health28 days ago
Ulefnersen clears Phase 3 hurdle for rare FUS-ALS, paving way for FDA review
Otsuka and Ionis reported that ulefnersen met its primary endpoint in a Phase 3 trial for FUS-ALS, a rare genetic form of amyotrophic lateral sclerosis. The drug, an RNA-targeted therapy designed to lower FUS protein levels, showed significant improvements in functional impairment and survival compared to placebo. With positive results on secondary endpoints and a favorable safety profile, the partners plan to seek expedited regulatory approval. This marks the second antisense therapy to demonstrate efficacy in a genetic form of ALS, following tofersen for SOD1-ALS.
