Positive Phase 1 trial results for SMT C1100 in Duchenne (Summit PLC)

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. 

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Sharon Hesterlee, Ph.D.
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Comment by amit gupta on December 13, 2012 at 11:41am

sharon, i thought they said they have to do a long-term safety study (12 months) in animals first before they can plan a next trial in dmd boys...if that is is true then a more realistic estimate of a trial in boys would be 2015.

Comment by Sharon Hesterlee on December 13, 2012 at 10:01am

Denise--they were a bit vague about time lines because they are still making decisions about how to structure the first study and putting funds together, but it will be in boys with DMD.  As Stephanie said, the Webinar will be available later today.


Comment by Stephanie Matthes on December 13, 2012 at 9:22am

Hi Denise -- We should have the webinar available online by the end of the day. I'll send you the link when it's ready!

Comment by Denise Bretsik on December 13, 2012 at 7:30am


I missed the webinar on the 11th. Has it been posted yet? I would really like to hear it. Did they talk about when and where they will start the next phase?

Thank you!

Comment by Sharon Hesterlee on November 2, 2012 at 3:27pm

Hi Denise--not yet.  I know that Summit is in the process of gathering data to figure out how best to design that first study.  Let me see if they will share their time line and I will get back to you (will post here)


Comment by Denise Bretsik on November 2, 2012 at 2:02pm

Hello, Sharon,

Has there been any updates on when the next clinical trial will begin or how long the next trial my take?


Comment by Sharon Hesterlee on October 22, 2012 at 4:48pm

Hi Amit--usually if there is no safety data available in children then you would do a phase I/II rather than a phase II.  It's almost academic in a way since most studies with new drugs will go through at least two phase II studies regardless of whether one of them is technically a phase I/II study.  What I'm trying to say is that I'm not sure it really adds to the time it takes to approve the drug in the long run--safety is just a bigger endpoint when it's a phase I/II.

Comment by amit gupta on October 22, 2012 at 4:23pm

sharon, what i asked was whether any drug that is intended for children (except gene therapy/stem cell type) needs to go thru pahse 1 (healthy adult trial for safety) and then again phase 1 in children, like the smtc trial that went thru phase 1 and you said needs to go thru phase 1/2, not just proceed to phase 2..

Comment by Sharon Hesterlee on October 22, 2012 at 2:08pm

Yes, good article.  And the short answer to your question Amit is no, not all drugs go through a phase I in healthy volunteers.  Any drug that would result in a healthy person being exposed to excessive risk or which would make a healthy person less healthy just by the nature of the intervention would not be tested in a traditional phase I.  For example, exon skipping drugs are not tested in healthy volunteers because you would be taking a full length dystrophin and making it less functional for those volunteers, potentially giving them Becker-like symptoms.  Also, gene therapy studies usually don't use healthy volunteers because you are adding in a copy of a healthy gene that isn't needed in a healthy volunteer--you might risk symptoms from making too much of that gene product.  But drugs that are not likely to alter the function of genes or processes in healthy people in a negative way by viture of how they are designed to work typically are tested in healthy volunteers first.  And as you suggest, the FDA prefers that drugs be tested in adults first before children, when possible.  Does that help?

Comment by Conrad Klahn on October 22, 2012 at 1:53pm

Here is a good article from the American Cancer Society explaining the Clinical Trial Phases:


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