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 leading biotechnology company headquartered in Carlsbad, California. Founded in 1989, Ionis has been pioneering the discovery and development of antisense drugs, with an impressive portfolio of over 1300 patents. Ionis focuses on creating innovative RNA-targeted therapies for complex diseases, particularly in the areas of cardiovascular, metabolic, neurological, and rare genetic disorders.
Ionis has successfully brought several groundbreaking medicines to market. Notable achievements include the development of Spinraza, in collaboration with Biogen, which was launched in 2016 to treat spinal muscular atrophy, a rare neuromuscular disorder. In 2023, Biogen also launched Qalsody for ALS, further extending the impact of Ionis' antisense technology.
Through its cardiovascular-focused subsidiary, Akcea Therapeutics, Ionis has introduced drugs like Tegsedi for ATTR amyloidosis and Waylivra in Europe for cardiology indications. The company continues to advance its pipeline with promising candidates such as olezarsen, an investigational RNA-targeted therapy for familial chylomicronemia syndrome (FCS). Ionis' ongoing Phase 3 trials for olezarsen have shown promising results, with significant reductions in triglyceride levels and improvements in related health outcomes.
Ionis' commitment to innovation and patient care is also evident in its robust financial health and strategic partnerships. With a deep understanding of disease biology and cutting-edge technology, Ionis continues to drive advancements in RNA therapies and gene editing, aiming to deliver life-changing medicines for patients around the world.
Recently, Ionis announced positive clinical trial results for several of its investigational drugs and plans to pursue regulatory approvals for these therapies. The company's pipeline is well-positioned to address unmet medical needs in various therapeutic areas, promising a steady flow of new treatments in the coming years.
For more detailed information about Ionis Pharmaceuticals, its products, and career opportunities, visit their official website at www.ionispharma.com.
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.
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