Roche to Develop Two Novel RNA-targeted Programs from Ionis for CNS Diseases

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Roche will gain exclusive worldwide rights to develop, manufacture, and commercialize Ionis’ investigational RNA-based therapeutic candidates for Alzheimer's and Huntington's disease.

Roche has entered into an agreement with Ionis Pharmaceuticals for two undisclosed early stage programs for RNA-targeting investigational therapeutics for treating Alzheimer's disease (AD) and Huntington's disease (HD). According to a Sept. 27, 2023 company press release, Roche gains exclusive worldwide rights and will be responsible for clinical development, manufacturing, and commercialization of the candidates worldwide, the latter of which is dependent upon regulatory approval.

Under the agreement, Ionis' expertise in discovering medicines that target the root cause of central nervous system diseases will work in tandem with Roche's global experience developing and commercializing therapies for nervous system disorders. Ionis will receive a $60 million upfront payment and will be eligible for development, regulatory, and commercial milestone payments as well as tiered royalties. Ionis will be responsible for advancing the two programs through pre-clinical studies, after which Roche will take over. Roche will have sole responsibility for clinical development and, if approved, commercialization worldwide.

This agreement builds upon a 10-year-long partnership between the companies, which started in 2013. In 2018, the companies entered another collaboration to develop an Ionis therapeutic candidate for immunoglobulin A nephropathy and geographic atrophy treatment.

"We are excited to expand our partnership with Roche … ," said Brett P. Monia, CEO, Ionis, in the company press release. "With this new collaboration, we are joining forces to accelerate the discovery and development of novel therapies for people living with Alzheimer's disease and Huntington's disease globally. Collaborating on these two programs enables Ionis to advance our wholly owned programs, including those in neurology, aligned with our strategic priorities."


"Our lasting partnership with Ionis … is a great example of two collaborators mutually benefiting from their relationship by complementing and learning from each other," said James Sabry, global head of Roche Pharma partnering, in the press release. "By expanding our alliance, we bring together the companies' combined knowledge of the science in Alzheimer's disease and Huntington's disease with Roche's proven capabilities in the development and commercialization of innovative treatments in neuroscience."

Source: Ionis Pharmaceuticals