Welcome to our dedicated page for Ionis Pharmaceuticals news (Ticker: IONS), a resource for investors and traders seeking the latest updates and insights on Ionis Pharmaceuticals stock.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) is a biotechnology company that focuses on RNA-targeted medicines for serious diseases, and its news flow reflects this emphasis on clinical, regulatory and commercial milestones. Company updates frequently highlight progress in neurology and cardiometabolic programs, as well as developments in rare conditions where few or no disease-modifying treatments exist.
Recent Ionis news has featured pivotal clinical trial readouts, such as Phase 3 CORE and CORE2 results for olezarsen in severe hypertriglyceridemia, showing large reductions in triglyceride levels and acute pancreatitis events with favorable safety and tolerability. The company has also reported positive pivotal data for zilganersen in Alexander disease, describing evidence of disease-modifying impact in this rare and often fatal neurological condition.
Regulatory and commercial milestones are another major theme. Ionis has announced FDA approval of TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome and DAWNZERA (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema in adults and adolescents 12 years and older. News items also cover FDA Breakthrough Therapy designations for olezarsen in severe hypertriglyceridemia and zilganersen in Alexander disease, as well as European regulatory opinions and anticipated launches.
Partnered program updates appear regularly, including GSK’s Phase 3 B-Well 1 and B-Well 2 results for bepirovirsen in chronic hepatitis B, Novartis’ Lp(a) HORIZON study of pelacarsen in cardiovascular disease, and AstraZeneca collaborations on WAINUA and eplontersen. Investors can also find coverage of Ionis’ financing activities, such as convertible note offerings, and its participation in healthcare and investor conferences.
For followers of IONS stock, this news page provides a centralized view of Ionis’ clinical trial milestones, FDA and EMA interactions, product launches, collaboration updates and capital markets events, helping readers understand how the company’s RNA-targeted pipeline and marketed medicines are evolving over time.
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 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.