Molecular Therapy Publication Highlights Sleeping Beauty Potential in Personalized TCR Gene Therapy

BOSTON, March 09, 2016 -- ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, today announced the publication of an article describing the use of Sleeping Beauty non-viral gene transfer technology to modify T cells for the targeting of neoantigens present within solid tumors. This approach unlocks the potential for rapid and inexpensive personalized T-cell receptor (TCR) gene therapy aimed at the unique mutations within a patient’s cancer cells. The article, titled “Stable, non-viral expression of mutated tumor neoantigen-specific T-cell receptors using the Sleeping Beauty transposon/transposase system,” was published in the journal Molecular Therapy (5 March 2016, doi:10.1038/mt.2016.51), and is available online. 

“The DNA plasmids from the Sleeping Beauty platform provide a customizable solution to personalized TCR gene therapy, which is needed to open the door to safely target solid tumors,” said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM and an author of the paper. “There is growing consensus that a non-viral approach to gene therapy will be essential to targeting individual neoantigens. In Sleeping Beauty, we have the only clinically validated non-viral gene transfer platform. This compels us to bring this important technology to patients.”

Neoantigens unique to each patient’s tumor can be recognized by autologous T cells through their T-cell receptors, but the low frequency and/or terminal differentiation of mutation-specific T cells may limit their utility as adoptive T-cell therapies. The publication describes how the Sleeping Beauty platform can be used to transfer and stably express TCR genes into T cells while retaining their proliferative potential. Indeed, the Sleeping Beauty gene transfer occurs without the need for cell division thus facilitating the genetic modification of minimally-differentiated T cells. The authors note that the Sleeping Beauty platform “can facilitate the use of personalized T cell therapy targeting unique neoantigens.”

The Sleeping Beauty transposon-transposase system was exclusively licensed by Intrexon Corporation (NYSE:XON) through the University of Texas MD Anderson Cancer Center and accessed as part of ZIOPHARM's collaboration with Intrexon.

About ZIOPHARM Oncology, Inc.:

ZIOPHARM Oncology is a Boston, Massachusetts-based biotechnology company employing novel gene expression, control and cell technologies to deliver safe, effective and scalable cell- and viral-based therapies for the treatment of cancer. The Company's synthetic immuno-oncology programs, in collaboration with Intrexon Corporation (NYSE:XON) and the MD Anderson Cancer Center, include chimeric antigen receptor T cell (CAR-T) and other adoptive cell based approaches that use non-viral gene transfer methods for broad scalability. The Company is advancing programs in multiple stages of development together with Intrexon Corporation's RheoSwitch Therapeutic System® technology, a switch to turn on and off, and more precisely modulate gene expression in order to improve therapeutic index. The Company's pipeline includes a number of cell-based therapeutics in both clinical and preclinical testing which are focused on hematologic and solid tumor malignancies.

Forward-Looking Safe-Harbor Statement:

This press release contains certain forward-looking information about ZIOPHARM Oncology, Inc. that is intended to be covered by the safe harbor for "forward-looking statements" provided by the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as "may," "will," "could," "expects," "plans," "anticipates," and "believes." These statements include, but are not limited to, statements regarding the progress, timing and results of preclinical and clinical trials involving the Company's drug candidates, and the progress of the Company's research and development programs. All of such statements are subject to certain risks and uncertainties, many of which are difficult to predict and generally beyond the control of the Company, that could cause actual results to differ materially from those expressed in, or implied by, the forward-looking statements. These risks and uncertainties include, but are not limited to: whether chimeric antigen receptor T cell (CAR T) approaches, Ad-RTS-IL-12, TCR and NK cell-based therapies, or any of our other therapeutic candidates will advance further in the pre-clinical or clinical trials process and whether and when, if at all, they will receive final approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies and for which indications; whether chimeric antigen receptor T cell (CAR T) approaches, Ad-RTS-IL-12, TCR and NK cell-based therapies, and our other therapeutic products will be successfully marketed if approved; the strength and enforceability of our intellectual property rights; competition from other pharmaceutical and biotechnology companies; and the other risk factors contained in our periodic and interim SEC reports filed from time to time with the Securities and Exchange Commission, including but not limited to, our Annual Report on Form 10-K for the fiscal year ended December 31, 2015. Readers are cautioned not to place undue reliance on these forward-looking statements that speak only as of the date hereof, and we do not undertake any obligation to revise and disseminate forward-looking statements to reflect events or circumstances after the date hereof, or to reflect the occurrence of or non-occurrence of any events.
Trademarks

RheoSwitch Therapeutic System^® (RTS^®) technology is a registered trademark of Intrexon Corporation.

Lori Ann Occhiogrosso
ZIOPHARM Oncology, Inc.
617-259-1987
[email protected]

David Pitts
Argot Partners
212-600-1902
[email protected]