Summit Corporation Outlines Clinical Development Plans for Utrophin Modulator Program for Duchenne Muscular Dystrophy

Published: Mar 21, 2013

Oxford, UK, 21 March 2013 – Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy (‘DMD’) and C. difficile infections, today outlines its future plans for the continued development of utrophin modulators for the treatment of DMD. Utrophin modulation is a disease modifying approach that has the potential to treat all genetic forms of DMD. Summit’s programme includes the lead candidate SMT C1100 that is expected to enter clinical trials in patients during H2 2013, and earlier-stage next generation utrophin modulators that are being developed to add further value to the programme. Summit is presenting its utrophin modulator programme at a major international conference being held later this Spring.

“Summit has a unique opportunity to develop a high-value franchise in utrophin modulation, an innovative therapeutic approach for DMD that targets all genetic forms of this devastating disease,” commented Glyn Edwards, Chief Executive Officer of Summit. “These clinical trials will be the first to evaluate utrophin modulation in patients, and they aim to quickly establish clinical proof of concept for SMT C1100 through the use of novel biomarkers developed to measure aspects of muscle health. The biomarker work, to be conducted side by side with our clinical development programme, will strengthen our DMD franchise and will enhance the commercial value of this asset.”

The proposed proof of concept patient trial will include two components: a dose-finding study in DMD patients to confirm translation of safety, tolerability and pharmacokinetics from an adult to a paediatric population, followed by a Phase 2 trial that will include clinical markers of muscle health as well as levels of utrophin expression and other novel biomarkers. The biomarker programme has commenced and includes the collaboration with Children’s National Medical Center of Washington DC, funded by the DMD organisation, The Foundation to Eradicate Duchenne, which was announced by Summit in February 2013. Summit is now engaged with the regulatory authorities and expects the dose finding study to start in H2 2013.

In preparation for the clinical studies, drug material manufacture and long-term regulatory toxicology studies will be commissioned. Summit has selected a specialist contractor capable of manufacturing and formulating GMP grade drug material for use in the long-term toxicology studies, the patient proof of concept trials, and ultimately any potential future registration trials and market use.

The Company will present its utrophin modulation programme and biomarker development at the Muscular Dystrophy Association Scientific Conference, 21-24 April 2013, Washington DC, US. A copy of the presentation will be made available on the Company website at that time.

About DMD and Utrophin Modulation

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 target all genetic forms of DMD. SMT C1100 is the Company’s leading utrophin modulator drug and it has successfully completed a Phase 1 healthy volunteer clinical trial in late 2012.

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 and follow Summit on Twitter (@summitplc).

For more information, please contact:


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

Nomura Code Securities

(Nominated Adviser and Joint broker)

Chris Collins / Jonathan Senior / Giles Balleny

Tel: +44 (0)20 7776 1200

Hybridan LLP

(Joint broker)

Claire Louise Noyce / Deepak Reddy

Tel: +44 (0)207 947 4350

Peckwater PR

(Financial public relations, UK)

Tarquin Edwards Tel: +44 (0)7879 458 364

MacDougall Biomedical Communications

(US media contact)

Michelle Avery Tel: +1 781-235-3060

Forward Looking Statements

This announcement contains "forward-looking statements", including, but not limited to, statements about the discovery, development and commercialisation of programme assets. These forward-looking statements are statements based on the Company’s current intentions, beliefs and expectations, which include, among other things, the Company’s results of operations, financial condition, prospects, growth, strategies and the industry in which the Company operates. No forward-looking statement is a guarantee of future performance and actual results could differ materially from those expressed or implied in the forward-looking statements. Accordingly, readers should not place undue reliance on forward-looking statements or information. Forward-looking statements and information by their nature involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements, or industry results, to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements or information. These include but are not limited to: adverse results in clinical or preclinical development studies; delays in obtaining regulatory approval; failure to obtain patent protection for inventions; commercial limitations imposed by patents owned or controlled by third parties; being unable to secure partnership agreements to develop and commercialise programme assets; being unable to secure the necessary funding to conduct any proposed research and development studies; and the ability to retain and recruit key personnel. The Company expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statement contained in this announcement to reflect any changes in expectations with regard thereto or any changes in events, conditions or circumstances on which any such statement is based, except as required by applicable law.

Michelle Avery, Ph.D.

Associate Account Executive

MacDougall Biomedical Communications


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