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.
Akcea Therapeutics, a subsidiary of Ionis Pharmaceuticals (NASDAQ: IONS), has received approval from the Scottish Medicines Consortium (SMC) for volanesorsen, enabling access via the NHS in Scotland for treating familial chylomicronaemia syndrome (FCS). This follows NICE's earlier approval in England. Volanesorsen is the only treatment available for FCS in the UK, aimed at reducing triglyceride levels significantly. The SMC's decision is a milestone for FCS patients, allowing access while additional clinical data is collected over three years.
Ionis Pharmaceuticals reported its third-quarter 2020 financial results, showing a net income of $5 million (non-GAAP) but a GAAP net loss of $31 million. The company achieved revenue growth, with commercial revenues of $95 million driven by SPINRAZA royalties of $74 million. The recent acquisition of Akcea strengthens Ionis's pipeline, projecting 10 or more marketing applications by 2025. Cash reserves stand at $2.3 billion post-acquisition, maintaining solid financial health.
Ionis Pharmaceuticals and Akcea Therapeutics have won the Prix Galien USA Award for the Best Biotechnology Product for 2020, recognizing TEGSEDI, the first self-administered treatment for the polyneuropathy of hereditary ATTR amyloidosis. Presented in a virtual ceremony on October 29, 2020, this prestigious award highlights significant advancements in biomedicine. Brett P. Monia emphasized TEGSEDI's role in improving the lives of patients affected by this severe disease. TEGSEDI is FDA-approved and offers hope for individuals suffering from hATTR amyloidosis.
Ionis Pharmaceuticals announced the dosing of the first patients with ION541, an investigational therapy for amyotrophic lateral sclerosis (ALS). This milestone is significant as ION541 targets ataxin-2 RNA, potentially reversing TDP-43 toxicity, a common characteristic of ALS. Supported by a $10 million payment from Biogen, this Phase 1/2 trial aims to address both familial and sporadic ALS cases. Ionis continues to advance its ALS pipeline, which includes tofersen and IONIS-C9Rx, enhancing its commitment to developing innovative therapies for this devastating condition.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) will host a live webcast on November 4, 2020, at 11:30 a.m. ET to discuss its third quarter 2020 financial results and provide updates on its pipeline and business progress. The webcast will be available on their website, www.ionispharma.com, with a replay for a limited time after the event.
Ionis Pharmaceuticals (NASDAQ: IONS) announced that IONIS-PKK-LRx is being tested in a Phase 2 clinical study aimed at reducing respiratory complications in COVID-19 patients. Conducted in Brazil, this study will enroll up to 110 patients across 25 hospitals. The primary endpoint is to monitor the number of days patients are alive and free of oxygen support over 15 days. IONIS-PKK-LRx targets bradykinin signaling, a key factor in severe COVID-19 cases. Additionally, promising data from a related HAE study has been published.
Ionis Pharmaceuticals (NASDAQ: IONS) announced significant results from a clinical trial of IONIS-ENAC-2.5Rx, an investigational antisense medicine for cystic fibrosis (CF). The trial demonstrated a mean 55.6% reduction in ENaC mRNA expression at a 75 mg dose (p<0.05), marking a breakthrough in aerosol delivery methods. This study lays the groundwork for future developments in treating CF and other lung diseases. The company plans to present additional data at the 2020 North American Cystic Fibrosis Conference from Oct. 21-23 and initiate a study for COPD later this year.
Ionis Pharmaceuticals (NASDAQ: IONS) announced that Frank Bennett, Ph.D., received the 2020 Lifetime Achievement Award from the Oligonucleotide Therapeutics Society. Dr. Bennett was honored for his significant contributions to antisense oligonucleotide drugs aimed at correcting severe genetic diseases. He is a founding member of Ionis and leads neurological programs. Additionally, Ionis scientists won the Paper of the Year Award for their research on a new antisense mechanism, reinforcing the company's leadership in RNA-targeted therapeutics.
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