A Phase II Study of Safety and Efficacy of AMX0035 in Adult Patients With Wolfram Syndrome

This study is an open label Phase II study to evaluate the safety and efficacy of AMX0035 in adults with Wolfram syndrome.

Key Inclusion Criteria:Definitive diagnosis of Wolfram syndromeInsulin dependent diabetes mellitus due to Wolfram syndromeAt least 17 years of ageParticipant must be willing to wear a CGM device for the duration of the studyKey Exclusion Criteria:Presence of pathologies that can alter the enterohepatic circulation of bile acids (e.g., ileal resection and stoma, regional ileitis)Any history of heart failure per New York Heart Association (NYHA)History of or family history of breast and/or ovarian cancerParticipant under severe salt restriction where the added salt intake due to treatment would put the patient at risk, in the Investigator's judgmentReceived treatment with any investigational drug or device within the 30 days (or 5 half-lives, whichever is longer) prior to first dose at Day 1Previous treatment with gene or cellular therapy