CARLSBAD, Calif., July 19, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it will host a live webcast on Thursday, August 1st at 11:30 a.m. Eastern Time to discuss its second quarter 2024 financial results. About Ionis Pharmaceuticals, Inc. Ionis Pharmaceuticals Investor Contact: D. Wade Walke, Ph.D. – [email protected] – 760-603-2331 SOURCE Ionis Pharmaceuticals, Inc. Webcast scheduled for Thursday, August 1 at 11:30 a.m. Eastern Time The webcast may be accessed at htt ...

Zilganersen is the first investigational medicine in clinical development for people with Alexander disease, a rare, life-threatening neurological condition AxD is estimated to occur in an estimated one in one million people in the U.S. and can present throughout life.1-3 The disease is a result of genetic variants in the glial fibrillary acidic protein (GFAP) gene that disrupt the structure and function of astrocytes in the brain. AxD is generally characterized by cognitive dysfunction and progressive neur ...

CARLSBAD, Calif., July 8, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) announced today that it will host a live webcast on Monday, July 22nd at 8:00 a.m. Eastern Time to discuss results from the Phase 1/2a HALOS study of ION582 for the treatment of people with Angelman syndrome. Results from the HALOS study will also be presented on Wednesday, July 24 at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium in Sandusky, Ohio. About Ionis Pharmaceuticals, Inc ...

Core Insights - Ionis Pharmaceuticals is advancing its investigational RNA-targeted therapy olezarsen for the treatment of familial chylomicronemia syndrome (FCS), with the FDA accepting its new drug application (NDA) [14] - The company has reported positive data from phase III studies for donidalorsen, showing significant reductions in monthly HAE attack rates [2] - Ionis has established partnerships with major pharmaceutical companies like Biogen, AstraZeneca, and Novartis for various drug candidates, enhancing its pipeline development [3][17] Pipeline Developments - Olezarsen is set to be Ionis' first independent product launch and the first FDA-approved treatment for FCS if approved [1] - The NDA for olezarsen is supported by results from the phase III BALANCE study, demonstrating significant triglyceride-lowering effects and reduced acute pancreatitis attacks in FCS patients [6] - Ionis has completed enrollment in three late-stage studies for olezarsen targeting severe hypertriglyceridemia (sHTG), with data expected in the second half of 2025 [5] Regulatory Progress - The FDA has granted priority review to the NDA for olezarsen, which shortens the review period by four months, with a decision anticipated by December 19, 2024 [10] - Ionis has received fast-track, orphan drug, and breakthrough therapy designations for olezarsen, which may provide up to seven years of market exclusivity post-approval [11] Financial Performance - Year-to-date, Ionis' stock has decreased by 7.1%, slightly better than the industry average decline of 7.8% [7] - The company currently holds a Zacks Rank 3 (Hold), indicating a neutral outlook [4]

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]