Ionis Pharmaceuticals Stock Price, News & Analysis

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.

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.