Ionis completes enrollment in pivotal trial evaluating zilganersen in people living with Alexander disease
2024-07-18 · Ionis Pharmaceuticals · original ionis.com ↗
Zilganersen is the first investigational medicine in clinical development for people with Alexander disease, a rare, life-threatening neurological condition CARLSBAD, Calif., July 18, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that it has completed enrollment in the pivotal trial of zilganersen (ION373), 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 (AxD). The primary endpoint is percent change from baseline in gait speed as assessed by the 10-Meter Walk Test (10MWT). Topline data are anticipated in the second half of 2025.
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