Pancreatic Ductal Adenocarcinoma (PDAC) is the most common and deadliest form of pancreatic cancer. It is an extremely difficult-to-treat disease with a median survival of less than 1 year and a dismal 5-year survival rate of 3-5%. This is primarily due to a rapid dissemination of the tumors and a lack of effective biomarkers for early detection which means that in general, the cancer is fairly advanced when initially diagnosed. Currently, the chemotherapeutic drug gemcitabine is the first-line treatment for PDAC. However, gemcitabine does not significantly prolong survival and patients commonly develop chemoresistance and relapse. Thus, there is an urgent need for the identification of effective, new therapeutic strategies for the treatment of PDAC. BioTheryX is developing a novel chemotherapeutic that inhibits Wnt signaling and eukaryotic translation initiation factor 5A (eIF5A) activity for the treatment of pancreatic ductal adenocarcinoma (PDAC). Preliminary data suggest the compound is well tolerated in mice. The goal of this Phase I proposal is to demonstrate efficacy of this compound in an orthotopic PDAC animal model and advancing the compound to a Phase II for additional preclinical studies with the ultimate goal of filing an IND a the end of Phase II.
The goal of this program is to develop and ultimately commercialize a novel small molecule drug for the treatment of pancreatic ductal adenocarcinoma (PDAC). PDAC is refractory to essentially all therapies to current treatments and has a media survival time of less than 1 year and a dismal 5 year survival rate of 3-5%.