Otsuka Pharmaceutical and Ionis Pharmaceuticals reported positive Phase III results for ulefnersen in the Fusion study for FUS-associated amyotrophic lateral sclerosis, a rare ALS subtype with no approved targeted therapies. The companies said the trial met key statistical targets, including improvements in function and survival measures, positioning ulefnersen for potential expedited regulatory discussions. Ulefnersen is an antisense oligonucleotide designed to address the underlying genetic driver in this patient population. With the Phase III readout now in hand, Otsuka and Ionis appear focused on translating efficacy signals into a regulatory path built around an unmet-need genomics segment of the ALS landscape.