Core Insights - Ionis Pharmaceuticals has completed Phase 3 enrollment for olezarsen, targeting severe hypertriglyceridemia, with results anticipated in the second half of 2025 [1][3] - The FDA has accepted the New Drug Application for olezarsen for familial chylomicronemia syndrome (FCS) under Priority Review, with an action date set for December 19, 2024 [1][12] - Olezarsen is designed to lower apoC-III production, a protein that regulates triglyceride metabolism, and is being evaluated for both FCS and severe hypertriglyceridemia [4][12] FDA Review Process - Priority Review designation indicates the potential for significant improvements in treatment, with FDA action expected within six months [2] - The application is based on positive results from the Balance Phase 3 study, presented at the 2024 ACC Annual Meeting and published in NEJM [2] Clinical Trials and Patient Demographics - Three Phase 3 trials (CORE, CORE2, ESSENCE) for severe hypertriglyceridemia have enrolled over 1,000 patients with triglyceride levels ≥500 mg/dL and over 1,400 patients with levels between 150 mg/dL and <500 mg/dL [3][7] - Severe hypertriglyceridemia affects over three million people in the U.S., posing risks of pancreatitis and cardiovascular issues [7][17] Market Need and Treatment Landscape - There are currently no FDA-approved therapies for FCS, and patients rely on restrictive diets for management [6][15] - Existing treatment guidelines recommend lifestyle changes and medications for triglyceride levels ≥500 mg/dL, but many patients require additional therapeutic options [17] Company Overview - Ionis Pharmaceuticals has a strong pipeline in RNA-targeted medicines and has developed five marketed medicines, focusing on serious diseases [18]

Year to date, Ionis' shares have lost 19.7% compared with the industry's 8.4 % fall. Ionis currently carries a Zacks Rank #3 (Hold).You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here. Ionis Pharmaceuticals (IONS) has entered into a licensing agreement with Otsuka Pharmaceutical. Per the terms, Otsuka will acquire exclusive rights to market its investigational late-stage hereditary angioedema (HAE) therapy, donidalorsen, in the Asia-Pacific region. Image Source: Zacks Investment ...

Core Insights - Ionis Pharmaceuticals has entered into a licensing agreement with Otsuka Pharmaceutical for donidalorsen, granting Otsuka exclusive rights in the Asia-Pacific region while Ionis retains primary responsibility for development [1][2] - Ionis plans to file a New Drug Application with the FDA for donidalorsen this year and will independently launch the drug in the U.S. if approved [2][3] - The agreement includes a $20 million upfront payment to Ionis, with additional milestone payments based on regulatory and sales targets, and tiered royalties similar to the European agreement [3] Company Overview - Ionis Pharmaceuticals specializes in RNA-targeted medicines and has a pipeline focused on neurology, cardiology, and other high-need areas, with five marketed medicines [7] - The company has built a robust commercial infrastructure in preparation for upcoming launches, including donidalorsen for hereditary angioedema (HAE) [3][4] Product Information - Donidalorsen is an investigational medicine designed to target prekallikrein (PKK), which is involved in the inflammatory response associated with HAE attacks [6] - HAE is a rare genetic condition affecting over 20,000 patients in the U.S. and Europe, characterized by recurrent severe swelling [5]

Company Overview - Ionis Pharmaceuticals has been innovating medicines for over three decades, focusing on serious diseases [3] - The company currently markets five medicines and has a strong pipeline in neurology, cardiology, and other high-need areas [3] - Ionis is a pioneer in RNA-targeted medicines and is advancing new approaches in gene editing [3] Upcoming Events - Ionis management will participate in a fireside chat at the virtual TD Cowen Genetic Medicines & RNA Summit on June 20, 2024 [1] - A live webcast of the presentation will be available on the Ionis website, with a replay accessible within 48 hours [2]

"We're delighted by the results from the OASIS clinical program, which we believe position donidalorsen to advance the prophylactic treatment paradigm for people living with HAE. Despite currently available therapies, people living with HAE still face significant disease burden and new prophylactic treatments are needed," said Brett Monia, Ph.D., chief executive officer of Ionis. "These data underscore the potential of donidalorsen to continually improve HAE attack rates and quality of life over time, posit ...

Core Insights - Ionis Pharmaceuticals will host a live webcast on May 31, 2024, at 8:00 a.m. Eastern Time to discuss the results of the donidalorsen Phase 3 OASIS-HAE and OASISplus studies [1] - The study results will be presented in three late-breaking oral presentations at the 2024 European Academy of Allergy and Clinical Immunology Annual Meeting in Valencia, Spain [1] Company Overview - Ionis Pharmaceuticals has been innovating medicines for over three decades, focusing on serious diseases [3] - The company currently markets five medicines and has a strong pipeline in neurology, cardiology, and other high-need areas [3] - As a pioneer in RNA-targeted medicines, Ionis is advancing RNA therapies and exploring new gene editing approaches [3]

Core Insights - Ionis Pharmaceuticals announced new subgroup analyses from the Phase 3 NEURO-TTRansform study of WAINUA™ (eplontersen), showing consistent benefits in neuropathy impairment and improved quality of life across different patient segments [1] - WAINUA was approved by the FDA in December 2023 for treating hereditary transthyretin-mediated amyloidosis polyneuropathy (hATTR-PN) in adults [1][4] - AstraZeneca and Ionis are collaborating on the commercialization of WAINUA in the U.S. and are pursuing regulatory approval in Europe and other regions [2] Study Details - The NEURO-TTRansform study is a global, open-label, randomized trial assessing the efficacy and safety of eplontersen in patients with ATTRv-PN, with evaluations at weeks 35, 66, and 85 [10] - Eplontersen is also being evaluated in the Phase 3 CARDIO-TTRansform study for patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), which is fully enrolled with over 1,400 patients, making it the largest study in this patient population [3] Product Information - WAINUA™ (eplontersen) is a LIgand-Conjugated Antisense (LICA) medicine designed to inhibit the production of transthyretin (TTR) protein, specifically indicated for treating polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults [4][5] - The most common adverse reactions reported in WAINUA-treated patients include decreased serum vitamin A levels (15%) and vomiting (9%) [7] Disease Context - Hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) is characterized by the accumulation of misfolded TTR protein in peripheral nerves, leading to debilitating nerve damage and progressive loss of motor functions, with a typical disability onset within five years of diagnosis [11]

Core Viewpoint - Ionis Pharmaceuticals and Biogen have decided to terminate the development of their investigational ALS drug, BIIB105 / ION541, due to failure to show efficacy in slowing disease progression as indicated by the ALSpire study results [1][2]. Group 1: Drug Development and Study Results - The decision to halt BIIB105's development was based on topline data from the phase I/II ALSpire study, which showed no reduction in plasma neurofilament light chain (NfL) levels in ALS patients after six months of treatment [2]. - Patients treated with BIIB105 did not show improvements in clinical outcome measures related to function, breathing, and strength [2]. - Ionis intends to advance ION582, an experimental drug for Angelman Syndrome, independently after Biogen opted out of licensing rights for this drug [3][6]. Group 2: Market Performance and Company Impact - Following the announcement, shares of Ionis Pharmaceuticals and Biogen fell by at least 2% in market capitalization [1]. - Year-to-date, Biogen's shares have decreased by 11.1%, while Ionis has seen a more significant decline of 25.6% [3][6]. - Despite the setbacks, Ionis reported positive topline results from the HALOS study for ION582, indicating safety and improvements in cognitive, communication, and motor functions [5][6]. Group 3: Ongoing Collaborations and Future Prospects - Biogen and Ionis have a long-standing partnership that has previously yielded successful drugs like Spinraza and Qalsody for treating ALS [7][8]. - Both companies continue to develop investigational medicines for neurodegenerative diseases, including Alzheimer's and Parkinson's [8].

Core Insights - Ionis Pharmaceuticals announced positive topline data from the HALOS Phase 1/2a study of ION582 for Angelman syndrome, showing safety, tolerability, and significant functional improvements in cognition, communication, and motor function [1][2][5] Company Developments - Ionis will independently advance ION582 and plans to discuss the Phase 1/2a results with regulatory authorities to design a pivotal program [2][4] - Biogen has chosen not to exercise its option to license ION582, allowing Ionis to retain full control over the drug's development [2] Clinical Study Details - The HALOS trial included a three-month multiple-ascending dose study with 51 patients aged 2-50, followed by a long-term extension evaluating higher doses for an additional 12 months [4][8] - Topline results indicated that approximately 65% of patients showed cognitive improvements on the Bayley-4 assessment at six months, with similar improvements in communication and motor skills [6][14] Safety and Efficacy - ION582 was found to be safe and well tolerated across all dose levels, with adverse events consistent with patient medical histories [6][8] - The study demonstrated consistent improvements across various measures, including the Bayley-4 and SAS-CGI-C scales, correlating with positive changes in EEG activity [6][14] Future Plans - Detailed results from the HALOS study are expected to be presented at the upcoming Angelman Syndrome Foundation meeting in July [8] - Ionis is committed to advancing ION582 into a pivotal study, aiming to address the significant unmet need for therapies in Angelman syndrome [4][8]

Core Insights - Ionis Pharmaceuticals will hold its 2024 virtual Annual Meeting of Stockholders on June 6, 2024, followed by a corporate update [1] - The company has a strong pipeline in neurology, cardiology, and other high-need areas, with five marketed medicines [1] Company Overview - Ionis Pharmaceuticals has been innovating medicines for serious diseases for three decades [1] - The company is a pioneer in RNA-targeted medicines and is advancing new approaches in gene editing [1] - Ionis emphasizes a deep understanding of disease biology and industry-leading technology to drive innovation [1] Meeting Details - The virtual Annual Meeting will take place from 5:00 p.m. to 5:15 p.m. ET, with a corporate update at 5:30 p.m. ET [3] - Stockholders of record as of April 8, 2024, are invited to participate and will receive a proxy card for access [3] - A help line will be available for technical assistance during the live event, which will not have a replay [3]