Assuming phase 1 is a success, how can parents help encourage Summit to consider US sites for phase 2?
******** PRESS RELEASE ***************
Today's press release announced that the manufacture of a new formulation of SMT C1100 is currently on-track and Summit plan to apply to the Medicines and Healthcare products Regulatory Agency (MHRA) for permission to conduct a phase 1 clinical trial by the end of next month. If permission is granted, a phase 1 trial in healthy volunteers will commence with results expected by the end of September.
The Muscular Dystrophy Campaign has funded Professor Dame Kay Davies' research into utrophin for more than 25 years and in collaboration with Summit they discovered and developed SMT C1100. Research has shown that treating a mouse model of Duchenne muscular dystrophy with SMT C1100 resulted in increased muscle strength and muscles that didn't tire as easily.
SMT C1100 was recently tested in a phase 1 clinical trial by US pharmaceutical company BioMarin, but unfortunately even at high doses only small amounts entered the bloodstream. Summit plc has since reformulated SMT C1100 into a form that should be better absorbed and therefore reach the muscles at the levels thought to be required to improve muscle function. The planned phase 1 trial will evaluate how well this new formulation of the drug is absorbed and will also monitor the safety of the drug.
A successful outcome from the phase 1 trial could lead to a phase 2 study in Duchenne muscular dystrophy patients starting in 2013.
The start of this clinical trial is especially exciting because, unlike other approaches such as exon skipping, this drug has the potential to treat all boys with Duchenne muscular dystrophy, regardless of what genetic mutation they have.
I just wanted to let you know that I've flagged this as a follow up discussion for PPMD, but there may be a delay in response time because both Pat and Sharon are attending the Duchenne Parent Project (Italy) meeting this week into next. You can expect a reply by the middle of next week!