Biotechs Intrexon, Ziopharm and MD Anderson Sign $100 Million CART PactBy
The biotechnologies companies, Intrexon Corporation and Ziopharm Oncology, have formed a broad exclusive licensing agreement with the University of Texas MD Anderson Cancer Center, including an exclusive sublicensing agreement through MD Anderson for intellectual property developed at the University of Minnesota for the development of non-viral adoptive cellular cancer immunotherapies.
Under the agreement, MD Anderson will receive $100 million; $50 million from each Intrexon and Ziopharm, payable in shares of their respective common stock, as well as a commitment of $15 million to $20 million annually over three years for researching and developing the technologies. The parties will enter into additional collaboration and technology transfer agreements to accelerate technology and clinical development.
The licensed technologies are from the laboratory of Laurence Cooper, MD, PhD, professor of pediatrics at MD Anderson, and Perry Hackett, PhD, professor within the College of Biological Sciences at Minnesota. The Cooper and Hackett laboratories have pioneered the design and clinical investigation of novel chimeric antigen receptor (CAR) T cell therapies using non-viral gene integration platforms. MD Anderson has built on this technology to deliver patient-derived T cells as well as approaches to generating products for universal off-the-shelf applications. When combined with Intrexon’s technology suite and Ziopharma’s clinically tested RheoSwitch Therapeutic System interleukin-12 modules, the resulting proprietary methods and technologies may help realize the promise of genetically modified CAR T cells by tightly controlling cell expansion and activation in the body and minimizing off-target effects and toxicity while maximizing therapeutic efficacy.
Using cell-engineering techniques and multigenic gene programs, the collaboration will implement non-viral adoptive cellular therapies based on designer cytokines and CARs under control of the RheoSwitch technology targeting both hematologic and solid tumor malignancies, with up to five CARs expected to enter the clinic in 2015 and off-the-shelf programs initiating in 2016.
Cooper, Hackett, and colleagues developed a non-viral DNA plasmid-based gene transfer system to modify T cells by creating a CAR that recognizes and binds to a specific cell surface protein on targeted malignant cells. The testing of this system at MD Anderson in humans paves the way for the rapid design and implementation of modified T cells that can be infused into patients with many types of malignancies.
Work continues in conjunction with MD Anderson’s Moon Shots Program, an initiative to accelerate the conversion of scientific discoveries into clinical advances and reduce cancer deaths, first targeting eight types of cancer. Cooper leads the Applied Cellular Therapeutics platform for the moon shots, providing expertise and new cellular therapy capabilities for both blood and solid tumor cancers. Clinical trials using non-viral adoptive cellular therapies are either under way or planned for specific moon shot cancers.
The shared infrastructure between MD Anderson, Intrexon, and Ziopharm enables two approaches to deliver commercially viable T cells to the bedside. The first develops a point-of-care approach with rapid assembly and infusion of autologous T cells. The second arises from the universal donor platform to infuse off-the-shelf T cells using activation and targeting-gene programs. The collaboration will advance these platforms in parallel with the most effective CAR-T products tested at MD Anderson graduating to multicenter trials.