Amylyx Pharmaceuticals, Inc. (NASDAQ:AMLX) ("Amylyx" or the "Company") today 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 (70–180 mg/dL), 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.