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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 that Roche is initiating a new Phase 2 trial to assess tominersen for Huntington's disease (HD) after halting the Phase 3 GENERATION HD1 study. Post-hoc analyses indicate potential benefits of tominersen for younger adult patients with lower disease burden. The trial's design will be revealed in future presentations starting January 20, 2022. Tominersen aims to reduce huntingtin protein levels, addressing the underlying cause of HD. This development is seen as a positive step for the HD community, according to Ionis executives.
Ionis Pharmaceuticals announced that CEO Brett P. Monia, Ph.D., will provide a company overview at the virtual 40th Annual J.P. Morgan Healthcare Conference on January 12, 2022, at 9:45 a.m. ET. A live webcast will be accessible via the Investors & Media section of their website, with a replay available within 48 hours. Ionis has over 30 years of leadership in RNA-targeted therapy and boasts a strong late-stage pipeline focusing on neurological and cardiometabolic treatments.
Biogen and Ionis Pharmaceuticals announced a strategic collaboration whereby Biogen exercised its option to acquire a worldwide, exclusive license to develop and commercialize BIIB115/ION306, an investigational antisense oligonucleotide targeting spinal muscular atrophy (SMA). This initiative aims to address unmet patient needs and is supported by a $60 million upfront payment from Biogen. Plans include advancing BIIB115 into clinical trials to evaluate its safety and efficacy, with Biogen handling all development costs.
Ionis Pharmaceuticals, a leader in RNA-targeted therapies, has finalized a collaboration agreement with AstraZeneca for the development and commercialization of eplontersen, an investigational treatment for transthyretin amyloidosis (ATTR). The agreement follows the end of the antitrust review period and includes an upfront payment of $200 million, along with potential milestone payments totaling up to $3.4 billion. Ionis will receive royalties ranging from low double-digit to mid-20s percentages based on region.
Ionis Pharmaceuticals has announced a strategic collaboration with AstraZeneca to develop and commercialize eplontersen, an investigational treatment for transthyretin amyloidosis (ATTR). The agreement includes a $200 million upfront payment, potential milestone payments of up to $485 million, and sales-related milestones up to $2.9 billion. Ionis will lead Phase 3 trials for hereditary ATTR and will manufacture eplontersen, while AstraZeneca will handle commercial supply outside the U.S. The collaboration aims to enhance patient access and shareholder value as they prepare for a potential NDA submission by the end of 2022.
Ionis Pharmaceuticals will host a virtual investor day on December 9, 2021, at 11:00 a.m. Eastern Time. The session aims to outline the company's strategy, advancements, and progress in commercializing its leading programs. It will feature discussions on key technological innovations and is expected to last around 2.5 hours. Interested investors can access the webcast through Ionis's official website, with a limited-time replay available.
Ionis has pioneered RNA-targeted therapies for over 30 years, with three marketed medicines and a strong late-stage pipeline.
Ionis Pharmaceuticals announced promising results from Pfizer's Phase 2b study of vupanorsen, showing statistically significant reductions in non-HDL cholesterol and triglycerides across all doses. This investigational therapy aims to reduce cardiovascular risks and severe hypertriglyceridemia. The study involved 286 participants and achieved its primary endpoint at 24 weeks. Despite some injection site reactions and increases in liver enzymes, there were no serious adverse events related to treatment. Full results are expected to be presented at a medical meeting next year.
Ionis Pharmaceuticals (NASDAQ: IONS) has announced the initiation of the Phase 3 OASIS-HAE study for donidalorsen, an investigational antisense drug targeting hereditary angioedema (HAE). This registrational trial aims to evaluate the efficacy of donidalorsen in reducing HAE attacks, with a participation of up to 84 patients. Positive Phase 2 data showed that donidalorsen was safe, well-tolerated, and resulted in a significant reduction of moderate to severe attacks. The company emphasizes its commitment to providing transformative treatments to the HAE patient community.
Ionis Pharmaceuticals (NASDAQ: IONS) announced presentations of its cardiovascular pipeline at the AHA Scientific Sessions from Nov. 13-15, 2021. Data from a Phase 1 study of ION449, aimed at lowering LDL-C levels, will feature prominently. ION449 is being developed in collaboration with AstraZeneca. Additionally, olezarsen, which targets triglyceride levels, will be discussed, with evaluations ongoing in two Phase 3 studies. A symposium on the prevalence of ATTR-CM in African American populations will also take place, highlighting health disparities.
Ionis Pharmaceuticals (Nasdaq: IONS) announced its participation in two upcoming virtual investor conferences. The events include the Stifel 2021 Healthcare Conference on November 15, 2021, and the Evercore ISI 4th Annual HealthCONx Conference on November 30, 2021. Details regarding presentation times may change, and updates will be available on their website. A live webcast of the presentations will be accessible in the Investors & Media section of Ionis' site, with replays available within 48 hours. For over 30 years, Ionis has led in RNA-targeted therapy, emphasizing their commitment to innovative treatments.
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