Rare Daily Staff

Summit Therapeutics said it would discontinue development of its experimental therapy to treat the progressive, rare, and fatal muscle disease Duchenne muscular dystrophy after it failed to meet its primary and secondary endpoints in a mid-stage clinical trial.

DMD is caused by a genetic mutation that results in a deficiency of dystrophin, a protein that is essential to healthy muscles. Summit Therapeutics had been developing its candidate ezutromid, an experimental drug that activates utrophin. Utrophin is a protein that serves a similar function to dystrophin during fetal development, but then shuts off.

The study, known as PhaseOut DMD, was a multi-center, open-label phase 2 clinical trial. A total of 40 boys with DMD were enrolled in the study with 38 completing the 48-week clinical trial. The primary endpoint was the change from baseline in magnetic resonance parameters related to the leg muscles. Biopsy measures evaluating utrophin and muscle damage were included as secondary endpoints.

Based on the outcome of the study, Summit said it is discontinuing its development of ezutromid and will be implementing cost reduction measures. Summit will now focus its operations on the development of its pipeline of new mechanism antibiotics.

The Company’s lead product candidate, ridinilazole, is expected to enter late-stage clinical trials for the treatment of C. difficile infection in the first quarter of 2019.

“These data come as a great disappointment to us and to all those living with DMD,” said Glyn Edwards, CEO of Summit. “While we believe utrophin modulation could still have a place in the treatment of DMD, it is clear that ezutromid is not providing a benefit for patients.”

The company said it plans to explore ways that information gathered as part of PhaseOut DMD can be made available to support other research activities in DMD for the benefit of the DMD community.

June 28, 2018
Photo: Glyn Edwards, CEO of Summit

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