Amylyx announces HELIOS clinical trial meets primary endpoint

Amylyx announced topline data from the Phase 2 open-label HELIOS clinical trial of AMX0035 in 12 adults living with Wolfram syndrome. Wolfram syndrome is a rare, progressive, monogenic disease impacting approximately 3,000 people in the U.S. HELIOS showed improvement in pancreatic function, as measured by C-peptide response after 24 weeks of treatment with AMX0035, the study’s primary efficacy endpoint, in contrast to the expected decrease in pancreatic function with disease progression. Similar overall improvements or stabilization were observed across all secondary endpoints, including hemoglobin A1c, time in target glucose range assessed by continuous glucose monitoring, and visual acuity. Patient- and physician-reported global impressions of change showed disease stability or improvement in all participants, meeting prespecified responder criteria. In addition, longer-term data for all participants who completed Week 36 and Week 48 assessments showed sustained improvement over time. Data from HELIOS are being presented at the International Society for Pediatric and Adolescent Diabetes 50th Annual Congress and during a webcast held by the company. The analysis performed includes Week 24 data for all 12 participants and data for all participants who completed their Week 36 and Week 48 assessments as of the data cutoff. 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 a surrogate marker of glycemic control, assessed using a mixed meal tolerance test at Week 24. Secondary and exploratory outcomes include the assessment of other diabetic measures and other domains affected by the disease. HELIOS showed improvements in its primary endpoint of C-peptide response with a change from baseline to Week 24 at 120 minutes of +3.8 minutes*ng/mL in the Intent to Treat group and +20.2 min*ng/mL in the Per Protocol group. In addition, as outlined in the table below, participants receiving AMX0035 had improved glycemic control, as measured by markers of glucose metabolism; improved visual acuity in some participants, as measured by the Snellen chart; and improvement or stabilization of the disease, as measured by the Clinician Reported Global Impression of Change and Patient Reported Global Impression of Change. The safety profile of AMX0035 in HELIOS was consistent with prior safety data. AMX0035 was generally well-tolerated. All adverse events were mild or moderate, and there were no serious AEs related to AMX0035 treatment. The most common AE was diarrhea.