Summit Therapeutics plc (NASDAQ: SMMT LON:SUMM) has identified biomarkers to relate muscle regeneration and the presence of utrophin protein in patients with Duchenne (DMD) and Becker muscular dystrophy (BMD).
DMD is caused by the absence of dystrophin protein and it is believed utrophin modulation, a naturally occurring protein similar to dystrophin, could slow or even stop the progression of DMD.
Summit is developing for a treatment for DMD using utrophin modulator therapies and sponsored the study at the Institute of Child Health at UCL alongside children’s charity Joining Jack
“Our data show it is possible to reproducibly measure utrophin protein and distinguish between repairing and mature muscle fibres at the single fibre level in DMD and BMD muscle biopsies,” said Jenny Morgan, Professor in Cell Biology at the ICH.
“This has led to important observations correlating regeneration and disease severity and so these biomarkers have application in clinical trials to potentially monitor activity of utrophin modulator therapies such as Summit's SMT C1100," she added.
Summit has just started a phase II proof of concept study for its SMT C1100 treatment.