, /PRNewswire/ -- (Nasdaq: ) 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.
AxD is estimated to occur in an estimated one in one million people in the U.S. and can present throughout life.
The disease is a result of genetic variants in the glial fibrillary acidic protein ( ) gene that disrupt the structure and function of astrocytes in the brain. AxD is generally characterized by cognitive dysfunction and progressive neurologic deterioration, including loss of independence and the ability to control muscles for large movements, swallowing and airway protection. Zilganersen is designed to stop the excess GFAP that accumulates because of disease-causing variants in the gene, with the goal of slowing or stabilizing disease progression in people living with AxD.
"Current approaches to disease management for Alexander disease can mitigate some symptoms of AxD but do not address the underlying cause or slow disease progression. Our zilganersen study is the first trial to evaluate an investigational medicine designed to address the underlying cause of Alexander disease," s.
