Therapeutic development – oral medication

In the past, I looked for existing drugs that could be beneficial for patients with Wolfram syndrome. This effort led to a few clinical trials, but a repurposed drug could be just a band-aid for Wolfram, and we need a new medication for Wolfram syndrome. I have been focusing my efforts on developing a new drug, AMX0035, for the treatment of Wolfram syndrome in collaboration with Amylyx Pharmaceuticals in Cambridge, Massachusetts. AMX0035 targets endoplasmic reticulum stress (a molecular mechanism of Wolfram) and mitochondrial dysfunction. A recent clinical trial of AMX0035 in patients with ALS, an adult-onset neurodegenerative disorder, was successful ( Our pre-clinical study using cell and rodent models of Wolfram syndrome was positive. We have deposited the data to the public server ( and plan to publish it in a medical research journal soon. 

US FDA granted an orphan drug designation of AMX0035 for the treatment of Wolfram syndrome in October 2020. We submitted our clinical trial plan to the US FDA and received their feedback in the late summer of 2020. We have examined our longitudinal study data and dantrolene clinical trial data extensively and have created an innovative clinical trial protocol to ensure the safety of our patients and assess the efficacy of AMX0035 accurately. I spend a certain amount of time every single day on this project with medical officers at Amylyx and my colleagues at Washington University. We have been communicating with US FDA and hope to start a trial late summer of 2021. Please stay tuned.

Fumi Urano