Dr. Fumihiko Urano
Washington University School of Medicine, USA
Dear Friends,
I hope you had a wonderful summer. As the number of patients referred to us continues to grow, our team feels an even greater urgency to develop safe and effective treatments for Wolfram syndrome. I’m pleased to share the latest updates with you:
Ongoing Clinical Trial of AMX0035 in patients with Wolfram Syndrome
We have been collaborating with Amylyx Pharmaceuticals to advance the development of AMX0035, a novel oral medication with the potential to halt or delay the progression of Wolfram syndrome, based on its mechanisms of action and pre-clinical studies. The trial began in April 2023, and the interim results were announced in April 2024. According to Amylyx, the interim analysis of the ongoing Phase 2 clinical trial of AMX0035 for Wolfram syndrome, which involved eight participants assessed at Week 24, revealed encouraging outcomes. Participants demonstrated improvements in pancreatic function and glycemic control, as indicated by C-peptide levels and other markers of glucose metabolism. This contrasts with the typical disease progression expected in Wolfram syndrome. All eight participants met the prespecified responder criteria, showing either improvement or stabilization of their condition, as assessed by both the Patient Global Impression of Change (PGIC) and Clinician Global Impression of Change (CGIC) scales. Furthermore, the majority of participants reported some improvement in vision. Importantly, AMX0035 was generally well-tolerated by all participants, supporting its potential as a therapeutic option for this rare and devastating disorder.