Department of Chemistry

Department of Chemistry University of Oxford

Exciting interim data from Summit's PhaseOut DMD clinical trial

Exciting interim data from Summit

Summit Therapeutics plc, an Oxford spin-out company based on pioneering work by Professor Steve Davies, Professor Angela Russell and Professor Dame Kay Davies (Department of Physiology, Anatomy and Genetics; DPAG) has announced that its candidate drug, ezutromid, currently in a Phase II clinical study for the treatment of Duchenne Muscular Dystrophy (DMD), has significantly reduced muscle damage in patients with DMD. The findings come after evaluation of the trial's 24-week interim data. DMD is a devastating disease caused by the absence of a large protein called dystrophin in all muscle cells; patients are generally wheelchair bound by the age of 12 and die in their twenties. At present there is no effective treatment, but Summit's approach is to identify a drug that will increase the amount of the dystrophin-related protein utrophin in muscle. The latest results with ezutromid have shown a statistically significant and meaningful reduction in muscle damage as measured by a 23% decrease in mean developmental myosin (biomarker of muscle damage) in muscle biopsies at 24 weeks compared to baseline. Further, a total of 14/22 patients showed a decrease in development myosin, with five of those showing a greater than 40% reduction. The research team here at the Chemistry Department in collaboration with DPAG and Summit Therapeutics as part of the UtroDMD Alliance is currently building on the exciting ezutromid data to develop second and third generation utrophin modulators, thus driving pre-clinical studies towards developing a best-in-class therapy for DMD patients worldwide. more........