Amylyx Completes HELIOS Enrollment for Wolfram Syndrome Trial

Amylyx Pharmaceuticals, a biotech firm listed on NASDAQ under the ticker AMLX, has successfully completed the enrollment phase for its Phase 2 clinical trial, known as HELIOS, which is investigating the efficacy of AMX0035 in treating Wolfram syndrome (WS). WS is a rare genetic disorder that leads to neurodegeneration and early mortality, with no current cure available. The trial has enrolled a total of 12 adult WS patients, and initial findings are expected to be released in the latter part of 2024.

Fumihiko Urano, the principal investigator for the HELIOS trial and a professor at Washington University School of Medicine, highlighted the significant unmet need for WS patients, noting the disease's progressive and often fatal nature. Urano's previous preclinical research indicated that AMX0035, a combination of sodium phenylbutyrate and taurursodiol, could potentially influence the disease pathways associated with WS.

The HELIOS trial is an open-label study aimed at assessing the safety, tolerability, and impact of AMX0035 on endocrinological, neurological, and ophthalmologic functions in adult WS patients. The data gathered will guide potential future trials by Amylyx for this patient population.

Further details regarding the HELIOS study can be accessed on the clinical trials registry website, under the identifier NCT05676034. In September 2022, Washington University School of Medicine researchers, in collaboration with Amylyx, published preclinical data in the Journal of Clinical Investigation Insight, which explored AMX0035's potential as a novel therapeutic approach for WS.

Camille L. Bedrosian, Amylyx's Chief Medical Officer, expressed excitement about the preclinical findings and the ongoing exploration of AMX0035's potential in treating WS, including within the HELIOS trial. She also acknowledged the contributions of the WS community, families, caregivers, experts, clinicians, researchers, and advocacy leaders in achieving the enrollment milestone.

In November 2020, the FDA granted orphan drug designation to AMX0035 for WS treatment. This designation is given to drugs intended for rare diseases affecting fewer than 200,000 people in the U.S., and it comes with financial incentives and potential market exclusivity upon regulatory approval.

Wolfram syndrome is an autosomal recessive condition characterized by early-onset diabetes, optic nerve atrophy, and neurodegeneration. Common symptoms include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorinal deafness, neurogenic bladder, and progressive neurological issues. The condition is associated with poor prognosis and premature death due to severe neurological disabilities.

AMX0035, also known as RELYVRIO in the U.S. and ALBRIOZA in Canada, is an oral combination therapy approved for the treatment of amyotrophic lateral sclerosis (ALS) in both countries. Amylyx is also researching its potential for other neurodegenerative diseases and exploring its use in different populations and regions.

Amylyx Pharmaceuticals is dedicated to discovering and developing innovative treatments for neurodegenerative diseases, with headquarters in Cambridge, Massachusetts, and operations in Canada, EMEA, and Japan.