Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) released topline results on Monday from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive, and often fatal neurological condition.

The neurological disorder destroys the myelin sheath protecting nerve fibers, caused by a mutation in the GFAP gene.

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) with favorable safety ...