Ionis reports positive pivotal study results for zilganersen in Alexander disease; NDA planned Q1 2026

IONIS PHARMACEUTICALS INC

Primary endpoint met: gait speed improved 33.3% vs control (p=0.0412) at week 61 in 50 mg cohort.
No approved disease-modifying treatments for Alexander disease; first investigational drug to show positive impact.
Safety profile favorable: most adverse events mild/moderate; SAEs numerically lower in zilganersen arm.
NDA submission to FDA targeted for Q1 2026; evaluating U.S. Expanded Access Program.

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