Amylyx Pharmaceuticals launches phase 2 trial for AMX0035 Wolfram syndrome therapy

Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss.

In September, Amylyx won FDA approval for AMX0035 (sodium phenylbutyrate and taurursodiol), marketed as Relyvrio, for adults with amyotrophic lateral sclerosis (ALS).

Both sodium phenylbutyrate and taurursodiol are smal molecules demonstrating neuroprotective effects in preclinical studies.

Trial design

The HELIOS is an exploratory open-label proof of biology study. Amylyx anticipates topline results from HELIOS in 2024.

The HELIOS trial is an exploratory open-label, single-group proof of biology study, with Amylyx anticipating topline results in 2024. The trial aims to assess the safety and efficacy of AMX0035 in…

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