Ionis Pharmaceuticals, Inc. develops RNA-targeted medicines and reports news around marketed medicines and a pipeline in neurology, cardiometabolic disease and other high-need areas. Company updates commonly address commercial performance for TRYNGOLZA, financial results, R&D revenue from partnerships, and progress across antisense oligonucleotide programs.
Clinical and regulatory news also covers programs such as olezarsen for severe hypertriglyceridemia, zilganersen for Alexander disease, and partner-developed bepirovirsen for chronic hepatitis B. Governance and investor-relations releases include annual meeting materials, board composition changes, and healthcare conference presentations.
Ionis Pharmaceuticals (Nasdaq: IONS) has dosed the first participant in ASCEND, an open-label Phase 1-2 study of ION337, an investigational RNA-targeted medicine for Dravet syndrome, a rare, severe childhood-onset neurological disorder. ASCEND will evaluate safety and tolerability in children aged 2–12 years with a clinical diagnosis of Dravet syndrome.
The study includes a 6‑month single ascending dose part, followed by a 24‑month multiple ascending dose part with intrathecal dosing of ION337 every 6 months, plus a 7‑month safety follow‑up. ION337 is Ionis’ first wholly owned medicine developed with its next‑generation N‑Methylacetamide (NMA) antisense technology, designed to enhance potency and support infrequent dosing.
Ionis (Nasdaq: IONS) and AstraZeneca reported that the Phase 3 CARDIO-TTRansform trial of eplontersen in transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its primary endpoint of CV mortality and recurrent CV events versus placebo.
A prespecified eplontersen monotherapy subgroup showed a nominally significant hazard ratio of 0.71, while no effect was seen in patients on stabilizers at baseline. Eplontersen achieved large, sustained TTR reductions and a favorable safety profile. Full data are planned for ESC Congress August 2026.
Ionis (Nasdaq: IONS) completed enrollment of the pivotal pediatric cohort in the global Phase 3 REVEAL study of obudanersen for Angelman syndrome. Cohort 1 enrolled 136 participants aged 2 to <18 years. The adult cohort is expected to finish enrollment in Q3 2026, with topline data anticipated in 2H 2027. Ionis also plans a Phase 3 CHAMPION study for UPD and imprinting defect genotypes before end of 2026.
Ionis (Nasdaq: IONS) signed a license deal granting Recordati exclusive rights to develop and commercialize zilganersen for Alexander disease in all countries outside the U.S. Ionis keeps U.S. rights and global development leadership.
Ionis receives a $30 million upfront payment, is eligible for milestones, and a tiered royalty of up to the mid‑20% range on annual net sales.
Ionis (Nasdaq: IONS) received FDA approval for TRYNGOLZA (olezarsen) as an adjunct to diet to reduce triglycerides and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG, TG ≥500 mg/dL). TRYNGOLZA, also indicated for familial chylomicronemia syndrome, is a once-monthly 50 mg or 80 mg self-administered autoinjector.
Phase 3 CORE and CORE2 data showed fasting triglyceride reductions up to 72% vs placebo at six months, sustained at 12 months, and acute pancreatitis events reduced by up to 91%. TRYNGOLZA will be available in the U.S. for sHTG in July.
Ionis (Nasdaq: IONS) appointed Ludwig Hantson, Ph.D., a veteran biopharmaceutical executive, to its Board of Directors, effective immediately. He previously served as CEO of Alexion and Baxalta and held senior roles at Baxter Bioscience, Novartis and Johnson & Johnson.
Ionis highlights his experience leading complex global organizations in rare and specialty diseases as it prepares for multiple new commercial launches and continued scaling as a fully integrated biotechnology company. Ionis also recently added Peter Reikes to the board and announced the retirement of directors Joseph Wender and Lynne Parshall.
Ionis (Nasdaq: IONS) will present new analyses from the Phase 3 CORE and CORE2 studies of olezarsen in severe hypertriglyceridemia (sHTG) at the 2026 ADA, NLA and ENDO congresses.
According to Ionis, prior CORE/CORE2 results showed highly statistically significant reductions in fasting triglycerides and acute pancreatitis events, with favorable safety and tolerability. Olezarsen’s U.S. FDA Priority Review has a target action date of June 30, 2026.
Ionis (Nasdaq: IONS) reported that partner GSK announced positive pivotal Phase 3 data for bepirovirsen in chronic hepatitis B. Six-month treatment achieved a 19% functional cure rate overall and 26% in patients with lower baseline HBsAg (≤1000 IU/mL), versus 0% with standard of care.
Bepirovirsen is under FDA priority review with Breakthrough and Fast Track status, and is also under review in Europe, Japan and China. Ionis is eligible for additional regulatory and sales milestones plus 10–12% tiered royalties on net sales.
Ionis (Nasdaq: IONS) announced that partner Biogen reported topline Phase 2 CELIA results for diranersen (BIIB080) in early Alzheimer’s disease. CELIA did not meet its primary dose-response endpoint on CDR-SB at Week 76, but showed tau biomarker reductions and cognitive effects across all studied doses.
Prespecified analyses indicated slowed clinical decline, especially at 60 mg every 24 weeks. Tau reductions in CSF and PET were maintained over the dosing period. Safety and tolerability were generally in line with the Phase 1b study, though serious adverse events were more frequent at the highest dose. Biogen plans to advance diranersen to registrational development.
Ionis (Nasdaq: IONS) will hold a virtual Annual Meeting of Stockholders and a corporate update on Thursday, June 4, 2026. The Annual Meeting is 5:00–5:15 p.m. ET and the corporate update at 5:30 p.m. ET, presented by CEO Brett P. Monia, Ph.D.
Stockholders of record at close of business on April 7, 2026 may join the Annual Meeting webcast at www.virtualshareholdermeeting.com/IONS2026. The corporate update webcast is available live and will have a limited-time archived replay at the event webcasts URL.