Otsuka/Ionis ALS drug clears Phase 3 primary endpoint, eyes expedited review
A Phase 3 trial of ulefnersen, the RNA-targeted ALS drug from Ionis and Otsuka, met its primary endpoint in the rare FUS-ALS form, with 73 of 89 enrolled patients in the primary analysis showing improved outcomes on functional and survival measures and favorable safety. Secondary endpoints and a biomarker signal also favored the drug, supporting discussions with regulators about expedited approval. The rarity of FUS-ALS limits market opportunity; Ionis will collect royalties on sales after a $10 million upfront license, with analysts forecasting modest long-term revenue compared with other Ionis programs.