If you saw yesterday’s news about Summit’s – a UK drug company – findings, you may have some questions about what this means and is next. This report refers to Summit’s compound that the pharmaceutical company Biomarin had in trial.  The compound failed and Biomarin stopped the trial and made the decision not to go forward.   The compound, which is a small molecule, SMT C1100 (orally available) and suggested to upregulate utrophin, originated from the laboratory of Kay Davies in collaboration with Summit. In this case, the compound apparently upregulated utrophin in the mdx mouse, but in humans the compound did not have the same effect. Summit has a long commitment to utrophin upregulation and continues to believe in this compound. After analyzing the compound, it was thought that the drug needed to be reformulated  in order to achieve an effect  in humans. Keep in mind ADME (absorption, distribution, metabolism, excretion) varies from species to species, sometimes requiring different formulations of the compound to achieve an effect.  Since Biomarin made the decision not to move forward, Summit (led by Jon Tinsley) analyzed the data and decided that the failure of the compound was due  to formulation of the drug.  Lee Sweeney, Sharon Hesterlee, and others have had several discussions with Jon Tinsley (Summit) about reformulating SMT C1100. Summit has submitted a proposal to PPMD, based on reformulating the compound and demonstrating efficacy in animals, with the goal of advancing this reformulated compound into trial.


For more about Summit, please visit PPMD's newly revamped Research Section for more on this and other promising therapies in the pipeline.

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Comment by cheryl cliff on March 16, 2011 at 4:03pm
Got it, thanks again.  Fingers crossed for a Phase I safety trial to get here and fast. 

Comment by Pat Furlong on March 16, 2011 at 1:13pm

Hi Bob,  Both Utrohin and Dystrophin can be measured.  What is unknown is how the amount of dystrophin or utrophin expressed corelates to improvements in function.      We need biomarkers in DMD, something we are able to measure that translates into x% improvement.  CK is not a biomarker because it fluctuates all over the map.


Cheryl - Biomarin did not report any safety signals.  That does not mean there are none, just that they did not see any at the time the trial was halted.   It is wonderful that Jon Tinsley/Summit has not given up on the compound.  If it is reformulated and moves to trial, they would start with Phase I safety.

Comment by John Smith on March 16, 2011 at 12:06pm

Thank you Pat.  There seems to be great challenges measuring Dystrophin levels in the body.  Are Utrophin levels more readily measured?   If they are more readily measured, would this tend to shorten, expedite, or bolster the drug trial process?

Comment by cheryl cliff on March 15, 2011 at 12:12pm
Thanks for clearing up the confusion Pat.  And a BIG hug to Summit for continuing to work on utrophin upregulation!!!  But Iam unsure on something, Summit mentioned in their release that they had non-toxic results with testing on healthy volunteers.  So, is it safe to assume they were referring to the reformulated compound?

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