Amylyx Pharmaceuticals (NASDAQ: AMLX), a developer of new treatments for neurodegenerative disease, on Wednesday released interim data from Phase 2 HELIOS trial showing improvements in pancreatic function and glycemic control in adults with Wolfram syndrome. The trial, assessing AMX0035, involved eight participants completing 24 weeks of treatment.

Results indicated positive impacts on C-peptide and glucose metabolism markers, contrary to the disease's typical progression. Majority of participants showed vision improvement. AMX0035 demonstrated good tolerance among participants.

AMX0035, an oral combination of sodium phenylbutyrate and taurursodiol, targets ER stress and mitochondrial dysfunction, potentially mitigating neurodegeneration. The FDA granted orphan drug designation to AMX0035 for Wolfram syndrome treatment in November 2020.

Wolfram syndrome, a rare genetic disease affecting around 3,000 people in the US, is characterized by diabetes, optic nerve atrophy and neurodegeneration.