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

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced interim data from the ongoing Phase 2 HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with Wolfram syndrome, a rare, progressive genetic disease impacting approximately 3,000 people in the U.S. The interim data from eight participants who have completed 24 weeks of treatment demonstrated that AMX0035 had a clinically meaningful effect on key outcomes measuring the progression of diabetes, visual decline, and overall disease burden in adult participants living with Wolfram syndrome.

“I have been studying Wolfram syndrome and caring for people with the disease for more than 20 years. This community has an urgent unmet need for disease modifying treatments. Outcomes for people with Wolfram syndrome consistently worsen over time, so disease stabilization alone is clinically meaningful for both patients and their doctors. The interim results from HELIOS that demonstrate improvement across multiple organ systems impacted by this progressive disease are encouraging,” said Fumihiko Urano, MD, PhD, Principal Investigator of the Phase 2 HELIOS clinical trial in Wolfram syndrome and the Samuel E. Schechter Professor of Medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine in St. Louis.

HELIOS is an ongoing, open-label Phase 2 study in 12 participants designed to evaluate if AMX0035 slows progression of diabetic, visual, and other measures in people living with Wolfram syndrome and to evaluate safety and tolerability. The primary efficacy endpoint of the trial measures change from baseline in C-peptide, an established, objective laboratory measure of pancreatic beta cell function and glycemic control, assessed using a Mixed Meal Tolerance Test (MMTT). Secondary and exploratory outcomes include the measurement of other diabetic responses and other domains affected by the disease. The interim analysis performed is based on a data cutoff as of March 5, 2024, which includes all participants who completed their Week 24 assessments as of the cutoff (n=8).