Non-confidential report

Valerion proposes to deliver MTM1 into skeletal muscle using MTM1 fused to an antibody delivery protein as an eventual therapy for patients with myotubular myopathy. This is a relatively early phase project and TACT welcomed the opportunity to review the proposal at this stage. The committee agreed with the applicant’s current plans to help identify the best population for the future clinical study and recognized that this could help define outcome measures most likely to detect change in a therapeutic trial. The full report offers recommendations on:

• The need for a definitive rigorous preclinical efficacy study
• Pre-clinical studies and suggested animal models that establish target engagement, dose, route of administration, dose frequency and effect on meaningful functional outcomes
• Patient populations for clinical studies including natural history data collection
• Interaction with regulatory bodies – in particular early on about moving from adult to paediatric clinical trials
• Selection of valid primary and secondary endpoints
• Biomarker identification
• Marketing considerations

To request a full copy of the report please contact the applicant, Deborah Ramsdell: deb.ramsdell@alopexx.com

Name of applicant: Deborah Ramsdell

Reviewed: October, 2016 in Miami, FL, USA