Otsuka Pharmaceutical and Ionis Pharmaceuticals said their experimental drug ulefnersen met the main goal of a late-stage trial in patients with FUS-ALS, a rare inherited form of amyotrophic lateral sclerosis. The therapy improved function and survival compared with placebo in the reported study. Most adverse effects were described as mild or moderate, with a favorable safety profile. The companies plan to discuss the results with the U.S. FDA and other regulators while exploring possible accelerated-approval pathways.
Source: https://www.reuters.com/business/healthcare-pharmaceuticals/otsuka-ionis-experimental-als-drug-meets-main-goal-late-stage-study-2026-09-22/