APE1/Ref-1 is a protein with a pleiotropic role in regulating multiple transcription factors involved in cancer cell signaling, as well as responding to oxidative and base DNA damage. Additionally, transcription factors important in the hypoxic response, including HIF-1, NFkB, are regulated by APE1/Ref-1.
APX3330 is a highly selective inhibitor of APE1/Ref-1 redox function. The anti-tumor effect of APX330 has been demonstrated in a variety of preclinical xenograft models of cancer and the human safety profile of APX3330 has been established in prior clinical studies by Eisai Pharmaceuticals, who sought to develop APX3330 in a non-cancer indication.
Apexian Pharmaceuticals now seeks to develop APX3330 as an orally administered anti-cancer agent targeting the APE1/Ref-1 protein.
A Phase I clinical trial evaluating APX3330 as a single agent in patients with advanced solid tumors will commence in early 2017. Please see https://clinicaltrials.gov/ for additional information regarding studies involving APX3330.