Signaling pathways are the means by which cells exchange instructional messages that regulate specific biological functions. Early in prenatal development, the instructional messages that direct the formation of tissues and organs are controlled by certain developmental signaling pathways, including the Hedgehog, bone morphogenetic protein, or BMP, and other pathways, which act by initiating cascades of gene signaling required for tissue formation and regulation. The body also uses these developmental signaling pathways to repair damage and regenerate tissues. For example, in damaged nerve tissue, our preclinical models demonstrate that activation of the Hedgehog pathway promotes repair and regeneration of nerve function, in part, by inducing the activation of a cascade of secondary signaling that promotes the growth of new cells and blood vessels.

The ability to modulate certain signaling pathways is of great interest to biotechnology and pharmaceutical companies as many diseases and disorders are now known to be associated with components of these signaling pathways. We are also using our signaling pathway-based preclinical drug development experience in our efforts to develop drug candidates that seek to target additional signaling pathways. For example, our Targeted Cancer Drug Development Platform consists of several proprietary cancer drug programs that target multiple signaling pathways other than the Hedgehog pathway. Our programs under this platform are primarily focused upon developing a number of proprietary small molecule inhibitor drug compounds, including CUDC-101.  Each compound is designed to inhibit one or more clinically validated cancer targets, that include both kinases and non-kinases.  We believe that focusing on clinically validated cancer targets should create a faster, less expensive and less risky development path since many of the specific cancer indications and drug profiles have already been well defined by clinical trials and/or marketed drugs.