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 leader pioneering RNA-targeted therapies through its antisense technology platform. This news hub provides investors and industry professionals with comprehensive updates on the company's clinical developments, strategic partnerships, and regulatory milestones.
Access real-time updates on Ionis' innovative research in neurology, cardiology, and rare diseases, including progress on therapies like Qalsody and Wainua. The page aggregates essential information including earnings reports, collaboration announcements with partners such as Biogen and AstraZeneca, and clinical trial outcomes.
Our curated collection serves as your primary source for tracking Ionis' advancements in antisense oligonucleotide development and commercialization strategies. Bookmark this page for streamlined access to verified updates about licensing agreements, patent developments, and therapeutic pipeline expansions.
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.
Ionis Pharmaceuticals announced the design of its pivotal Phase 3 REVEAL trial for ION582, following successful alignment with the FDA. The trial, targeting Angelman syndrome (AS), will enroll approximately 200 children and adults with maternal UBE3A gene deletion or mutation. The study will use Bayley-4 expressive communication as the primary endpoint, with patients randomized 2:1 to active therapy or placebo. The Phase 2 HALOS study showed promising results, with 97% of participants in medium and high dose groups showing improvement in overall AS symptoms. The Phase 3 trial is planned to begin in H1 2025.