SUMMIT ANNOUNCES FIRST PATIENT DOSED IN A PHASE 1B CLINICAL TRIAL OF SMT C1100 FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

SUMMIT ANNOUNCES FIRST PATIENT DOSED IN A PHASE 1B CLINICAL TRIAL OF SMT C1100 FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY 

Oxford, UK, 9 December 2013 – Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy (‘DMD’) and C. difficileinfection, announces that the first DMD patient has been enrolled and dosed in a Phase 1b clinical trial of the oral, small molecule utrophin modulator SMT C1100.  

“SMT C1100 is a promising treatment that has the potential to improve the life of all patients with DMD, irrespective of the underlying dystrophin fault,” commented Dr David Roblin, Summit’s Chief Medical Officer.  “There is an urgent need to develop effective medicines for DMD patients and this study forms an important step in our clinical plans towards establishing SMT C1100 and utrophin modulation as a viable treatment that could slow or even stop the progression of this devastating condition.”

About the Phase 1b Clinical Trial
The Phase 1b trial is a dose-escalating, open-label study that is being conducted in paediatric patients with DMD.  The primary endpoint of the trial is the evaluation of the safety and tolerability of SMT C1100. The study will also measure blood plasma concentration levels of SMT C1100 to determine the dose to be used in a subsequent Phase 2 proof of concept efficacy trial.  

The trial is enrolling 12 patients aged between 5 and 11 years, divided equally into three dose cohorts.  Each cohort will receive daily oral doses of SMT C1100 for a total of ten days with a review taking place after each cohort has completed dosing.  The trial is being conducted at up to four NHS hospitals in the UK with the Chief Investigator being Professor Francesco Muntoni at Great Ormond Street Hospital, London.

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Notes to Editors

About DMD, Utrophin Modulation and SMT C1100
DMD is a progressive muscle wasting disease that affects around 50,000 boys in the developed world.  It is caused by different faults in the gene that encodes dystrophin, a protein that is essential for the healthy function of all muscle.  There is currently no cure for DMD and life expectancy is around mid-twenties.  Utrophin protein performs essentially the same functional role as dystrophin in developing muscle fibres and studies have shown that maintaining utrophin expression has the potential to slow down or even stop the progression of DMD, regardless of the underlying dystrophin mutation.  It is also expected to be complementary to other therapeutic approaches in clinical trials.  SMT C1100, an orally administered small molecule, is Summit’s most advanced utrophin modulator.  Non-clinical studies showed SMT C1100 increases utrophin protein levels in skeletal and cardiac muscle resulting in a significant reduction in the dystrophic muscle pathology.  SMT C1100 has successfully completed a Phase 1 trial in healthy volunteers with the drug shown to be safe and achieved expected therapeutic blood levels.

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 atwww.summitplc.com and follow Summit on Twitter (@summitplc).