Ulefnersen Shows Positive Phase 3 Signal in FUS-ALS

Ionis reports positive topline results from the randomized, placebo-controlled phase 3 FUSION study of genetically targeted antisense therapy ulefnersen in rapidly progressive FUS-ALS, including functional/survival, neurofilament, and survival signals. The primary population was only 73 patients, so full results, peer review, regulatory assessment, durability, and safety are still needed.