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Oxford, UK – Summit Therapeutics plc , the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and
Ezutromid is an orally administered small molecule that is designed to modulate utrophin, a protein that is structurally and functionally similar to the dystrophin protein. Dystrophin is essential for the healthy function of all muscles but is missing in patients with DMD. Utrophin modulation is a potential disease-modifying approach that could treat all boys and young men with DMD, regardless of their underlying dystrophin gene mutation.
“Ezutromid has garnered considerable interest from the patient, family and healthcare communities, which we believe is due to its potential to slow or stop progression of DMD regardless of the underlying dystrophin gene mutation,” said Ralf Rosskamp, MD, Chief Medical Officer of Summit. “The enrolment of the first patient in PhaseOut DMD is a significant milestone in the clinical development of ezutromid, with the aim of studying long-term dosing of ezutromid in boys with DMD. We look forward to the possibility of demonstrating ezutromid’s effect on utrophin with the initial set of 24-week biopsy data.”
The Company anticipates reporting data periodically during this trial with 24-week muscle biopsy data from the first group of patients enrolled expected to be reported in January 2017.