Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis Pharmaceuticals (Nasdaq: IONS) announced positive topline results from the pivotal study of zilganersen in treating Alexander disease (AxD), a rare and often fatal neurological condition. The drug demonstrated statistically significant improvement in the primary endpoint of gait speed, showing a 33.3% mean difference (p=0.0412) compared to control at week 61.

The study showed consistent benefits across key secondary endpoints, including improvements in patient-reported outcomes and clinical assessments. Zilganersen demonstrated a favorable safety profile, with most adverse events being mild to moderate. The company plans to submit a New Drug Application (NDA) in Q1 2026 and is considering an Expanded Access Program in the U.S.

Ionis Pharmaceuticals (Nasdaq: IONS) and Sobi® have received European Union approval for TRYNGOLZA® (olezarsen) to treat familial chylomicronemia syndrome (FCS). The approval is based on the successful Phase 3 Balance study results, which demonstrated significant reduction in fasting triglyceride levels and acute pancreatitis events over 12 months.

FCS is a rare genetic condition affecting up to 13 people per million in the EU, characterized by severely high triglyceride levels exceeding 880 mg/dL. TRYNGOLZA will be commercialized by Sobi in regions outside the U.S., Canada, and China. The drug is also being evaluated for severe hypertriglyceridemia (sHTG), with positive Phase 3 results announced in September 2025.

Ionis Pharmaceuticals (Nasdaq: IONS) celebrates the 10th anniversary of its Surf Away+ adaptive event, expanding its reach to include multiple neurological disease communities. The event, originally focused on providing adaptive surfing for people with spinal muscular atrophy (SMA), now welcomes individuals from Alexander disease (AxD), Amyotrophic Lateral Sclerosis (ALS), and Angelman syndrome (AS) communities.

The 2025 event in Oceanside, CA, expects over 200 attendees and features diverse activities including adaptive surfing, sitting volleyball, wheelchair pickleball, and sensory-friendly experiences. The program, supported by 100+ Ionis volunteers, partners with the Challenged Athletes Foundation (CAF) and ADAPT Functional Movement Center to provide comprehensive support and resources for participants and caregivers.

Ionis Pharmaceuticals (Nasdaq: IONS) has received FDA Breakthrough Therapy designation for ION582, its investigational treatment for Angelman syndrome, a rare neurological disease characterized by severe intellectual disability and seizures. The designation was granted based on promising results from the Phase 1/2 HALOS study, which demonstrated clinical improvements across multiple functional domains.

The company is currently conducting the Phase 3 REVEAL study, expected to complete enrollment in 2026. The study will evaluate ION582 in children and adults with Angelman syndrome who have maternal UBE3A gene deletion or mutation. This breakthrough designation could expedite the review process for what could be the first disease-modifying treatment for Angelman syndrome patients.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced its upcoming Innovation Day event scheduled for October 7, 2025, in New York. The event will feature presentations from the company's leadership team and include special guest Dr. Robert D. Fishberg, a distinguished expert in severe hypertriglyceridemia, familial chylomicronemia syndrome, and lipid disorders.

The event will run from 8:30 AM to 12:30 PM ET and will be accessible via webcast. Interested parties can register online, and a replay will be available for a limited time on Ionis' website investor events section.

Ionis Pharmaceuticals (Nasdaq: IONS) has announced its participation in three upcoming investor conferences in September 2025. The company's management will engage in fireside chats at the 2025 Wells Fargo Healthcare Conference (September 4), the H.C. Wainwright 27th Annual Global Investment Conference (September 8), and the Morgan Stanley 23rd Annual Global Healthcare Conference (September 9).

Investors can access live webcasts of these presentations through the Investors & Media section of Ionis's website. Replay recordings will be available within 48 hours and archived for a limited time.

Ionis Pharmaceuticals (NASDAQ:IONS) announced breakthrough results from Phase 3 CORE and CORE2 studies of olezarsen in treating severe hypertriglyceridemia (sHTG). The drug demonstrated a 72% placebo-adjusted reduction in fasting triglycerides and an unprecedented 85% reduction in acute pancreatitis events (p=0.0002).

The largest pivotal program for sHTG, involving nearly 1,100 patients, showed significant efficacy at both 80mg and 50mg monthly doses. The drug maintained a favorable safety profile, with over 90% of patients choosing to continue in the open-label extension study. Ionis plans to submit a supplemental new drug application (sNDA) by year-end.

Additionally, Phase 3 Essence study results for moderate hypertriglyceridemia were published in The New England Journal of Medicine, demonstrating significant triglyceride reduction and meeting all key secondary endpoints.

Ionis Pharmaceuticals (Nasdaq: IONS) has received FDA approval for DAWNZERA™ (donidalorsen), the first RNA-targeted prophylactic treatment for hereditary angioedema (HAE) in patients 12 years and older. The drug demonstrated an 81% reduction in monthly HAE attack rates compared to placebo in Phase 3 trials, increasing to 87% from the second dose.

DAWNZERA offers the longest dosing interval available, administered via subcutaneous autoinjector every 4 or 8 weeks. The treatment showed a 94% total mean attack rate reduction after one year in extension studies. Notably, 84% of patients preferred DAWNZERA over prior treatments, citing better disease control and improved administration experience.

This marks Ionis' second independent product launch in nine months, with DAWNZERA becoming commercially available in the coming days, supported by the Ionis Every Step™ patient support program.

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.