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Amylyx Pharmaceuticals Announces Interim Data From Phase 2 HELIOS Clinical Trial Of AMX0035 In Adults With Wolfram Syndrome

Benzinga ·  Apr 10 09:02

- Interim analysis, including eight participants assessed at Week 24, demonstrated improvements in pancreatic function and glycemic control, as measured by C-peptide and other markers of glucose metabolism, rather than worsening typically expected with disease progression

- All eight participants met prespecified responder criteria demonstrating either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change (PGIC) and the Clinician Reported Global Impression of Change (CGIC) scales

- Majority of participants reported some improvement in vision

- AMX0035 was generally well-tolerated in all participants

- Interim data to be presented during a webcast today at 1:30 p.m. Eastern Time

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