Ionis, Otsuka ALS Drug Meets Main Goal in Phase 3 Trial, Improving Survival

09:03 AM EDT, 09/22/2026 (MT Newswires) -- Ionis Pharmaceuticals and Otsuka Pharmaceutical said Tuesday that their late-stage study of ulefnersen successfully met its primary endpoint, significantly improving survival and function in patients with a rare form of amyotrophic lateral sclerosis, or ALS. The trial also met key secondary goals, significantly lowering a key nerve damage marker and delaying death or the need for permanent ventilation, the companies said. Ionis and Otsuka plan to discuss the results with the US Food and Drug Administration and other health authorities regarding potential expedited regulatory pathways for drug approval. Shares of Ionis Pharmaceuticals were 1.7% higher in Tuesday premarket activity.