Shots:

• Following COMP’s positive opinion, the EC has granted ODD to AMX0035 (oral combination of sodium phenylbutyrate & taurursodiol) to treat Wolfram syndrome
• AMX0035 is being assessed under a P-II (HELIOS) study for its safety, tolerability & efficacy plus for evaluating endocrinological, neurological & ophthalmologic function in adults (n=12) with Wolfram syndrome
• Interim analysis of the study in 8 subjects at wk.24 showed improved pancreatic function & glycemic control; all 8 reached responder criteria, showing either improved or stabilized disease as per PGIC & CGIC scales, with most reporting improved vision. Topline data from all 12 subjects at wk.24 are expected in H2'24