Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals (Nasdaq: IONS) reported strong Q2 2025 financial results, with total revenue doubling to $452 million compared to $225 million in Q2 2024. The company's TRYNGOLZA™ generated $19 million in net product sales in Q2 2025. Based on strong performance, Ionis increased its 2025 financial guidance, now expecting total revenue of $825-850 million, up from previous guidance of $725-750 million.

Key upcoming catalysts include the anticipated approval of donidalorsen for hereditary angioedema (HAE) next month and Phase 3 data from the CORE and CORE2 studies in severe hypertriglyceridemia expected in September 2025. The company's partnered medicine WAINUA™ generated $44 million in Q2 sales, while SPINRAZA® contributed $54 million in royalty revenue.

Ionis Pharmaceuticals (Nasdaq: IONS) and Sobi® announced that the European Medicines Agency's CHMP has recommended approval for TRYNGOLZA® (olezarsen) to treat familial chylomicronemia syndrome (FCS) in the EU. The decision follows successful Phase 3 Balance study results showing significant triglyceride reductions and fewer acute pancreatitis events.

The treatment demonstrated sustained triglyceride reduction over 12 months with favorable safety. FCS, affecting up to 13 people per million in the EU, is a rare genetic condition causing severe hypertriglyceridemia with triglyceride levels often exceeding 880 mg/dL. The European Commission's final approval decision is expected by Q4 2025.

Sobi will handle commercialization outside the U.S., Canada, and China, leveraging their existing market expertise from Waylivra. TRYNGOLZA was previously approved in the U.S. in December 2024.

Ionis Pharmaceuticals (Nasdaq: IONS) announced positive Phase 3 OASISplus study results for donidalorsen in hereditary angioedema (HAE) patients, published in JACI In Practice. The study demonstrated that patients switching from other prophylactic treatments to donidalorsen experienced a 62% further reduction in mean monthly HAE attack rate from baseline.

Key findings include 84% of patients preferring donidalorsen over prior treatments, with 93% reporting well-controlled disease compared to 67% at baseline. The drug showed positive results across different prior treatments, with attack rate reductions of 65%, 41%, and 73% for patients switching from lanadelumab, C1INH, and berotralstat, respectively.

Donidalorsen is currently under FDA review with a PDUFA date of August 21, 2025, positioning it as a potential first-in-class prophylactic treatment for HAE in patients 12 years and older.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced it will host a webcast to discuss its second quarter 2025 financial results and provide updates on key programs. The event is scheduled for Wednesday, July 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 limited time following the event.

Ionis Pharmaceuticals (NASDAQ:IONS) announced positive interim Phase 1 results for salanersen (ION306/BIIB115), their novel antisense oligonucleotide for treating spinal muscular atrophy (SMA). The drug, licensed to Biogen, showed promising results with once-yearly dosing potential.

Key findings include 70% reduction in neurofilament light chain at 6 months, sustained through one year. In a subgroup of 8 participants aged 2-12, half achieved new WHO motor milestones, with mean improvements of 3.3 points in HFMSE and 5.3 points in RULM. Both tested doses (40mg and 80mg) were generally well-tolerated.

Based on these positive results, Biogen is engaging with global regulators to advance salanersen to Phase 3 studies.

Ionis Pharmaceuticals announced that Dr. Richard Geary, Executive VP and Chief Development Officer, will retire in January 2026 after a 30-year tenure. Dr. Holly Kordasiewicz, currently SVP of Neurology, will succeed him. During his tenure, Dr. Geary led numerous development programs resulting in six FDA and EU approvals, including TRYNGOLZA. Dr. Kordasiewicz, with 20 years of R&D experience, currently oversees Ionis' neurology program, which includes eight wholly owned medicines in clinical development. She played a key role in developing QALSODY for ALS and IONIS-MAPTRx for Alzheimer's disease. Dr. Geary will continue as a strategic consultant throughout 2026 to ensure a smooth transition.

Ionis Pharmaceuticals has initiated its Phase 3 REVEAL clinical trial for ION582, targeting Angelman syndrome (AS), a rare neurodevelopmental disorder. The global study will evaluate approximately 200 children and adults with AS having UBE3A gene deletion or mutation. Participants will be randomized 2:1 to receive either ION582 (40mg or 80mg quarterly doses) or placebo over 52 weeks, followed by a 2-year extension where all participants receive ION582. The primary endpoint focuses on expressive communication improvement using Bayley-4 assessment. The study builds on positive Phase 1/2 HALOS results, which showed consistent clinical improvements across functional domains. Study completion is expected in 2026.

Ionis Pharmaceuticals (NASDAQ: IONS) reported positive topline results from the Essence study of olezarsen in patients with moderate hypertriglyceridemia. The drug achieved its primary endpoint with significant triglyceride reductions of 61% and 58% versus placebo at 80mg and 50mg monthly doses respectively (p<0.0001). The study included nearly 1,500 participants, most on standard lipid-lowering medicines. Olezarsen met all key secondary endpoints, with most participants reaching normal triglyceride levels (<150mg/dL). The drug showed a favorable safety profile, with mild injection site reactions as the most common side effect. Following FDA approval of TRYNGOLZA for FCS, Ionis expects Phase 3 CORE and CORE2 study results for severe hypertriglyceridemia (sHTG) in Q3 2025, potentially leading to an sNDA filing by year-end.

Ionis Pharmaceuticals (NASDAQ: IONS) has announced its upcoming 2025 virtual Annual Meeting of Stockholders, scheduled for Thursday, June 5, 2025. The event will consist of two parts: the Annual Meeting from 5:00-5:15 PM ET, followed by a corporate update at 5:30 PM ET. Stockholders of record as of April 7, 2025 can participate in the virtual meeting through www.virtualshareholdermeeting.com/IONS2025 using their 16-digit control number from their brokerage firm's proxy card. CEO Brett P. Monia will deliver a corporate update presentation with a Q&A session, accessible to all interested parties via webcast. While the stockholder meeting won't be recorded, the corporate update will have an archived replay available for a limited time.