ZIOPHARM & Intrexon Announce Worldwide Partnership for Synthetic Biology DNA-Based Oncology Therapeutics


ZIOPHARM Oncology, Inc., a small molecule late-stage oncology drug development company, and Intrexon Corporation, a next-generation synthetic biology company, recently announced a global exclusive channel partnership in oncology in which ZIOPHARM will develop and commercialize DNA-based therapeutics using Intrexon’s UltraVector Technology. Under the partnership, ZIOPHARM will utilize Intrexon’s advanced transgene engineering platform for the controlled and precise cellular production of anti-cancer effectors.

ZIOPHARM will have rights to Intrexon’s entire human in vivo effector platform within the field of oncology, which includes two lead clinical-stage product candidates, one of which is in an advanced Phase I study and another which will be the subject of an IND filing during the first half of 2011.

Intrexon employs its modular genetic engineering platform in the areas of therapeutics, protein production, industrial, and agriculture products. The exclusive channel partnership between Intrexon and ZIOPHARM has been established specifically for the field of human oncologic therapeutics. Under the partnership, Intrexon remains responsible for technology discovery efforts and managing the patent estate as well as for certain aspects of manufacturing. ZIOPHARM will be responsible for conducting preclinical and clinical development of candidates, as well as for other aspects of manufacturing and the commercialization of the candidates.

Intrexon’s core synthetic biology technology is designed to create Better DNA at industrial scale, enabling unprecedented control over the function and output of living cells by providing external control over in vivo activation and regulation of potent effectors. This platform, called UltraVector, provides speed, flexibility, consistency, and precision to the design, production, and testing of rationally designed complex transgenes and their encoded genetic circuits. These qualities allow an iterative and rational approach to transgene design, which can be continually engineered until their performance is optimized. Through this process, Intrexon is able to overcome the challenges inherent in current therapeutic strategies, including recombinant protein therapies and constitutive gene therapies, thereby enhancing capabilities, improving safety and lowering cost for human therapeutics.

The lead oncology product candidate developed using Intrexon’s technologies is currently in Phase Ib clinical study for metastatic melanoma. ZIOPHARM expects to submit an IND application with US FDA for a second oncology product candidate in the first half of this year.

“Controllable, scalable synthetic biology, the tightly regulated delivery of therapeutic proteins from within the body, is an aspirational and disruptive technology that Intrexon has brought from scientific theory to medical application,” said Jonathan Lewis, MD, PhD, CEO and CMO of ZIOPHARM. “As the sole channel partner for in vivo therapeutic candidates for human oncology, ZIOPHARM plans to leverage this technology for next-generation products targeting key pathways used by cancers to grow and metastasize. Intrexon has developed a technology that is uniquely flexible, scalable and controllable, adding significantly to our small molecule drug development capabilities and our ability to translate science to the patient using our world-class global team.”

“We are very pleased to collaborate with ZIOPHARM, which, under the leadership of Jonathan Lewis, is building an industry-leading oncology company with a strategic vision regarding cancer medicine. ZIOPHARM’s oncology expertise, development capabilities, as well as its excellent reputation within the oncology community make ZIOPHARM an exceptional investment for Intrexon and ideal partner to rapidly achieve the full therapeutic benefit and commercial potential of Intrexon’s disruptive technologies,” added RJ Kirk, Intrexon’s Chairman and CEO. “This collaboration leverages the capabilities and strengths of each partner and has the potential to create significant value for shareholders.”