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) 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.
Ionis Pharmaceuticals will host a live webcast on September 2 at 10:00 a.m. ET to discuss its cardio-renal franchise. The 90-minute session will feature presentations from its executives, including Brett P. Monia and Sam Tsimikas. Key topics will include advances in RNA-targeted drug discovery and specific medicines aimed at treating triglyceride-driven diseases, thrombosis, and treatment-resistant hypertension. The webcast can be accessed at www.ionispharma.com.
On August 24, 2020, Akcea Therapeutics and Ionis Pharmaceuticals announced that data from the Phase 2 clinical trial of vupanorsen (AKCEA-ANGPTL3-LRx) will be presented at the ESC Congress 2020, scheduled for August 29-September 1, 2020. Vupanorsen is an investigational therapy targeting cardiovascular diseases by reducing ANGPTL3 protein levels in patients with conditions such as hypertriglyceridemia and type 2 diabetes. The companies previously secured a $250 million licensing deal with Pfizer, which will assume further development responsibilities.
Akcea Therapeutics and Ionis Pharmaceuticals announced that data from the Phase 2 study of AKCEA-APOCIII-LRx will be presented at the ESC Congress 2020 from August 29 to September 1, 2020. The drug aims to reduce apolipoprotein C-III levels, a protein linked to triglyceride regulation and cardiovascular risk. Previous studies showed promising results, with dose-dependent reductions in triglycerides and increases in HDL-C. This presentation may influence the drug's market potential and investor outlook.
Akcea Therapeutics has announced new pricing and reimbursement terms for WAYLIVRA® (volanesorsen) in Germany, effective August 15, 2020. This renegotiation, required 12 months post-commercialization, highlights the drug's value for patients with familial chylomicronemia syndrome (FCS) at high risk for pancreatitis. WAYLIVRA is now the only therapy approved for FCS in the EU, following its launch in August 2019. Akcea aims to expand WAYLIVRA's availability to more European countries, supporting the needs of patients suffering from this ultra-rare disease.
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