We currently have seven marketed medicines to treat serious diseases: TRYNGOLZA (olezarsen), DAWNZERA (donidalorsen), WAINUA (eplontersen), SPINRAZA (nusinersen), QALSODY (tofersen), TEGSEDI (inotersen) and WAYLIVRA (volanesorsen). Marketed Medicines ● TRYNGOLZA is approved in the United States, or U.S., as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome, or FCS, and is approved in the European Union, or EU, for patients with genetically confirmed FCS.
Cash figures as of 2026-06-30. Database snapshot: 2026-11-07.
Share structure
Common
166.2M
Pre-funded warrants
0.0M
Options
0.0M
RSUs
0.0M
Warrants
0.0M
Converts
2.0M
Conv preferred
0.0M
ESPP reserve
—
FDSO
168.2M
Cap table as of 2026-06-30.
Price history
Loading IONS daily closes…
Loading price history…
Next catalyst
Donidalorsen · Jan 12 - Feb 23 in 9 wks · Phase 3 · Hereditary Angioedema
Recently reported
Olezarsen Phase 3Positive · 2026-10-16
CORE · CORE2 · severe hypertriglyceridemia
Primary: Placebo-adjusted mean reduction in fasting triglycerides in the Phase 3 CORE (NCT05079919) and CORE2 (NCT05552326) studies in severe hypertriglyceridemia — Met the primary endpoint, with a placebo-adjusted mean reduction in fasting triglycerides of up to 72% at six months, sustained through 12 months
· CORE n=617 and CORE2 n=446, randomized to 50 mg or 80 mg olezarsen or placebo every four weeks for 12 months
· 85% reduction in adjudicated acute pancreatitis events over 12 months in a pooled analysis
· At baseline, fasting triglycerides >=880 mg/dL in 47% of CORE participants and 37% of CORE2 participants
· CHMP positive opinion for an indication extension in adults with fasting triglycerides >=880 mg/dL (>=10 mmol/L)
· European Commission decision expected in Q4 2026
Safety: Favorable safety and tolerability profile stated; most common adverse reactions in sHTG were injection site reactions and liver enzyme increases
Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Status note Once-monthly self-administered LICA medicine approved in the U.S. to reduce triglycerides and the risk of acute pancreatitis in adults with sHTG and in FCS. FDA granted priority review of olezarsen for sHTG. Wholly owned; positioned for independent launch.
Partnerships Otsuka Pharmaceutical Co., Ltd.: commercialization of DAWNZERA in Europe (2023) and Asia-Pacific (2024)
Indications
Hereditary angioedema (HAE)(Approved)
Status note RNA-targeted medicine approved in the U.S. for prophylaxis to prevent HAE attacks in adults and pediatric patients 12+; also approved in the EU and UK for routine prevention of recurrent HAE attacks. European Commission approved DAWNZERA in the EU in Q1 2026.
Partnerships Biogen: commercialization; Ionis earns royalties on net sales
Indications
Spinal muscular atrophy (SMA)(Approved)
Status note Antisense medicine for SMA. Higher-dose SPINRAZA approved and launched in the U.S. and EU. Ionis earns royalty payments on net sales (portion of SPINRAZA royalties monetized via Royalty Pharma agreement).
Status note Once-monthly subcutaneous LICA medicine approved in numerous countries including the U.S., EU, UK, Canada, and China for polyneuropathy of hereditary transthyretin-mediated amyloidosis. In July 2026, the CARDIO-TTRansform trial in ATTR-CM missed its primary efficacy endpoint (announced with AstraZeneca).
Status note Received FDA accelerated approval in April 2023 and EMA marketing authorization under exceptional circumstances in May 2024 for adult patients with SOD1-ALS. Biogen is evaluating tofersen for presymptomatic SOD1-ALS (stage not stated).
Partnerships Swedish Orphan Biovitrum AB (Sobi): distribution agreement; Ionis supplies finished goods and earns commercial revenue
Indications
Familial chylomicronemia syndrome (FCS) at high risk for pancreatitis(Approved)
Status note Once-weekly subcutaneous medicine approved in Europe and Brazil as an adjunct to diet in adult patients with genetically confirmed FCS at high risk for pancreatitis. Sold in Europe through the Sobi distribution agreement.
Partnerships Recordati AG: commercialization of zilganersen for AxD in countries outside the U.S. (agreement June 2026; up to $75M in upfront + milestone payments)
Indications
Alexander disease (AxD)(Phase 3)
Catalysts
— September 22, 2026
Status note Investigational medicine for AxD. Regulatory submission based on the Phase 3 portion of a clinical study; FDA granted Priority Review with PDUFA action date September 22, 2026.
Partnerships GSK: GSK is developing bepirovirsen under the 2010 collaboration; CHB collaboration agreement amended Q2 2026 with increased milestones
Indications
Chronic hepatitis B (CHB)(Phase 3)
Catalysts
— October 26, 2026
Status note Medicine in development for CHB. Japanese MHLW accepted an NDA for review in Q1 2026; FDA granted Priority Review with PDUFA action date October 26, 2026.
Status note Medicine in development for patients with elevated lipoprotein(a)-driven CVD. Ongoing Phase 3 Lp(a) HORIZON cardiovascular outcome study conducted by Novartis.
Partnerships Biogen: licensed salanersen under the 2017 SMA collaboration (option exercised 2021); Biogen initiated Phase 3 in Q2 2026
Indications
Spinal muscular atrophy (SMA)(Phase 3)
Status note Medicine in development for SMA. Biogen advanced salanersen into Phase 3 in Q2 2026 (triggering a $45M milestone); FDA granted Breakthrough Therapy designation.
Partnerships Ono: advanced sapablursen into a pivotal clinical trial in Q2 2026 (triggering a $20M milestone)
Indications
Polycythemia vera (PV)(Phase 3)
Status note Medicine in development for PV. Advanced into Phase 3 in Q2 2026; received FDA Fast Track, Orphan Drug, and Breakthrough Therapy designations for PV.
Status note RNA-targeted medicine for FUS-ALS. Phase 3 FUSION study (initiated April 2021) ongoing in juvenile and adult patients with FUS-ALS; global commercialization rights licensed out (partner not named in excerpts).
Per-asset detail extracted from the 10-Q filed 2026-08-04. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (20 as sponsor, 2 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.