Amylyx Pharmaceuticals Announces Peer-Reviewed Publication of Phase 2 Open-Label HELIOS Trial Data for AMX0035 in The Journal of Clinical Investigation

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CAMBRIDGE, Mass., May 15, 2026--(BUSINESS WIRE)--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. "Wolfram syndrome is a rare genetic disease that presents significant challenges for patients and their families. It typically begins in childhood, with insulin-requiring diabetes and progressive optic nerve changes affecting vision, and over time can involve broader neurological symptoms that increasingly affect daily life, with no disease-modifying treatments approved today," said Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS trial and the Samuel E. Schechter Professor of Medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine in St. Louis. "In this context, the publication of the HELIOS results at Weeks 24 and 48 is highly encouraging. The observed objective improvement in pancreatic function and visual acuity stabilization over 48 weeks, combined with a favorable safety profile, reinforces AMX0035’s potential to meaningfully alter the trajectory of this disorder." "We are excited to share the peer-reviewed publication of these results, which details the clinical findings of AMX0035 at Week 24 and 48 across both the e...

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