Ionis Pharmaceuticals, Inc. develops RNA-targeted medicines and reports news around marketed medicines and a pipeline in neurology, cardiometabolic disease and other high-need areas. Company updates commonly address commercial performance for TRYNGOLZA, financial results, R&D revenue from partnerships, and progress across antisense oligonucleotide programs.
Clinical and regulatory news also covers programs such as olezarsen for severe hypertriglyceridemia, zilganersen for Alexander disease, and partner-developed bepirovirsen for chronic hepatitis B. Governance and investor-relations releases include annual meeting materials, board composition changes, and healthcare conference presentations.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) will host a live webcast on February 24 at 11:30 a.m. ET to discuss its fourth quarter and full year 2021 financial results. The webcast will also cover the company's pipeline and business progress. Access the webcast and replay through the company's investor relations page. Ionis is a leader in RNA-targeted therapy, offering three marketed medicines and a robust late-stage pipeline focused on neurological and cardiometabolic diseases.
Pfizer and Ionis Pharmaceuticals announced the termination of the clinical development program for vupanorsen (PF-07285557), an investigational antisense therapy aimed at cardiovascular risk reduction and severe hypertriglyceridemia. Despite meeting its primary endpoint of reducing non-HDL cholesterol and triglycerides in a Phase 2b study, the results were insufficient to justify further development. Additionally, vupanorsen was linked to dose-dependent liver fat increases and elevated liver enzymes. Pfizer will return development rights to Ionis, which licensed vupanorsen in November 2019.
Ionis Pharmaceuticals has received orphan drug designation from the FDA for eplontersen, an investigational antisense therapy aimed at treating transthyretin-mediated amyloidosis. This designation provides Ionis with key incentives, including tax credits and seven years of market exclusivity after approval. Eplontersen, currently in Phase 3 trials, targets both hereditary and non-hereditary forms of the disease, which can lead to severe complications such as heart failure and nerve damage. The company's collaboration with AstraZeneca further supports the development of this promising treatment.
Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) has appointed Joseph T. Baroldi as executive vice president and chief business officer, effective January 31, 2022, and Eric P. Bastings, M.D., as vice president of development strategy. Baroldi, with over 20 years of experience, previously served as COO at Avidity Biosciences and held various roles at Ionis. Dr. Bastings brings over 20 years of regulatory experience from the FDA. The appointments aim to enhance business development and clinical leadership, crucial for advancing Ionis' innovative neurology pipeline.
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.