Amylyx: First person dosed in AMX0035 trial for Wolfram Syndrome treatment

Amylyx Pharmaceuticals announced that the first participant has been dosed in the HELIOS study, a Phase 2 clinical trial of AMX0035 and taurursodiol for the treatment of Wolfram syndrome . HELIOS is an exploratory open-label proof of biology study assessing the effect of AMX0035 safety and tolerability, and various measures of endocrinological, neurological and ophthalmologic function. Amylyx anticipates topline results from HELIOS in 2024. "WS is a rare, progressive, and often fatal neurodegenerative disease. Preclinical data showed that a combination treatment of two chemicals, namely AMX0035, may restore cellular functioning in a cellular model of WS," said Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS clinical trial, and Professor of Medicine and of Pathology and Immunology in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine. "There continues to be a significant unmet need for people living with WS and we look forward to building upon our preclinical findings as part of HELIOS to explore the safety, tolerability, and preliminary clinical activity in people living with WS."