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Amylyx reports positive results for failed ALS drug in Wolfram trial

Amyyx Pharmaceuticals has released promising interim results from its Phase II trial of Relyvrio (AMX0035) for Wolfram syndrome, a rare disease.

This announcement follows challenges, including the withdrawal of the drug from the ALS market after a Phase III trial failed to meet endpoints, despite FDA approval in 2022.

The Phase II HELIOS trial (NCT05676034) focuses on assessing AMX0035's safety and various functions affected by Wolfram syndrome.

Primary endpoints include changes in C-peptide levels, a measure of pancreatic beta cell function, and glycemic control via a Mixed Meal Tolerance Test (MMTT). Secondary outcomes evaluate diabetic responses and other disease-affected domains.

Interim data from eight of 12 patients show significant improvements in diabetes progression, visual decline, and overall disease burden.

AMX0035's safety profile remained consistent, with mostly mild or moderate adverse events, the most common being diarrhoea.

AMX0035, a combination of sodium phenylbutyrate and taurursodiol, shows promise in cellular function enhancement and is also investigated for progressive supranuclear palsy (PSP).