Oxford, UK, 10 October 2012 – Summit (AIM: SUMM), a UK drug discovery company, announces
positive top-line results from a Phase 1 clinical trial of SMT C1100 for the treatment of Duchenne
Muscular Dystrophy (‘DMD’), a fatal muscle wasting disease for which there is currently no cure. SMT
C1100, an oral small molecule, is a potential disease modifying drug that works to increase or
upregulate the amount of a naturally occurring protein called utrophin. These data will be presented at
the 17th Annual Congress of the World Muscle Society, 9-13 October 2012, Perth Australia.
The Phase 1 dose-escalating trial was conducted in healthy volunteers and evaluated a new aqueous
formulation of SMT C1100. The trial met its primary endpoints with results showing the formulation to
be safe and well tolerated at all doses. Importantly, the new formulation also demonstrated improved
levels of bioavailability (absorption) of the drug that were above those anticipated to be needed to
achieve clinical efficacy. These results are strongly supportive for the progression of SMT C1100 into
patient clinical trials.
“The positive outcome from this Phase 1 trial is a significant step forward for DMD and our utrophin
upregulator drug SMT C1100,” commented Glyn Edwards, Chief Executive Officer of Summit.
“Utrophin upregulation is an important mechanism for treating DMD as it is expected to have broad
applicability for all patients regardless of the specific genetic fault causing their disease. We are highly
encouraged that the new formulation of SMT C1100 shows improved bioavailability as we believe it
supports the progression of this potential breakthrough treatment into DMD patient clinical trials.”
The double-blind, placebo-controlled trial examined single and multiple ascending oral doses of a
nanoparticle aqueous suspension formulation of SMT C1100 in a total of 48 healthy volunteers. When
single doses were increased from 50mg/kg up to 400mg/kg, SMT C1100 blood plasma levels were
increased for several hours above those required to give a 50% increase in utrophin levels in vitro
measured in cells taken from DMD patients. In addition, the new formulation resulted in higher blood
plasma concentrations of SMT C1100 when compared with results from a previous Phase 1 healthy
volunteer study that used a different formulation. Analysis of the multiple ascending dose groups
remains on-going and results are expected to be reported later this year.
A copy of the presentation being given at the World Muscle Society Congress will be available from
Summit’s website, www.summitplc.com.
The Phase 1 trial has been supported by a group of US DMD foundations: the Muscular Dystrophy
Association, Charley’s Fund, Cure Duchenne, the Foundation to Eradicate Duchenne, Nash Avery
Foundation and Parent Project Muscular Dystrophy.
About SMT C1100
SMT C1100 is designed to upregulate and maintain the production of utrophin. Utrophin is a protein
that is highly expressed in foetal and regenerating muscle but decreases as the muscle fibre mature
and is eventually replaced by dystrophin, a similar protein that maintains the integrity and healthy
function of muscles. Patients with DMD are unable to make dystrophin, resulting in muscle fibre
degeneration. However, if utrophin is continually expressed in the mature fibre, it can functionally
replace dystrophin and is expected to overcome the deficit in patients with DMD. This approach is
expected to be a universal treatment for all DMD patients regardless of whether the disease was
caused by an inherited or spontaneous mutation. Summit has demonstrated in non-clinical efficacy
studies that SMT C1100 is capable of increasing utrophin to restore and maintain the healthy function
of muscles including the heart and diaphragm. SMT C1100 has been granted orphan drug status in
Europe and the US.
Summit is an Oxford, UK based drug discovery 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:
Glyn Edwards / Richard Pye
Tel: +44 (0)1235 443 951
Singer Capital Markets
(Nominated Adviser and Joint broker)
Shaun Dobson / Jenny Wyllie
Tel: +44 (0)203 205 7500
Claire Louise Noyce / Deepak Reddy
Tel: +44 (0)207 947 4350
(Financial public relations, UK)
Tel: +44 (0)7879 458 364
MacDougall Biomedical Communications
(U.S. media contact)
Tel: +1 781-235-3060
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