Summit Corporation plc
('Summit' or 'the Company')
REPEAT DOSING OF SMT C1100 FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY MEETS
ENDPOINTS IN PHASE 1 CLINICAL TRIAL
* New formulation delivers drug levels that are predicted to significantly
increase utrophin production
* Summit to progress utrophin upregulator into next stages of development
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.