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Summit completes patient enrollment in phase 2 trial of ezutromid in DMD

Summit Therapeutics has completed enrolment in PhaseOut DMD, a phase 2 proof of concept clinical trial of ezutromid, in Duchenne muscular dystrophy (DMD) patients.

PhaseOut DMD 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 has triggered a $22 million milestone payment as part of the Company's licence and collaboration agreement with Sarepta Therapeutics, Inc.

"Completing enrolment in PhaseOut DMD is a major step in the development of ezutromid as we seek to understand if this utrophin modulator has positive effects on muscle structure leading to changes in muscle health and function in patients with DMD," said Dr David Roblin, Chief Operating Officer and President of R&D of Summit.

"Utrophin modulation has potential as a universal treatment option for patients with DMD, and we look forward to the 24-week data readout expected in the first quarter of 2018."

PhaseOut DMD is a 48-week open-label trial that has enrolled 40 patients at sites in the UK and the US. As part of the trial, each patient undergoes two biopsies, a baseline biopsy on enrolment 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.