Oxford, UK, 7 November 2012 – Summit (AIM: SUMM), a UK drug discovery company, announces
that the repeat dosing of the utrophin upregulator SMT C1100 for the treatment of the fatal muscle-
wasting disease Duchenne Muscular Dystrophy (‘DMD’) has successfully met the endpoints as part of
a Phase 1 clinical trial in healthy volunteers. The trial evaluated a new formulation of SMT C1100 and
the results showed that upon repeat dosing, concentrations of the drug achieved in the blood plasma,
stabilised at levels that from preclinical studies are expected to significantly increase utrophin protein
production. The new formulation was also shown to be safe and well-tolerated in this Phase 1 trial.
SMT C1100 is a potential disease-modifying, oral small-molecule that works by upregulating
(increasing) the amount of a naturally occurring protein called utrophin to maintain the healthy function
of muscles. These data strongly support the progression of SMT C1100 into the next stages of
development that includes biomarker and long-term safety studies, which will be required before a
DMD patient efficacy trial could commence. The latest results will be presented at the 2012 Action
Duchenne Conference, 9-10 November, London UK.
“Utrophin upregulation is a unique approach for treating DMD because it could benefit all DMD
patients, regardless of their underlying genetic fault,” commented Glyn Edwards, Chief Executive
Officer of Summit. “We are highly encouraged by these results, as the new formulation achieves
blood concentrations that have the potential to significantly increase utrophin levels, with the outcome
of maintaining the healthy function of muscles in patients with DMD. The results therefore strongly
support continuing clinical evaluation of SMT C1100.”
The double blind, placebo-controlled Phase 1 trial examined a new nanoparticle aqueous suspension
of SMT C1100 in a total of 48 healthy volunteers. The previously reported results from the single
ascending dose cohort showed SMT C1100 to be safe and well-tolerated at all doses. These new data
are being reported from the repeat dosing cohort where the volunteers received 100mg/kg twice daily
for nine days. These results show that in all volunteers the blood plasma concentration of SMT C1100
stabilised after four days of dosing above the required level expected to increase utrophin protein
production by 50% for at least 14 hours a day in a preclinical model. The plasma levels achieved were
equivalent to those that gave significant therapeutic benefit in the gold standard disease model of
DMD.
A copy of the presentation being given at the Action Duchenne conference will be available on
Summit’s website after the event.
The Phase 1 trial has received funding from 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 & Utrophin Upregulation
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 switching utrophin production back-on 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.
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 and follow Summit on Twitter (@summitplc).
For more information, please contact:
Summit
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
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
tarquin.edwards@peckwaterpr.co.uk
MacDougall Biomedical Communications
(U.S. media contact)
Michelle Avery
Tel: +1 781-235-3060
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