Amylyx Pharmaceuticals Announces Interim Data From Ongoing Phase 2 Helios Clinical Trial
Objectives
Summarize two types of Wolfram syndrome and related disorders.
Share lessons and stories from past and current Wolfram Syndrome clinical studies, including both achievements and obstacles.
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