On September 22, 2025, Ionis Pharmaceuticals, Inc. announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD). AxD is a rare, progressive, and often fatal neurological condition with no approved disease-modifying treatments.
Zilganersen 50 mg demonstrated statistically significant and clinically meaningful stabilization on the primary endpoint of gait speed, as assessed by the 10-Meter Walk Test (10MWT), compared to control at week 61 (mean difference 33.3%, p=0.0412). The drug also showed consistent favorable trends across key secondary endpoints, indicating slowed disease progression, stabilization, or improvement.
These unprecedented results highlight zilganersen's potential to transform the future treatment landscape for AxD by directly targeting the underlying cause of the disease. Ionis plans to submit a new drug application (NDA) to the U.S. Food and Drug Administration in Q1 2026, offering long-awaited hope for patients and families.
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