Following closely on the heels of the good news about Sarepta’s exon 51 skipping strategy, PPMD is pleased to report that Summit PLC has been able to achieve sufficient plasma levels of its reformulated utrophin-upregulating drug to justify a phase I/II trial in Duchenne (Read Summit's Press Release). At PPMD we have always believed in “hedging our bets” and investing widely in different therapies, some of which are mutation specific and some of which are not. Unlike exon-skipping strategies, upregulating utrophin is not a mutation specific strategy and, if effective, could potentially benefit anyone with Duchenne. To date we have funded the screening and identification of a utrophin upregulating compound at PTC Therapeutics, which is now in the preclinical validation stage; Tivorsan’s biglycan compound which seems to work by concentrating utrophin at the muscle cell membrane; and we contributed $250,000 to Summit PLC to fund the manufacture of its reformulated utrophin-upregulating compound SMT-C1100. Some of you may recall that the SMT-C1100 had been acquired by Biomarin who gave the drug back after they were unable to achieve high enough plasma levels of the drug with the original formulation and Summit struggled to find support to reformulate the drug and try again.
The Summit PLC project is unique in that so many different funders in the Duchenne world came together to support it and we all worked together to share our reviews and information (with full permission from the company). Other significant funders of this project included Charley’s Fund, Cure Duchenne, the Foundation to Eradicate Duchenne, the Muscular Dystrophy Association, and the Nash Avery Foundation. Never doubt that this community can make a difference—a phase I/II Duchenne trial with this drug is only possible due to the support of the community.
Sharon Hesterlee, Ph.D.
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