ONCOPREX® Nanoparticle Delivery System

The First Systemic Gene Therapy Delivery Platform Used in Humans for Cancer

Our oncology drug development program utilizes our unique, proprietary non-viral ONCOPREX Nanoparticle Delivery System, which we believe is the first systemic gene therapy delivery platform used for cancer in humans. This platform, originally developed through collaborative research between the University of Texas MD Anderson Cancer Center and the National Institutes of Health, has been optimized to work with our initial product candidate, REQORSA™ immunogene therapy.

Designed to Deliver Cancer-Fighting Genes Systemically

The ONCOPREX platform has been designed and optimized to deliver cancer-fighting genes into the patient’s body systemically. Using this system, we encapsulate plasmids that express tumor suppressor genes within lipid nanoparticles and intravenously administer the encapsulated plasmids which are taken up by the tumor cells, after which the tumor suppressor genes express proteins that are missing or found in low quantities in the tumor cells. Our nanoparticles are non-immunogenic, allowing repetitive therapeutic dosing and have been clinically shown to deliver molecular kinase inhibitors effectively.

Enhanced Safety and Efficacy Design

We have administered REQORSA to more than 50 patients in Phase 1 and 2 clinical trials using our systemic, proprietary, non-viral delivery system.

A Phase 1 clinical trial showed that systemic, intravenous therapy using the ONCOPREX Nanoparticle Delivery System was shown to selectively and preferentially target primary and metastatic tumor cells, resulting in clinically significant anticancer activity. The nanoparticles are non-immunogenic, allowing repetitive therapeutic dosing and providing extended half-life in the circulation.

Our earlier clinical trials have also shown that the ONCOPREX Nanoparticle Delivery System is well tolerated in humans and can safely deliver high therapeutic doses. We believe the ONC-001 clinical trial was the first systemic gene therapy clinical trial using a nanoparticle delivery system to deliver a tumor suppressor gene.