Ionis Reports Positive Zilganersen Pivotal Study Results for Alexander Disease

Ionis Pharmaceuticals announced additional positive results from the pivotal study of zilganersen in children and adults with Alexander disease (AxD). The study met its primary endpoint, demonstrating statistically significant and clinically meaningful stabilization of gait speed in individuals aged 5 and older at Week 61. Furthermore, new data indicated that zilganersen may improve gross motor function in younger children aged 2-4 years. These findings build on previously reported topline data and will be presented at the 2026 American Academy of Neurology annual meeting.