Ionis Pharmaceuticals Stock Price, News & Analysis

Ionis (Nasdaq: IONS) will present detailed Phase 3 results for olezarsen in severe hypertriglyceridemia (sHTG) as a late-breaking abstract at AHA Scientific Sessions on November 8, 2025. The CORE and CORE2 pivotal studies showed a highly statistically significant reduction in fasting triglycerides and fewer acute pancreatitis events, with reported favorable safety and tolerability. Ionis plans to submit a supplemental NDA to the U.S. FDA by the end of the year. The presentation (CORE-TIMI 72a and CORE2-TIMI 72b) is scheduled for 9:40–9:50 a.m. ET and a company webcast to discuss results will be held on November 8 at 3:00 p.m. ET, with a limited-time replay available.

Ionis (Nasdaq: IONS) was ranked #2 in Science magazine’s 2025 Top Employer survey, marking the company’s first year of eligibility for the list.

The ranking cited industry-leading innovation, alignment of company and employee values, employee loyalty, and a culture of respect. The survey collected about 5,500 completed responses and evaluated firms on 24 characteristics.

The company highlighted its three-decade culture, scientific focus, and mission-driven workforce and directed readers to its careers page for more information.

Ionis (NASDAQ: IONS) announced that chief executive officer Brett P. Monia, Ph.D. will receive the 2025 Lifetime Achievement Award from the Oligonucleotide Therapeutics Society (OTS).

Monia, a founding member of OTS who joined Ionis in 1989 and became CEO in 2020, has led programs that produced multiple FDA‑approved oligonucleotide medicines and advanced more than 40 clinical programs across neurological, cardiometabolic and rare diseases. He has authored over 250 publications and holds more than 100 patents.

The award will be presented at the 21st Annual OTS Meeting in Budapest on October 21, 2025, where Ionis will also present preclinical and clinical research via oral and poster sessions.

Ionis (Nasdaq: IONS) will host a live webcast on Wednesday, October 29, 2025 at 11:30 a.m. Eastern Time to discuss third quarter 2025 financial results and progress on key programs.

Investors can access the live webcast and a limited-time replay at https://ir.ionis.com/events-and-presentations/upcoming-events. The company will cover Q3 2025 results and program updates during the presentation.

Ionis (Nasdaq: IONS) hosted its 2025 Innovation Day highlighting recent independent launches, late‑stage data and a path to sustained positive cash flow.

Key points: two independent launches in under nine months; two more independent launches planned for 2026; four partner launches by end of 2027; TRYNGOLZA showed up to 72% fasting triglyceride reduction and 85% reduction in acute pancreatitis events; planned sNDA submission by year‑end; zilganersen pivotal positivity with NDA planned Q1 2026; ~$2B cash guidance (2025) and expected cash‑flow breakeven in 2028.

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.