Image Source: Zacks Investment Research Olezarsen is one of Ionis' wholly-owned pipeline candidates, which it intends to launch independently in the United States. The drug's commercial launch is part of management's broad strategy to deliver a steady flow of wholly-owned medicines to patients. If approved, the drug will not only be Ionis' first independent product launch but also the first FDA-approved treatment for FCS. Last month, Ionis reported positive data from two phase III studies — OASIS-HAE and OA ...

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 ...

Otsuka will be responsible for commercialization efforts for donidalorsen across both Asia Pacific and Europe Ionis plans to independently bring donidalorsen to U.S. patients, if approved CARLSBAD, Calif., June 18, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered into a license agreement with Otsuka Pharmaceutical Co., Ltd. (Otsuka) under which Otsuka obtains exclusive rights across the Asia-Pacific region for donidalorsen, an investigational RNAtargeted pr ...

CARLSBAD, Calif., June 17, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that management will participate in a fireside chat at the virtual TD Cowen Genetic Medicines & RNA Summit on Thursday, June 20, 2024. A live webcast of this presentation can be accessed on the Investors & Media section of the Ionis website at https://ir.ionis.com/events-and-presentations/upcoming-events. A replay will be available on the Ionis website within 48 hours and will be archived for a limited ...

"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 ...

Webcast scheduled for Friday, May 31 at 8:00 a.m. Eastern Time CARLSBAD, Calif., May 28, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Friday, May 31th at 8:00 a.m. Eastern Time to discuss the donidalorsen Phase 3 OASIS-HAE and OASISplus (Open-Label Extension and Switch cohorts) study results that will be presented in three late-breaking oral presentations at the 2024 European Academy of Allergy and Clinical Immunology (EAACI) Annual Meet ...

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].

Part 1 of the HALOS trial was a three-month, multiple-ascending dose (MAD) study in 51 patients aged 2-50, which evaluated three doses of ION582. All eligible patients transitioned into the Part 2 long-term extension (LTE) portion of the study, which is evaluating the two higher doses of ION582 for an additional 12 months. Part 3 of the study will evaluate eligible patients for an additional three years. Topline results were available for all patients at four months (one month after last MAD dose), and six ...