Ionis Pharmaceuticals reported positive pivotal Phase 3 trial results for zilganersen, an antisense oligonucleotide aimed at treating Alexander disease, a rare neurogenetic disorder. The drug demonstrated significant improvement in gait speed over control, meeting its primary endpoint. The data support a planned NDA submission slated for early 2026. This marks the first clinical-stage drug candidate specifically targeting this often fatal disease and offers a new therapeutic hope for patients.