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 company that focuses on RNA-targeted medicines for serious diseases, and its news flow reflects this emphasis on clinical, regulatory and commercial milestones. Company updates frequently highlight progress in neurology and cardiometabolic programs, as well as developments in rare conditions where few or no disease-modifying treatments exist.
Recent Ionis news has featured pivotal clinical trial readouts, such as Phase 3 CORE and CORE2 results for olezarsen in severe hypertriglyceridemia, showing large reductions in triglyceride levels and acute pancreatitis events with favorable safety and tolerability. The company has also reported positive pivotal data for zilganersen in Alexander disease, describing evidence of disease-modifying impact in this rare and often fatal neurological condition.
Regulatory and commercial milestones are another major theme. Ionis has announced FDA approval of TRYNGOLZA (olezarsen) for familial chylomicronemia syndrome and DAWNZERA (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema in adults and adolescents 12 years and older. News items also cover FDA Breakthrough Therapy designations for olezarsen in severe hypertriglyceridemia and zilganersen in Alexander disease, as well as European regulatory opinions and anticipated launches.
Partnered program updates appear regularly, including GSK’s Phase 3 B-Well 1 and B-Well 2 results for bepirovirsen in chronic hepatitis B, Novartis’ Lp(a) HORIZON study of pelacarsen in cardiovascular disease, and AstraZeneca collaborations on WAINUA and eplontersen. Investors can also find coverage of Ionis’ financing activities, such as convertible note offerings, and its participation in healthcare and investor conferences.
For followers of IONS stock, this news page provides a centralized view of Ionis’ clinical trial milestones, FDA and EMA interactions, product launches, collaboration updates and capital markets events, helping readers understand how the company’s RNA-targeted pipeline and marketed medicines are evolving over time.
Ionis Pharmaceuticals has scheduled a live webcast to discuss its Q3 2021 financial results and business progress on November 3, 2021, at 11:30 a.m. ET. This session will provide insights into the company's pipeline developments and financial performance, contributing to its position in the RNA-targeted therapy market. The webcast is accessible to interested parties via Ionis's official website, with a replay available for a limited time afterwards.
Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced the topline results from Biogen's Phase 3 VALOR study of tofersen, an antisense medicine for SOD1 amyotrophic lateral sclerosis (ALS). Although the primary endpoint was not met, secondary measures indicated trends favoring tofersen. The study involved 108 participants, showing 38% and 67% differences in key biomarkers for faster and slower progressors, respectively. Biogen plans to engage regulatory bodies for next steps, while expanding its early access program for SOD1-ALS patients.
Ionis Pharmaceuticals (NASDAQ: IONS) will present 14 posters and two symposiums at the 3rd European ATTR Amyloidosis meeting from September 6-8, 2021. Highlighting advancements in its ATTR amyloidosis portfolio, the presentations will focus on the investigational antisense medicine, eplontersen, which is being evaluated in two phase 3 trials for transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN). Ionis aims to enhance treatment options for patients impacted by this disease.
Ionis Pharmaceuticals (Nasdaq: IONS) announced its participation in upcoming virtual investor conferences, including the 2021 Wells Fargo Healthcare Conference on September 10, the Morgan Stanley 19th Annual Global Healthcare Conference on September 13, and the Bank of America Global Healthcare Conference 2021 on September 15. Presentation details may change, with updates available on the company's website. A live webcast will be offered, with replays accessible within 48 hours. Ionis is a leader in RNA-targeted therapy, with three marketed medicines and a strong pipeline.
Ionis Pharmaceuticals reported Q2 2021 financial results, with total revenues of $126 million and a net loss of $36 million (non-GAAP). The company is well capitalized, ending the quarter with $2.1 billion in cash and investments. Key advancements include full enrollment in the Phase 3 NEURO-TTRansform study and ongoing studies for tofersen and pelacarsen. Ionis aims for more than $600 million in revenue for 2021, while operational changes have streamlined costs. The company anticipates significant data readouts in H2 2021, potentially leading to numerous new product launches by 2026.
Ionis Pharmaceuticals announced significant progress for pelacarsen, a potential treatment for lipoprotein(a)-driven cardiovascular disease, as the Lp(a) HORIZON study reached 50% enrollment with 4,000 participants. This Phase 3 study aims to support the drug's indication for reducing cardiovascular risk. Ionis received a $25 million milestone payment from Novartis and is eligible for up to $675 million in additional milestones, along with mid-teens to low 20% royalties on pelacarsen sales. This investigational medicine utilizes LICA technology to target elevated Lp(a) levels, a significant risk factor for cardiovascular diseases.
Ionis Pharmaceuticals (NASDAQ: IONS) has successfully completed enrollment in its pivotal Phase 3 NEURO-TTRansform study, exceeding its target with over 160 patients participating. The study focuses on eplontersen for treating hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). Eplontersen, an investigational drug using Ionis' LICA technology, showed up to 94% reductions in transthyretin (TTR) in Phase 1 trials. An interim analysis is expected by mid-2022, with a potential NDA filing by year-end 2022.
Biogen and Ionis Pharmaceuticals reported positive topline data from a Phase 1b clinical trial of BIIB080, aimed at treating mild Alzheimer's disease. The trial demonstrated significant dose-dependent reductions in tau protein levels in cerebrospinal fluid, achieving mean reductions of 30% to 49% across various dosing schedules. All participants completed the treatment phases, with no serious adverse events reported. These findings underscore the potential of BIIB080 and Ionis' antisense technology in addressing neurodegenerative diseases, paving the way for further clinical investigations.
Ionis Pharmaceuticals (NASDAQ: IONS) announced a live webcast scheduled for August 4, 2021, at 11:30 a.m. ET, to discuss its Q2 2021 financial results along with updates on its pipeline and business progress. Ionis has been a leader in RNA-targeted therapy for over 30 years, with three marketed medicines and a strong late-stage pipeline. The webcast will be accessible via their investor relations page, and a replay will be available for a limited time.
Ionis Pharmaceuticals has entered an exclusive licensing agreement with Bicycle Therapeutics to enhance its LIgand Conjugated Antisense (LICA) medicines. The deal grants Ionis access to Bicycle's proprietary Bicycles, targeting transferrin receptor 1 to improve delivery of oligonucleotides. Ionis made a $45 million upfront payment, which includes a license fee and equity investment. This partnership aims to boost Ionis' drug discovery capabilities, potentially leading to better treatment options for patients.