SUMMIT AND CHILDREN'S NATIONAL MEDICAL CENTER ENTER UTROPHIN BIOMARKER 

COLLABORATION FOR DUCHENNE MUSCULAR DYSTROPHY 

  * Collaboration Supported by Grant from Foundation to Eradicate Duchenne 

 

Oxford, UK, 6 February 2013 - Summit (AIM: SUMM), a drug discovery and 

development company advancing therapies for Duchenne Muscular Dystrophy ('DMD') 

and C. difficile infections, announces that it has entered into a collaboration 

with Dr Yetrib Hathout from Children's National Medical Center in Washington DC, 

for the development of utrophin biomarkers for DMD.  The collaboration is being 

financially supported by a grant from the Foundation to Eradicate Duchenne and 

is part of a comprehensive biomarker programme being undertaken by Summit to 

advance its utrophin modulator programme for DMD.  In late 2012, Summit reported 

that in a Phase 1 trial in healthy volunteers, its lead candidate SMT C1100 was 

safe and well-tolerated. 

 

"We are delighted to be working with Dr Hathout on developing new biomarkers 

that will help advance our understanding of DMD while supporting the progress of 

Summit's utrophin modulator programme," commented Glyn Edwards, Chief Executive 

Officer of Summit.  "Developing biomarker indicators capable of accurately 

measuring utrophin protein levels in muscle will be vital in helping to confirm 

the activity of our clinical candidate SMT C1100 in future patient trials.  We 

thank the Foundation to Eradicate Duchenne for their continuing support in 

advancing this important medical research." 

 

Joel Wood, President of the Foundation to Eradicate Duchenne added, "We are 

committed to ensuring that urgently needed treatments have the best possible 

chance of successfully progressing through clinical trials. As such, we are 

pleased to provide funding to support Summit's utrophin modulation programme, 

which is a novel and promising approach for treating all genetic forms of DMD." 

 

DMD is caused by genetic mutations that prevent patients from making the 

structural protein dystrophin, which leads to progressive muscle wasting and is 

ultimately fatal.  Summit is pioneering utrophin modulation to stimulate 

production of utrophin, a functionally similar protein to dystrophin that is 

expressed in foetal and regenerating muscle, and which has the potential to 

restore and maintain healthy muscle function.  This disease modifying approach 

would benefit all DMD patients, regardless of the underlying genetic fault 

causing their illness.  SMT C1100 is the Company's leading utrophin modulator 

drug and successfully completed a Phase 1 clinical trial in late 2012. 

 

The development of new biomarkers that accurately quantify utrophin protein 

levels in DMD muscles will play an important role in providing evidence for the 

potential effectiveness of Summit's utrophin modulator drugs in future patient 

clinical trials.  Dr Hathout, a Principal Investigator at the Center for Genetic 

Medicine Research at Children's National, will apply his expertise in cutting- 

edge proteomic techniques to develop a sensitive, robust mass-spectrometry based 

assay that can quantitatively measure utrophin protein levels in biopsies of DMD 

muscle.  This collaboration is part of Summit's comprehensive biomarker 

programme developing a range of assays that will measure biological endpoints to 

demonstrate muscle benefit after treatment with small molecule utrophin 

modulators. 

 

                                    - END - 

 

Notes to Editors 

 

About Children's National Medical Center 

Children's  National  Medical  Center,  located  in  Washington, DC, is a proven 

leader in the development of innovative new treatments for childhood illness and 

injury.  Children's has  been serving  the nation's  children for more than 135 

years.  Children's National is proudly ranked among the best pediatric hospitals 

in  America  by  US  News  &  World  Report  and  the  Leapfrog  Group. For more 

information, visit www.childrensnational.org. Children's Research Institute, the 

academic   arm   of   Children's   National   Medical  Center,  encompasses  the 

translational,  clinical,  and  community  research  efforts of the institution. 

Learn more about our research programs at www.childrensnational.org/research. 

 

About Foundation to Eradicate Duchenne 

The  Foundation to Eradicate Duchenne Muscular Dystrophy ('FED') is a non-profit 

organisation founded in 2002 to provide funding into research for treatments and 

ultimately a cure for Duchenne Muscular Dystrophy ('DMD').  THE FED raises money 

to  support  the  world's  only  clinical  trials network for DMD.  The clinical 

trials network is known as CINRG and works with scientists all over the world on 

therapies for DMD. 

 

About Summit 

Summit is an Oxford, UK based drug discovery and development Company targeting 

high-value areas of unmet medical need including Duchenne Muscular Dystrophy and 

C. difficile infection. Summit is listed on the AIM market of the London Stock 

Exchange and trades under the ticker symbol SUMM. Further information is 

available at www.summitplc.com. 

 

 

For more information, please contact: 

 

 Summit 

 Glyn Edwards / Richard Pye                   Tel: +44 (0)1235 443 951 

 

 

 Nomura Code Securities 

 (Nominated Adviser and Joint broker) 

 Chris Collins / Jonathan Senior / Giles      Tel: +44 (0)20 7776 1200 

 Balleny 

 

 

 Hybridan LLP 

 (Joint broker) 

 Claire Louise Noyce / Deepak Reddy           Tel: +44 (0)207 947 4350 

 

 

 Peckwater PR 

 (Financial public relations, UK)             Tel: +44 (0)7879 458 364 

 Tarquin Edwards                              tarquin.edwards@peckwaterpr.co.uk 

 

 

 MacDougall Biomedical Communications 

 (US media contact)                           Tel: +1 781-235-3060 

 Michelle Avery 

 

 

 

Forward Looking Statements 

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U.S. Private Securities Litigation Reform Act of 1995. Forward-looking 

statements can be identified by words such as "anticipates", "intends", "plans", 

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regarding our business, the economy and other future conditions. Because 

forward-looking statements relate to the future, by their nature, they are 

subject to inherent uncertainties, risks and changes in circumstances that are 

difficult to predict. The Company's actual results may differ materially from 

those contemplated by the forward-looking statements. The Company cautions you 

therefore that you should not rely on any of these forward-looking statements as 

statements of historical fact or as guarantees or assurances of future 

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conditions. 

 

 

 

 

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Source: Summit Corporation PLC via Thomson Reuters ONE 

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