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.
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Ionis Pharmaceuticals has announced that its partner Biogen received FDA approval for QALSODY (tofersen) as the first treatment targeting a genetic cause of amyotrophic lateral sclerosis (ALS)SOD1 gene mutation. This accelerated approval is based on a significant reduction of the neurofilament light chain, a biomarker linked to neuronal damage in ALS patients treated with QALSODY. The drug is administered via injection and aims to address the severe impact of SOD1 mutations, with ongoing trials to confirm clinical benefits. Notably, QALSODY joins another Ionis-developed drug, SPINRAZA, in tackling rare neurodegenerative diseases. However, approval is contingent on further demonstration of clinical efficacy from ongoing studies.
Ionis Pharmaceuticals announced successful results from its Phase 3 NEURO-TTRansform study for eplontersen, aimed at treating hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The study met all co-primary and secondary endpoints, with a significant reduction of 82% in serum TTR concentration among treated patients compared to an 11% reduction in the placebo group (p<0.0001). Eplontersen also halted disease progression and improved quality of life, as measured by neuropathy impairment and quality of life scores. The findings, presented at AAN 2023, highlight eplontersen's potential as a new treatment option for patients with this fatal disease. The FDA has accepted a New Drug Application for eplontersen, with a PDUFA date set for December 22, 2023, while the company plans further regulatory submissions in Europe and beyond.
Ionis Pharmaceuticals has announced a live webcast scheduled for May 3, 2023, at 11:30 a.m. Eastern Time, to discuss its first quarter 2023 financial results. This event will provide insights into the company's financial performance, emphasizing its leadership in RNA-targeted therapies. Ionis has over three decades of experience and currently boasts three marketed medicines alongside a robust late-stage pipeline focused on cardiovascular and neurological treatments. Stakeholders can access the webcast and follow up with a replay through Ionis' investor relations page.
Ionis Pharmaceuticals announced new Phase 1b clinical data for IONIS-MAPTRx (BIIB080), revealing a dose-dependent reduction of tau protein in cerebrospinal fluid (CSF) in early-stage Alzheimer's patients. The study, presented at AD/PD 2023, showed a significant decrease in tau pathology measured by positron emission tomography (PET) at week 25 through week 100. In all dose groups, there was a 60% reduction in baseline CSF tau levels by study's end. The primary focus was on safety, with mild to moderate adverse events reported. The ongoing Phase 2 CELIA study for IONIS-MAPTRx is currently recruiting participants in the U.S.
Ionis Pharmaceuticals (NASDAQ: IONS) announced participation in upcoming investor conferences, enhancing its visibility in the healthcare sector. Notable events include the Guggenheim Virtual Genomic Medicines and Rare Disease Days on April 4, 2023, and the 22nd Annual Needham Virtual Healthcare Conference on April 17, 2023. A live webcast of the presentations will be available on the Ionis website, with replays accessible within 48 hours.
With over 30 years in RNA-targeted therapy, Ionis is a leader in genetic medicine, possessing three marketed medicines and a strong late-stage pipeline focusing on cardiovascular and neurological conditions.
Ionis Pharmaceuticals reported positive topline results from the 66-week analysis of the Phase 3 NEURO-TTRansform study for eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The study met co-primary endpoints, showing significant reductions in TTR levels and improved neuropathy symptoms and quality of life. Results will be shared at the AAN Annual Meeting in April, with U.S. FDA accepting a New Drug Application for eplontersen, targeting a decision by December 22, 2023. The data indicates eplontersen may fulfill a critical need for new treatments for this severe condition.
Ionis Pharmaceuticals announced a favorable outcome from the FDA's Advisory Committee meeting regarding tofersen, a treatment for SOD1 amyotrophic lateral sclerosis (ALS). The committee voted 9-0 that the evidence suggests a reduction in neurofilament light chain (NfL) levels indicates potential clinical benefits. However, there was mixed support for traditional approval, with 3 in favor, 5 against, and 1 abstention. If approved by the FDA, expected by April 25, 2023, tofersen would be the first treatment targeting a genetic cause of ALS. This represents a significant advancement in ALS therapeutics, as there are currently no genetically targeted treatments available.
Ionis Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for eplontersen, an investigational treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The application is set for a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA indicated no review issues and will not hold an advisory committee meeting. The NDA is based on the Phase 3 NEURO-TTRansform study, which showed significant reductions in serum transthyretin (TTR) levels and improved quality of life for patients. Eplontersen has already received Orphan Drug Designation.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced its upcoming presentations at three investor conferences. These include the Cowen 43rd Annual Health Care Conference on March 7, 2023, the Oppenheimer 33rd Annual Virtual Healthcare Conference on March 15, 2023, and the Stifel 2023 Virtual CNS Days on March 29, 2023. Investors can access a live webcast during the events via the Investors & Media section on the Ionis website, with replays available shortly after. Ionis has been a pioneer in RNA-targeted therapy for over 30 years, focusing on innovative genetic medicine therapies.