CARLSBAD, Calif., Jan. 18, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that its partner, Roche, is designing a new Phase 2 trial to evaluate tominersen in Huntington's disease (HD). After halting dosing in the Phase 3 GENERATION HD1 study, exploratory post-hoc analyses suggest tominersen may benefit younger adult patients with lower disease burden. These results require confirmation in a randomized, placebo-controlled study.
An experimental drug developed by Ionis Pharmaceuticals and Roche could offer a way forward to treat Huntington's disease, nearly three decades after researchers first pinpointed the gene behind the hereditary neurodegenerative disorder.