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.
Alcyone Therapeutics has received FDA approval to continue enrollment in the PIERRE pivotal IDE clinical study for the ThecaFlex DRx™ System. This implantable device is being investigated for routine subcutaneous administration of nusinersen to treat spinal muscular atrophy (SMA). The first stage, involving 10 SMA patients, has been completed with no device-related adverse events. The FDA has approved enrollment of an additional 80 SMA patients across 30 centers in the U.S. and Europe.
Key observations include:
- Implantation duration: 1-2 hours
- Hospital discharge: Within 24 hours
- Infusion procedure: Less than 30 minutes
- Actual nusinersen infusion: Less than 10 minutes
The ThecaFlex system aims to provide a less invasive, more accessible alternative to repeat lumbar puncture for SMA patients receiving SPINRAZA® (nusinersen) therapy.
Ionis Pharmaceuticals (Nasdaq: IONS) announced positive results from the Phase 1/2 HALOS study of ION582 in people with Angelman syndrome (AS). The study showed consistent clinical improvement in communication, cognition, and motor function. Key findings include:
- 97% of patients in medium and high dose groups showed improvement in overall AS symptoms
- Improvements exceeded those observed in natural history studies
- ION582 demonstrated favorable safety and tolerability at all dose levels
Ionis plans to initiate Phase 3 development in H1 2025. The company views ION582 as a potential cornerstone of its next wave of transformational medicines for neurological conditions.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced a live webcast scheduled for Thursday, August 1st at 11:30 a.m. Eastern Time to discuss its second quarter 2024 financial results. Interested parties can access the webcast through the company's investor relations website at https://ir.ionis.com/events-and-presentations/upcoming-events. A replay of the webcast will be available for a time at the same address. This event provides an opportunity for investors and analysts to gain insights into Ionis' financial performance and potentially its future outlook.
Ionis Pharmaceuticals (Nasdaq: IONS) has completed enrollment in the pivotal trial of zilganersen (ION373), an investigational RNA-targeted medicine for Alexander disease (AxD). This rare, progressive neurological disorder affects an estimated one in one million people in the U.S. The trial's primary endpoint is percent change from baseline in gait speed, assessed by the 10-Meter Walk Test. Topline data are expected in the second half of 2025.
Zilganersen aims to address the underlying cause of AxD by targeting excess GFAP accumulation resulting from genetic variants in the GFAP gene. This approach could potentially slow or stabilize disease progression in AxD patients, who currently lack treatments that address the root cause of their condition.
Ionis Pharmaceuticals (NASDAQ: IONS) has announced a live webcast scheduled for Monday, July 22, at 8:00 a.m. Eastern Time. This webcast will discuss the results from the Phase 1/2a HALOS study of ION582, a treatment for Angelman syndrome. The findings will also be presented at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium on Wednesday, July 24, in Sandusky, Ohio. Interested parties can access the webcast via the provided link, with a replay available for a time.
Ionis Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for treating familial chylomicronemia syndrome (FCS). The FDA has granted Priority Review with a PDUFA date set for December 19, 2024. Olezarsen has also received Fast Track, Orphan Drug, and Breakthrough Therapy designations.
Additionally, Ionis completed Phase 3 enrollment for three studies (CORE, CORE2, and ESSENCE) evaluating olezarsen for severe hypertriglyceridemia (sHTG), with results anticipated in the second half of 2025. These studies involve over 2,400 patients and aim to address the life-threatening complications associated with sHTG.
The NDA is supported by positive Phase 3 study results presented at the 2024 ACC Annual Meeting and published in the New England Journal of Medicine.
Ionis Pharmaceuticals has entered an expanded licensing agreement with Otsuka Pharmaceutical for the investigational RNA-targeted medicine donidalorsen, aimed at treating hereditary angioedema (HAE).
Under the agreement, Otsuka gains exclusive rights to commercialize donidalorsen in the Asia-Pacific region while also handling regulatory filings and localized development. Ionis will continue to manage global development and plans to submit a New Drug Application to the FDA this year for U.S. approval, aiming for an independent U.S. launch.
Previously, Otsuka secured licensing for Europe and is preparing a Marketing Authorization Application to the EMA this year. Ionis will receive a $20 million upfront payment and milestone payments from Otsuka, along with tiered royalties.
Recent positive results from Phase 3 trials were presented at the EAACI Annual Congress and published in The New England Journal of Medicine.
Ionis Pharmaceuticals announced that its management will participate in a fireside chat at the TD Cowen Genetic Medicines & RNA Summit on June 20, 2024. The event will be virtual, and a live webcast will be available on the Ionis website. Interested parties can access the webcast on the Investors & Media section of the website, with a replay available within 48 hours for a time.
Ionis Pharmaceuticals revealed positive Phase 3 results for donidalorsen in treating hereditary angioedema (HAE). The OASIS-HAE and OASISplus studies showed significant reductions in mean monthly HAE attack rates, with a >90% reduction over one year. Patients switching from other treatments saw a 62% further reduction, and 84% preferred donidalorsen. Favorable safety and tolerability were reported across all cohorts. Ionis plans to seek regulatory approval based on these results. These findings will be presented at the EAACI Congress 2024, and Ionis aims to launch donidalorsen as part of its independent commercial pipeline.
Ionis Pharmaceuticals (NASDAQ: IONS) announced a live webcast scheduled for May 31, 2024, at 8:00 a.m. Eastern Time to discuss the donidalorsen Phase 3 OASIS-HAE study results. These results will be presented during three late-breaking oral presentations at the 2024 EAACI Annual Meeting in Valencia, Spain. The webcast and a -time replay can be accessed at the Ionis website.