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AMYLYX Phase 1/2 Clinical Trial

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.

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Amylyx Pharmaceuticals Announces First Participant Dosed in Phase 2 Study of AMX0035 for the Treatment of 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).

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Amylyx Pharmaceuticals Announces Interim Data From Ongoing Phase 2 Helios Clinical Trial

Objectives

  1. Summarize two types of Wolfram syndrome and related disorders.
  2. Share lessons and stories from past and current Wolfram Syndrome clinical studies, including both achievements and obstacles.
  3. Emphasize the need for cooperation with patient organizations and industry partners to support the development of new therapies.

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