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 participation in key investor conferences featuring fireside chats this September 2022. These events include the Wells Fargo Healthcare Conference on September 7, Citi's BioPharma Conference on September 8, and the Morgan Stanley Global Healthcare Conference on September 12. Live webcasts will be available on the Ionis website, with replays accessible within 48 hours post-event.
Ionis leads in RNA-targeted therapies, with three marketed medicines and a strong pipeline in cardiovascular and neurological sectors.
Ionis Pharmaceuticals reported positive Phase 3 data for eplontersen targeting hereditary ATTR polyneuropathy, with an NDA filing expected in H2 2022. The FDA granted priority review for tofersen, with a decision anticipated by January 25, 2023. The company achieved $134 million in Q2 2022 revenues, reflecting a 15% year-over-year increase, although commercial revenue declined 12% due to decreased SPINRAZA royalties. Ionis ended Q2 with $2 billion in cash, maintaining financial stability to support its late-stage pipeline.
Ionis Pharmaceuticals has announced positive topline results from its Phase 2b RE-THINC ESRD study of fesomersen, an investigational therapy targeting patients with end-stage renal disease on hemodialysis. The study achieved its primary goal, with no increase in major bleeding incidents compared to placebo. Fesomersen, demonstrating significant reductions in Factor XI activity levels, was deemed safe and well-tolerated. This outcome underlines its potential as a novel anti-thrombotic treatment, addressing a critical unmet need for patients at high risk for thromboembolic events and bleeding complications.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced a live webcast on August 9 at 11:30 a.m. Eastern Time to discuss its second quarter financial results and update on its pipeline and business progress. The event underscores Ionis's commitment to transparency and communication with investors regarding its financial performance and future strategies. The webcast will be accessible on their website, and a replay will be available for a limited time.
Ionis Pharmaceuticals announced that the FDA has accepted its New Drug Application for tofersen, aimed at treating SOD1-ALS, a severe genetic form of ALS affecting around 330 people in the U.S. If approved, tofersen will be the first treatment targeting a genetic cause of this disease. Data from 12-month studies indicate earlier tofersen initiation slowed decline across multiple clinical measures and significantly reduced neurofilament levels, reflecting neurodegeneration. The FDA has granted priority review with a target action date of January 25, 2023.
Ionis Pharmaceuticals has announced the completion of patient enrollment in the pivotal Phase 3 Lp(a) HORIZON study of pelacarsen, with 8,325 participants. Pelacarsen targets elevated lipoprotein(a) levels, a significant risk factor for cardiovascular disease. This study, conducted by Novartis, aims to assess the safety and efficacy of pelacarsen in reducing cardiovascular events in patients with elevated Lp(a). There are currently no approved therapies for effectively lowering Lp(a), and topline results are expected in 2025.
Ionis Pharmaceuticals (Nasdaq: IONS) will participate in a virtual fireside chat at the H.C. Wainwright 1st Annual Hereditary Angioedema Conference on July 20, 2022. Investors can access the live webcast on the Ionis website; a replay will be available within 48 hours post-event. Ionis has been a leader in RNA-targeted therapy for over 30 years, with a strong pipeline and three marketed medicines. The company aims to revolutionize standards of care through innovative therapies.
Ionis Pharmaceuticals announced that Roche will advance the investigational drug IONIS-FB-LRx into a Phase 3 study for treating IgA nephropathy (IgAN), following positive results from a Phase 2 trial. This study showed a significant reduction in 24-hour urinary protein levels after 29 weeks. The Phase 2 study also demonstrated a favorable safety profile. Ionis will receive $55 million for licensing the drug and achieving a milestone. This partnership reinforces Ionis' commitment to addressing unmet needs in kidney diseases.
Ionis Pharmaceuticals announced positive interim results from the Phase 2b B-Clear study of bepirovirsen for chronic hepatitis B treatment, presented at the EASL International Liver Congress 2022. The study showed a virologic response (VR) in 28% of patients on nucleoside analogue treatment and 29% of those not on treatment after 24 weeks. GSK plans to initiate a Phase 3 study in the first half of 2023. Bepirovirsen aims to offer a functional cure for chronic hepatitis B, impacting approximately 300 million people globally.
Ionis Pharmaceuticals (Nasdaq: IONS) announced positive topline results from the Phase 3 NEURO-TTRansform study of eplontersen for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The 35-week interim analysis showed statistically significant improvements in both the modified Neuropathy Impairment Score +7 and Norfolk Quality of Life Questionnaire-Diabetic Neuropathy. Eplontersen also proved to have a favorable safety profile. Ionis and AstraZeneca plan to submit a New Drug Application to the FDA this year.