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.