Susan's questions to Amylyx Pharmaceuticals Inc (AMLX) leadership • Q1 2025
Question
An analyst from Bank of America Securities, on behalf of Tim Anderson, asked about the ongoing discussions with the FDA regarding the Phase III trial design for AMX0035 in Wolfram syndrome. She also inquired what specific insights the upcoming 48-week HELIOS data would provide to inform these regulatory discussions.
Answer
Chief Medical Officer Dr. Camille Bedrosian stated that Amylyx does not disclose details of its FDA interactions but confirmed the 48-week data is crucial for informing the Phase III design. She guided listeners to expect updates on endpoints similar to the 24-week data, including C-peptide response, glycemic measures, and visual acuity, noting that stabilization in this progressive disease is significant. She emphasized this is the first-ever trial in Wolfram, so there is no existing template.