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 (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.
Ionis Pharmaceuticals (NASDAQ: IONS) announced new data presentations for donidalorsen, their investigational RNA-targeted medicine for hereditary angioedema (HAE), at the 2025 AAAAI/WAO Joint Congress. The drug's New Drug Application is under FDA review with a PDUFA date of August 21, 2025.
The presentations will showcase additional data from the Phase 3 OASIS and OASISplus studies, along with three-year data from the Phase 2 open-label extension study. The company will present 11 posters covering various aspects including efficacy in adolescent patients, disease control after switching from prior treatments, quality-of-life improvements, and patient-reported outcomes.
The data demonstrates sustained HAE attack rate reduction and improved quality of life in patients previously on other prophylactic treatments, with the convenience of monthly or bi-monthly self-administration via autoinjector. If approved, donidalorsen would be Ionis' second independent commercial launch.
Ionis Pharmaceuticals (NASDAQ: IONS) reported its Q4 and full year 2024 financial results, marking significant milestones with multiple product launches. The company's total revenue for 2024 was $705 million, down from $788 million in 2023, with operating expenses of $1,180 million.
Key highlights include the launch of TRYNGOLZA for familial chylomicronemia syndrome and WAINUA for hereditary transthyretin-mediated amyloidosis, which generated $85 million in sales with $20 million in royalty revenue. SPINRAZA generated global sales of $1.6 billion, resulting in $216 million in royalty revenue.
The company maintains a strong financial position with $2.3 billion in cash and investments as of December 31, 2024. For 2025, Ionis projects revenue exceeding $600 million and expects to maintain cash reserves of approximately $1.7 billion.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced a webcast scheduled for Wednesday, February 19th at 11:30 a.m. Eastern Time. During this event, the company will discuss its fourth quarter and full year 2024 financial results. The webcast will be accessible through the company's investor relations website, and a replay will be available for a time afterward.
Ionis Pharmaceuticals (IONS) enters 2025 with significant momentum following the U.S. approval and launch of TRYNGOLZA™, the first-ever therapy for familial chylomicronemia syndrome (FCS). The company anticipates four independent product launches over the next three years, plus four additional launches from partnered programs. Key 2025 milestones include potential European approval for TRYNGOLZA, expected FDA decision on donidalorsen for hereditary angioedema by August 21, and continued progress with WAINUA™ in collaboration with AstraZeneca.
The company plans to advance seven clinical-stage therapies for neurological diseases, including Phase 3 trials for ION582 in Angelman syndrome and data readouts for zilganersen in Alexander disease. Ionis expects increasing product and royalty revenue to drive positive cash flow, marking its transition to a fully integrated commercial-stage biotechnology company.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced that CEO Brett P. Monia, Ph.D., will deliver a company overview presentation at the 43rd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled for Wednesday, January 15, 2025, at 10:30 a.m. PT.
Investors and interested parties can access a live webcast of the presentation through the Investors & Media section of the Ionis website. The presentation recording will be made available on the company's website within 48 hours and will remain accessible for a time.
Ionis Pharmaceuticals (NASDAQ: IONS) announced FDA approval of TRYNGOLZA™ (olezarsen), the first-ever treatment for adults with familial chylomicronemia syndrome (FCS). The drug, administered monthly via auto-injector, demonstrated a 42.5% placebo-adjusted mean reduction in triglyceride levels at six months and 57% at 12 months in Phase 3 Balance trial. TRYNGOLZA significantly reduced acute pancreatitis events, with only one episode in the treatment group versus 11 episodes in the placebo group.
The treatment showed a favorable safety profile and will be available in the U.S. before year end. This approval marks Ionis's evolution into a commercial-stage biotechnology company, with TRYNGOLZA being the first of four planned launches over the next three years.
Ionis Pharmaceuticals (NASDAQ: IONS) has announced its participation in six major healthcare investor conferences throughout November and December 2024. The company's management will engage in fireside chats at events including Guggenheim's Inaugural Healthcare Conference, UBS Global Healthcare Conference, Stifel Healthcare Conference, Jefferies London Healthcare Conference, Citi's Global Healthcare Conference, and Piper Sandler Annual Healthcare Conference. Live webcasts will be available on the Ionis website's Investors & Media section, with replays accessible within 48 hours and archived for a time.
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