Interim Phase 2 data for AMX0035 shows clinically meaningful effects in Wolfram syndrome (n=8)

Amylyx Pharmaceuticals, Inc.

C-peptide AUC increased +15.6 ng*min/mL at Week 24; 7 of 8 participants had shorter time to peak C-peptide.
HbA1c reduced by 0.26% on average; 6 of 8 improved; time in target glucose range improved by +7.1%.
Visual acuity improved in 5 of 8 participants; one participant changed from legally blind to sighted.
All 8 participants showed disease stability or improvement per CGIC and PGIC; no serious AEs related to AMX0035.

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