Amylyx Reports 48-Week HELIOS Trial Results Showing AMX0035 Benefits in Wolfram Syndrome

Amylyx Pharmaceuticals announced positive 48-week results from its Phase 2 HELIOS trial evaluating AMX0035 in adults with Wolfram syndrome, a rare genetic disorder. The trial showed continued improvement in pancreatic beta cell function, building on prior data from Week 24 that demonstrated gains in C-peptide levels following a mixed-meal tolerance test. These findings were presented at the Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) in Copenhagen.

“The results of the Phase 2, open-label HELIOS trial continue to demonstrate that AMX0035 has the potential to favorably alter the trajectory of Wolfram syndrome, a progressive disorder with no approved treatment options. The consistency of the Week 48 results across multiple measures of disease progression that meaningfully impact the daily lives of those living with Wolfram syndrome, including pancreatic function, glycemic control, and vision, reinforces the previously reported Week 24 findings. Additionally, the majority of participants reported meaningful improvement in at least one Wolfram syndrome-related symptom during interviews, underscoring the real-world importance of these results,” said Fumihiko Urano, MD, PhD, Principal Investigator of the Phase 2 HELIOS clinical trial in Wolfram syndrome 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.

Beyond pancreatic function, patients treated with AMX0035 showed stable or improved glycemic control, visual acuity, and reduced symptom burden through Week 48. Measures such as HbA1c and continuous glucose monitoring indicated sustained metabolic benefits, and all participants with available data met responder criteria on both Patient and Clinician Global Impression of Change (PGI-C and CGI-C). Additionally, qualitative interviews supported perceived improvements in symptoms, with no serious adverse events reported.

“These long-term results reinforce both our positive data at Week 24 and our belief in the potential of AMX0035 to stabilize and even improve key manifestations of Wolfram syndrome, a relentlessly progressive disorder,” said Camille L. Bedrosian, MD, Chief Medical Officer at Amylyx. “With these findings, we are focused on working closely with the FDA to inform the design of a Phase 3 trial. Our ultimate aim is to address the unmet needs that are still a reality for people living with this devastating disorder. We want to thank the Wolfram syndrome community for their continued collaboration and support.”

The HELIOS trial (NCT05676034), a single-arm Phase 2 study, is investigating the safety and efficacy of AMX0035 a combination of sodium phenylbutyrate and taurursodiol in treating Wolfram syndrome. The treatment has received Orphan Drug Designation from both the U.S. FDA and the European Commission, and the latest data support its potential as a disease-modifying therapy for this underserved condition.