KX2-391 is a new cancer drug that is in Phase 1 clinical trials and was discovered and is being developed by Kinex Pharmaceuticals (Applicant). During the Phase 1 trial, plasma, white blood cells (PBMC's) and tumor biopsy tissue will be collected. These clinical samples will eventually be evaluated for KX2-391 related biomarkers which will provide tools to monitor the efficacy of the drug and to potentially identify patients who are most likely to benefit from this treatment. In addition, these biomarkers will be helpful in designing Phase 2 and Phase 3 clinical trials, and monitoring their success. The experiments proposed in this grant application are focused on identifying and validating KX2-391 associated biomarkers by first using mouse models for various human cancers such as colon, pancreas, prostate and breast. KX2-391 has been shown to be efficacious in mouse models for these types of cancer and these types of cancer are being considered for Phase 2 clinical trials. Once potential biomarkers are identified in the animal studies, they will be used to analyze human samples. The goal of biomarker research is to identify genes or proteins that can be used to detect disease, monitor progress to a therapy, monitor treatment compliance, determine treatment efficacy, predict response to treatment and to stage disease. These goals are certainly ambitious;however the potential payoff would be tremendous. Funding research to provide these benefits to patients is clearly warranted.
The research that is proposed in this grant application will support the further development of a novel cancer drug that has been shown to inhibit both primary tumor growth and metastasis. The drug is currently in Phase 1 clinical trials where safety and pharmacokinetics are being evaluated as primary endpoints. Tissue samples from patients in the Phase 1 trial are being procured for the development of biomarker tests to measure drug efficacy and to identify potential patient populations who are sensitive to this drug.