoriasis burden across scalp, itch, and quality-of-life measures. ONWARD3: highest complete skin clearance seen (54% PASI 100) at Week 48; patients switching from placebo or apremilast achieved rapid responses comparable to continuous treatment at Week 48. On track to submit NDA for envudeucitinib in moderate-to-severe plaque psoriasis in 4Q 2026.

8-K filed (items 1.01, 1.02, 2.03, 3.02: financing-related)

8-K filed (items 1.01: financing-related)

FDA granted Fast Track designation to VIR-5500 (PSMA-targeted PRO-XTEN dual-masked T-cell engager, co-developed with Astellas) for late-line metastatic castration-resistant prostate cancer; Phase 3 planned 2027.

Resubmitted the apitegromab Marketing Authorisation Application to EMA for SMA (children and adults), replacing the Catalent Indiana fill-finish facility with the alternate site that supported the Sep-2026 US ISEMBYLD approval; CHMP opinion expected H1 2027.

8-K filed (items 1.01, 3.02: financing-related)

Phase 3 RAPIDe-3 results (NCT06343779) published in The Lancet: oral deucrictibant IR 20 mg for on-demand treatment of HAE attacks met the primary endpoint and all 11 secondary endpoints with high statistical significance; time to onset of symptom relief 1.28 hours and to complete symptom resolution 11.95 hours vs placebo; most attacks controlled with a single capsule; well tolerated, no safety signals. IR-capsule NDA under FDA review with PDUFA target April 23, 2027; MAA under EMA review; RAPIDe-2 open-label extension ongoing.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

Viatris agreed to acquire Pacira BioSciences for $36.50/share in cash (~$1.65B equity value, ~45% premium), definitive agreement announced Oct 8; expected to close by end of 2026, subject to customary conditions.

8-K filed (items 1.01: financing-related)

Announced expanded Phase 1b safety and efficacy data for givastomig (CLDN18.2 x 4-1BB bispecific) combined with nivolumab and mFOLFOX6 in previously untreated HER2-negative CLDN18.2+ PD-L1+ gastroesophageal adenocarcinoma, to be presented as poster 2945P at ESMO Congress 2026 (Oct 23-27, Madrid); global randomized Phase 2 (NCT07432295) underway following positive Phase 1b topline; FDA Fast Track designation for the indication.

8-K filed (items 1.01, 2.03, 3.02: financing-related)

Dosed the first patient in EQUINOX-HFpEF, a Phase 2 trial of EDG-15400 (oral selective cardiac sarcomere modulator) in adults with symptomatic HFpEF and LVEF >=50% (~90 participants planned).

Received regulatory and ethics (HREC) approval in Australia to begin first-in-human Phase 1/2 Strive trial of EDIT-401 (in vivo gene editing medicine targeting ApoC3) in patients with hyperlipidemia, including heterozygous familial hypercholesterolemia (HeFH).

To present late-breaking clinical data from the Phase 1b trial of CTX310 (CRISPR/Cas9 in vivo gene-editing therapy targeting ANGPTL3) in patients with severe hypertriglyceridemia as a late-breaking oral presentation (LBS.05, New Frontiers in Cardiometabolic Risk Reduction) at AHA Scientific Sessions 2026 on Monday, November 9, 2026, 8:00 a.m. CST; data embargoed until presentation. Abstract: "CRISPR-Cas9 Gene Editing Therapy Targeting ANGPTL3 for Severe Hypertriglyceridemia: Results From Phase 1b Trial."

First patient dosed in planned registrational Phase 2 study of CLN-049 (novel FLT3xCD3 T cell engager) in relapsed/refractory acute myeloid leukemia.

FDA granted Fast Track Designation to [212Pb]PSV359 for adults with locally advanced, unresectable or metastatic FAP-positive solitary fibrous tumors; in ongoing Phase 1/2a study.

Positive Phase 3 EXCALIBER-RRMM topline: ZENBEXUS + daratumumab + dexamethasone vs daratumumab/bortezomib/dexamethasone in RRMM (n=800): median PFS 42 vs 20 months, HR 0.49, p<0.000001, at median 23-month follow-up; builds on the MRD-neg CR dual-primary endpoint that supported FDA approval; detailed results at ASH.

Exclusive license to Ono Pharma for extracellular IgG degraders BHV-1300/BHV-1310/BHV-1320 in Japan, South Korea, Taiwan and ASEAN: up to $100M near-term ($80M at closing + $20M milestone within ~1 year), ~20% royalties; BHV-1300 in Phase 3 for Graves' disease.

Submitted supplemental NDA to FDA to expand Ameluz (aminolevulinic acid HCl) red-light PDT to mild-to-moderate actinic keratoses on the full body (extremities, neck, trunk; treatment fields up to ~240 cm² with adjusted illumination protocol), extending the label beyond face/scalp.

Positive topline results from Phase 2 FB102-301 study (NCT06982963, 126 adults with celiac disease under 8-week gluten challenge): met primary endpoint with statistically significant change from baseline in villus height-to-crypt depth ratio (Vh:Cd) at day 78 vs placebo (p=0.0176); no new safety signals. argenx plans to advance FB102 into Phase 3.

Discontinuing Phase 3 UNITY study of efgartigimod subcutaneous in moderate-to-severe Sjögren's disease: independent Data Monitoring Committee recommended stopping for futility after interim analysis, concluding the study is unable to meet its primary endpoint (change in clinESSDAI at Week 48). Safety consistent with established efgartigimod profile; no new safety signals.

Ministry of Health of Armenia granted the first-ever marketing authorizations for Botvymo (botensilimab, BOT) and Evamplix (balstilimab, BAL), each approved for use in combination, for previously treated adult MSS metastatic colorectal cancer without active liver metastases. First approvals of either medicine anywhere in the world; effective Oct 2, 2026; each conditional, valid one year and renewable. Agenus to evaluate approvals in other Eurasian Economic Union countries.

Spin-out Neurosterix (Addex holds 20% equity) completed Phase 1 clinical studies of NTX-253, a muscarinic M4 positive allosteric modulator for schizophrenia, and plans to advance into Phase 2 in schizophrenia patients; Phase 1 was a multi-part single/multiple ascending-dose design in healthy volunteers and schizophrenia patients yielding safety and pharmacokinetic data.

Positive Phase 2 ZUPREME-2 topline (NCT06926842): petrelintide met its primary endpoint with up to 9.2% mean body-weight reduction at week 28 vs 2.0% with placebo (all three once-weekly dose arms statistically significant), plus placebo-adjusted HbA1c reductions of 0.61-0.88%, in 220 US adults with overweight/obesity and type 2 diabetes. Tolerability comparable to placebo (GI-related discontinuation 1.9% vs 1.7%); no unexpected safety signals. Zealand and Roche have started the Phase 3a program (ZUPREME-3/4/5, ~7,000 participants total). Announced October 7, 2026.

FDA granted Orphan Drug Designation to HPPD (HPP8668), vTv's oral first-in-class Nrf2/Bach1 modulator, for sickle cell disease. Preclinical Townes mouse-model data (Augusta University): orally active and well tolerated, increased fetal hemoglobin comparable to or exceeding hydroxyurea, reduced oxidative stress and RBC sickling. CEO Paul Sekhri: designation strengthens HPPD partnering discussions; lead program cadisegliatin (T1D, Ph3 CATT1) remains the priority.

UK MHRA revoked the marketing authorization for IXCHIQ (single-shot chikungunya vaccine) for travelers, following a further benefit-risk assessment by the UK Commission on Human Medicines; based on review of previous safety data, no newly identified safety signal. Valneva disagrees but will not appeal, citing immaterial financial/commercial impact (not actively distributed in UK for months, no product on market, no recall costs). Licenses in EU, Canada, Brazil and other territories unaffected; EMA and Health Canada recently maintained authorizations with updated product information.

8-K: underwriting agreement (Oct 5) with Jefferies/TD Securities/Leerink/Stifel — closing leg of the priced $350M @ $85 follow-on (424B5 filed Oct 6).

First patient dosed in a Phase 2a clinical trial in Australia evaluating GlyphAgo (SPT-320, oral prodrug of agomelatine) in adults with generalized anxiety disorder (GAD) and sleep disturbance; six-week double-blind proof-of-pharmacology trial (ISI sleep outcomes, HAM-A, CGI-S); topline data expected early 2028. Seaport also plans a global randomized Phase 2/3 in GAD in 1H 2027 with topline by end of 2028.

8-K: 1-for-25 reverse stock split effective Oct 5, 2026 to regain Nasdaq $1.00 minimum-bid compliance.

First patient enrolled in investigator-initiated PRISM-TAMP trial (University of Vermont-sponsored, NCT07477418) evaluating neoadjuvant gemcitabine delivered via the Trans-Arterial Micro-Perfusion (TAMP) platform before surgery in borderline resectable pancreatic ductal adenocarcinoma; ~10 patients planned, endpoints tumor regression, intraoperative surgical margins, safety/tolerability. First RenovoRx-supported IIT to advance to enrollment; second patient progressing toward TAMP treatment.

Highlights from Oct 6 investor webinar: FDA-aligned pivotal Phase 2 trial to enroll 12 male patients at recalibrated dose (first 3 treated count toward efficacy population); success requires >=7 patients meeting both co-primary endpoints at 12 months (myocardial LAMP2 expression >= Grade 1, LV mass index reduction >= 10% from baseline) supporting potential accelerated approval; first 3 patients under modified protocol showed no thrombotic microangiopathy/capillary leak syndrome in initial observation period (preliminary); longer-term Phase 1 follow-up: all 6 patients with myocardial LAMP2 expression and >=10% LVMI reduction at ~12 months; at 3-7 years LVMI below baseline in 5/6 (>=10% reduction in 4/6) with sustained cardiac biomarker reductions and QoL improvements; pivotal trial dosing expected complete mid-2027, topline mid-2028.

8-K: agreed to sell its Rare Pediatric Disease Priority Review Voucher (awarded Aug 19, 2026 on GENGLYCOS approval) for $210M cash; 20% of gross payable to NIH.

424B3: proxy statement/prospectus supplement — Pulmatrix special meeting postponed to Oct 26, 2026.

FDA approved Tukysa (tucatinib) + trastuzumab + pertuzumab as front-line maintenance treatment for adults with unresectable locally advanced or metastatic HER2-positive breast cancer following induction therapy. Approval based on Phase 3 HER2CLIMB-05: 35.9% reduction in risk of progression or death; median PFS 24.9 months vs 16.3 months.

FDA approved Rhapsido (remibrutinib) as the first treatment for symptomatic dermographism in adults not adequately controlled with antihistamines, expanding use beyond chronic spontaneous urticaria. Phase 3 RemIND: 29.3% of patients achieved complete hive resolution at 12 weeks vs 14% on placebo; improvements seen as early as week 2.

First patient enrolled in Phase 1b/2 NX-5948-203 study of oral BTK degrader bexobrutideg (NX-5948, co-developed with Roche) in combination with venetoclax in patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL); initial cohort in relapsed/refractory CLL with potential expansion to first-line and other B-cell malignancies.

424B3: prospectus supplement no. 9 — 3,245,595 common shares issuable upon exercise (Medicus Pharma).

Patient enrollment milestones in NSCLC trials: enrollment completed in Part C of Phase 2 THIO-101 study of ateganosine (sequenced with checkpoint inhibitor); 65% of 2026 enrollment target reached in pivotal Phase 3 THIO-104 study.

424B3: resale prospectus — 751,879 shares by selling stockholder PoC Capital (clinical trial funding agreement); company receives no proceeds.

Health Canada approved TLANDO capsules for testosterone replacement therapy in adult males with hypogonadism; North America commercial rights held by licensing partner Verity Pharma; Canadian launch planned by end of 2026.

Four abstracts accepted for MSToronto2026 (Oct 21-23) disclosing new data: Phase 2 CALLIPER OLE in progressive MS - high proportion transitioned to OLE with low discontinuation; trends toward lower 24-week confirmed disability worsening maintained through extension week 48, confirmed disability improvement more frequent; post-hoc analysis: vidofludimus calcium significantly reduced EBV-specific T-cell receptor signatures (consistent with lower EBV reactivation) with reductions linked to improved SDMT cognitive performance, driven by PPMS patients; bidirectional confirmed-disability-change endpoint (worsening + improvement) statistically significant via CALLIPER data and GPC methodology; preclinical: Nurr1 required for neuronal survival effect in vitro, reduced plasma NfL in EAE models.

The Hague specialized Dutch patent court found Merck infringing Halozyme's MDASE (rHuPH20) patent EP 2,797,622 and enjoined MSD BV from manufacturing or selling Keytruda SC in Belgium, Denmark, France, Ireland, Italy, Sweden, Switzerland and the Netherlands; court rejected Merck's invalidity arguments. Separate from the December 2025 German preliminary injunction.

8-K: third loan to Pronghorn Resources — aggregate $3.5M secured convertible note ($2M + $964K + $536K).

Positive 3-year topline from long-term Phase 3 extension study GLK-202-03 of Epioxa in keratoconus: sustained 1.9 D Kmax improvement, 29%/20% of eyes gaining >=2/>=3 lines of BCVA at year 3; no SAEs or treatment-related AEs.

S-1: resale registration for private-placement selling-stockholder shares (Decoy Therapeutics); not a primary offering.

Board initiated formal strategic process (Tungsten Advisors retained as exclusive financial advisor) to maximize value of orphan thrombo-inflammatory portfolio; options include asset licensing or sale, partnerships, portfolio transactions, and business combinations. Follows FDA alignment on Phase 3 development pathway for CAD-1005 in heparin-induced thrombocytopenia.

STAT+ reports Caribou is shutting down (wind-down), a day after the company announced a strategic-alternatives review, discontinuation of both allogeneic CAR-T programs (vispa-cel for r/r B-NHL, CB-011 for r/r multiple myeloma), and workforce reductions; $113.8M cash/securities as of June 30, 2026.

FDA granted plozasiran entry into the STAR (Split Real Time Application Review) pilot program for the planned sHTG supplemental NDA, qualifying the supplement for 6-month Priority Review without redeeming a PRV; Arrowhead retains the PRV purchased Aug 2026. sNDA submission targeted by year-end 2026.

Final Phase 1 data for oral LRRK2 PROTAC degrader ARV-102 in 24 Parkinson's patients (20-80 mg x 28 days vs placebo): dose-dependent improvement in saccadic hypometria (objective eye-movement measure), decreases in endolysosomal/neuroinflammatory biomarkers, increases in synaptic-integrity biomarkers; CSF LRRK2 reductions associated with oculomotor and pathway improvements. Presented as late-breaker at the 2026 International Congress of Parkinson's Disease and Movement Disorders (Seoul).

FDA granted two Breakthrough Therapy Designations for telisotuzumab adizutecan (Temab-A; ABBV-400), a next-generation c-Met-directed ADC with a novel topoisomerase 1 inhibitor payload: (1) in combination with bevacizumab for refractory metastatic colorectal cancer previously treated with fluoropyrimidine, irinotecan, oxaliplatin, anti-VEGF mAb and, if indicated, anti-EGFR mAb; (2) as monotherapy for locally advanced/metastatic EGFR wild-type, c-Met protein-expressing, non-squamous NSCLC after platinum chemotherapy and anti-PD-(L)1 therapy. Designations based on first-in-human study M21-404 (NCT05029882); the first BTDs for Temab-A, bringing AbbVie's ADC portfolio to four BTDs.

8-K filed (items 1.01, 3.02: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Plans January 2027 data readout for hospitalized babesiosis trial (NCT06207370): after scheduled Oct 1, 2026 interim analysis (primary endpoint time to sustained clinical resolution), DSMB recommended completing originally planned N=33 enrollment, but company will conclude enrollment at 30 and unblind now (limited incremental power from 3 more patients; babesiosis seasonality could delay unblinding to fall 2027). No safety signals. Primary/secondary endpoint results expected January 2027. Intends to request pre-sNDA meeting with FDA in January 2027 to discuss an sNDA for tafenoquine in babesiosis, proposing NAT-based molecular clearance as surrogate endpoint for accelerated approval. Expanded-access study (NCT06478641) confirmed molecular cure in first 3 patients; final screening data from 2 more patients expected early November 2026. Announced October 6, 2026 (8:10am ET).

Shelf registration amended

Completed a Phase 1 study of SCY-770, a first-in-class selective oral direct AMPK activator for autosomal dominant polycystic kidney disease (ADPKD): well tolerated with a favorable safety profile in healthy adult participants at higher doses than previously evaluated; pharmacokinetics characterized to support Phase 2 dose selection. Phase 2 proof-of-concept study in ADPKD patients remains on track to initiate in Q4 2026. Announced October 6, 2026 (7:30am ET).

Repligen Completes Acquisition of BioLife Solutions (BLFS) - merger closed prior to market open Oct 6, 2026 following BioLife shareholder approval at the Oct 5 special meeting; BioLife holders receive $11.25 cash + 0.1442 Repligen shares per BioLife share (~$1.5B enterprise value, 64% stock / 36% cash); BLFS halted Oct 5 after-hours and suspended effective Oct 7, 2026.

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 2.03, 3.02: financing-related)

Registration statement filed (primary offering)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

Announced statistically significant final 3-year overall survival benefit in the Phase 2b trial in fully resected pulmonary metastatic osteosarcoma: 72.7% 3-year OS in OST-HER2-treated patients vs 45.8% in a comparable combined historical control (p=0.0006; 41 patients enrolled, 4 lost to follow-up). Preparing early market access submissions in the US, UK, Europe and Australia; WHO accepted brand name Herlystic.

First patients enrolled in the Phase 3 NEPTUNE pivotal trial of urcosimod for neuropathic corneal pain (NCP) - a global ~111-patient study of a single-dose regimen; first patients enrolled by Dr. Melissa Toyos (Toyos Clinic, Nashville). NCP has no FDA-approved treatment. Announced October 6, 2026 (7:00am ET).

Announced publication in the Journal of Hepatology of a pre-specified secondary analysis of the Phase 3 MAESTRO-NASH trial demonstrating consistent Rezdiffra efficacy across common genetic risk variants associated with MASH progression, including improvements in histology and MRI-PDFF (Oct 6, 2026, GlobeNewswire).

Reported positive interim efficacy data from the ongoing Phase 2 trial of ENV-105 (carotuximab, anti-CD105) in combination with apalutamide in metastatic castration-resistant prostate cancer (mCRPC): statistically significant PFS difference, median PFS 17.7 months (ENV-105 + apalutamide) vs 2 months (apalutamide alone), p=0.0008. Protocol modified to enroll earlier (PSA rise after upfront androgen signaling inhibitor; randomized to enzalutamide +/- ENV-105); Phase 2 completion targeted Q4 2027 or earlier (Oct 6, 2026, Business Wire).

Dosed the first patient in the Phase 2a RESET trial (NCT07592767) of JOTROL (investigational trans-resveratrol micellar formulation) in Parkinson's disease - a multi-center, randomized, double-blind, placebo-controlled study; coordinating PI Charbel Moussa (Georgetown). IND cleared November 2025 on preclinical neuroprotection data. Separately announced exploration of strategic options to maximize shareholder value. Announced October 6, 2026 (11:15am ET).

Registration statement amended

Reached FDA alignment on the final design of ENABLE-2, the pivotal Phase 3 trial of relcobatinib (ELVN-001) in previously treated chronic myeloid leukemia (CML). ENABLE-2 will randomize ~450 adults (1L+ treated with one or more TKIs) 1:1 to relcobatinib 80 mg QD vs investigator-selected second-generation TKI (dasatinib, nilotinib, or bosutinib); primary endpoint MMR at Week 24, key secondary MMR at Week 96. USAN adopted 'relcobatinib' as the nonproprietary name for ELVN-001. Trial initiation on track before year-end 2026. Announced October 6, 2026 (4:05pm ET).

Board approved a process to evaluate strategic alternatives (merger, acquisition, business combination or other transactions; Wedbush engaged as exclusive advisor); will discontinue further clinical development of vispa-cel (ANTLER Ph1, r/r B-NHL) and CB-011 (CaMMouflage Ph1, r/r multiple myeloma) and implement a substantial workforce reduction expected to be mostly complete in Q4 2026; $113.8M cash, cash equivalents and marketable securities as of June 30, 2026.

8-K filed (items 1.01: financing-related)

Offering prospectus supplement filed

FDA accepted for filing the NDA for oral infigratinib for children with achondroplasia with Priority Review; PDUFA target action date February 4, 2027. Based on Phase 3 PROPEL 3: met primary and key secondary endpoints - annualized height velocity treatment effect +2.10 cm/yr (p<0.0001), and the first statistically significant improvement in body proportionality and arm span in an achondroplasia Phase 3 trial. If approved, would be the first and only oral therapy for achondroplasia; BridgeBio plans U.S. launch upon approval. Announced October 6, 2026 (7:30am ET).

Negative Phase 3 topline from FURVENT (global pivotal Phase 3, NCT05607550): firmonertinib failed to meet the requirement for progression-free survival in previously untreated patients with advanced non-small cell lung cancer. Shares halted in premarket trading (Oct 6, 2026, company IR release).

Registration statement filed (primary offering)

Completed enrollment in the Phase 3 CATT1 study (NCT06334133) of cadisegliatin as adjunctive therapy to insulin in type 1 diabetes: 166 patients randomized (cadisegliatin 800 mg QD, 800 mg BID, or placebo) in the randomized, double-blind, placebo-controlled study evaluating incidence of level 2/3 hypoglycemic events over six months. Cadisegliatin holds FDA Breakthrough Therapy designation; topline results expected mid 2027 (Oct 5, 2026, GlobeNewswire).

8-K filed (items 1.01, 2.03: financing-related)

Announced commencement of underwritten public offering of common stock (size not disclosed); 30-day underwriter option for up to an additional 15% of shares offered; Jefferies, TD Cowen, Leerink Partners, Stifel joint bookrunners. Proceeds to advance Gastroenterology/Rheumatology/Dermatology programs (preclinical studies, clinical trials, manufacturing, Phase 3 readiness) and a new program advancing SPY072 into late-stage development for hidradenitis suppurativa; remainder for R&D, working capital and general corporate purposes (Oct 5, 2026, GlobeNewswire).

Announced pricing of underwritten public offering: 4,117,648 shares of common stock at $85.00/share for ~$350.0M gross proceeds (30-day underwriter option for up to ~$52.5M additional); expected close Oct 7, 2026. Proceeds to advance GI, rheumatology and dermatology programs incl. Phase 3 readiness and SPY072 in hidradenitis suppurativa.

Offering prospectus filed (possible follow-on or ATM takedown)

Completed successful meeting with FDA aligning on planned U.S. NDA for zorevunersen in Dravet syndrome: FDA accepted multicomponent assessment of four prespecified Vineland-3 subdomains (expressive communication, receptive communication, interpersonal relationships, personal skills) analyzed via MMRM for cognition/behavior changes in Phase 3 EMPEROR (NCT06872125). 5 years of Phase 1/2a + OLE safety/efficacy data discussed. EMPEROR readout anticipated Q3 2027; data to be submitted through 1H 2027 to complete NDA submission in 2H 2027 supporting potential U.S. approval/launch in early 2028. Announced October 5, 2026.

FDA recommended a pre-BLA meeting as precursor to a Biologics License Application for ersodetug in congenital hyperinsulinism after reviewing Phase 3 sunRIZE data, which failed its prespecified primary endpoint (behavioral confounders acknowledged); topline results from the Phase 3 upLIFT study in tumor hyperinsulinism expected this quarter; sunRIZE open-label extension continues with high retention (Oct 5, 2026, 7:00am ET GlobeNewswire).

Shelf registration amended

Dosed the first patient in the global Phase 1/2 first-in-human trial of PM577a, an investigational in vivo Prime Editor designed as a one-time curative therapy for H1069Q-mutated Wilson disease (NCT07748403; open-label, adults and adolescents; initial data expected 2027). FDA also granted Rare Pediatric Disease designation to PM577 for Wilson disease (Oct 5, 2026, 8:00am ET GlobeNewswire). First clinical study of an in vivo Prime Editing therapy.

Registration statement filed (primary offering)

Vaxcyte reported positive topline data from OPUS-1 (NCT07284654), the pivotal adult Phase 3 noninferiority trial of VAX-31, its 31-valent pneumococcal conjugate vaccine candidate for the prevention of invasive pneumococcal disease and pneumonia in adults (Oct 5, 2026, 6:00am ET GlobeNewswire release; data presented on the company's 8:00am ET webcast). VAX-31 met ALL prespecified co-primary immunogenicity endpoints: noninferiority (OPA GMR lower-bound 95% CI >0.667) on all 28 serotypes shared with PCV20 (Prevnar 20) and/or PCV21 (Capvaxive) -- 11/11 triple-shared, 9/9 VAX-31+PCV20-only, 8/8 VAX-31+PCV21-only; superiority (LBCI >2.0) on all 3 serotypes unique to VAX-31 plus cross-reactive serotype 20B; and immunobridging noninferiority in adults 18-49 vs 50-64 on all 32 serotype comparisons. Vs PCV21, serotypes 3 and 12F missed the >0.667 bar but met the prespecified >0.5 key-secondary threshold. Safety profile similar to PCV20/PCV21 with no vaccine-related serious adverse events and no discontinuations. Trial: randomized, double-blind, active-controlled; 4,047 pneumococcal-naive US adults (>=50, n=3,572 randomized 1:1:1 VAX-31/PCV20/PCV21; 18-49, n=475 randomized 3:1 VAX-31/PCV20). Next: OPUS-2 and OPUS-3 readouts H1 2027, BLA submission planned H1 2028.

Announced commencement of two separate underwritten public offerings: $500M of common stock + pre-funded warrants, and $500M aggregate principal amount of convertible senior notes due 2032 (mature Oct 15, 2032); 30-day underwriter options for up to $75M additional common stock and $75M additional notes; the two offerings are not contingent on each other. Proceeds to fund VAX-31 adult (OPUS-2/OPUS-3 enrolled) and infant Phase 2 programs, manufacturing scale-up and inventory, VAX-31 adult US commercial launch prep, other pipeline R&D and general corporate purposes (Oct 5, 2026, 4:28 PM ET, GlobeNewswire).

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01: financing-related)

First cohort dosed in the Phase 1/2 ANCHOR trial of KRRO-121, a GalNAc-conjugated RNA-editing oligonucleotide for hyperammonemia in urea cycle disorders (UCDs) and hepatic encephalopathy: a single 2 mg/kg dose was administered to the first cohort of healthy volunteers in Australia. Part 1 is a placebo-controlled SAD/MAD study in up to 50 healthy volunteers (SAD started September 2026); Part 2 will enroll up to 15 UCD patients of any mutational background, with patient dosing expected to start 1H 2027 and an interim readout expected 2H 2027 (Oct 5, 2026, GlobeNewswire).

8-K filed (items 1.01, 2.03, 3.03: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 2.03, 3.02: financing-related)

Positive Phase 3 topline from EPCORE DLBCL-2 (fixed-duration epcoritamab + R-CHOP vs R-CHOP alone in newly diagnosed DLBCL, IPI 2-5): statistically significant and clinically meaningful PFS improvement; risk of progression or death reduced 51% (HR 0.49 [95% CI 0.36, 0.67]; p<0.0001). Safety generally well tolerated, consistent with the individual agents. First Phase 3 bispecific-antibody combination to show a PFS benefit in frontline DLBCL; companies will engage global regulators (Oct 5, 2026, GlobeNewswire).

Received Human Research Ethics Committee (HREC) approval and Therapeutic Goods Administration (TGA) clearance under Australia's Clinical Trial Notification (CTN) scheme for the first-in-human Phase 1 trial of EQ504, a potent and selective AhR (aryl hydrocarbon receptor) modulator. Randomized, double-blind, placebo-controlled SAD/MAD study in healthy volunteers evaluating safety, tolerability, PK/PD for proof of mechanism; dosing expected to begin by early November 2026 with topline Phase 1 proof-of-mechanism data anticipated ~6 months after study initiation. Subject to supportive data, a placebo-controlled proof-of-concept study in moderately to severely active ulcerative colitis is planned for 2H 2027 (Oct 5, 2026, GlobeNewswire).

FDA cleared the IND for CR-002, a PD-L1-targeted topoisomerase-inhibitor antibody-drug conjugate for advanced solid tumors (Oct 5, 2026, 7:30am ET GlobeNewswire). Global Phase 1/2 APOLLO trial (in collaboration with Adcendo) planned to start dosing by end of 2026, initial clinical data expected H2 2027.

Presented 24-month HOPE-3 open-label extension data for deramiocel in Duchenne muscular dystrophy (late-breaking poster, World Muscle Society 2026, Hiroshima) focused on upper-limb function (PUL 2.0): delayed-start group LS mean change -2.05 (n=52) in placebo year vs -0.49 (n=49) on deramiocel (76% reduction in decline rate, diff 1.56, 95% CI 0.47-2.64); early-start group -0.95 (n=54) vs -0.89 (n=49), stable. Month 24 observed vs natural-history predicted: placebo arm -3.76 vs -5.35; deramiocel arm -3.42 vs -5.88. ITT 106 randomized, 98 entered OLE (49/arm). Data were part of the deramiocel BLA major amendment; PDUFA November 22, 2026. PPMD webinar Oct 7. Announced October 5, 2026.

Regulatory update following Type C meeting with FDA (3Q 2026) on the BB-301 pivotal study in oculopharyngeal muscular dystrophy (OPMD): FDA requested additional available data including the Benitec-sponsored OPMD Natural History Study, which Benitec will provide in the coming weeks; FDA agreed to review on a timeline allowing confirmation of the final pivotal study design in 1Q 2027. Pivotal enrollment still planned for mid-2027. Interim Phase 1b/2a results (12-month follow-up for all 6 Cohort 1 patients, interim follow-up for all 3 Cohort 2 patients) accepted as late-breaking poster at ESGCT Oct 27-30, 2026. Announced October 5, 2026.

Month 12 exploratory analyses from the Phase 3 FORTIFY trial of BBP-418 in LGMD2I/R9 (FKRP-related limb-girdle muscular dystrophy) showed disease-modifying potential: 54% of treated patients had stable or improved LVEF vs 25% on placebo; mean glycosylated alpha-dystroglycan rose above carrier levels; 100% of patients with elevated baseline HS troponin I returned to normal range by Month 12 (Oct 5, 2026, 7:30am ET GlobeNewswire). FDA granted Priority Review; PDUFA date November 27, 2026.

New analyses of the randomized double-blind placebo-controlled Phase 2 ATH434-201 study (N=61 mITT) in multiple system atrophy, presented at the 2026 International Congress of Parkinson's Disease and Movement Disorders: 50 mg ATH434 slowed functional decline by ~52% vs placebo on the 11-item UMSARS Part I (-4.64-point difference at Week 52, p=0.032 after adjusting for baseline CSF NfL); baseline CSF neurofilament light predicted decline rate, supporting its use as a Phase 3 covariate (Oct 5, 2026, 7:05am ET GlobeNewswire).

Positive Phase 1a safety, tolerability and target engagement data for ABI-6250 to be presented as two posters (one a Poster of Distinction) at AASLD The Liver Meeting 2026 (Nov 5-9): randomized blinded Phase 1a in healthy participants, well tolerated with no SAEs, dose-dependent reversible increases in total/conjugated bile acids consistent with NTCP target engagement; supports plans to initiate Phase 2 studies in chronic HDV infection and cholestatic liver diseases (Oct 5, 2026, GlobeNewswire).

First case of stable disease for 3 months in the ongoing Phase 1 dose-escalating trial (NCT05316129) of lira-cel in recurrent ovarian cancer: the patient in cohort 5 (highest dose to date, 1e7 CAR+ cells/kg after lymphodepletion) showed no progression at 90 days; no dose-limiting toxicities observed in any cohort to date; multiple patients surviving >1 year including one >2 years (Oct 5, 2026, PR Newswire).

FDA approved a supplement to the Biologics License Application (BLA) for AVT02 (adalimumab-ryvk), marketed in the U.S. as SIMLANDI, Alvotech's biosimilar to Humira (Oct 5, 2026 GlobeNewswire release; dateline Reykjavik). The approval enables AVT02 drug substance for U.S. commercial supply to be manufactured in a second production suite (DSM2) at the company's Reykjavik, Iceland facility, in addition to the first suite -- effectively doubling the drug substance manufacturing capacity available to support U.S. supply. The release states the approval 'further confirms resolution of FDA inspection-related matters at the Reykjavik site.'

Alector licensed its Parkinson's drug candidate AL050 (GBA1 / glucocerebrosidase-targeted) to Roche under a license agreement: $100 million upfront to Alector plus up to $1.17 billion in milestone payments; ALEC shares up ~20% on the news (Oct 5, 2026, Endpoints News).

8-K filed (items 1.01: financing-related)

Positive Phase 3 topline from EPCORE DLBCL-2 (fixed-duration epcoritamab + R-CHOP vs R-CHOP alone in newly diagnosed DLBCL, IPI 2-5): statistically significant and clinically meaningful PFS improvement; risk of progression or death reduced 51% (HR 0.49 [95% CI 0.36, 0.67]; p<0.0001). Safety generally well tolerated, consistent with the individual agents. First Phase 3 bispecific-antibody combination to show a PFS benefit in frontline DLBCL; companies will engage global regulators (Oct 5, 2026, GlobeNewswire).

Announced a webcast and conference call on Monday, October 5, 2026 at 8:00 a.m. ET (dial 800-445-7795 domestic / 785-424-1699 international, ID PCVX1005; live webcast on the vaxcyte.com IR page, archived at least 30 days) to present topline safety, tolerability and immunogenicity data from OPUS-1, the pivotal adult Phase 3 trial of VAX-31, the company's 31-valent pneumococcal conjugate vaccine (PCV) candidate for prevention of invasive pneumococcal disease and pneumococcal pneumonia. This release announces the webcast only; the data itself lands Oct 5 pre-market.

Phase 1/2 RAINFOL-01 Part C readout for rinatabart sesutecan (Rina-S, FRa-targeted TOPO1 ADC) in platinum-resistant ovarian cancer (n=109): confirmed ORR 45.9% incl. 5 complete responses, median duration of response 12.1 months, median PFS 9.5 months; activity regardless of FRa expression incl. low/non-expressors and regardless of prior mirvetuximab; presented as late-breaking oral at IGCS Congress 2026. Four Phase 3 RAINFOL trials ongoing (RAINFOL-02 in PROC NCT06619236; -03 endometrial; -04 PSOC maintenance; -07 2L PSOC).

Announced 3-year follow-up results from the ovarian cancer cohort of the Phase 1b C-800-01 trial (NCT03860272) for botensilimab (BOT, Fc-enhanced anti-CTLA-4) + balstilimab (BAL, anti-PD-1) in recurrent ovarian cancer, presented at IGCS 2026: estimated 3-year overall survival 48% (n=35 evaluable), unchanged from the 2-year estimate, in a heavily pretreated population (median 4 prior lines; ~3/4 platinum-resistant/refractory); 11 of 44 treated patients alive and off all therapy at last follow-up.

Announced new molecular profiling and external control arm analyses of Phase 2 RAMP 201 (NCT04625270) for avutometinib + defactinib (AVMAPKI + FAKZYNJA) in recurrent low-grade serous ovarian cancer (LGSOC), presented at IGCS 2026: triple wild-type (KRAS/NRAS/BRAF) population showed confirmed ORR 18% (7/39) with 13-month mPFS; KRAS wild-type with NRAS/BRAF mutations ORR 22% (2/9). External control vs GOG 281 conventional care: KRAS-mutated weighted ORR 38.7% vs 0% (mPFS 22.1 vs 6.5 mo); KRAS wild-type weighted ORR 22.7% vs 7.9% (mPFS 11.3 vs 7.7 mo). Reinforces approved KRAS-mutated use and supports expansion into KRAS wild-type LGSOC beyond the May 2025 accelerated approval.

Offering prospectus supplement filed

New ecopipam data ahead of MDS Congress (Seoul, Oct 4-8): post-hoc analysis showed ~70% of pediatric Tourette patients had clinically meaningful tic reduction within 8 weeks; interim 18-month analysis of ongoing open-label extension showed mean 45.6% reduction in tic severity scores with no new safety signals; subgroup analyses found common concurrent psychiatric conditions did not negatively impact efficacy or safety. Ecopipam NDA under FDA Priority Review (accepted Aug 19).

FDA cleared IND application for SZN-8141 (bifunctional Wnt-pathway-activating/anti-VEGF antibody) for diabetic macular edema; DUET Phase 1b/2a trial to start by year-end 2026 with initial data in H2 2027. IND clearance also unlocks the second tranche of the $175M PIPE financing.

Announced clinical trial collaboration agreement with AstraZeneca and Daiichi Sankyo to evaluate ivonescimab (PD-1/VEGF bispecific) in combination with Datroway (datopotamab deruxtecan, TROP2-directed ADC) across multiple solid tumor types including lung and breast cancer, starting with a Phase III trial in first-line triple-negative breast cancer. Each company contributes its therapy and shares trial costs; each retains development and commercial rights to its own medicine. Builds on AZ's $2B strategic equity investment in Summit.

8-K filed (items 1.01, 3.02: financing-related)

Registration statement filed (primary offering)

EMA validated the Marketing Authorisation Application (MAA) for rebisufligene etisparvovec (UX111), single-dose intravenous AAV9 gene therapy for MPS IIIA (Sanfilippo syndrome Type A); validation confirms the application is complete enough to begin formal scientific review (not an approval). Therapy holds PRIME and Orphan Drug designations from EMA; FDA approved it on September 17, 2026. Company also engaging UK MHRA and Saudi FDA on geographic expansion.

Announced closing of public offering of up to $282.6M.

Presented detailed Phase 3 results in a late-breaking oral at EADV Congress 2026 (Vienna) for LITFULO (ritlecitinib) in nonsegmental vitiligo: TRANQUILLO 2 (100 mg QD, n=1,567 adults) and TRANQUILLO (50 mg QD, n=607 ages 12+) met co-primary endpoints at Week 52 - F-VASI75 21.86%/12.47% vs 2.40%/2.48% placebo; T-VASI50 13.02%/8.98% vs 2.40%/1.98% placebo; repigmentation improving from Week 24 through Week 52 with greater disease stabilization; safety consistent with the alopecia areata profile, no new signals. Plans global regulatory filings (FDA + EMA). Largest Phase 3 program to date for an oral systemic therapy in NSV.

Licensing and option agreement with China's Abogen Biosciences for ABO2203, an mRNA-encoded CD19xCD3 T-cell engager designed to deplete B cells in vivo for autoimmune disease: $575M upfront, up to $7.2B in development/regulatory/commercial milestones (~$7.8B total) plus royalties. Exclusive worldwide license to ABO2203 plus exclusive options on additional RNA-platform programs. ABO2203 is the first mRNA-encoded TCE to enter clinical evaluation for autoimmune disease (Phase 1 in autoimmune disease at Ruijin Hospital since 2024; early ITP data showed B-cell depletion without cytokine release syndrome). Deal follows Novartis' September pause of an autoimmune CAR-T program over three deaths.

FDA extended the BLA review of denecimig (hemophilia A, with or without inhibitors) past the expected Q3 2026 decision due to ongoing manufacturing facility remediation after a pre-license inspection; no new timeline communicated. No deficiencies identified in clinical efficacy/safety data from the FRONTIER trial program; denecimig received a positive CHMP opinion on Sept 17, 2026. Novo still targets US launch in 1H 2027 pending approval; 2026 financial outlook unchanged.

Offering prospectus supplement filed

FDA approved an expanded indication for Jaypirca (pirtobrutinib), the first-and-only non-covalent BTK inhibitor, for previously untreated CLL/SLL with no known 17p deletion (first-line use). Based on Phase 3 BRUIN CLL-313 (n=282, pirtobrutinib vs bendamustine+rituximab): IRC-assessed PFS HR=0.20 (95% CI 0.11-0.37, p<0.0001), median PFS not reached vs 33.5 months for BR; ORR 94% vs 81%. Safety consistent with established profile (dose reductions 3.6%, permanent discontinuations 4.3%). Data presented at ASH Dec 2025 and published in JCO.

Shelf registration filed (enables future raises incl. ATM)

Phase 3 ICONIC-TOTAL Week 112 (2-year) late-breaking data at EADV Congress 2026 for icotrokinra (ICOTYDE, first targeted oral IL-23R peptide) in high-impact plaque psoriasis (scalp/genital/hands-feet): IGA 0/1 rose from 57% at Week 16 to 67% at Week 24 to 70% at Week 112; site-specific complete clearance 60% scalp, 89% genital, 63% hands/feet; mNAPSI mean improvement 71% at Week 112; safety consistent with established profile, no new signals. ICOTYDE is already approved in the US, Europe, Japan and China.

8-K filed (items 1.02: financing-related)

Registration statement filed (primary offering)

Offering prospectus supplement filed

Registration statement filed (primary offering)

Announced five-year data from the Phase 3 PROPEL open-label extension of POMBILITI (cipaglucosidase alfa) + OPFOLDA (miglustat) in late-onset Pompe disease (n=82 treated continuously from PROPEL baseline), presented as late-breaker at WMS 2026 Hiroshima: durable motor function (mean %predicted 6MWD +0.7% in ERT-experienced, +10.5% in ERT-naive) and pulmonary stabilization; no new safety signals (51.2% treatment-related AEs, 5 discontinued for safety). Also shared Phase 1/2 update for nivudirsen (BMN 351) in DMD (near full-length dystrophin expression, favorable tolerability).

Offering prospectus supplement filed

Reported 52-week Phase 2 data from Part A of the ongoing Phase 2/3 AMETHYST study of litifilimab (BIIB059, anti-BDCA2) in cutaneous lupus erythematosus (n=93 adults with active subacute/chronic CLE refractory or intolerant to antimalarials): clear/almost clear skin (CLA-IGA-R erythema 0-1) at Week 52 in 27.2% on litifilimab from study start (vs 19.0% at Week 24); CLASI-70 at Week 52 28.8% (vs 21.7% at Week 24); participants who switched from placebo to litifilimab at Week 24 showed onset of improvement as early as 4 weeks, with ~1 in 3 (33.7%) achieving clear/almost clear skin by Week 52. Generally well tolerated with no new safety signals (serious AEs 3.4% in extended treatment period). Phase 3 part remains blinded with results expected H1 2027. Data presented at EADV 2026.

FDA alignment on registrational pathway for WVE-006 (GalNAc-RNA editing oligonucleotide) in alpha-1 antitrypsin deficiency (AATD): single 2-year registrational trial to support full approval, with 1-year interim analysis for potential accelerated approval based on AATD biomarkers; FDA supports Wave's LC-MS assay to measure M-AAT (wild-type) and Z-AAT (mutant) protein. WVE-006 is a first-in-class RNA editing candidate aimed at correcting the root cause of AATD (no approved therapy addresses both lung and liver manifestations).

Announced plans to evaluate SL-325 (DR3-blocking antibody) in RECEPTIVE-HS1, a randomized, double-blind, placebo-controlled Phase 2 trial (~150 patients, 1:1:1 low/high dose/placebo, primary HiSCR50 at Week 16) in moderate-to-severe hidradenitis suppurativa (second Phase 2 indication after Crohn's disease); IND submission expected Oct 2026, Week-16 data expected 1H28; appointed Shoba Ravichandran, M.D. (ex-Novartis, led Cosentyx HS approval) as SVP Clinical Development; cash runway into 2029 unchanged.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01: financing-related)

Announced TUDRIQEV (vusolimogene oderparepvec-wtpg; formerly RP1) is now commercially available in the US in combination with nivolumab for unresectable advanced cutaneous melanoma progressing on PD-1 blockade (ReplimuneConnect Plus support program live); first commercial product following early-August FDA accelerated approval (IGNYTE ORR 24.2%, median DoR 14.1 mo); confirmatory Phase 3 IGNYTE-3 (NCT06264180) ongoing.

Announced first results for investigational ubamatamab (MUC16xCD3 bispecific) in low-grade serous ovarian cancer (LGSOC) in a late-breaking plenary at IGCS 2026: from an ongoing Phase 1/2 trial, the 11 patients treated with 800 mg monotherapy (recurrent, heavily pre-treated, median 4 prior lines) showed 73% ORR (all partial responses), 9-month median duration of response, and 11-month median PFS. CRS in 90% (all grade 1/2); no TEAEs resulted in death. Enrolling a single-arm potentially registrational LGSOC cohort (~100 patients) and planning a confirmatory randomized Phase 3; FDA discussion planned in coming months.

Reported Phase 2 COURAGE data for anti-GDF8 (myostatin) antibody trevogrumab in obesity (BMI>=30): vs semaglutide alone (placebo+semaglutide), lean mass by DXA was preserved dose-dependently (trevogrumab 75mg: -3.3% vs -6.7% at 26 wks, 50.7% relative preservation; -4.2% vs -7.3% at 52 wks, 42.5% preservation) with no meaningful enhancement of total weight loss; MRI substudy showed 69-72% muscle preservation at 52 wks; generally well tolerated (77% AE rate vs 82% placebo). Presented at EASD 2026, in press with The Lancet. Planning a Phase 2 trial in older adults with obesity plus decreased muscle mass/strength.

Sanofi to pay Regeneron $1B upfront plus up to $7B in milestones (total up to $8B) to expand the immunology alliance with 4 next-generation long-acting antibodies: lead is IL-13 mAb REGN20423 (currently in Phase 1 in atopic dermatitis), plus 3 preclinical assets (IL-4xIL-13 bispecific, anti-IL-4, anti-IL-4R-alpha) expected to enter clinic in 2027; Regeneron also has an option to add Sanofi's lunsekimig (TSLPxIL-13 bispecific, Phase 3 in COPD) after data read. Regeneron leads R&D, Sanofi leads global commercialization, 50/50 global cost and profit split; agreement settles prior Dupixent collaboration litigation. Aimed at a Dupixent successor ahead of the 2031 US patent expiry.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Detailed Phase 2 results for tilrekimig (PF-07275315, trispecific IL-4/IL-13/TSLP antibody) in moderate-to-severe atopic dermatitis presented at EADV: EASI-75 at week 16 of 62.5% vs 19.9% placebo (Stage 1, 450mg Q2W, p=0.0008) and 58.5%/61.0%/47.8% vs 9.1% placebo across Q4W doses (Stage 2); no dose-dependent safety signals; Phase 3 AD (incl. dupilumab comparator) and asthma trials initiated, Ph2b/3 in COPD ongoing

Surpassed full enrollment in the BACKBEAT Global Pivotal Trial (NCT06059638) of AVIM Therapy with strategic collaborator Medtronic in pacemaker-indicated patients with uncontrolled hypertension: 330 patients randomized as of Sept 30, 2026 at 81 sites in 13 countries, exceeding the 316-patient target (284 evaluable needed for primary 24-hr ambulatory SBP analysis; >90% power to detect >=5 mmHg difference); primary endpoint data targeted for late-breaking presentation at a major CV conference in Q2 2027 to support future marketing applications.

Long-term durability data for rezpegaldesleukin presented at EADV Congress 2026: REZOLVE-AA Phase 2b (severe-to-very-severe alopecia areata, 52-week extension cohort) — SALT Score <=20 maintained in 75% (6/8) at 4 months off treatment and 63% (5/8) at 6 months off treatment; SALT Score <=10 deepened from 7% at end of 52 weeks to 19% after 6 months off treatment; data support Q4W/Q12W maintenance dosing. REZOLVE-AD Phase 2b maintenance (moderate-to-severe atopic dermatitis): durable EASI-75/EASI-90/vIGA-AD 0/1/Itch NRS at week 52 with monthly and quarterly dosing; EASI-100 increased 2-5x (Q4W 4%->22%, Q12W 9%->18% among re-randomized). Safety consistent with prior results. Third ZENITH AD Phase 3 initiated (treatment-experienced AD); ZENITH AA Phase 3 (850 pts, SALT <=20 at Week 52 primary) to initiate early 2027. Investor call 8:00 AM ET Oct 1.

Offering prospectus supplement filed

Announced positive data from GFORCE-1, a Phase 1 study of MRT-8102 (NEK7-directed molecular glue degrader for NLRP3/IL-1-driven inflammatory diseases) in obese subjects at elevated CVD risk: robust and sustained NEK7 degradation across 5/20/40 mg QD doses with similar biomarker reductions at all doses; HMGB1, calprotectin, S100A12, SAA and IL-1beta normalized to healthy-volunteer levels; safe and well tolerated over 4 weeks dosing + 4 weeks follow-up with no SAEs and AE rates comparable to placebo. Phase 2 trials planned across coronary artery disease (GFORCE-2), gout (GEMINI-1), and moderate-to-severe hidradenitis suppurativa (GALAXY-1). Company held conference call Oct 1, 8:00 a.m. ET.

Presented positive Phase 1b open-label extension data for rexaceract (GT-02287, oral brain-penetrant GCase allosteric modulator) in Parkinson's (MDS Congress 2026 poster): 12 evaluable participants completed 12 months dosing with MDS-UPDRS motor scores stable to baseline (no clinically meaningful progression; ~6 points/yr expected in early PD), both high- and low-CSF-GluSph groups; GluSph dropped toward healthy levels in high-baseline group; no new safety signals, lower TEAE rate in extension; new Phase 2 protocol to FDA in coming weeks, Phase 2 initiation guided 1Q27.

Discontinued FHD-909 (SMARCA2/4 synthetic lethality, Lilly-partnered) after Phase 1 dose-escalation data showed insufficient efficacy in SMARCA4-mutant lung/solid tumors; Lilly collaboration (2021, up to $1.6B) ends with no further activities, selective SMARCA2 degrader also dropped; ~40% workforce reduction (board approved Sept 30), cash runway extended into 2H 2029; shares -45% premarket.

Evommune Presents Late-Breaking Phase 2a Proof-of-Concept Data for EVO301 in Atopic Dermatitis at EADV 2026 Congress - EVO301 met primary endpoint: 55% LS mean EASI reduction at Week 12 vs 22% placebo (p<0.01), responses deepening 8 weeks after two IV doses; global Phase 2b (subcutaneous) on track mid-2027. EVO301 now lead AD program after EVO756 Phase 2b miss.

Initiated NDA submission under FDA Real-Time Oncology Review for zipalertinib plus chemotherapy in first-line EGFR exon 20 insertion-mutant NSCLC, based on Phase 3 REZILIENT3 (met primary endpoint; longest median PFS seen in this setting).

Announced publication in the Journal for ImmunoTherapy of Cancer of final Phase 2a results (NCT04495153) for aglatimagene besadenovec plus valacyclovir in unresectable stage III/IV NSCLC with inadequate response to ICI: median overall survival 24.5 months in the evaluable population (n=46), 21.5 months in the progressive-disease-on-ICI cohort (n=41), vs 9.8-11.8 month historical docetaxel benchmark; 37% of progressive-disease patients alive beyond 24 months. Non-squamous histology predicted greater benefit (mOS 25.4 months in non-squamous progressive cohort). Generally well tolerated (13.7% grade 3 TRAEs, no dose-limiting toxicities). Supports the ongoing pivotal Phase 3 AURORA trial, enrolling globally at ~150 sites.

Updated AlloNK (AB-101) + rituximab Phase 2a data in Sjogren's disease presented at ISSjD 2026 (July 2, 2026 cutoff): first 11 SjD patients with >=6 months follow-up, mean ClinESSDAI improved 6.9 points from 14.6 and mean ESSPRI improved 3.5 points from 8.0 at 6 months (both past minimal-clinically-important-improvement thresholds); 82%/55%/73% improved >=1 point in dryness/fatigue/pain; mean stimulated salivary flow rose 0.4 to 1.1 mL/min (above >1.0 normal threshold) in the 8 hyposalivation patients. Safety across expanded 73-patient basket (SjD, RA, SSc, myositis): no CRS, ICANS, GvHD, or discontinuations due to AEs; 11% SAEs; most common TEAEs nausea 40%, headache 27%, lymphopenia 25%; supports outpatient administration.

Initiated the second Phase 1b trial (BActinium-2) of AKY-2519 ([225Ac]Ac-AKY-2519, miniprotein radioconjugate targeting B7-H3) in NSCLC and other B7-H3-expressing solid tumors (3 dose levels, multicenter open-label); BActinium-1 (mCRPC) enrollment ongoing with preliminary data expected 2027; ASCO 2026 first-in-human imaging/dosimetry showed robust tumor uptake with limited normal tissue exposure.

Positive topline from 48-week open-label extension (Part B) of Phase 2a STENOVA trial of ontunisertib 200 mg BID in fibrostenosing Crohn's disease: favorable safety through 60 weeks, 3% annualized FSCD-related event rate (one EBD, no surgeries), stricture stabilization trend. Supports initiation of global NOV-ERA Phase 2b in FSCD in coming months.

Underwriters fully exercised the IPO over-allotment option for 3,937,500 shares at $17.00/sh; issuance closed Oct 1, 2026; total IPO: 30,187,500 shares, ~$513.2M aggregate gross proceeds.

Late-breaking Phase 3 Viti-Up-1/Viti-Up-2 data at EADV 2026: RINVOQ (upadacitinib) 15 mg showed continued repigmentation through 76 weeks in non-segmental vitiligo.

Detailed results from VIRAGE Phase 2b trial of VCN-01 (zabilugene almadenorepvec, oncolytic adenovirus) + gemcitabine/nab-paclitaxel in metastatic pancreatic cancer published in Nature Medicine (n=112 randomized): improved OS, PFS and duration of response vs chemo alone, greater with 2 doses 3 months apart; VIRAGE2 Phase 2a testing more frequent dosing initiated (first patient treated Aug 2026)

FDA granted Fast Track designation for the BiPASS program evaluating 68Ga-PSMA-PET imaging (Illuccix / Gozellix) alongside MRI for prostate cancer detection prior to biopsy (announced Sept 30, 2026, PRNewswire). Telix has completed enrollment in the Phase 3 BiPASS study (NCT07052214) and has aligned with FDA on an NDA pathway for the pre-biopsy indication; submission planned. Fast Track enables more frequent FDA engagement and may expedite review of the planned NDA.

Positive late-breaking Phase 3 head-to-head results for zasocitinib (TAK-279, oral TYK2 inhibitor) vs deucravacitinib (BMS Sotyktu) in moderate-to-severe plaque psoriasis, presented at EADV 2026: statistical superiority across the primary and all key secondary efficacy endpoints; complete skin clearance (PASI 100) at week 16 in 36.5% (n=301) vs 13.9% (n=303, p<0.001), PASI 90 62.5% vs 34.0%, sPGA 0 43.2% vs 18.5%; higher rates of complete clearance from week 8; no new safety signals. Long-term data from pivotal LATITUDE PsO 3001/3002 showed durable clearance through week 52 (up to 93% of week-24 responders maintaining). Zasocitinib under review by FDA and EMA for moderate-to-severe plaque psoriasis.

New ELEVIDYS (delandistrogene moxeparvovec) data at WMS 2026: durable functional benefit over 2 years vs well-matched external control in ambulatory Duchenne patients treated at ages 8-12 (where decline is most pronounced); robust dystrophin expression and sarcolemmal localization 12 weeks post-infusion in patients treated at ages 2-3; safety profile consistent, no new signals

Positive full 52-week results from license partner Ascletis' Phase 3 open-label extension of denifanstat in moderate-to-severe acne in China (ASC40-304, 240 rollover patients, up to 52 weeks exposure), presented at EADV 2026: IGA treatment success 57.8%/55.6% (denifanstat/placebo-crossover), total lesion count -73.0%/-70.7%, inflammatory lesions -78.1%/-75.8%; no permanent AE-related discontinuations, no drug-related SAEs. U.S. AURORA Phase 3 patient screening starts October 2026.

Offering prospectus supplement filed

Topline results from pivotal Phase 3 TransportNPC study (CTD-TCNPC-301) of Trappsol Cyclo (IV hydroxypropyl-beta-cyclodextrin) in 94 patients with Niemann-Pick Disease Type C: primary endpoint (change in 4-domain NPC Clinical Severity Scale at Week 96) showed a directionally favorable effect (64% slowing, LS mean difference -0.81 points, 95% CI -2.04 to 0.41) but did not reach statistical significance (p=0.19). Prespecified analysis in patients on background miglustat and/or leucine (~83%, n=78) was statistically significant (71% slowing, difference -1.11 points, 95% CI -2.19 to -0.02; p=0.046). Well tolerated; no new safety signals. Company remains on track for NDA submission in Q4 2026 following pre-NDA meeting. Separate overall survival analysis vs INPDR external controls suggested 85% mortality risk reduction (HR 0.154, p=0.044).

Offering prospectus supplement filed

Offering prospectus supplement filed

8-K filed (items 1.01, 3.02: financing-related)

Completed enrollment in BASECAMP Phase 2 trial of forazapadin in Duchenne muscular dystrophy (ambulatory boys 7-10; 1:1:1 to forazapadin 60mg/120mg/placebo, exceeded enrollment target in <9 months across 18 sites); topline data timing revised to Q1 2027; company cites potential accelerated approval pathway discussions with FDA

Tulisokibart (anti-TL1A mAb) met primary endpoint of HiSCR50 at week 16 in Phase 2b MK-7240-012 trial in moderate-to-severe hidradenitis suppurativa (72% high dose, 64% medium dose vs 35% placebo); key secondary endpoints met; first positive Phase 2 anti-TL1A data in dermatology; presented at EADV 2026

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 3.02: financing-related)

U.S. District Court for the District of Delaware ruled Liquidia's Yutrepia (inhaled treprostinil dry powder) infringes claims 1 and 14 of United Therapeutics' U.S. Patent No. 11,826,327 covering inhaled treprostinil for PH-ILD; court found the 4 other asserted claims not infringed. Both sides to submit proposed remedies within one week; UTHR seeking injunctive relief. UTHR +13% (~$540.54), LQDA -50% (~$3.18B market value lost). Liquidia plans appeal and will ask FDA to remove the PH-ILD indication from Yutrepia's label.

Phase 2 eloraTZP results for eloralintide (amylin receptor agonist) + tirzepatide combo in obesity/type 2 diabetes, presented at EASD 2026 Milan (48 wks, n=367): highest combo dose lost 23.3% body weight vs 14.8% on tirzepatide alone and 11.1% on eloralintide alone; A1C down up to 2.9% vs 2.4% on tirzepatide alone. Discontinuation 10.8-27% in combo arms (vs 2.9% tirzepatide, 16.7% placebo), mostly GI during dose escalation. Lilly plans to start Phase 3 in Q4 2026.

Detailed Phase 3 ACHIEVE-4 results for Foundayo (orforglipron, oral GLP-1) vs titrated insulin glargine in adults with type 2 diabetes + obesity/overweight at increased CV risk (n~1,000+, largest and longest orforglipron study): MACE-4 met prespecified non-inferiority (HR 0.84, 95% CI 0.59-1.20); CV death 53% lower (HR 0.47, CI 0.25-0.90), all-cause death 57% lower (HR 0.43, CI 0.25-0.75); waist -7.0 cm, SBP -4.4 mmHg, hsCRP -38.9% at week 52; sustained A1c reduction and weight loss; presented at EASD 2026 and published simultaneously in The Lancet

Completed enrollment in KaiNETIC global Phase 3 program of ribupatide injection (KAI-9531, once-weekly dual GLP-1/GIP receptor agonist) for obesity/overweight: three double-blind, randomized, placebo-controlled trials (KaiNETIC-1/2/3) at >200 sites across 11 countries, doses up to 10 mg weekly over 76 weeks; topline data now expected mid-2028

8-K filed (items 1.01: financing-related)

Phase 1b/2a RESOLVE trial 52-week durability update for EP-104GI in eosinophilic esophagitis (EoE): 9 of 14 patients (64%) in clinical remission 52 weeks after a single esophageal-wall injection; Cohort 9 mean SDI reduction 3.0 at 36 and 52 weeks; meaningful histologic improvement (EoEHSS Stage -0.26, Grade -0.27 at 36 weeks in Cohort 8b); no treatment-emergent SAEs; Phase 2b interim data expected December 2026.

LPA1 class read-through: BMS amended the admilparant Phase 3 ALOFT LTE for liver-injury monitoring after a 'limited number' of liver events including a participant death from liver injury (causality unclear; unblinded monitors kept the study going); CTNM shares fell sharply in late-day trading Tuesday before rebounding early Wednesday; RBC/Leerink call the pullback overdone, Stifel notes CTNM's differentiated design + low 10 mg/day dose; ALOFT readout expected by end of 2026

Positive preliminary topline Phase 2 data from Seabreeze STAT COPD (CBP-201-207, n=159): single subcutaneous dose of rademikibart as add-on to standard of care in acute COPD exacerbations with type 2 inflammation cut treatment failure 81% vs placebo through Week 4 (p=0.0122), new moderate-to-severe exacerbations 85% (p=0.030), new ED visits/hospitalizations 100% (p=0.0137); 70 mL FEV1 improvement (p=0.1607, n.s.); lower AE incidence than placebo. Company to engage FDA on a Phase 3 registrational program.

Presented 24-month HOPE-3 open-label extension data for deramiocel in Duchenne muscular dystrophy at World Muscle Society 2026 (Hiroshima): 82 of 106 randomized patients reached 24 months (40 on deramiocel from start, 42 placebo-to-crossover). Delayed-start group PUL 2.0 decline slowed from 2.05 points in the placebo year to 0.49 points after crossover (76% reduction); early-treated group declined 0.95 points in year 1 and 0.89 in year 2; both groups beat an external natural-history model. Data were part of the recent major amendment to the deramiocel BLA; PDUFA target action date November 22, 2026. Shares +10.5% on the data.

Offering prospectus filed (possible follow-on or ATM takedown)

Integrated clinical and biomarker data for aglatimagene besadenovec in intermediate-risk localized prostate cancer (GlobeNewswire, Sep 30 2026): Ph3 PrTK03 (635 pts, median 58.0 mo follow-up) showed 41% reduction in risk of prostate cancer recurrence or prostate cancer-specific death vs placebo + EBRT (HR 0.59; 95% CI 0.41-0.84; p=0.0034); exploratory: 90% improvement in time to metastasis (HR 0.10), 52% in time to biochemical failure, 49% in time to salvage therapy; negative biopsy rate 76.6% vs 60.7% control (p=0.0026). Ph2 PrTK05 early data: systemic expansion/activation of circulating cytotoxic CD8 T-cell populations after local treatment. PrTK05 biodistribution/shedding data on track for inclusion in planned Q4 2026 BLA submission for localized prostate cancer.

8-K filed (items 1.01, 3.02: financing-related)

BMS amended the Phase 3 ALOFT long-term extension protocol for enhanced liver-injury monitoring (liver injury now an 'important potential risk', EU amendment May) after a 'limited number' of liver-related safety events including a participant death from liver injury / multi-organ failure; spokesperson confirmed Sep 30; causality unclear, unblinded monitors recommended the study continue; ALOFT readout expected by end of 2026 (admilparant, LPA1R inhibitor in IPF/PPF)

FDA approved expanded indication for CAMZYOS (mavacamten) for symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults and pediatric patients weighing 30 kg (66 lbs) or more - the only FDA-approved oHCM therapy for a pediatric population. Approval based on Phase 3 SCOUT-HCM (NCT06253221) in 44 adolescents aged 12 to <18: primary endpoint Valsalva LVOT gradient change at week 28 was -48.5 mmHg on mavacamten vs -0.5 mmHg on placebo (difference -48.0 mmHg, p<.001); resting LVOT gradient difference -47.0 mmHg.

FDA approved expanded indication for CAMZYOS (mavacamten) for symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms in adults and pediatric patients weighing 30 kg (66 lbs) or more — the only FDA-approved oHCM therapy in a pediatric population. Approval based on positive Phase 3 SCOUT-HCM results (44 adolescents aged 12 to <18 years; Valsalva LVOT gradient -48.0 mmHg vs placebo at week 28, P<.001). BMS announced September 30, 2026.

8-K filed (items 1.01: financing-related)

Detailed Phase 2 data for daxdilimab (ILT7-targeting mAb) in discoid lupus (n=72) presented at EADV 2026: low dose produced 6-point placebo-adjusted reduction in CLASI-A severity score; both doses met primary and key secondary endpoints (61.7%/62.1% achieved >=50% CLASI-A reduction vs 23.7% placebo)

Received official minutes from its FDA Biosimilar Biological Product Development (BPD) Type 2b meeting on ABP-450, its proposed biosimilar to BOTOX (onabotulinumtoxinA) for therapeutic use: FDA agreed in principle to AEON's two-stage comparative pharmacodynamic study using CMAP measurements and said previously completed Daewoong head-to-head studies vs BOTOX may be submitted as supportive evidence with scientific bridging; AEON plans an IND for the CMAP PD study in Q4 2026 and study start in Q1 2027, and does not plan a standalone comparative efficacy study at this time. Because the minutes do not preclude a 351(k) BLA path, the two-year milestone warrants (July 2026) accelerate and will expire Nov 16, 2026 unless exercised earlier.

Positive Phase 2 APEX Part B results for zumilokibart (half-life-extended anti-IL-13 antibody, acquired via Apogee) in moderate-to-severe atopic dermatitis, late breaker at EADV 2026: all three dose regimens met the primary endpoint (EASI-75 at Week 16 vs placebo; n=346 randomized 1:1:1:1 over 16-week induction); mid- and high-dose regimens also beat placebo across key secondaries (EASI-90/100, I-NRS4); mid-dose selected for Phase 3 and evaluation of extended dosing intervals (half-life ~77 days).

Positive topline Phase 3 VP-VEC-162-3502 (NCT04652882, n=43, 28-day, double-blind placebo-controlled) for HETLIOZ (tasimelteon) 20 mg in Delayed Sleep-Wake Phase Disorder: sleep onset shifted 42.5 min earlier vs 5.4 min on placebo, a 37.1-minute difference (p=0.022, primary endpoint met); 60% (12/20) advanced sleep timing >=30 min vs 15% (3/20) placebo (p=0.008); no new safety signals. Vanda plans an sNDA; if approved it would be the first FDA-approved DSWPD treatment.

Resubmitted NDA for oxylanthanum carbonate (OLC), oral phosphate binder for hyperphosphatemia in CKD patients on dialysis, after June 2026 CRL citing third-party manufacturing deficiencies (no clinical efficacy/safety concerns raised). Resubmission adds CMC data from a new manufacturing vendor (FDA-inspected March 2024, NAI status) plus 12-month stability data and in-vitro bridging data. Expects FDA acceptance within 30 days and a new 6-month PDUFA clock (~March 2027). Cash runway into 2H 2027.

Favorable TX2100 Phase 1a topline (n=48, randomized double-blind placebo-controlled SAD in healthy volunteers): generally safe/well-tolerated across all 6 dose cohorts (IV + SC), no deaths, SAEs or severe TEAEs; PK showed target-mediated drug disposition confirming APJ target engagement; supports once-weekly and Q2W SC dosing. FDA granted Fast Track designation for TX2100 in HHT (Sept 2026) and cleared the IND (Aug 2026). Open-label Phase 1b in HHT patients planned to start by year-end 2026; placebo-controlled Phase 2 in moderate-to-severe HHT in early 2027. Full data at CureHHT conference Oct 12-16.

8-K filed (items 1.01, 3.02: financing-related)

Offering prospectus supplement filed

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01: financing-related)

Additional Descartes-08 retreatment data in generalized myasthenia gravis presented at the MGFA Scientific Session (AANEM 2026): case series of 5 Phase 2b patients retreated after MG symptom recurrence at least 12 months post-initial course showed mean MG-ADL reduction of 6.6 points at Month 12 following retreatment; median interval between end of initial treatment course and first retreatment infusion was 16.6 months; no new safety signals. Topline data from the Phase 3 AURORA trial of Descartes-08 in MG expected 1Q27.

Additional AMT-130 (ifezuntirgene inilparvovec) Phase I/II data in Huntington's disease: in 12 high-dose patients at 48 months, cUHDRS showed 44% slowing of disease progression (p=0.144, non-significant) and TFC 61% slowing (nominal p=0.008); updated 36-month analysis in all 15 high-dose patients (regulatory anchor for the submitted BLA) showed 80% cUHDRS slowing (nominal p=0.005) and 67% TFC slowing (nominal p=0.011); post-hoc analysis with the prior external control showed 54% cUHDRS slowing (nominal p=0.041) and 68% TFC slowing (nominal p=0.001) at 48 months. Clear dose-dependent response. Five high-dose patients (17%) had treatment-related SAEs of CNS inflammation, all fully resolved; one suicide in a low-dose patient ~5 years post-treatment, assessed as unrelated.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 3.03: financing-related)

Submitted an optimized seamless Phase 2b/3 registration-intent protocol to FDA for Teverelix, its long-acting GnRH antagonist, in advanced prostate cancer patients at increased cardiovascular risk. Stage-gated design begins with ~80 randomized patients (replacing the previously FDA-cleared 40-patient open-label Phase 2b dose-optimization study), expanding to ~608 total once predefined continuation criteria are met - down from ~1,500 planned patients - without Medicus independently funding an additional study; creates a defined, capital-efficient, partner-ready registrational pathway.

8-K filed (items 3.02: financing-related)

Detailed Phase 3 TRIUMPH-2 results for retatrutide (GIP/GLP-1/glucagon triple hormone receptor agonist) in adults with type 2 diabetes and obesity/overweight: at 80 weeks, 12 mg lost an average of 49.6 lbs (20.8%) vs 4.0% placebo; A1C reduced up to 1.6% (9/12 mg), up to 40.0% reached A1C <5.7%; 59.5% on 12 mg no longer met BMI obesity criteria. Results presented at EASD 2026 Milan and simultaneously published in The Lancet. Lilly plans BLA submission in Q1 2027.

8-K filed (items 1.01: financing-related)

Positive topline LIBERTY Phase 3 (n=210, randomized, double-blind, active-controlled): VYD2311 met all primary and key secondary endpoints vs mRNA COVID vaccine COMIRNATY - TEAE/ISR/hypersensitivity 56.5% vs 91.4% at 6d (p<0.0001), 60.9% vs 91.4% at 56d (p<0.0001); systemic AEs 44.1% vs 68.1% (p=0.008); no Grade >2 VYD2311-related AEs, no hypersensitivity/anaphylaxis. VYD2311+mRNA added ~2.5x to vaccine-induced neutralizing titers with no immunologic interference. Plans BLA via Accelerated Approval on DECLARATION+LIBERTY after Type C meeting; DECLARATION safety/immunogenicity topline expected October 2026 (efficacy unblinded post-approval).

8-K filed (items 1.01, 3.02: financing-related)

Interim NEXICART-2 update (n=45, registrational-design US Phase 2, NCT06097832): NXC-201 sterically-optimized BCMA CAR-T produced an 89% (40/45) complete response rate by independent review committee; all 25 new relapsed/refractory AL amyloidosis patients are in CR or MRD-negative; all MRD-negative patients to date reached CR within one year, potentially lifting the CR rate to 98% (44/45); no relapses in any patient reaching CR or MRD-negativity; no neurotoxicity or enterocolitis observed. NEXICART-2 final readout and BLA submission planned 2027. NXC-201 holds FDA Breakthrough Therapy and RMAT designations.

Priced underwritten registered offering of 11,363,637 common shares at $11.00/share for ~$125M gross proceeds (closing ~Sep 30, 2026; J.P. Morgan sole bookrunner; Eventide, Janus Henderson, Ridgeback, Wellington participating); net proceeds to fund NXC-201 development, working capital and general corporate purposes. Offering under S-3 shelf (File No. 333-292665).

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Additional one-year data from Phase 1/2 ACHIEVE trial of zeleciment basivarsen (z-basivarsen, DYNE-101) in DM1: pooled MAD group showed functional improvement vs baseline, vs placebo, and vs matched natural history across multiple measures at 12 months (5xSTS -1.2s, 10MWR -0.3s; QMT total and hand grip +4.8% predicted; MDHI total -25.2%); vHOT improved 3.2s at 6 months (vs +0.4s placebo). Safety data through Apr 20, 2026 favorable, no related SAEs. Data presented at WMS and AANEM 2026. ACHIEVE registrational expansion cohort topline expected Q1 2027, intended to support US Accelerated Approval submission Q3 2027.

Submitted BLA to FDA for VIASKIN Peanut Patch in children aged 4-7 years (peanut allergy); requested Priority Review (Breakthrough Therapy Designation previously granted). Supported by positive Phase 3 VITESSE trial (n=654, largest immunotherapy trial in this population).

Phase 2 CLOVER WaM subset data for iopofosine I 131 in Waldenstrom macroglobulinemia patients refractory/resistant to BTK inhibitor therapy: major response rate 79.2%, overall response rate 87.5%, duration of response exceeding 16 months in subset treated immediately post-BTKi (heavily pretreated, >=2 prior lines). Company reaffirmed confirmatory Phase 3 dosing early 2027 and NDA for accelerated approval in 1H27 (Breakthrough Therapy Designation; expects 6-month review).

FDA granted Fast Track designation to plinabulin + docetaxel for advanced/metastatic non-squamous NSCLC without actionable genomic alterations after progression on anti-PD-(L)1 and platinum chemotherapy (2L/3L setting, same population as the global Phase 3 DUBLIN-4 trial, n~442, OS primary endpoint, prespecified interim at 221 PFS events). Company also announced a strategic transaction selling its majority equity interest in its Chinese subsidiary to Biolin Investment Limited, which will fund the China portion (~50% of enrollment) of DUBLIN-4, substantially reducing cash requirements toward the interim analysis.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Offering prospectus supplement filed

Atara and Pierre Fabre resubmitted BLA for Ebvallo (tabelecleucel) in EBV+ post-transplant lymphoproliferative disease after FDA agreed single-arm ALLELE study with historical control may serve as adequate and well-controlled; rejected twice before (Jan 2025 and Jan 2026 CRLs); analysts flag possible Class 2 resubmission with ~March 29, 2027 action date

Intent to submit Formal Dispute Resolution Request (FDRR) to FDA, reviewed by the Office of New Drugs, to appeal the March 16, 2026 Complete Response Letter that denied approval of reproxalap NDA for dry eye disease (third CRL; nine adequate and well-controlled trials submitted across three NDAs). FDRR submission and OND meeting expected Q4 2026. Also updated cash runway guidance into 2029 on $45.1M cash as of June 30, 2026.

Announced publication in Gynecologic Oncology (online; Oct 2026 print issue) of two-year median follow-up from Phase 2 RAMP 201 of AVMAPKI FAKZYNJA CO-PACK (avutometinib + defactinib) in recurrent low-grade serous ovarian cancer: median follow-up 24.9 mo, mDoR 31.1 mo, mPFS 12.9 mo, no new safety signals, 12% AE discontinuation; KRAS wild-type mDoR improved 9.2 to 12 mo vs July 2025 primary analysis; also presented at SGO 2026 plenary

8-K filed (items 1.01, 2.03: financing-related)

8-K filed (items 1.01: financing-related)

FDA approved DEGEVMA (denosumab-adet) as a biosimilar to Xgeva (denosumab) across all reference indications (bone complications in advanced cancer with bone metastases, giant cell tumor of bone, hypercalcemia of malignancy); Teva's second FDA-approved biosimilar in 2026, completing its denosumab biosimilar portfolio alongside PONLIMSI.

AstraZeneca $2.0B strategic equity investment: ~108,955 convertible preferred shares (1:1,000 conversion) at $18.36 common-equivalent per share (10% premium to prior week's 5-day VWAP), expected to close by end of week. Paired with clinical collaboration to evaluate AZ's sonesitatug vedotin with Summit's ivonescimab (PD-1xVEGF bispecific) in GI cancers (shared costs, each retains commercial rights, no milestones/royalties) plus non-binding MOU on additional ivonescimab + AZ medicine combos. Shares up ~15-18% after hours. FDA decision on ivonescimab ES-SCLC accelerated-approval BLA due by Nov 14.

Announced Sep 30 KOL event to review 52-week data from license partner Ascletis' Phase 3 open-label extension of denifanstat in moderate-to-severe acne in China (improvements in all efficacy endpoints beyond 12-week results; generally well-tolerated) and planned US Phase 3 AURORA trial (initiation expected Q4 2026).

Awarded BARDA contract providing up to ~$214M in non-dilutive funding to develop SCY-247 (second-generation fungerp antifungal) for serious invasive fungal infections; base period ~$18.5M advances oral + IV formulations into Phase 2 in invasive candidiasis; if all options exercised, funds through NDA for treatment of invasive candidiasis and prevention of invasive fungal infections in high-risk patients; contract up to 10 years; SCY-770 ADPKD program and cash runway into 2029 unchanged.

8-K filed (items 1.01: financing-related)

Updated Phase 1a follow-up data for TTX-MC138 in advanced solid tumors (data cutoff Sep 1, 2026): 71.4% stable disease in 14 efficacy-evaluable patients; 3 patients progression-free at one year; primary endpoint met; no dose-limiting toxicities

FDA alignment on potential registrational path for pelareorep in second-line RAS-mutant, microsatellite-stable metastatic colorectal cancer: pivotal Part B expansion of randomized REO 033 could support accelerated approval based on ORR, with PFS underpinning possible full approval; substantially all Part A clinical sites now activated with multiple patients enrolled (pelareorep + FOLFIRI/bevacizumab vs FOLFIRI/bevacizumab, ORR primary); interim Part A clinical update expected by year-end 2026.

8-K filed (items 3.03: financing-related)

Phase 2 MN-001-NATG-202 topline (tipelukast, n=40, hypertriglyceridemia/NAFLD with T2DM): triglyceride reduction vs placebo missed at week 24 (p=0.113) but was significant early at week 4 (p=0.015); significant HDL-C (p=0.0048) and HDL-P (p=0.018) increases; favorable safety; company calls it proof-of-concept, larger study to follow

8-K filed (items 1.01: financing-related)

Phase 3 AZURE-1 met primary endpoint in chronic HDV: 56% on brelovitug 300 mg QW and 45% on brelovitug 900 mg Q4W achieved virologic response + ALT normalization at Week 24 vs 0% on delayed treatment (p<0.0001 both); 48-week Ph2b data showed deepening viral suppression with increased ALT normalization. BLA planned H1 2027; US commercial launch targeted Q4 2027.

Peer-reviewed publication of pooled cardiac safety analysis of Annamycin across five clinical trials (Frontiers in Cardiovascular Medicine): no clinical or subclinical cardiotoxicity observed.

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01: financing-related)

DAYBREAK Phase 3 met primary endpoints for both Zenkuda (tarcocimab tedromer) and tabirafusp-ted (KSI-501) in wet AMD: non-inferior vision gains vs aflibercept at year one (Zenkuda p=0.0007; tabirafusp-ted vision p=0.0036, anatomical p<0.0001); 54% of Zenkuda patients on 6-month dosing at year one with 0% intraocular inflammation. Multi-indication BLA planned 4Q2026 across five positive Phase 3 studies.

8-K filed (items 1.01, 3.02: financing-related)

AstraZeneca submitted a US NDA for ORPATHYS (savolitinib) + TAGRISSO in MET-overexpressed/amplified EGFR-mutated NSCLC after EGFR-TKI progression. Supported by the global SAFFRON Phase 3 (338 pts, 230 centers, 29 countries), which showed statistically significant and clinically meaningful PFS and OS improvement vs doublet platinum chemo in patients progressed on TAGRISSO; no new safety signals. Full data to be presented in a Presidential Symposium at ESMO 2026. The combo is already approved in China on SACHI Phase 3.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

4D-150 advanced into Phase 3 with initiation of 4SIGHT trial in diabetic macular edema (N=514); updated 2-year SPECTRA data: mean BCVA gain +10.8 letters, CST reduction -176 µm.

Pharmacokinetic results with new neflamapimod 50 mg oral tablet formulation; FDA removed the drug-exposure limit on neflamapimod.

8-K filed (items 1.01, 2.03, 3.02: financing-related)

Announced Phase 3 DRAGON topline results for tinlarebant (oral RBP4 antagonist) in adolescent Stargardt disease will be presented at Euretina on Oct 4, 2026 (Free Paper 47); DRAGON met its primary endpoint and tinlarebant NDA was accepted by FDA in Aug 2026 with Priority Review, PDUFA Feb 12, 2027

Biogen discontinues Phase 3 LIGHTHOUSE study of BIIB122 (DNL151, LRRK2 inhibitor, partnered with Denali Therapeutics) in Parkinson's patients with LRRK2 pathogenic mutations. Decision not safety/efficacy-related: trial complexity and long timeline (readout not expected until 2031). Focus shifts to the earlier-readout Phase 2b LUMA study, whose protocol is amended to enroll eligible LRRK2-mutation patients; enrolled LIGHTHOUSE participants can roll into LUMA. Biogen/Denali 'remain committed' to BIIB122.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Nature Medicine published ASPEN-06 Phase 2 data for evorpacept + trastuzumab/ramucirumab/paclitaxel in HER2-positive gastric/GEJ cancer: 40.3% ORR vs 26.6% control, durable clinical benefit.

Offering prospectus filed

8-K filed (items 3.02: financing-related)

Positive Phase 1 safety/efficacy data for prula-cel (formerly ADI-001) in SLE with/without lupus nephritis (n=22 evaluable): no CRS >Grade 2, no IEC-HS, no ICANS; 54% DORIS remission at 12 months and 50% CRR; all patients discontinued immunosuppressants. Pivotal lupus nephritis trial start-up planned 4Q2026.

Mirum to present topline results on September 28, 2026 (investor call 8:30 AM ET) from AZURE-1 Phase 3 study of brelovitug in chronic hepatitis delta, plus 48-week data from the Phase 2b portion.

Jury awarded Nektar $90M in its suit against Eli Lilly over the REZPEG/neural-cytokine licensing dispute (implied-covenant claim); a win for Nektar but a far smaller sum than the ~$1B sought. Lilly conceded a data error but contested intent/causation/damages. Award is appealable; cash years out.

8-K filed (items 3.02: financing-related)

Priced upsized concurrent offering: 7,857,143 shares of common stock at $35.00/share (up from $200M to $275M, net proceeds ~$258.2M), settling Sep 25, 2026; 30-day underwriter option for 1,178,571 more shares.

Priced upsized concurrent offering: $225M of 2.00% convertible senior notes due 2032 (up from $200M, net proceeds ~$218M), settling Sep 25, 2026; 30-day underwriter option for $33.75M more. Proceeds fund VK2735/VK3019 development.

Shelf registration filed (enables future raises incl. ATM)

Offering prospectus supplement filed

Offering prospectus supplement filed

FDA accepted sNDA and granted Priority Review for lower doses of Joenja (leniolisib) for children aged 4+ weighing 13-27 kg with APDS; PDUFA target action date January 30, 2027. Supported by positive Phase III data in children 4-11.

Bahamas Longevity and Regenerative Therapies Act (LARTA) Board granted provisional approval plus priority designation for OCU400 (modifier gene therapy) in retinitis pigmentosa; Ocugen to supply via expanded access with first patient targeted within 90 days of full approval. Bahamas-only provisional pathway, not FDA/EMA; Phase 3 topline still guided 1Q 2027.

Completed Health Canada Pre-NDS process for PrimeC (ALS) with alignment on filing content; targeting December 2026 New Drug Submission filing; pursuing optimized US strategy (smaller/shorter PARAGON pivotal, active comparator vs edaravone, potential accelerated-approval discussion with FDA)

Merck and Daiichi Sankyo voluntarily withdrew the US application seeking accelerated approval for ifinatamab deruxtecan in extensive-stage small cell lung cancer after discussions with the FDA, which determined mid-stage clinical data did not satisfy requirements for an early green light.

FDA approved Atebrioz (zilurgisertib) for FOP patients aged 12+ (once-daily oral ALK2 inhibitor, licensed from Incyte; PDUFA was 2026-09-26, approved a day early): PROGRESS pivotal Ph2 (n=63) showed new heterotopic ossification volume -3.2 cm3 vs +24.6 cm3 on placebo at week 24 (nominal p=0.004); 3rd FDA-approved FOP therapy after Sohonos and Pasatru; launch planned October 2026

Shelf registration filed (enables future raises incl. ATM)

FDA approved Olumiant (baricitinib) for pediatric patients 12+ with severe alopecia areata, expanding the label beyond adult severe AA (2022) and RA; based on BRAVE-AA-PEDS Phase 3 (NCT05723198): SALT<=20 at Wk36 in 42% (4mg)/27% (2mg) vs 5% placebo, SALT<=10 in 37%/21% vs 2%

Five-year follow-up data from CARTITUDE-2 Cohort A (n=20) of CARVYKTI (cilta-cel) in RRMM after 1-3 prior lines (presented at IMS 2026): 10 of 20 patients (50%) alive and progression-free without maintenance therapy at 5 years after a single infusion; median PFS 60.5 months; 5-year OS rate 69.2%; median OS not reached; safety consistent with known profile (no new neurotoxicity)

Shelf registration filed (enables future raises incl. ATM)

Positive Phase 4 STAR topline: TREMFYA (guselkumab) met primary (BASDAI at Wk24) and major secondary endpoints (ASDAS-CRP, MRI-confirmed sacroiliac inflammation) in biologic-naive adults with active PsA and axial involvement (NCT04929210, n=411); first dedicated IL-23 inhibitor study in MRI-confirmed axial PsA; safety consistent with established profile

Long-term CARTITUDE-2 cohort A data (Phase 2, n=20, early-line R/R multiple myeloma) presented at IMS 2026: 50% of patients remained alive and progression-free at 5 years after a single CARVYKTI (cilta-cel) infusion with no maintenance therapy; median PFS 60.5 mo, 5-yr OS 69.2%

8-K filed (items 1.01, 1.02, 3.02: financing-related)

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 3.03: financing-related)

First presentation of Ph3 EXCALIBER-RRMM (NCT04975997) results for ZENBEXUS (iberdomide) + daratumumab/dex in RRMM at IMS 2026 (simultaneous Lancet Oncology): MRD-negative CR 41.1% vs 20.7% on DVd (p<0.0001, OR 2.75); ORR 88.9% vs 76.1%; CR or better 45.9% vs 24.9%; dual primary endpoints are MRD-neg CR and PFS

First presentation of prespecified analysis from pivotal Phase 3 EXCALIBER-RRMM (NCT04975997) of iberdomide + daratumumab + dexamethasone (ZDd) vs DVd in relapsed/refractory multiple myeloma: statistically significant and clinically meaningful improvement in MRD-negative complete response, 41.1% vs 20.7% (p<0.0001, OR 2.75) at median 15.7 months follow-up in 420 evaluable patients; ORR 88.9% vs 76.1%, CR or better 45.9% vs 24.9%. Presented as late-breaking oral (LBA12) at IMS 2026 Glasgow and simultaneously published in The Lancet Oncology. Dual primary endpoints are MRD-negative CR and PFS; PFS confirmatory analysis ongoing.

FDA accepted sBLA and granted Priority Review for perioperative IMFINZI (durvalumab) plus neoadjuvant enfortumab vedotin in cisplatin-ineligible/declined muscle-invasive bladder cancer, based on VOLGA Phase 3 interim (statistically significant event-free and overall survival benefit vs cystectomy +/- adjuvant); PDUFA expected Q4 2026, full data at a forthcoming meeting.

FDA approved Juvmo (tavapadon), once-daily oral D1/D5 dopamine receptor partial agonist, for Parkinson's disease in adults (AbbVie gained it via the $8.7B Cerevel Therapeutics acquisition in 2023)

Offering prospectus supplement filed

Phase 3 upLIFT (ersodetug, tumor HI): 7th of 8 enrolled participants met primary-endpoint responder criterion (one withdrew to hospice, counted as non-responder); topline guided before end of 2026

Positive 3-year durability data from Phase 2 ALTITUDE study of surabgene lomparvovec (sura-vec, ABBV-RGX-314) in diabetic retinopathy, presented at Retina Society 59th Annual Scientific Meeting: 60% of Dose Level 3 NPDR participants (6/10) with >2-step DRSS improvement at 3 years, no vision-threatening events, no additional DR treatment; no intraocular inflammation with short-course prophylactic topical steroids; no new safety signals through 3 years (n=17).

FDA cleared IND application for PM647, an investigational in vivo Prime Editor for alpha-1 antitrypsin deficiency (AATD): Phase 1/2 global open-label single-arm first-in-human study of a one-time IV infusion planned, initially enrolling adults with lung-only AATD then expanding to a liver-disease cohort; initial clinical data expected 2027; second liver-franchise IND clearance after PM577a (Wilson disease)

U.S. FDA clearance for protocol amendment to J&J-led Phase 3 NANORAY-312 study of JNJ-1900 (NBTXR3) in locally advanced platinum-ineligible head and neck cancer: planned interim analysis eliminated and final analysis modified to fewer events so it reads out sooner

FDA cleared IND application for forazapadin in facioscapulohumeral muscular dystrophy (FSHD), expanding the program into a second degenerative muscle disease; Phase 2 study expected to begin Q4 2026; FSHD Canada Foundation to provide up to $5M non-dilutive financing toward clinical development

Positive topline from pivotal Phase 2b/3 BRUNELLO trial of remigromig (MK-3000, tri-specific Wnt-pathway agonist, from the $3B EyeBio acquisition) in diabetic macular edema (n=984): both doses (0.5/0.8 mg) non-inferior to ranibizumab (Lucentis) on Week-52 BCVA; higher proliferative diabetic retinopathy, vitreous hemorrhage and treatment discontinuation rates in remigromig arms; full data at AAO October 2026; second Phase 2b/3 trial ongoing

FDA approved WELIREG (belzutifan) + LENVIMA (lenvatinib) for adults with advanced ccRCC following a PD-1/PD-L1 inhibitor (~10 days ahead of the Oct 4 PDUFA target date); approval based on Phase 3 LITESPARK-011 vs cabozantinib: mPFS 14.6 vs 10.6 mo, ORR 53% vs 40% (p=.0002), mOS 33.7 vs 28.6 mo

Completed last-patient-last-visit (LPLV) in the double-blind portion of the Phase 2b/3 COMBAT-ALS trial of MN-166 (ibudilast) in ALS (234 patients randomized at US/Canada sites, primary endpoint CAFS); topline results from the double-blind portion expected by end of 2026; full-study LPLV expected March 2027

FDA approved new potency assay (T-cell Proliferation Inhibition BioAssay, TIBA) for release and stability monitoring of all new commercial lots of Ryoncil (remestemcel-L-rknd), to be used alongside existing IL-2Rα inhibition and CAP assays

FDA approved Onswik (insulin efsitora alfa-gobe), once-weekly basal insulin injection for adults with type 2 diabetes (QWINT Ph3 program n>3,400; non-inferior A1C vs glargine/degludec); 4th global approval after EMA/Mexico/Japan

Positive one-year topline durability data from registrational KYSA-8 trial of miv-cel (mivocabtagene autoleucel) in stiff person syndrome (n=26; T25FW median improvement 49% at month 12, p<0.0001; 92% free of chronic immunotherapies) + positive longer-term follow-up from Phase 2 portion of KYSA-6 in generalized myasthenia gravis (responses sustained up to 1.5 yrs); rolling BLA submission on track for completion in Q4 2026

Kodiak to present topline results on September 28, 2026 (webcast 8:30 AM ET) from DAYBREAK pivotal Phase 3 study of Zenkuda (tarcocimab tedromer) and KSI-501 (tabirafusp alfa tedromer) in wet age-related macular degeneration.

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 3.02: financing-related)

R&D Day: final OS from randomized Ph2 OVATION 2 of IMNN-001 in newly diagnosed advanced ovarian cancer - mOS 45.1 mo vs 30.4 mo SOC (+14.7 mo, widened from 11.1 mo at Jul 2024 interim); 24.2-mo benefit in PARP-maintenance subgroup (65.6 vs 41.4 mo); no CRS or systemic IL-12 toxicities; pivotal Ph3 OVATION 3 enrolling ahead of plan, targeting enrollment completion 1Q29

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 2.03: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

Shelf registration filed (enables future raises incl. ATM)

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01, 3.02: financing-related)

Reached alignment with FDA on protocol for global Phase 3 study of efzofitimod in pulmonary sarcoidosis with restrictive lung disease: randomized, double-blind, placebo-controlled, ~372 pts, 5.0 mg/kg IV q3w, primary endpoint change in FVC at week 48; study-related activities to begin Q4 2026

Phase 3-enabling topline from Phase 2 portion of RADIANT study of remlifanserin (5-HT2A inverse agonist) in Alzheimer's disease psychosis: 60 mg narrowly missed primary endpoint (SAPS-H+D -12.6 vs -10.4 placebo, ES 0.26, p=0.0603); key secondary CGI-S-ADP significant nominal (p=0.0077, ES 0.37); 30 mg showed minimal improvement; favorable safety, no QT signal; Acadia to continue the two ongoing Phase 3 ADP studies and remove the 30 mg arm

Acadia Pharmaceuticals Announces Phase 3 Enabling Topline Results from Phase 2 RADIANT Study of Remlifanserin for the Treatment of Alzheimer’s Disease Psychosis (ADP) — Meaningful and consistent efficacy trends across endpoints support continuing with Phase 3 development of 60 mg once daily...

8-K filed (items 1.01, 2.03: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 3.03: financing-related)

8-K filed (items 1.01: financing-related)

FDA approved LYRFIGTU (lirafugratinib) for previously treated FGFR2 fusion/rearrangement-positive cholangiocarcinoma, 4 days ahead of the Sep 27 PDUFA; Relay out-licensed lirafugratinib worldwide to partner Elevar Therapeutics (HLB subsidiary) in Dec 2024 for $75M upfront + regulatory milestones and up to $425M commercial milestones, so a regulatory milestone payment is likely triggered; approval based on Ph1/2 ReFocus ORR 46%, mDOR 11.8 months (NCT04526106)

8-K filed (items 1.01: financing-related)

Offering prospectus supplement filed

8-K filed (items 1.01: financing-related)

Positive Week 28 data from EVERLAST-A Phase 2a trial of ORKA-001 (half-life-extended IL-23p19 mAb, 600 mg at Wk 0/4) in moderate-to-severe plaque psoriasis: PASI 100 71.4% (45/63, up from 63.5% at Wk 16) and PASI 90 87.3% at Wk 28 with no dosing after Week 4; IGA 0/1 84.1%; placebo-crossover arm reached PASI 100 40.0% at equivalent post-dose timepoint (vs 42.9% active arm); safety consistent with IL-23 class, no ISR, 2 unrelated SAEs; 52-wk data expected Dec 2026, EVERLAST-B Ph2b data in 4Q 2026

Offering prospectus supplement filed

OF LONGEVERON INC., DATED SEPTEMBER 16, 2026 Exhibit 99.1 Longeveron Announces Topline Results from ELPIS II Phase 2b Clinical Trial Evaluating Laromestrocel as a Potential Treatment for Hypoplastic Left Heart Syndrome (HLHS) ------------------- Company Announces Exploration of All Options to...

8-K filed (items 1.01, 2.03: financing-related)

8-K filed (items 1.01, 2.03: financing-related)

Positive prespecified interim results from partner Roche's Phase 3 IMAgINATION study of sefaxersen in primary IgA nephropathy: met primary endpoint (statistically significant, clinically meaningful UPCR proteinuria reduction vs placebo at 37 weeks); no new safety signals; study continues blinded to week 105 eGFR readout [NCT05797610, per the release]

Ionis announces positive interim data from partner Roche’s Phase 3 IMAgINATION study in IgA nephropathy (IgAN) – Sefaxersen demonstrated statistically significant and clinically meaningful reductions in proteinuria versus placebo at 37 weeks, showing best-in-class potential – – IgAN is a...

Topline results from IMVT-1402 (imeroprubart) proof-of-concept study (NCT06980805, 57 patients) in cutaneous lupus erythematosus: missed primary endpoint (CLASI-A percent change from baseline at Week 12, not statistically significant); positive trends with deeper IgG reductions but did not meet internal bar — discontinuing development in CLE. Favorable safety profile; all other programs (Graves, DTR RA, MG, CIDP, Sjogren's) on track.

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

Submitted first NDA to FDA for seralutinib in PAH (supported by Phase 3 PROSERA as single adequate well-controlled study + Phase 2 TORREY confirmatory evidence; FDA pre-NDA minutes framed PROSERA significance/magnitude as review issues, not filing issues; filing decision expected later this year)

Evommune Announces Late-Breaker Presentation of Phase 2a Data for EVO301 in Atopic Dermatitis at European Academy of Dermatology & Venereology 2026 Congress Palo Alto, Calif., and New York, September 23, 2026 – Evommune, Inc. (NYSE: EVMN) (“Evommune” or the “Company”), a clinical-stage...

8-K filed (items 1.01, 3.02: financing-related)

MAVERIC Phase 3 (CardiolRx in recurrent pericarditis) hit original 110-pt enrollment target ahead of schedule; steering committee recommends expanding to up to 150 pts after blinded review showed lower-than-expected recurrence events; topline now guided mid-2027 (was Q1 2027); cash runway into Q1 2028

Armata Pharmaceuticals Receives $3.7 Million in Additional Non-Dilutive Funding from U.S. Department of War to Support Advancement of AP-SA02 Phase 3 Development Initial Phase 3-focused funding to support advancement of AP-SA02 in complicated Staphylococcus aureus bacteremia New commitment is...

Encouraging interim results from ongoing Phase 2 study of ARCT-810 in OTC deficiency: generally safe/well tolerated with encouraging activity across key urea cycle biomarkers; per FDA advice, next-gen candidate ARCT-2601 (LUNAR 2.0 delivery platform, >30-fold higher protein expression in NHP studies) to be integrated into the ongoing Phase 2 study by YE 2026; also announced acquisition of AI discovery company myNEO

8-K filed (items 3.03: financing-related)

FDA final approval of ANDA for Everolimus Tablets for Oral Suspension 2mg/3mg/5mg (generic Afinitor Disperz); ~$123M US annual sales per IQVIA MAT July 2026

Positive week-100 safety & immunogenicity results from Part 1 of VacSYn Phase 2 trial of ACI-7104 in early-stage Parkinson's disease: all primary endpoints met (safe/well tolerated, 100% immunogenicity response after 3 immunizations, CSF antibody penetration with target-engagement signal); Part 2 design headed to FDA review early 2027 [NCT06015841, per the release]

Preliminary positive data from open-label extension portion of PALISADE-4 Phase 3 study of fasedienol for acute treatment of social anxiety disorder

Positive VK2735 maintenance-study topline: 22% placebo-adjusted weight loss at Week 33 (17.5 mg weekly, no plateau); EOW dosing retained 97% of Week 21 loss, monthly 90% vs 61% placebo; GI AE rates similar to placebo

Viking Therapeutics Announces Positive Topline Results from Maintenance Study of GLP-1/GIP Agonist VK2735 Demonstrating the Promise of Multiple Maintenance Dosing Regimens 22% Placebo-Adjusted Weight Loss Reported at Week 33 Following Weekly Dosing of VK2735 at 17.5 mg; No Plateau Observed...

8-K filed (items 1.01: financing-related)

Shelf registration amended

Health Canada approval of IMCIVREE (setmelanotide) for weight management in adult and pediatric patients 4+ with acquired hypothalamic obesity

Quoin Pharmaceuticals Announces FDA Fast Track Designation for QRX003 for the Treatment of Peeling Skin Syndrome – First-Ever Fast Track Designation Granted for a Peeling Skin Syndrome Therapy – Second Fast Track Designation Granted to QRX003, in Addition to Netherton Syndrome – Fast Track...

Positive Ph2 topline for leniolisib in genetically defined PIDs with immune dysregulation linked to PI3Kd signaling: generally well-tolerated with clinical improvements incl. mean 26.4% spleen volume reduction (n=13, single-arm dose-escalation); late-breaking abstract accepted at ESID 2026; separate Ph2 leniolisib in CVID topline expected Q4 2026

PDS0101 VERSATILE-002 Phase 2 results published in JAMA Oncology: PDS0101 + pembrolizumab in 1L HPV16+ R/M HNSCC (ICI-naive CPS>=1, n=53 mITT): mOS 39.3 mo vs 17.9 mo best published pembro SoC; ORR 36% vs 19% SoC; 13.8% Gr>=3 TRAEs; Ph3 planned, partnership strategy pursued

PDS Biotech Announces Publication of VERSATILE-002 Phase 2 Clinical Trial Results of PDS0101 with pembrolizumab in HPV16-Positive Recurrent and/or Metastatic (R/M) Head and Neck Cancer in JAMA Oncology 39.3 months median overall survival in first-line (1L) R/M immune checkpoint inhibitor naïve...

European Commission marketing authorization for Ubeslo (obicetrapib 10 mg) and Evlarco (obicetrapib + ezetimibe fixed-dose) in primary hypercholesterolemia/mixed dyslipidemia — first global regulatory approval of obicetrapib (with Menarini)

FDA approves WINREVAIR label update to include Phase 3 HYPERION data in adults with newly diagnosed PAH (76% reduction in clinical worsening risk, HR 0.24, p<0.0001)

THIO-104 pivotal Phase 3 of ateganosine (telomere-targeting) as 3L NSCLC reached 65 randomized patients across 38 sites in 6 countries; on track for >100 by year-end; most recent assessment of ateganosine + checkpoint inhibitor showed 90.5% interim DCR in heavily pretreated 3L NSCLC in Phase 2 THIO-101

8-K filed (items 1.01: financing-related)

FDA final approval of BRAVNETSA (lutetium Lu 177 dotatate) for SSTR-positive GEP-NETs; first radioligand therapy approved via ANDA pathway; FDA-determined bioequivalent and therapeutically equivalent to LUTATHERA

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Positive Ph3 FUSION topline: ulefnersen met primary endpoint (joint-rank functional impairment + survival, p=0.0005) in FUS-ALS; first treatment to target the genetic cause of FUS-ALS and show disease-modifying potential; Ionis/Otsuka to discuss expedited regulatory pathways with FDA

IDMC recommends ongoing Ph2 minimal residual disease (MRD) study of IMNN-001 continue without modification after favorable safety review (no CRS, systemic toxicities or serious immune-related AEs); also no new safety concerns in pivotal Ph3 OVATION 3 trial

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01, 2.03, 3.02: financing-related)

FDA lifted clinical hold on simufilam IND, enabling planned Phase 2a proof-of-concept study in TSC-related epilepsy (16-wk randomized double-blind, ~40 pts, screening from Q1 2027)

Updated long-term Phase 2 BESTOW extension study results presented at the International Congress of the Transplantation Society

FDA regulatory update on tovecimig BLA in previously treated advanced biliary tract cancer (BTC): FDA recommended Compass conduct a trial demonstrating a survival benefit before submitting a BLA; Compass disagrees (cites positive COMPANION-002 Phase 2/3: ORR 18.0% vs 5.3% p=0.0228, median PFS 4.7 vs 2.6 mo HR 0.44 p<0.0001; OS confounded by crossover, not significant) and intends to engage FDA while continuing BLA preparation

Positive Ph3 EMBARQ-CSU1/CSU2 topline: barzolvolimab met Week 12 UAS7 primary + all key secondaries in CSU (n=1,939); Week 12 complete response 42.1-45.7% vs 9.3-12.6% placebo; BLA planned 2027

Celldex Announces Positive Results from Phase 3 EMBARQ-CSU1 and EMBARQ-CSU2 Studies of Barzolvolimab Which Met Primary and All Key Secondary Endpoints · Best-in-disease results showed rapid, profound, sustained efficacy, supporting barzolvolimab’s potential to be a transformational therapy in...

8-K filed (items 1.01: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Offering prospectus filed

News Release bioAffinity Technologies to Present Positive Clinical and Veteran-Focused Data on CyPath® Lung at NAVREF 2026 Annual Conference Presentations demonstrate beneficial role for CyPath Lung in veteran lung cancer care NAVREF 2026 focuses on innovative research to improve healthcare for...

Offering prospectus supplement filed

Positive Ph3 OASIZ 301 topline: dazodalibep met Week 48 ESSDAI primary endpoint in moderate-to-severe systemic Sjogren's disease (n~621; improvement from Wk4, sustained); no FDA-approved therapies in Sjogren's; OASIZ 303 symptomatic study completes Q4 2026

European Commission approval of RINVOQ (upadacitinib) for children with active polyarticular juvenile idiopathic arthritis

Positive Phase 2b AMPLIFIED results for inaxaplin 45mg QD in AMKD: -42.7% UACR at Wk13 in modest-proteinuria cohort (N=22), -17.3% in T2D cohort (N=17); safe, no inaxaplin-related SAEs; AMPLITUDE Phase 2/3 fully enrolled, interim analysis data early 2027 (could support accelerated approval)

Offering prospectus supplement filed

8-K filed (items 1.01: financing-related)

8-K filed (items 3.03: financing-related)

Positive topline from pivotal Phase 3 Study 451 of CAPLYTA (lumateperone 42mg) in bipolar I manic episodes (YMRS -4.8 LSMD vs placebo, p<0.0001, effect as early as day 3); second pivotal study (Study 452) completed, analysis underway; not yet approved for mania

8-K filed (items 3.03: financing-related)

FDA granted Priority Review to ARIKAYCE sNDA for full approval in MAC lung disease (Ph3b ENCORE met primary RSS + secondary culture conversion endpoints); PDUFA target Jan 28, 2027; Japan PMDA review planned Q4 2026

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01, 3.02, 3.03: financing-related)

Cue Biopharma Announces Positive Topline Results from CUE-221 Phase 2 Study in Chronic Spontaneous Urticaria Results support potential for differentiated clinical impact for patients suffering from chronic hives and advancement to Phase 2b/3 registration enabling study • Primary endpoint and the...

Reported positive Phase 1b proof-of-concept results for ALKS 7290 (oral orexin-2 receptor agonist) in adults with ADHD: dose-dependent, clinically meaningful AISRS reductions (median -14.0 pts at 20mg, -19.0 pts at 50mg from ~39 baseline) at day 14; first OX2R agonist to show ADHD symptom benefit; Phase 2 study (NCT07755410, n~312) enrolling, first patient dosed Sep 2026, data expected 2027

Alkermes Contacts: For Investors: Sandy Coombs, +1 781 609 6377 For Media: Gretchen Murphy,+1 781 609 6419 Alkermes Announces Positive Phase 1b Results Demonstrating Clinical Proof-of-Concept for ALKS 7290 in Adults With Attention-Deficit Hyperactivity Disorder (ADHD) — ALKS 7290 is the First...

Positive Ph2 CUE-221 topline in CSU (n=145, China): HSS7=0 at Wk12 met at all doses (54%/53%/43% vs 11% placebo, p<0.005 top two; 41% omalizumab); key secondary UAS7=0 met at 4mg/kg (46% vs 11%); durable benefit 12 weeks off drug; Phase 2b/3 planned

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Registration statement amended

FDA full approval of Inluriyo (imlunestrant) + Verzenio (abemaciclib) for ER+/HER2-/ESR1-mutated advanced/metastatic breast cancer after >=1 line of endocrine therapy; Ph3 EMBER-3 ESR1-mutated subgroup (n=159): mPFS 11.1 vs 5.5 mo (HR 0.53); second Inluriyo approval in <1 yr (monotherapy approved Sep 2025); Guardant360 CDx companion diagnostic also approved

Registration statement filed (primary offering)

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

8-K filed (items 3.03: financing-related)

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

8-K filed (items 1.01, 2.03, 3.02: financing-related)

Registration statement filed (primary offering)

8-K filed (items 1.01, 3.02: financing-related)

FDA approved lanreotide injection 120mg/0.5mL (generic referencing Somatuline Depot) with CGT designation; immediate launch; reference US sales $983M (12mo to Jul 2026).

NDA submitted for azetukalner in focal seizures (X-TOLE/X-TOLE2); voluntary pause of new enrollment in Ph3 psychiatry studies (X-NOVA2 MDD, X-CEED BPD) after neuropsychiatric AEs incl psychosis, in consultation with DSMB; epilepsy studies unaffected; X-NOVA2 ~360/450 pts powered, topline Q1 2027

8-K filed (items 1.01: financing-related)

Telomir Announces Peer-Reviewed Publication Showing Telomir-Zn Restores Visual Function and Retinal Structure in a Model of Age-Related Macular Degeneration (AMD) Findings Strengthen Telomir-Zn’s Oncology-Focused Platform While Demonstrating Broader Relevance of Its Metal-Dependent Epigenetic...

8-K filed (items 3.03: financing-related)

FDA standard full approval of FAYUVI (rebisufligene etisparvovec-hopf, UX111) for pediatric MPS IIIA (Sanfilippo syndrome Type A): first-ever treatment for the disease; Transpher A pivotal trial + long-term follow-up (~8 yrs) showed +23.5 pt Bayley-III cognitive score vs natural history (p<0.0001); 2nd gene therapy and 6th overall FDA approval for Ultragenyx; Priority Review Voucher received; commercial product to Qualified Treatment Centers within 30-60 days

FDA approved sNDA for IBTROZI (taletrectinib) with updated label: TRUST-I TKI-naive median DOR 49.7 months (longest reported for approved ROS1 TKI); approval came ~4 months ahead of PDUFA; no new safety signals

NeOnc Executives Invest Approximately $629,000 in Open-Market Stock Purchases Following Positive NEO100 Phase 2a Results CEO Amir Heshmatpour has purchased 111,000 shares for approximately $418,700 since the Company announced positive NEO100 Phase 2a results CALABASAS, Calif., Sept. 16, 2026 –...

MIRA Pharmaceuticals Advances Ketamir-2 to Phase 2a in Chemotherapy-Induced Peripheral Neuropathy (CIPN) Following FDA Review Protocol Submitted for IRB Review at a Leading Cancer Research Institution, Advancing the Study Toward Site Initiation MIAMI, Sept. 17, 2026 (GLOBE NEWSWIRE) — MIRA...

8-K filed (items 1.01, 3.02: financing-related)

OF ISPIRE TECHNOLOGY ISSUED ON SEPTEMBER 15, 2026 Exhibit 99.1 Ispire Technology Inc. Reports Fourth Quarter and Fiscal Year 2026 Financial Results Q4 Revenue Increased 33% Year-over-year and 43% Sequentially to $26.7 Million Full Year Operating Cash Burn Improves by $6.8 Million, Signaling...

8-K filed (items 2.03: financing-related)

Registration statement filed (primary offering)

Offering prospectus filed (possible follow-on or ATM takedown)

Resubmitted the Anaphylm (dibutepinephrine) sublingual-film NDA to FDA after the Jan 30 2026 CRL (human-factors + confirmatory PK studies completed; pouch-opening time 17s -> 3s, no protocol-defined use errors in self-administration arm); FDA to set review classification and timeline after initial review

Offering prospectus supplement filed

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 1.01, 2.03: financing-related)

8-K filed (items 3.02: financing-related)

Shelf registration amended

Discontinues VHB937 (lifonebart) development in ALS after Phase 2 ASTRALS trial (n=251, early-stage ALS) missed primary composite endpoint (survival without permanent ventilation + ALSFRS-R change) and secondary endpoints (respiratory decline, NfL); disclosed in an Aug 25 community letter, confirmed by Novartis Sep 16 (Endpoints/Reuters); program continues in Alzheimer's (Phase 2 enrolling)

ELPIS II Phase 2b topline missed primary endpoint (RVEF change at Month 12; LS mean diff -0.7 pp, p=0.8336) for laromestrocel in HLHS; exploratory endpoints non-significant; company to explore strategic options + cost containment

8-K filed (items 1.01: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01: financing-related)

8-K filed (items 3.02: financing-related)

Positive final topline results from the Phase 3 ORIGIN 3 trial of TRUTAKNA (atacicept) in IgA nephropathy (IgAN) (n=428): met all prespecified endpoints; placebo-adjusted eGFR change +5.6 mL/min/1.73m2 at 52 wks (p<0.0001), annualized slope +5.0 through 104 wks; composite kidney disease progression HR 0.24 (76% risk reduction, 11 vs 38 events); sBLA planned Q4 2026.

Vera Therapeutics Announces TRUTAKNA ™ (atacicept-vymj) Stabilized eGFR and Prevented Kidney Disease Progression Through Two Years in ORIGIN 3 Final Efficacy Analysis in IgA Nephropathy • TRUTAKNA, the first and only FDA-approved BAFF and APRIL inhibitor, met all prespecified endpoints,...

Offering prospectus filed (possible follow-on or ATM takedown)

8-K filed (items 1.01, 3.02: financing-related)

FDA alignment on pivotal Phase 2 trial of RP-A501 in Danon disease: 12-patient pivotal population (first 3 treated under modified protocol count), recalibrated dose 3.8e13 GC/kg, 12-month co-primary endpoints (myocardial LAMP2 expression + >=10% LV mass index reduction) intended to support accelerated approval; no TMA or capillary leak in first 3 patients; remaining 9 to be dosed by mid-2027

Rocket Pharmaceuticals Announces FDA Alignment on Path to Complete Pivotal Phase 2 Trial of RP-A501 in Danon Disease FDA confirms pivotal efficacy population of 12 patients, including the first three treated under the modified protocol FDA review of initial safety data supports continued...

Offering prospectus supplement filed

8-K filed (items 1.01, 3.02: financing-related)

8-K filed (items 3.03: financing-related)

8-K filed (items 1.02: financing-related)

8-K filed (items 1.01, 3.02, 3.03: financing-related)

8-K filed (items 1.01, 3.02: financing-related)

FDA accepted NDA for bezuclastinib in Advanced Systemic Mastocytosis (AdvSM); PDUFA target action date June 29, 2027; NDAs for NonAdvSM (PDUFA Dec 30, 2026) and GIST (PDUFA Nov 30, 2026) also under review

Mixed Ph2 Seabreeze STAT asthma topline for rademikibart (anti-IL-4Ralpha, n=160): primary treatment-failure rate cut 66% vs placebo but missed significance (p=0.153); key secondary post-BD FEV1 +130mL placebo-adjusted at Day 7, p=0.023; safe; Seabreeze STAT COPD topline later this month; FDA alignment meeting on Ph3 program planned

BlossomHill Therapeutics Presents Updated Data from Ongoing Phase 1/2 SOLARA Trial Demonstrating Encouraging Anti-Tumor Activity of OMNI-EGFR ™ Inhibitor BH-30643 in EGFR C797S-Positive NSCLC at IASLC 2026 World Conference on Lung Cancer 45% objective response rate and 88% disease control rate...

ADDRESS-LC Phase 2 topline for bezisterim in Long COVID (signal-finding, n=203): no endpoint stat-sig in overall ITT; statistically significant improvements in pre-specified high-symptom-burden subgroups; safety similar to placebo; confirmatory Phase 3 planned

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

Offering prospectus supplement filed

8-K filed (items 1.01: financing-related)

8-K filed (items 1.01: financing-related)

FDA approved Pixclara (floretyrosine F 18), the first FDA-approved FET-PET imaging drug for glioma, to differentiate recurrent/progressive glioma from treatment-related change in adults and pediatric patients 1 month and older; NDA resubmission after a prior CRL (PDUFA target was Sep 11, 2026). Pixclara (TLX101-Px) also the subject of a Phase 3 registrational study for brain metastases indication expansion.

8-K filed (items 3.02: financing-related)

Telomir Announces Research with The Ohio State University Demonstrating In-Vitro Anti-Cancer Activity in Human and Canine Osteosarcoma Models Preclinical Findings Support Parallel Development Pathway in Canine Osteosarcoma as TNBC Program Advances Toward Site Initiation MIAMI, Sept. 14 (GLOBE...

Scholar Rock Announces FDA Approval of ISEMBYLD™ (apitegromab-mstn), the First and Only Muscle-Targeted Treatment for Children and Adults with Spinal Muscular Atrophy (SMA) September 11, 2026 · ISEMBYLD is approved for use in all adults and children ≥2 years of age with SMA who are currently...

Discontinues SION-719 as add-on to Trikafta after Phase 2a PreciSION CF missed key activity endpoint (sweat chloride); pivots to SION-451 + SION-2222 dual combination Phase 2a proof-of-concept trial (AscenSION CF, expected Q1 2027); ~46% workforce reduction with ~$6.4M restructuring charges; cash runway extended into H2 2029

FDA granted Breakthrough Therapy Designation to RASONQUE (daraxonrasib) in combination with gemcitabine + nab-paclitaxel for first-line (treatment-naive) metastatic pancreatic adenocarcinoma. Based on Phase 1/2 RMC-GI-102 data (58% confirmed response rate in the treatment-naive cohort per company; manageable safety consistent with known profiles). Informs RASolute 303 Phase 3 evaluating RASONQUE mono + combo vs GnP alone in previously untreated mPDAC. Third BTD for RASONQUE, fifth across the RAS(ON) portfolio.

8-K filed (items 1.02: financing-related)

8-K filed (items 1.01: financing-related)

Offering prospectus supplement filed

8-K filed (items 1.02: financing-related)

Lipocine Announces Initiation of BLOOM Phase 3 Study of LPCN 1154 for Postpartum Depression ● Study informed by encouraging exploratory efficacy findings and a favorable safety profile in the prior Phase 3 trial ● Approximately 120 participants with severe postpartum depression (PPD) planned;...

Positive Ph3 Panorama topline for DT120 (lysergide) ODT in GAD (n=245, single 100 ug dose): HAM-A -5.1 placebo-adjusted at Wk12 (p<0.0001, d=0.64), met primary + all key secondaries; 3rd positive Ph3 for DT120 (Emerge MDD Jun, Voyage GAD Aug); pre-NDA mtg 4Q26, NDA filing 1H27

Definium Therapeutics Announces Positive Topline Results from Phase 3 Panorama Study of DT120 ODT in Generalized Anxiety Disorder Study met primary and all key secondary efficacy endpoints; Panorama is the second positive Phase 3 trial for DT120 ODT in GAD and the third positive Phase 3 trial...

Phase 2 MOONLIGHT-1 of JNJ-5120/PIPE-307 (J&J-partnered M1 antagonist) in MDD missed primary endpoint (MADRS change at Day 5 vs placebo); well tolerated, no new safety signals; J&J continuing to evaluate prespecified exploratory endpoints.

8-K filed (items 1.01: financing-related)

Corbus Pharmaceuticals Announces Positive Topline Data from CANYON-1 Study of Daily Oral CRB-913 for the Treatment of Obesity • CRB-913 demonstrated statistically significant and clinically meaningful mean weight loss of 5% at 60 mg after 12 weeks with no plateau observed • CRB-913 was generally...

Zipalertinib Plus Chemotherapy Demonstrates 6-Month Median Progression-Free Survival Benefit in REZILIENT3 Phase 3 Trial in First-Line EGFR Exon 20 Insertion Mutation Non-Small Cell Lung Cancer Results from the Phase 3 REZILIENT3 trial were featured in a Presidential Symposium at WCLC 2026 and...

Perspective Therapeutics Announces Clinical Collaboration and Supply Agreement with Merck to Evaluate [212Pb]PSV359 in Combination with Keytruda® (Pembrolizumab) in FAP-α Positive Solid Tumors SEATTLE, September 14, 2026 -- Perspective Therapeutics, Inc. (“Perspective,” the “Company,” “we,”...

First median OS data from non-pivotal stage 1 of PRESERVE-003 Phase 3 (NCT05671510) of gotistobart (BNT316/ONC-392) in previously treated squamous NSCLC, presented at WCLC 2026: mOS 18.5 vs 10.0 months vs docetaxel (HR 0.56, nominal p=0.0295, n=87, 25.4-mo median follow-up); safety consistent with prior data (grade >=3 TRAEs 44.4% vs 48.8%); pivotal stage 2 ongoing at >160 sites globally.

Shelf registration filed (enables future raises incl. ATM)

8-K filed (items 1.01: financing-related)

Offering prospectus filed (possible follow-on or ATM takedown)

Biofrontera Announces FDA Approval of Ameluz ® Red Light PDT for the Treatment of Superficial Basal Cell Carcinoma Ameluz used in conjunction with the RhodoLED lamp becomes the first and only photodynamic therapy approved in the United States to treat a skin cancer, creating an additional growth...

Shelf registration filed (enables future raises incl. ATM)

Armata Pharmaceuticals Receives U.S. FDA Breakthrough Therapy Designation for AP-SA02 Designation is driven by compelling Phase 2 clinical data in patients with complicated Staphylococcus aureus bacteremia showing AP-SA02 may offer substantial improvement over available therapies Potentially...

8-K filed (items 1.01, 3.02: financing-related)

Positive Phase 1 topline for AGMB-447 (inhaled lung-restricted ALK5 inhibitor) in IPF patients: generally favorable safety/tolerability, efficient lung restriction with low systemic exposure, robust ALK5 target engagement. CTA for INSPIRIA Phase 2 in IPF submitted; initiation intended before year-end 2026.

Final OS analysis of Phase 3 PAPILLON: first-line IV RYBREVANT (amivantamab) + chemo in EGFR exon 20 insertion NSCLC; median OS 34.3 vs 27.9 mo chemo alone (HR 0.87, 95% CI 0.66-1.14, p=0.307; 76% crossover to RYBREVANT in control arm); crossover-adjusted HR 0.57 (nominal p=0.003, 43% reduction in death risk); PFS2 28.3 vs 17.5 mo (HR 0.59, p<0.0001); longest reported mOS in this population (~2x historical median); presented at WCLC 2026 Presidential Symposium (NCT04538664).

Positive detailed Ph3 REZILIENT3 results for zipalertinib + chemo in 1L EGFR ex20ins NSCLC (n=279): mPFS 14.5 vs 8.5 mo (HR 0.50, 95% CI 0.34-0.73, p=0.00015), ORR 65.0% vs 40.3% (p<0.0001); interim OS HR 0.72 at 30% maturity; data featured in WCLC 2026 Presidential Symposium

Interim TACTI-004 root-cause findings: subtle structural differences (e.g. N-glycan) identified between 2,000 L-scale efti used in TACTI-004 and 200 L-scale efti used in earlier successful studies, potentially explaining the divergent immune-activation profile; clinical and trial-execution factors ruled out; new 200 L manufacturing run contracted; root-cause analysis ongoing with a further update promised. Development refocused on HNSCC (CPS<1) and neoadjuvant STS; next trial starts targeted 2H CY2027.

FDA extended the PDUFA action date for the zanzalintinib + atezolizumab (Tecentriq) NDA in previously treated metastatic colorectal cancer by 3 months to March 3, 2027, after classifying updated safety/efficacy data submitted in response to an FDA information request as a major amendment (disclosed via 8-K; original date Dec 3, 2026)

NOTICE OF PENDENCY AND PROPOSED SETTLEMENT OF DERIVATIVE MATTERS TO: ALL PERSONS OR ENTITIES WHO HOLD OR BENEFICIALLY OWN, DIRECTLY OR INDIRECTLY, ACADIA PHARMACEUTICALS, INC. (“ACADIA” OR THE “COMPANY”) COMMON STOCK AS OF JULY 9, 2026 (“CURRENT ACADIA STOCKHOLDERS”) PLEASE READ THIS NOTICE...

Positive topline from Phase 3 LUNA (M24-859, NCT06806293, n=468): atogepant for preventive treatment of menstrual migraine met the primary endpoint (reduction in migraine days during the perimenstrual period, averaged across 3 menstrual cycles) and all 8 ranked secondary endpoints vs placebo (p<0.0001 across the board); average reduction 1.20 days on atogepant vs 0.40 on placebo. Safety consistent with atogepant's known profile, no new signals. First positive Phase 3 of a CGRP-targeted treatment specifically in menstrual migraine; AbbVie plans submissions to health authorities worldwide.

Quoin Pharmaceuticals Announces FDA Grants Rare Pediatric Disease Designation for QRX003 in Peeling Skin Syndrome - Second Rare Pediatric Disease (RPD) Designation for QRX003 - FDA Previously Granted RPD Designation for QRX003 in Netherton Syndrome - If a New Drug Application (NDA) for QRX003 Is...

Prelude Therapeutics Receives FDA Clearance of Investigational New Drug (IND) Application for PRT13722, Its First-in-Class Oral KAT6A Selective Degrader Phase 1 study of PRT13722 in patients with HR+ breast cancer anticipated to begin enrolling patients in the fourth quarter of 2026. WILMINGTON,...

Published abridged final Phase 2 CSR for SKNJCT-003 (NCT06608238) SkinJect in nodular BCC (n=90, randomized double-blind, device-only microneedle comparator): selected 200 mcg D-MNA patch as the proposed pivotal dose; longer follow-up showed continued improvement in clinical and histological clearance. FDA issued Study May Proceed letter for NDA-enabling registrational Phase 2b SKNJCT-005 in Gorlin syndrome (up to ~50 pts); ODD and RPD designation applications submitted (potential PRV).

Disc Medicine Presents Initial Results from RESTORE-PV Phase 2 Trial in Patients with Polycythemia Vera (PV) at the 14 th Society of Hematologic Oncology (SOHO) Annual Meeting • Initial results from RESTORE-PV show DISC-3405 increased hepcidin and lowered serum iron, translating to controlled...

FDA imposed a partial clinical hold on BHV-7000 (opakalim), the selective Kv7.2/7.3 potassium-channel activator in pivotal testing for focal epilepsy, after rodent studies found BHV-7000 metabolites of unclear human risk; new-patient enrollment is paused while already-enrolled participants continue treatment. One of the two pivotal studies is fully enrolled and Biohaven still expects topline data this year. The hold landed days after SK Biopharmaceuticals agreed to license BHV-7000 for $350M upfront (+$50M next year, up to $150M in milestones).

Pyxis Oncology Announces Positive Updated Data from Phase 1 Monotherapy Study of Micvotabart Pelidotin (MICVO) in Second-Line and Beyond Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma (2L+ R/M HNSCC) MICVO demonstrated rapid and deep responses, with a 36% confirmed objective response...

Lexaria Announces Exercise of Warrants for Approximately $5.9 Million in Gross Proceeds Tuesday, 08 September 2026 08:00 AM Topic: Financing KELOWNA, BC / ACCESS Newswire / September 8, 2026 / Lexaria Bioscience Corp. (NASDAQ:LEXX)(NASDAQ:LEXXW) (the "Company" or "Lexaria"), a global innovator...

Opus Genetics Announces Positive Low Dose Cohort 1 Data from Phase 1/2 Clinical Trial of OPGx-BEST1 and Successful FDA Type C Meeting with Potential Phase 3 Dosing in 2027 OPGx-BEST1 demonstrated a favorable safety and tolerability profile with no serious adverse events or dose-limiting...

Evommune Announces Top-line Results from EVO756 Phase 2b Trial in Moderate-to-Severe Atopic Dermatitis ‑ Trial did not meet the primary endpoint of percent change in Eczema Area and Severity Index (EASI) score from baseline at Week 12; EVO756 was generally well tolerated ‑ EVO301, the Company’s...

Cocrystal Pharma Announces Last Subject Dosed in Phase 1b Human Challenge Study Evaluating CDI-988 for Norovirus Prevention and Treatment No serious adverse events reported to date Preliminary efficacy results expected in late 2026 or early 2027 MIAMI, Fla. And BOTHELL, Wash., September 09, 2026...

Positive topline 52-week open-label Part C data from Phase 3 COMP005 trial of COMP360 (synthetic psilocybin) in TRD (n=258): additional 25 mg dose extended durability to 1 year, avg 13-point MADRS reduction from baseline at Week 52, 40-45% responders / ~30% remitters at 6 weeks post Part C dose, no new safety findings; rolling NDA submission on track for Q4 2026 completion, commercial launch expected H1 2027

Aspire-Lakewood Holdings Board Approves $10 Million Share Repurchase Program Brings total authorized repurchase to $10 million Reflects leadership’s view that Aspire-Lakewood’s share price does not reflect the Company’s financial strength or long-term growth opportunity Increased authorization...

Positive outcome from FDA Type C meeting for global Phase 3 4WARD trial of mavorixafor in chronic neutropenia (NCT06056297): FDA agreed to reduce sample size 176 -> 126 participants while maintaining power for co-primary endpoints (annualized infection rate reduction + positive ANC response); safety database acceptable to support sNDA; enrollment completion expected by year-end 2026, topline in H1 2028

Investor and Media Contact: Samuel Fisch Executive Director, Investor Relations and Corporate Communications Email: [email protected] Veru Advances Oral Sabizabulin Following Positive Preclinical Data Showing Potent Anticancer Activity in Human Daraxonrasib (Revolution Medicines’...

Roivant Announces Positive Results from PHocus Study of Mosliciguat in Patients with Pulmonary Hypertension Associated with Interstitial Lung Disease (PH-ILD) and Unveils Ongoing Phase 3 PHrontier Study • PHocus met its primary endpoint, demonstrating a clinically meaningful and statistically...

Positive topline from pivotal Phase 3 CHAPTER-3 (NCT06669754) of deucrictibant XR 40mg QD for HAE prophylaxis (n=85, 55 XR/30 pbo, 24 wks): primary endpoint met, 83% attack-rate reduction vs placebo (p<0.0001) across all three HAE types, 87% in type 1/2; all secondary endpoints met with statistical significance; well tolerated. First and only prophylaxis Ph3 study in all three HAE types; MAAs planned from 1H2027.

Announced interim 3-year overall survival analysis from the Phase 2b trial in fully resected pulmonary metastatic osteosarcoma: 71.2% 3-year OS in OST-HER2-treated patients vs 45.8% in a comparable combined historical control (p=0.002; 41 patients enrolled, 6 lost to follow-up; two patients not yet at the 3-year timepoint). No new deaths since the 2.5-year analysis.

Phase 3 HARBOR trial of delpacibart etedesiran (del-desiran) in myotonic dystrophy type 1 missed primary vHOT endpoint (hand myotonia); ~150 patients (NCT06411288); activity seen in secondaries/exploratory endpoints

Intellia Therapeutics Announces FDA Acceptance of Biologics License Application with Priority Review for Lonvoguran Ziclumeran (Lonvo-z) for Hereditary Angioedema (HAE) • FDA sets Prescription Drug User Fee Act (PDUFA) date of March 10, 2027 • Positions lonvo-z to be the world’s first in vivo...

Positive primary endpoint results from the randomized controlled Phase 2 portion of the HexAgon study: INBRX-106 (hexavalent OX40 agonist) + pembrolizumab delivered cORR 48.3% vs 26.5% for pembrolizumab alone in 1L PD-L1+ (CPS>=20) HNSCC (n=63 evaluable), interim median PFS 9.6 vs 4.9 months; Ph2 expansion of ~50 HPV+ patients planned as potential accelerated-approval path.

Structure Therapeutics Reports Positive Clinical Data Across Oral Small Molecule Amylin and GLP-1 Programs for Chronic Weight Management ACCG-2671 (oral small molecule amylin receptor agonist) demonstrated a ~6-day half-life, no serious adverse events, and evidence of target engagement including...

Monte Rosa Therapeutics Announces Milestone Achievement Associated with Initiation of Phase 2 Clinical Study for VAV1-directed Molecular Glue Degrader MRT-6160 (DDY391) Phase 2 study to be conducted in participants with Sjögren’s disease Novartis conducting study under global exclusive...

Definium Therapeutics Receives Breakthrough Therapy Designation for DT120 (lysergide) Orally Disintegrating Tablet (ODT) in Major Depressive Disorder (MDD) Following Positive Phase 3 Results Second FDA Breakthrough Therapy designation for DT120 reinforces its potential across MDD and generalized...

Positive topline from registrational Phase 2 QUINTESSENTIAL (NCT06297226) of arlocabtagene autoleucel (arlo-cel, GPRC5D-directed CAR T) in quadruple-class exposed RRMM post prior BCMA therapy: met primary endpoint (statistically significant and clinically meaningful ORR at >=4 prior lines) and key secondary CRR; safety consistent with CAR T / GPRC5D class. Results to be presented at upcoming medical meeting.

BBOT Announces New BBO-8520 Data; Highlights Strategic Focus on 2L+ NSCLC BBO-8520 Combination as Well as BBO-11818 and BBO-10203 Combinations in KRAS-Mutant Cancers • BBO-8520 plus pembrolizumab demonstrated a 75% ORR at 500 mg QD and 53% ORR across dose levels in 2L+ KRAS G12C...

Azitra, Inc. to Present at the H.C. Wainwright 28 th Annual Global Investment Conference BRANFORD, Conn. – September 8, 2026 — Azitra, Inc. (“Azitra” or the “Company”) (NYSE American: AZTR), a clinical-stage biopharmaceutical company focused on developing innovative therapies for precision...

Full results from replicate Phase 3 OBERON (NCT05166889) and TITANIA (NCT05158387) of tozorakimab (anti-IL-33) 300mg Q4W in symptomatic COPD: met the primary endpoint with statistically significant reductions in annualized moderate/severe exacerbations vs placebo on inhaled standard of care -- 29% (OBERON) and 34% (TITANIA) in former smokers, 30% and 29% in the overall population; first biologic to show efficacy across blood eosinophil thresholds (23% below 150, 34% >=150, 43% >=300 cells/uL). Presented as ERS 2026 late-breaker and published in NEJM. BLA accepted for FDA Priority Review as add-on COPD maintenance, PDUFA anticipated Q1 2027.

ASPIRE BIOPHARMA HOLDINGS, INC. ANNOUNCES CORPORATE NAME CHANGE TO ASPIRE-LAKEWOOD HOLDINGS, INC. ESTERO, FL / September 8, 2026 Aspire Biopharma Holdings, Inc. (Nasdaq: ASBP) (“Aspire” or the “Company”), today announced that it has changed its corporate name from “Aspire Biopharma Holdings,...

Phase 3 Lp(a)HORIZON trial of pelacarsen (Lp(a)-lowering ASO, Ionis-licensed) missed primary MACE endpoint despite lowering Lp(a); 8,323 patients

Ionis partner Novartis announces Lp(a)HORIZON Phase 3 topline results for pelacarsen in patients with elevated Lp(a) and established cardiovascular disease (CVD) CARLSBAD, Calif., September 4, 2026 -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) partner Novartis today announced that the pelacarsen...

FDA accelerated approval of ETCAMAH (camizestrant) + CDK4/6 inhibitor for HR+/HER2- advanced breast cancer upon ESR1 mutation detection during AI + CDK4/6 inhibitor therapy; based on Ph3 SERENA-6 (56% reduction in risk of progression/death vs AI + CDK4/6, ASCO 2025/NEJM); first FDA approval guided by ctDNA resistance-mutation detection before radiographic progression; Guardant360 CDx companion diagnostic authorized

Explanatory Note Teva Pharmaceutical Industries Limited (“Teva” or the “Company”) is filing this Exhibit 99.1 to recast historical segment information as set forth in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the Securities and Exchange...

China NMPA approval of etripamil nasal spray for adult PSVT (marketed in US as CARDAMYST): first regulatory approval of etripamil outside the US; triggers $2.0M milestone payment from Everest Medicines (exclusive Greater China licensee; up to $98M more milestones + tiered royalties to 18%); based on RAPID and China JX02002 data

Milestone Pharmaceuticals Announces NMPA Approval of Etripamil Nasal Spray for PSVT in China, Marking First Regulatory Approval of Etripamil Outside of the United States Everest Medicines to Lead Commercialization in Greater China, Bringing Novel Innovation to Estimated 3-6 Million Chinese...

FDA Grants Fast Track Designation to YUTREPIA® for the Treatment of Raynaud's Phenomenon Associated with Systemic Sclerosis · Fast Track designation underscores the significant unmet need for patients with systemic sclerosis-associated Raynaud's phenomenon (SSc-RP), a debilitating manifestation...

FDA approved ZANVASTRO (zilganersen) for Alexander disease in pediatric and adult patients — first and only disease-modifying treatment for AxD; RNA-targeted ASO, 50mg quarterly intrathecal injection; pivotal study showed 33.3% LS mean difference in gait-speed stabilization vs control at Wk61; Rare Pediatric Disease Priority Review Voucher awarded; Ionis' first independent neurology launch and third wholly-owned marketed drug.

bioAffinity Technologies to Participate in the H.C. Wainwright 28 th Annual Global Investment Conference SAN ANTONIO, Texas – September 3, 2026 – bioAffinity Technologies, Inc . (Nasdaq: BIAF; BIAFW) , a biotechnology company focused on noninvasive diagnostics for the early detection of lung...

Aptevo Reports 93% Clinical Benefit Rate with Mipletamig Triplet in Difficult-to-Treat TP53-Mutated Frontline AML Remission results nearly double published ven/aza outcomes in patients with historically limited treatment success SEATTLE, WA – September 3, 2026 – Aptevo Therapeutics Inc. (Nasdaq:...

Positive Phase 3 CERVINO topline for etentamig (BCMA x CD3 T-cell engager) in triple-class exposed relapsed/refractory multiple myeloma: ORR 74.0% vs 45.7% (p<0.0001), PFS HR 0.40 (p<0.0001); 393 patients, median follow-up 11.4 months; 12-month OS 87.9% vs 72.0%

AbbVie Completes Acquisition of Apogee Therapeutics – Acquisition deepens AbbVie's robust immunology pipeline with diverse assets targeting dermatologic, respiratory and other inflammatory and immunological diseases – Apogee's lead asset, zumilokibart, is a late-stage, half-life extended...

Positive Phase 2a topline for TEV-408 (anti-IL-15 mAb) in celiac disease (n=50, gluten challenge): met primary endpoint, Vh:Cd LS mean change -0.43 vs -0.88 placebo (treatment diff 0.45, p<0.05); favorable IEL density effect; lower GI symptom scores; well tolerated, no safety signals.

Phase 3 Aspire trial of apazunersen (GTX-102) in Angelman syndrome missed primary endpoint (change from baseline in Bayley-4 cognitive raw score) and key secondary endpoint (net response in Multidomain Responder Index); company evaluating program disposition and expense reductions

BLA submitted to FDA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130), one-time gene therapy for Huntington's disease; priority review requested (6-mo review after 60-day filing period). MAA also submitted to UK MHRA. Both supported by 3-year Phase 1/2 data showing 75% slowing of disease progression (cUHDRS vs Enroll-HD external control, p=0.003).

DATED SEPTEMBER 1, 2026 Exhibit 99.1 NorthStrive Biosciences Expands AI-Driven Drug Discovery Program with Yuva Biosciences, Advancing Four AI-Discovered Compounds Toward Lead Nomination in Cardiac Disease and Obesity Pursuant to a First Amendment to the Development and License Agreement,...

Upstream Bio Announces Pivotal Phase 3 Development Plans for Verekitug in Both Severe Asthma and CRSwNP – Following productive FDA discussions, Upstream Bio will study 400mg of verekitug administered every 12 weeks, versus placebo, in severe asthma and CRSwNP with one pivotal Phase 3 trial in...

Positive Phase 3 REMODEL-1 and REMODEL-2 topline for remibrutinib (oral BTK inhibitor) in relapsing multiple sclerosis: both trials beat teriflunomide on annualized relapse rate and all key secondary MRI endpoints; ~2,000 patients combined; no Hy's Law liver signal; late-breaker at MSToronto2026, global filings planned

MIRA Pharmaceuticals Advances Non-Opioid Oral MIRA-55 for Inflammatory Pain Toward IND Positive Toxicology Results From 7-Day Rat and Dog Studies Characterize Tolerability and Inform Dose Selection for Next Phase of IND-Enabling Program, Targeting Q1 2027 IND Submission MIAMI, Sept. 1, 2026...

Alumis Announces Topline Results from Envudeucitinib Phase 2b Trial in Systemic Lupus Erythematosus (SLE) – LUMUS trial did not meet its primary and secondary endpoints in the overall trial population – – Robust responses in the prespecified subgroup of patients with high interferon gene...

Adicet Bio Announces Completion of Last Patient Visit for Planned Analysis of prula-cel (formerly ADI-001) Study in Patients with Systemic Lupus Erythematosus With or Without Lupus Nephritis and Provides Corporate Update Prula-cel topline results from planned analysis expected September 2026 FDA...

FDA approved MIMRYLO (rusfertide), first-in-class hepcidin mimetic, for erythrocytosis in adults with polycythemia vera (licensed to Takeda); Phase 3 VERIFY (n=293): 76.9% response (phlebotomy-free, weeks 20-32) vs 32.9% placebo. Approval triggers $275M in milestones to PTGX ($200M opt-out fee + $75M approval milestone) + tiered royalties 14-29% + up to $875M in future milestones. NCCN Category 1 designation across PV risk categories followed Sep 18.

Terminated Phase 2 BNT122-01 trial (NCT04486378) of autogene cevumeran (BNT122) as adjuvant monotherapy in ctDNA-positive, surgically resected high-risk Stage II/III colorectal cancer after DSMB identified numerical imbalance in overall survival (more deaths in vaccine arm, per Genentech spokesperson to Fierce Biotech) and concluded continuation unlikely to change efficacy outcome; no new safety signals; futility boundary had been crossed Oct 2025; jointly developed with Genentech, termination decided in consultation with them; BNT122 development continues in combination settings (e.g. pancreatic cancer)

FDA approved LISRAYA (brepocitinib) 30 mg, once-daily oral TYK2/JAK1 inhibitor, for adults with dermatomyositis — first targeted therapy approved for DM (off Ph3 VALOR; Priority Review + Orphan Drug Designation); available in the US immediately

FDA approved RASONQUE (daraxonrasib), first broad RAS-targeted medicine, for adults with metastatic pancreatic adenocarcinoma with at least one prior systemic therapy or not candidates for multiagent systemic therapy. Approval based on Phase 3 RASolute 302: mOS 13.2 vs 6.7 months vs chemotherapy, risk of death reduced 60%, significant improvements across all primary and key secondary endpoints; once-daily oral tablets; WAC $39,800 per 30-day supply.

Phase 3 HARMONi-GI1 topline positive (announced by Akeso): ivonescimab (SMT112) + chemo beat durvalumab + chemo in first-line advanced biliary tract cancer (BTC) — statistically significant and clinically meaningful OS superiority at pre-specified interim analysis; key secondary PFS and ORR also met. Single-region China study sponsored by Akeso; ivonescimab approved in China for NSCLC, investigational in Summit territories. Summit confirmed ESMO 2026 Presidential Symposium II presentation (Oct 25).

Paused 8 autoimmune/neurology studies of rapcabtagene autoleucel (rap-cel) CD19 CAR-T after 3 deaths from immune effector cell-associated hemophagocytic lymphohistiocytosis-like syndrome (IEC-HS); screening/randomization/dosing suspended; oncology CAR-T studies unaffected

2026 Animal Study #2 (GLP-1-A26-2) dog PK complete (18 arms, oral amycretin + retatrutide, DehydraTECH 3.0 vs 2.0): best amycretin tablet reached 1.65% absolute bioavailability (+481% vs DHT2.0 reference tablet); best retatrutide tablet improved absorption +4,552% vs DHT2.0 reference tablet (0.10% absolute bioavailability; DHT3.0 capsules 0.26%). DHT3.0 outperformed with sodium caprate vs SNAC for both drugs.

FDA approved PASATRU (garetosmab-grts) for adults with fibrodysplasia ossificans progressiva (FOP): first FDA-approved treatment shown in a placebo-controlled trial to reduce new heterotopic ossification (HO) lesions + clinician-assessed flare-ups (Ph3 OPTIMA n=63: 90%/94% fewer new HO lesions at 56 wks for 10/3 mg/kg vs placebo; 88% fewer flare-ups at 10 mg/kg); monthly IV 10 mg/kg; EU submission under review

FDA accelerated approval of GENGLYCOS (pariglasgene brecaparvovec-opnr) gene therapy for glycogen storage disease type Ia in patients age 8+; Phase 3 GlucoGene showed reduced cornstarch requirements vs placebo (p<0.001); company's first gene-therapy approval

Merck and Moderna announced positive topline results from Phase 3 INTerpath-001 of intismeran autogene (mRNA-4157/V940, individualized neoantigen therapy) + KEYTRUDA as adjuvant therapy in completely resected stage IIB-IV melanoma: met primary endpoint of recurrence-free survival (RFS) and key secondary distant metastasis-free survival (DMFS) vs KEYTRUDA alone at pre-specified interim analysis; first positive Phase 3 readout for an individualized neoantigen therapy. Safety consistent, no new signals; trial continues for overall survival; data to be presented at an upcoming medical meeting.

Positive Phase 3 LUCIDITY topline for avexitide (first-in-class GLP-1 receptor antagonist, Breakthrough Therapy Designation) in post-bariatric hypoglycemia (n=78 post-RYGB, 16 weeks): met the primary endpoint (FDA-agreed) with 55% reduction in composite rate of Level 2/3 hypoglycemic events vs placebo (p=0.000003); all secondary endpoints met (SMBG Level 2, CGM Level 2, adjudicated Level 3); generally well-tolerated, no treatment-related SAEs; NDA submission planned by end of 2026; would be first FDA-approved therapy for PBH.

AstraZeneca discontinuing Phase 3 eVOLVE-Lung02 trial (NCT05984277) of volrustomig (MEDI5752, PD-1xCTLA-4 bispecific) + chemotherapy vs pembrolizumab + chemotherapy as 1L treatment in mNSCLC with PD-L1 <50% (n=895) after IDMC concluded the regimen was unlikely to meet either dual primary endpoint (PFS, OS in PD-L1-negative <1% population). No new safety signals. Other volrustomig Phase 3 trials (cervical, HNSCC, mesothelioma) continue as planned.

Positive Phase 3 ALKIVIA topline for VYVGART Hytrulo (efgartigimod) in autoimmune myositis: met primary endpoint of mean Total Improvement Score at Week 52 in combined IMNM+DM population (15.4-point advantage vs placebo, p=0.0011); first Phase 3 to show significant improvement in IMNM. DM subgroup +14.5 points but p=0.1093 (not significant).

FDA accepted the Biologics License Application (BLA) for obexelimab for the treatment of IgG4-related disease (IgG4-RD); PDUFA target action date of May 27, 2027. Announced in the Q2 2026 results release.

Positive Ph3 Voyage topline for DT120 (lysergide) ODT in GAD (single 100 ug dose): HAM-A -5.4 placebo-adjusted at Wk12 (p<0.0001, d=0.81), met primary + all key secondaries; rapid onset by Day 2; 2nd positive Ph3 for DT120 after Emerge (MDD, Jun 22); Panorama GAD Ph3 topline followed Sep 14

Strategic refocus: PDSB will discontinue the PDS0101 Phase 3 VERSATILE-003 trial and cease internal investment in PDS0101 (pursuing strategic partnerships/external funding instead), prioritizing PDS0301 tumor-targeted IL-12 immunocytokine. NCI-collab Phase 2 in metastatic MSS/pMMR CRC with liver mets showed 71% ORR at 6 months and 80% 24-month survival (>380 patients treated; benchmarks mOS <10 mo, ORR <25%). Randomized Phase 2b mCRC program designed with FDA feedback. (Letter to shareholders, GlobeNewswire Aug 11)

Positive topline human-factors validation + PK study results for Anaphylm (dibutepinephrine) sublingual film: revised packaging addressed all CRL deficiencies (pouch-open failures 26/166 -> 1/105, median open time 17s -> 3s); top-of-tongue PK arm met endpoints with clinically meaningful PD; supports planned NDA resubmission <8 months from Jan 2026 CRL

Positive Phase 2 topline (Phase 1/2 portion, NCT07118891) for ABCL635, NK3R antagonist antibody for moderate-to-severe vasomotor symptoms of menopause: single 600mg SC dose, n=92, met primary endpoints at Week 4 — 83% reduction in hot-flash frequency vs 33% placebo (placebo-adjusted -5.3 events/day, p<0.001) and 58% severity reduction vs 12% placebo; significant from Week 1 through Week 4; sleep and patient global impression improved; no discontinuations due to adverse events.

FDA accelerated approval of TUDRIQEV (oncolytic immunotherapy, formerly RP1) plus nivolumab for unresectable advanced cutaneous melanoma after anti-PD-1 progression; IGNYTE (n=91): ORR 24.2%, median duration of response 14.1 months

Positive Phase 2 RECLAIM topline for pemvidutide in moderate-to-severe alcohol use disorder: heavy drinking days/week -4.20 vs -2.75 placebo (treatment effect -1.45, p=0.0014); 2-level WHO risk reduction 64.4% vs 34.8% (p=0.0049); zero heavy-drinking days 42.2% vs 17.4% (p=0.0066)

FDA granted traditional approval to Retevmo (selpercatinib) for locally advanced/metastatic RET fusion-positive solid tumors (adults and pediatric 2+, post-systemic therapy or no satisfactory alternative), converting the 2022/2024 accelerated approvals; off LIBRETTO-001 (ORR 47%, mDOR 24.5 mo) + LIBRETTO-121

Positive 36-week topline results from Part B of the SIGNAL-AA Phase 2a trial of bempikibart (anti-IL-7Rα) in severe/very severe alopecia areata (n=33, SALT 50-100): mean SALT reduction 35.3% (mITT); SALT-20 response 40.0% mITT (10/25) / 30.3% ITT (10/33); SALT30 44.0%/33.3%, SALT50 44.0%/33.3%; no SAEs, no Grade 3+ related AEs, ISR most common TEAE (36.3%, all mild); registration-directed program planned H1 2027

CARDIO-TTRansform Phase 3 of eplontersen (Wainua) in ATTR-CM (n=1,432) did not meet the primary endpoint (composite CV mortality + recurrent CV events through week 140; rate ratio 0.89, 95% CI 0.73-1.09, p=0.277); prespecified monotherapy subgroup (no baseline stabilizer) nominally significant (HR 0.71), no treatment effect in baseline-stabilizer users; well tolerated; full data at ESC Aug 2026

Positive Ph3 Emerge topline for DT120 (lysergide) ODT in MDD (n=149, single 100 ug dose): MADRS -8.1 placebo-adjusted at Wk6 (p<0.0001), met primary + all key secondaries; rapid onset (Wk1 -14.2) durable to Wk12 (-7.3); response 35% vs 7%, remission 24% vs 3%; no suicidality signal; 1st positive Ph3 for DT120; 2nd MDD Ph3 (Ascend) ongoing

Discontinuation of Phase 3 KEYNOTE-D46/EVOKE-03 (NCT05609968): Trodelvy + Keytruda vs Keytruda monotherapy in previously untreated PD-L1 TPS >=50% metastatic NSCLC, on external Data Monitoring Committee recommendation; PFS final analysis showed numerical improvement but not statistically significant, statistically significant OS at final analysis judged unlikely; no new safety signals; setback for Trodelvy expansion into first-line lung cancer

Bristol Myers Squibb discontinued development of ORM-6151, the GSPT1-targeting degrader-antibody conjugate acquired from Orum Therapeutics in 2023 for $100M upfront (+$80M milestones), after reviewing Phase 1 data in AML/MDS (announced via Orum regulatory filing Sep 17; no reversion rights to Orum, upfront retained).

Positive Ph3 ACACIA-HCM: met dual primary endpoints; KCCQ-CSS +3.0 pts vs placebo (p=0.021), pVO2 +0.67 mL/kg/min (p=0.003) at Wk36; key secondaries favored aficamten.

Positive topline from global Phase 3 HAELO trial of lonvo-z (lonvoguran ziclumeran, one-time in vivo CRISPR editing of KLKB1) in hereditary angioedema (n=80): 87% reduction in mean monthly HAE attacks vs placebo (0.26 vs 2.10, weeks 5-28, p<0.0001); 62% of patients entirely attack-free and therapy-free vs 11% placebo (p<0.0001); all key secondaries met (on-demand attack rate -89%, moderate/severe -91%); no serious adverse events; first-ever Phase 3 data for an in vivo gene editing therapy; rolling BLA initiated April 2026 (BLA accepted with Priority Review Sep 2026)

FDA issued a Complete Response Letter for the TrenibotE BLA; CRL relates to manufacturing processes only — no safety or efficacy concerns identified, and no additional clinical studies requested. AbbVie expects to submit its response in the coming months. TrenibotE is a first-in-class botulinum neurotoxin serotype E (rapid onset ~8 hours, duration 2-3 weeks) for moderate-to-severe glabellar lines; program supported by two pivotal Phase 3 studies plus an open-label safety study in 2,100+ patients.

FDA removed the partial clinical hold on the Phase 2 LINNET study of lorigerlimab; new enrollment resumes under a revised protocol with added risk-mitigation for hematologic and cardiac toxicities; mid-2026 clinical update still guided.

FDA placed a partial clinical hold on the Phase 2 LINNET study of lorigerlimab (PD-1 x CTLA-4 bispecific DART) in platinum-resistant ovarian and clear-cell gynecologic cancers after 4 serious safety events incl. 1 treatment-related death (Gr4 neutropenia + septic shock); new enrollment paused, current participants continue.