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Summit publishes biomarkers to quantify utrophin protein and regeneration of muscle fibres in DMD

SUMMIT Therapeutics plc (NASDAQ: SMMT, AIM: SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy ('DMD') andClostridium difficile infection, announces the online publication on the development of imaging techniques designed to quantify utrophin protein and muscle fibre regeneration in muscle biopsies from patients with DMD and Becker muscular dystrophy ('BMD'). 

This collaborative research was conducted on Summit's behalf by biomarker experts, Professor Jenny Morgan, Professor Caroline Sewry and Professor Francesco Muntoni at the Institute of Child Health, University College London with financial support from the UK charity Joining Jack.

"Summit Therapeutics' approach to DMD aims to maintain expression of utrophin, a protein typically found in developing and repairing muscle fibres, so that it can replace the missing dystrophin protein in mature muscle fibre," commented Professor Jenny Morgan, Professor in Cell Biology, the Dubowitz Neuromuscular Centre at the University College London Institute of Child Health. "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.  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."

The research was published in the peer reviewed journal PLoS ONE in the paper entitled "Correlation of utrophin levels with the dystrophin protein complex and muscle fibre regeneration in Duchenne and Becker muscular dystrophy muscle biopsies." The developed assays allowed the absolute quantification of regenerating muscle fibres within a biopsy section, and for the first time, the researchers observed a correlation between the percentage of regenerating muscle fibres with differences in clinical severity between patients with DMD and BMD from whom the biopsy was taken. The results also showed a significant correlation between utrophin and B-dystroglycan protein levels at the membrane of individual fibres. This observation is important as B-dystroglycan (a member of the dystrophin associated protein complex) is required to bind to utrophin or dystrophin in healthy muscle fibres to maintain function.

Alex Johnson from Joining Jack added, "Our mission is to provide support towards the development of promising new therapies for DMD that can make a difference to our boys and families living with this disease on a daily basis. We are pleased to have supported this important work on development of new biomarkers and look forward to them being available for use in future trials of utrophin modulator treatments."

A copy of the publication is now available from Summit's website: www.summitplc.com/publications.

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