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 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.
Ionis Pharmaceuticals (Nasdaq: IONS) will present new subgroup analyses from its Phase 3 NEURO-TTRansform study of WAINUA™ (eplontersen) at the 2024 International Symposium on Amyloidosis (ISA). The study highlights consistent benefits in neuropathy impairment and quality of life across various patient segments. WAINUA, approved by the FDA in December 2023 for treating hATTR-PN, will have several notable presentations on May 29, 2024. Additionally, eplontersen is being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This study, with over 1,400 patients, is the largest in this patient population.
Ionis Pharmaceuticals and Biogen announced the discontinuation of BIIB105 (ION541), an investigational antisense oligonucleotide for amyotrophic lateral sclerosis (ALS), based on Phase 1/2 ALSpire study results. Despite demonstrating statistically significant reductions in cerebrospinal fluid ATXN2 protein, BIIB105 did not lower plasma neurofilament light chain (NfL) levels, nor did it impact clinical outcomes such as function, breathing, and strength over a 6-month period. Common adverse events included procedural pain, headache, and fall, with higher discontinuation rates in the BIIB105 group. Further analyses of the data are ongoing, and results will be presented at the European Network to Cure ALS (ENCALS) meeting in June 2024.
Ionis Pharmaceuticals announced positive topline results from its Phase 1/2a trial of ION582 for Angelman syndrome, a serious neurodevelopmental disorder. The HALOS study showed ION582 to be safe and well-tolerated, with consistent improvements across cognition, communication, and motor functions. Approximately 65-70% of patients showed improvements in key functional areas at six months. Ionis plans to advance ION582 into a pivotal trial and present detailed data at an upcoming medical meeting. However, Biogen has opted not to license ION582.
Ionis Pharmaceuticals announced it will hold its 2024 virtual Annual Meeting of Stockholders on June 6, 2024. The event will start at 5:00 p.m. ET, with stockholders of record as of April 8, 2024, invited to participate. A general corporate update, including a Q&A session, will follow at 5:30 p.m. ET, presented by CEO Brett P. Monia. Stockholders will receive proxy cards needed to log in, vote, and submit questions. Technical assistance will be available. The event will be webcast live, with an archived replay available later.
Ionis Pharmaceuticals, Inc. reported its first quarter 2024 financial results, highlighting positive Phase 3 data for HAE and regulatory submissions for olezarsen. The company remains on track to achieve its 2024 financial guidance, with revenue totaling $119 million and operating expenses increasing due to strategic investments in new products. Ionis' cash and short-term investments stand at $2.2 billion, supporting planned upcoming launches and reaffirmed financial guidance.
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