Ionis Pharmaceuticals completes enrollment in trial of zilganers in AxD

Ionis Pharmaceuticals has completed enrollment in the pivotal trial of zilganersen, an investigational RNA-targeted medicine in development for the treatment of children and adults with a rare, progressive and ultimately fatal neurological disorder known as Alexander disease, or AxD. The primary endpoint is percent change from baseline in gait speed as assessed by the 10-Meter Walk Test. Topline data are anticipated in the second half of 2025. AxD is estimated to occur in an estimated one in one million people in the U.S. and can present throughout life.Zilganersen is designed to stop the excess GFAP that accumulates because of disease-causing variants in the GFAP gene, with the goal of slowing or stabilizing disease progression in people living with AxD.