Post by Lucas on Oct 10, 2012 15:06:22 GMT -5
SUMMIT ANNOUNCES POSITIVE PHASE 1 TRIAL RESULTS FOR SMT C1100 FOR TREATMENT OF
DUCHENNE MUSCULAR DYSTROPHY
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 17(th) 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.
DUCHENNE MUSCULAR DYSTROPHY
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 17(th) 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.