Welcome to our dedicated page for Ionis Pharmaceuticals news (Ticker: IONS), a resource for investors and traders seeking the latest updates and insights on Ionis Pharmaceuticals stock.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) is a biotechnology company that focuses on RNA-targeted medicines for serious diseases, and its news flow reflects this emphasis on clinical, regulatory and commercial milestones. Company updates frequently highlight progress in neurology and cardiometabolic programs, as well as developments in rare conditions where few or no disease-modifying treatments exist.
Recent Ionis news has featured pivotal clinical trial readouts, such as Phase 3 CORE and CORE2 results for olezarsen in severe hypertriglyceridemia, showing large reductions in triglyceride levels and acute pancreatitis events with favorable safety and tolerability. The company has also reported positive pivotal data for zilganersen in Alexander disease, describing evidence of disease-modifying impact in this rare and often fatal neurological condition.
Regulatory and commercial milestones are another major theme. Ionis has announced FDA approval of TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome and DAWNZERA (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema in adults and adolescents 12 years and older. News items also cover FDA Breakthrough Therapy designations for olezarsen in severe hypertriglyceridemia and zilganersen in Alexander disease, as well as European regulatory opinions and anticipated launches.
Partnered program updates appear regularly, including GSK’s Phase 3 B-Well 1 and B-Well 2 results for bepirovirsen in chronic hepatitis B, Novartis’ Lp(a) HORIZON study of pelacarsen in cardiovascular disease, and AstraZeneca collaborations on WAINUA and eplontersen. Investors can also find coverage of Ionis’ financing activities, such as convertible note offerings, and its participation in healthcare and investor conferences.
For followers of IONS stock, this news page provides a centralized view of Ionis’ clinical trial milestones, FDA and EMA interactions, product launches, collaboration updates and capital markets events, helping readers understand how the company’s RNA-targeted pipeline and marketed medicines are evolving over time.
Ionis Pharmaceuticals reported strong Q1 2025 financial results, with revenue increasing 10% to $132 million compared to Q1 2024. The company's first independent product launch, TRYNGOLZA, generated over $6 million in its first quarter. Total commercial revenue grew 28% year-over-year.
The company significantly increased its 2025 financial guidance, raising revenue projections from >$600 million to $725-750 million. Operating loss guidance improved to <$375 million, with expected year-end cash position of ~$1.9 billion.
Key highlights include:
- SPINRAZA generated global sales of $424 million with $48 million in royalty revenue
- WAINUA achieved $39 million in sales with $9 million in royalties
- Donidalorsen launch expected in 2025 with PDUFA date of August 21
- Phase 3 results for olezarsen expected in Q3 2025
Ionis Pharmaceuticals revealed significant findings from a Harris Poll survey about hereditary angioedema (HAE) patient experiences. The survey, involving 150 HAE patients and 228 allergists/immunologists in the U.S., highlighted substantial treatment gaps and patient dissatisfaction.
Key findings show that 91% of HAE patients are interested in trying new prophylactic therapies, while 65% feel they haven't found their optimal treatment. Despite 85% of healthcare providers claiming to understand HAE's impact, 60% of patients wish their doctors better understood their daily challenges.
The survey exposed concerning statistics: 72% of patients make daily life tradeoffs due to unpredictable attacks, 67% live in fear of attacks, and 89% reported missing activities in the past year. Only 36% of patients use standardized tools to assess HAE control, indicating a significant communication gap between patients and healthcare providers.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced a scheduled webcast to discuss its first quarter 2025 financial results. The event will take place on Wednesday, April 30th at 11:30 a.m. Eastern Time. Investors and interested parties can access the webcast through the company's investor relations website at ir.ionis.com. A replay of the presentation will be made available for a time on the same platform.
Ionis Pharmaceuticals (NASDAQ: IONS) has announced a webinar focused on severe hypertriglyceridemia (sHTG), scheduled for Monday, April 14 at 11:00 a.m. ET. The event will feature an expert panel discussion addressing the risks, clinical presentation, and significant unmet medical needs associated with sHTG.
The panel will be led by Dr. Sam Tsimikas, senior vice president of global cardiovascular development at Ionis, and includes distinguished experts in lipidology, cardiology, and endocrinology:
- Dr. Seth Baum - Chief Medical Officer, Flourish Research
- Dr. Alan S. Brown - Director, Lipid Clinic at Advocate Lutheran General Hospital
- Dr. Savitha Subramanian - Medical Director, Lipid Clinic at University of Washington Medical Center
The webinar will be accessible through the company's investor relations website, with a replay available for a time.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced its management's upcoming participation in three major healthcare investor conferences in Spring 2025. The company will engage in fireside chats at the:
- 24th Annual Needham Virtual Healthcare Conference on April 7, 2025
- Bank of America 2025 Health Care Conference on May 14, 2025
- RBC Capital Markets Global Healthcare Conference on May 21, 2025
All presentations will be accessible via live webcast through the Investors & Media section of Ionis's website. Replay recordings will be made available within 48 hours of each presentation and will remain accessible for a time.
Ionis Pharmaceuticals (NASDAQ: IONS) has expanded its partnership with Sobi, granting exclusive rights to commercialize olezarsen outside the U.S., Canada, and China for treating familial chylomicronemia syndrome (FCS) and severely elevated triglycerides.
Key highlights:
- TRYNGOLZA™ (olezarsen) received FDA approval in December 2024 as the first treatment for FCS in the U.S.
- European Medicines Agency (EMA) review is ongoing with potential approval expected this year
- Ionis will receive an upfront payment, milestone-based payments, and tiered royalties up to mid-20% on annual net sales
- Sobi will handle regulatory submissions and commercialization in their designated territories
Three Phase 3 clinical trials (CORE, CORE2, and ESSENCE) are evaluating olezarsen for severe hypertriglyceridemia (sHTG), with data expected in mid-2025 and second half of 2025.
Ionis Pharmaceuticals (IONS) and Ono Pharmaceutical have entered into a global license agreement for sapablursen, an RNA-targeted medicine for polycythemia vera (PV). Under the agreement, Ono obtains exclusive global rights for development and commercialization of the drug.
The deal includes a $280 million upfront payment to Ionis, with potential additional payments of up to $660 million based on development, regulatory and sales milestones. Ionis will also receive mid-teen percentage royalties on annual net sales.
Sapablursen is currently in Phase 2 IMPRSSION study and has received Fast Track designation and orphan drug designation from the FDA in 2024. Ionis will complete the ongoing Phase 2 study, while Ono will handle subsequent development, regulatory filings, and commercialization.
Ionis Pharmaceuticals (IONS) announced the European Union approval of WAINZUA (eplontersen) for treating hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or 2 polyneuropathy (ATTRv-PN). The approval follows successful NEURO-TTRansform Phase 3 trial results through 66 weeks.
WAINZUA, marketed as WAINUA in the U.S., is the EU's only approved self-administered monthly auto-injector treatment for ATTRv-PN. The drug demonstrated consistent benefits in serum transthyretin concentration, neuropathy impairment, and quality of life versus placebo, while maintaining a favorable safety profile.
This marks WAINZUA's second major approval, following previous authorizations in North America and the UK. The treatment is being jointly developed and commercialized by Ionis and AstraZeneca in the U.S., with AstraZeneca holding exclusive rights for other global markets.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced its management team's upcoming participation in several investor conferences in March 2025. The schedule includes:
- Raymond James 46th Annual Institutional Investors Conference (March 4)
- TD Cowen 45th Annual Health Care Conference (March 4)
- Leerink Partners Global Healthcare Conference (March 11)
- Barclays 27th Annual Global Healthcare Conference (March 12)
- UBS Virtual CNS Day (March 17)
- Stifel Virtual CNS Forum (March 19)
Live webcasts of these presentations will be available on the Investors & Media section of the Ionis website at https://ir.ionis.com/events-and-presentations/upcoming-events. Replays will be accessible within 48 hours and archived for a time.