The University of Texas MD Anderson Cancer Center has established a broad exclusive licensing agreement with Intrexon Corporation and ZIOPHARM Oncology, both of which are focused on the development of non-viral adoptive cellular cancer immunotherapies. MD Anderson’s new agreement with the companies includes an exclusive sub-licensing for intellectual property designed at the University of Minnesota.
The terms of the agreement stipulate a $100 million payment, half from Intrexon and the other half from ZIOPHARM, in shares of their common stocks, in addition to three years of payments between $15 and $20 million granted annually to support the research, which will be conducted at MD Anderson Cancer Center’s facilities.
The technologies included in the license agreement are the result of a pioneering collaborative investigation between Laurence Cooper, M.D., Ph.D., a professor of Pediatrics at MD Anderson, and a professor in the College of Biological Sciences at Minnesota, Perry Hackett, Ph.D. The two scientists have been working on the design and clinical investigation of novel chimeric antigen receptor (CAR) T cell therapies using non-viral gene integration platforms.
MD Anderson has used the technology to provide T-cell therapies to oncology patients, in addition to novel therapeutic approaches for the development of medical products for global off-the-shelf application. “Genetically engineering our patients’ immune-system T cells to efficiently attack and destroy cancer cells represents one of the most exciting approaches with curative potential in oncology today,” said MD Anderson president Ron DePinho, M.D.
The non-viral DNA plasmid-based gene transfer system designed by Cooper and Hackett aims to alter T cells through the creation of a CAR able to determine and bind a particular cell surface protein on targeted malignant cells. The study of the technology in humans being planned to start at MD Anderson is expected to be the first steps for both the design and implementation of modified T cells for the treatment of patients with several types of different conditions.
“The promise of controlled, cell-based immuno-oncology therapy is that we can achieve dramatic, long duration anti-cancer results while keeping patients out of intensive care during treatment. As importantly, we can reproduce these results in a globally scalable and economically viable way,” explained the chief executive officer of ZIOPHARM Oncology, Jonathan Lewis, M.D., Ph.D.
The technology is expected to improve genetically modified CAR T cells, in combination with a technology suite from Intrexon and a clinically tested RheoSwitch Therapeutic System-interleukin-12 modules from ZIOPHARM. The combination of both companies’ technologies allows for close monitoring of expansion and activation, as well as reducing off-target effects and toxicity.
“We believe coupling MD Anderson’s unique CAR T cell approach with the powerful technologies of ZIOPHARM and Intrexon will allow us to build T cells that hit cancer harder, with greater precision, under tighter control and with potentially fewer side effects for patients,” explained DePinho. “This agreement ranks as one of MD Anderson’s most substantial collaborations and will provide significant resources to fuel its mission of Making Cancer History.”
“We are proud to see Perry Hackett’s discovery and development work on Sleeping Beauty, a non-viral DNA plasmid-based gene transfer system, in conjunction with Dr. Cooper’s expertise in immunotherapies, to provide this breakthrough in oncology,” said Brian Hermann, VP of research at the University of Minnesota.
The collaboration is meant to join both novel cell engineering techniques and multigenic gene programs, in order to apply novel non-viral adoptive cellular therapies based on cytokines and CARs. Within this year, the partners expect to be able to stimulate a next-generation immunology pipeline, and to initiate off-the-shelf programs in the next year.
“It is a shared vision to maximize the speed and breadth of multigenic innovation for patients through the use of nimble, non-viral DNA cell manufacturing strategies that can further overcome viral packaging constraints and economic limitations,” added the senior vice president and head of Intrexon’s health sector, Gregory Frost, Ph.D. “Collectively, this will assemble the most advanced set of technologies to empower the strongest adoptive cell therapy pipeline that can drive innovation through multiple horizons and patient populations.”
“The human application of T cell therapies provides cancer patients with new hope, and the alignment of MD Anderson’s immunotherapy and translational programs with Intrexon and ZIOPHARM will help make that hope a reality,” MD Anderson’s Cooper said. “These two interconnected companies have first-in-class genetic tools and systems to reprogram cells and the management and regulatory expertise to undertake development of potent and focused cell-based immunotherapies.”
With this in mind, MD Anderson, Intrexon and ZIOPHARM will be working in a shared infrastructure focused on two approaches to enable the provision of viable T cells to oncology patients. While the first is based on a point-of-care approach with rapid assembly and infusion of autologous T cells, the second is based on a universal donor platform that infuses off-the-shelf T cells using innovative activation and targeting gene programs that precisely recognize and systemically combat malignancies.
“The MD Anderson Cancer Center has long been a leader in cancer therapy, in terms of innovation, patient care, and the highest quality research. As part of our commitment to this important partnership and the acceleration of translational medicine, ZIOPHARM will build a base of operations in Houston to join and collaborate with the academic and medical community around this world-class institution,” added the CEO of ZIOPHARM Oncology, Jonathan Lewis.