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 (Nasdaq: IONS) said management will appear at six investor conferences in late February and March 2026, offering fireside chats and webcasts.
Dates include Feb 26, Mar 2, Mar 9, Mar 11 and Mar 17, 2026, with live webcasts and replays available on the company's investor website and archived for a limited time.
Ionis (NASDAQ: IONS) changed the start time for its fourth quarter and full year 2025 financial results webcast to 8:30 a.m. Eastern Time on Wednesday, February 25, 2026. The date of the call is unchanged and a replay will be available for a limited time at the company's investor events page.
Ionis (Nasdaq: IONS) will host a live webcast on Wednesday, February 25, 2026 at 11:30 a.m. Eastern Time to discuss fourth quarter and full year 2025 financial results and to highlight progress on key programs. A replay will be available for a limited time on the investor events page.
Ionis (Nasdaq: IONS) and Otsuka announced that the European Commission approved DAWNZERA (donidalorsen) for routine prevention of hereditary angioedema (HAE) in adults and adolescents 12+ in the EU.
Approval follows positive Phase 3 OASIS-HAE and OASISplus data, including a 94% overall mean monthly attack rate reduction at one year in the OASISplus open-label extension. DAWNZERA is self-administered by subcutaneous autoinjector every four or eight weeks. Ionis is eligible for a $15 million milestone payment and tiered royalties up to 30% on net sales; Otsuka holds exclusive rights in Europe and Asia Pacific.
Ionis (Nasdaq: IONS) reported 2025 momentum and set 2026 catalysts including two planned independent launches and multiple Phase 3 readouts. TRYNGOLZA (olezarsen) generated preliminary U.S. net product sales of $105M in 2025 and a U.S. sNDA for severe hypertriglyceridemia was submitted; peak annual TRYNGOLZA net sales guidance was raised to >$2 billion. Positive pivotal Phase 3 topline results were announced for bepirovirsen in chronic hepatitis B with planned global regulatory submissions in 2026. Ionis expects a total of five Phase 3 readouts and four NDA/sNDA submissions in 2026, plus launches for DAWNZERA, olezarsen (sHTG) and zilganersen (Alexander disease), and targets cash flow breakeven in 2028.
Ionis (Nasdaq: IONS) partner GSK announced positive topline Phase 3 results for bepirovirsen from the B-Well 1 and B-Well 2 trials. The studies enrolled >1,800 patients across 29 countries and met the primary endpoint, with bepirovirsen plus standard of care showing a statistically significant and clinically meaningful functional cure rate versus standard of care alone.
The announcement notes an acceptable safety profile consistent with prior studies, planned global regulatory filings beginning Q1 2026, and existing Fast Track/Breakthrough/SENKU designations. Under the 2019 license, Ionis is eligible for $150M in remaining milestones and 10–12% royalties on net sales.
Ionis (NASDAQ: IONS) will present a company overview at the 44th Annual J.P. Morgan Healthcare Conference. CEO Brett P. Monia, Ph.D., will speak on January 13, 2026 at 8:15 a.m. PT. A live webcast will be available on the company Investors & Media website at https://ir.ionis.com/events, with a replay posted within 48 hours and archived for a limited time.
Ionis (Nasdaq: IONS) announced that the U.S. FDA granted Breakthrough Therapy designation to zilganersen for Alexander disease (AxD), a rare, progressive neurological disorder with no approved disease-modifying treatments.
The FDA decision is supported by pivotal study topline results: zilganersen 50 mg improved 10-Meter Walk Test gait speed by 33.3% versus control (p=0.0412) at week 61, with favorable safety and consistent secondary endpoint benefits. Ionis plans an NDA submission in Q1 2026 and is working to start a U.S. Expanded Access Program.
Ionis said this designation may expedite review and follows a recent Breakthrough Therapy designation for another program, underscoring the company’s late-stage pipeline focus.
Ionis (Nasdaq: IONS) announced that the U.S. FDA granted Breakthrough Therapy designation to olezarsen for use as an adjunct to diet to reduce triglycerides in adults with severe hypertriglyceridemia (sHTG; TG ≥500 mg/dL) on December 1, 2025.
The designation is based on Phase 3 CORE and CORE2 results showing up to 72% placebo‑adjusted triglyceride reduction, an 85% reduction in acute pancreatitis events, and nearly 90% of treated patients achieving TG <500 mg/dL. Data were published in The New England Journal of Medicine and presented at AHA. Ionis said it is on track to submit a supplemental new drug application to the FDA by the end of the year.
Ionis Pharmaceuticals (Nasdaq: IONS) and Otsuka announced the CHMP has adopted a positive opinion for DAWNZERA (donidalorsen) for routine prevention of hereditary angioedema (HAE) in adults and adolescents aged 12 and older.
The opinion is now with the European Commission, with a decision expected in Q1 2026. CHMP based its recommendation on Phase 3 OASIS-HAE and OASISplus results showing a significant, sustained reduction in mean monthly HAE attack rate, including with autoinjector self-administration. DAWNZERA received FDA approval in August 2025.