Amylyx’s AMX0035 Shows Promising Results In Phase 2 Trial For Wolfram Syndrome

Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) announced promising results from its Phase 2 HELIOS clinical trial evaluating AMX0035 (a combination of sodium phenylbutyrate [PB] and taurursodiol [TURSO]) in 12 adult patients with Wolfram syndrome, a rare genetic disorder affecting roughly 3,000 people in the U.S. After 24 weeks of treatment, the trial demonstrated an improvement in pancreatic function, as measured by C-peptide response, which was the primary endpoint. 

This finding contrasts with the usual decline seen as the disease progresses. The trial also showed improvements or stability in secondary endpoints, such as hemoglobin A1c levels, glucose monitoring, and visual acuity. Both patients and physicians reported either stability or improvement in overall condition, aligning with predefined responder criteria. Additionally, participants who completed assessments at Weeks 36 (n=10) and 48 (n=6) continued to show sustained improvements. These findings were presented today at the International Society for Pediatric and Adolescent Diabetes (ISPAD) 50th Annual Congress and during an Amylyx-hosted webcast.

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 stated, “The topline results of HELIOS indicate that AMX0035 has the potential to favorably change the trajectory of Wolfram syndrome, a progressive disease with no approved treatment options. These results build on the interim data presented in April of this year and show an improvement on multiple measures of pancreatic beta cell function, glycemic control, and vision. In addition, the participants who reached their Week 36 or Week 48 assessments demonstrated sustained improvement over baseline in C-peptide and HbA1c, which are objective laboratory measures of pancreatic function and glycemic control. These data are encouraging since Wolfram syndrome is a progressive disease.”

The analysis includes data from all 12 participants at Week 24, as well as those who completed assessments at Week 36 (n=10) and Week 48 (n=6) by the data cutoff. The main goal of the study was to measure the change in C-peptide levels, a key indicator of pancreatic beta cell function and a marker for glycemic control, using a mixed meal tolerance test (MMTT) at Week 24. The trial also evaluated secondary and exploratory outcomes related to diabetes management and other areas affected by the disease.

Results from the HELIOS trial revealed improvements in C-peptide response, with a Week 24 change from baseline at 120 minutes of +3.8 minng/mL [SE: 19.3] in the Intent to Treat group (N=12) and +20.2 minng/mL [SE: 11.2] in the Per Protocol group (N=11). Participants treated with AMX0035 also showed better glycemic control, enhanced visual acuity in some cases (measured using the Snellen chart), and either stabilization or improvement of their condition, based on both clinician and patient-reported global impressions of change.