Amylyx Reports Positive Long-Term Phase 2 HELIOS Trial Results for AMX0035 in Wolfram Syndrome

Amylyx Pharmaceuticals, a company specializing in investigational therapies for neurodegenerative diseases, has reported promising results from a Phase 2 trial of their drug AMX0035 in patients with Wolfram syndrome. This rare genetic disorder, characterized by symptoms such as diabetes, vision loss, and neurological degeneration, currently has no approved treatments. The trial, known as HELIOS, has shown that AMX0035 could potentially slow or improve disease progression in individuals living with Wolfram syndrome.

The HELIOS trial, conducted over 48 weeks, involved adult participants diagnosed with the syndrome. Initial results at Week 24 had already pointed to improvements in pancreatic function, as evaluated through a mixed-meal tolerance test. The results at Week 48 further confirmed these findings, showing that AMX0035 led to sustained stabilization or improvement in pancreatic beta cell function, glycemic control, and vision. These outcomes were measured using standardized tests like hemoglobin A1c for glycemic control and visual acuity tests, with all participants meeting responder criteria for either improvement or no change in their symptoms.

Participants also reported improvements in their overall symptom burden through patient and clinician global impression scales. The qualitative interviews conducted during the study provided additional support for the drug's positive impact on symptoms associated with Wolfram syndrome. Importantly, AMX0035 was generally well-tolerated, with all adverse events being mild or moderate and no serious adverse events linked to the treatment.

The trial's principal investigator, Fumihiko Urano, MD, PhD, highlighted that the results underscore AMX0035's potential to alter the progressive nature of Wolfram syndrome, reinforcing previous findings of its efficacy. Camille L. Bedrosian, MD, the Chief Medical Officer at Amylyx, emphasized the company's commitment to collaborating with the U.S. Food and Drug Administration (FDA) to design a Phase 3 trial, aiming to address unmet needs within the Wolfram syndrome community.

HELIOS is a single-arm, open-label trial designed to assess AMX0035's safety and efficacy in treating Wolfram syndrome, with a focus on endocrinological, neurological, and ophthalmological functions. The FDA and the European Commission have granted Orphan Drug Designation to AMX0035 for this indication, recognizing its potential as a treatment for a disorder that affects approximately 3,000 individuals in the U.S.

Amylyx Pharmaceuticals created AMX0035 as a combined oral therapy of sodium phenylbutyrate (PB) and taurursodiol (TURSO). The drug aims to mitigate neurodegeneration by targeting pathways leading to cell death. Amylyx believes that by addressing endoplasmic reticulum stress and mitochondrial dysfunction, AMX0035 can effectively reduce the impact of neurodegenerative diseases like Wolfram syndrome and progressive supranuclear palsy.

The company remains optimistic about its financial future, with an expected cash runway extending through the end of 2026. As they continue to focus on advancing treatments for diseases with high unmet needs, Amylyx remains dedicated to their mission of delivering impactful solutions for complex health challenges.