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Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) is a leading biotechnology company headquartered in Carlsbad, California. Founded in 1989, Ionis has been pioneering the discovery and development of antisense drugs, with an impressive portfolio of over 1300 patents. Ionis focuses on creating innovative RNA-targeted therapies for complex diseases, particularly in the areas of cardiovascular, metabolic, neurological, and rare genetic disorders.
Ionis has successfully brought several groundbreaking medicines to market. Notable achievements include the development of Spinraza, in collaboration with Biogen, which was launched in 2016 to treat spinal muscular atrophy, a rare neuromuscular disorder. In 2023, Biogen also launched Qalsody for ALS, further extending the impact of Ionis' antisense technology.
Through its cardiovascular-focused subsidiary, Akcea Therapeutics, Ionis has introduced drugs like Tegsedi for ATTR amyloidosis and Waylivra in Europe for cardiology indications. The company continues to advance its pipeline with promising candidates such as olezarsen, an investigational RNA-targeted therapy for familial chylomicronemia syndrome (FCS). Ionis' ongoing Phase 3 trials for olezarsen have shown promising results, with significant reductions in triglyceride levels and improvements in related health outcomes.
Ionis' commitment to innovation and patient care is also evident in its robust financial health and strategic partnerships. With a deep understanding of disease biology and cutting-edge technology, Ionis continues to drive advancements in RNA therapies and gene editing, aiming to deliver life-changing medicines for patients around the world.
Recently, Ionis announced positive clinical trial results for several of its investigational drugs and plans to pursue regulatory approvals for these therapies. The company's pipeline is well-positioned to address unmet medical needs in various therapeutic areas, promising a steady flow of new treatments in the coming years.
For more detailed information about Ionis Pharmaceuticals, its products, and career opportunities, visit their official website at www.ionispharma.com.
Ionis Pharmaceuticals (Nasdaq: IONS) announced positive results from the Phase 1/2 HALOS study of ION582 in people with Angelman syndrome (AS). The study showed consistent clinical improvement in communication, cognition, and motor function. Key findings include:
- 97% of patients in medium and high dose groups showed improvement in overall AS symptoms
- Improvements exceeded those observed in natural history studies
- ION582 demonstrated favorable safety and tolerability at all dose levels
Ionis plans to initiate Phase 3 development in H1 2025. The company views ION582 as a potential cornerstone of its next wave of transformational medicines for neurological conditions.
Ionis Pharmaceuticals (Nasdaq: IONS) has announced a live webcast scheduled for Thursday, August 1st at 11:30 a.m. Eastern Time to discuss its second quarter 2024 financial results. Interested parties can access the webcast through the company's investor relations website at https://ir.ionis.com/events-and-presentations/upcoming-events. A replay of the webcast will be available for a time at the same address. This event provides an opportunity for investors and analysts to gain insights into Ionis' financial performance and potentially its future outlook.
Ionis Pharmaceuticals (Nasdaq: IONS) has completed enrollment in the pivotal trial of zilganersen (ION373), an investigational RNA-targeted medicine for Alexander disease (AxD). This rare, progressive neurological disorder affects an estimated one in one million people in the U.S. The trial's primary endpoint is percent change from baseline in gait speed, assessed by the 10-Meter Walk Test. Topline data are expected in the second half of 2025.
Zilganersen aims to address the underlying cause of AxD by targeting excess GFAP accumulation resulting from genetic variants in the GFAP gene. This approach could potentially slow or stabilize disease progression in AxD patients, who currently lack treatments that address the root cause of their condition.
Ionis Pharmaceuticals (NASDAQ: IONS) has announced a live webcast scheduled for Monday, July 22, at 8:00 a.m. Eastern Time. This webcast will discuss the results from the Phase 1/2a HALOS study of ION582, a treatment for Angelman syndrome. The findings will also be presented at the 2024 Angelman Syndrome Foundation (ASF) Family Conference and Research Symposium on Wednesday, July 24, in Sandusky, Ohio. Interested parties can access the webcast via the provided link, with a replay available for a time.
Ionis Pharmaceuticals announced the FDA's acceptance of its New Drug Application (NDA) for olezarsen, an investigational RNA-targeted medicine for treating familial chylomicronemia syndrome (FCS). The FDA has granted Priority Review with a PDUFA date set for December 19, 2024. Olezarsen has also received Fast Track, Orphan Drug, and Breakthrough Therapy designations.
Additionally, Ionis completed Phase 3 enrollment for three studies (CORE, CORE2, and ESSENCE) evaluating olezarsen for severe hypertriglyceridemia (sHTG), with results anticipated in the second half of 2025. These studies involve over 2,400 patients and aim to address the life-threatening complications associated with sHTG.
The NDA is supported by positive Phase 3 study results presented at the 2024 ACC Annual Meeting and published in the New England Journal of Medicine.
Ionis Pharmaceuticals has entered an expanded licensing agreement with Otsuka Pharmaceutical for the investigational RNA-targeted medicine donidalorsen, aimed at treating hereditary angioedema (HAE).
Under the agreement, Otsuka gains exclusive rights to commercialize donidalorsen in the Asia-Pacific region while also handling regulatory filings and localized development. Ionis will continue to manage global development and plans to submit a New Drug Application to the FDA this year for U.S. approval, aiming for an independent U.S. launch.
Previously, Otsuka secured licensing for Europe and is preparing a Marketing Authorization Application to the EMA this year. Ionis will receive a $20 million upfront payment and milestone payments from Otsuka, along with tiered royalties.
Recent positive results from Phase 3 trials were presented at the EAACI Annual Congress and published in The New England Journal of Medicine.
Ionis Pharmaceuticals announced that its management will participate in a fireside chat at the TD Cowen Genetic Medicines & RNA Summit on June 20, 2024. The event will be virtual, and a live webcast will be available on the Ionis website. Interested parties can access the webcast on the Investors & Media section of the website, with a replay available within 48 hours for a time.
Ionis Pharmaceuticals revealed positive Phase 3 results for donidalorsen in treating hereditary angioedema (HAE). The OASIS-HAE and OASISplus studies showed significant reductions in mean monthly HAE attack rates, with a >90% reduction over one year. Patients switching from other treatments saw a 62% further reduction, and 84% preferred donidalorsen. Favorable safety and tolerability were reported across all cohorts. Ionis plans to seek regulatory approval based on these results. These findings will be presented at the EAACI Congress 2024, and Ionis aims to launch donidalorsen as part of its independent commercial pipeline.
Ionis Pharmaceuticals (NASDAQ: IONS) announced a live webcast scheduled for May 31, 2024, at 8:00 a.m. Eastern Time to discuss the donidalorsen Phase 3 OASIS-HAE study results. These results will be presented during three late-breaking oral presentations at the 2024 EAACI Annual Meeting in Valencia, Spain. The webcast and a -time replay can be accessed at the Ionis website.
Ionis Pharmaceuticals (Nasdaq: IONS) will present new subgroup analyses from its Phase 3 NEURO-TTRansform study of WAINUA™ (eplontersen) at the 2024 International Symposium on Amyloidosis (ISA). The study highlights consistent benefits in neuropathy impairment and quality of life across various patient segments. WAINUA, approved by the FDA in December 2023 for treating hATTR-PN, will have several notable presentations on May 29, 2024. Additionally, eplontersen is being evaluated in the Phase 3 CARDIO-TTRansform study for transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This study, with over 1,400 patients, is the largest in this patient population.
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