Amylyx Publishes Positive Phase 2 Data for AMX0035 in Wolfram Syndrome — Amylyx Pharmaceuticals, Inc.

Event summary

Week 24 and Week 48 results from the Phase 2 open-label HELIOS trial of AMX0035 in Wolfram syndrome published in The Journal of Clinical Investigation on May 15, 2026.
Trial showed continued improvements in pancreatic beta cell function through Week 48 and stabilization of visual acuity.
Secondary measures of glycemic control (HbA1c and time in target glucose range) improved from baseline at Weeks 24 and 48.
AMX0035 received Orphan Drug Designation for Wolfram syndrome from the FDA in November 2020 and the European Commission in August 2024.

The big picture

Amylyx's publication of positive Phase 2 data for AMX0035 in Wolfram syndrome reinforces its strategy of targeting neurodegenerative and endocrine disorders with high unmet needs. The results position AMX0035 as a potential first-in-class treatment, aligning with broader industry trends toward precision therapies for rare genetic diseases. With Orphan Drug Designation secured in key markets, Amylyx is poised to advance AMX0035 through later-stage development, though execution risks remain given the small patient population and regulatory hurdles.

What we're watching

Regulatory Pathway: Whether the positive Phase 2 data will accelerate FDA discussions for a Phase 3 trial in Wolfram syndrome.
Clinical Validation: The pace at which longer-term data from the HELIOS trial (up to 208 weeks) will be presented and its impact on AMX0035's profile.
Market Opportunity: How Amylyx positions AMX0035 in the rare disease space, given the lack of approved treatments for Wolfram syndrome.