Amylyx Pharmaceuticals Publishes Phase 2 Trial Data for AMX0035 in Wolfram Syndrome
Amylyx Pharmaceuticals has announced the publication of results from its Phase 2 open-label HELIOS trial in The Journal of Clinical Investigation. The trial evaluated AMX0035, a combination of sodium phenylbutyrate and taurursodiol, in adults with Wolfram syndrome. The study showed improvements in pancreatic beta cell function and glycemic control over 48 weeks, with stabilization in visual acuity. The trial's findings suggest AMX0035's potential to alter the progression of Wolfram syndrome, a rare genetic disorder characterized by diabetes and neurodegeneration. The drug was generally well-tolerated, with no serious adverse events reported. 
