Ulefnersen Meets FUS ALS Phase Three Endpoint

Company Ionis / Otsuka Event Type Clinical Trial Data Readout (Source Gap Catch-Up) Modality Antisense Oligonucleotide Asset Ulefnersen (ION363) Target FUS pre-mRNA Disease Area FUS-Mutated Amyotrophic Lateral Sclerosis Summary Ionis and Otsuka announced on 22 September that intrathecal ulefnersen met the prespecified primary endpoint in…