Summit Announces Publication on Biomarkers to Quantify Utrophin Protein and Regeneration of Muscle Fibres in DMD

OXFORD, United Kingdom, March 15, 2016 -- Summit Therapeutics plc (NASDAQ:SMMT) (AIM:SUMM), the drug discovery and development company advancing therapies for Duchenne muscular dystrophy (‘DMD’) and Clostridium 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 β-dystroglycan protein levels at the membrane of individual fibres. This observation is important as β-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.

Notes to Editors

About Joining Jack
Joining Jack was founded in 2012 by ex-Wigan Warriors Rugby League player Andy Johnson and his wife Alex, following the devastating news that their son, Jack, had been diagnosed with Duchenne Muscular Dystrophy (DMD). The money that Joining Jack raises, through the kind support and donations of others, goes to help relieve the needs of sufferers of DMD by funding research into methods of treatment and cure of the condition. For more information on Joining Jack, and to keep up to date with any charity news or events, please visit www.joiningjack.org, follow us on Twitter (@alljoinjack), or find us on Facebook – www.facebook.com/joiningjack

About Summit
Summit is a biopharmaceutical company focused on the discovery, development and commercialisation of novel medicines for indications for which there are no existing or only inadequate therapies. Summit is conducting clinical programmes focused on the genetic disease Duchenne muscular dystrophy and the infectious disease C. difficile infection. Further information is available at www.summitplc.com and Summit can be followed on Twitter (@summitplc).

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Forward-looking Statements
Any statements in this press release about Summit’s future expectations, plans and prospects, including but not limited to, statements about the clinical and preclinical development of Summit’s product candidates, the therapeutic potential of Summit’s product candidates, and the design, timing of initiation, and completion of and availability of data from clinical trials, and other statements containing the words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "would," and similar expressions, constitute forward looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation of future clinical trials, availability and timing of data from on-going and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials or preclinical studies will be indicative of the results of later clinical trials, expectations for regulatory approvals, availability of funding sufficient for Summit’s foreseeable and unforeseeable operating expenses and capital expenditure requirements and other factors discussed in the "Risk Factors" section of filings that Summit makes with the Securities and Exchange Commission including Summit’s Annual Report on Form 20-F for the fiscal year ended January 31, 2015.  Accordingly readers should not place undue reliance on forward looking statements or information. In addition, any forward looking statements included in this press release represent Summit’s views only as of the date of this release and should not be relied upon as representing Summit’s views as of any subsequent date. Summit specifically disclaims any obligation to update any forward-looking statements included in this press release.

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