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Ionis Pharmaceuticals (IONS) enters agreement with Roche for two novel RNA-targeted programs for Alzheimer's disease and Huntington's disease

September 27, 2023 7:00 AM EDT

Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced that it has entered an agreement with Roche for two undisclosed early-stage programs for RNA-targeting investigational medicines for the treatment of Alzheimer's disease (AD) and Huntington's disease (HD). Roche gains exclusive worldwide rights and will be responsible for clinical development, manufacturing, and commercialization of the medicines if they receive regulatory approval. The companies will leverage Ionis' expertise discovering medicines that target the root cause of central nervous system diseases and Roche's global experience developing and commercializing therapies for nervous system disorders.

"We are excited to expand our partnership with Roche, a global leader in developing and delivering innovative treatments to patients," said Brett P. Monia, Ph.D., Ionis' chief executive officer. "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, a leader in RNA-targeted therapeutics, is a great example of two collaborators mutually benefiting from their relationship by complementing and learning from each other," said James Sabry, Ph.D., global head of Roche Pharma partnering. "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."

Under terms of the agreement, Ionis will have responsibility to advance the two programs through pre-clinical studies; Roche will have sole responsibility for clinical development and, if approved, commercialization worldwide. Ionis will receive a $60 million upfront payment from Roche and is eligible to receive development, regulatory and commercial milestone payments, and tiered royalties.

Ionis and Roche have been strong partners for the past 10 years, starting with their collaboration in 2013 for tominersen, an Ionis-discovered investigational medicine for HD. Tominersen, designed to reduce the production of all forms of the huntingtin protein, is currently being evaluated by Roche in a Phase 2 proof of concept study (GENERATION HD2) assessing safety, biomarker and efficacy trends in people with prodromal or early manifest HD. In addition to tominersen, both companies are working together on a pre-clinical stage mutant HTT selective antisense oligonucleotide for HD. The companies entered another collaboration in 2018 to develop IONIS-FB-LRx targeting Factor B for the treatment of IgA nephropathy (IgAN) and geographic atrophy (GA). IONIS-FB-LRx is currently in a Phase 2 study for GA, led by Ionis, and a Phase 3 study for IgAN led by Roche.



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