Ionis Pharmaceuticals Stock Price, News & Analysis

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.

Ionis Pharmaceuticals announced that Executive VP and CSO C. Frank Bennett has received the 23rd Jacob and Louise Gabbay Award for his contributions to antisense therapy. The award honors significant advances in biomedical sciences and includes a cash prize and medallion. Bennett shares the recognition with Adrian R. Krainer and is acknowledged for his pivotal role in developing SPINRAZA, the key treatment for spinal muscular atrophy. He is a founding member of Ionis and has received several prestigious awards for his ongoing research in RNA-targeted therapies.

Ionis Pharmaceuticals (NASDAQ: IONS) announced the presentation of its advances in ATTR amyloidosis therapies at the 2021 Peripheral Nerve Society Annual Meeting, scheduled for June 12-13 and 25-27. The company highlights its commitment to innovating antisense therapies for this condition. Key presentations include a plenary lecture by Dr. Frank Bennett and a panel discussion moderated by Dr. Kemi Olugemo. Additionally, multiple on-demand posters will address various aspects of TTR amyloidosis treatment, showcasing Ionis' ongoing research and development efforts in the field.

Ionis Pharmaceuticals has announced significant leadership changes in its board of directors. Joseph Loscalzo, M.D., Ph.D., has been appointed chairman, succeeding Stanley Crooke, who has retired but will remain as a scientific advisor. Allene M. Diaz has also joined the board. Dr. Loscalzo's background includes extensive experience at Harvard Medical School and numerous accolades in cardiovascular science. Diaz brings a wealth of experience from her previous roles in GlaxoSmithKline and other firms. These changes aim to bolster Ionis's strategy towards delivering over 12 novel RNA-targeted therapies by 2026.