Ezutromid: Utrophin modulation as a potential treatment for Becker muscular dystrophy

Jon Tinsley, Summit

29th October 2016

Summit is developing ezutromid (previously known as SMT C1100), an orally administered small molecule for the treatment of Duchenne muscular dystrophy, that is currently in a phase 2 trial in ambulant patients.  Ezutromid is designed to modulate utrophin expression to compensate for the lack of dystrophin in Duchenne muscular dystrophy patients and thereby restore and maintain healthy muscle function. Summit submitted a proposal that aims to assess the impact of ezutromid on the modulation of utrophin expression in muscle fibres in a population of Becker muscular dystrophy patients. TACT welcomed the opportunity to review this application and its full report offers recommendations on future trial design, including selection of trial population and appropriate functional outcomes. Advice is also given on recommended further pre-clinical studies.

The committee felt that this orally-administered compound has a good safety and tolerability profile. They also advise the applicant on addressing:


  • the need to better understand the effect of existing Becker-like dystrophin on the mechanism of action of ezutromid
  • establishing the amount of utrophin needed to provide a clinical benefit
  • the challenges of conducting a clinical trial in a highly heterogeneous Becker population
  • the opportunity presented by any future study to better understand the natural history of Becker muscular dystrophy


To request a full copy of the report please contact the applicant Jon Tinsley: Jon.Tinsley@summitplc.com


Utrophin modulation as a potential treatment for Becker muscular dystrophy

21 Apr 2017