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 biotechnology leader pioneering RNA-targeted therapies through its antisense technology platform. This news hub provides investors and industry professionals with comprehensive updates on the company's clinical developments, strategic partnerships, and regulatory milestones.
Access real-time updates on Ionis' innovative research in neurology, cardiology, and rare diseases, including progress on therapies like Qalsody and Wainua. The page aggregates essential information including earnings reports, collaboration announcements with partners such as Biogen and AstraZeneca, and clinical trial outcomes.
Our curated collection serves as your primary source for tracking Ionis' advancements in antisense oligonucleotide development and commercialization strategies. Bookmark this page for streamlined access to verified updates about licensing agreements, patent developments, and therapeutic pipeline expansions.
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.
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.