Summit Corporation plc
('Summit' or 'the Company')
SUMMIT REPORTS PRELIMINARY RESULTS FROM PHASE 1B CLINICAL TRIAL OF SMT C1100 FOR DUCHENNE MUSCULAR DYSTROPHY
Oxford, UK, 21 May 2014 - Summit (AIM: SUMM), a drug discovery and development company advancing therapies for Duchenne Muscular Dystrophy ('DMD') and C. difficile infection, announces that SMT C1100 for the treatment of DMD has successfully met its primary endpoint of safety and tolerability in a Phase 1b clinical trial in patients with the disease. SMT C1100 is an oral small molecule utrophin modulator that has the potential to treat all patients with DMD, regardless of the underlying dystrophin fault causing the disease.
The Phase 1b dose-escalating trial was conducted in 12 patients with DMD aged between 5 and 11 years old. These preliminary results show that SMT C1100 was safe and well tolerated at all doses tested in the study, and that there were no issues with patient compliance. All the boys had variable blood plasma concentrations of SMT C1100 with only two of the boys achieving concentrations similar to those of the adult volunteers in the 2012 Phase 1 study. Initial evidence suggests that the variability in drug uptake may be due to differences in diet and to other disease-related factors.
The non-placebo controlled trial also measured creatine kinase ('CK') levels, an enzyme that is associated with muscle fibre damage and elevated in boys with DMD. In the majority of patients there was a reduction in CK levels during dosing with SMT C1100. These data are consistent with non-clinical in vivo efficacy studies in the mdx model of DMD that showed SMT C1100 reduced CK levels after only 15 days.
"This first-ever trial of a utrophin modulator in DMD patients demonstrated the excellent safety profile of SMT C1100 with the study successfully achieving its primary endpoint," commented Glyn Edwards, Chief Executive Officer of Summit. "In addition, the positive impact on the enzyme markers of muscle health was both unexpected and exciting with these data potentially representing SMT C1100's first signs of activity in DMD patients."
These preliminary trial data will be reviewed further by Summit and is expected to lead to a revision of future clinical trial plans in order to determine the optimal way, either through dietary means or drug formulation changes, to address the drug uptake differences between DMD patients and healthy volunteers. The next patient study is now expected to start in Q4 2014.
Glyn Edwards continued, "Our goal with utrophin modulation is to treat all patients with DMD. Armed with a greater understanding of the importance of diet and other disease related factors, our future clinical trial plans will be modified and will increase the potential likelihood of achieving this goal."
A webcast presentation by Glyn Edwards on the preliminary Phase 1b results is available by clicking on the following link: http://www.brrmedia.co.uk/event/123676?popup=true
About the Phase 1b Clinical Trial
The Phase 1b trial was a dose-escalating, open-label study conducted in paediatric patients with DMD to evaluate safety, tolerability and drug exposure. The trial enrolled 12 patients aged between 5 and 11 years, divided equally into three dose cohorts. Each cohort received daily oral doses of SMT C1100 for a total of ten days with a dose escalating safety review taking place after each cohort had completed dosing. The trial was conducted at four NHS hospitals in the UK with the Chief Investigator being Professor Francesco Muntoni at Great Ormond Street Hospital, London. Further information about the trial is available at www.clintrial.gov.
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Notes to Editors
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 and follow Summit on Twitter (@summitplc).
For more information, please contact:
Glyn Edwards / Richard Pye
|Tel: +44 (0)1235 443 951|
| Cairn Financial Advisers LLP|
Liam Murray / Tony Rawlinson
|Tel: +44 (0)20 77148 7900|
| N+1 Singer |
Aubrey Powell / Will Goode
|Tel: +44 (0)20 7496 3000|
| Peckwater PR|
(Financial public relations, UK)
Tel: +44 (0)7879 458 364
| MacDougall Biomedical Communications|
(US media contact)
|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.