Amylyx announces results from Phase 2 HELIOS trial of AMX0035

Amylyx (AMLX) Pharmaceuticals announced positive Week 48 data from the Phase 2 open-label HELIOS clinical trial of AMX0035 in adults living with Wolfram syndrome. These results were presented at the Joint Congress of the European Society for Pediatric Endocrinology, ESPE, and the European Society of Endocrinology, ESE, in Copenhagen, Denmark. Consistent with the HELIOS trial’s previously presented primary efficacy outcome of improvement in pancreatic function, as measured by C-peptide response to a mixed-meal tolerance test at Week 24, treatment with AMX0035 through Week 48 demonstrated continued and sustained improvement in pancreatic beta cell function. Treatment with AMX0035 from Week 24 to Week 48 also showed sustained improvements or stabilization in glycemic control, as measured by hemoglobin A1c and time in target glucose range assessed by continuous glucose monitoring, as well as visual acuity. All participants with available measurements met the responder criteria, defined as either improvement or no change, on both the Patient Global Impression of Change and Clinician Global Impression of Chang at Weeks 24 and 48, indicating stability or improvement in their Wolfram syndrome-related symptoms.