CureFAKtor Pharmaceuticals, a privately-held biopharmaceutical company, has reported data demonstrating analogs of novel focal adhesion kinase (FAK) inhibitor CFAK-C4 disrupted FAK-vascular endothelial growth factor receptor 3 (FAK-VEGFR3) interaction and inhibited pancreatic tumor growth.

Data showed that oral small molecule FAK inhibitor C4 analogs disrupt FAK-VEGFR3 interaction and dramatically inhibit pancreatic tumor growth at low concentrations as single agents, as well as in combination with gemcitabine, demonstrating synergistic effect. In vivo, 30-day treatment with low doses of the four analogs led to massive reduction in tumor growth when treated as single agents (60%) and in combination with gemcitabine (80%) (P<0.05). The C4 analogs displayed enhanced potency, minimum toxicity and high specificity for the target site.

A previous CureFAKtor study pinpointed the site of interaction of VEGFR-3 and FAK to create the small molecule drug capable of disrupting signaling and causing death of many types of cancer cells.

CureFAKtor is planning a phase I clinical study of CFAK-C4 in combination with gemcitabine chemotherapy in 2012, which received Orphan Drug Designation by the FDA.