Amylyx Pharmaceuticals, Inc. Announces Positive Long-Term Results from Phase 2 Helios Clinical Trial of AMX0035 in People with Wolfram Syndrome

Published: 2025-05-12 07:00:00 ET AMLX

Published on 05/12/2025 at 07:00, updated on 05/12/2025 at 08:05

Amylyx Pharmaceuticals, Inc. announced positive Week 48 data from the Phase 2 open-label HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine) 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 and are available on the "Presentations" page of the Amylyx website. All participants with available measurements met the responder criteria, defined as either improvement or no change, on both the Patient Global Impression of Change (PGI-C) and Clinician Global Impression of Change (CGI-C) at weeks 24 and 48, indicating stability or improvement in their Wolfram syndrome-related symptoms. Results from qualitative on-study interviews further supported the potential positive impact of AMX0035 on symptom burden.

Safety data were consistent with safety data from prior studies of AMX0035. 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, reinforce the previously reported Week 24 findings.