Amylyx Pharmaceuticals, Inc. today announced positive Week 48 data from the Phase 2 open-label HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with Wolfram syndrome. These results were presented at the Joint Congress of the European Society for Pediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) in Copenhagen, Denmark and are available on the “Presentations” page of the Amylyx website.
Study Overview
Official Title
A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome
Brief Summary
This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.
CAMBRIDGE, Mass.–(BUSINESS WIRE)– Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the first participant has been dosed in the HELIOS study, a Phase 2 clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO]) for the treatment of Wolfram syndrome (WS).
Objectives
- Summarize two types of Wolfram syndrome and related disorders.
- Share lessons and stories from past and current Wolfram Syndrome clinical studies, including both achievements and obstacles.
- Emphasize the need for cooperation with patient organizations and industry partners to support the development of new therapies.
About the Snow Foundation
The Snow Foundation is a collective voice for Wolfram syndrome patients, working towards a cure for Wolfram syndrome and developing novel therapies for diabetes, vision loss, hearing loss and neurodegeneration.
Rare Diseases…Common Problems
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