Otsuka and Ionis Pharmaceuticals said a Phase 3 ALS study hit its primary endpoint, positioning the partners to discuss expedited regulatory pathways. The program uses the RNA-based candidate ulefnersen (licensed by Ionis to Otsuka), with the trial outcome framed as a key milestone for advancing discussions with the FDA. The companies’ next steps likely center on assembling the full regulatory package for accelerated review and clarifying the evidence needed for an expedited decision. For ALS, where therapeutic options remain limited, a Phase 3 primary endpoint win can materially shift expectations for near-term commercialization. The development also underscores continued progress for antisense/targeted RNA approaches in neurodegeneration, especially where survival endpoints and progression measures can drive regulatory leverage.