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 (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.
Ionis Pharmaceuticals (NASDAQ: IONS) and Genuity Science have announced a multi-year collaboration focused on accelerating the development of therapeutics for up to 20 diseases. Genuity Science will leverage whole-genome sequencing and deep phenotype data, enhancing Ionis' antisense technology to expedite drug development. The partnership involves upfront payments, milestone incentives, and royalties, aimed at increasing discovery opportunities and understanding genetic disease complexities.
Ionis Pharmaceuticals (NASDAQ: IONS) announced that the FDA has granted orphan drug designation to ION373 for treating Alexander disease, a rare neurodegenerative disease. This designation is crucial as it highlights the urgent need for effective treatments for this debilitating condition, which affects roughly one in one million births.
The FDA's Orphan Drug Act offers significant incentives, including a seven-year market exclusivity after approval. Ionis previously received similar status from the EMA, reflecting global recognition of ION373's potential in addressing unmet medical needs.
Ionis Pharmaceuticals (Nasdaq: IONS) announced its participation in several virtual investor conferences. Management will present at the Wells Fargo Healthcare Conference on September 10, 2020, followed by the Morgan Stanley Global Healthcare Conference on September 14, 2020, the BofA Global Healthcare Conference on September 18, 2020, and the Oppenheimer Life Sciences Summit on September 22, 2020. The presentation details will be available on Ionis' website, with live webcasts and later replays offered. Ionis leads in RNA-targeted drug development.
Ionis Pharmaceuticals (NASDAQ: IONS) has published promising results from a compassionate-use study of its investigational drugs, IONIS-PKKRx and IONIS-PKK-LRx, for treating severe hereditary angioedema (HAE). The study shows these antisense medicines effectively reduced plasma prekallikrein activity and cut breakthrough attack rates in patients. Notably, one patient achieved complete resolution of symptoms. Ionis is advancing the development of IONIS-PKK-LRx, which offers a potential monthly treatment option, aiming to improve patient outcomes.
Akcea Therapeutics and Ionis Pharmaceuticals announced positive results from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-LRx) at the ESC Congress 2020. The trial showed significant reductions in triglyceride levels, achieving a maximum reduction of 53% with the 80 mg dose. The therapy demonstrated a favorable safety profile, with injection site reactions being the most common adverse events. This investigational antisense therapy targets ANGPTL3 to reduce cardiovascular risks. Pfizer plans further Phase 2b studies to optimize dosing.
Akcea Therapeutics (NASDAQ: AKCA) and Ionis Pharmaceuticals (NASDAQ: IONS) announced positive results from the Phase 2 study of AKCEA-APOCIII-LRx, presented at the ESC Congress 2020. The study demonstrated significant reductions in triglyceride levels (TG) and apoC-III, with a notable 62% reduction in TG at the highest dose administered. Furthermore, 91% of patients achieved TG levels below 150 mg/dL at six months. The drug exhibited a favorable safety profile, with plans for a Phase 3 trial targeting familial chylomicronemia syndrome (FCS) later this year.
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