AMX0035 Gains Orphan Drug Status for Wolfram Syndrome

For the purpose of treating Wolfram syndrome, Amylyx Pharmaceuticals has been given Orphan Drug Designation for AMX0035, a patented combination of taurursodiol (TURSO) and sodium phenylbutyrate (PB). A uncommon, progressive, and monogenic condition called Wolfram syndrome is characterised by optic nerve atrophy, deafness, diabetes insipidus, childhood-onset diabetes mellitus, and neurodegeneration. In 2020, the FDA designated AMX0035 as an orphan drug to treat Wolfram syndrome.

Amylyx reported encouraging results from an interim analysis of its Phase 2 HELIOS research, which showed that AMX0035 improved glycaemic management and pancreatic function in eight Wolfram syndrome subjects evaluated at Week 24. Based on the Patient Reported Global Impression of Change (PGIC) and Clinical Reported Global Impression of Change (CGIC) ratings, all eight individuals demonstrated either improvement or stabilisation of their condition, meeting predetermined responder criteria. Most participants said their vision had improved somewhat.

This autumn, at Week 24, the business plans to provide topline statistics from all 12 participants. Because WFS1 mutations and ER stress are clearly linked, Wolfram syndrome is regarded as a model endoplasmic reticulum stress illness. It is thought that AMX0035 targets mitochondrial malfunction and ER stress. At Week 24, initial results from HELIOS revealed stabilisation or even improvement across key endpoints.