Amylyx Publishes AMX0035 Clinical Trial Results

Amylyx Pharmaceuticals announced that Week 24 and Week 48 results from the Phase 2 open-label HELIOS clinical trial of AMX0035, an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol, in adults living with Wolfram syndrome have been published in The Journal of Clinical Investigation, a peer-reviewed medical journal. The publication reports Week 24 and Week 48 results from the Phase 2 open-label HELIOS trial previously presented in part at medical meetings. Consistent with earlier disclosures, the findings indicate continued improvements observed in pancreatic beta cell function through Week 48, as measured by C-peptide response to a mixed-meal tolerance test. Additionally, secondary measures of glycemic control, including HbA1c and time in target glucose range, demonstrated improvement from baseline at Weeks 24 and 48, while best-corrected visual acuity trended toward stabilization over the 48-week period. Participant and Clinician Global Impression of Change assessments also classified participants with available data as responders, defined by improvement or disease stabilization. The peer-reviewed publication also details qualitative interviews that reinforced the clinical relevance of these objective outcomes, with participants indicating meaningful improvements in diabetes and vision problems. Furthermore, the published safety findings remained consistent with prior clinical experience with AMX0035, with reported adverse events being mild or moderate and no serious adverse events related to treatment. HELIOS is a single-site, single-arm, open-label, Phase 2 clinical trial investigating AMX0035 in 12 adults living with Wolfram syndrome for up to 208 weeks followed by a four-week safety follow-up. The Company expects to present additional longer-term data from HELIOS at an upcoming scientific meeting.