Amylyx Pharmaceuticals Announces Interim Data From Ongoing Phase 2 HELIOS Clinical Trial Demonstrating Improvements in Pancreatic Function and Glycemic Control with AMX0035 in People with Wolfram Syndrome - Seite 2

In this interim analysis of eight participants treated with AMX0035, increases were observed on average in the primary outcome of total C-peptide response (C-peptide AUC change from baseline) including in the 90-minute response at Week 24 (+15.6 ng*min/mL, 95% CI: [1.3, 30.0]). In Wolfram syndrome, progressive decline would have been expected on this measure. Additionally, seven out of eight participants demonstrated at least a 30-minute shorter time to peak C-peptide response. In Wolfram syndrome, a progressive increase in time to peak C-peptide response, indicating slower pancreatic response, and reduced total C-peptide response would have been expected.

“The improvements in C-peptide, an objective laboratory measure, observed in our HELIOS trial are promising and differ from the normal course of Wolfram syndrome despite best supportive care,” said Camille L. Bedrosian, MD, Chief Medical Officer of Amylyx. “The majority of people with Wolfram syndrome carry mutations in the WFS1 gene, which encodes a protein that spans the membrane of the endoplasmic reticulum (ER) called wolframin. Loss of wolframin function leads to ER stress and impaired mitochondrial dynamics, which in turn leads to dysfunction and apoptosis of cells. Because of the clear link between WFS1 mutations and ER stress, Wolfram syndrome is considered a prototypical ER stress disorder. AMX0035 is believed to target both ER stress and mitochondrial dysfunction. We believe today’s interim results support the compelling science behind the mechanism of action of AMX0035 and its potential to help people living with Wolfram syndrome.”

“The HELIOS trial began as a result of a multi-year collaboration with the Wolfram syndrome community and follows strong preclinical data with clear effects in cellular and animal models. We thank the Wolfram community, including those participating in HELIOS, their loved ones, and the clinical staff at Washington University School of Medicine in St. Louis, for their support of this important trial,” said Justin Klee and Joshua Cohen, Co-CEOs of Amylyx. “We are planning to engage with regulatory authorities to align on the development path in Wolfram syndrome. We expect topline data for all 12 participants at Week 24 in the second half of this year.”