Amylyx posts Phase 2 HELIOS data showing AMX0035 benefits in Wolfram syndrome

Amylyx Pharmaceuticals, Inc.

Amylyx Pharmaceuticals, Inc.

AMLX

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  • Amylyx Pharmaceuticals announced peer-reviewed publication of Week 24 and Week 48 results from Phase 2 open-label HELIOS trial of AMX0035 in adults with Wolfram syndrome; portions of data were previously presented at medical meetings.
  • Results indicated stabilization or improvement across measures tied to disease progression, including pancreatic function, glucose control, vision, and overall symptom burden.
  • Drug was generally well tolerated, consistent with earlier safety disclosures, with no treatment-related serious adverse events reported.
  • Company is working with FDA on Phase 3 trial in Wolfram syndrome; additional longer-term HELIOS data are expected at an upcoming scientific meeting.


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