Amylyx Pharmaceuticals Announces Positive Long-Term Results from Phase 2 HELIOS Clinical Trial of AMX0035 in People with Wolfram Syndrome - Seite 2

“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.”

HELIOS (NCT05676034) is a single-site, single-arm, open-label, Phase 2 trial designed to evaluate the safety and tolerability of AMX0035, as well as its effects on various measures of endocrinological, neurological, and ophthalmological function in adult participants living with Wolfram syndrome. The U.S. Food and Drug Administration (FDA) and the European Commission granted Orphan Drug Designation to AMX0035 for the treatment of Wolfram syndrome in November 2020 and August 2024, respectively.

As outlined in the table below, results through Week 48 of the HELIOS trial showed treatment with AMX0035 resulted in improvement or stabilization across measures of glycemic control, visual acuity, and overall symptom burden with AMX0035. Notably, HELIOS showed improvements in the primary endpoint of C-peptide response, as measured by area under the curve from 0-120 minutes during a Mixed Meal Tolerance Test (MMTT). In the Per Protocol group, the mean change from baseline to Week 24 was 20.2 min*ng/mL [standard error (SE) 11.2], and, at Week 48, the mean change from baseline was +34.5 minutes ng/mL(min*ng/mL) [13.0]; descriptive p-value=0.0263). Long-term data at Week 48 included 10 participants in the Per Protocol population with genetically confirmed Wolfram syndrome (one participant was excluded from the Per Protocol population due to not meeting the study inclusion criteria of genetically confirmed Wolfram syndrome) and 11 participants in the Intent-to-Treat (ITT) population. One participant discontinued between Week 24 and Week 48 for reasons unrelated to safety.