IONS (Ionis Pharmaceuticals Inc.) stock: $44.44, $7.39B market cap, $2.05B cash, $7.14B EV, $9.38B fully diluted market cap. Next expected readout: Dec-26. Lead program: Donidalorsen (Hereditary Angioedema), Phase 3. Data from Fully Diluted's biotech stocks database.
Pipeline assets
TRYNGOLZA
Approved · Active Modality: antisense oligonucleotide (LICA)
Aliases Olezarsen
Target apoC-III
Indications- Severe hypertriglyceridemia (sHTG) (Approved)
- Familial chylomicronemia syndrome (FCS) (Approved)
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.
DAWNZERA
Approved · Active Modality: antisense oligonucleotide
Aliases Donidalorsen
Target PKK
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.
SPINRAZA
Approved · Active Modality: antisense oligonucleotide
Aliases Nusinersen
Target SMN2
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).
WAINUA
Approved · Active Modality: antisense oligonucleotide (LICA)
Aliases Eplontersen
Target TTR
Partnerships AstraZeneca: joint development and commercialization for ATTR (shared development, commercialization, and medical affairs)
Indications- Polyneuropathy of hereditary transthyretin-mediated amyloidosis (Approved)
- Transthyretin amyloidosis cardiomyopathy (ATTR-CM) (Phase 3)
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).
QALSODY
Approved · Active Modality: antisense oligonucleotide
Aliases Tofersen
Target SOD1
Partnerships Biogen: development/commercialization; Ionis earns royalties on net sales
Indications- SOD1 amyotrophic lateral sclerosis (SOD1-ALS) (Approved)
- Presymptomatic SOD1-ALS (Stage not disclosed)
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).
TEGSEDI
Approved · Active Modality: antisense oligonucleotide
Aliases Inotersen
Target TTR
Partnerships Swedish Orphan Biovitrum AB (Sobi): distribution agreement; Ionis supplies finished goods and earns commercial revenue
Indications- ATTRv polyneuropathy (ATTRv-PN) (Approved)
Status note Once-weekly subcutaneous medicine approved in Europe and Brazil for ATTRv-PN. Sold in Europe through the Sobi distribution agreement.
WAYLIVRA
Approved · Active Modality: antisense oligonucleotide
Aliases Volanesorsen
Target apoC-III
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.
Zilganersen
Phase 3 · Active Modality: antisense oligonucleotide
Target GFAP
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)
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.
Obudanersen
Phase 3 · Active Modality: antisense oligonucleotide
Aliases ION582
Target UBE3A
Indications- AS (rare genetic neurological disorder) (Phase 3)
Status note RNA-targeted medicine. Phase 3 REVEAL study enrollment completed July 2026; open-label Phase 1/2 HALOS study continuing.
Bepirovirsen
Phase 3 · Active Modality: antisense oligonucleotide
Target HBV
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)
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.
Pelacarsen
Phase 3 · Active Modality: antisense oligonucleotide
Target Apo(a)
Partnerships Novartis: developing pelacarsen, including conducting the Phase 3 Lp(a) HORIZON cardiovascular outcome study
Indications- Lipoprotein(a)-driven cardiovascular disease (Phase 3)
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.
Salanersen
Phase 3 · Active Modality: antisense oligonucleotide
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.
Sapablursen
Phase 3 · Active Modality: antisense oligonucleotide
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.
Sefaxersen
Phase 3 · Active Modality: antisense oligonucleotide
Target FB
Partnerships Hoffmann-La Roche / F. Hoffmann-La Roche Ltd: developing sefaxersen for IgAN
Indications- IgA nephropathy (IgAN) (Phase 3)
Status note Medicine in development for IgAN. Roche advanced sefaxersen into Phase 3 in Q2 2023.
Ulefnersen
Phase 3 · Active Modality: antisense oligonucleotide
Target FUS
Indications- FUS amyotrophic lateral sclerosis (FUS-ALS) (Phase 3)
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.