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

• Ionis reported positive topline Phase 3 results for ulefnersen in FUS-ALS, supporting a potential first disease-modifying genetic treatment.
• The study showed better outcomes on a combined measure of function and survival versus placebo, supporting a disease-progression impact.
• Safety profile looked favorable, with most adverse events described as mild to moderate.
• Detailed trial results will be presented at a future medical congress, with a peer-reviewed publication planned in coming months.
• Otsuka plans to seek expedited regulatory submission pathways, starting with discussions with the FDA and other global regulators.

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. Ionis Pharmaceuticals Inc. published the original content used to generate this news brief via Business Wire (Ref. ID: 202609220800BIZWIRE_USPR_____20260922_BW427197) on September 22, 2026, and is solely responsible for the information contained therein.