Amylyx Pharmaceuticals Announces Completion of Enrollment in HELIOS, a Phase 2 Study of AMX0035 for the Treatment of Wolfram Syndrome

CAMBRIDGE, Mass. — Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX)  today announced it has completed enrollment of its Phase 2 HELIOS trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS), a rare genetic disease that typically results in neurodegeneration and premature death, which has no known cure. The trial has enrolled 12 adult participants living with WS, and preliminary results are anticipated in the second half of 2024.

“The unmet need for people living with WS remains significant. WS is progressive, often fatal, and difficult to diagnose, with current treatments focused mostly on helping to address symptoms,” said Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS clinical trial and Professor of Medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine. “Our preclinical findings showed that AMX0035 may impact relevant WS disease pathways and manifestations, and we look forward to the preliminary results from HELIOS anticipated later this year.”

HELIOS is an open-label proof of biology trial designed to study the effect of AMX0035 on safety and tolerability, and various measures of endocrinological, neurological, and ophthalmologic function in adult participants living with WS. Data from this initial study will inform potential future Amylyx trials for individuals with WS.

In September 2022, researchers from Washington University School of Medicine in St. Louis, in collaboration with Amylyx, published preclinical data in the peer-reviewed Journal of Clinical Investigation Insight. The data explored the potential of AMX0035 as a novel therapeutic approach for WS and provided initial proof-of-concept for its therapeutic development in WS.

“Little progress has been made in the treatment of WS, and we are excited by the potential we have been seeing in the preclinical work with AMX0035,” said Camille L. Bedrosian, MD, Chief Medical Officer of Amylyx. “These preclinical findings suggest the possibilities of AMX0035 for individuals with WS that we continue to explore, including in the HELIOS trial. We are grateful to those who partnered to complete this important clinical trial enrollment milestone, in particular, the WS community, their families and caregivers, experts, clinicians, researchers, and advocacy leaders.”

Amylyx announced that the FDA granted orphan drug designation to AMX0035 for the treatment of WS in November 2020. The FDA may grant this designation to drugs and biologics intended to treat a rare disease or condition affecting fewer than 200,000 persons in the U.S. Orphan designation qualifies a company for certain benefits, including financial incentives to support clinical development and the potential for seven years of market exclusivity in the U.S. upon regulatory approval.

 

About Wolfram Syndrome

Wolfram syndrome (WS) is an autosomal recessive neurodegenerative disease characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration. Common manifestations of WS include diabetes mellitus, optic nerve atrophy, central diabetes insipidus, sensorineural deafness, neurogenic bladder, and progressive neurologic difficulties. Genetic and experimental evidence suggest that endoplasmic reticulum (ER) dysfunction is a critical pathogenic component of WS. The prognosis of WS is poor, and many people with the disease die prematurely with severe neurological disabilities.

 

About AMX0035 / RELYVRIO^®/ ALBRIOZA™ / ALBRIOZA^®

AMX0035 is an oral, fixed-dose combination of sodium phenylbutyrate and taurursodiol (known as ursodoxicoltaurine outside of the U.S.). It is approved as RELYVRIO^® to treat amyotrophic lateral sclerosis (ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. AMX0035 is being studied for the potential treatment of other neurodegenerative diseases, and Amylyx is exploring its treatment in other populations and regions. The formulation of RELYVRIO, ALBRIOZA, and AMX0035 is identical.

 

About Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada, EMEA, and Japan.