Ionis Pharmaceuticals Stock Price, News & Analysis

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.

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.