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 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.
Ionis Pharmaceuticals announced that the FDA granted orphan drug and rare pediatric disease designations to its investigational drug ION582, targeting Angelman syndrome. This neurogenetic disorder affects 1 in 12,000 to 20,000 people, resulting in severe developmental delays and caregiver dependence. The orphan drug status offers incentives such as tax credits and market exclusivity. ION582 is currently in a Phase 1/2 clinical trial with 44 participants. The company aims to expedite its development process to meet urgent patient needs.
The Phase 3 VALOR study of tofersen, an investigational therapy for SOD1-ALS, revealed that early initiation significantly slowed declines in clinical function, respiratory function, muscle strength, and quality of life. The 12-month data demonstrated a 33% reduction in SOD1 protein levels and a 51% decrease in neurofilament, indicating reduced neurodegeneration. Additionally, early initiation was correlated with a lower risk of death or permanent ventilation. However, the initial six-month study did not meet its primary endpoint.
Ionis Pharmaceuticals (Nasdaq: IONS), located in Carlsbad, Calif., will present at the RBC Capital Markets Global Healthcare Conference on May 18, 2022. A live webcast of the presentation will be available on Ionis's website, with a replay accessible within 48 hours after the event. Ionis has over 30 years of experience in RNA-targeted therapy and boasts a strong pipeline focusing on cardiovascular and neurological franchises.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) reported a strong start to 2022, achieving $142 million in total revenues for Q1, representing over 25% growth year-over-year. The firm remains on track to meet its 2022 financial guidance. Key highlights include positive data from studies of eplontersen, ION449, and donidalorsen. Operating expenses totaled $173 million on a non-GAAP basis, yielding a net loss of $39 million. Ionis maintains a robust cash position with $2.1 billion available for continued investments in its late-stage pipeline, positioning the company for future growth.
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