Summit Therapeutics (SMMT) said Monday it has completed enrollment in PhaseOut DMD, a phase 2 proof of concept clinical trial of the utrophin modulator, ezutromid, in patients with Duchenne muscular dystrophy (DMD). The study aims to provide proof of concept for ezutromid through measures of a number of endpoints related to muscle structure, health and function. The Company believes the trial could provide valuable insight into utrophin modulation as a potential disease-modifying treatment for all patients with DMD, regardless of the underlying dystrophin mutation.
With the dosing of the last patient in the trial, the company said it has triggered a $22 million milestone payment as part of its licence and collaboration agreement with Sarepta Therapeutics (SRPT). The 48-week open-label trial has enrolled 40 patients at sites in the UK and the U.S. As part of the trial, each patient undergoes two biopsies, a baseline biopsy on enrollment and a second either at 24 or 48 weeks. In the first quarter of 2018, Summit expects to report 24-week biopsy analysis from approximately 20 patients, as well as 24-week MRI and functional data from all 40 patients enrolled in the trial. Top-line data from the complete 48-week trial are expected in the third quarter of 2018.