Investigational oral therapy improves pancreatic function, symptoms in Wolfram syndrome

An oral therapy to treat Wolfram syndrome significantly improved pancreatic functions and induced improvement in symptoms at 24 and 48 weeks for most participants in a phase 2 trial.

• AMX0035 significantly improved C-peptide response at 48 weeks in a small group of adults with Wolfram syndrome.
• Symptom improvement was observed in nine of 10 adults at 48 weeks.

There are no FDA-approved treatments for Wolfram syndrome, a rare neurogenerative disease that impacts multiple organ systems, according to Fumihiko Urano, MD, PhD, the Samuel E. Schechter Professor of Medicine and professor of pathology and immunology at Washington University School of Medicine. In findings from the HELIOS trial presented at the Joint Congress of the European Society for Paediatric Endocrinology and European Society of Endocrinology, Urano and colleagues showed that AMX0035 (Amylyx Pharmaceuticals), an oral combination therapy of sodium phenylbutyrate and taurursodiol, was associated with improvements in multiple measures of Wolfram syndrome progression at 24 and 48 weeks. As Healio previously reported, the FDA granted AMX0035 orphan drug designation for Wolfram syndrome in 2020.