Roche, Ionis forge new pact targeting Alzheimer's, Huntington's diseases

27 Sep 2023
Phase 2OligonucleotidePhase 3License out/in
Roche entered an agreement with Ionis Pharmaceuticals centred on two undisclosed early-stage programmes for RNA treatments targeting Alzheimer's disease and Huntington's disease. As part of the deal announced Wednesday, Ionis will receive a $60-million upfront payment, and also stands to gain unspecified development, regulatory and commercial milestones, plus tiered royalties.
"Our lasting partnership with Ionis…is a great example of two collaborators mutually benefiting from their relationship by complementing and learning from each other," remarked James Sabry, global head of Roche Pharma partnering. The Swiss drugmaker has a long-standing partnership with Ionis on tominersen, which Roche last year decided to continue to advance in Huntington's disease despite work being halted in 2021 after the experimental antisense oligonucleotide failed to show evidence of clinical benefit in a Phase III trial.
Besides tominersen, the companies are working together on a preclinical mutant HTT selective antisense oligonucleotide for Huntington's. They entered another collaboration in 2018 to develop IONIS-FB-LRx targeting Factor B for the treatment of IgA nephropathy and geographic atrophy (GA). That compound is currently in a Phase II study for GA, led by Ionis, and a Phase III study for IgA nephropathy, led by Roche.
Meanwhile, Roche's earlier foray into Alzheimer's was marked by the notable failure of the anti-amyloid antibody gantenerumab last November in two trials from the GRADUATE programme.
"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 commercialisation of innovative treatments in neuroscience," Sabry said Wednesday. As part of the new deal, Ionis will have responsibility to advance the two programmes through preclinical testing, while Roche gains exclusive worldwide rights and will assume clinical development, manufacturing and commercialisation of the medicines if they obtain regulatory approval.
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