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

Core Insights - Amylyx Pharmaceuticals has completed enrollment for its Phase 2 HELIOS trial of AMX0035 for treating Wolfram syndrome, a rare genetic disease with no known cure, involving 12 adult participants, with preliminary results expected in the second half of 2024 [1][2] Group 1: Clinical Trial Details - The HELIOS trial is an open-label proof of biology study aimed at assessing the safety, tolerability, and effects of AMX0035 on various functions in adults with Wolfram syndrome [2] - Preliminary findings from the HELIOS trial are anticipated later this year, which will inform future trials for individuals with Wolfram syndrome [2][4] Group 2: Background on Wolfram Syndrome - Wolfram syndrome is characterized by childhood-onset diabetes, optic nerve atrophy, and neurodegeneration, leading to a poor prognosis and premature death in many cases [5] - The disease is difficult to diagnose and currently has limited treatment options focused mainly on symptom management [2][5] Group 3: AMX0035 Overview - AMX0035 is a fixed-dose combination of sodium phenylbutyrate and taurursodiol, currently approved for treating amyotrophic lateral sclerosis (ALS) under the brand name RELYVRIO in the U.S. and ALBRIOZA in Canada [6] - The drug is being explored for potential treatment in other neurodegenerative diseases, including Wolfram syndrome [6] Group 4: Regulatory Status - The FDA granted orphan drug designation to AMX0035 for Wolfram syndrome in November 2020, which provides certain benefits, including financial incentives and potential market exclusivity upon regulatory approval [4]