Summit Therapeutics has announced that the clinical development program for ezutromid for the treatment of  Duchenne muscular dystrophy (DMD) is being discontinued as the treatment did not meet its primary or secondary endpoints.

Ezutromid, an oral utrophin modulator, was assessed in the 48-week PhaseOut DMD trial which included 40 boys with DMD. The primary endpoint was the change from baseline in magnetic resonance parameters related to the leg muscles. The secondary endpoint consisted of biopsy measures evaluating utrophin and muscle damage, with patients having 2 biopsies, 1 at baseline and a second at either 24- or 48-weeks. Statistical decreases in developmental myosin and magnetic resonance T2 measures were seen after 24 weeks of treatment, but were not seen after 48 weeks. 

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“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,” said Glyn Edwards, CEO of Summit. The Company will now discontinue development of ezutromid and work with trial investigators to bring the current trial and extension to conclusion. They stated that the information gathered in the trial will be made available for further research into DMD. 

Ezutromid was granted Orphan Drug designation and Fast Track status for an expedited review by the Food and Drug in 2016.

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