Otsuka says Phase 3 FUSION trial shows ulefnersen improves function and survival in FUS-ALS

• Otsuka reported positive topline Phase 3 results for ulefnersen in FUS-ALS, a rare genetic form of amyotrophic lateral sclerosis.
• The trial met its main goal, showing better overall outcomes than placebo on a combined measure of function and survival.
• Secondary findings supported a potential disease-modifying effect, reinforcing the case for a targeted treatment in this patient subset.
• Safety looked favorable, with most adverse events described as mild or moderate.
• Detailed results will be presented at a future medical congress, ahead of planned regulatory discussions aimed at expedited submission pathways.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. Otsuka Holdings Co. Ltd. published the original content used to generate this news brief via Business Wire (Ref. ID: 202609220800BIZWIRE_USPR_____20260922_BW352423) on September 22, 2026, and is solely responsible for the information contained therein.