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Amylyx Pharmaceuticals Announces Interim Data From Ongoing Phase 2 Helios Clinical Trial

Objectives

  1. Summarize two types of Wolfram syndrome and related disorders.
  2. Share lessons and stories from past and current Wolfram Syndrome clinical studies, including both achievements and obstacles.
  3. Emphasize the need for cooperation with patient organizations and industry partners to support the development of new therapies.

Key Takeaways

Strong Scientific Rationale

  • Wolfram syndrome is a progressive, genetic disease caused by mutations in WFS1 that cause endoplasmic reticulum (ER) stress and impaired mitochondrial dynamics
  • AMX0035 has been shown to simultaneously mitigate ER stress and mitochondrial dysfunction
  • Preclinical data have demonstrated the efficacy of AMX0035 in cell lines, patientderived cells, and mouse models

Compelling Data Support AMX0035’s Potential in Wolfram Syndrome

  • Interim analysis demonstrated improvement 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 participants met prespecified responder criteria demonstrating either improvement or stabilization of disease according to both patient- and clinician-reported scales
  • Majority of participants reported some improvement in vision
  • AMX0035 was generally well-tolerated in all participants

Urgent Unmet Need

  • There are currently no disease-modifying therapies for Wolfram syndrome; treatment strategies focus on symptom management
  • Wolfram syndrome impacts ~3,000 people in the U.S. and results in premature death

AMX0035 Wolfram Syndrome Program Next Steps

  • Continue to progress the Phase 2 HELIOS study and evaluate options for future development
  • Planning to engage with regulatory authorities to align on the development path
  • Topline data for all 12 participants at Week 24 are expected in the second half of 2024