Every upcoming biotech catalyst in one place: PDUFA dates, trial readouts, and regulatory filings across 268 tickers. For the 90-day tactical view, see the Readout Radar. Provisional calendar, staged Oct 31, 2026 from the 9.8.26 event sheet — timings update as new information lands.
2026ANNXAnnexon Biosciences ANX1502 — Cold Agglutinin Disease (CAD) Phase 1
Proof-of-concept update 2026→ readout Fall’26.
2026ARWRArrowhead Pharmaceuticals ARO-CFB — Complement Mediated Disease Phase 1
Initial Phase 1 data expected 2025, data 2026?.
2026BBOTBridgeBio Oncology Therapeutics BBO-11818 - mono & combos incl. +pembro or +chemo or +cetuximab or +BBO-10203 — KRAS-mutant solid tumors (KONQUER-101) Phase 1
Monotherapy dose-escalation. Ongoing, cetuximab combo first patient in H1’26, 10203 combo first patient in Jul-Aug 2026, Dec 2025 data cut-off: initial safety data and efficacy anecdote, expansion and combo cohorts planned for 2026,…
Monotherapy dose-escalation. Ongoing, cetuximab combo first patient in H1’26, 10203 combo first patient in Jul-Aug 2026, Dec 2025 data cut-off: initial safety data and efficacy anecdote, expansion and combo cohorts planned for 2026, additional monotherapy/combo efficacy and safety data H2’26.
Trastuzumab pamirtecan (BNT323, DB-1303) — Multiple solid tumors Phase 1/2
Pumitamig combo data 2026.
Elfetabart drozuntecan (BNT324, DB-1311) — Multiple solid tumors Phase 1/2
2L+ mCRPC data at ASCOGU’26 including post-RLT activity, pumitamig combo data 2026.
BNT325 (DB-1305) — Multiple solid tumors Phase 1/2
Pumitamig combo data in TNBC 2026.
Gotistobart (BNT316, ONC-392) — 2L aPD-(L)1-R/R metastatic SQ NSCLC (PRESERVE-003) Phase 2/3
Positive phase Dec 2, 2025. Stage /phase 2 OS HR 0.46, phase 3 IA1 2026 [restricted to SQ, unfav. Risk/reward in NSQ→ discont.], longer OS follow-up data at WCLC’26.
2026COGTCogent Biosciences CGT4859 — FGFR2/3-altered solid tumors, cholangiocarcinoma Phase 1/2
Phase 1 data 2026, primary completion H1’27.
2026CRBUCaribou Biosciences Vispa-cel (CB-010, vispacabtagene regedleucel) — R/R B-cell NHL (ANTLER) Phase 1
Long-term follow-up 2026 (n=84 dosed) for the allogeneic CAR-T vispa-cel, using partial HLA matching for persistence and young donors for potency. November 2025: 2L LBCL with 4+ HLA matches at 80M cells (n=22), ORR 82%/CR 64%; 2–3L LBCL…
Long-term follow-up 2026 (n=84 dosed) for the allogeneic CAR-T vispa-cel, using partial HLA matching for persistence and young donors for potency. November 2025: 2L LBCL with 4+ HLA matches at 80M cells (n=22), ORR 82%/CR 64%; 2–3L LBCL with 2+ matches at 40–120M cells (n=35), ORR 86%/CR 63%. EHA 2026 optimized product (donors under 30, ≥2 HLA matches, n=27 2L LBCL): ORR 82%/CR 67%, median PFS 17.1 months; grade 3+ CRS 4%, infections 22%, prolonged cytopenias 21%, IEC-HS 4%, with one fatal IEC-HS and one possibly-related PML death. Ten batches estimated to cover 99% of patients with 2+ HLA matches.
2026IDYAIDEAYA Biosciences IDE397 + TRODELVY — Bladder Cancer, Lung Cancer (MTAP deletion) Phase 1/2
Combo deprioritized [bladder data prev. Due 2026], trial terminated, potential additional combos with other TopoI ADCs including IDE034 in the future.
IM-1021 — R/R solid tumors & B-cell lymphoma (ROR1+) Phase 1
Initial Phase 1 data expected 2026→ Aug 2026 update: “objective responses observed in B-cell lymphoma at multiple dose levels” → initial lymphoma data 2026 [so likely at ASH’26].
Anzu-cel (IMA203) — Uveal melanoma Phase 1/2
Phase 1/2 data expected update 2026.
IMA203CD8 — Gynecologic cancers, other solid cancers Phase 1
Dose-escalation phase 1a data at ASCO’26 at relevant doses → ORR 63%; cORR 50% in gyn cancers and ORR 67% and cORR 64% in synovial sarcoma, dose-escalation completed mid-2026, RP2D 2026, updated phase 1 data across multiple PRAME+ solids…
Dose-escalation phase 1a data at ASCO’26 at relevant doses → ORR 63%; cORR 50% in gyn cancers and ORR 67% and cORR 64% in synovial sarcoma, dose-escalation completed mid-2026, RP2D 2026, updated phase 1 data across multiple PRAME+ solids at ESMO’26.
2026IONSIonis Pharmaceuticals ION717 — Prion disease Phase 2
Phase 2 data readout expected 2026.
2026KYTXKyverna Therapeutics KYV-101 (miv-cel, mivocabtagene autoleucel) — Lupus nephritis (KYSA-1 & KYSA-3) Phase 1/2
Phase 1 data 2026, indication deprioritized H1’26 to focus on SPS/gMG/PMS.
2026LXEOLexeo Therapeutics LX2020 — Arrhythmogenic cardiomyopathy (PKP2-ACM) – (HEROIC-PKP2) Phase 1/2
HEROIC-PKP2 gene therapy data (n=10; 3 at low dose 2e13 vg/kg, 7 at high dose 6e13 vg/kg): NSVTs and PVCs reduced or stabilized in the majority, including 22% mean NSVT improvement and 14% mean PVC improvement in 5 high-dose patients at…
HEROIC-PKP2 gene therapy data (n=10; 3 at low dose 2e13 vg/kg, 7 at high dose 6e13 vg/kg): NSVTs and PVCs reduced or stabilized in the majority, including 22% mean NSVT improvement and 14% mean PVC improvement in 5 high-dose patients at latest visit; PGIC improved in 80%. Biopsy results pending (n=2); 12-month data for all high-dose patients expected Q4’26 alongside regulatory engagement.
mRNA-4359 — Advanced solid tumors Phase 2
Phase 2 data readout expected potential 2026.
mRNA-3927 — Propionic acidemia (PA) Phase 2
Registrational, readout potential 2026.
2026NRIXNurix Therapeutics GS-6791 (NX-0479) — RA, inflammatory diseases Phase 1
First patient in H1’26 but no record yet on ct.gov, potential results 2026, results to inform Nurix’s option for 50/50 co-dev and US profit share.
2026RVMDRevolution Medicines Zoldonrasib (RMC-9805) — KRAS G12D advanced solid tumors (incl. NSCLC & PDAC) Phase 1
Zoldon + daraxon data 2026.
2026SLNSilence Therapeutics Zerlasiran (SLN360) — Elevated lipoprotein(a) Phase 2
Phase 3 pending partner, expecting readthrough from Novartis 1st CVOT phase 3 readout of Lpa agent planned for 2026.
2026VNDAVanda Pharmaceuticals Tasilmelteon (HETLIOZ) — Delayed Sleep Phase Disorder Phase 3
Phase 3 topline readout expected 2026.
ABS-201 — Androgenetic alopecia (HEADLINE) Phase 1/2
First patient in Dec 2025, phase 1 SAD part readout Jun 2026: good safety and t1/2 ~65d+ supporting Q2M-Q3M dosing, phase 2 part 13-week readout H2’26 and 26-week readout early 2027.
H2’26ACADACADIA Pharmaceuticals Trofinetide (DAYBUE, DAYBU) — Rett syndrome Marketed
US, negative CHMP end-Feb 2026 in EU; re-exam sought → positive CHMP end-Jun 2026 → EC approval Aug 2026, JP phase 3 readout H2’26.
H2’26ACTUActuate Therapeutics Elraglusib + RASi — metastatic PDAC Preclinical
In vitro proof-of-concept using tumor models of RAS resistance, in vivo proof-of-concept and translational biomarker analysis, initial PC data Q2’26→ mid-2026, additional data H2’26.
H2’26ACTUActuate Therapeutics Elraglusib + mFOLFIRINOX + retifanlimab — 1L metastatic PDAC (RiLEY) Phase 1
Primary completion expected H2’26.
H2’26AGIOAgios Pharmaceuticals AG-181 — Phenylketonuria Phase 1
First patient in March 2026, proof-of-concept data H2’26, primary completion 2028.
Olanzapine + samidorphan (LIBALVI) — Schizophrenia, Bipolar I disorder (pediatric) - (ENLIGHTEN-Youth) Phase 3
Primary completion expected H2’26.
H2’26ALNYAlnylam Pharmaceuticals ALN-6400 — Bleeding disorders, Hereditary Hemorrhagic Telangiectasia (HHT) Phase 1/2
Healthy volunteer phase 1 data H1’26→ H2’26 and HTT H2’26.
H2’26ALNYAlnylam Pharmaceuticals ALN-2232 — Obesity & Weight Management Phase 1
Phase 1 data expected H2’26.
ARV-393 – mono & combos + glofitamab — R/R NHL Phase 1
Dose-escalation. Ongoing, interim monotherapy dose-escalation data in R/R NHL H2’26 [ASH’26?]. additional monotherapy dose-escalation data in B-/T-CL mid-2027, first patient in H1’26 in combo with glofitamab in DLBCL. data mid-2027, Aug…
Dose-escalation. Ongoing, interim monotherapy dose-escalation data in R/R NHL H2’26 [ASH’26?]. additional monotherapy dose-escalation data in B-/T-CL mid-2027, first patient in H1’26 in combo with glofitamab in DLBCL. data mid-2027, Aug 2026: responses in B-/T-CLs at dose levels < predicted effective exposure level.
ARV-806 — KRAS G12D solid tumors incl. PDAC, CRC, NSCLC Phase 1/2
Dose-escalation. Completed, initial monotherapy dose-escalation data 2026→ H2’26.
H2’26ARWRArrowhead Pharmaceuticals ARO-DIMER-PA — Mixed Hyperlipidemia Phase 1/2
First Phase 1/2 data expected H2’26.
H2’26BBIOBridgeBio Pharma Low-dose infigratinib — Hypochondroplasia - children & adolescents (ACCEL2/3) Phase 2/3
Phase 2 readout H2’26.
H2’26BBOTBridgeBio Oncology Therapeutics BBO-8520 - mono & + pembrolizumab or + BBO-10203 — KRAS G12C-mutant NSCLC (ONKORAS-101) Phase 1
Pembro combo ongoing, 10203 combo first patient in Apr 2026, Jan 2026 update. Nov 2025 data cut-off: 65% ORR in n=12 across all dose levels. Monotherapy, additional pembro combo efficacy and safety data H2’26.
H2’26BBOTBridgeBio Oncology Therapeutics BBO-11818 - mono & combos incl. +pembro or +chemo or +cetuximab or +BBO-10203 — KRAS-mutant solid tumors (KONQUER-101) Phase 1
Monotherapy dose-escalation. Ongoing, cetuximab combo first patient in H1’26, 10203 combo first patient in Jul-Aug 2026, Dec 2025 data cut-off: initial safety data and efficacy anecdote, expansion and combo cohorts planned for 2026,…
Monotherapy dose-escalation. Ongoing, cetuximab combo first patient in H1’26, 10203 combo first patient in Jul-Aug 2026, Dec 2025 data cut-off: initial safety data and efficacy anecdote, expansion and combo cohorts planned for 2026, additional monotherapy/combo efficacy and safety data H2’26.
H2’26BBOTBridgeBio Oncology Therapeutics BBO-10203 mono & +trastu or +fulv±ribociclib or +FOLFOX+bevac — Solid tumors incl. HR+ BC, HER2+ HR- BC, CRC (RASPI3Kα signaling) - (BREAKER-101) Phase 1
Dec 2025 data cut-off: initial safety data and efficacy anecdote, combo data in HER2+ BC, HR+/HER2- PIK3CAm BC and KRASm CRC H2’26.
H2’26BCAXBicara Therapeutics Ficerafusp alfa (BCA101) mono or + pembrolizumab — 3L+ RAS/BRAF-wt MSS mCRC – (BCA101X1101 / KN-E28) Phase 1
Expansion cohort from FIH phase 1, data from expansion cohort in monotherapy and +pembro H2’26.
H2’26BEAMBeam Therapeutics Risto-cel - formerly BEAM-101 — Sickle cell disease (BEACON) Phase 1/2
Registrational BEACON study in sickle cell disease; dosing complete in adolescents and adults, updated data expected H2’26 with BLA filing targeted year-end 2026. ASH 2025 data (n=31): rapid engraftment, no severe vaso-occlusive crises,…
Registrational BEACON study in sickle cell disease; dosing complete in adolescents and adults, updated data expected H2’26 with BLA filing targeted year-end 2026. ASH 2025 data (n=31): rapid engraftment, no severe vaso-occlusive crises, mean PBMC editing 67% at month 6 rising to 73% by month 12, rapid and sustained HbF increases with HbS reductions, and anemia resolution in 100% after transfused blood washed out; safety consistent with busulfan conditioning and autologous transplant. Published in NEJM April 2026.
BHV-2000 (Taldefgrobep Alfa) — Obesity Phase 2
Last patient in March 2026, topline H2’26, primary endpoint = % BW change at week 24.
Opakalim — Focal Epilepsy (RISE 3) Phase 2/3
Last patient in Jun 2026, topline H2’26.
BGE-102 — Pts w/ Elevated Cardiovascular Risk (QUELL-CV) Phase 2
RCT, first patient in Jun 2026, topline H2’26 [year-end 2026]. Primary endpoint = % change in hsCRP, potential phase 3 first patient in year-end 2027.
H2’26BMRNBioMarin Pharmaceutical BMN 351 — DMD (exon 51 amenable) Phase 1/2
Full data 6 and 9mg H1’26 at MDA’26, 12mg topline H2’26.
Rese-cel (CABA-201) — Pemphigus vulgaris (RESET-PV) Phase 1/2
Proof-of-concept data in n=3 witho LD, durability witho LD H2’26.
H2’26CGEMCullinan Therapeutics CLN-049 — R/R AML, R/R MDS Phase 1
Dose-escalation data ASH’25, updated dose-escalation data H2’26 [ASH’26 likely], dose expansion first patient in Q2’26, RP2D determination by Q4’26.
Cretostimogene grenadenorepvec (CG0070) ± gemcitabine — High-Risk NMIBC incl. BCG-naive, BCG-exposed, BCG-unresponsive (CORE‑008) Phase 2
Phase 2 in high-risk NMIBC across BCG-naive, BCG-exposed and BCG-unresponsive cohorts. Cohort CX data at AUA 2026 (cretostimogene + gemcitabine, sequential vs concurrent; 65% BCG-exposed/35% BCG-unresponsive): high-grade event-free…
Phase 2 in high-risk NMIBC across BCG-naive, BCG-exposed and BCG-unresponsive cohorts. Cohort CX data at AUA 2026 (cretostimogene + gemcitabine, sequential vs concurrent; 65% BCG-exposed/35% BCG-unresponsive): high-grade event-free survival 96% at 3 months and 89.5% at 6 months with no difference between schedules; CR at any time 86% in the CIS population and 92% ITT. Cohort C topline and cohort B data expected H2’26; cohort A monotherapy data (December 2025, SUO) showed 88% CR in BCG-naive CIS (n=25) with no grade 3+ adverse events.
H2’26CHRSCoherus Oncology Tagmokitug + toripalimab — 4L+ CRC w/o liver mets Phase 1/2
Fully enrolled, initial data H2’26.
H2’26CHRSCoherus Oncology Tagmokitug + toripalimab — 2L HNSCC Phase 1
Initial Phase 1 data expected mid-2026→ H2’26.
H2’26CHRSCoherus Oncology Tagmokitug + toripalimab — 2L gastric cancer, GEJ, EAC Phase 1
Initial Phase 1 data expected mid-2026→ H2’26.
H2’26CHRSCoherus Oncology Tagmokitug + toripalimab ± chemo — 1L/2L ESCC Phase 1
Initial Phase 1 data expected H2’26.
H2’26CHRSCoherus Oncology Casdozokitug + toripalimab+bevacizumab — 1L unresectable HCC Phase 2
Fully enrolled, initial data mid-2026→ H2’26.
H2’26CRSPCRISPR Therapeutics CTX611 (SRSD107) — Total Knee Arthroplasty (TKA) Phase 2
Topline/update H2’26, potential additional indications AF; VTE; CKD; PVD; CAT.
H2’26DNTHDianthus Therapeutics DNTH212 — Systemic lupus erythematosus (SLE) / healthy volunteers (CN) Phase 1
Healthy volunteer study in China. First patient in Dec 2025, Part A topline in healthy volunteers H2’26.
H2’26EIKNEikon Therapeutics EIK1003 mono & combos — Ovarian cancer (+chemo), Breast cancer (+chemo), Prostate Cancer (+ARPI), PDAC Phase 1/2
Two monotherapy dose levels (20 and 60 mg) evaluated to set the Phase 2 dose; first patient in for cohort 1D (EIK1003 + platinum chemotherapy + paclitaxel) in breast and ovarian cancer expected H2’26. ESMO 2026 will carry updated cohort 1A…
Two monotherapy dose levels (20 and 60 mg) evaluated to set the Phase 2 dose; first patient in for cohort 1D (EIK1003 + platinum chemotherapy + paclitaxel) in breast and ovarian cancer expected H2’26. ESMO 2026 will carry updated cohort 1A (monotherapy) and 1C (with paclitaxel) data plus initial cohort 1B (with abiraterone + prednisone) safety and preliminary efficacy in advanced prostate cancer. ASCO 2026: ORR 14% monotherapy (n=49) and 24.5% with paclitaxel (n=53).
Detalimogene voraplasmid — BCG-unresponsive NMIBC with CIS ± papillary disease (LEGEND Cohort 1 – pivotal, single-arm) Phase 2
Pivotal LEGEND readout in BCG-unresponsive NMIBC with CIS expected H2’26, pending sufficient 12-month CR events; May 2026 interim (n=124, without surfactant): CR at any time 54%, 6-month CR 46%. Primary endpoint was changed in H2’25 after…
Pivotal LEGEND readout in BCG-unresponsive NMIBC with CIS expected H2’26, pending sufficient 12-month CR events; May 2026 interim (n=124, without surfactant): CR at any time 54%, 6-month CR 46%. Primary endpoint was changed in H2’25 after FDA interaction from CR at 12 months to CR at any time, with duration of response as key secondary. BLA filing targeted H2’26; a surfactant-containing cohort started May 2026 while other cohorts were paused for cash preservation.
ERAS-4001 — KRASm solid tumors (BOREALIS-1) Phase 1
Preliminary monotherapy data H2’26, monotherapy expansion and comb dose-escalation FPIs 2027.
H2’26ETONEton Pharmaceuticals ET-700, zinc acetate ER — Wilson disease Phase 1
Pilot study first patient in Apr 2026, RCT vs zinc acetate [IR], readout H2’26, potential transition to pivotal trial early 2027.
Zanzalintinib + nivolumab — 1L Non-ccRCC (STELLAR-304) Phase 3
Phase 3 topline readout expected [PFS+ORR] mid-2026→ H2’26, OS secondary.
H2’26GYREGyre Therapeutics Pirfenidone capsule (ETUARY) — Pneumoconiosis (CN) Phase 3
China, readout H2’26.
H2’26IDYAIDEAYA Biosciences IDE034 — Solid tumors Phase 1
First patient in end-Feb 2026, data H2’26/early 2027.
Brenetafusp (IMC-F106C) mono & combos — Solid tumors incl. OC, NSCLC, melanoma Phase 1/2
First-in-human program for the ImmTAC brenetafusp in HLA-A02+ solid tumors. ASCO 2026 melanoma data (n=66, IO-experienced unresectable/metastatic): ORR 12%, DCR 52%, median OS 14.3 months on monotherapy, confirming the 160 mcg RP3D for…
First-in-human program for the ImmTAC brenetafusp in HLA-A02+ solid tumors. ASCO 2026 melanoma data (n=66, IO-experienced unresectable/metastatic): ORR 12%, DCR 52%, median OS 14.3 months on monotherapy, confirming the 160 mcg RP3D for PRISM-MEL-001; median OS was 14.7 months in primary PD-1-resistant patients. Brenetafusp + pembrolizumab showed numerically higher ORR/DCR than monotherapy (33% ORR in 6 primary-resistant patients); ovarian cancer activity seen, with bevacizumab and NSCLC combos ongoing. Additional data expected H2’26.
IMC-P115C — PRAME+ Solid tumors Phase 1
Initial dose-escalation data H2’26.
IMVT-1402 — Difficult-To-Treat (D2T RA), Anti-Citrullinated Protein Antibodies/ACPA+ RA Phase 2
Pt1 = 16-week open-label and Pt2=re-randomization of Pt1 responders at week 14-16 for 12-week RCT, May 2026 Pt1 update: ’-50-70 at week 16 73-54.5-36% [NRI] and 72-53-37% in JAKi+TNFi failure subgrp, readout H2’26.
IMVT-1402 — CLE Phase 2
Proof-of-concept readout H2’26.
INCA033989 + ruxolitinib — 1L Myelofibrosis with CALR mutation Phase 1/2
Phase 1 topline H2’26.
INCB160058 — JAK2 V617F-mutated MPNs Phase 2
Phase 2 data expected H2’26.
Povorcitinib — Asthma Phase 2
Proof-of-concept data H2’26.
JAK2 V617F inh. next gen — JAK2 V617F-mutated MPNs Preclinical
PC data H2’26.
Lacutamab (IPH4102) — PTCL (KILT) Phase 2
LYSARC IST, readout H2’26.
Monalizumab + durvalumab — Unresectable Stage III NSCLC after non-progression to cCRT (PACIFIC-9) Phase 3
Multi-arm RCT based on COAST phase 2, topline H2’26.
H2’26JANXJanux Therapeutics JANX011 — Autoimmune Diseases Phase 1
Primary completion expected H2’26.
Ziftomenib + gilteritinib/FLAG-IDA/LDAC — NPM1-m/FLT3m AML (KOMET-008) Phase 1
Dose-escalation, preliminary NMP1m/FLT3m gilte combo data H2’26.
H2’26KYTXKyverna Therapeutics KYV-101 (miv-cel, mivocabtagene autoleucel) — Progressive Multiple Sclerosis (KYSA-7, ISTs) Phase 2
Proof-of-concept data in n=5 with up to 12 months follow-up at conference in May 2026, additional IST phase 1 data H2’26, dev. strategy update early 2027.
H2’26LYELLyell Immunopharma Ronde-Cel (rondecabtagene autoleucel, LYL314) — 3L+ CD19-CAR-T-naïve aggressive LBCL (PiNACLE) Phase 3
Single-arm pivotal phase 1/2 expansion, update H2’26, pivotal data readout mid-2027, NDA filing 2027, phase 1/2 update at EHA’26 in n=108: no grade 3+ CRS; 8% grade 3+ ICANS including 8% with prophy vs 16% no-prophy; no change in cell…
Single-arm pivotal phase 1/2 expansion, update H2’26, pivotal data readout mid-2027, NDA filing 2027, phase 1/2 update at EHA’26 in n=108: no grade 3+ CRS; 8% grade 3+ ICANS including 8% with prophy vs 16% no-prophy; no change in cell expansion with prophy; 97% manuf. success rate.
H2’26LYELLyell Immunopharma LYL273 (GCC19CART) — 3L+ mCRC Phase 1/2
Initial report of 50% ORR in 3L+ mCRC at data cut-off end-Oct 2025, updated data Jun 2026: GI prophylaxis reduced grade 2+ diarrhea/colitis from 55% witho prophy to 10% with prophy, MTD N.R., phase 1 protocol amended for seamless expansion…
Initial report of 50% ORR in 3L+ mCRC at data cut-off end-Oct 2025, updated data Jun 2026: GI prophylaxis reduced grade 2+ diarrhea/colitis from 55% witho prophy to 10% with prophy, MTD N.R., phase 1 protocol amended for seamless expansion into potential pivotal single-arm phase 2 pending regulatory alignment and for new cohort [2L and RT combo], next update including additional data H2’26, pivotal trial first patient in H1’27.
H2’26MLTXMoonLake Immunotherapeutics Sonelokimab — HS (VELA-1 & VELA-2) Phase 3
IHS4-100 24.4/27.7%, BLA filing end-Sept 2026 [from H2’26] including VELA-TEEN data → potential Priority Review.
Ragistomig - mono & + aPD-(L)1 — R/R Solid Tumors Phase 1
Phase 1 topline readout expected H2’26.
Uliledlimab + aPD-(L)1 ± chemo — 1L+ mNSCLC Phase 1/2
PFS data H2’26 in later lines of therapy.
H2’26NMRANeumora Therapeutics NMRA-898 — Schizophrenia Phase 1
SAD in healthy volunteers, MAD in healthy volunteers and schizo patients, comprehensive M4 franchise update mid-2026 potential moving forward with 1 or both programs→ data H2’26.
Tacabrutideg, BGB‑16673 — R/R MCL; R/R CLL; R/R WM (CaDAnCe-101) Phase 2
Potential registrational data for AA H2’26 in R/R CLL, update at ASCO’26 and ’: R/R CLL/SLL ORR 85% and 2-year PFS 54%; R-R WM MRR 76% and VGPR 30% and 15 months-PFS 70%, BTK inhibitor-naïve CLL: ORR 86% and 6 months-PFS 100%.
BGB‑58067 mono & combos (+tisle+chemo) — Lung cancer, solid tumors Phase 1
Phase 1 data expected H2’26, combo part initiated Q1’26 in 1L NSCLC.
BG‑C477 — Lung cancer, GI cancers Phase 1
‘Promising’ data H2’26, pivotal trials planned.
H2’26ORICORIC Pharmaceuticals Enozertinib (ORIC-114) - mono or + SC amivantamab — EGFR ex20ins-mut NSCLC (1L: mono & + SC amivantamab, 2L: mono) Phase 1
Phase 1 data expected H2’26 in 1L EGFR ex20ins monotherapy and +SC amivantamab, potential phase 3 Redwood-1 in monotherapy or combo 2027+.
H2’26PBYIPuma Biotechnology Alisertib — 2L+ SCLC following on or after 1 prior Pt-based CT & anti-PD-L1 combo (ALISCA-Lung1) Phase 2
Interim data May 2026: ORR 6.7-11.5% across 50-60mg BID dose levels, 70 mg BID cohort initiated in 2026, presented at conference Q4’26, additional data H2’26/H1’27, expansion of enrollment in chemo-naïve patients H2’26 to obtain more data…
Interim data May 2026: ORR 6.7-11.5% across 50-60mg BID dose levels, 70 mg BID cohort initiated in 2026, presented at conference Q4’26, additional data H2’26/H1’27, expansion of enrollment in chemo-naïve patients H2’26 to obtain more data in biomarker focused pops, primary completion H2’27.
H2’26PCRXPacira BioSciences Triamcinolone ER (ZILRETTA) — Shoulder osteoarthritis pain Phase 3
Phase 3 topline readout expected H2’26.
H2’26RIGLRigel Pharmaceuticals R289 — Lower-risk MDS Phase 1
Phase 1 data expected at ASH’25, dose-expansion data; RP2D H2’26, expansion last patient in H2’26.
H2’26RNAAvidity Biosciences Del-desiran — Myotonic dystrophy type 1 (DM1) - (HARBOR) Phase 3
Phase 3 data readout expected H2’26.
H2’26ROIVRoivant Sciences IMVT-1402 — Difficult-to-Treat Rheumatoid Arthritis (D2T RA ie after failure to 2+ AT incl. biologics and/or DMARDs) Phase 2
Pt1 = 16-week open-label and Pt2=re-randomization of Pt1 responders at week 14-16 for 12-week RCT, May 2026 Pt1 update: ’-50-70 at week 16 73-54.5-36% [NRI] and 72-53-37% in JAKi+TNFi failure subgrp, readout H2’26.
H2’26ROIVRoivant Sciences IMVT-1402 — Cutaneous Lupus Erythematosus Phase 2
Phase 2 data readout expected H2’26.
H2’26RVMDRevolution Medicines RMC-5127 — KRAS G12V advanced solid tumors Phase 1
Monotherapy RP2D H2’26 but data disclosure 2027.
H2’26RXRXRecursion Pharmaceuticals REC-1245 — Solid tumors & lymphoma (Biomarker-enriched) Phase 1
Early safety/PK/PD data May 2026 supporting dose-escalation with goal to determine the RP2D, additional dose-escalation data H2’26.
H2’26RXRXRecursion Pharmaceuticals REC-4881 — Familial Adenomatous Polyposis (FAP) – (TUPELO) Phase 1/2
Preliminary efficacy data from phase 2 part Dec 2025 → next update H1’27, additional phase 2 data in FAP at leading hereditary GI conference Nov 2026, FDA interactions H1’26 and regulatory update H2’26, potential expansion into adults.
H2’26TARAProtara Therapeutics TARA-002 (Systemic Admin.) — HR NMIBC (ADVANCED-2) Preclinical
Phase 2 cohort C and D planned, PC data H2’26.
H2’26TLSATiziana Life Sciences Foralumab (TZLS-401) mono & +lecanemab / donanemab — Alzheimer’s disease Phase 2
First patient in Dec 2025, interim data H2’26.
H2’26TNGXTango Therapeutics Vopimetostat (TNG462) — MTAP‑deleted cancers (PDAC, NSCLC, other non‑CNS cancers) Phase 1/2
Dose expansion, 2L PDAC phase 3 in monotherapy first patient in 2026, monotherapy data in NSCLC 2026→ H2’26.
H2’26TNGXTango Therapeutics Vopimetostat (TNG462) + RAS(ON) inh. daraxonrasib or zoldonrasib — 2L+ MTAP-del RASm PDAC & NSCLC (any RAS with darax & KRAS G12Dm with zoldon) Phase 1/2
Initial dose-escalation. Combos data Jun 2026: 92% ORR; 100% DCR; 6 months-PFS 90% in n=12 PDAC and 100% ORR in n=3 NSCLC in combo with 100mg darax. 52% ORR; 37% cORR; 96% DCR; 6 months-PFS 74% in combo with 600-1200mg zoldon, combo data…
Initial dose-escalation. Combos data Jun 2026: 92% ORR; 100% DCR; 6 months-PFS 90% in n=12 PDAC and 100% ORR in n=3 NSCLC in combo with 100mg darax. 52% ORR; 37% cORR; 96% DCR; 6 months-PFS 74% in combo with 600-1200mg zoldon, combo data presented at ESMO’26, pivotal strategy in 1L PDAC freeze H2’26 with prioritization for the darax combo.
H2’26TNGXTango Therapeutics TNG456 — MTAP‑del glioblastoma Phase 1/2
Initial dose-escalation data 2026→ H2’26.
H2’26TRDAEntrada Therapeutics VX-670 — Myotonic dystrophy type 1 (DM1) – (GALILEO) Phase 1/2
SAD completed, MAD ongoing, enrollment and dosing completion mid-2026, readout H2’26.
H2’26VIRVir Biotechnology VIR-5818 ± pembrolizumab — HER2+ Solid tumors incl. Breast & GI Cancers Phase 1
Dose-escalation data H2’26 in monotherapy and + pembro, primary completion H1’28.
H2’26VRDNViridian Therapeutics VRDN-008 — Autoimmune disorders Phase 1
Healthy volunteer data H2’26, “VRDN-008 showed a longer half-life and more sustained IgG reduction vs efgartigimod in high-dose NHP study”.
Influvac HD — Influenza (EU) Phase 3
EU readout H2’26.
Norelgestromin+Low Ehinyl Estradiol Weekly Patch — Contraception (US) Registration
PDUFA decision expected H2’26.
H2’26XENEXenon Pharmaceuticals XEN1120 — Pain (Acute) Phase 1
SAD/MAD in healthy volunteers, readout 2026→ H2’26.
H2’26XENEXenon Pharmaceuticals XEN1701 — Pain (Acute) Phase 1
SAD/MAD in healthy volunteers, readout 2026→ H2’26.
JNJ-9401 — Prostate cancer Phase 1
Pasritamig combo data H2’26.
ZL-1503 — Moderate-to-severe atopic dermatitis Phase 1
Part A SAD data [4 dose levels] in healthy volunteers H2’26 from global portion, Part B MAD data [2 dose levels] in AtD H1’27.
Pasritamig (JNJ-78278343) ± JNJ-9401 — Later-lines mCRPC (After 1+ ARPI, 2+ taxanes, 1+ PSMA RLT, PARPi if eligible) – (KLK2-comPAS) Phase 3
Primary completion expected ~mid-2028, phase 1 combo data with JNJ-9401 H2’26.
Q4’26ABCLAbCellera Biologics ABCL575 — Atopic dermatitis Phase 1
Phase 1 topline readout expected Q4’26, negative readthrough on the class via Amgen/Sanofi.
Q4’26ACRSAclaris Therapeutics Bosakitug (ATI-045) — Atopic Dermatitis (Moderate to Severe) Phase 2
RCT, last patient in March 2026, topline Q4’26.
Q4’26AKBAAkebia Therapeutics Vadadustat (VAFSEO) — Anemia due to CKD (VOCAL) Phase 3
Phase 3 topline readout expected Q4’26.
Q4’26AKBAAkebia Therapeutics AKB-9090 — Cardiac surgery-associated AKI Phase 1
Primary completion expected Q4’26.
Empasiprubart — Multifocal motor neuropathy (MMN) – (EMPASSION) Phase 3
Phase 3 data readout expected Q4’26.
Q4’26ARQTArcutis Biotherapeutics Roflumilast foam 0.3% (ARQ-151) — Non-Segmental Vitiligo (pediatric) Phase 2
IST, open-label proof-of-concept, topline Q4’26 along with decision on potential next step.
Q4’26ARWRArrowhead Pharmaceuticals ARO-ALK7 — Obesity Phase 1
Phase 1 data expected Jan 2026 and later in 2026→ Q4’26.
Q4’26AXSMAxsome Therapeutics SUNOSI (solriamfetol) — Bonge Eating Disorder (ENGAGE) Phase 3
Phase 3 topline readout expected H2’26→ Q4’26.
Q4’26BCRXBioCryst Pharmaceuticals BCX17725 — Netherton Syndrome (adults & ados) Phase 1
Primary completion expected Q4’26.
Q4’26CADLCandel Therapeutics CAN-3110 (linoserpaturev) — Recurrent high-grade glioma (lesion 1+ CM, Arm A: dose-esc/exp 1x10e6 to 1x10e9 PFU / Arm B: cyclophosphamide pre-admin at 3x10e8 or 6x10e9 PFU / Arm C: up to 6 inj. at 1x10e8 or 1x10e9 PFU) Phase 1
Arms A and B OS data available, Arm C interim OS H2’25 with data cut-off mid-Aug 2025, Arm C mature OS data Q4’26, IND cleared Q1’26 for a dose-finding phase 2. Prep work ongoing for initiation.
Q4’26CGEMCullinan Therapeutics CLN-978 — SLE (OUTRACE-SLE) Phase 1
Initial safety and B-cell depletion data at EULAR’26 in n=14: reduction in hSLEDAI 71% including 36% DORIS remission. Peripheral B-cell deplation <LLOQ in 50% at ≥20µg, additional data Q4’26, expansion phase 2 first patient in early 2027…
Initial safety and B-cell depletion data at EULAR’26 in n=14: reduction in hSLEDAI 71% including 36% DORIS remission. Peripheral B-cell deplation <LLOQ in 50% at ≥20µg, additional data Q4’26, expansion phase 2 first patient in early 2027 in SLE and LN.
Q4’26CGEMCullinan Therapeutics CLN-978 — SjD (OUTRACE-SjD) Phase 1
Inital safety and B-cell depletion fo single dose + dose-escalation/efficacy data Q4’26.
Q4’26CGEMCullinan Therapeutics Velinotamig — Autoimmune diseases (CN) Phase 1
Genrix China trial, Jun 2026 update: rapid and marked reductions in SLEDAI-2K scores and proteinuria in first n=2 SLE at 10µg/kg target dose. Complete renal response in both [SLEDAI-2K 16 at bsln→ 0 at week 8 and 14 at blsn→ 2 at week 8],…
Genrix China trial, Jun 2026 update: rapid and marked reductions in SLEDAI-2K scores and proteinuria in first n=2 SLE at 10µg/kg target dose. Complete renal response in both [SLEDAI-2K 16 at bsln→ 0 at week 8 and 14 at blsn→ 2 at week 8], initial data Q4’26.
Eti-cel (UCART20x22) — R/R B-cell malignancies (NatHaLi-01) Phase 1
Dose-escalation phase 1 data at ASH’25, first patient in Q1’26 in LD IL-2 cohort, full phase 1 dataset Q4’26 including data from LD IL-2 cohort, EHA’26 update: ORR 88%; CR 63% in n=8 from optimal dose cohort.
CLYM116 — IgA nephropathy Phase 1
Initial PK/PD data in healthy volunteers Sept 2026: t1/2 29d; near-complete APRIL suppression with single 320mg dose; 60-75% IgA Gd-IgA1 suppression through 12-week supporting Q12W-Q3M dosing, additional phase 1 data Q4’26; China phase 1…
Initial PK/PD data in healthy volunteers Sept 2026: t1/2 29d; near-complete APRIL suppression with single 320mg dose; 60-75% IgA Gd-IgA1 suppression through 12-week supporting Q12W-Q3M dosing, additional phase 1 data Q4’26; China phase 1 data by Mabworks Q4’26.
Q4’26CMPXCompass Therapeutics CTX-8371 — Post-CPI solid tumors Phase 1
ICI-XP/refractory pop, phase 1 dose-escalation data at ASCO’26: ORR 20% in n=15 across all DLS and ORR 33% in n=6 at 2 highest dose levels 3 and 10mg/kg. Durable resp., cohort expansion [TNBC/HL/NSCLC] data dosed at either 3 or 10mg/kg in…
ICI-XP/refractory pop, phase 1 dose-escalation data at ASCO’26: ORR 20% in n=15 across all DLS and ORR 33% in n=6 at 2 highest dose levels 3 and 10mg/kg. Durable resp., cohort expansion [TNBC/HL/NSCLC] data dosed at either 3 or 10mg/kg in Q4’26.
Q4’26CMPXCompass Therapeutics CTX-10726 — Solid tumors Phase 1
First patient in Q1’26, initial data H2’26→ Q4’26.
Q4’26CORTCorcept Therapeutics Relacorilant — Cushing syndrome (hypercortisolism) – (GRACE) Registration
Phase 3 toplined positive May 2024, CRL Dec 2025 due to insufficient benefit/risk ratio → resubmission Jun 2026 with PDUFA Dec 17, 2026. Timing updated Oct 7, 2026 per company guidance: now guides Q4’26 (was Dec’26).
Oct’26DMACDiaMedica Therapeutics DM199 (rinvecalinase alfa) — Fetal growth restriction (IST Part 3) Phase 2
IST, IV/SC, first patient in 2025, cohort 1 topline Sept 2026 [n=6] [Oct 2026 check] Sept KOL call on first-cohort (n=6, 5 ug/kg) FGR topline rescheduled to a virtual KOL webinar Oct 23, 2026 (announced Sept 30, 2026); dosing has begun in…
IST, IV/SC, first patient in 2025, cohort 1 topline Sept 2026 [n=6] [Oct 2026 check] Sept KOL call on first-cohort (n=6, 5 ug/kg) FGR topline rescheduled to a virtual KOL webinar Oct 23, 2026 (announced Sept 30, 2026); dosing has begun in cohort 2 at 10 ug/kg.
Q4’26DNTHDianthus Therapeutics Claseprubart (DNTH103) — Multifocal motor neuropathy (MMN) Phase 2
RCT, topline H2’26→ Dec 2026, potential readthrough from ARGX empa’s MMN phase 3 readout Q4’26.
Q4’26DSGNDesign Therapeutics DT-216P2 — Friedreich ataxia (RESTORE-FA) Phase 1/2
May 2026 4-week data (n=16, weekly IV across 0.1–1 mg/kg): mean +6.4 points on mFARS, +2.7 on Upright Stability, >5-point PROMIS fatigue improvement; frataxin mRNA +65% in blood at week 4 and +42% in muscle. Protocol amended August 2026: 1…
May 2026 4-week data (n=16, weekly IV across 0.1–1 mg/kg): mean +6.4 points on mFARS, +2.7 on Upright Stability, >5-point PROMIS fatigue improvement; frataxin mRNA +65% in blood at week 4 and +42% in muscle. Protocol amended August 2026: 1 mg/kg as go-forward dose with 10 patients in a 12-week cohort, endogenous blood frataxin protein percent change specified as the efficacy primary endpoint, and exploration of doses above 1 mg/kg. 12-week frataxin data moved to Q1’27; registrational plan update expected Q4’26.
Q4’26ENTAEnanta Pharmaceuticals EDO-978 — CSU Phase 1
First patient in Apr 2026, topline Q4’26.
Q4’26FDMT4D Molecular Therapeutics 4D-150 — DME (SPECTRA) Phase 1/2
2-Year data expected H2’26→ Q4’26.
Epcoritamab (EPKINLY) + R-CHOP — 1L ND DLBCL (EPCORE DLBCL-2) Phase 3
Phase 3 data readout expected 2026→ Q4’26.
MCLA-158 (petosemtamab) + pembrolizumab — 1L PD-L1+ R/M HNSCC (LiGeR-HN1) Phase 3
Phase 3 data readout expected H2’26→ Q4’26.
Q4’26GPCRStructure Therapeutics Aleniglipron (GSBR-1290) — Obesity (with T2D), cardiovascular, metabolic Phase 2
Phase 2 data readout expected Q4’26, 2.5->180mg titration.
Alpha‑1 MP — Pulmonary Emphysema due to Alpha‑1 antitrypsin deficiency (SPARTA) Phase 3
Phase 3 topline readout expected Q4’26.
Q4’26HRMYHarmony Biosciences BP1.15205, BP-205 — Narcolepsy & other sleep/wake disorders Phase 1
Aug 2026 update including SAD topline in healthy volunteers: short Tmax 30-75min. mean t1/2 ~ 25hrs across dose levels supporting OD admin. dose proportional PK. no gender diff, MAD data in healthy volunteers Q4’26. Phase 1b in…
Aug 2026 update including SAD topline in healthy volunteers: short Tmax 30-75min. mean t1/2 ~ 25hrs across dose levels supporting OD admin. dose proportional PK. no gender diff, MAD data in healthy volunteers Q4’26. Phase 1b in sleep-deprived healthy volunteer first patient in Q3’26 with data early 2027, multiple phase 2s first patient in mid-2027.
Ozekibart (INBRX-109) + FOLFIRI — 3-4L Adv. CRC Phase 1
Update Apr 2026 with ORR 20% mPFS 5.5 months in 70% 4L+ pop, FDA meeting H2’26 for 1L pivotal trial plans, 2 additional phase 1 CRC cohort initiated Q2’26 including oze +FOLFIRI+beva in 2L and oze+Lonsurf+beva in 3-4L with interim results…
Update Apr 2026 with ORR 20% mPFS 5.5 months in 70% 4L+ pop, FDA meeting H2’26 for 1L pivotal trial plans, 2 additional phase 1 CRC cohort initiated Q2’26 including oze +FOLFIRI+beva in 2L and oze+Lonsurf+beva in 3-4L with interim results Q1’27, company seeking options for AA in 4L+ mCRC and EwS → FDA meeting Q4’26 to discuss plans to initiate a 1L CRC registrational trial and potential for AA in 4L CRC.
Axatilimab + ruxolitinib — 1L chronic GVHD Phase 2
Phase 2 topline readout expected Q4’26.
Ruxolitinib cream (Opzelura) — Hidradenitis suppurativa - mild/moderate (TRuE-HS 1&2) Phase 3
Phase 3 topline readout expected Q4’26.
Povorcitinib — Prurigo nodularis - moderate/severe (STOP-PN1 & 2) Phase 3
Phase 3 data expected mid-2026→ Q4’26.
DISC-3405 — SCD Phase 1
Initial Phase 1 data expected H2’26→ Q4’26.
KB803 — Ocular complications of DEB (IOLOITE) Phase 3
Phase 3 topline readout expected Q4’26.
Q4’26LXEOLexeo Therapeutics LX2020 — Arrhythmogenic cardiomyopathy (PKP2-ACM) – (HEROIC-PKP2) Phase 1/2
HEROIC-PKP2 gene therapy data (n=10; 3 at low dose 2e13 vg/kg, 7 at high dose 6e13 vg/kg): NSVTs and PVCs reduced or stabilized in the majority, including 22% mean NSVT improvement and 14% mean PVC improvement in 5 high-dose patients at…
HEROIC-PKP2 gene therapy data (n=10; 3 at low dose 2e13 vg/kg, 7 at high dose 6e13 vg/kg): NSVTs and PVCs reduced or stabilized in the majority, including 22% mean NSVT improvement and 14% mean PVC improvement in 5 high-dose patients at latest visit; PGIC improved in 80%. Biopsy results pending (n=2); 12-month data for all high-dose patients expected Q4’26 alongside regulatory engagement.
Q4’26LXRXLexicon Pharmaceuticals Sotagliflozin (ZYNQUISTA) — Type 1 diabetes (adjunct to insulin) – (STENO1) Registration
CRL Dec 2024, resubmission 2026→ Q4’26 based on STENO1 DK IST data where sota is approaching the patient exposure and safety data requirements prev. IDed by FDA as being ‘adequate’ to support an NDA resubmission.
MBX 4291 — Obesity Phase 1
MAD/Part B in BMI ≥30 → titration with 4x 30mgQW then single 120mg Q1M with t1/2 ~26d with gradual accum. “supportive of true Q1M dosing”; mean BW loss 7% [range 0-16%] at week 8 in n=6 active and 2 placebo; diarrhea through week 8 in n=1…
MAD/Part B in BMI ≥30 → titration with 4x 30mgQW then single 120mg Q1M with t1/2 ~26d with gradual accum. “supportive of true Q1M dosing”; mean BW loss 7% [range 0-16%] at week 8 in n=6 active and 2 placebo; diarrhea through week 8 in n=1 and no nausea or vomiting in 1st MAD cohort, 12-week MAD/Part C results Q4’26.
Q4’26MLTXMoonLake Immunotherapeutics Sonelokimab — PsA (+axSpA) (P-OLARIS) Phase 2
Interim readout Q4’26.
Q4’26NAMSNewAmsterdam Pharma Obicetrapib (TA-8995) — LDL-C lowering in ASCVD / HeFH (LDL-C ≥ 55 mg/dL) [BROADWAY] Marketed
EU, positive CHMP Jul 2026, potential DE launch Q4’26.
Q4’26NAMSNewAmsterdam Pharma Obicetrapib (TA-8995) — LDL-C lowering in HeFH (LDL-C ≥ 70 mg/dL) [BROOKLYN] Marketed
EU, positive CHMP Jul 2026, potential DE launch Q4’26.
Q4’26PBYIPuma Biotechnology Alisertib — 2L+ SCLC following on or after 1 prior Pt-based CT & anti-PD-L1 combo (ALISCA-Lung1) Phase 2
Interim data May 2026: ORR 6.7-11.5% across 50-60mg BID dose levels, 70 mg BID cohort initiated in 2026, presented at conference Q4’26, additional data H2’26/H1’27, expansion of enrollment in chemo-naïve patients H2’26 to obtain more data…
Interim data May 2026: ORR 6.7-11.5% across 50-60mg BID dose levels, 70 mg BID cohort initiated in 2026, presented at conference Q4’26, additional data H2’26/H1’27, expansion of enrollment in chemo-naïve patients H2’26 to obtain more data in biomarker focused pops, primary completion H2’27.
Q4’26PCRXPacira BioSciences iovera° system — Upper limb spasticity N/A
Registrational device trial, topline Q4’26.
PRGN-2009 + pembrolizumab — R/M cervical cancer Phase 2
Primary completion expected Q4’26.
Leniolisib (JOENJA) — Primary Immune Deficiencies (PIDs) with genetically defined immune dysregulation linked to PI3Kδ Phase 2
Phase 2 topline readout expected Q4’26.
Leniolisib (JOENJA) — CVID with immune dysregulation Phase 2
Phase 2 topline readout expected Q4’26.
Q4’26PRAXPraxis Precision Medicines Relutrigine — DEE (Phenotypic, Pediatric & Adults, regardless of etiology) – (EMERALD) Phase 3
Phase 3 topline readout expected Q4’26.
UX701 (rivunatpagene miziparvovec) — Wilson disease (Cyprus2+) Phase 1/2/3
Registrational, dose-finding stage data Q4’26, primary completion Q1’29.
RP2 mono & + atezolizumab+beva (HCC) or + durvalumab (BTC) — Locally adv. unresectable or metastatic HCC after after aPD-(L)1 failure, or BTC maintenance Phase 2
First patient in Q4’25 in BTC, preliminary data in HCC Q4’26, protocol amended to include RP2 monotherapy with data by year-end 2026.
Surabgene lomparvovec (sura-vec, ABBV-RGX-314) — Wet AMD (ATMOSPHERE, ASCENT) Phase 3
Phase 3 topline readout expected Q4’26, filings H1’27.
Q4’26SABSSAB Biotherapeutics SAB-142 — Delaying progression of type 1 diabetes in new-onset T1D pts (Stage 3, ≤100d from diagnosis, 5-40y) – (SAFEGUARD) Phase 2
Registrational, n=159 overall, Part A = dose-ranging study in n=12 adults fully enrolled Q1’26, Part B = dose-ranging RCT in n=147 pediatric+ados+adults with last patient in year-end 2026/Q4’26, topline H2’27.
Q4’26SLSSELLAS Life Sciences Tambiciclib (SLS009, GFH009) + ven-aza — 1L High-risk AML (coh. 1: pts unlikely to benefit from ven-aza based on molecular profiling, coh. 2 [early resistance]: pts with confirmed lack of response after 2C of ven-aza) Phase 2
First patient in Q1’26, n=28/80 enrolled, topline Q4’26.
Q4’26SNDXSyndax Pharmaceuticals Revumenib (Revuforj) — R/R acute leukemia with KMT2A translocation (AUGMENT-101-2A), R/R NPM1m AML (AUGMENT-101-2C) Marketed
New data in 1L; maintenance; RW; R-R setting 2H26 and 2027+, publication of R/R NUP98r data Q4’26.
Q4’26SNDXSyndax Pharmaceuticals Revumenib (Revuforj) — Myelofibrosis Phase 1/2
Proof-of-concept in MF to inform SNDX-62122 dec in this indication, PFI Q4’26, proof-of-concept data H2’27.
Molgramostim inhalation solution (MOLBREEVI) — Autoimmune pulmonary alveolar proteinosis (aPAP) Registration
PDUFA decision expected Nov 22, 2026, 3 months PDUFA date extension Apr 2026 after newly submitted data considered ‘major amendment’, MAAs under review by EMA and MHRA with potential approvals Q1’27 and Q4’26 respectively, presented at…
PDUFA decision expected Nov 22, 2026, 3 months PDUFA date extension Apr 2026 after newly submitted data considered ‘major amendment’, MAAs under review by EMA and MHRA with potential approvals Q1’27 and Q4’26 respectively, presented at ATS’26: continuous LT improvement in lung function and respiratory HR-QoL.
Q4’26TARAProtara Therapeutics IV Choline Chloride — Choline for parenteral support patients (THRIVE-3) Phase 3
Interim data H2’26→ Q4’26.
Q4’26TNYATenaya Therapeutics TN-201 — MYBPC3-associated hypertrophic cardiomyopathy (MyPEAK-1) Phase 1/2
MyPEAK-1 gene therapy in MYBPC3-associated HCM: initial n=6 data at AHA 2025 (3–6e13 vg/kg); FDA clinical hold (November 2025, immunosuppression monitoring standardization) lifted December 2025. Expansion cohort at 6e13 vg/kg with June…
MyPEAK-1 gene therapy in MYBPC3-associated HCM: initial n=6 data at AHA 2025 (3–6e13 vg/kg); FDA clinical hold (November 2025, immunosuppression monitoring standardization) lifted December 2025. Expansion cohort at 6e13 vg/kg with June 2026 interim: LVMI improvement 4–21% durable to 2 years, NYHA class improved by at least 1 in 5/6, meaningful KCCQ and 6-minute walk gains; all patients tapered off immunosuppressants. Data with ~2-year follow-up at 3e13 and ~1-year at 6e13 expected Q4’26 plus a regulatory update.
Q4’26TNYATenaya Therapeutics TN-401 — PKP2-associated arrhythmogenic right ventricular cardiomyopathy (RIDGE-1) Phase 1
TN-401 gene therapy in PKP2-associated ARVC (n=6, 3–6e13 vg/kg): data at ASCGT May 2026 showed PVC reductions of 31–84% at weeks 50–52 versus baseline, with substantial NSVT decreases as early as week 20 in the two high-burden patients.…
TN-401 gene therapy in PKP2-associated ARVC (n=6, 3–6e13 vg/kg): data at ASCGT May 2026 showed PVC reductions of 31–84% at weeks 50–52 versus baseline, with substantial NSVT decreases as early as week 20 in the two high-burden patients. Most frequent treatment-related events were transient troponin/transaminase elevations, including one grade 3 liver enzyme elevation from a medication error with steroid interruption. Additional 6e13 data and a regulatory update expected Q4’26.
Q4’26TRAXFirst Tracks Bio. ANB101 — Inflammatory diseases Phase 1
Phase 1a in healthy volunteers completed, potential dev. approach informed by clinical and translational evidence + competitor registrational data in SLE and CLE anticipated in Q4’26/early 2027.
Q4’26VIRVir Biotechnology Tobevibart + elebsiran — Hepatitis delta (HDV) - (ECLIPSE 1) Phase 3
Phase 3 topline readout expected Q4’26, Jan 2026 update including SOLSTICE phase 2 72-week and partial 96-week data: 88% undetectable HDV RNA and 90% with HBsAg<10 IU/mL by week 24. Maintained through week 96, presented at EASL’26.
MR-141 (phentolamine ophthalmic solution 0.75%) — Presbyopia (US) Registration
PDUFA decision expected mid-Oct 2026.
Obexelimab (XmAb5871) — Systemic lupus erythematosus (SLE) – (SunStone) Phase 2
Phase 2 topline readout expected Q4’26 in overall and biomarker pops, potential SLE phase 3 SPI H1’27.
Xanomeline-trospium (KarXT) — Psychosis in Alzheimer’s disease (ADEPT-1/2/3/4) Phase 3
Primary completion expected Q4’26.
Xanomeline-trospium (KarXT) — Manic episodes in Bipolar Disorder Type I (BALSAL-1/2) Phase 3
Primary completion expected Q4’26.
Zocilurtatug pelitecan (ZL-1310) mono & combos (+ atezo ± Pt-chemo) — ES-SCLC (1L induction, 1L maintenance, 2L+) Phase 1
Multiple prior data supporting phase 3 advancement, Part 1 monotherapy dose-escalation in 2L+ [0.8-2.8mg/kg] at ENA’25, 54% icORR in 2L+ at AACR’26 from Part 2 expansion [1.2 and 1.6mg/kg], data with atezo ± chemo in 1L H2’26 → 1L phase 3…
Multiple prior data supporting phase 3 advancement, Part 1 monotherapy dose-escalation in 2L+ [0.8-2.8mg/kg] at ENA’25, 54% icORR in 2L+ at AACR’26 from Part 2 expansion [1.2 and 1.6mg/kg], data with atezo ± chemo in 1L H2’26 → 1L phase 3 go/no-go Q4’26, additional data at ESMO’26 from doublet combo dose-escalation Part 1B and from triplet combo dose-escalation Part 1C, Part 3 = doublet expansion at 1.6mg/kg.
Tibulizumab (ZB-106) — Hidradenitis suppurativa (TibuSHIELD) Phase 2
Enrollment expanded Jun 2026 to n=225 from 180 ‘due to faster-than-expected enrollment’ ‘to improve the study’s power’, topline Q4’26.
Nov’26BBIOBridgeBio Pharma BBP-418 — Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) – (FORTIFY) Registration
NDA filed end-March 2026, PDUFA Nov 27, 2026 with Priority Review, positive Phase Oct 3, 2025.
Nov'26BOLTBolt Biotherapeutics BDC-4182 — Gastric/GEJ cancer (CLDN18.2) Phase 1/2
Initial Phase 1 dose-escalation data guided with Q3 2026 results (company PR Aug 11, 2026); Q3 report estimated ~Nov 11, 2026 from past reporting schedule, not company-confirmed. NCT06921837.
Aficamten (MYQORZO) — Obstructive hypertrophic cardiomyopathy (oHCM) - (MAPLE-HCM) Registration
sNDA filed, PDUFA Nov 14, 2026, label expansion superiority to beta-blocker, phase 3 head-to-head metoprolol with positive topline May 2025.
Molgramostim inhalation solution (MOLBREEVI) — Autoimmune pulmonary alveolar proteinosis (aPAP) Registration
PDUFA decision expected Nov 22, 2026, 3 months PDUFA date extension Apr 2026 after newly submitted data considered ‘major amendment’, MAAs under review by EMA and MHRA with potential approvals Q1’27 and Q4’26 respectively, presented at…
PDUFA decision expected Nov 22, 2026, 3 months PDUFA date extension Apr 2026 after newly submitted data considered ‘major amendment’, MAAs under review by EMA and MHRA with potential approvals Q1’27 and Q4’26 respectively, presented at ATS’26: continuous LT improvement in lung function and respiratory HR-QoL.
Povetacicept — IgA nephropathy (RAINIER) Phase 3
Vertex trial but China contribution by ZLAB, positive phase 3 week 36 topline from Vertex global phase March 3, 2026, PDUFA Nov 30, 2026 for AA, China filing date by ZLAB?
YE’26AAPGAscentage Pharma Lisaftoclax (APG-2575) + BTKi — R/R CLL/SLL with sub-optimal BTKi response (Add-on) – (GLORA – Global) Phase 3
Primary completion expected year-end 2026.
ACI-35.030 (JNJ-2056) — Alzheimer’s Disease (Reτain) Phase 2
Primary completion expected year-end 2026.
Alixorexton (ALKS 2680) — Idiopathic hypersomnia (Vibrance-3) Phase 2
Phase 2 topline readout expected ‘around year-end 2026’.
Imsidolimab — Generalized Pustular Psoriasis Registration
PDUFA decision expected Dec 12, 2026.
Tenapanor (IBSRELA) — Chronic Idiopathic Constipation (CIC) – (ACCEL) Phase 3
First patient in Jan 2026, last patient in by year-end 2026, topline H2’27, primary endpoint=constipation PROs.
YE’26ARWRArrowhead Pharmaceuticals SRP-1004 (ARO-ATXN2) — Spinocerebellar Ataxia 2 Phase 1/2
Primary completion expected year-end 2026.
YE’26AUTLAutolus Therapeutics Obe-cel (AUCATZYL) — SLE with kidney manifestation (CARLYSLE) Phase 1
Follow-up data at ACR’26 in n=6: DORIS 83.3%; complete renal remission 50% with 50x10e6 cells dose level → “data supporting progression into LN phase 2”, cohorts ongoing in ados 12-17y and at higher dose levels, longer follow-up data…
Follow-up data at ACR’26 in n=6: DORIS 83.3%; complete renal remission 50% with 50x10e6 cells dose level → “data supporting progression into LN phase 2”, cohorts ongoing in ados 12-17y and at higher dose levels, longer follow-up data year-end 2026.
YE’26AUTLAutolus Therapeutics AUTO1/22 — R/R ALL - pediatric (CARPALL) Phase 1
Primary completion expected year-end 2026.
YE’26AUTLAutolus Therapeutics AUTO6NG — R/R Neuroblastoma (MAGNETO) Phase 1
Primary completion expected year-end 2026.
YE’26AUTLAutolus Therapeutics AUTO8 — Light-chain Amyloidosis (ALARIC) Phase 1
Initial Phase 1 data expected by year-end 2026 in n~10.
YE’26BBIOBridgeBio Pharma BBP-812 — Canavan Disease (CANaspire) Phase 1/2
Primary completion expected year-end 2026.
BGE-102 — Pts w/ Elevated Cardiovascular Risk (QUELL-CV) Phase 2
RCT, first patient in Jun 2026, topline H2’26 [year-end 2026]. Primary endpoint = % change in hsCRP, potential phase 3 first patient in year-end 2027.
Gotistobart (BNT316, ONC-392) + PLUVICTO — 2L+ mCRPC Phase 1/2
Primary completion expected year-end 2026.
Dec’26CGEMCullinan Therapeutics CLN-978 — RA (OUTRACE-RA) Phase 1
Phase 1 OUTRACE-RA: initial data at EULAR 2026 (n=7) showed disease activity improvement in 71%, including one DAS28-ESR remission at the single 30 µg target dose, with peripheral B-cell depletion below LLOQ in 67% at ≥20 µg. Multi-dose…
Phase 1 OUTRACE-RA: initial data at EULAR 2026 (n=7) showed disease activity improvement in 71%, including one DAS28-ESR remission at the single 30 µg target dose, with peripheral B-cell depletion below LLOQ in 67% at ≥20 µg. Multi-dose readout slipped from Q3’26 to December 2026 and will be bundled with SLE and Sjögren’s data; the November 8 ACR Convergence poster will instead cover velinotamig. Phase 2 expansion first patient in is guided for early 2027.
Dec’26COGTCogent Biosciences Bezuclastinib (CGT9486) — Nonadvanced systemic mastocytosis (NonAdvSM) - (SUMMIT) Registration
PDUFA decision expected Dec 30, 2026, NDA filed end-Dec 2025 under RTOR, registrational phase 2 data at AAAAI’26.
YE’26CORTCorcept Therapeutics Nenocorilant + nivolumab — Solid tumors (SYNERGY) Phase 1
First patient in 2026, data by year-end 2026.
YE’26CORTCorcept Therapeutics Miricorilant — Biopsy-confirmed or presumed Metabolic dysfunction–associated steatohepatitis (MASH) – (MONARCH) Phase 2
Phase 2 data readout expected by year-end 2026.
YE’26CRVSCorvus Pharmaceuticals Soquelitinib (CPI-818) — Moderate-to-severe AtD (CN) Phase 1/2
China, phase 2 accrual to be potential adjusted based on phase 1b data, cohort 1 results year-end 2026 at 100mg BID and 200mg OD.
YE’26DRUGBright Minds Biosciences BMB-101 — Prader-Willi syndrome (PWS) – (NOVA) Phase 2
Primary completion expected year-end 2026.
YE’26GLSIGreenwich LifeSciences GLSI-100 — Recurrence prevention in HLA-A*02+ HER2/neu+ breast cancer at high risk & after perioperative treatment (Flamingo-01) [N.B. HLA-A*02- pts go into an open-label arm] Phase 3
Primary completion expected year-end 2026.
Amivantamab (RYBREVANT) - SC — 2L+ anti-EGFR-naive HPV- R/M HNSCC after prog. on or after aPD-(L)1 & Pt-chemo (OrigAMI-4) Registration
sBLA filed with Priority Review → potential PDUFA year-end 2026, based on registrational phase 2 portion of phase 1/2 [single-arm]: 42% ORR by BiCR; 56% of responses lasted ≥6 months; 47% ORR bu inv.; mPFS 6.8ùp and mOS 12.5 months.
IMC-R117C – mono & combos (chemo, aVEGF, TKIs) — Colorectal & GI cancers Phase 1/2
Primary completion expected year-end 2026.
Povorcitinib — Hidradenitis suppurativa - moderate/severe Registration
Filing early 2026, potential EU approval late 2026 and US approval early 2027.
Selcodebart (DISC-0974) — Anemia of myelofibrosis (RALLY-MF) Phase 1/2
ASCO’26 update in n=50 evaluable: Major response 55% on non-transfusion dose-dependent cohort and 64% in lightly transfused [TD Low] cohort; 50% in heavily transfused [TD High] cohort; ORR 72%. Similar rate regardless of concomitant JAKi,…
ASCO’26 update in n=50 evaluable: Major response 55% on non-transfusion dose-dependent cohort and 64% in lightly transfused [TD Low] cohort; 50% in heavily transfused [TD High] cohort; ORR 72%. Similar rate regardless of concomitant JAKi, updated phase 2 data H2’26→ Q4’26 [assumedly ASH’26], end-of-Phase-2 meeting by year-end 2026.
YE’26JAZZJazz Pharmaceuticals Zanidatamab — 2L+ HER2+ solid tumors – (DiscovHER PAN-206) Phase 2
Primary completion expected year-end 2026.
Injectable KB707 — Solid tumors incl. BCC, melanoma, Gorlin syndrome (OPAL-1) Phase 1/2
Phase 1/2 data expected of Gorlin syndrome cohort by year-end 2026.
YE’26LKFTLakefront Biotherapeutics Gamgertamig (OM336, CM336) — Autoimmune Cytopenia, AIHA, ITP, APS (global) Phase 1
Phase 1 data readout expected year-end 2026.
late’26MAZEMaze Therapeutics MZE829 — APOL1-Mediated Kidney Disease (AMKD) – (HORIZON) Phase 2
Positive proof-of-concept end March 2026 but n=12/15 evaluated for efficacy and differences in effect size across subgrps, data from all 3 cohorts of n=10-15 late 2026/early 2027 including AMKD witho diabetes. AMKD with diabetes. AMKD with…
Positive proof-of-concept end March 2026 but n=12/15 evaluated for efficacy and differences in effect size across subgrps, data from all 3 cohorts of n=10-15 late 2026/early 2027 including AMKD witho diabetes. AMKD with diabetes. AMKD with FSGS, pivotal trial being planned.
Dec’26MLYSMineralys Therapeutics Lorundrostat — Hypertension – uncontrolled (LAUNCH-HTN) Registration
PDUFA decision expected Dec 22, 2026. No Priority Review, positive topline March 2025 with full data at ACC’25, 50mg lorun: 16.9 mmHg reduction in SBP by AOBP at week 6 ie 9.1 mmHg placebo-adj reduction; 19 mmHg reduction in SBP by AOBP at…
PDUFA decision expected Dec 22, 2026. No Priority Review, positive topline March 2025 with full data at ACC’25, 50mg lorun: 16.9 mmHg reduction in SBP by AOBP at week 6 ie 9.1 mmHg placebo-adj reduction; 19 mmHg reduction in SBP by AOBP at week 12 ie 11.7 mmHg placebo-adj reduction, no improvement with 50100mg titration.
YE’26MNPRMonopar Therapeutics MNPR-101-Lu — uPAR-expressing adv. solid tumors Phase 1
Primary completion expected year-end 2026.
YE’26NAMSNewAmsterdam Pharma Obicetrapib (TA-8995) — LDL-C lowering in ASCVD / HeFH (LDL-C ≥ 55 mg/dL) [PREVAIL CVOT] Phase 3
Phase 3 topline readout expected year-end 2026 at the earliest→ IA performed Q4’26 with results Q1’27, trial to continue to reach the min # of events after year-end 2026.
YE’26NAMSNewAmsterdam Pharma Obicetrapib (TA-8995) — LDL-C lowering in T2D / Metabolic Syndrome (LDL-C ≥ 100 mg/dL) [RUBENS] Phase 3
Phase 3 topline readout expected by year-end 2026.
YE’26NAMSNewAmsterdam Pharma Obicetrapib (TA-8995) — Lp(a) lowering (Lp(a) ≥ 125 nmol/L or 50-125 nmol/L) [VINCENT] Phase 2
Phase 2 topline readout expected year-end 2026.
YE’26NERVMinerva Neurosciences Seltorexant — MDD (with insomnia symptoms) Phase 3
Primary completion expected year-end 2026.
YE’26NMRANeumora Therapeutics NMRA-215 – mono & combo — Obesity Preclinical
FIH start H1’26, biomarker data Q3’26, BW loss data by year-end 2026.
NXP900 + osimertinib — EGFRm NSCLC (coh. 1) Phase 1
Primary completion expected year-end 2026.
YE’26OVIDOvid Therapeutics OV329 — Drug-resistant focal onset seizures (adults) Phase 1
First patient in Q2’26, open-label photo paroxysmal response [PPR] study, first patient in Q3’26, readout ~year-end 2026, photo stimulation study to evaluate the anti-convulsant effect of 3 dose levels of OV329 including 5-7-9mg.
YE’26OVIDOvid Therapeutics OV4071 — Ketamine challenge with biomarkers Preclinical
Ketamine challenge phase 1 study. FIH H2’26, goal = further characterize potential PD effects and proof-of-mechanism using electrophysiology and biomarkers, results year-end 2026/early 2027.
YE’26PRQRProQR Therapeutics AX-0810 — Cholestatic diseases, biliary atresia (BA) Phase 1
Phase 1 in healthy volunteers (3, 6, 9 mg/kg): June 2026 target-engagement data (n=22, cohorts 1–2) showed clear NTCP engagement with dose-dependent serum total bile acid increases up to 8x, above the company’s predefined 2x threshold,…
Phase 1 in healthy volunteers (3, 6, 9 mg/kg): June 2026 target-engagement data (n=22, cohorts 1–2) showed clear NTCP engagement with dose-dependent serum total bile acid increases up to 8x, above the company’s predefined 2x threshold, plus statistically significant increases in urinary conjugated bile acid excretion and post-challenge TUDCA; no hormone changes. Biliary atresia selected as lead indication, with a China investigator-initiated trial in pediatric patients to inform a potential pivotal Phase 2 (first patient in mid’27, interim mid’28). 9 mg/kg data by year-end 2026; initial IIT data H1’27.
YE’26PRQRProQR Therapeutics AX-0811 — Cholestatic diseases, biliary atresia (BA) Preclinical
CTA filing mid-2026, initial clinical data [target engagement] in healthy volunteers by year-end 2026.
YE’26QNCXQuince Therapeutics AM-001 — Bronchiolitis Obliterans Syndrome post-lung transplant (BOS) Phase 2
Primary completion expected year-end 2026.
YE’26RANIRani Therapeutics RT-114 — Obesity Phase 1
Primary completion expected year-end 2026.
RP2 mono & + atezolizumab+beva (HCC) or + durvalumab (BTC) — Locally adv. unresectable or metastatic HCC after after aPD-(L)1 failure, or BTC maintenance Phase 2
First patient in Q4’25 in BTC, preliminary data in HCC Q4’26, protocol amended to include RP2 monotherapy with data by year-end 2026.
YE’26RNACCartesian Therapeutics Descartes-08 — AChR+ generalized myasthenia gravis (AURORA) Phase 3
Primary completion expected year-end 2026.
Ersodetug — Tumor hyperinsulinism (upLIFT) Phase 3
Open-label, topline H2’26, positive IA at 50% enrollment Jun 2026: 50 GIR reduction in 6/8 patients. Timing updated Oct 7, 2026 per company guidance: now guides YE’26 (was H2’26).
YE’26SLSSELLAS Life Sciences Tambiciclib (SLS009, GFH009) + Zanubrutinib — R/R DLBCL Phase 1/2
Primary completion expected year-end 2026.
YE’26SNDXSyndax Pharmaceuticals Revumenib (Revuforj) + ven+ASTX727 — 1L unfit & R/R NPM1m, KMT2Ar, NUP98r acute leukemias (SAVE) Phase 1/2
Primary completion expected year-end 2026.
YE’26SNDXSyndax Pharmaceuticals Revumenib (Revuforj) + venetoclax — NPM1m, KMT2A-r, NUP98r AML w/ MRD ≥ 0.1% by MFC (Break Through Cancer) Phase 1/2
Primary completion expected year-end 2026.
YE’26SRPTSarepta Therapeutics Eteplirsen (EXONDYS 51) — DMD (exon 51 skipping) - (MIS51ON) Phase 3
Phase 3 data readout expected year-end 2026.
YE’26SRPTSarepta Therapeutics SRP-1004 (ARO-ATXN2) — Spinocerebellar ataxia type 2 (SCA2) Phase 1/2
Primary completion expected year-end 2026.
SRK‑439 — Rare neuromuscular disorders Phase 1
Phase 1 topline readout expected H2’26→ late 2026 in healthy volunteers.
YE’26SUPNSupernus Pharmaceuticals SPN-820 / SPN-821 — Major depressive disorder (MDD) Phase 2
Primary completion expected year-end 2026.
Ublituximab - SC — Relapsing MS (RMS) Phase 3
Last patient in Apr 2026, topline year-end 2026/early 2027, non-inferiority SC Q2M and SC Q3M vs ubli IV.
Azer-cel (azercabtagene zapreleucel) — B-cell disorders incl. MS Phase 1
Primary completion expected year-end 2026.
YE’26TYRATyra Biosciences Dabogratinib (TYRA-300) — FGFR3-altered mUC & solid tumors (SURF301) Phase 1/2
Primary completion expected year-end 2026.
Dec’26VNDAVanda Pharmaceuticals Tasilmelteon (HETLIOZ) — Jet Lag Disorder, Insomnia Registration
2nd CRL Jan 2026 in Jet Lag after 1st CRL in 2018, formal evidentiary public hearing granted. 5d hearing guided for Dec 2026, negative re-exam CHMP March 2026.
Dec’26VNDAVanda Pharmaceuticals Imsidolimab — Generalized Pustular Psoriasis (GEMINI-1 & 2) Registration
Filed Q4’25, PDUFA Dec 12, 2026 → no Priority Review.
YE’26VTVTvTv Therapeutics Azeliragon + CRT — 1L GBM Phase 1/2
Primary completion expected year-end 2026.
YE’26VTVTvTv Therapeutics Azeliragon — Reduction of Cardiotoxicity in Breast Cancer Phase 1/2
Primary completion expected year-end 2026.
ZB021 — I&I, Psoriasis Phase 1
China SAD/MAD phase 1 in healthy volunteers by InnoCare, first patient in during H1’26, data by year-end 2026, then proof-of-concept in psoriasis by Zenas in North America with results 2027 after China phase 1 completion.
H1’27AAPGAscentage Pharma Olverembatinib (HPQ1351) — 2L SDH-deficient GIST (POLARIS-3 - Global/Multi TBC) Phase 3
Primary completion expected H1’27.
H1’27AAPGAscentage Pharma Arilzomadin (APG-115) ± Lisaftoclax (APG-2575) — Pediatric Solid Tumors, Neuroblastoma, RMS (CN) Phase 1/2
China, primary completion H1’27, initial data at ASCO’26 n=17: 1 CR in ped. Rhabdomyosarcoma *RMS* in monotherapy and ORR 23.5% in combo including 1 CR in Ewing sarcoma.
H1’27AAPGAscentage Pharma APG-2449 ± chemo — PROC, adv. solid tumors (CN) Phase 1
Primary completion expected H1’27.
H1’27ABEOAbeona Therapeutics TSHA-102 — Rett syndrome (REVEAL & ASPIRE) Phase 3
Taysha trials, seamless phase 1/2/3 REVEAL and phase 3 ASPIRE, primary completion 2031, REVEAL 6 months IA topline report H1’27 but availability early 2027 then Taysha to discuss the data with FDA. FDA feedback on next steps toward BLA…
Taysha trials, seamless phase 1/2/3 REVEAL and phase 3 ASPIRE, primary completion 2031, REVEAL 6 months IA topline report H1’27 but availability early 2027 then Taysha to discuss the data with FDA. FDA feedback on next steps toward BLA submission H1’27, BLA submission to include ASPIRE safety data to support broad label in ≥2y pop.
H1’27ACHVAchieve Life Sciences Cytisinicline — Nicotine dependence, smoking cessation (ORCA-2 & -3 & ORCA-OL) Registration
PDUFA decision expected Jun 20, 2026 → Apr 2026 update: company expecting to receive a CRL after observations after inspection → CRL Jun 2026 for CMC deficiencies at 3rd party manufacturer; final product labeling also not completed by end…
PDUFA decision expected Jun 20, 2026 → Apr 2026 update: company expecting to receive a CRL after observations after inspection → CRL Jun 2026 for CMC deficiencies at 3rd party manufacturer; final product labeling also not completed by end of review → tech xfer to Adare → resubmission Q4’26 with Adare as primary manufacturer, filing based on positive data from 2x phase 3 and ORCA-OL extension, potential approval/launch H1’27.
ACI-19764 — Neuroinflammation, Pts w/ CVD risks Phase 1
SAD MAD in healthy volunteers, first patient in end-Feb 2026, Aug 2026 update: good safety/tol in SAD/MAD in healthy volunteers up to 20mg/d. T1/2> 30hrs. evidence of brain penetration based on CSF exposure. Doses ≤10mg/d achieved PK…
SAD MAD in healthy volunteers, first patient in end-Feb 2026, Aug 2026 update: good safety/tol in SAD/MAD in healthy volunteers up to 20mg/d. T1/2> 30hrs. evidence of brain penetration based on CSF exposure. Doses ≤10mg/d achieved PK concentrations >IC90. dose dose-dependent IL-1B release inh. Dosing started in patients with cardiovascular disease risk [elevated hsCRP and either T2D and/or obesity]→ initial results by year-end 2026/H2’26 in this pop., additional data H1’27.
AKY-1189 — Nectin-4 expressing solid tumors incl. mUC, mBC, NSCLC, CRC, cervical cancer Phase 1
Preliminary data Q1’27, primary completion year-end 2027.
Q1’27ALNYAlnylam Pharmaceuticals Elebsiran + tobevibart — Hepatitis D virus infection (ECLIPSE-1/2/3) Phase 3
Phase 3 data readout expected Q4’27 and Q1’27.
H1’27ALNYAlnylam Pharmaceuticals ALN-4324 — Type 2 diabetes mellitus Phase 2
Primary completion expected H1’27.
H1’27ALNYAlnylam Pharmaceuticals ALN-4324 — Overweight/Obesity + T2D Phase 1/2
Primary completion expected H1’27.
Pemvidutide — Alcohol-associated liver disease (ALD) – (RESTORE) Phase 2
Primary completion expected H1’27.
Efgartigimod — Systemic sclerosis Phase 2
Primary completion expected Q1’27.
Efgartigimod — Primary immune thrombocytopenia (ITP) (ADVANCE-NEXT - US) Phase 3
Phase 3 data readout expected Q4’26→ H1’27.
Q1’27ARQTArcutis Biotherapeutics Roflumilast foam 0.3% (ARQ-151) — Hidradenitis Suppurativa (HS) Phase 2
IST, open-label proof-of-concept, topline Q1’27 along with decision on potential next step.
Luxdegalutamide (ARV-766, JSB462) – mono & + abiraterone — mCRPC, metastasis Prostate Cancer incl. pts naïve to novel hormonal agents Phase 1/2
Primary completion expected H1’27.
Q1’27AURAAura Biosciences Belzupacap sarotalocan (bel-sar) — Metastases to the choroid Phase 2
Proof-of-concept data 2026 → update Q1’27 including guidance on trial completion.
Q1’27AURAAura Biosciences Belzupacap sarotalocan (bel-sar) — Ocular surface cancers, retinoblastoma (pediatric) Phase 1
Proof-of-concept data 2026 → update Q1’27 including guidance on trial completion.
BIIB145 — Autoimmune diseases Phase 1
Primary completion expected Q1’27.
Felzartamab — AMR Phase 3
Phase 3 data readout expected mid-2027→ H1’27.
Pumitamig (BNT327, BMS986545) + paclitaxel — 2L SCLC (CN) Phase 3
Primary completion expected Q1’27.
Pumitamig (BNT327, BMS986545) + chemo — 2L neuroendocrine neoplasms (CN) Phase 2
Primary completion expected Q1’27.
Q1’27CBIOCrescent Biopharma CR-001 — Advanced solid tumors (ASCEND) - global Phase 1/2
Preliminary dose-escalation and backfill cohort data Q1’27 including 1L NSCLC backfill cohort, initial combo data with SOC chemos in 1L and 2L+ ~mid-2027.
Q1’27CBIOCrescent Biopharma CR-003 (SKB105) — Advanced solid tumors - CN Phase 1/2
First patient in Q1’26, proof-of-concept data Q1’27.
BIIB145 — Autoimmune diseases Phase 1
Primary completion expected Q1’27.
H1’27CHRSCoherus Oncology Tagmokitug + pasritamig — 3L+ mCRPC Phase 1
First patient in Fall’26, initial data H1’27.
CLYM116 — IgA nephropathy (NAVIGATE) Phase 2
RCT, 200mg loading dose + 400mg Q8W or Q12W, intial data H1’27, phase 2 intended to support the advancement of Q12W regimen into phase 3 [first patient in 2027], high concentration 200 mg/mL formulation developed enabling 400mg SC…
RCT, 200mg loading dose + 400mg Q8W or Q12W, intial data H1’27, phase 2 intended to support the advancement of Q12W regimen into phase 3 [first patient in 2027], high concentration 200 mg/mL formulation developed enabling 400mg SC injection with PFS. autoinjector in dev.
H1’27CMPXCompass Therapeutics CTX-009 (tovecimig) + durva+chemo — 1L Biliary Tract Cancer Phase 1/2
Primary completion expected H1’27.
H1’27CTMXCytomX Therapeutics CX-801 + pembrolizumab — Adv. melanoma Phase 1
Monotherapy dose-escalation. DL4 ongoing, combo dose-escalation. DL3 cleared, data by H2’26→ initial melanoma combo data H1’27.
H1’27DNLIDenali Therapeutics BIIB122 (DNL151) — Parkinson’s disease (carriers of a pathogenic LRRK2 variant) - (BEACON) Phase 2
Denali trial, readout H1’27.
H1’27DNLIDenali Therapeutics DNL628 (OTV:MAPT) — Alzheimer’s disease Phase 1
CTA cleared Jan 2026, initial biomarker data from AD patients H1’27.
Q1’27DYNDyne Therapeutics Zeleciment rostudirsen (DYNE-251) — DMD (exon 51) – (DELIVER) Registration
PDUFA decision expected Jan 21, 2027 with Priority Review, registrational phase 1/2 expansion cohort for AA, NDA filed for AA based on dystrophin surrogate end May 2026 at 20mg/kg Q4W, potential launch Q1’27, data presented at MDA early…
PDUFA decision expected Jan 21, 2027 with Priority Review, registrational phase 1/2 expansion cohort for AA, NDA filed for AA based on dystrophin surrogate end May 2026 at 20mg/kg Q4W, potential launch Q1’27, data presented at MDA early March 2026.
Q1’27ELVNEnliven Therapeutics ELVN-002 + trastuzumab or + trastuzumab+chemo — HER2+ solid tumors (+trastu), HER2+ CRC & mBC (+trastu+chemo) Phase 1
Primary completion expected Q1’27.
H1’27EPRXEupraxia Pharmaceuticals EP-104GI — Eosinophilic esophagitis (EoE) – (RESOLVE ph2b portion) Phase 2
Phase 2 topline readout expected H2’26→ Q4’26 including clinical and histological endpts, followed by end-of-Phase-2 meeting and potential phase 3 H1’27.
Q1’27EWTXEdgewise Therapeutics Sevasemten — Duchenne muscular dystrophy (LYNX in 4-9y & FOX in 6-14y) Phase 2
Primary completion expected Q1’27.
H1’27FDMT4D Molecular Therapeutics 4D-150 — Wet AMD (newly/recently diagnosed) - (4FRONT-1 & 2) Phase 3
Randomized against aflibercept 2 mg; enrollment completed February 2026 (4FRONT-1) and June 2026 (4FRONT-2), ~4 months ahead of plan. 52-week topline H1’27 for 4FRONT-1, H2’27 for 4FRONT-2. PRISM Phase 2b two-year data (July 2026, n=45):…
Randomized against aflibercept 2 mg; enrollment completed February 2026 (4FRONT-1) and June 2026 (4FRONT-2), ~4 months ahead of plan. 52-week topline H1’27 for 4FRONT-1, H2’27 for 4FRONT-2. PRISM Phase 2b two-year data (July 2026, n=45): 3e10 vg/eye selected as RP3D with maintained BCVA, CST control, and durable treatment-burden reduction; mild intraocular inflammation in 2.8% within 28 weeks, no new inflammation with 2–4+ years follow-up (n=71 safety population), and no 4D-150-related hypotony, endophthalmitis, vasculitis, or choroidal effusions.
Imetelstat + ruxolinitib — 1L INT-1/INT-2/HR myelofibrosis (IMproveMF) Phase 1
Primary completion expected Q1’27.
Rinatabart sesutecan (Rina-S) — 2-4L Platinum Resistant Ovarian Cancer (RAINFOL-02) Phase 3
Primary completion expected Q1’27.
MCLA-158 (petosemtamab) — 2-3L R/M HNSCC (LiGeR-HN2) Phase 3
Phase 3 data readout expected H2’26→ Q1’27.
H1’27HRMYHarmony Biosciences EPX-100 (clemizole HCl) — Dravet syndrome (DS) – (ARGUS-3) Phase 3
Phase 3 topline readout expected H1’27, OLE data at AAN’26.
H1’27HRMYHarmony Biosciences EPX-100 (clemizole HCl) — Lennox-Gastaut syndrome (LGS) – (LIGHTHOUSE) Phase 3
Phase 3 topline readout expected H1’27.
IMC-M113V — HIV (functional cure) – (STRIVE) Phase 1/2
Initial Phase 1/2 data expected at CROI’25 at 60-120-300mcg: significant reduction in cell-associated HIV Gag RNA; trend of reduction in intact HIV DNA after multiple doses; viral rebound delayed in some patients, data H1’27 from 1200mcg…
Initial Phase 1/2 data expected at CROI’25 at 60-120-300mcg: significant reduction in cell-associated HIV Gag RNA; trend of reduction in intact HIV DNA after multiple doses; viral rebound delayed in some patients, data H1’27 from 1200mcg cohort.
H1’27IONSIonis Pharmaceuticals Tonlamarsen — Recent hospitalization and concurrent episode of Acute Severe Hypertension (KARDINAL-ASH) Phase 2
First patient in Jun 2026, topline H1’27.
H1’27IOVAIovance Biotherapeutics Lifileucel, LN-144 (AMTAGVI) — Melanoma (post-anti-PD-1) - (C-144-01 coh. 2 & 4) Marketed
MAA resub. 2026, potential UK/CA approvals H1’16, potential CH approval H1’27.
H1’27IOVAIovance Biotherapeutics Lifileucel — Cutaneous Squamous Cell Carcinoma (CSCC), Merkel cell carcinoma (MCC) – multiple ISTs Phase 2
ISTs, initial data H1’27 in CSCC and MCC.
IPH5301 ± chemo+trastuzumab — Solid tumors (CHANCES) Phase 1
Primary completion expected Q1’27.
H1’27JANXJanux Therapeutics JANX007 – mono & + darolutamide — mCRPC Phase 1
Phase 1a data in n=Dec 16, 2024, cohort expansion in taxane-naïve mCRPC initiated Q2’25, daro combo expansion cohort added H1’26, additional data H1’27.
H1’27JAZZJazz Pharmaceuticals Dordaviprone (ONC201) — NDH3 K27M-mutant diffuse glioma (ACTION) Phase 3
Phase 3 data readout expected late 2026; early 2027 → OS IA H1’27.
H1’27KROSKeros Therapeutics KER-065 — DMD Phase 2
First patient in Aug 2026, data H1’27.
Inhaled KB707 + pembro — NSCLC (KYANITE-1) Phase 1/2
Initial combo data at ASCO’26: ORR 31%; DCR 75%; durable responses [monotherapy: ORR 36%], longer follow-up data H1’27 along with registrational plans.
KB801 — Neurotrophic keratitis (EMERALD-1) Phase 1/2
Registrational, readout 2026→ last patient in by year-end 2026 so readout likely delayed to H1’27.
Q1’27LQDALiquidia Corporation LIQ861 (YUTREPIA) — SSc-RP (RE-WARM) Phase 2
Primary completion expected Q1’27.
H1’27LQDALiquidia Corporation LIQ861 (YUTREPIA) — PAH (SPRINT) Phase 4
Primary completion expected H1’27.
Q1’27LXRXLexicon Pharmaceuticals Sotagliflozin — Hypertrophic cardiomyopathy (oHCM & nHCM) – (SONATA-HCM) Phase 3
Fully enrolled, topline Q1’27.
Q1’27LXRXLexicon Pharmaceuticals LX9851 — Obesity & associated metabolic disorders Phase 1
Primary completion expected Q1’27.
VDPHL01 — Androgenetic Alopecia, Pattern hair loss (females) - (Study 306) Phase 2/3
4.5 mg once- or twice-daily; topline H1’27 on 6-month target-area hair count, with patient-reported outcomes assessed in the Phase 3 portion. Positive open-label Phase 2 ‘Study 207’ data July 2026 (6 months): ‘improved’ or ‘much improved’…
4.5 mg once- or twice-daily; topline H1’27 on 6-month target-area hair count, with patient-reported outcomes assessed in the Phase 3 portion. Positive open-label Phase 2 ‘Study 207’ data July 2026 (6 months): ‘improved’ or ‘much improved’ at month 6 in 88.9% (once-daily) and 90% (twice-daily), mean non-vellus hair-count gains of 22.7 and 23.3 hairs/cm² respectively, with onset as early as month 2.
Q1’27MIRMMirum Pharmaceuticals Volixibat — Primary biliary cholangitis (PBC) – (VANTAGE) Phase 2
Potential registrational, interim data Jun 2024, phase 2b fully enrolled as of Aug 2026, topline Q1’27.
Inhaled human insulin (AFREZZA) — Newly Diagnosed T1D (6-17y) – (INHALE-1st) Phase 2
Primary completion expected Q1’27.
Q1’27NRIXNurix Therapeutics Bexobrutideg (NX-5948) — R/R B-cell malignancies (NX-5498-301) Phase 1
Primary completion expected Q1’27.
NXP900 — YES1/SRC-driven solid tumors: NSCLC - adeno (coh. 1), NSCLC - SCC (coh. 2), RCC (coh. 3), mesothelioma (coh. 4), other (coh. 5) Phase 1
Primary completion expected Q1’27.
OCU400 — Retinitis pigmentosa (gene-agnostic) - (liMeliGhT) Phase 3
Last patient in early-March 2026, rolling submission initiation planned Q3’26 and completion mid-2027, 1-year topline Q1’27, primary endpoint =.
Q1’27OCULOcular Therapeutix OTX-TKI (AXPAXLI) — Wet AMD (SOL-1 & SOL-R) Phase 3
SOL-1 positive topline mid-February 2026 (superiority to Eylea standard dose at week 36); 505(b)(2) filing planned Q4’26 on SOL-1 one-year data plus SOL-R interim safety. August 2026 SOL-R protocol amendment to maximize the label: new key…
SOL-1 positive topline mid-February 2026 (superiority to Eylea standard dose at week 36); 505(b)(2) filing planned Q4’26 on SOL-1 one-year data plus SOL-R interim safety. August 2026 SOL-R protocol amendment to maximize the label: new key secondary of superiority to aflibercept 8 mg (Eylea HD) at week 96 and prevention of fibrosis/atrophy versus aflibercept 2 mg, pushing the non-inferiority SOL-R topline to Q1’28 from Q1’27.
Q1’27OCULOcular Therapeutix OTX-TKI (AXPAXLI) — NPDR incl. DME (HELIOS-2 & HELIOS-3) Phase 3
Primary completion expected Q1’27.
Palazestrant (OP-1250) — 2-3L ER+/HER2- metastatic breast cancer (OPERA-03) Phase 3
Phase 3 topline readout expected Fall’26→ Q1’27, ESMO’26 phase 1 data: mPFS 4.6m and CBR 40% in overall pop; mPFS 5.6 months and CBR 52% in ESR1m pop. MPFS 7.2 months and CBR 48% in 2-3L overall pop; mPFS 7.3 months and CBR 59% in 2-3L…
Phase 3 topline readout expected Fall’26→ Q1’27, ESMO’26 phase 1 data: mPFS 4.6m and CBR 40% in overall pop; mPFS 5.6 months and CBR 52% in ESR1m pop. MPFS 7.2 months and CBR 48% in 2-3L overall pop; mPFS 7.3 months and CBR 59% in 2-3L ESR1m pop.
H1’27PBYIPuma Biotechnology Alisertib — 2L+ SCLC following on or after 1 prior Pt-based CT & anti-PD-L1 combo (ALISCA-Lung1) Phase 2
Interim data May 2026: ORR 6.7-11.5% across 50-60mg BID dose levels, 70 mg BID cohort initiated in 2026, presented at conference Q4’26, additional data H2’26/H1’27, expansion of enrollment in chemo-naïve patients H2’26 to obtain more data…
Interim data May 2026: ORR 6.7-11.5% across 50-60mg BID dose levels, 70 mg BID cohort initiated in 2026, presented at conference Q4’26, additional data H2’26/H1’27, expansion of enrollment in chemo-naïve patients H2’26 to obtain more data in biomarker focused pops, primary completion H2’27.
Deucrictibant (IR) — Hereditary angioedema (HAE) – on‑demand (acute attacks) – (RAPIDe-3) Registration
Positive phase 3 topline Dec 2025, presented at AAAAI’26, PDUFA Apr 23, 2027, also under review by EMA. Timing updated Oct 7, 2026 per company guidance: now guides H1’27 (was Apr’27).
H1’27PRQRProQR Therapeutics AX-0810 — Cholestatic diseases, biliary atresia (BA) Phase 1
Phase 1 in healthy volunteers (3, 6, 9 mg/kg): June 2026 target-engagement data (n=22, cohorts 1–2) showed clear NTCP engagement with dose-dependent serum total bile acid increases up to 8x, above the company’s predefined 2x threshold,…
Phase 1 in healthy volunteers (3, 6, 9 mg/kg): June 2026 target-engagement data (n=22, cohorts 1–2) showed clear NTCP engagement with dose-dependent serum total bile acid increases up to 8x, above the company’s predefined 2x threshold, plus statistically significant increases in urinary conjugated bile acid excretion and post-challenge TUDCA; no hormone changes. Biliary atresia selected as lead indication, with a China investigator-initiated trial in pediatric patients to inform a potential pivotal Phase 2 (first patient in mid’27, interim mid’28). 9 mg/kg data by year-end 2026; initial IIT data H1’27.
H1’27PRQRProQR Therapeutics AX-0422 — Hurler Syndrome Preclinical
CTA filing and first patient in early 2027, initial clinical data [biomarkers] H1’27.
Moponetug, BMS-986446 (PRX005) — Alzheimer’s disease (TargetTau-1) Phase 2
Primary completion expected H1’27.
H1’27RCUSArcus Biosciences Quemliclustat +GnP — 1L mPDAC (PRISM-1) Phase 3
Last patient in Sept 2025, readout [OS] H1’27.
Surabgene lomparvovec (sura-vec, ABBV-RGX-314) — Wet AMD (ATMOSPHERE, ASCENT) Phase 3
Phase 3 topline readout expected Q4’26, filings H1’27.
H1’27RNACCartesian Therapeutics Descartes-08 — Myositis incl. Antisynthetase Syndrome (ASyS) and dermatomyositis (DM) – (TRITON) Phase 2
Potential registrational RCT. blinded DSMB IA on efficacy at week 24 after n=10 to select the final sample size, first patient in Apr 2026, data from subset of patients H1’27 to inform path to registration [primary completion year-end…
Potential registrational RCT. blinded DSMB IA on efficacy at week 24 after n=10 to select the final sample size, first patient in Apr 2026, data from subset of patients H1’27 to inform path to registration [primary completion year-end 2027].
H1’27RNACCartesian Therapeutics In vivo Descartes-08 — Generalized myasthenia gravis Phase 1
First patient in H2’26, initial data H1’27, dose-escalation. IV infusions at multiple dose levels using BOIN adaptive design, approach potential eliminating ex vivo manuf. step.
H1’27RXRXRecursion Pharmaceuticals REC-617 – mono/combo — Advanced solid tumors Phase 1/2
Combo safety/PK data H1’27.
H1’27RXRXRecursion Pharmaceuticals REC-3565 — B-cell malignancies Phase 1
Safety/PK data H1’27.
SEP‑479 — Hypoparathyroidism Phase 1
First patient in Q1’26, PK supportive of OD dosing, Aug 2026 update: dosing for MAD cohorts extended to 14d “to fully characterize steady-state PKs”, SAD/MAD topline year-end 2026/early 2027 → Q1’27.
Q1’27SGMTSagimet Biosciences TVB-3567 — Solid tumors Phase 1
Primary completion expected Q1’27.
H1’27SLDBSolid Biosciences SGT-003 — DMD - US (INSPIRE) Phase 1/2
Primary completion expected H1’27.
Q1’27SPRYARS Pharmaceuticals ARS-2 (intranasal epinephrine) — Acute Flares in CSU/urticaria Phase 2
First patient in Q3’25, interim data Q1’27 [primary completion mid-2027].
Q1’27SRPTSarepta Therapeutics Delandistrogene moxeparvovec, SRP-9001 (ELEVIDYS) — DMD (Ambulatory & non-ambulatory, various age, screening, comedication - (ENDEAVOR) Phase 1
Cohort 8 12-week readout H2’26→ Q1’27 after delay in enrollment.
H1’27STOKStoke Therapeutics STK-002 — Autosomal dominant optic atrophy (ADOA) Phase 1
Ph 1 ADOA patients, safety/efficacy data H1’27.
Molgramostim inhalation solution (MOLBREEVI) — Autoimmune pulmonary alveolar proteinosis (aPAP) Registration
PDUFA decision expected Nov 22, 2026, 3 months PDUFA date extension Apr 2026 after newly submitted data considered ‘major amendment’, MAAs under review by EMA and MHRA with potential approvals Q1’27 and Q4’26 respectively, presented at…
PDUFA decision expected Nov 22, 2026, 3 months PDUFA date extension Apr 2026 after newly submitted data considered ‘major amendment’, MAAs under review by EMA and MHRA with potential approvals Q1’27 and Q4’26 respectively, presented at ATS’26: continuous LT improvement in lung function and respiratory HR-QoL.
H1’27TARSTarsus Pharmaceuticals TP-05 — Lyme disease (Calliope) Phase 2
RCT, first patient in Marc 2026 and last patient in Q2’26, topline H1’27.
H1’27TARSTarsus Pharmaceuticals TP-04 — Ocular rosacea (KORE) Phase 2
Phase 2 topline readout expected H1’27.
Q1’27TECXTectonic Therapeutic TX45 — Group 2 PH-HFpEF (CpcPH-enriched) – (APEX) Phase 2
Last patient in Jun 2026, trial including enriched pop. Of more severe dzz subtype [pre- and post-capillary pulmonary hypertension *CpcPH*] with pulmonary vascular resistance *PVR*>3 Wood Units *WU* at bsln. Enriched pop. ~70% of overall…
Last patient in Jun 2026, trial including enriched pop. Of more severe dzz subtype [pre- and post-capillary pulmonary hypertension *CpcPH*] with pulmonary vascular resistance *PVR*>3 Wood Units *WU* at bsln. Enriched pop. ~70% of overall patients enrolled, IDMC recommended Jul 2026 to continue the study witho modification, readout 2026→ early Q1’27.
Q1’27TRDAEntrada Therapeutics ENTR-601-44 — DMD (exon 44 skipping, ambulatory pts) – (ELEVATE-44-201) Phase 1/2
Positive cohort 1 MAD topline May 2026 (EU/UK): lower-than-expected Cmax and AUC in pediatric patients versus healthy volunteers and adult NHPs at 8 mg/kg, but statistically significant functional improvement on time-to-rise velocity (mean…
Positive cohort 1 MAD topline May 2026 (EU/UK): lower-than-expected Cmax and AUC in pediatric patients versus healthy volunteers and adult NHPs at 8 mg/kg, but statistically significant functional improvement on time-to-rise velocity (mean +0.115 vs placebo), 2.36% dystrophin increase over 4% baseline, and 2.31% exon skipping over 2.66% baseline. Open-label cohort 1 data by year-end 2026; cohort 2 at 12 mg/kg slipped to Q1’27, with cohort 3 at 18 mg/kg if needed; company to re-engage FDA on a higher US starting dose.
H1’27TRDAEntrada Therapeutics ENTR-601-45 — DMD (exon 45 skipping, ambulatory pts) – (ELEVATE-45-201) Phase 1/2
Q6W dosing, 5mg/kg cleared by DMC Jun 2026→ recom to escalate to 10mg/kg in cohort 2, EU cohort 1 MAD PK+dystro data mid-2026→ Oct 2026, data from cohort 2 MAD H1’27, cohort 3 MAD [15mg/kg] if needed.
UGN-104 — Low‑grade Upper Tract UC (LG‑UTUC) Phase 3
Primary completion expected Q1’27.
Q1’27VIRVir Biotechnology Tobevibart + elebsiran — Hepatitis delta (HDV) Not Virologically Suppressed With Bulevirtide - (ECLIPSE 2) Phase 3
Phase 3 topline readout expected Q1’27.
Q1’27VIRVir Biotechnology Tobevibart + elebsiran — Hepatitis delta (HDV) - (ECLIPSE 3, H2H Bulevirtide) Phase 2
Phase 2 topline readout expected Q1’27, part of pivotal program.
H1’27VKTXViking Therapeutics VK2735 - SC — Obesity (maintenance) Phase 1
SC maintenance readout Q3’26, additional cohorts disclosed Apr 2026 [10 and 15mg Q4W+5 and 10mg Q2W added], oral maintenance to proceed after SC maintenance with data H1’27.
Q1’27VNDAVanda Pharmaceuticals VCA-894A — CMT2S Phase 1/2
Primary completion expected Q1’27.
H1’27VNDAVanda Pharmaceuticals Milsaperidone (BYSANTI) — MDD (adjunct) Phase 3
Phase 3 topline readout expected 2026→ Q1’27→ H1’27.
Telitacicept — Generalized myasthenia gravis (Global) – (UPSTREAM MG, formerly RemeMG) Phase 3
Phase 3 topline readout expected H1’27.
Selatogrel — Acute MI / acute coronary syndrome (SOS-AMI) Phase 3
Last patient in H2’26, readout H1’27.
Cenerimod — Systemic lupus erythematosus (SLE) – (OPUS-1&-2) Phase 3
Last patient in H2’26, readout H1’27.
H1’27VTVTvTv Therapeutics Cadisegliatin (TTP399) – hybrid closed loop — T1D (Hybrid CATT1) Phase 2
Primary completion expected H1’27.
H1’27XENEXenon Pharmaceuticals Azetukalner (XEN1101) — Primary generalized tonic clonic seizures (X-ACKT) Phase 3
Primary completion expected H1’27.
H1’27XENEXenon Pharmaceuticals Azetukalner (XEN1101) — Major depressive disorder (X-NOVA2 & X-NOVA3) Phase 3
X-NOVA2 topline H1’27 [vs primary completion Q3’26 on ct.gov], X-NOVA3 primary completion Q2’27 but data disclosure seemingly after that point in time.
Obexelimab (XmAb5871) — Systemic lupus erythematosus (SLE) – (SunStone) Phase 2
Phase 2 topline readout expected Q4’26 in overall and biomarker pops, potential SLE phase 3 SPI H1’27.
ZL-1109 (elegrobart, VRDN-003) — TED (Active/Chronic) - (CN) Phase 3
Primary completion expected Q1’27.
ZL-1503 — Moderate-to-severe atopic dermatitis Phase 1
Part A SAD data [4 dose levels] in healthy volunteers H2’26 from global portion, Part B MAD data [2 dose levels] in AtD H1’27.
ZL-6201 — Solid tumors incl. sarcoma Phase 1
Global, proof-of-concept data 2027→ dose-escalation data H1’27.
Tibulizumab (ZB-106) — Systemic sclerosis (TibuSURE) Phase 2
Over-enrolled, topline H1’27.
early’27ABEOAbeona Therapeutics TSHA-102 — Rett syndrome (REVEAL & ASPIRE) Phase 3
Taysha trials, seamless phase 1/2/3 REVEAL and phase 3 ASPIRE, primary completion 2031, REVEAL 6 months IA topline report H1’27 but availability early 2027 then Taysha to discuss the data with FDA. FDA feedback on next steps toward BLA…
Taysha trials, seamless phase 1/2/3 REVEAL and phase 3 ASPIRE, primary completion 2031, REVEAL 6 months IA topline report H1’27 but availability early 2027 then Taysha to discuss the data with FDA. FDA feedback on next steps toward BLA submission H1’27, BLA submission to include ASPIRE safety data to support broad label in ≥2y pop.
ABS-201 — Androgenetic alopecia (HEADLINE) Phase 1/2
First patient in Dec 2025, phase 1 SAD part readout Jun 2026: good safety and t1/2 ~65d+ supporting Q2M-Q3M dosing, phase 2 part 13-week readout H2’26 and 26-week readout early 2027.
early’27ALNYAlnylam Pharmaceuticals Cemdisiran + pozelimab — Paroxysmal nocturnal hemoglobinuria (ACCESS-1/-2/-EXT) Phase 3
Primary completion expected early 2027.
early’27BBIOBridgeBio Pharma Encaleret — Autosomal dominant hypocalcemia type 1 (CALIBRATE) Registration
PDUFA decision expected May 8, 2027, NDA filed mid-May 2026, launch early 2027 → shift since no Priority Review, positive phase 3 topline Oct 2025.
early’27ETONEton Pharmaceuticals ET-700, zinc acetate ER — Wilson disease Phase 1
Pilot study first patient in Apr 2026, RCT vs zinc acetate [IR], readout H2’26, potential transition to pivotal trial early 2027.
early’27HRMYHarmony Biosciences BP1.15205, BP-205 — Narcolepsy & other sleep/wake disorders Phase 1
Aug 2026 update including SAD topline in healthy volunteers: short Tmax 30-75min. mean t1/2 ~ 25hrs across dose levels supporting OD admin. dose proportional PK. no gender diff, MAD data in healthy volunteers Q4’26. Phase 1b in…
Aug 2026 update including SAD topline in healthy volunteers: short Tmax 30-75min. mean t1/2 ~ 25hrs across dose levels supporting OD admin. dose proportional PK. no gender diff, MAD data in healthy volunteers Q4’26. Phase 1b in sleep-deprived healthy volunteer first patient in Q3’26 with data early 2027, multiple phase 2s first patient in mid-2027.
early’27IDYAIDEAYA Biosciences IDE034 — Solid tumors Phase 1
First patient in end-Feb 2026, data H2’26/early 2027.
INCA035784 — Myelofibrosis or ET with CALR mutation Phase 1/2
Phase 1 data early 2027.
Axatilimab — 1L chronic GVHD Phase 3
Phase 3 data readout expected early 2027.
INCA33890 — Solid tumors Phase 1
Primary completion expected early 2027.
INCA33890 mono & combos (chemo, INCB161734, others) — Solid tumors w/ KRAS G12D muts Phase 1
Primary completion expected early 2027.
INCB161734 mono & combos (chemo, INCA33890, others) — KRAS G12D-mutated solid tumors Phase 1
Primary completion expected early 2027, phase 1 data at ESMO’26 for 734+GnP/mFOLFIRINOX in 1L PDAC and 734 ±cetux in 2L+ CRC.
Povorcitinib — Hidradenitis suppurativa - moderate/severe Registration
Filing early 2026, potential EU approval late 2026 and US approval early 2027.
early’27IRONDisc Medicine Bitopertin — Erythropoietic porphyrias (EPP & XLP) – (APOLLO) Phase 3
Confirmatory, readout Q4’26 [vs early 2027] and n=183 [from 150 planned].
early’27KYTXKyverna Therapeutics KYV-101 (miv-cel, mivocabtagene autoleucel) — Progressive Multiple Sclerosis (KYSA-7, ISTs) Phase 2
Proof-of-concept data in n=5 with up to 12 months follow-up at conference in May 2026, additional IST phase 1 data H2’26, dev. strategy update early 2027.
early’27MAZEMaze Therapeutics MZE829 — APOL1-Mediated Kidney Disease (AMKD) – (HORIZON) Phase 2
Positive proof-of-concept end March 2026 but n=12/15 evaluated for efficacy and differences in effect size across subgrps, data from all 3 cohorts of n=10-15 late 2026/early 2027 including AMKD witho diabetes. AMKD with diabetes. AMKD with…
Positive proof-of-concept end March 2026 but n=12/15 evaluated for efficacy and differences in effect size across subgrps, data from all 3 cohorts of n=10-15 late 2026/early 2027 including AMKD witho diabetes. AMKD with diabetes. AMKD with FSGS, pivotal trial being planned.
early’27OVIDOvid Therapeutics OV4071 — Ketamine challenge with biomarkers Preclinical
Ketamine challenge phase 1 study. FIH H2’26, goal = further characterize potential PD effects and proof-of-mechanism using electrophysiology and biomarkers, results year-end 2026/early 2027.
early’27TGTXTG Therapeutics Ublituximab - SC — Relapsing MS (RMS) Phase 3
Last patient in Apr 2026, topline year-end 2026/early 2027, non-inferiority SC Q2M and SC Q3M vs ubli IV.
early’27TRAXFirst Tracks Bio. ANB101 — Inflammatory diseases Phase 1
Phase 1a in healthy volunteers completed, potential dev. approach informed by clinical and translational evidence + competitor registrational data in SLE and CLE anticipated in Q4’26/early 2027.
Q2’27ARWRArrowhead Pharmaceuticals Daplusiran/Tomligisiran (ARO-HBV) — Hepatitis B Virus (HBV) – (B-UNITED) Phase 2
Primary completion expected Q2’27.
Q2’27IDYAIDEAYA Biosciences Darovasertib (IDE196) — Uveal melanoma (Neoadjuvant & pot. Adjuvant) – (OptimUM-09) Phase 2
Primary completion expected Q2’27.
Ruxolitinib cream (Opzelura) — Pediatric vitiligo - mild/moderate Phase 3
Primary completion expected Q2’27.
Q2’27MISTMilestone Pharmaceuticals Etripamil nasal spray (CARDAMYST) — Paroxysmal supraventricular tachycardia (PSVT) - Pediatrics (NODE-202) Phase 2
Primary completion expected Q2’27.
Rilparencel — Type 2 diabetes CKD (Stage 3b/4) – prevent/delay ESKD (PROACT 1) Phase 3
AA-supporting cohort fully enrolled; full enrollment H2’26, AA-enabling surrogate endpoint [eGFR slope] readout Q2’27, confirmatory composite time-to-event analysis topline H2’29.
Q2’27TLSATiziana Life Sciences Foralumab (TZLS-401) — Multiple System Atrophy (MSA) Phase 2
Primary completion expected Q2’27.
Q2’27VKTXViking Therapeutics VK2735 - SC — Obesity ± T2D (VANQUISH-1/-2) Phase 3
VQ1 78-week readout potential Q2’27, VQ2 78-week readout potential Q4’27, both fully enrolled as of Apr 2026, 42-week LTE.
Q2’27XENEXenon Pharmaceuticals Azetukalner (XEN1101) — Major depressive disorder (X-NOVA2 & X-NOVA3) Phase 3
X-NOVA2 topline H1’27 [vs primary completion Q3’26 on ct.gov], X-NOVA3 primary completion Q2’27 but data disclosure seemingly after that point in time.
XmAb412 — IBD, UC,CD Phase 1
Primary completion expected Q2’27.
Obefazimod (ABX464) — Crohn's disease (ENHACE-CD) Phase 2
12-Week induction topline late 2026→ mid-2027.
2027ACRSAclaris Therapeutics ATI-2138 — Alopecia Areata Phase 2
First patient in H1’26 with topline 2027 → AA deprioritized, LP selected as lead indication.
Jun’27AKBAAkebia Therapeutics Praliciguat — Focal Segmental Glomerulosclerosis (FSGS) Phase 2
Primary completion expected Jun 2027.
2027AKBAAkebia Therapeutics Ebribafusp (AKB-097, ADX-097) — C3G Glomerulopathy, IgAN, LN Phase 2
Basket trial, first patient in Aug 2026, initial data 2027, primary completion Q1’28.
AKY-2519 — mCRPC (PLUVICTO-naïve & PLUVICTO-XP cohorts) - (BActinium-1) Phase 1
First patient in May 2026, preliminary data 2027, primary completion year-end 2027.
mid’27ALNYAlnylam Pharmaceuticals ALN-6400 — Von Willebrand Disease Phase 2
Primary completion expected mid-2027.
Feb’27ARQTArcutis Biotherapeutics Roflumilast cream 0.05%, ARQ-151 (ZORYVE) — Atopic dermatitis (children 3-24mo) – (INTEGUMENT-INFANT) Registration
PDUFA decision expected Feb 23, 2027, sNDA filed end-Apr 2026, phase 2 positive topline Feb 2026.
ARV-393 – mono & combos + glofitamab — R/R NHL Phase 1
Dose-escalation. Ongoing, interim monotherapy dose-escalation data in R/R NHL H2’26 [ASH’26?]. additional monotherapy dose-escalation data in B-/T-CL mid-2027, first patient in H1’26 in combo with glofitamab in DLBCL. data mid-2027, Aug…
Dose-escalation. Ongoing, interim monotherapy dose-escalation data in R/R NHL H2’26 [ASH’26?]. additional monotherapy dose-escalation data in B-/T-CL mid-2027, first patient in H1’26 in combo with glofitamab in DLBCL. data mid-2027, Aug 2026: responses in B-/T-CLs at dose levels < predicted effective exposure level.
mid’27ARWRArrowhead Pharmaceuticals ARO-MAPT — Alzheimer’s Disease / Taupathies Phase 1/2
Primary completion expected mid-2027.
mid’27ASNDAscendis Pharma TransCon PTH — Hypoparathyroidism (adults – dosing expansion ≥30μg/d) – (PaTHway60) Phase 3
Primary completion expected mid-2027.
May’27AXSMAxsome Therapeutics AXS‑12 (reboxetine) — Narcolepsy Registration
NDA filing around end-Q1’26, PDUFA May 1, 2027, phase 3s toplined positive in 2025.
2027AXSMAxsome Therapeutics SUNOSI (solriamfetol) — Shift Work Disorder (SUSTAIN) Phase 3
Phase 3 topline readout expected 2026→ 2027.
Felzartamab — IgA nephropathy Phase 3
Primary completion expected mid-2027.
BGE-102 ± anti-VEGF — DME (QUELL-DME) Phase 1/2
First patient in mid-2026, topline mid-2027.
Tinlarebant (LBS-008) — Stargardt disease (STGD1) – (DRAGON I) Registration
PDUFA decision expected Feb 12, 2027 with Priority Review, rolling submission initiated in Apr 2026 under BTD. completion Jun 2026, positive 24 months topline Dec 2025; positive secondary endpts at ASRS’26.
Tinlarebant (LBS-008) — Geographic atrophy (GA) – (PHOENIX) Phase 3
Primary completion expected mid-2027.
feb’27BMRNBioMarin Pharmaceutical Vosoritide (VOXZOGO) — Achondroplasia (children with open growth plates) Marketed
PDUFA decision expected 28 feb’27 for conversion to full approval.
2027BMRNBioMarin Pharmaceutical BMN 333 — Achondroplasia Phase 2/3
Phase 2/3 data expected update 2027.
Pumitamig (BNT327, BMS986545) + chemo — 1/2L+ ES-SCLC (coh1: 1L; coh2: 2L: coh3: 3L) Phase 2
Primary completion expected mid-2027.
Pumitamig (BNT327, BMS986545) + GnP — 1L PDAC (BNT327-09) Phase 2
Primary completion expected 2027.
Pumitamig (BNT327, BMS986545) + chemo — 1L MSS unresectable mCRC (CN) Phase 2
Primary completion expected 2027.
Rese-cel (CABA-201) — Dermatomyositis (RESET-Myositis) Phase 1/2/3
Registrational RESET-Myositis cohort for the CD19 CAR-T rese-cel: EULAR 2026 update (n=10 dermatomyositis + antisynthetase syndrome) showed 80% meeting the primary endpoint, with 83% of 6 dermatomyositis patients achieving an…
Registrational RESET-Myositis cohort for the CD19 CAR-T rese-cel: EULAR 2026 update (n=10 dermatomyositis + antisynthetase syndrome) showed 80% meeting the primary endpoint, with 83% of 6 dermatomyositis patients achieving an immunomodulator-free moderate-to-major TIS response at week 16, maintained through up to 1.5 years follow-up. Potential BLA filing H2’27 on a 17-patient single-arm cohort including outpatient dosing; topline mid’27 on moderate/major TIS response off immunomodulators.
mid’27CBIOCrescent Biopharma CR-001 — Advanced solid tumors (ASCEND) - global Phase 1/2
Preliminary dose-escalation and backfill cohort data Q1’27 including 1L NSCLC backfill cohort, initial combo data with SOC chemos in 1L and 2L+ ~mid-2027.
mid’27CBIOCrescent Biopharma CR-001 — Advanced solid tumors - CN Phase 1/2
First patient in H1’26→ Q2’26, initial data ~mid-2027.
mid’27CBIOCrescent Biopharma CR-001 + sac-TMT — Advanced NSCLC - CN Phase 2
Kelun China trial, first patient in H2’26, initial data mid-2027.
Feb’27CGEMCullinan Therapeutics Zipalertinib — 2L+ EGFR ex20ins-mut NSCLC after CT or amivantamab-CT (REZILIENT1) Registration
Registrational phase 2b, rolling NDA filing completion Q1’26, PDUFA Feb 27, 2027.
2027CGTXCognition Therapeutics Zervimesine (CT1812) incl. background anti-Abeta mAbs — Alzheimer’s disease (early to mild) – (START) Phase 2
Phase 2 topline readout expected H2’27/data 2027, 15% of patients on background anti-Abeta mAbs.
2027CRVSCorvus Pharmaceuticals Soquelitinib (CPI-818) — Asthma Phase 1
First patient in 2026, readout 2027.
2027CRVSCorvus Pharmaceuticals Soquelitinib (CPI-818) — HS Phase 2
First patient in 2026, readout 2027.
2027DNLIDenali Therapeutics TAK-594 / DNL593 (PTV:PGRN) — Frontotemporal dementia-GRN Phase 1/2
Fully enrolled; n=40, initial data 2026→ year-end 2026→ 2027 allowing for period to assess treatment effect on biomarkers including NfL.
2027DNLIDenali Therapeutics DNL952 (ETV:GAA) — Pompe disease Phase 1
IND clinical hold lifted Jan 2026, first patient in Q2’26 initial data 2027.
2027DSGNDesign Therapeutics DT-168 — Fuchs endothelial corneal dystrophy Phase 2
Biomarker trial, corneal endothelium biomarker data H2’26→ 2027 due to delay in supply of DT-168 blow-fill-seal eye droppers.
2027DSGNDesign Therapeutics DT-818 — Myotonic dystrophy type 1 Phase 1
MAD phase 1 first patient in end-Jun 2026 in DM1 patients, mis-splicing correction data 2027.
Jan’27DYNDyne Therapeutics Zeleciment rostudirsen (DYNE-251) — DMD (exon 51) – (DELIVER) Registration
PDUFA decision expected Jan 21, 2027 with Priority Review, registrational phase 1/2 expansion cohort for AA, NDA filed for AA based on dystrophin surrogate end May 2026 at 20mg/kg Q4W, potential launch Q1’27, data presented at MDA early…
PDUFA decision expected Jan 21, 2027 with Priority Review, registrational phase 1/2 expansion cohort for AA, NDA filed for AA based on dystrophin surrogate end May 2026 at 20mg/kg Q4W, potential launch Q1’27, data presented at MDA early March 2026.
EDIT-401 — Hyperlipidemia, ASCVD Preclinical
PC proof-of-concept data May 2026 at EASC [~90%+ reductions in LDL-C, Lp(a), and ApoB in non-human primates], IND/CTA filing mid-2026→ filed in AU/NZ Aug 2026, 2-part in vivo human proof-of-concept data from phase 1/2 by year-end 2026→…
PC proof-of-concept data May 2026 at EASC [~90%+ reductions in LDL-C, Lp(a), and ApoB in non-human primates], IND/CTA filing mid-2026→ filed in AU/NZ Aug 2026, 2-part in vivo human proof-of-concept data from phase 1/2 by year-end 2026→ Q1’27 with complete dose-finding part 1 topline 2027.
2027ENTAEnanta Pharmaceuticals Zelicapavir — RSV (pediatrics) – (LOTUS) Phase 2
First patient in Aug 2026, phase 2b in pediatric patients, topline 2027, 1st phase 2 presented at ESCMID’26. ‘data supporting further clinical dev’.
2027ENTAEnanta Pharmaceuticals Zelicapavir — RSV (high-risk adults) – (RESOLVE) Phase 2/3
Seeking FDA alignement in Q2’26 with phase 3 first patient in H2’26 → Jun 2026 program: phase 2b/3 first patient in Q4’26 in HR adults with phase 2b part topline 2027, 1st phase 2b RSVHR presented at ESCMID’26.
ERAS-4001 — KRASm solid tumors (BOREALIS-1) Phase 1
Preliminary monotherapy data H2’26, monotherapy expansion and comb dose-escalation FPIs 2027.
mid’27FULCFulcrum Therapeutics SLTE-1009 — Migraine Phase 1
First patient in in AU Jul 2026, safety/PK topline mid-2027.
mid’27GYREGyre Therapeutics Hydronidone (F351) — Chronic hepatitis B (CHB)-associated liver fibrosis (CN) Registration
China NDA filing accepted for review May 2026 for conditional approval, priority review granted→ potential China approval ~mid-2027.
mid’27HRMYHarmony Biosciences Pitolisant (WAKIX) — Prader-Willi syndrome (PWS) Phase 3
Phase 3 topline readout expected 2026→ mid-2027.
2027HRMYHarmony Biosciences Pitolisant HD — Fatigue in Narcolepsy (ONSTRIDE 1) Phase 3
Phase 3 data readout expected 2027.
2027HRMYHarmony Biosciences Pitolisant HD — Sleep Inertia in Idiopathic Hypersomnia (ONSTRIDE 2) Phase 3
Phase 3 data readout expected 2027.
IMA402 — Melanoma, gynecologic cancers, others Phase 1
Dose-exp, final RP2D H1’26, update from larger n in melanoma/gyn. Cancers in monotherapy and ICI combo H2’26 → potential conversion of Phase 1b cohorts into registrational Phase 2 trials, additional phase 1b cohorts in monotherapy and…
Dose-exp, final RP2D H1’26, update from larger n in melanoma/gyn. Cancers in monotherapy and ICI combo H2’26 → potential conversion of Phase 1b cohorts into registrational Phase 2 trials, additional phase 1b cohorts in monotherapy and combos [ICI or SOC] in later and earlier LoT opened mid-2026 including sqNSCLC cohort evaluating 401+402 combo with data 2027, phase 1b data at RP2D range across multiple cancers at ESMO’26.
IMVT-1402 — Graves’ Disease Phase 3
Phase 3 data readout expected 2027.
IMVT-1402 — gMG Phase 3
Primary completion expected 2027.
Ozekibart (INBRX-109) — Unresectable metastatic conventional chondrosarcoma Registration
Registrational phase 2, positive topline Sept 2025, BLA filed Apr 2026 and PDUFA Apr 14, 2027.
INBRX-106 + pembrolizumab — Stage II-III TNBC (Neoadjuvant) Phase 2
Primary completion expected mid-2027.
Lacutamab (IPH4102) — CTCL incl. Sézary syndrome & mycosis fungoides (TELLOMAK) Phase 2
Potential registrational, last data at ASCO’25, potential AA sought in 2L SS post-moga in 2027 once confirmatory study is ‘well underway’ [FDA flexibility guided for CTCL].
Bitopertin — Erythropoietic porphyrias (EPP & XLP) Phase 3
CNPV award, PFUDA pushed to early Feb 2026 → CRL received Feb 2026. FDA requesting APOLLO phase 3 data from year-end 2026/Q4’26 readout for traditional approval pathway, then CRL response submission and potential US approval mid-2027.
Selcodebart (DISC-0974) — Anemia associated with IBD Phase 2
First patient in Feb 2026, initial data 2027 [primary completion Q4’26].
mid’27JAZZJazz Pharmaceuticals JZP351 + intensive chemo (cyta+dauno) — 1L ND AML (Intermediate-/High-risk) – (AMLSG 30-18) Phase 3
Primary completion expected mid-2027.
JADE-101 — IgA nephropathy (JUNIPER) Phase 2
First patient in May 2026, interim data 2027.
JADE-201 — RA Phase 1
First patient in May 2026, SAD in RA patients, interim data 2027 [primary completion H2’27].
mid’27KLRAKailera Therapeutics Ribupatide HD (KAI-9531, HRS9531) — Obesity w/o T2D (BMI ≥35) - US Phase 2
RCT. 10-13-16-20mg x48 weeks, fully enrolled, readout 2027→ mid-2027.
2027KLRAKailera Therapeutics KAI-4729 (HRS-4729) — Obesity Preclinical
Global phase 1 first patient in 2026 with topline 2027, positive China SAD/MAD phase May 1, 2026 [1-4-8-12mg QW vs ribu 4mg QW vs placebo QW]: mean BW loss at week 12 “up to” 16% at 12mg vs 5.4% placebo.
KB408 — Alpha-1 antitrypsin deficiency-lung disease (AATD-LD) – (SERPENTINE-1) Phase 1/2
Cohort 2B repeat dose data 2026→ 2027.
2027LKFTLakefront Biotherapeutics Gamgertamig (OM336, CM336) — Seropositive AIDs, Active Sjogren's Disease, Idiopathic Inflammatory Myopathy (global) Phase 1
Phase 1 data readout expected 2027.
2027LKFTLakefront Biotherapeutics Gamgertamig (OM336, CM336) — Autoimmune Cytopenia, AIHA, ITP, APS (CN) Phase 1/2
Primary completion expected 2027.
mid’27LYELLyell Immunopharma Ronde-Cel (rondecabtagene autoleucel, LYL314) — 3L+ CD19-CAR-T-naïve aggressive LBCL (PiNACLE) Phase 3
Single-arm pivotal phase 1/2 expansion, update H2’26, pivotal data readout mid-2027, NDA filing 2027, phase 1/2 update at EHA’26 in n=108: no grade 3+ CRS; 8% grade 3+ ICANS including 8% with prophy vs 16% no-prophy; no change in cell…
Single-arm pivotal phase 1/2 expansion, update H2’26, pivotal data readout mid-2027, NDA filing 2027, phase 1/2 update at EHA’26 in n=108: no grade 3+ CRS; 8% grade 3+ ICANS including 8% with prophy vs 16% no-prophy; no change in cell expansion with prophy; 97% manuf. success rate.
2027MAZEMaze Therapeutics MZE782 — PKU (CIPheR) Phase 2
First patient in during Q2’26, topline 2027.
MPC-06-ID (rexlemestrocel-L) + Hyaluronic Acid — Chronic Low Back Pain (CLBP) due to DDD (MSB-DR004) Phase 3
1st phase 3 MSB-DR003 toplined Feb 2021, MSB-DR004 12 months readout est. H1’27→ topline confirmed mid-2027.
2027NBIXNeurocrine Biosciences NBI-569 — Alzheimer’s Psychosis Phase 1
First Phase 1 data expected 2027.
2027NBIXNeurocrine Biosciences NBI-986 — Movement disorders Phase 1
Initial Phase 1 data expected 2026→ 2027.
2027NBIXNeurocrine Biosciences NBI-890 — Tardive dyskinesia Phase 2
RCT, 1st data 2027; primary completion Q1’27.
2027NBIXNeurocrine Biosciences NBI-355 — Epilepsy Phase 1
Initial Phase 1 data expected 2026→ 2027.
2027NBIXNeurocrine Biosciences NBIP-1435 — Congenital adrenal hyperplasia Phase 1
Phase 1 data readout expected 20206, phase 2 first patient in 2026 and readout 2027.
2027NBIXNeurocrine Biosciences NBIM-1112 — CNS indications Phase 1
First Phase 1 data expected 2027.
2027NBIXNeurocrine Biosciences NBIB-223 — Friedreich’s Ataxia Phase 1
‘Initiating’ as of Aug 2026, 1st data 2027.
2027NBIXNeurocrine Biosciences NBIP-2118 — Obesity Phase 1
First patient in May 2026, initial data 2027.
2027NMRANeumora Therapeutics NMRA-215 – mono & combo — Obesity Preclinical
FIH start H1’26, biomarker data Q3’26, BW loss data by year-end 2026 [Oct 2026 check] FIH program delayed vs sheet: IND filing planned Q4 2026 with Phase 1 start targeted end-2026/Q1 2027 (2026 8-Ks); repeat 13-wk rat toxicity favorable…
FIH start H1’26, biomarker data Q3’26, BW loss data by year-end 2026 [Oct 2026 check] FIH program delayed vs sheet: IND filing planned Q4 2026 with Phase 1 start targeted end-2026/Q1 2027 (2026 8-Ks); repeat 13-wk rat toxicity favorable (July 27, 2026). No human biomarker data yet.
2027NTHINeOnc Technologies NEO100 — Meningioma Phase 2
Phase 2 data readout expected 2027.
Mar’27NTLAIntellia Therapeutics Lonvoguran ziclumeran (lonvo-z, NTLA-2002) — HAE (HAELO) Registration
BLA rolling submission initiated end-Apr 2026 with completion H2’26, positive phase 3 topline end-Apr 2026 [primary and all key secondary endpts met. 87% attack reduction over 6 months. 62% attack-free vs 11% placebo], data presented at…
BLA rolling submission initiated end-Apr 2026 with completion H2’26, positive phase 3 topline end-Apr 2026 [primary and all key secondary endpts met. 87% attack reduction over 6 months. 62% attack-free vs 11% placebo], data presented at EAACI Jun 2026 + NEJM published, potential approval/launch H1’27. Timing updated Oct 7, 2026 per company guidance: now guides Mar’27 (was H1’27).
2027NTLAIntellia Therapeutics AVC-201 — AML Phase 1
Primary completion expected 2027.
OCU400 — Retinitis pigmentosa (gene-agnostic) - (liMeliGhT) Phase 3
Last patient in early-March 2026, rolling submission initiation planned Q3’26 and completion mid-2027, 1-year topline Q1’27, primary endpoint =.
OP-3136 – mono & +palazestrant (mBC) or +fulvestrant (mBC) or +darolutamide (mCRPC) — ER+/HER2- metastatic breast cancer, mCRPC, lung cancer Phase 1
Primary completion expected H2’27, preliminary monotherapy data at ASCO’26 in n=19 across dose levels up 45mg OD: shrinkage in 68%; ORR 15.8% and cORR 10.5%; rapid sustained and sig reduction in lys23 of histone H3 *direct KAT6 target*;…
Primary completion expected H2’27, preliminary monotherapy data at ASCO’26 in n=19 across dose levels up 45mg OD: shrinkage in 68%; ORR 15.8% and cORR 10.5%; rapid sustained and sig reduction in lys23 of histone H3 *direct KAT6 target*; dysgeusia 81% [56% grade 1, 25% grade 2]. Anemia 38% including 6% grade 3 -neutropenia 34% including 28% grade 3, initial combo data mid-2027, mCRPC combo first patient in Q4’26.
2027ORICORIC Pharmaceuticals Enozertinib (ORIC-114) - mono or + SC amivantamab — EGFR ex20ins-mut NSCLC (1L: mono & + SC amivantamab, 2L: mono) Phase 1
Phase 1 data expected H2’26 in 1L EGFR ex20ins monotherapy and +SC amivantamab, potential phase 3 Redwood-1 in monotherapy or combo 2027+.
2027ORICORIC Pharmaceuticals Enozertinib (ORIC-114) — Atypical EGFR/PAAC muts NSCLC (mono, 1L & 2L+) Phase 1
1L monotherapy data at ESMO’26, potential monotherapy phase 3 Redwood-1 2027+.
PGN-EDODM1 — Myotonic dystrophy type 1, DM1 (ages 16-60y) – (FREEDOM2-DM1) Phase 2
FREEDOM2-DM1 in myotonic dystrophy type 1: FDA partial clinical hold (March 2026, pharmacology/toxicology findings) with the trial continuing ex-US. 5 mg/kg data (n=8) showed weak splicing correction and efficacy; August 2026 DSMB…
FREEDOM2-DM1 in myotonic dystrophy type 1: FDA partial clinical hold (March 2026, pharmacology/toxicology findings) with the trial continuing ex-US. 5 mg/kg data (n=8) showed weak splicing correction and efficacy; August 2026 DSMB recommended advancing to a 12.5 mg/kg MAD cohort after reviewing 10 mg/kg data, and escalating the open-label extension from 5 to 10 mg/kg (n=16 in OLE). 10 mg/kg data (n=8) slipped to November 2026, 12.5 mg/kg data H1’27, then an end-of-Phase-2 meeting; OLE update early January 2027.
Deucrictibant — Acquired angioedema due to C1 inhibitor deficiency (AAE-C1INH) – (CREAATE) Phase 3
Primary completion expected mid-2027.
Jan’27PRAXPraxis Precision Medicines Ulixacaltamide (PRAX‑944) — Essential tremor (ESSENTIAL3) Registration
PDUFA decision expected Jan 29, 2027, positive phase 3 topline Oct 2025, Aug 2026 update: mid-cycle review completed; no AdCom contemplated; no Form 483 observations after inspection.
PM577 — Wilson’s disease (ATP7B p.H1069Q [anchor], R778L [follow-on]) Preclinical
NZ CTA clearance Jun 2026, US IND cleared Jul 2026, phase 1/2 first patient in 2026→ H2’26, initial data 2027.
PM647 — Alpha-1 antitrypsin deficiency (AATD) due to E342K (Pi*Z) or D341H (Little Rock) mutations Preclinical
IND &/or CTA mid-2026→ Q3’26, phase 1 first patient in 2026, initial data 2027.
mid’27RCUSArcus Biosciences Casdatifan mono & +zimberelimab, +zim+ipi, +tivozanib, +ivonescimab,+axitinib, +undiscl. — 1L & 2L+ ccRCC (ARC-20) Phase 1
ARC-20 multi-cohort program for the HIF-2α inhibitor casdatifan ± zimberelimab, ipilimumab, tivozanib, or ivonescimab in 1L and 2L+ clear-cell RCC. Monotherapy data at ASCO-GU February 2026 (100 mg QD): confirmed ORR 45%, median PFS 15.1…
ARC-20 multi-cohort program for the HIF-2α inhibitor casdatifan ± zimberelimab, ipilimumab, tivozanib, or ivonescimab in 1L and 2L+ clear-cell RCC. Monotherapy data at ASCO-GU February 2026 (100 mg QD): confirmed ORR 45%, median PFS 15.1 months; pooled n=121, cORR 35%, mPFS 12.2 months. Multiple line-of-therapy readouts H2’26 including first casdatifan + zimberelimab efficacy in 1L; undisclosed non-TKI combo revealed as ivonescimab (July 2026) with first patient in by year-end 2026 and 1L data by mid’27.
mid’27RCUSArcus Biosciences AB102 — Atopic Dermatitis, CSU Phase 1
FIH Aug 2026, CSU POCFPI mid-2027 with data 2027-28.
2027RCUSArcus Biosciences AB102 — Atopic Dermatitis, CSU Phase 1
FIH Aug 2026, CSU POCFPI mid-2027 with data 2027-28.
IMVT-1402 — Graves' Disease Phase 3
Phase 3 data readout expected 2027.
IMVT-1402 — Myasthenia Gravis Phase 3
Phase 3 data readout expected 2027.
2027RVMDRevolution Medicines Daraxonrasib, RMC-6236 (Rasonque) — Previously treated RAS-mutant NSCLC (RASolve 301) – 200mg mono vs docetaxel Phase 3
Initial readout 2027.
2027RVMDRevolution Medicines RMC-5127 — KRAS G12V advanced solid tumors Phase 1
Monotherapy RP2D H2’26 but data disclosure 2027.
mid’27SLDBSolid Biosciences SGT-501 — RYR2-mediated Catecholaminergic polymorphic ventricular tachycardia (CPVT) –(ARTEMIS) Phase 1
Primary completion expected mid-2027.
mid’27SMMTSummit Therapeutics Ivonescimab (SMT112) + casdatifan — 1L & 2L+ ccRCC (ARC-20) Phase 1
Multi cohorts, ivo combo Jul 2026 with data by mid-2027.
2027SNDXSyndax Pharmaceuticals Revumenib (Revuforj) — R/R acute leukemia with KMT2A translocation (AUGMENT-101-2A), R/R NPM1m AML (AUGMENT-101-2C) Marketed
New data in 1L; maintenance; RW; R-R setting 2H26 and 2027+, publication of R/R NUP98r data Q4’26.
Feb’27SRPTSarepta Therapeutics Eteplirsen (EXONDYS 51), Golodirsen (VYONDYS 53), Casimersen (AMONDYS 45) — DMD (exon 51/53/45 skipping) Marketed
PDUFA decision expected Feb 27, 2027 for the sNDAs of VYONDYS 53 and AMONDYS 45 aimed at conversion of AA to full approval.
Aug’27SUPNSupernus Pharmaceuticals SPN-817 — Focal epilepsy (RENAISSANCE 2) Phase 2
Primary completion expected Aug 2027.
2027SYRESpyre Therapeutics SPY003 — Ulcerative colitis (SKYLINE platform trial) Phase 2
Part A [open label] induction readout Q2’26→ Q3’26→ Sep 2026, Part B [RCT] induction 2027.
mid’27TNXPTonix Pharmaceuticals TNX-102 SL — Acute stress reaction (ASR), Acute stress disorder (ASD), prophylaxis against development of PTSD (OASIS) Phase 2
IIT, funded by US DOD, topline H2’26→ mid-2027.
Dabogratinib (TYRA-300) — Low Grade Upper-Tract UC (SURF303) Phase 2
Open-label; no control, 60-80-TBD mg, first patient in Dec 2025, initial data 2027, primary completion 2030.
Jun’27UTHRUnited Therapeutics Ralinepag — PAH (ADVANCE OUTCOMES ) Registration
PDUFA decision expected Jun 24, 2027, positive phase 3 topline March 2026.
2027UTHRUnited Therapeutics Treprostinil Soft Mist Inhaler — PAH Registration
US approval expected in 2027.
2027UTHRUnited Therapeutics Treprostinil Soft Mist Inhaler — PH-ILD Registration
US approval expected in 2027.
Avutometinib + defactinib — R/R LGSOC (RAMP 301) Phase 3
Phase 3 topline readout expected by mid-2027.
Apr’27VTVTvTv Therapeutics Cadisegliatin (TTP399) — T1D (adjunct to insulin) – (CATT1) Phase 3
Primary completion expected Apr 2027.
2027VYGRVoyager Therapeutics NBIB-223 — Friedreich's ataxia Phase 1
‘Initiating’ as of Aug 2026, 1st data 2027.
2027XENEXenon Pharmaceuticals NBI-921355, NBI-355 — Epilepsy Phase 1
Initial Phase 1 data expected 2026→ 2027.
ZB021 — I&I, Psoriasis Phase 1
China SAD/MAD phase 1 in healthy volunteers by InnoCare, first patient in during H1’26, data by year-end 2026, then proof-of-concept in psoriasis by Zenas in North America with results 2027 after China phase 1 completion.
ZB022 — I&I Preclinical
Phase 1 first patient in 2026→ 2027 with potential safety/PK data 2027.
ZB014 — Systemic lupus erythematosus (SLE) – (SunStone) Preclinical
Phase 1 first patient in 2027 with potential safety/PK data 2027.
Xanomeline-trospium (KarXT) + SOC — Manic episodes in Bipolar Disorder Type I (NCT07140913) Phase 3
Primary completion expected mid-2027.
Q3’27AAPGAscentage Pharma Lisaftoclax (APG-2575) + acalabrutinib — 1L CLL/SLL – (GLORA-2 – Multi) Phase 3
Primary completion expected Q3’27.
H2’27AAPGAscentage Pharma Arilzomadin (APG-115) + selumetinib — Neurofibromatosis Type 1 (NF1), R/R MPNST (MEKMDM2 - US) Phase 1/2
Primary completion expected H2’27.
Q3’27ACADACADIA Pharmaceuticals ACP‑211 — MDD with inadequate response to antidepressant [add. Indications: TRD & others] Phase 2
Primary completion expected Q3’27.
Q3’27ALNYAlnylam Pharmaceuticals ALN-ANG3 — Diabetic Kidney Disease Phase 2
Primary completion expected Q3’27.
Q3’27ALNYAlnylam Pharmaceuticals Mivelsiran — Cerebral amyloid angiopathy (cAPPricorn-1) Phase 2
Primary completion expected Q3’27.
Q3’27ALNYAlnylam Pharmaceuticals Rapirosiran — MASH (NASHGEN-2) Phase 2
Primary completion expected Q3’27.
Q3’27ANNXAnnexon Biosciences Vonaprument (ANX007) — Dry AMD with Geographic atrophy (GA) – (ARCHER II) Phase 3
M15 topline H2’26 → M15 primary endpoint readout Q4’26 and M24 primary endpoint Q3’27 with program expanded with M15; M24 dual primary endpoint strategy instead of M15 primary endpoint only [success can be claimed based on either M15 or…
M15 topline H2’26 → M15 primary endpoint readout Q4’26 and M24 primary endpoint Q3’27 with program expanded with M15; M24 dual primary endpoint strategy instead of M15 primary endpoint only [success can be claimed based on either M15 or M24], discontinuations <10% and compliance >95%, launch of OLE.
H2’27APVOAptevo Therapeutics Mipletamig (APVO436) + ven+aza — 1L AML (RAINIER) Phase 1/2
Primary completion expected H2’27.
Tenapanor (IBSRELA) — Chronic Idiopathic Constipation (CIC) – (ACCEL) Phase 3
First patient in Jan 2026, last patient in by year-end 2026, topline H2’27, primary endpoint=constipation PROs.
Efgartigimod — Sjogren’s disease (UNITY) Phase 3
Phase 3 data readout expected H2’27.
Empasiprubart — Chronic inflammatory demyelinating polyneuropathy (CIDP) – (EMVIGORATE & EMNERGIZE) Phase 3
Phase 3 data readout expected H2’27.
Luxdegalutamide (ARV-766, JSB462) + Pluvicto — mCRPC after ≥1 ARPI & 0-2 taxane regimens (CJSB462B12201) Phase 2
Primary completion expected Q3’27.
Q3’27ARWRArrowhead Pharmaceuticals Zodasiran (ARO-ANG3) — HoFH (Adults) - (YOSEMITE) Phase 3
Last patient in Jul 2026, topline Q3’27.
Q3’27ARWRArrowhead Pharmaceuticals Zodasiran (ARO-ANG3) — HoFH (Ados) - (SPRUCE) Phase 3
Primary completion expected Q3’27.
TransCon CNP — Hypochondroplasia (2-18y) – (teACH) Phase 3
Primary completion expected Q3’27.
TransCon IL-2 β/γ mono & combos (+pembro and/or TransCon TLR7/8 Agonist or others) — Solid Tumors (Systemic) - (IL-Believe) Phase 2
Primary completion expected Q3’27.
H2’27AURAAura Biosciences Belzupacap sarotalocan (bel-sar) — 1L choroidal melanoma with small or indeterminate lesions (CoMpass) Phase 3
Last patient in Jun 2026, topline H2’27.
H2’27AUTLAutolus Therapeutics Obe-cel (AUCATZYL) — Progressive MS (BOBCAT) Phase 1
First patient in Oct 2025, initial data by year-end 2026→ Q1’27 at ECTRIMS [data in n=12], then full phase 1 data H2’27 in n=18.
Q3’27BCRXBioCryst Pharmaceuticals Navenibart — Hereditary angioedema (HAE) attacks (ALPHA-ORBIT & LTE) Phase 3
Last patient in Jun 2026, topline Q3’27, BLA filing by year-end 2027.
Zorevunersen (STK-001) — Dravet syndrome Phase 3
Phase 1/2 results in NEJM March 2026, readout mid-2027→ Q3’27.
H2’27CBIOCrescent Biopharma CR-001 + CR-003 — Advanced solid tumors - CN Preclinical
Phase 1/2 first patient in H2’26→ H1’27 in China for CR-001+003, initial data H2’27.
H2’27CBIOCrescent Biopharma CR-002 — Advanced solid tumors - global Preclinical
Phase 1 first patient in H2’26, proof-of-concept data H2’27.
H2’27CBIOCrescent Biopharma CR-003 (SKB105) — Advanced solid tumors - global Phase 1
Phase 1 first patient in H2’26 with proof-of-concept data H2’27.
Cemsidomide + dex — 4L+ R/R MM (MOMEMTUM) Phase 2
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at…
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at RP2D of 100µg [also MTD] and ORR 40% at 75µg and ORR 36% across all dose levels; ORR 53% in prior CAR-T or TCE and 47% in Prior BCMA and 47% in 6L+ at RP2D. mDoR 7.9 months across all dose levels, phase 1 biomarker and translational data Sept 2026 at IMS.
H2’27CGTXCognition Therapeutics Zervimesine (CT1812) incl. background anti-Abeta mAbs — Alzheimer’s disease (early to mild) – (START) Phase 2
Phase 2 topline readout expected H2’27/data 2027, 15% of patients on background anti-Abeta mAbs.
Budoprutug - IV — Immune thrombocytopenia (ITP) Phase 1/2
Primary completion expected Q3’27.
Q3’27CRDFCardiff Oncology Onvansertib (NMS-1286937) — 2L SCLC Phase 2
Primary completion expected Q3’27.
Omecamtiv mecarbil — HFrEF (LVEF < 30% within 6mo, NT-proBNP ≥ 1000pg/mL) - (COMET-HF) Phase 3
Primary completion expected H2’27.
H2’27ETRAElectra Therapeutics Ipsoprubart (pan-SIRP mAb) — SURPASS — secondary hemophagocytic lymphohistiocytosis (sHLH) Phase 2/3
Registrational program (SURPASS) enrollment completion expected H2 2027 per 424B4 prospectus; FDA Breakthrough Therapy + EMA PRIME designations.
H2’27FDMT4D Molecular Therapeutics 4D-150 — Wet AMD (newly/recently diagnosed) - (4FRONT-1 & 2) Phase 3
Randomized against aflibercept 2 mg; enrollment completed February 2026 (4FRONT-1) and June 2026 (4FRONT-2), ~4 months ahead of plan. 52-week topline H1’27 for 4FRONT-1, H2’27 for 4FRONT-2. PRISM Phase 2b two-year data (July 2026, n=45):…
Randomized against aflibercept 2 mg; enrollment completed February 2026 (4FRONT-1) and June 2026 (4FRONT-2), ~4 months ahead of plan. 52-week topline H1’27 for 4FRONT-1, H2’27 for 4FRONT-2. PRISM Phase 2b two-year data (July 2026, n=45): 3e10 vg/eye selected as RP3D with maintained BCVA, CST control, and durable treatment-burden reduction; mild intraocular inflammation in 2.8% within 28 weeks, no new inflammation with 2–4+ years follow-up (n=71 safety population), and no 4D-150-related hypotony, endophthalmitis, vasculitis, or choroidal effusions.
H2’27GLUEMonte Rosa Therapeutics MRT-2359 + apalutamide — AR-mut mCRPC (MODeFIRe-1) Phase 2
Primary completion expected H2’27.
Talquetamab (TALVEY) + D or DP — 2L+ R/R MM (MonumenTAL-3) Phase 3
Primary completion expected H2’27.
Alpha‑1 Proteinase Inhibitor SC — Alpha‑1 antitrypsin deficiency (SWIFT-SC) Phase 3
Primary completion expected Q3’27.
H2’27IKTInhibikase Therapeutics IkT-001 — PAH (IMPROVE-PAH) Phase 3
First patient in Apr 2026, Part A interim safety H1’27, Part A last patient in and Part B FI H2’27, Part A topline mid-2028 [PVR], Part B last patient in year-end 2028, Part B topline mid-2029 [6-minute walk distance].
Brenetafusp (IMC-F106C) + nivolumab — 1L adv. cut. melanoma (PRISM-MEL-001) Phase 3
Primary completion expected H2’27.
Tafasitamab (Monjuvi / Minjuvi) — Primary Autoimmune Blood Cell Disorders, ITP, wAIHA Phase 2
Primary completion expected Q3’27.
Q3’27IONSIonis Pharmaceuticals ION775 — TG reduction in severe hypertriglyceridemia (sHTG) Phase 2
First patient in May 2026, primary completion Q3’27, phase 1 data at ESC’26: robust TG lowering with potential for extended dosing intervals.
Q3’27IONSIonis Pharmaceuticals ION440 — MECP2 duplication syndrome Phase 2
Primary completion expected Q3’27.
Q3’27IONSIonis Pharmaceuticals ION464 — Multiple system atrophy and Parkinson's disease Phase 2
Primary completion expected Q3’27.
Q3’27IONSIonis Pharmaceuticals Opemalirsen (AZD2373) — APOL1-mediated CKD (AMKD) Phase 2
Primary completion expected Q3’27.
H2’27IONSIonis Pharmaceuticals Obudanersen, ION582 — Angelman syndrome (REVEAL) Phase 3
Last patient in Jul 2026, topline H2’27.
H2’27JBIOJade Biosciences JADE-301 — Dermatomyositis Preclinical
FIH in healthy volunteers planned H1’27→ Q4’26, interim data H2’27.
Q3’27LQDALiquidia Corporation LIQ861 (YUTREPIA) — PH-ILD (ASCENT coh. B) Marketed
Primary completion expected Q3’27.
H2’27LXEOLexeo Therapeutics LX2006 — Friedreich ataxia cardiomyopathy (with abnormal LVMI ) - (SUNRISE-FA 2) Phase 3
Open-label pivotal trial to support accelerated approval in Friedreich ataxia cardiomyopathy; first patient in June 2026, topline H2’27. Primary endpoint is LVMI by MRI at month 6, powered to detect ≥15% effect size; FDA recommended…
Open-label pivotal trial to support accelerated approval in Friedreich ataxia cardiomyopathy; first patient in June 2026, topline H2’27. Primary endpoint is LVMI by MRI at month 6, powered to detect ≥15% effect size; FDA recommended dropping cardiac frataxin protein co-primary as proof of mechanism is established. Key secondaries include mFARS, KCCQ, high-sensitivity troponin-I, and lateral wall thickness; CLARITY-FA natural history study ongoing.
Q3’27MAZEMaze Therapeutics S-606001 (MZE001) + ERT — Pompe disease (Late-onset) Phase 2
Primary completion expected Q3’27.
Q3’27MDGLMadrigal Pharmaceuticals Resmetirom (Rezdiffra) — Post-Liver Transplant Patients With MASH (from recurrent MASH or de novo MASH) Phase 2
Primary completion expected Q3’27.
H2’27MIRMMirum Pharmaceuticals MRM‑3379 — Fragile X syndrome (BLOOM) Phase 2
Primary completion expected H2’27.
H2’27MPLTMapLight Therapeutics ML-007C-MA — Alzheimer’s disease psychosis Phase 2
Phase 2 topline readout expected H2’27 [vs primary completion Q1’27].
mRNA-1469 — Bundibugyo Ebolavirus vaccine Phase 1
Primary completion expected Q3’27.
H2’27NAMSNewAmsterdam Pharma Obicetrapib + ezetimibe FDC — LDL-C lowering in ASCVD / HeFH – Coronary Plaque Volume Study (LDL-C ≥ 70 mg/dL & non-calcified coronary plaque >75mm3) [REMBRANDT] Phase 3
Primary completion expected H2’27.
Q3’27NBIXNeurocrine Biosciences Osavampator (NBI-7845, TAK-653) — Major depressive disorder (inadequate response) Phase 3
Primary completion expected Q3’27.
Q3’27NBIXNeurocrine Biosciences NBI-570 — Schizophrenia Phase 2
Primary completion expected Q3’27.
H2’27NBIXNeurocrine Biosciences Direclidine (NBI-568) — Schizophrenia (Acute) Phase 3
Primary completion expected H2’27.
Givastomig + nivolumab + chemo — 1L CLDN18.2+ Gastric Cancer (GEA) Phase 2
RCT, first patient in Feb 2026, PFS topline H2’27.
H2’27NERVMinerva Neurosciences Roluperidone (MIN-101) — Negative symptoms of schizophrenia (C19 study) Phase 3
Confirmatory, phase A = 12-week RCT of rolu vs placebo; phase B = 40-week RCT of rolu vs 3 SOC antipsychotics [risperidone, aripiprazole, olanzapine], first patient in end-March 2026, 12-week Part A topline H2’27, 52-week Part B topline…
Confirmatory, phase A = 12-week RCT of rolu vs placebo; phase B = 40-week RCT of rolu vs 3 SOC antipsychotics [risperidone, aripiprazole, olanzapine], first patient in end-March 2026, 12-week Part A topline H2’27, 52-week Part B topline H2’28.
NGN-401 — Rett syndrome (3y+) - (Embolden) Phase 1/2/3
Converted from Phase 1/2 to Phase 3 (Embolden); topline H2’27 with PPQ manufacturing runs completing year-end 2026 ahead of BLA submission. June 2026 Phase 1/2 data (all n=10, 12–30 months follow-up): CGI-I improvement in 100%, ≥1…
Converted from Phase 1/2 to Phase 3 (Embolden); topline H2’27 with PPQ manufacturing runs completing year-end 2026 ahead of BLA submission. June 2026 Phase 1/2 data (all n=10, 12–30 months follow-up): CGI-I improvement in 100%, ≥1 developmental milestone gained in 100% (mean 4.7 per patient), milestone gains deepening over time with no plateau and none lost through 30 months; 70% gained ≥2 milestones with improvement across ≥2 core Rett domains, in both pediatric and adolescent/adult patients; clinically meaningful gains on the Rett gross motor and hand function scales.
Q3’27NTLAIntellia Therapeutics Nexiguran ziclumeran (nex-z, NTLA-2001) — ATTR-PN (MAGNITUDE-2) Phase 3
Primary completion expected Q3’27.
NXP900 + lorlatinib — ALK+ NSCLC (coh. 2) Phase 1
Primary completion expected Q3’27.
OP-3136 – mono & +palazestrant (mBC) or +fulvestrant (mBC) or +darolutamide (mCRPC) — ER+/HER2- metastatic breast cancer, mCRPC, lung cancer Phase 1
Primary completion expected H2’27, preliminary monotherapy data at ASCO’26 in n=19 across dose levels up 45mg OD: shrinkage in 68%; ORR 15.8% and cORR 10.5%; rapid sustained and sig reduction in lys23 of histone H3 *direct KAT6 target*;…
Primary completion expected H2’27, preliminary monotherapy data at ASCO’26 in n=19 across dose levels up 45mg OD: shrinkage in 68%; ORR 15.8% and cORR 10.5%; rapid sustained and sig reduction in lys23 of histone H3 *direct KAT6 target*; dysgeusia 81% [56% grade 1, 25% grade 2]. Anemia 38% including 6% grade 3 -neutropenia 34% including 28% grade 3, initial combo data mid-2027, mCRPC combo first patient in Q4’26.
MDX2301 — COVID-19 (Therapeutic & Prophylaxis) Phase 1
Primary completion expected Q3’27.
H2’27OVIDOvid Therapeutics OV329 — Drug-resistant focal onset seizures (adults) Phase 2
RCT, first patient in Q2’26 in AU, readout mid-2027/Q3’27→ H2’27, 3-5-7mg tested in phase 1 → 5 and 7mg in phase 2.
KL1333 — mtDNA-driven primary mitochondrial disease (e.g., m.3243A>G) Phase 2/3
Primary completion expected H2’27.
H2’27PVLAPalvella Therapeutics QTORIN rapamycin 3.9% topical — Clinically Significant Angiokeratomas Phase 2
First patient in May 2026, topline H2’27.
Q3’27RAPPRapport Therapeutics RAP-219 — Focal onset seizures (FOCUS-1 & 2 - Global) Phase 3
Primary completion expected Q3’27.
H2’27RXRXRecursion Pharmaceuticals REC-4539 — Solid tumors incl. SCLC (ENLYGHT) Phase 1
Strategic pause → phase 1 first patient in May 2026, safety/PK data H1’27→ H2’27.
H2’27SABSSAB Biotherapeutics SAB-142 — Delaying progression of type 1 diabetes in new-onset T1D pts (Stage 3, ≤100d from diagnosis, 5-40y) – (SAFEGUARD) Phase 2
Registrational, n=159 overall, Part A = dose-ranging study in n=12 adults fully enrolled Q1’26, Part B = dose-ranging RCT in n=147 pediatric+ados+adults with last patient in year-end 2026/Q4’26, topline H2’27.
H2’27SNDXSyndax Pharmaceuticals Revumenib (Revuforj) — Myelofibrosis Phase 1/2
Proof-of-concept in MF to inform SNDX-62122 dec in this indication, PFI Q4’26, proof-of-concept data H2’27.
Q3’27SRPTSarepta Therapeutics Delandistrogene moxeparvovec, SRP-9001 (ELEVIDYS) — DMD (4y+, ambulatory) Marketed
Primary completion expected Q3’27.
H2’27TLSATiziana Life Sciences Foralumab (TZLS-401) — ALS (BANYAN) Phase 2
Primary completion expected H2’27.
Q3’27TRAXFirst Tracks Bio. ANB033 — Eosinophilic Esophagitis Phase 1
First patient in Q1’26, topline mid-2027→ Q3’27.
Q3’27TRVITrevi Therapeutics Nalbuphine ER (HADUVIO) — Refractory chronic cough (RCC) – (LAKE) Phase 2
Primary completion expected Q3’27.
H2’27TRVITrevi Therapeutics Nalbuphine ER (HADUVIO) — Chronic cough in IPF (OCEAN-2) Phase 3
First patient in Q3’26, 2-week titration+12-week fixed dosing, topline H2’27– primary endpoint= relative change from bsln in 24hr Cough Freq at week 14.
H2’27TVTXTravere Therapeutics Pegtibatinase (TVT-058) — Classical homocystinuria (HCU) – (HARMONY) Phase 3
First patient in Dec 2023 → enrollment voluntary paused Sept 2024 due to CMC/scale-up issues and re-initiated early 2026 with first patient in Apr 2026, topline H2’27.
Verekitug — COPD (Venture) Phase 2
Last patient in Jun 2026, topline H2’27.
H2’27UTHRUnited Therapeutics Inhaled treprostinil (Tyvaso) — Progressive pulmonary fibrosis (TETON-PPF) Phase 3
Last patient in Aug 2026, week 52 topline H2’27.
H2’27VYGRVoyager Therapeutics VY7523 — Alzheimer's disease Phase 1/2
Initial tau PET imaging data from MAD in Alz patients H2’26→ Q4’26, safety potential early 2027 and biomarker data H2’27 dpding on enrollment.
H2’27XFORX4 Pharmaceuticals XOLREMDI (Mavorixafor) — Chronic neutropenic disorders (4WARD) Phase 3
Last patient in Q3’26, topline H2’27.
XmAb942 — Ulcerative colitis (XENITH-UC) Phase 2
12-Week induction topline H2’27, Q4W after initial week 0-week 2 loading like afimkibart and tulisokibart but aiming at Q12W/Q3M dosing in maintenance vs Q4W for afim/tuliso. Duvakitug being Q2W induction and Q4W maintenance.
Tumor Treating Fields (OPTUNE) — 1L Pancreatic cancer Registration
Potential China approval H2’27.
Efgartigimod (VYVGART) — Sjogren’s disease (UNITY) Phase 3
Argenx trial, readout 2027→ H2’27.
Pasritamig (JNJ-78278343) + docataxel — 2L+ mCRPC (After 1-2 ARPI) – (KLK2-PASenger) Phase 3
Primary completion expected H2’27, phase 1 data at ASCO-GU’26.
Q4’27ALNYAlnylam Pharmaceuticals Elebsiran + tobevibart — Hepatitis D virus infection (ECLIPSE-1/2/3) Phase 3
Phase 3 data readout expected Q4’27 and Q1’27.
Omaveloxolone (SKYCLARYS) — Friedreich ataxia (Pediatric) Phase 3
Primary completion expected Q4’27.
DURAVYU — COMO/CAPRI — diabetic macular edema Phase 3
COMO and CAPRI Phase 3 DME trials fully enrolled (480+ patients in 5 months); topline 56-week data anticipated Q4 2027.
IMG-007 — Atopic dermatitis (moderate-to-severe) Phase 2
Amended protocol [linked to Kaposi sarcoma?], topline Q4’27.
Q4’27VKTXViking Therapeutics VK2735 - SC — Obesity ± T2D (VANQUISH-1/-2) Phase 3
VQ1 78-week readout potential Q2’27, VQ2 78-week readout potential Q4’27, both fully enrolled as of Apr 2026, 42-week LTE.
YE’27AAPGAscentage Pharma Olverembatinib (HPQ1351) + chemo — 1L Ph+ ALL (POLARIS-1 - Global) Phase 3
Primary completion expected year-end 2027.
YE’27AAPGAscentage Pharma APG-5918 — Anemia (CN) Phase 1
Primary completion expected year-end 2027.
YE’27AAPGAscentage Pharma APG-2449 — Adv. solid tumors (CN) Phase 1
Primary completion expected year-end 2027.
YE’27ALNYAlnylam Pharmaceuticals Nucresiran — ATTR-PN (TRITON-PN) Phase 3
Primary completion expected year-end 2027.
YE’27ALNYAlnylam Pharmaceuticals Cemdisiran + pozelimab — Geographic atrophy (SIENNA) Phase 3
Primary completion expected year-end 2027.
Efgartigimod — Antibody mediated rejection (AMR) Phase 2
Primary completion expected year-end 2027.
YE’27ARWRArrowhead Pharmaceuticals ARO-RAGE — Inflammatory Pulmonary Diseases, Allergen induced mild asthma Phase 2
Primary completion expected year-end 2027.
TransCon CNP — Hypochondroplasia (0-2y) – (reACHin) Phase 2
Primary completion expected year-end 2027.
YE’27AUTLAutolus Therapeutics Obe-cel (AUCATZYL) — R/R ALL – pediatric (CATULUS) Phase 1/2
Pivotal, phase 1 data at ASH’25: ORR 95.5%; CRR 90.9%, phase 2 last patient in H1’27 and topline year-end 2027.
YE’27AXSMAxsome Therapeutics Auvelity (AXS‑05) — Smocking Cessation (SUMMIT) Phase 2/3
Primary completion expected year-end 2027.
YE’27BBIOBridgeBio Pharma Acoramidis (Attruby) — Transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) Phase 3
Primary completion expected year-end 2027.
BNT3212 ± pumitamig — Multiple solid tumors Phase 1/2
Primary completion expected year-end 2027.
Gedatolisib + fulvestrant + palbociclib/ribociclib — 1L ET-resistant (inoperable) HR+ HER2- adv./metastatic Breast Cancer after prog. from adj. & no prior systemic therapy - (VIKTORIA-2) Phase 3
Primary completion expected year-end 2027.
YE’27CORTCorcept Therapeutics Relacorilant + nab-paclitaxel — 2-3L Cervical Cancer (STELLA) Phase 2
First patient in Q2’26, IST by RCAGY-GINECO, data by year-end 2027.
YE’27CORTCorcept Therapeutics Relacorilant + GnP — 1L PDAC (TRIDENT) Phase 2
First patient in 2026, data by year-end 2027.
YE’27DNLIDenali Therapeutics Tividenofusp alfa, DNL310, ETV:IDS (AVLAYAH) — MPS II (Hunter syndrome, presymptomatic or symptomatic pediatric >=5kg prior to adv. neurologic impairment) Marketed
Primary completion expected year-end 2027.
Amivantamab (RYBREVANT) + FOLFIRI — 2L+ KRAS/NRAS/BRAF-wt mCRC after Chemo±anti-VEGF (OrigAMI-3) Phase 3
Primary completion expected year-end 2027.
Xembify — CIDP Phase 3
Primary completion expected year-end 2027.
Atebimetinib (IMM-1-104) + cemiplimab — 1L NSCLC Phase 2
First patient in H2’26, preliminary data year-end 2027.
YE’27IONSIonis Pharmaceuticals ION826 (AZD4063) — Myocardial disease Phase 1
Primary completion expected year-end 2027.
late’27JAZZJazz Pharmaceuticals Zanidatamab + chemo — Previously T-DXd treated mBC (EmpowHER) Phase 3
Phase 3 data readout expected late 2027; early 2028.
YE’27JAZZJazz Pharmaceuticals JZP898 ± pembrolizumab — Solid tumors Phase 1
Primary completion expected year-end 2027.
YE’27KNSAKiniksa Pharmaceuticals KPL-387 — Recurrent pericarditis (Well controlled on SOC therapy) – (KPL-387-C212) Phase 2
Primary completion expected year-end 2027.
late’27KYMRKymera Therapeutics KT-621 — Asthma (BREADTH) Phase 2
Phase 2 data readout expected late 2027.
NBTXR3 (+RT) — Treatment-Naïve Stage II-III Esophageal Cancer Phase 1
Primary completion expected year-end 2027.
YE’27NTLAIntellia Therapeutics Nexiguran ziclumeran (nex-z, NTLA-2001) — ATTR-CM (MAGNITUDE) Phase 3
Primary completion expected year-end 2027.
Palazestrant (OP-1250) + ribocliclib — 1L or 2L+ ER+/HER2- mBC Phase 1/2
Primary completion expected year-end 2027.
Palazestrant (OP-1250) + palbocliclib — 1L or 2L+ ER+/HER2- mBC Phase 1/2
Primary completion expected year-end 2027.
Palazestrant (OP-1250) + atirmocliclib — ER1L or 2L+ ER+/HER2- mBC Phase 1/2
Primary completion expected year-end 2027.
Palazestrant (OP-1250) + alpelisib — PI3Km ER+/HER2- metastatic breast cancer Phase 1/2
Primary completion expected year-end 2027.
Palazestrant (OP-1250) + everolimus — 1L or 2L+ ER+/HER2- mBC Phase 1/2
Primary completion expected year-end 2027.
OPK-88006 injectable — MASH [, Obesity] Phase 1/2
Primary completion expected year-end 2027.
YE’27PBYIPuma Biotechnology Alisertib + fulvestrant/ET — Chemo-naïve HR+ HER2- metastatic Breast Cancer (TBCRC-041 / ALISCA-Breast1) Phase 2
Primary completion expected year-end 2027.
YE’27RNACCartesian Therapeutics Descartes-08 — Childhood-onset SLE, ANCA-associated Vasculitis (AAV), Juvenile Myasthenia (jMG) Gravis, Juvenile Dermatomyositis (JDM) - (HELIOS) Phase 1/2
Primary completion expected year-end 2027.
YE’27RYTMRhythm Pharmaceuticals RM‑718 — Obesity, MC4R Pathway Impairment Phase 1/2
Primary completion expected year-end 2027.
YE’27SNDXSyndax Pharmaceuticals Revumenib (Revuforj) + ‘7+3’ IC — 1L fit NPM1m, KMT2A-r, NUP98r acute leukemias Phase 1
Primary completion expected year-end 2027.
YE’27SRPTSarepta Therapeutics SRP-1005 (ARO-HTT) — Huntington’s Disease (INSIGHTT) Phase 1
Primary completion expected year-end 2027.
late’27SYRESpyre Therapeutics SPY072 + bimekizumab — HS (SKYLIGHT platform trial) Phase 2
First patient in Aug 2026, RCT of 072+bimeki vs bimeki, week 16 HiSCR75 topline late 2027/early 2028, proof-of-concept for 772 combo of 072+007.
YE’27TECXTectonic Therapeutic TX45 — Group 3 PH-ILD Phase 2
Primary completion expected year-end 2027.
YE’27VERAVera Therapeutics Atacicept — Glomerular Diseases incl. PMN, FSGS, MCD (PIONEER) Phase 2
Primary completion expected year-end 2027.
YE’27VNDAVanda Pharmaceuticals Iloperidone (FANAPT) - LAI — Bipolar I Disorder, Schizophrenia Phase 3
Primary completion expected year-end 2027.
YE’27VNDAVanda Pharmaceuticals Tasilmelteon (HETLIOZ) — Insomnia – pediatric Phase 3
Primary completion expected year-end 2027.
YE’27VNDAVanda Pharmaceuticals VTR-297 (Tricostatin A) — Hematological Malignancies Phase 1
Primary completion expected year-end 2027.
XmAb541 — Ovarian cancer Phase 1
Primary completion expected year-end 2027.
Zanidatamab (ZIIHERA) + chemo — Previously T‑DXd treated HER2+ breast cancer (EmpowHER-303) Phase 3
Last patient in H1’27, topline late 2027; early 2028.
H1’28AAPGAscentage Pharma Lisaftoclax (APG-2575) + azacitidine — 1L Elderly Unfit AML – (GLORA-3 – Multi) Phase 3
Primary completion expected H1’28.
Q1’28ACADACADIA Pharmaceuticals remlifanserin, ACP-204 — Lewy body dementia with psychosis Phase 2
Primary completion expected Q1’28.
H1’28ALNYAlnylam Pharmaceuticals Zilebesiran + REVERSIR — Hypertension (Pretreated w/ Zilebesiran) Phase 2
Primary completion expected H1’28.
Q1’28ARWRArrowhead Pharmaceuticals Fazirsiran (ARO-AAT) — Alpha-1 Liver Disease (AATD-Liver) Phase 3
Primary completion expected Q1’28.
H1’28ARWRArrowhead Pharmaceuticals Olpasiran (AMG 890) — MACE Reduction in Pts w/ ASCVD+elevated Lp(a) - (OCEAN(a)) Phase 3
Primary completion expected H1’28.
Q1’28AVBPArriVent BioPharma ARR-217 (MRG007) – mono & + bevacizumab — GI tumors Phase 1
Primary completion expected Q1’28.
H1’28AXSMAxsome Therapeutics AXS‑14 (esreboxetine) — Fibromyalgia (FORWARD) Phase 3
Primary completion expected H1’28.
H1’28BCAXBicara Therapeutics Ficerafusp alfa (BCA101) + pembrolizumab — 1L R/M HPV- HNSCC (FORTIFI-HN01) Phase 2/3
Primary completion expected H1’28.
BHV-1300 — Graves’ Disease, RA Phase 3
Primary completion expected Q1’28.
Tinlarebant (LBS-008) — Stargardt disease (STGD1) – (DRAGON II) Phase 1/2/3
Primary completion expected Q1’28.
BNT116 + cemiplimab — 1L PD-L1 ≥50% mNSCLC Phase 2
Primary completion expected H1’28.
Trastuzumab pamirtecan (BNT323, DB-1303) — 2L+ HER2+ EC (coh1: IHC 1+/2+ & coh2: 3+) – (Fern-EC-01) Phase 3
Primary completion expected H1’28.
Teclistamab (TECVAYLI) + R — 1L ND TE MM maintenance after ASCT (MajesTEC-4) Phase 3
Primary completion expected H1’28.
Savolitinib + osimertinib — NSCLC (1L; EGFRm MET-oe) – CN (SANOVO) Phase 3
Primary completion expected H1’28.
Surufatinib (Sulanda) + camrelizumab+GnP — PDAC (1L) – CN Phase 2/3
Primary completion expected H1’28.
HMPL-760 + R-GemOx — DLBCL (2L; R/R) – CN Phase 3
Primary completion expected H1’28.
Tebentafusp (KIMMTRAK) — 2L+ adv. cutaneous melanoma (TEBE-AM) Phase 3
Primary completion expected Q1’28.
Q1’28IOVAIovance Biotherapeutics Lifileucel + pembrolizumab — 1L melanoma (TILVANCE-301) Phase 3
Primary completion expected Q1’28.
H1’28JANXJanux Therapeutics JANX014 — mCRPC Phase 1
Primary completion expected H1’28.
H1’28JAZZJazz Pharmaceuticals JZP815 — Solid tumors and hematologic malignancies Phase 1
Primary completion expected H1’28.
Q1’28KLRAKailera Therapeutics Ribupatide (KAI-9531, HRS9531) — Obesity w/o T2D (BMI ≥30, BMI ≥27 with a comorbidity) – (KaiNETIC-1 - Global) Phase 3
Primary completion expected Q1’28.
Q1’28KLRAKailera Therapeutics Ribupatide (KAI-9531, HRS9531) — Obesity w/ T2D (BMI ≥27) – (KaiNETIC-2 - Global) Phase 3
Primary completion expected Q1’28.
Q1’28KLRAKailera Therapeutics Ribupatide (KAI-9531, HRS9531) — Obesity w/o T2D (BMI ≥35) – (KaiNETIC-3 -Global) Phase 3
Primary completion expected Q1’28.
H1’28KROSKeros Therapeutics Elritercept (TAK-226, KER-050) — 2L TD anemia with very low/low/intermediate-risk MDS (RENEW) Phase 3
Primary completion expected H1’28.
Q1’28LBRXLB Pharmaceuticals LB-102 – Oral — Bipolar I depression (ILLUMINATE-1) Phase 2
Phase 2 topline readout expected Q1’28.
Canvuparatide (MBX 2109) — Chronic hypoparathyroidism (oPTimize) Phase 3
Primary completion expected Q1’28.
Remestemcel-L (RYONCIL) + ruxolitinib — 2L Steroid-refractory acute Gr III-IV GvHD - adults Phase 3
Primary completion expected Q1’28.
MNKD-201 (nintedanib DPI) — IPF (INFLO-2) Phase 2
Primary completion expected Q1’28.
Q1’28NBIXNeurocrine Biosciences Direclidine (NBI-568) — Bipolar mania Phase 2
Primary completion expected Q1’28.
H1’28NBIXNeurocrine Biosciences Crinecerfont (CRENESSITY) — CAH (pediatric, <4y for classic CAH) Phase 2
Primary completion expected H1’28.
NBTXR3 (+Radscopal RT or anti-PD-1) — Solid Tumors Phase 1/2
Primary completion expected Q1’28.
H1’28NKTRNektar Therapeutics Rezpegaldesleukin — Atopic dermatitis (ZENITH AD-1/-2) Phase 3
Primary completion expected H1’28.
H1’28NKTRNektar Therapeutics Rezpegaldesleukin — New onset stage 3 T1D (TrialNet) Phase 2
Primary completion expected H1’28.
Q1’28NRIXNurix Therapeutics Bexobrutideg (NX-5948) — 3L+ Triple-XP R/R CLL/SLL after prog. to BTKi & BCL-2i (DAYBreak CLL-201) Phase 2
Primary completion expected Q1’28.
MDX2003 — Solid tumors Phase 1
Primary completion expected Q1’28.
Q1’28ORICORIC Pharmaceuticals Rinzimetostat (ORIC-944) + darolutamide — mCRPC after abiraterone (Himalayas-1)) Phase 3
Primary completion expected Q1’28.
H1’28OVIDOvid Therapeutics OV4071 — Acute schizophrenia Preclinical
Phase 1 first patient in Q2’26, Phase 1b proof-of-concept study first patient in H1’27, topline H1’28.
Q1’28PBYIPuma Biotechnology Alisertib + paclitaxel — 2L+ SCLC (ALISCA-Lung2) Phase 1/2
Primary completion expected Q1’28.
PRGN-2009 + chemo — Neoadjuvant HPV-associated oropharyngeal cancer / HNSCC Phase 2
Primary completion expected Q1’28.
Q1’28QNCXQuince Therapeutics AM-001 (add-on to SOC) — PH-ILD & PA still symptomatic despite background therapy Phase 2
AM-001 (from the Orphai acquisition, May 2026) as add-on to standard of care in PH-ILD and PAH symptomatic on background therapy. Positive Phase 2a topline May 2026 (n=10, 6 evaluable, all on background therapy): primary endpoint change in…
AM-001 (from the Orphai acquisition, May 2026) as add-on to standard of care in PH-ILD and PAH symptomatic on background therapy. Positive Phase 2a topline May 2026 (n=10, 6 evaluable, all on background therapy): primary endpoint change in predicted VO2max at week 24 +5.4%, FVC +4.8%, 6-minute walk +81.3 m, NT-proBNP −29% (no statistics provided); PH-ILD subgroup (n=4) similar. Phase 2b first patient in mid’26, readout Q1’28.
Q1’28QNCXQuince Therapeutics AM-001 — PH-ILD Phase 1
From Orphai acquisition May 2026, planned phase 2b topline Q1’28.
H1’28RLAYRelay Therapeutics Zovegalisib (RLY-2608) + ET (fulvestrant) — 2L+ PIK3CA-mutated HR+/HER2- adv. breast cancer (ReDiscover-2) Phase 3
Primary completion expected H1’28.
Q1’28SLDBSolid Biosciences SGT-212 — Friedreich's ataxia (FALCON) Phase 1
Primary completion expected Q1’28.
SL-325 — CD (RECEPTIVE-CD1) Phase 2
CD phase 2b first patient in Q3’26. RCT of 2 dose levels IV vs placebo. 12-week induction and 40-week maintenance. Induction primary endpoint = ER at week 12 and clinically rem. At week 12 as key secondary endpt. Induction week 12 topline…
CD phase 2b first patient in Q3’26. RCT of 2 dose levels IV vs placebo. 12-week induction and 40-week maintenance. Induction primary endpoint = ER at week 12 and clinically rem. At week 12 as key secondary endpt. Induction week 12 topline H1’28.
Q1’28TYRATyra Biosciences Dabogratinib (TYRA-300) — FGFR3-altered Low Grade Intermediate-Risk NMIBC (SURF302) Phase 2
Primary completion expected Q1’28.
Q1’28TYRATyra Biosciences TYRA-430 — FGF19+/FGFR4-driven adv solid tumors incl. HCC -FGF/FGFR+ all comers in Part A & FGF19+/- HCC in Part B coh. 1 (SURF431) Phase 1
Primary completion expected Q1’28.
early’28ASNDAscendis Pharma TransCon hGH — Turner syndrome, Idiopathic Short Stature (ISS), SHOX Deficiency, Small for Gestational Age (SGA) (children2-17y) – (HighLiGHts) Phase 3
Primary completion expected early 2028.
early’28CCCCC4 Therapeutics Cemsidomide + elranatamab + dex — 2L+ R/R MM Phase 1
First patient in Apt’26→ Q2’26, ‘incremental’ dose-escalation update H2’26, full dose-escalation data mid-2027, additional combo plans mid-2026, potential pivotal phase 3 by early 2028 in combo with a BCMA TCE.
Axatilimab + steroids — 1L chronic GVHD Phase 3
Phase 3 topline readout expected early 2028.
INS1201 — Duchenne muscular dystrophy (DMD) – (ASCEND) Phase 1
Primary completion expected early 2028.
early’28JAZZJazz Pharmaceuticals Zanidatamab + chemo — Previously T-DXd treated mBC (EmpowHER) Phase 3
Phase 3 data readout expected late 2027; early 2028.
early’28JAZZJazz Pharmaceuticals Zanidatamab + chemo — Neoadj. HER2+ breast cancer (EmpowHER 208) Phase 2
Primary completion expected early 2028.
early’28PCRXPacira BioSciences PCRX-2002 (AMT-143) — Post-surgical pain Phase 1
Phase 2 first patient in 2026→ year-end 2026, topline year-end 2027/early 2028.
early’28SNDXSyndax Pharmaceuticals Axatilimab (Niktimvo) + CS — 1L cGVHD (AXemplify-357) Phase 3
Incyte RCT, topline early 2028, primary completion Q3’27.
early’28SYRESpyre Therapeutics SPY072 + bimekizumab — HS (SKYLIGHT platform trial) Phase 2
First patient in Aug 2026, RCT of 072+bimeki vs bimeki, week 16 HiSCR75 topline late 2027/early 2028, proof-of-concept for 772 combo of 072+007.
Zanidatamab (ZIIHERA) + chemo — Previously T‑DXd treated HER2+ breast cancer (EmpowHER-303) Phase 3
Last patient in H1’27, topline late 2027; early 2028.
Q2’28ARQTArcutis Biotherapeutics ARQ-234 — Atopic dermatitis Phase 1
Primary completion expected Q2’28.
Tacabrutideg, BGB‑16673 — R/R CLL; R/R CLL/SLL (incl vs pirto) – (CaDAnCe-302/-303-/-304) Phase 3
Primary completion expected Q2’28.
Q2’28RYTMRhythm Pharmaceuticals Bivamelagon (LB54640) — Acquired hypothalamic obesity Phase 2
Primary completion expected Q2’28.
Zocilurtatug pelitecan (ZL-1310) tarlatamab ± durvalumab — 1L & 2L+ ES-SCLC (doublet in 2L+, triplet in 1L+) - (DeLLphi-313) Phase 1
Primary completion expected Q2’28.
mid’28ALLOAllogene Therapeutics Cema-cel (ALLO-501A) — Large B-cell lymphoma (1L consolidation in MRD+ pts) – (ALPHA3) Phase 2
Potential pivotal, positive non-futility IA on MRD clearance rate Apr 2026 [58% vs 16%. 25-30% delta sought → ~51% actual], EFS IA mid-2027, last patient in by year-end 2027, primary EFS readout mid-2028, 90% power to detect HR 0.5.
Dostarlimab (JEMPERLI) — Multiple Cancers incl. EC, dMMR/MSI-H Pan Tumors Marketed
Positive AZUR-1 pivotal Phase 2 topline July 2026 in untreated stage II–III dMMR/MSI-H locally advanced rectal cancer: meaningful sustained 12-month clinical CR rate, accepted under Project Orbis with Priority Review and PDUFA February…
Positive AZUR-1 pivotal Phase 2 topline July 2026 in untreated stage II–III dMMR/MSI-H locally advanced rectal cancer: meaningful sustained 12-month clinical CR rate, accepted under Project Orbis with Priority Review and PDUFA February 2027. AZUR-2 Phase 3 (dostarlimab in resectable dMMR/MSI-H colon cancer) topline 2028; AZUR-4 Phase 2 (dostarlimab + chemo in MMRp/MSS colon cancer) topline Q4’26; JADE pivotal Phase 3 (PD-L1-high HNSCC post-chemoradiation) topline 2028.
mid’28ARWRArrowhead Pharmaceuticals Olpasiran (AMG 890) — Plaque reduction in Pts w/ ASCVD+elevated Lp(a) - (OCEAN(a)-CCTA) Phase 3
Primary completion expected mid-2028.
2028AUTLAutolus Therapeutics Obe-cel (AUCATZYL) — Refractory LN (LUMINA) Phase 2
Pivotal, first patient in Q1’26, data 2028 [primary completion Q3’28], RCT phase 3 after potential AA with LUMINA.
mid’28AXSMAxsome Therapeutics SUNOSI (solriamfetol) — Attention Deficit Hyperactivity Disorder (children) – (FOCUS-2 in 6-12y / FOCUS-3 in 12-18y) Phase 3
Primary completion expected mid-2028.
Dapirolizumab pegol — SLE (PHOENYCS FLY) Phase 3
Primary completion expected mid-2028.
Pumitamig (BNT327, BMS986545) + chemo — 1L ES-SCLC (ROSETTA Lung-01) Phase 3
Phase 3 data readout expected 2028, head-to-head atezo+chemo.
Pumitamig (BNT327, BMS986545) + TMZ — 2L GBM (BNT327-10) Phase 2
Primary completion expected 2028.
Pumitamig (BNT327, BMS986545) + chemos — 1-2L Adv. TNBC Phase 2
Primary completion expected 2028.
2028CBIOCrescent Biopharma CR-001 + CR-002 — Advanced solid tumors - global Preclinical
Combo phase 1/2 first patient in H1’27→ 2028 for CR001+002, initial data year-end 2027→ delayed to 2028+.
mid’28CCCCC4 Therapeutics Cemsidomide + dex — 4L+ R/R MM (MOMEMTUM) Phase 2
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at…
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at RP2D of 100µg [also MTD] and ORR 40% at 75µg and ORR 36% across all dose levels; ORR 53% in prior CAR-T or TCE and 47% in Prior BCMA and 47% in 6L+ at RP2D. mDoR 7.9 months across all dose levels, phase 1 biomarker and translational data Sept 2026 at IMS.
2028CTNMContineum Therapeutics PIPE-791 — Idiopathic pulmonary fibrosis (IPF) Phase 2
26-Week RCT, first patient in Q1’26, potential readthrough from BMS LPA1 phase 3 in IPF, readout 2028.
2028ETONEton Pharmaceuticals ASN-001 (timolol topical gel) — Proliferating superficial infantile hemangiomas Phase 2/3
US rights acquisition Aug 2026, positive phase 2/3 trial results showing week 24 elimination or near-elimination rates of 56% *BID* and 42% *TID* vs 15% placebo, bioav. Bridging study to be run by Eton for filing. NDA filing H2’27 for…
US rights acquisition Aug 2026, positive phase 2/3 trial results showing week 24 elimination or near-elimination rates of 56% *BID* and 42% *TID* vs 15% placebo, bioav. Bridging study to be run by Eton for filing. NDA filing H2’27 for potential approval and launch 2028.
IMG-007 — Alopecia areata Phase 2
First patient in 2026, initial data 2028.
mid’28IKTInhibikase Therapeutics IkT-001 — PAH (IMPROVE-PAH) Phase 3
First patient in Apr 2026, Part A interim safety H1’27, Part A last patient in and Part B FI H2’27, Part A topline mid-2028 [PVR], Part B last patient in year-end 2028, Part B topline mid-2029 [6-minute walk distance].
Atebimetinib (IMM-1-104) + mGnP — 1L mPDAC (MAPKeeper 301) Phase 3
RCT, atebi+mGNP vs GnP, first patient in mid-2026, topline [OS] mid-2028.
IMVT-1402 — SjD Phase 2
Primary completion expected 2028.
IMVT-1402 — CIDP Phase 2
Primary completion expected 2028.
mid’28IONSIonis Pharmaceuticals ION356 — Pelizaeus-Merzbacher disease Phase 2
Primary completion expected mid-2028.
IPH4502 — Adv solid tumors incl. mUC, NSCLC, mBC, OC, GC/GEJ/esophageal, CRC Phase 1
Primary completion expected 2028.
2028MPLTMapLight Therapeutics ML-007C-MA — Schizophrenia - Acute Exacerbation of psychosis (ZEPHYR-2) Phase 3
Pending FDA alignment, topline 2028.
mid’28NKTRNektar Therapeutics Dapirolizumab pegol — SLE (PHOENYCS FLY) Phase 3
Primary completion expected mid-2028.
mid’28OVIDOvid Therapeutics OV4071 — Psychosis associated with Parkinson’s disease (PDP) & Lewy body dementia (LBDP) Phase 1
First patient in May 2026, phase 1 in healthy volunteers, Phase 2 proof-of-concept study first patient in H1’27, topline ~mid-2028.
2028PCRXPacira BioSciences Enekinragene inzadenovec (PCRX-201) — Knee osteoarthritis pain (ASCEND) Phase 2
Part A in n~45; 3 dose grps stratified by K-L Grade, Part B in n~90; 3 grps stratified by K-L Grade with enrollment in Part B dose-dependent upon availability of study drug with manuf. process 2→ confirmed end-Jul 2026, Part A last patient…
Part A in n~45; 3 dose grps stratified by K-L Grade, Part B in n~90; 3 grps stratified by K-L Grade with enrollment in Part B dose-dependent upon availability of study drug with manuf. process 2→ confirmed end-Jul 2026, Part A last patient in Jun 2026→ Part A topline year-end 2026, Part B topline 2028+.
mid’28PRQRProQR Therapeutics AX-0810 — Cholestatic diseases, biliary atresia (BA) Phase 1
Phase 1 in healthy volunteers (3, 6, 9 mg/kg): June 2026 target-engagement data (n=22, cohorts 1–2) showed clear NTCP engagement with dose-dependent serum total bile acid increases up to 8x, above the company’s predefined 2x threshold,…
Phase 1 in healthy volunteers (3, 6, 9 mg/kg): June 2026 target-engagement data (n=22, cohorts 1–2) showed clear NTCP engagement with dose-dependent serum total bile acid increases up to 8x, above the company’s predefined 2x threshold, plus statistically significant increases in urinary conjugated bile acid excretion and post-challenge TUDCA; no hormone changes. Biliary atresia selected as lead indication, with a China investigator-initiated trial in pediatric patients to inform a potential pivotal Phase 2 (first patient in mid’27, interim mid’28). 9 mg/kg data by year-end 2026; initial IIT data H1’27.
2028RNAAvidity Biosciences Del-brax — Facioscapulohumeral muscular dystrophy (FSHD) – (FORTITUDE-3) Phase 3
Phase 3 data readout expected 2028.
IMVT-1402 — Chronic Inflammatory Demyelinating Polyneuropathy Phase 2
Assumedly registrational, readout 2028.
IMVT-1402 — Sjögren’s Disease Phase 2
Phase 2 data readout expected 2028.
mid’28RVMDRevolution Medicines Daraxonrasib, RMC-6236 (Rasonque) — 1L mPDAC (RASolute 303) - 300mg mono, 200mg+GnP then 300mg mono vs GnP Phase 3
Primary completion expected mid-2028.
2028TARSTarsus Pharmaceuticals IRX-101 — Post-procedural pain & corneal tox in pts receiving IVT therapy (COMFORT) Phase 3
From iRenix Medical acquisition Jul 2026, potential new phase 3 first patient in H1’27 and topline 2028, 1st phase 2/3 RELIEF→ statistically significant reductions in post-procedural pain and corneal fluorescein staining [measure of…
From iRenix Medical acquisition Jul 2026, potential new phase 3 first patient in H1’27 and topline 2028, 1st phase 2/3 RELIEF→ statistically significant reductions in post-procedural pain and corneal fluorescein staining [measure of corneal surface damage] vs povidone-iodine aka Betadine.
2028UTHRUnited Therapeutics UKidney — End-stage renal disease (EXPAND) Phase 1/2
Primary completion expected 2028.
mid’28VERAVera Therapeutics Atacicept (TRUTAKNA) — IgA nephropathy (ORIGIN EXTEND) Phase 3
Primary completion expected mid-2028.
mid’28XENEXenon Pharmaceuticals Azetukalner (XEN1101) — Bipolar depression (X-CEED & X-CEED OLE) Phase 3
Primary completion expected mid-2028.
Plamotamab — Rheumatoid arthritis Phase 1
Primary completion expected mid-2028.
Zocilurtatug pelitecan (ZL-1310) — Relapsed ES-SCLC (DLLEVATE) Phase 3
Primary completion expected mid-2028.
Pasritamig (JNJ-78278343) ± JNJ-9401 — Later-lines mCRPC (After 1+ ARPI, 2+ taxanes, 1+ PSMA RLT, PARPi if eligible) – (KLK2-comPAS) Phase 3
Primary completion expected ~mid-2028, phase 1 combo data with JNJ-9401 H2’26.
Q3’28AAPGAscentage Pharma Lisaftoclax (APG-2575) – mono / combos — AML/MDS incl. venetoclax-refractory pts (CN) Phase 1/2
Primary completion expected Q3’28.
Efgartigimod alfa IV + empasiprubart IV — AChR+ gMG w/ partial resp. on VYVGART (ADAPT-FORWARD 1) Phase 2
Primary completion expected Q3’28.
BHV-8000 — Parkinson’s Disease Phase 2/3
Primary completion expected H2’28.
Pumitamig (BNT327, BMS986545) + docetaxel — 2L Adv./metastatic NSCLC Phase 2
Primary completion expected H2’28.
Q3’28CGEMCullinan Therapeutics Zipalertinib — NSCLC (1L/2L+ brain mets, 1L EGFR ex20ins, 2L uncommon EGFR muts incl. PACC) - (REZILIENT2) Phase 2
Primary completion expected Q3’28.
Q3’28DYNDyne Therapeutics Zeleciment basivarsen (DYNE-101) — Myotonic dystrophy type 1 (DM1) – (HARMONIA) Phase 3
Primary completion expected Q3’28.
Trimodulin — Severe community‑acquired pneumonia (sCAP) Phase 3
Primary completion expected Q3’28.
INCA036873 — B-cell and T-cell lymphomas Phase 1
Primary completion expected Q3’28.
INCB161734 — KRAS G12D-mutated mPDAC (DAWN-303) Phase 3
Primary completion expected Q3’28.
Tabirafusp tedromer (KSI-501) - 50mg/mL - Q8W & personalized Q4W-Q24W — DME (ALTO) Phase 3
Primary completion expected Q3’28.
Q3’28MAZEMaze Therapeutics S-606001 (MZE001) — Pompe disease (Late-onset) Phase 1
Primary completion expected Q3’28.
VIS-101 — Wet AMD Phase 2
Primary completion expected H2’28.
H2’28NERVMinerva Neurosciences Roluperidone (MIN-101) — Negative symptoms of schizophrenia (C19 study) Phase 3
Confirmatory, phase A = 12-week RCT of rolu vs placebo; phase B = 40-week RCT of rolu vs 3 SOC antipsychotics [risperidone, aripiprazole, olanzapine], first patient in end-March 2026, 12-week Part A topline H2’27, 52-week Part B topline…
Confirmatory, phase A = 12-week RCT of rolu vs placebo; phase B = 40-week RCT of rolu vs 3 SOC antipsychotics [risperidone, aripiprazole, olanzapine], first patient in end-March 2026, 12-week Part A topline H2’27, 52-week Part B topline H2’28.
OCU410 — Geographic Atrophy from Dry AMD (ArMaDa3) Phase 3
Primary completion expected Q3’28.
Sonrotoclax (BGB‑11417) + zanubrutinib — R/R MCL (CELESTIAL-RRMCL) Phase 3
Primary completion expected Q3’28.
Sonrotoclax (BGB‑11417) + zanubrutinib — 1L CLL/SLL (Fixed Duration) Phase 3
Primary completion expected H2’28.
V350A / V350B (MDX2201) — Infectious Mono & EBV-related cancers Phase 1
Primary completion expected Q3’28.
MDX2001 — Solid tumors Phase 1/2
Primary completion expected Q3’28.
Leniolisib (JOENJA) — APDS (ages 1-6y) Phase 3
Primary completion expected H2’28.
Q3’28RCKTRocket Pharmaceuticals RP-A601 — PKP2-associated Arrhythmogenic CardioMyopathy Phase 1
Primary completion expected Q3’28.
H2’28SNDXSyndax Pharmaceuticals Revumenib (Revuforj) + ven+aza — 1L fit KMT2A-r acute leukemia (RAVEN) Phase 2
Primary completion expected H2’28.
Apitegromab - IV — SMA in toddlers who have received an approved SMN1-tgted GT or who are receiving ongoing treatment with an approved SMN2-tgted therapy – ages <2y (OPAL) Phase 2
Primary completion expected H2’28.
H2’28VNDAVanda Pharmaceuticals Ponesimod (PONVORY) — Psoriasis (Moderate-to-Severe) Phase 3
Primary completion expected H2’28.
XmAb657 — Idiopathic inflammatory myopathy (IIM), Sjögren’s disease, SSc Phase 1
Primary completion expected H2’28.
Xanomeline-trospium (KarXT) — Alzheimer’s disease (MINDSET-1 & 2) Phase 3
Primary completion expected Q3’28.
ADI‑212 — Prostate cancer (mCRPC) Preclinical
Primary completion expected Q4’28.
Lecanemab (LEQEMBI) — Preclinical AD Phase 3
Primary completion expected Q4’28.
Q4’28IRWDIronwood Pharmaceuticals Apraglutide — Short bowel syndrome with intestinal failure (SBS-IF) – (STARS-2) Phase 3
Primary completion expected Q4’28.
Q4’28ORKAOruka Therapeutics ORKA-002 — HS (ORCA-SPLASH) Phase 2
Primary completion expected Q4’28.
Q4’28QNCXQuince Therapeutics AM-001 — Sarcoidosis Associated PH (SAPH) Phase 1
From Orphai acquisition May 2026, planned phase 2 topline Q4’28.
YE’28AVBPArriVent BioPharma Firmonertinib — Adjuvant Stage I-III EGFR uncommon muts incl. Ex20ins/PACC/classical-like (FIRMOST - CN) Phase 3
Primary completion expected year-end 2028.
YE’28BBIOBridgeBio Pharma Encaleret — Autosomal dominant hypocalcemia type 1 – pediatric (CALIBRATE-PEDS) Phase 2/3
Primary completion expected year-end 2028.
Felzartamab — Primary membranous nephropathy Phase 3
Primary completion expected year-end 2028.
Cemsidomide + dex — 4L+ R/R MM (MOMEMTUM) Phase 2
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at…
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at RP2D of 100µg [also MTD] and ORR 40% at 75µg and ORR 36% across all dose levels; ORR 53% in prior CAR-T or TCE and 47% in Prior BCMA and 47% in 6L+ at RP2D. mDoR 7.9 months across all dose levels, phase 1 biomarker and translational data Sept 2026 at IMS.
Teclistamab (TECVAYLI) — 4L+ 3x-Class-Refractory R/R MM (Trilogy-4) Phase 3
Primary completion expected year-end 2028.
Rinatabart sesutecan (Rina-S) — 2-4L Endometrial Cancer (RAINFOL-03) Phase 3
Primary completion expected year-end 2028.
Rinatabart sesutecan (Rina-S) + maintenance SOC — Maintenance in Recurrent Pt-sensitive Ovarian Cancer After 2L Pt-based Chemo Doublet (RAINFOL-04) Phase 3
Primary completion expected year-end 2028.
Rinatabart sesutecan (Rina-S) — Adv. NSCLC (RAINFOL-05) Phase 2
Primary completion expected year-end 2028.
Amivantamab (RYBREVANT) + mFOLFOX6/FOLFIRI — 1L KRAS/NRAS/BRAF-wt left-sided mCRC (OrigAMI-2) Phase 3
Primary completion expected year-end 2028.
Seralutinib — Pulmonary hypertension associated with interstitial lung disease (PH-ILD) – (SERANATA) Phase 3
Primary completion expected year-end 2028.
GIGA-2339 — Hepatitis B virus Phase 1
Primary completion expected year-end 2028.
Ranosidenib (HMPL-306) — AML (2L; R/R) – CN (RAPHAEL) Phase 3
Primary completion expected year-end 2028.
HMPL-A251 — Adv. HER2+ solid tumors – Global Phase 1/2
Primary completion expected year-end 2028.
HMPL-A580 — Adv. solid tumors incl. NSCLC, CRC, HNSCC, ESCC - Global Phase 1/2
Primary completion expected year-end 2028.
HMPL-A830 — Adv. solid tumors - CN Phase 1/2
Primary completion expected year-end 2028.
Ozekibart (INBRX-109) + irinotecan + TMZ (for EwS) — Solid tumors incl. CRC & 2-3L Adv. Ewing sarcoma Phase 1
Primary completion expected year-end 2028.
INCB123667 — PROC with cyclin E1 overexpression (MAESTRA 2) Phase 3
Primary completion expected year-end 2028.
INCB123667 — PROC with cyclin E1 overexpression (MAESTRA 1) Phase 2
Primary completion expected year-end 2028.
INCA33890 + chemo+beva — 1L MSS mCRC (NEXERA-303) Phase 3
Primary completion expected year-end 2028.
Treprostinil palmitil inhalation powder (TPIP) — Pulmonary hypertension associated with interstitial lung diseases (PH-ILD) Phase 3
Primary completion expected year-end 2028.
Treprostinil palmitil inhalation powder (TPIP) — Pulmonary arterial hypertension (PAH) Phase 3
Primary completion expected year-end 2028.
YE’28JAZZJazz Pharmaceuticals Zanidatamab + SOC chemo ± PD-1/L-1 — 1L BTC (HERIZON-BTC-302) Phase 3
Primary completion expected year-end 2028.
YE’28JAZZJazz Pharmaceuticals Zanidatamab + tucatinib — Breast cancer (PRE-I-SPY/I-SPY-P1) Phase 1
Primary completion expected year-end 2028.
YE’28JAZZJazz Pharmaceuticals JZP3507 (ONC206) — Pheochromocytoma, Paraganglioma Phase 2
Primary completion expected year-end 2028.
YE’28MOLNMolecular Partners MP0317 + Gem-cis-durvalumab — Adv. 1L Cholangiocarcinoma / BTC (TACTIC) Phase 2
Primary completion expected year-end 2028.
Palazestrant (OP-1250) + ribocliclib — 1L ER+/HER2- metastatic breast cancer (OPERA-02) Phase 3
Primary completion expected year-end 2028.
Apitegromab - IV — FSHD (FORGE) Phase 2
Primary completion expected year-end 2028.
Telitacicept — Primary Sjögren’s disease (Global) – (UPSTREAM SjD) Phase 3
Primary completion expected year-end 2028.
XmAb541 + XmAb808 — Adv. solid tumors incl. HG Serous Ovarian cancer Phase 1
Primary completion expected year-end 2028.
Povetacicept — PMN (OLYMPUS) Phase 2/3
Primary completion expected year-end 2028.
Efgartigimod — Autoimmune Encephalitis (AIE) Phase 2
Primary completion expected Q1’29.
Q1’29AUPHAurinia Pharmaceuticals Voclosporin (LUPKYNIS) + belimumab or obinutuzumab or anifrolumab — Lupus nephritis (PRESERVE) Phase 4
Primary completion expected Q1’29.
Q1’29AUPHAurinia Pharmaceuticals Aritinercept (AUR200) — gMG Phase 1/2
Primary completion expected Q1’29.
H1’29AUTLAutolus Therapeutics AUTO8 — MM (MCARTY) Phase 1
Primary completion expected H1’29.
H1’29AVBPArriVent BioPharma ARR-002 — OC, EC Phase 1
First patient in H2’26→ Q3’26, primary completion H1’29, PC data at AADCR’26 with Aarvik.
Q1’29EIKNEikon Therapeutics EIK1005 — MSI-high adv. solid tumors Phase 1/2
Primary completion expected Q1’29.
Fruquintinib + sintilimab — EMC (2L; confirmatory) – CN (FRUSICA-3) Phase 3
Primary completion expected Q1’29.
INBRX-106 + pembrolizumab — 1L R/M PD-L1 CPS≥20 HNSCC Phase 2/3
Primary completion expected H1’29.
H1’29JAZZJazz Pharmaceuticals Zanidatamab + pembrolizumab+chemo — 1L HER2+ PD-L1+ GEA (ZANGEA) Phase 2
Primary completion expected H1’29.
H1’29JAZZJazz Pharmaceuticals Recombinant Erwinia asparaginase + chemo — ND high-risk ALL/LBL Phase 2
Primary completion expected H1’29.
H1’29KROSKeros Therapeutics Elritercept (TAK-226, KER-050) — 1L anemia with transfusion-dpdt ESA-naïve very low to intermediate risk MDS (ELRiSE MDS) Phase 3
Primary completion expected H1’29.
H1’29LBRXLB Pharmaceuticals LB-102 – Oral — MDD (Adjunct setting) Phase 2
First patient in early 2027, topline H1’29.
VRd followed by cilta-cel (Carvykti) — TI NDMM (CARTITUDE-5) Phase 3
Primary completion expected Q1’29.
Q1’29SABSSAB Biotherapeutics SAB-142 — Delaying progression of type 1 diabetes in T1D pts (Stage 3, ≤2y from diagnosis, 5-40y) – (PRISE-hATG) Phase 3
Primary completion expected Q1’29.
Q1’29SLDBSolid Biosciences SGT-003 — DMD (ambulant 7-11y) - (IMPACT DUCHENNE) Phase 3
Primary completion expected Q1’29.
H1’29SMMTSummit Therapeutics Ivonescimab (SMT112) + daraxonrasib or elironrasib or zoldonrasib — Solid Tumors incl. NSCLC, PDAC, CRC (global, RevMed-sponsored) – (RMC-APEX-103) Phase 1/2
Primary completion expected H1’29.
Povetacicept — Generalized Myasthenia Gravis (ETNA) Phase 2
Primary completion expected Q1’29.
Q1’29ZVRAZevra Therapeutics Celiprolol — COL3A1+ Vascular Ehlers-Danlos syndrome (vEDS) – (DiSCOVER) Phase 3
Primary completion expected Q1’29.
Latarcibart (VGA039) — Von Willebrand disease (VWD) – (VIVID-6) Phase 3
Phase 3 topline readout expected early 2029, from Vega Tx acquisition Jun 2026, phase 1/2 data at ISTH’26.
early’29MISTMilestone Pharmaceuticals Etripamil nasal spray — Atrial fibrillation with rapid ventricular rate (AFib-RVR) - (ReVeRA-301) Phase 3
Primary completion expected early 2029.
mid’29AAPGAscentage Pharma Lisaftoclax (APG-2575) + azacitidine — 1L HR-MDS (GLORA-4 – Global) Phase 3
Primary completion expected mid-2029.
ACI-24.060 — Alzheimer’s Disease (Prodromal), Down Syndrome (non-demented but with amyloid pathology) - (ABATE) Phase 1/2
Primary completion expected 2029.
Pemvidutide — MASH (F2-F3, biopsy-confirmed cohort & NIT-confirmed cohort) – (PERFORMA) Phase 3
First patient in Aug 2026, 52-week topline 2029.
2029AVBPArriVent BioPharma Firmonertinib — 1L+ EGFR PACC-mut NSCLC (ALPACCA) Phase 3
Primary completion expected 2029.
Pumitamig (BNT327, BMS986545) + chemo — 1L Adv. NSCLC (SQ & NSQ substudies) - (ROSETTA Lung-02) Phase 2/3
Primary completion expected 2029.
Pumitamig (BNT327, BMS986545) + physician’s choice chemo — 1L Adv. PD-L1 CPS<10% TNBC – (ROSETTA Breast-01) Phase 3
Primary completion expected 2029.
Pumitamig (BNT327, BMS986545) mono or + ipi — 1L Adv. or Unresectable HCC (ROSETTA HCC-206) Phase 1/2
Primary completion expected 2029.
Pumitamig (BNT327, BMS986545) mono or + ipi or + cabo or + casdatifan — 1L Adv. RCC (ROSETTA RCC-208) Phase 1/2
Primary completion expected 2029.
Elfetabart drozuntecan (BNT324, DB-1311) — 1L ARPI-XP chemo/taxane-naïve mCRPC – (BNT324-03) Phase 3
Primary completion expected 2029.
αβ T Cells, CD19 HD allo CAR-T — Autoimmune diseases Phase 1
Primary completion expected 2029.
Zanzalintinib — 1-2L NET (STELLAR-311) Phase 2/3
Primary completion expected mid-2029.
Amivantamab (RYBREVANT) + SOC pembro+chemo — 1L R/M HNSCC (OrigAMI-5) Phase 3
Primary completion expected mid-2029.
mid’29IKTInhibikase Therapeutics IkT-001 — PAH (IMPROVE-PAH) Phase 3
First patient in Apr 2026, Part A interim safety H1’27, Part A last patient in and Part B FI H2’27, Part A topline mid-2028 [PVR], Part B last patient in year-end 2028, Part B topline mid-2029 [6-minute walk distance].
2029NTLAIntellia Therapeutics AVC-203 — B-Cell malignancies (QUADvance) Phase 1/2
Primary completion expected 2029.
Prasinezumab — Parkinson’s disease (PARAISO) Phase 3
Primary completion expected mid-2029.
Coramitug (PRX004) — ATTR amyloidosis (CLEOPATTRA) Phase 3
Primary completion expected mid-2029.
mid’29RCKTRocket Pharmaceuticals RP-A701 — BAG3-associated Dilated CardioMyopathy Phase 1
Primary completion expected mid-2029.
2029RCUSArcus Biosciences Casdatifan + Pumitamig (BNT327, BMS986545) — 1L Adv. RCC (ROSETTA RCC-208) Phase 1/2
Primary completion expected 2029.
mid’29RIGLRigel Pharmaceuticals Olutasidenib (REZLIDHIA) + TMZ — 1L maintenance in mIDH1 high-grade glioma incl. DIPG (after RT induction) - (TarGet-D) Phase 2
Primary completion expected mid-2029.
mid’29SNDXSyndax Pharmaceuticals Revumenib (Revuforj) + ‘7+3’ IC — 1L fit [IC-eligible] NPM1m AML (REVEAL-ND NMP1) Phase 3
Primary completion expected mid-2029.
2029UTHRUnited Therapeutics UThymoKidney — End-stage renal disease (EXTEND) Phase 1/2
Primary completion expected 2029.
XP‑8121 (levothyroxine QW SC) — Hypothyroidism Phase 2
Weekly subcutaneous levothyroxine; Phase 3 first patient in H2’26, last patient in 2028, topline 2029. Positive Phase 2 topline May 2024 (open-label crossover): predictable bioavailability with sustained levels, TSH/T4 normalization using…
Weekly subcutaneous levothyroxine; Phase 3 first patient in H2’26, last patient in 2028, topline 2029. Positive Phase 2 topline May 2024 (open-label crossover): predictable bioavailability with sustained levels, TSH/T4 normalization using 45% less drug than oral daily equivalent, 4x target dose conversion factor when switching, higher treatment satisfaction, and 72% patient preference for the subcutaneous route.
Luxdegalutamide (ARV-766, JSB462) + tulmimetostat (DZR123) — Taxane-naïve mCRPC (TulmiSTAR-01) Phase 1/2
Primary completion expected H2’29.
IOV-4001 — Stage III-IV Melanoma (post-anti-PD-1) – (IOV-GM1-201 coh. 1) Phase 1/2
Primary completion expected H2’29.
IOV-4001 — 2-4L NSCLC (incl. post-anti-PD-1) - (IOV-GM1-201 coh. 2) Phase 1/2
Primary completion expected H2’29.
H2’29IOVAIovance Biotherapeutics IOV-4001 — Stage III-IV Melanoma (post-anti-PD-1) – (IOV-GM1-201 coh. 1) Phase 1/2
Primary completion expected H2’29.
H2’29IOVAIovance Biotherapeutics IOV-4001 — 2-4L NSCLC (incl. post-anti-PD-1) - (IOV-GM1-201 coh. 2) Phase 1/2
Primary completion expected H2’29.
Q3’29LQDALiquidia Corporation L606 — PH-ILD (Re-Spire - Global) Phase 3
Primary completion expected Q3’29.
Q3’29LRMRLarimar Therapeutics Nomlabofusp — Friedreich’s ataxia Phase 3
Primary completion expected Q3’29.
H2’29NRIXNurix Therapeutics Bexobrutideg (NX-5948) — 2L+ R/R CLL/SLL after prior covalent BTKi (DAYBreak CLL-306) Phase 3
Primary completion expected H2’29.
Sonrotoclax (BGB‑11417) + obinutuzumab — R/R CLL/SLL (CELESTIAL-RRCLL) Phase 3
Primary completion expected H2’29.
Rilparencel — Type 2 diabetes CKD (Stage 3b/4) – prevent/delay ESKD (PROACT 1) Phase 3
AA-supporting cohort fully enrolled; full enrollment H2’26, AA-enabling surrogate endpoint [eGFR slope] readout Q2’27, confirmatory composite time-to-event analysis topline H2’29.
H2’29TARSTarsus Pharmaceuticals Gildeuretinol (ALK-001) — Stargardt disease (NORTHSTAR) Phase 3
From the Alkeus acquisition (August 2026); topline H2’29. Primary endpoint is rate of retinal atrophic lesion growth over 24 months, with low-light visual acuity as secondary. Earlier data: statistically significant 29.5% slower annualized…
From the Alkeus acquisition (August 2026); topline H2’29. Primary endpoint is rate of retinal atrophic lesion growth over 24 months, with low-light visual acuity as secondary. Earlier data: statistically significant 29.5% slower annualized lesion growth versus placebo/natural history (n=50, TEASE-1); significant low-light VA loss 87% less likely versus placebo (TEASE-2); no treatment-related effects on night vision, dark adaptation, or color vision.
UGN-501 (ICVB-1042) — High‑grade NMIBC Preclinical
Primary completion expected H2’29.
H2’29VIRVir Biotechnology VIR-5525 ± pembrolizumab — EGFR+ Solid tumors incl. NSCLC, CRC, HNSCC, cSCC Phase 1
Primary completion expected H2’29.
Xanomeline-trospium (KarXT) mono or + KarEC — Autism-Related Irritability (NCT07284745 & NCT07285798) Phase 3
Primary completion expected Q3’29.
YE’29AAPGAscentage Pharma APG-3288 — R/R NHL, R/R CLL/SLL [+ Autoimmune Diseases] Phase 1
Primary completion expected year-end 2029.
YE’29ACTUActuate Therapeutics Elraglusib — R/R solid tumors incl. Melanoma, NSCLCn CRC, PDAC (Actuate-2601) Phase 1/2
Primary completion expected year-end 2029.
YE’29IOVAIovance Biotherapeutics Lifileucel, LN-144 (AMTAGVI) — Endometrial cancer (post-anti-PD-1) - (IOV-END-201) Phase 2
Primary completion expected year-end 2029.
YE’29KROSKeros Therapeutics Elritercept (TAK-226, KER-050) – addon to ruxolitinib — Anemia in myelofibrosis pts on ruxolitinib (ELRISE MF) Phase 3
Primary completion expected year-end 2029.
YE’29LYELLyell Immunopharma Ronde-Cel (rondecabtagene autoleucel, LYL314) — 2L aggressive LBCL (PiNACLE-H2H liso-cell or axi-cel) Phase 3
Primary completion expected year-end 2029.
YE’29RLMDRelmada Therapeutics NDV-01 — IR-NMIBC (Coh. 1), recurrent HR-NMIBC + CIS ± papillary disease after 1-2 prior LoT for BCG-unresp. NMIBC (Coh. 2a), recurrent HR-NMIBC w/o CIS + HG papillary disease after 1-2 prior LoTs for BCG-unresp. NMIBC (Coh. 2b) - (RESCUE) Phase 3
Primary completion expected year-end 2029.
YE’29SNDXSyndax Pharmaceuticals Revumenib (Revuforj) + ven+aza — 1L unfit [IC-ineligible] NPM1m or KMT2A-r(earranged) AML (EVOLVE-2) Phase 3
Primary completion expected year-end 2029.
Xanomeline-trospium (KarXT) — Schizophrenia (EMERGENT TEEN) Phase 3
Primary completion expected year-end 2029.
2030ALLOAllogene Therapeutics ALLO-316 — R/R RCC (TRAVERSE) Phase 1
Primary completion expected 2030.
2030ALNYAlnylam Pharmaceuticals Nucresiran — ATTR-CM (TRITON-CM) Phase 3
Primary completion expected 2030.
2030ALNYAlnylam Pharmaceuticals Zilebesiran — Hypertension (ZENITH) Phase 3
Primary completion expected 2030.
2030ALNYAlnylam Pharmaceuticals Mivelsiran — Down syndrome-associated AD Phase 2
Primary completion expected 2030.
Pumitamig (BNT327, BMS986545) — Stage III unresectable NSCLC (Adj. / PACIFIC setting) - (ROSETTA Lung-201) Phase 3
Primary completion expected 2030.
Pumitamig (BNT327, BMS986545) + chemo — 1L HER2- PD-L1+ Gastric/GEJ Cancer (ROSETTA Gastric-204) Phase 2/3
Primary completion expected 2030.
Pumitamig (BNT327, BMS986545) + chemo — 1L MSS unresectable mCRC – (ROSETTA CRC-203) Phase 2/3
Primary completion expected 2030.
BNT314, GEN1059 ± pumitamig+chemo — mCRC (1L & 2L+) Phase 1/2
Primary completion expected 2030.
Zanzalintinib + belzutifan — 2L+ adv. RCC after prog. on aPD-(L)1 & VEGFR TKIs in sequence or combo (LITESPARK-034) Phase 3
Primary completion expected 2030.
Imetelstat + aza or ven-aza — R/R AML (IMAGINE) Phase 1
Primary completion expected 2030.
2030IDYAIDEAYA Biosciences Darovasertib (IDE196) — Primary Uveal melanoma (Neoadjuvant) – (OptimUM-10) Phase 3
Primary completion expected 2030.
IMC-S118AI — T1D Phase 1
Primary completion expected 2030.
INS1202 — SOD1 Amyotrophic lateral sclerosis (ALS) Phase 1
Primary completion expected 2030.
2030IONSIonis Pharmaceuticals ION337 — Dravet Syndrome (ASCEND) Phase 1/2
Primary completion expected 2030.
2030IOVAIovance Biotherapeutics Lifileucel, LN-144 (AMTAGVI) — 2L NSCLC (post-chemo & post-anti-PD-1) - (IOV-LUN-202 coh. 1 & 2) Phase 2
Primary completion expected 2030.
2030IOVAIovance Biotherapeutics Lifileucel, LN-144 (AMTAGVI) — 2-4L NSCLC (incl. post-anti-PD-1) - (IOV-LUN-202 coh. 3B) Phase 2
Primary completion expected 2030.
2030IOVAIovance Biotherapeutics LN-145 Gen 3 + core biopsy — NSCLC (2L post-chemo & anti-PD-1) - (IOV-LUN-202 Coh. 3) Phase 2
Primary completion expected 2030.
Monalizumab + durva+chemo (neaoadj.) & +durva (adj.) — Neoadjuvant NSCLC & other tumors (NeoCOAST-2) Phase 2
Primary completion expected 2030.
2030JAZZJazz Pharmaceuticals Zanidatamab + SOC — Breast cancer (I-SPY 2) Phase 2
Primary completion expected 2030.
mRNA-4157 (V940) + pembrolizumab — Adjuvant in Fully resected Stage II-IIIB NSCLC (INTerpath-002) Phase 3
Primary completion expected 2030.
PRGN-2009 + pembrolizumab — 1L HPV-associated oropharyngeal cancer / HNSCC Phase 2
Primary completion expected 2030.
MORF-057, LY4268989 – mono & + mirikizumab — UC (moderate-to-severe) Phase 2
Primary completion expected 2030.
Ublituximab (BRIUMVI) - IV — Relapsing MS (RMS) – pediatric Phase 2/3
Primary completion expected 2030.
2030TSHATaysha Gene Therapies TSHA-105 — SLC13A5 Citrate Transporter Disorder Phase 1/2
Primary completion expected 2030.
Dabogratinib (TYRA-300) — Achondroplasia (Children) - (BEACH301) Phase 2
Primary completion expected 2030.
2030VCELVericel Corporation MACI ankle — Talus cartilage/osteochondral lesions – ankle (17-65y) - (MASCOT) Phase 3
Primary completion expected 2030.
Orelabrutinib — Primary progressive multiple sclerosis (PPMS) – (PriMroSe) Phase 3
Primary completion expected 2030.
Orelabrutinib — Non-active Secondary progressive multiple sclerosis (naSPMS) – (Monarch) Phase 3
Primary completion expected 2030.
Xanomeline-trospium (KarXT) + KarEC — Schizophrenia, Autism-Related Irritability (NCT07424404) Phase 3
Primary completion expected 2030.
YE’30DYNDyne Therapeutics Zeleciment rostudirsen (DYNE-251) — DMD (exon 51) – (FORZETTO) Phase 3
Primary completion expected year-end 2030.
Tenapanor (IBSRELA) — IBS-C (12-17y) – (TEN-01-304) Phase 3
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
Tenapanor (IBSRELA) — IBS-C (6-11y) – (TEN-01-202) Phase 2
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
BIIB142 — Autoimmune diseases Phase 1
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
Q3’26 OverdueCCCCC4 Therapeutics BIIB142 — Autoimmune diseases Phase 1
Primary completion expected Q3’26 [Oct 2026 check] Sheet error: BIIB142 is Biogen's oral IRAK4 degrader, originated from the 2018 Biogen-C4T collaboration; C4T is only eligible for milestones. No Phase 1 readout reported by Biogen. Deep…
Primary completion expected Q3’26 [Oct 2026 check] Sheet error: BIIB142 is Biogen's oral IRAK4 degrader, originated from the 2018 Biogen-C4T collaboration; C4T is only eligible for milestones. No Phase 1 readout reported by Biogen. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
Q3’26 OverdueCRDFCardiff Oncology Onvansertib (NMS-1286937) + NALIRIFOX — 1L mPDAC / biomarker-selected Phase 2
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
OCU200 — DME / DR / Wet AMD Phase 1
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
Q3’26 OverdueTYRATyra Biosciences TYRA-200 — Adv solid tumors - FGF/FGFR+ all comers in Part A & FGFRi-XP ICC w/ FGFR2 alterations in Part B (SURF201) Phase 1
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
S4V2 — Shigellosis (adults 18-50y) Phase 2
Human challenge data 2026→ mid-2026→ Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
S4V2 — Shigellosis (infants 8-10mo) Phase 2
Phase 2 data readout expected H1’26→ mid-2026→ Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
Q3’26 OverdueVNDAVanda Pharmaceuticals VTR-297 (Tricostatin A) — Onychomycosis Phase 2
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
Q3’26 OverdueVNDAVanda Pharmaceuticals VSJ-110 — Dry Eye Disease Phase 2
Primary completion expected Q3’26. Deep check Oct 7, 2026: no company disclosure, SEC filing, or registry results update found.
2026FBRXForte Biosciences FB102 — Alopecia areata Phase 1
✓ Acquired by argenx ($2.2B, closed Aug 27, 2026); ticker delisted — catalyst retired as no longer applicable.
Phase 1 data readout expected 2026.
2026IONSIonis Pharmaceuticals Ulefnersen — Amyotrophic lateral sclerosis (caused by FUS gene) Phase 3
✓ 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…
Phase 3 data readout expected 2026. 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…
Phase 3 data readout expected 2026. 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... Reported Sept 22, 2026.
2026IONSIonis Pharmaceuticals Sefaxersen (RG6299) — IgA Nephropathy (IgAN) Phase 3
✓ 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…
Phase 3 data readout expected 2026. 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…
Phase 3 data readout expected 2026. 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... Reported Sept 23, 2026.
H2’26AARDAardvark Therapeutics ARD-101 — Hypothalamic obesity (HONOR) Phase 2
✓ FDA lifted the clinical hold on the IND for ARD-101 for hyperphagia in Prader-Willi syndrome (PWS): clinical development may resume. (discontinuation, Oct 22, 2026.)
First patient in initially planned H2’25 with preliminary or interim data H2’26, assumedly also paused or on clinical hold. FDA lifted the clinical hold on the IND for ARD-101 for hyperphagia in Prader-Willi syndrome (PWS): clinical…
First patient in initially planned H2’25 with preliminary or interim data H2’26, assumedly also paused or on clinical hold. FDA lifted the clinical hold on the IND for ARD-101 for hyperphagia in Prader-Willi syndrome (PWS): clinical development may resume. (discontinuation, Oct 22, 2026.)
Prulacabtagene leucel (prula-cel, ADI‑001) — Autoimmune diseases (LN & SLE, SSc, IIM / SPS, AAV, RA) Phase 1
✓ 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…
First patient in Q4’24, last patient in early Sept 2026, comprehensive clinical update including data from n=22 LN and SLE patients with 6 months+ follow-up of which n=13 with 12 months+ follow-up, topline Q3’26→ Sept 2026, prep. For an LN…
First patient in Q4’24, last patient in early Sept 2026, comprehensive clinical update including data from n=22 LN and SLE patients with 6 months+ follow-up of which n=13 with 12 months+ follow-up, topline Q3’26→ Sept 2026, prep. For an LN or LN+SLE pivotal trial to start H2’26 after “productive” FDA interactions, clinical data in SSc H2’26. 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... Reported Sept 28, 2026.
ACI-7104.056 — Parkinson’s Disease (VacSyn) Phase 2
✓ 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%…
Final week 104 Part 1 readout H2’26, trial extended Jun 2026 for 2 years of additional follow-up, positive immune/biomarket data Dec 2025: 100% responder rate as per Ab response against immunizing a-syn target antigen; Ab titers in serum >…
Final week 104 Part 1 readout H2’26, trial extended Jun 2026 for 2 years of additional follow-up, positive immune/biomarket data Dec 2025: 100% responder rate as per Ab response against immunizing a-syn target antigen; Ab titers in serum > 500x higher vs placebo at week 76 ie 2 weeks 6th immunization; presence of immunizing IgGs in CSF; NfL stabilization vs increase in placebo. 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... Reported Sept 23, 2026.
H2’26ACRSAclaris Therapeutics ATI-052 — Severe asthma Phase 1
✓ ATI-052: 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,…
First patient in Feb 2026, d29 topline H2’26, phase 2b planned. ATI-052: 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…
First patient in Feb 2026, d29 topline H2’26, phase 2b planned. ATI-052: 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... (discontinuation, Oct 27, 2026.)
H2’26APGEApogee Therapeutics APG279 (APG777 + APG990) — Atopic dermatitis Phase 1
✓ Acquired by AbbVie ($10.9B, closed Sep 3, 2026); ticker delisted — catalyst retired as no longer applicable.
Head-to-head dupilimumab, 24-week interim data H2’26.
Q3’26ARCTArcturus Therapeutics ARCT-810 (LUNAR-OTC) — Ornithine transcarbamylase (OTC) deficiency Phase 2
✓ 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,…
EU phase 2 in n=8 completed [6 ARCT-810 and 2 placebo], US phase 2 in up to n=6 ongoing → data released Jun 2025 on n=8 EU and n=3 US, alignment with regulatory agencies on pivotal strategy H2’26, data and regulatory plan Q3’26.…
EU phase 2 in n=8 completed [6 ARCT-810 and 2 placebo], US phase 2 in up to n=6 ongoing → data released Jun 2025 on n=8 EU and n=3 US, alignment with regulatory agencies on pivotal strategy H2’26, data and regulatory plan Q3’26. 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.
H2’26AVBPArriVent BioPharma Firmonertinib — 1L EGFR Ex20ins-mut NSCLC (FURVENT) Phase 3
✓ 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…
Fully enrolled, topline early 2026 → mid-2026 → H2’26. 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…
Fully enrolled, topline early 2026 → mid-2026 → H2’26. 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. Reported Oct 6, 2026.
H2’26BEAMBeam Therapeutics BEAM-301 — Glycogen storage disease Ia (R83C-mut) Phase 1/2
✓ 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,…
DL1 complete and DL2 ongoing, initial data 2026→ H2’26. 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…
DL1 complete and DL2 ongoing, initial data 2026→ H2’26. 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... (data, Oct 13, 2026.)
Lecanemab SC-AI (LEQEMBI IQLIK) — Early AD (Initiation) Registration
✓ FDA approved the LEQEMBI IQLIK sBLA (once-weekly SC lecanemab autoinjector) as an initiation dose for early AD on July 13, 2026; US launch planned for late Aug 2026.
PDUFA decision expected mid-end-May 2026, EU decision Q2-Q3’26. FDA approved the LEQEMBI IQLIK sBLA (once-weekly SC lecanemab autoinjector) as an initiation dose for early AD on July 13, 2026; US launch planned for late Aug 2026.
Gotistobart (BNT316, ONC-392) + pembrolizumab — Platinum-resistant ovarian cancer Phase 2
✓ 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…
Primary completion expected Q3’26. 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…
Primary completion expected Q3’26. 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.
Budoprutug - IV — Primary membranous nephropathy (PMN) Phase 2
✓ 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…
Initial Phase 2 data expected H2’26, primary completion H2’27. 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,…
Initial Phase 2 data expected H2’26, primary completion H2’27. 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... Reported Oct 26, 2026.
H2’26CORTCorcept Therapeutics Relacorilant + nab-paclitaxel + bevacizumab — PROC (BELLA Part A), PSOC ater prog. on PARPi (BELLA Part B), 2-3L EC (BELLA Part C) Phase 2
✓ 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…
BELLA Part A/B/C data by year-end 2026→ H2’26. 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…
BELLA Part A/B/C data by year-end 2026→ H2’26. 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... Reported Oct 26, 2026.
H2’26CRBUCaribou Biosciences CB-011 — R/R multiple myeloma (CaMMouflage) Phase 1
✓ Board approved a process to evaluate strategic alternatives (merger, acquisition, business combination or other transactions; Wedbush engaged as exclusive advisor); will discontinue further clinical…
450m cohort expansion data and dose-escalation long-term follow-up H2’26 in BCMA-naïve and BCMA-XP, HLA class II matching+intensified LD, n=12 BCMA-naïve at RDE of 450m+intensified LD: ORR 92%; CR-sCR 75%; MRD- 91%. EHA’26 update: 50% in…
450m cohort expansion data and dose-escalation long-term follow-up H2’26 in BCMA-naïve and BCMA-XP, HLA class II matching+intensified LD, n=12 BCMA-naïve at RDE of 450m+intensified LD: ORR 92%; CR-sCR 75%; MRD- 91%. EHA’26 update: 50% in ≥CR at 15m; 1 treatment-related death from IEC-AH out of n=35 treated with selected LD regimen; 25% grade 3+ infections and 8% grade 3+ IEC-HS and 42% grade 3+ prolonged cytopenias in n=12 BCMA-naïve at RDE+intensified LD. 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... Reported Oct 6, 2026.
H2’26DAWNDay One Biopharmaceuticals DAY301 (MTX-13) — Solid tumors Phase 1
✓ Acquired by Servier ($2.5B, closed Apr 23, 2026); ticker delisted — catalyst retired as no longer applicable.
Initial Phase 1 data expected H2’26.
H2’26DBVTDBV Technologies VIASKIN peanut patch (DBV712) — Peanut allergy - ages 1-3y (EPITOPE, EPITOPE-OLE, COMFORT Toddlers) Phase 3
✓ DBV submitted BLA to FDA for Viaskin Peanut patch in children aged 4-7 years, supported by the successful Phase 3 VITESSE trial. Reported Sept 29, 2026.
EPITOPE completed, COMFORT Toddlers phase 3 last patient in Q2’26? and topline H2’26 [potential shift into Q1’27?], AA filing H2’26 [potential shift into Q1’27?]. DBV submitted BLA to FDA for Viaskin Peanut patch in children aged 4-7…
EPITOPE completed, COMFORT Toddlers phase 3 last patient in Q2’26? and topline H2’26 [potential shift into Q1’27?], AA filing H2’26 [potential shift into Q1’27?]. DBV submitted BLA to FDA for Viaskin Peanut patch in children aged 4-7 years, supported by the successful Phase 3 VITESSE trial. Reported Sept 29, 2026.
Q3’26ELVNEnliven Therapeutics ELVN-002 — HER2m NSCLC Phase 1
✓ Enliven discontinued ELVN-002 development and is exploring strategic alternatives for the program (per Q1 2026 10-Q); the Ph1a/1b HER2m NSCLC primary-completion readout did not occur.
Primary completion expected Q3’26. Enliven discontinued ELVN-002 development and is exploring strategic alternatives for the program (per Q1 2026 10-Q); the Ph1a/1b HER2m NSCLC primary-completion readout did not occur.
H2’26GLUEMonte Rosa Therapeutics MRT-8102 — IL-1β/NLRP3 driven inflammatory diseases (GFORCE-1) Phase 1
✓ 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…
Positive hsCRP reductions in SAD/MAD Jan 2026 in patients with elevated cardiovascular disease risks, positive SAD; MAD safety March 2026, expansion Part 3 hsCRP proof-of-concept readout H2’26. Announced positive data from GFORCE-1, a…
Positive hsCRP reductions in SAD/MAD Jan 2026 in patients with elevated cardiovascular disease risks, positive SAD; MAD safety March 2026, expansion Part 3 hsCRP proof-of-concept readout H2’26. 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... Reported Oct 1, 2026.
H2’26IDYAIDEAYA Biosciences IDE849 (SHR-4849) ± IDE161 or + durvalumab — SCLC, NETs, NECs, melanoma Phase 1
✓ 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…
Combo with IDE161 first patient in March 2026, monotherapy dose-escalation data H2’26, combo dose-escalation initiation after monotherapy dose-escalation completion, China ORR/PFS/12 months-OS data in n~100 SCLC and NEC at ESMO’26 by…
Combo with IDE161 first patient in March 2026, monotherapy dose-escalation data H2’26, combo dose-escalation initiation after monotherapy dose-escalation completion, China ORR/PFS/12 months-OS data in n~100 SCLC and NEC at ESMO’26 by Hengui, expansion cohort ongoing at 2.4 and 3.5 mg/kg IV Q3W to determine the RP3D. 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... Reported Oct 23, 2026.
Eftilagimod alfa + pembrolizumab + chemo — 1L NSCLC (TACTI-004) Phase 3
✓ 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,…
Futile mid-March 2026, Jul 2026 update: ORR 42.9% efti+SOC vs 55.1% SOC control. Similar outcomes in bot hSQ a NSQ; no superiority in any TPS subgrp. Negative outcomes deemed to different immunological resp. Vs prev. Studies, additional…
Futile mid-March 2026, Jul 2026 update: ORR 42.9% efti+SOC vs 55.1% SOC control. Similar outcomes in bot hSQ a NSQ; no superiority in any TPS subgrp. Negative outcomes deemed to different immunological resp. Vs prev. Studies, additional data Q3’26. 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.
DISC-3405 — Polycythemia vera (RESTORE-PV) Phase 2
✓ 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…
Initial Phase 2 data expected H2’26→ Q3’26, SOHO’26: safety in n=40 cohorts A/B and PK/PD/efficacy in n=20 cohort A. Disc Medicine Presents Initial Results from RESTORE-PV Phase 2 Trial in Patients with Polycythemia Vera (PV) at the 14 th…
Initial Phase 2 data expected H2’26→ Q3’26, SOHO’26: safety in n=40 cohorts A/B and PK/PD/efficacy in n=20 cohort A. 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...
VYD2311 — COVID-19 prevention (DECLARATION) Phase 3
✓ 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…
First patient in late 2025, trial upsized by +5Apr 00, 2026 in addition to 1818 initially planned, topline mid-2026 → Q3’26 → ~end-Q3’26. Positive topline LIBERTY Phase 3 (n=210, randomized, double-blind, active-controlled): VYD2311 met…
First patient in late 2025, trial upsized by +5Apr 00, 2026 in addition to 1818 initially planned, topline mid-2026 → Q3’26 → ~end-Q3’26. 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).
Q3’26KYTXKyverna Therapeutics KYV-101 (miv-cel, mivocabtagene autoleucel) + LD — Myasthenia gravis (KYSA-6) Phase 2/3
✓ 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;…
Additional phase 2 long-term follow-up data Q3’26. 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,…
Additional phase 2 long-term follow-up data Q3’26. 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.
Q3’26KYTXKyverna Therapeutics KYV-101 (miv-cel, mivocabtagene autoleucel) + LD — Stiff person syndrome (KYSA-8) Registration
✓ 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;…
Rolling BLA filing initiated mid-May 2026 and expected to be completed Q4’26, registrational single-arm phase 2, positive topline data mid-Dec 2025 [median 46% improvement On Timed 25-Foot Walk at week 16 in n=26], LBA presented at AAN’26,…
Rolling BLA filing initiated mid-May 2026 and expected to be completed Q4’26, registrational single-arm phase 2, positive topline data mid-Dec 2025 [median 46% improvement On Timed 25-Foot Walk at week 16 in n=26], LBA presented at AAN’26, 1y follow-up data H2’26→ Q3’26. 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.
VDPHL01 — Androgenetic Alopecia, Pattern hair loss (males) - (Study 304) Phase 3
✓ VDPHL01: veradermics to share topline results on October 19, 2026 (investor call and live webcast 8:00 a.m. (topline, Oct 18, 2026.)
2nd trial. 8.5mg BID and OD, 6 months TAHC topline H2’26. VDPHL01: veradermics to share topline results on October 19, 2026 (investor call and live webcast 8:00 a.m. (topline, Oct 18, 2026.)
OCU400 — Retinitis pigmentosa (gene-agnostic) - (liMeliGhT) Phase 3
✓ 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…
Last patient in early-March 2026, rolling submission initiation planned Q3’26 and completion mid-2027, 1-year topline Q1’27, primary endpoint =. Bahamas Longevity and Regenerative Therapies Act (LARTA) Board granted provisional approval…
Last patient in early-March 2026, rolling submission initiation planned Q3’26 and completion mid-2027, 1-year topline Q1’27, primary endpoint =. 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.
Deucrictibant (XR) — Hereditary angioedema (HAE) – prophylaxis (12y+) - (CHAPTER-3/4) Phase 3
✓ 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…
CHAPTER-3 topline Q3’26, CHAPTER-4 = phase 3 OLE, LTE data from CHAPTER-1 phase 2 at AAAAI’26. 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):…
CHAPTER-3 topline Q3’26, CHAPTER-4 = phase 3 OLE, LTE data from CHAPTER-1 phase 2 at AAAAI’26. 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.
H2’26ROIVRoivant Sciences Brepocitinib — Non-Infectious Uveitis Phase 3
✓ brepocitinib: 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…
Phase 3 data readout expected H2’26. brepocitinib: 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…
Phase 3 data readout expected H2’26. brepocitinib: 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... (topline, Oct 21, 2026.)
Q3’26TECXTectonic Therapeutic TX2100 — Hereditary hemorrhagic telangiectasia (HHT) Phase 1
✓ 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…
Phase 1 first patient in Feb 2026 in healthy volunteers. 6 dose levels completed as of Aug 2026, topline end Q3’26, then phase 2 first patient in early 2027. Favorable TX2100 Phase 1a topline (n=48, randomized double-blind…
Phase 1 first patient in Feb 2026 in healthy volunteers. 6 dose levels completed as of Aug 2026, topline end Q3’26, then phase 2 first patient in early 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.
Q3’26VKTXViking Therapeutics VK2735 - SC — Obesity (maintenance) Phase 1
✓ 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…
SC maintenance readout Q3’26, additional cohorts disclosed Apr 2026 [10 and 15mg Q4W+5 and 10mg Q2W added], oral maintenance to proceed after SC maintenance with data H1’27. Positive VK2735 maintenance-study topline: 22% placebo-adjusted…
SC maintenance readout Q3’26, additional cohorts disclosed Apr 2026 [10 and 15mg Q4W+5 and 10mg Q2W added], oral maintenance to proceed after SC maintenance with data H1’27. 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.
Sept’26ABEOAbeona Therapeutics UX111 (ebisufligene etisparvovec) — MPS IIIA (Sanfilippo syndrome type A) Registration
✓ FDA approved FAYUVI (rebisufligene etisparvovec, formerly UX111) for Sanfilippo syndrome type A (MPS IIIA) on Sept 18, 2026; Ultragenyx took it through late-stage development and approval after…
Re-filing end Jan 2026 based on Ultragenyx phase 1/2 dataset, PDUFA Sept 19, 2026. FDA approved FAYUVI (rebisufligene etisparvovec, formerly UX111) for Sanfilippo syndrome type A (MPS IIIA) on Sept 18, 2026; Ultragenyx took it through…
Re-filing end Jan 2026 based on Ultragenyx phase 1/2 dataset, PDUFA Sept 19, 2026. FDA approved FAYUVI (rebisufligene etisparvovec, formerly UX111) for Sanfilippo syndrome type A (MPS IIIA) on Sept 18, 2026; Ultragenyx took it through late-stage development and approval after out-licensing from Abeona (Abeona's ABO-102) in 2022.
Sept’26CCCCC4 Therapeutics Cemsidomide + dex — 4L+ R/R MM (MOMEMTUM) Phase 2
✓ C4 presented cemsidomide biomarker data at the IMS annual meeting on Sept 25, 2026: Ph1 cemsidomide+dex showed coordinated T-cell/NK-cell activation with strongest signals at 75/100 ug; first Ph1b…
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at…
Registrational, first patient in Feb 2026, last patient in ~Q1’27, RP2D 100µg, phase 2 initial ORR/DoR/safety H2’27, efficacy/safety topline mid-2028, potential NDA filing in 4L+ by year-end 2028, phase 1 data update at EHA’26: ORR 53% at RP2D of 100µg [also MTD] and ORR 40% at 75µg and ORR 36% across all dose levels; ORR 53% in prior CAR-T or TCE and 47% in Prior BCMA and 47% in 6L+ at RP2D. mDoR 7.9 months across all dose levels, phase 1 biomarker and translational data Sept 2026 at IMS. C4 presented cemsidomide biomarker data at the IMS annual meeting on Sept 25, 2026: Ph1 cemsidomide+dex showed coordinated T-cell/NK-cell activation with strongest signals at 75/100 ug; first Ph1b combo-with-elranatamab dose level (75 ug) declared safe, advancing to expansion/escalation.
CLYM116 — IgA nephropathy Phase 1
✓ Climb Bio reported Ph1 healthy-volunteer data Sept 3, 2026 (N=46): ~29-day half-life (3x sibeprenlimab), >90% free APRIL and 60-75% IgA/Gd-IgA1/IgM suppression from a single 320 mg SC dose, well…
Initial PK/PD data in healthy volunteers Sept 2026: t1/2 29d; near-complete APRIL suppression with single 320mg dose; 60-75% IgA Gd-IgA1 suppression through 12-week supporting Q12W-Q3M dosing, additional phase 1 data Q4’26; China phase 1…
Initial PK/PD data in healthy volunteers Sept 2026: t1/2 29d; near-complete APRIL suppression with single 320mg dose; 60-75% IgA Gd-IgA1 suppression through 12-week supporting Q12W-Q3M dosing, additional phase 1 data Q4’26; China phase 1 data by Mabworks Q4’26. Climb Bio reported Ph1 healthy-volunteer data Sept 3, 2026 (N=46): ~29-day half-life (3x sibeprenlimab), >90% free APRIL and 60-75% IgA/Gd-IgA1/IgM suppression from a single 320 mg SC dose, well tolerated; supports Q12W dosing in the ongoing NAVIGATE-2 Ph2.
Zilurgisertib — Fibrodysplasia ossificans progressiva Registration
✓ FDA approved zilurgisertib (Atebrioz, Mirum) for FOP in patients 12+ on Sept 25, 2026, one day ahead of the Sept 26 PDUFA; Mirum licensed the oral ALK2 inhibitor from Incyte.
PDUFA decision expected Sept 26, 2026 with Priority Review, positive registrational phase 2 topline Jun 2026 and at ENDO’26. FDA approved zilurgisertib (Atebrioz, Mirum) for FOP in patients 12+ on Sept 25, 2026, one day ahead of the Sept…
PDUFA decision expected Sept 26, 2026 with Priority Review, positive registrational phase 2 topline Jun 2026 and at ENDO’26. FDA approved zilurgisertib (Atebrioz, Mirum) for FOP in patients 12+ on Sept 25, 2026, one day ahead of the Sept 26 PDUFA; Mirum licensed the oral ALK2 inhibitor from Incyte.
Sept’26MIRMMirum Pharmaceuticals Zilurgisertib — Fibrodysplasia ossificans progressiva Registration
✓ 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…
PDUFA decision expected Sept 26, 2026 with Priority Review, positive registrational phase 2 topline Jun 2026 and at ENDO’26, in-licensed from Incyte Q2’26. FDA approved Atebrioz (zilurgisertib) for FOP patients aged 12+ (once-daily oral…
PDUFA decision expected Sept 26, 2026 with Priority Review, positive registrational phase 2 topline Jun 2026 and at ENDO’26, in-licensed from Incyte Q2’26. 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.
UX111 (ebisufligene etisparvovec) — MPS IIIA (Sanfilippo syndrome type A) Registration
✓ 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 +…
Re-filing end Jan 2026 based on phase 1/2 dataset, PDUFA Sept 19, 2026. FDA standard full approval of FAYUVI (rebisufligene etisparvovec-hopf, UX111) for pediatric MPS IIIA (Sanfilippo syndrome Type A): first-ever treatment for the…
Re-filing end Jan 2026 based on phase 1/2 dataset, PDUFA Sept 19, 2026. 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.
Apitegromab - IV — Spinal muscular atrophy (SMA) – ages 2y+ Registration
✓ 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 ·…
Potential EU approval mid-2026 → shifted to ‘later in 2026’ pending FDA reclassification of Catalent Indiana post inspection [or even later if switch to fill’n’finish back-up], ‘constructive’ meeting between FDA and Catalent Indiana…
Potential EU approval mid-2026 → shifted to ‘later in 2026’ pending FDA reclassification of Catalent Indiana post inspection [or even later if switch to fill’n’finish back-up], ‘constructive’ meeting between FDA and Catalent Indiana early-Q1’26, resubmission end-March 2026 to FDA after reinspection [review with 2nd fill’n’finish site]. PDUFA Sept 30, 2026, CRL Sept 2025 due to CMC deficiencies. 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...
Oct’26ACADACADIA Pharmaceuticals remlifanserin, ACP-204 — Alzheimer's disease psychosis (RADIANT) Phase 2/3
✓ 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…
Phase 2 topline Aug-Oct 2026 timeframe→ Sept-Oct 2026, phase 3 screening started. 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…
Phase 2 topline Aug-Oct 2026 timeframe→ Sept-Oct 2026, phase 3 screening started. 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... Reported Sept 24, 2026.
Oct’26ALNYAlnylam Pharmaceuticals ALN-HTT02 — Huntington’s disease Phase 1
✓ 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…
Initial Phase 1 data expected Oct 2026 at EHDN. 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…
Initial Phase 1 data expected Oct 2026 at EHDN. 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... Reported Oct 23, 2026.
VLS-01 (DMT) — Treatment-resistant depression (TRD) – (ELUMINA) Phase 2
✓ Acquired by Eli Lilly ($2.8B, closed Sep 11, 2026); ticker delisted — catalyst retired as no longer applicable.
Last patient in Jul 2026, phase 2b topline Q4’26.
Q4’26CRNXCrinetics Pharmaceuticals Atumelnant (CRN04894) — ACTH‑dependent Cushing’s syndrome Phase 1/2
✓ Acquired by Vertex ($10.0B, closed Sep 1, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected Q4’26.
DURAVYU (vorolanib intravitreal insert) — LUCIA — wet age-related macular degeneration Phase 3
✓ Missed noninferiority on BCVA vs aflibercept in full dataset; stock fell ~67%. Reported Aug 17, 2026.
LUCIA Phase 3 topline expected Q4 2026 (company guidance Sep 10, 2026). Second wet-AMD pivotal trial after LUGANO missed its BCVA non-inferiority primary Aug 17, 2026; company targets NDA 1H27 contingent on LUCIA. Missed noninferiority on…
LUCIA Phase 3 topline expected Q4 2026 (company guidance Sep 10, 2026). Second wet-AMD pivotal trial after LUGANO missed its BCVA non-inferiority primary Aug 17, 2026; company targets NDA 1H27 contingent on LUCIA. Missed noninferiority on BCVA vs aflibercept in full dataset; stock fell ~67%. Reported Aug 17, 2026.
Rinatabart sesutecan (Rina-S) — 2-4L Pt-Resistant Ovarian Cancer (RAINFOL-01) Phase 1/2
✓ 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. Reported Oct 3, 2026.
Potential registrational phase 2 readout H2’26→ Q4’26. 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. Reported Oct 3,…
Potential registrational phase 2 readout H2’26→ Q4’26. 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. Reported Oct 3, 2026.
Oct’26IONSIonis Pharmaceuticals Bepirovirsen, GSK3228836 (Hibsago) — Hepatitis B Virus Infection (B-Well 1 & 2) Marketed
✓ HIBSAGO (bepirovirsen): ionis Pharmaceuticals announced that partner GSK received FDA approval of HIBSAGO (bepirovirsen) for the treatment of chronic hepatitis B (CHB) virus infection in combination…
JP approval Aug 2026, PDUFA Oct 26, 2026. Priority Review, positive phase 3 topline Jan 2016, EASL’26 data: ~20% functional cure at week 72 vs 0% placebo in overall pop.; 26% vs 0% in lower bsln viral activity. HIBSAGO (bepirovirsen):…
JP approval Aug 2026, PDUFA Oct 26, 2026. Priority Review, positive phase 3 topline Jan 2016, EASL’26 data: ~20% functional cure at week 72 vs 0% placebo in overall pop.; 26% vs 0% in lower bsln viral activity. HIBSAGO (bepirovirsen): 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... (approval, Oct 20, 2026.)
Bitopertin — Erythropoietic porphyrias (EPP & XLP) – (APOLLO) Phase 3
✓ 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…
Confirmatory, readout Q4’26 [vs early 2027] and n=183 [from 150 planned]. 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…
Confirmatory, readout Q4’26 [vs early 2027] and n=183 [from 150 planned]. 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%... Reported Oct 19, 2026.
Q4’26KYMRKymera Therapeutics KT-579 — Autoimmune diseases (e.g., SLE, IBD, RA) Phase 1
✓ 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,…
First patient in Q1’26, readout H2’26→ Q4’26 in healthy volunteers. 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…
First patient in Q1’26, readout H2’26→ Q4’26 in healthy volunteers. 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... Reported Oct 29, 2026.
Q4’26MIRMMirum Pharmaceuticals Maralixibat (LIVMARLI) — Cholestatic pruritus (due to add. rare cholestatic conditions) – (LIVMARLI EXPAND) Phase 3
✓ 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…
Phase 3 topline readout expected Q4’26. 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…
Phase 3 topline readout expected Q4’26. 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... Reported Oct 12, 2026.
VAX-31 — Pneumococcal disease prevention (IPD, 50y+) – (OPUS-1: H2H Capvaxive & Prevnar 20, OPUS-2: with or 1mo after HD flu vax, OPUS-3: pop. prev. vaccinated with lower-valency pneumococcal vax) Phase 3
✓ Vaxcyte reported positive topline data from OPUS-1, the pivotal adult Phase 3 noninferiority trial of VAX-31 (31-valent pneumococcal conjugate vaccine). Reported Oct 5, 2026.
All 3 studies fully enrolled, OPUS-1 = non-inferiority phase 3 safety/tol and immunogenicity readout Q4’26, OPUS-2/3 readouts H1’27. Vaxcyte reported positive topline data from OPUS-1, the pivotal adult Phase 3 noninferiority trial of…
All 3 studies fully enrolled, OPUS-1 = non-inferiority phase 3 safety/tol and immunogenicity readout Q4’26, OPUS-2/3 readouts H1’27. Vaxcyte reported positive topline data from OPUS-1, the pivotal adult Phase 3 noninferiority trial of VAX-31 (31-valent pneumococcal conjugate vaccine). Reported Oct 5, 2026.
VAX-31 — Pneumococcal disease prevention (IPD, 50y+) – (OPUS-1: H2H Capvaxive & Prevnar 20, OPUS-2: with or 1mo after HD flu vax, OPUS-3: pop. prev. vaccinated with lower-valency pneumococcal vax) Phase 3
✓ Vaxcyte reported positive topline data from OPUS-1, the pivotal adult Phase 3 noninferiority trial of VAX-31 (31-valent pneumococcal conjugate vaccine). Reported Oct 5, 2026.
All 3 studies fully enrolled, OPUS-1 = non-inferiority phase 3 safety/tol and immunogenicity readout Q4’26, OPUS-2/3 readouts H1’27. Timing updated Oct 7, 2026 per company guidance: now guides Oct’26 (was H1’27). Vaxcyte reported positive…
All 3 studies fully enrolled, OPUS-1 = non-inferiority phase 3 safety/tol and immunogenicity readout Q4’26, OPUS-2/3 readouts H1’27. Timing updated Oct 7, 2026 per company guidance: now guides Oct’26 (was H1’27). Vaxcyte reported positive topline data from OPUS-1, the pivotal adult Phase 3 noninferiority trial of VAX-31 (31-valent pneumococcal conjugate vaccine). Reported Oct 5, 2026.
Oct’26RAPPRapport Therapeutics RAP-219 — Bipolar disorder (mania) Phase 2
✓ 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,…
Phase 2 topline readout expected Oct 2026, SAP modified and target enrollment increased for the phase 2 to potential be considered as confirmatory evidence of effectiveness. Positive Phase 2 topline results for RAP-219 (TARPγ8-selective…
Phase 2 topline readout expected Oct 2026, SAP modified and target enrollment increased for the phase 2 to potential be considered as confirmatory evidence of effectiveness. 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... Reported Oct 20, 2026.
Q4’26SNDXSyndax Pharmaceuticals Axatilimab (Niktimvo) + ruxo — 1L cGVHD Phase 2
✓ Phase 2 MAXPIRe topline in idiopathic pulmonary fibrosis (IPF): trial did not meet its primary endpoint (annualized rate of FVC decline). Reported Oct 12, 2026.
Incyte RCT, data Q4’26, primary completion mid-2027. Phase 2 MAXPIRe topline in idiopathic pulmonary fibrosis (IPF): trial did not meet its primary endpoint (annualized rate of FVC decline). Reported Oct 12, 2026.
Q4’26SNDXSyndax Pharmaceuticals Axatilimab (Niktimvo) — IPF (MAXPIRe) Phase 2
✓ Phase 2 MAXPIRe topline in idiopathic pulmonary fibrosis (IPF): trial did not meet its primary endpoint (annualized rate of FVC decline). Reported Oct 12, 2026.
Syndax trial, topline Q4’26, primary endpoint = annualized rate of decline of FVC over 26 weeks. Phase 2 MAXPIRe topline in idiopathic pulmonary fibrosis (IPF): trial did not meet its primary endpoint (annualized rate of FVC decline).…
Syndax trial, topline Q4’26, primary endpoint = annualized rate of decline of FVC over 26 weeks. Phase 2 MAXPIRe topline in idiopathic pulmonary fibrosis (IPF): trial did not meet its primary endpoint (annualized rate of FVC decline). Reported Oct 12, 2026.
Q4’26TRAXFirst Tracks Bio. ANB033 — Celiac Disease Phase 1
✓ ANB033 (CD122 antagonist): pOSITIVE top-line results from Cohort 1 (gluten challenge, n=30) of the Phase 1b ANB033 trial in celiac disease. (topline, Oct 12, 2026.)
Cohort 1 [gluten challenge] topline Q4’26, positive read-throughs from TEVA a-IL15 and Forte’s a-CD122 programs Jul 2026 in vitiligo. ANB033 (CD122 antagonist): pOSITIVE top-line results from Cohort 1 (gluten challenge, n=30) of the Phase…
Cohort 1 [gluten challenge] topline Q4’26, positive read-throughs from TEVA a-IL15 and Forte’s a-CD122 programs Jul 2026 in vitiligo. ANB033 (CD122 antagonist): pOSITIVE top-line results from Cohort 1 (gluten challenge, n=30) of the Phase 1b ANB033 trial in celiac disease. (topline, Oct 12, 2026.)
Q4’26VNDAVanda Pharmaceuticals Tradipitant (NEREUS) — Vomiting induced by GLP-1 RAs (The Thetis) Phase 3
✓ 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…
First patient in Apr 2026, topline Q4’26. 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…
First patient in Apr 2026, topline Q4’26. 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... Reported Oct 27, 2026.
Neladalkib (NVL-655) — TKI-pretreated adv. ALK+ NSCLC (ALKove-1) Registration
✓ Acquired by GSK ($10.6B, closed Jul 15, 2026); ticker delisted — catalyst retired as no longer applicable.
PDUFA decision expected Nov 27, 2026, Priority Review, NDA filed early Apr 2026 based on registrational phase 2 data, alignment on filing in TKI pre-treated ALK+ NSCLC prev. Reached, positive phase 2 topline Nov 2025: BICR ORR 31%; 12…
PDUFA decision expected Nov 27, 2026, Priority Review, NDA filed early Apr 2026 based on registrational phase 2 data, alignment on filing in TKI pre-treated ALK+ NSCLC prev. Reached, positive phase 2 topline Nov 2025: BICR ORR 31%; 12 months-DoR 64%; 18 months-DoR 53% in overall pop. BICR ORR 46%; 12 months-DoR 80%; 18 months-DoR 60% in TKI pre-treated but lorlatinib-naïve pop; intracranial responses seen, oral presented at ASCO’26.
Anito-cel (anitocabtagene autoleucel) — 4L+ R/R MM (iMMagine-1) Registration
✓ Acquired by Gilead ($7.8B, closed Apr 28, 2026); ticker delisted — catalyst retired as no longer applicable.
Registrational phase 2, positive data at ASH’25, PDUFA Dec 23, 2026.
YE’26CNTACentessa Pharmaceuticals Cleminorexton (ORX750) — Idiopathic hypersomnia (CRYSTAL-1) Phase 2
✓ Acquired by Eli Lilly (up to $7.8B, closed Jun 24, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected year-end 2026.
YE’26CRDFCardiff Oncology Onvansertib (NMS-1286937) + palcitaxel — 2L TNBC Phase 1/2
✓ 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). (topline, Oct 21,…
Primary completion expected year-end 2026. 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). (topline, Oct…
Primary completion expected year-end 2026. 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). (topline, Oct 21, 2026.)
YE’26MDGLMadrigal Pharmaceuticals Resmetirom (Rezdiffra) — MASH (NASH) with Compensated Cirrhosis (F4c) – (MAESTRO-NASH-OUTCOMES) Phase 3
✓ 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…
Primary completion expected year-end 2026. 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…
Primary completion expected year-end 2026. 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... Reported Oct 6, 2026.
YE’26VNDAVanda Pharmaceuticals VQW-765 — Social Performance Anxiety Phase 3
✓ 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…
Phase 3 topline readout expected 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…
Phase 3 topline readout expected 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... Reported Oct 14, 2026.
rF1V vaccine + CpG 1018 — Plague Phase 2
✓ Acquired by Sanofi ($2.2B, closed Feb 10, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected Q1’27.
H1’27RAPTRAPT Therapeutics Ozureprubart (RPT904) — Food allergy (prestIgE) Phase 2
✓ Acquired by GSK ($2.2B, closed Mar 3, 2026); ticker delisted — catalyst retired as no longer applicable.
Phase 2 data readout expected H1’27.
early’27ATXAstria Therapeutics Navenibart (STAR-0215) — Hereditary angioedema (ALPHA-ORBIT & ORBIT-EXPANSE) Phase 3
✓ Astria Therapeutics (ATXS) acquired by BioCryst ($700M, closed Jan 23, 2026); ticker delisted — catalyst retired.
ALPHA-ORBIT topline early 2027.
early’27CDTXCidara Therapeutics CD388 — Seasonal influenza (Prophylaxis, subjects with high-risk co-morbidities, immune compromised status, adults 65y+) Phase 3
✓ Acquired by Merck ($9.2B, closed Jan 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected early 2027.
Q2’27CRNXCrinetics Pharmaceuticals Atumelnant (CRN04894) — Congenital adrenal hyperplasia (adults) – (CALM-CAH) Phase 3
✓ Acquired by Vertex ($10.0B, closed Sep 1, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected Q2’27.
2027APLSApellis Pharmaceuticals APL-3007 + SYFOVRE — Geographic atrophy (GA) Phase 2
✓ Acquired by Biogen ($5.6B, closed May 2026); ticker delisted — catalyst retired as no longer applicable.
Phase 2 topline readout expected 2027.
mid’27CNTACentessa Pharmaceuticals ORX489 — Narcolepsy / sleep-wake disorders Phase 1
✓ Acquired by Eli Lilly (up to $7.8B, closed Jun 24, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected mid-2027.
mid’27DAWNDay One Biopharmaceuticals DAY101, tovorafenib (OJEMDA) — Frontline RAF-altered pLGG (FIREFLY-2) Phase 3
✓ Acquired by Servier ($2.5B, closed Apr 23, 2026); ticker delisted — catalyst retired as no longer applicable.
Last patient in H2’26→ May 2026, topline mid-2027.
H2’27APGEApogee Therapeutics Zumilokibart (APG777) — Eosinophilic Esophagitis (ELEVATE) Phase 2
✓ Acquired by AbbVie ($10.9B, closed Sep 3, 2026); ticker delisted — catalyst retired as no longer applicable.
First patient in H2’26, topline H2’27, long-term follow-up data H2’28.
H2’27APLSApellis Pharmaceuticals Pegcetacoplan — Delayed graft function (DGF) Phase 3
✓ Acquired by Biogen ($5.6B, closed May 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected H2’27.
Q3’27CNTACentessa Pharmaceuticals Cleminorexton (ORX750) — Narcolepsy type 1 & type 2, idiopathic hypersinsomnia [optional coh.] (SAPPHIRE) Phase 2/3
✓ Acquired by Eli Lilly (up to $7.8B, closed Jun 24, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected Q3’27.
Q3’27CRNXCrinetics Pharmaceuticals Paltusotine — Carcinoid syndrome (NETs) – (CAREFNDR) Phase 3
✓ Acquired by Vertex ($10.0B, closed Sep 1, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected Q3’27.
H2’27CRNXCrinetics Pharmaceuticals CRN09682 — NETs and SST2‑expressing tumors (BRAVESST2) Phase 1/2
✓ Acquired by Vertex ($10.0B, closed Sep 1, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected H2’27.
Z-1018 — Herpes zoster (shingles) Phase 1/2
✓ Acquired by Sanofi ($2.2B, closed Feb 10, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected year-end 2027.
H1’28APGEApogee Therapeutics Zumilokibart (APG777) — Atopic dermatitis (Adventure-1/-2) Phase 3
✓ Acquired by AbbVie ($10.9B, closed Sep 3, 2026); ticker delisted — catalyst retired as no longer applicable.
AtD phase 3 first patient in H2’26, readout H1’28, MD selected in phase 2b.
2028FOLDAmicus Therapeutics BMN 820 (DMX‑200) + background ARB SOC — Focal segmental glomerulosclerosis (FSGS) – (ACTION3) Phase 3
✓ Acquired by BioMarin ($4.8B, closed Apr 27, 2026); ticker delisted — catalyst retired as no longer applicable.
Phase 3 topline readout expected 2028 [proteinuria agreed with FDA as appropriate endpoint to support registration], run by Dimerix.
Anito-cel (anitocabtagene autoleucel) — 2-4L R/R MM (iMMagine-3) Phase 3
✓ Acquired by Gilead ($7.8B, closed Apr 28, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected Q3’28.
H2’28APGEApogee Therapeutics Zumilokibart (APG777) + Topical CS — Atopic dermatitis (Adventure-TCS) Phase 3
✓ Acquired by AbbVie ($10.9B, closed Sep 3, 2026); ticker delisted — catalyst retired as no longer applicable.
AtD phase 3 first patient in H2’26, readout H2’28, MD selected in phase 2b.
H2’28APGEApogee Therapeutics Zumilokibart (APG777) — Eosinophilic Esophagitis (ELEVATE) Phase 2
✓ Acquired by AbbVie ($10.9B, closed Sep 3, 2026); ticker delisted — catalyst retired as no longer applicable.
First patient in H2’26, topline H2’27, long-term follow-up data H2’28.
2029APLSApellis Pharmaceuticals Pegcetacoplan — FSGS Phase 2/3
✓ Acquired by Biogen ($5.6B, closed May 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected 2029.
Neladalkib (NVL-655) — TKI-naïve adv. ALK+ NSCLC (ALKAZAR) Phase 3
✓ Acquired by GSK ($10.6B, closed Jul 15, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected year-end 2029.
2030CRNXCrinetics Pharmaceuticals Atumelnant (CRN04894) — Congenital adrenal hyperplasia (pediatric) – (Balance-CAH) Phase 2/3
✓ Acquired by Vertex ($10.0B, closed Sep 1, 2026); ticker delisted — catalyst retired as no longer applicable.
Primary completion expected 2030.