HELIOS Phase 2: AMX0035 improves C-peptide AUC by +3.8 min*ng/mL at Week 24 in Wolfram syndrome

Amylyx Pharmaceuticals, Inc.

Primary endpoint: C-peptide AUC improved at Week 24 (ITT +3.8 min*ng/mL; Per Protocol +20.2) and sustained through Week 48 (+36.7).
Glycemic control: HbA1c decreased (-0.09% at Week 24 to -0.35% at Week 36); time in target glucose range increased (+5.2% to +12.3%).
All 12 participants showed stability/improvement on CGIC and PGIC; no serious AEs related to AMX0035; diarrhea most common.
FDA and EU granted Orphan Drug Designation; company plans to meet FDA to discuss Phase 3, update in 2025.

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