A Targeted Anti-Cancer Therapy
RX-3117 is a novel, oral, small molecule nucleoside prodrug which is activated (phosphorylated) by UCK2 which is predominantly expressed in cancer cells. Once activated, it is incorporated into the DNA or RNA of cancer cells inducing apoptotic cell death. Because UCK2 is overexpressed in multiple human tumors but has a very limited presence in normal tissues, RX-3117 offers the potential for a targeted anti-cancer therapy with an improved efficacy and safety profile relative to other nucleoside analogues. RX-3117 has been shown to have efficacy in preclinical models and in clinical trials against tumors that have developed resistance to other nucleoside analogues such as gemcitabine. RX-3117 has therapeutic potential in a broad range of cancers, including pancreatic, bladder, colon, and lung cancer. RX-3117 has received "orphan drug designation" for pancreatic cancer from the U.S. Food and Drug Administration (FDA) and from the European Commission (EC).