Apexan Pharmaceuticals has announced that The National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) has awarded a $2.3 million grant to Dr. Reuben Kapur and Dr. Mark R. Kelley, investigators in the Wells Center for Pediatric Research at the IU School of Medicine, to conduct studies that focus on preleukemic stem cells bearing specific mutations and their progression to full-blown cancer in the context of diabetes. The funds will also be used to examine and assess cancer progression in animal models under conditions of hyperglycemia, or high blood sugar, over the next four years. In these studies, they will build on their recently published work in Cell Stem Cell, demonstrating that APX3330 blocks this progression under conditions of inflammation.
APX3330 is a lead compound of Apexian Pharmaceuticals, a clinical stage drug development company focused on advancing APX3330 for the treatment of diseases mediated by the APE1/Ref-1 protein. Dr. Kelley is the chief scientific officer of Apexian.
Results of Apexian’s recently completed Phase I clinical trial of APX3330 in patients with advanced, end-stage solid tumors were presented at the annual meeting of the American Society of Clinical Oncology (ASCO). The study confirmed the safety profile of APX3330 when given chronically as an orally-administered pill and identified a recommended phase II dose for further clinical development of APX3330 in a variety of possible indications.
Tumor biopsy data from the study confirmed the mechanism of action of APX3330 interacting with its molecular target, the APE1/Ref-1 protein, a gatekeeper for transcription factor activity. According to Dr. Kelley,
Given the importance of the APE1/Ref-1 protein in a variety of disease processes, including cancer, diabetic macular edema (DME) and inflammatory bowel disease (ulcerative colitis) and others, the phase I results confirm APX3330’s ability to control the drug target in humans. The NHLBI grant will be used to increase our understanding of the links between diabetes and cancer, allowing us to explore the potential of APX3330 in these areas of high unmet medical need.
Apexian Pharmaceuticals is a patient-centered biotechnology company dedicated to developing novel compounds that target the APE1 protein in multiple indications including cancer. Apexian’s research is grounded and driven by the belief that “better is always possible.”