- Clinical Trials
Our oncology program utilizes our unique, proprietary non-viral ONCOPREX nanoparticle delivery system. 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.
The ONCOPREX platform has been designed and optimized to deliver cancer-fighting genes into the patient’s body. These cancer-fighting genes are encapsulated in nanoscale hollow spheres called “nanoparticles,” which are then administered intravenously and taken up by tumor cells where they express proteins that are missing or found in low quantities. Our nanoparticles are non-immunogenic, allowing repetitive therapeutic dosing and have been clinically shown to deliver molecular kinase inhibitors effectively.
Our nanoparticle non-viral delivery system, which has been used in our clinical trials for the treatment of non-small cell lung cancer (NSCLC), is designed to be small enough to cross tight barriers in the lungs but large enough to avoid liver accumulation and clearance. The nanoparticles have been shown to be taken up by tumor cells after REQORSA administration at up to 33 times the rate they are taken up by normal cells. The cationic charge of the lipid nanoparticles targets cancer cells, which facilitates endocytosis. Once inside the cancer cell, the TUSC2 gene activates signaling pathways that result in cell destruction or apoptosis.
We have administered REQORSA to more than 50 patients in Phase I and II clinical trials using our proprietary non-viral delivery system.
A Phase I clinical trial showed that 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.
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 this was the first systemic gene therapy clinical trial that used a nanoparticle delivery system for delivering a tumor suppressor gene.