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ZIOPHARM Announces Plans for Phase I Clinical Trial with CD33 CAR-T Cell Therapy Targeting Acute Myeloid Leukemia

BOSTON, July 12, 2016 (GLOBE NEWSWIRE) -- ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, today announced plans for a Phase I adoptive cellular therapy clinical trial utilizing autologous T cells transduced with lentivirus to express a CD33-specific chimeric antigen receptor (CAR) in patients with relapsed or refractory acute myeloid leukemia (AML). The upcoming trial will be the second initiated at The University of Texas MD Anderson Cancer Center under the research and development agreement among ZIOPHARM, Intrexon Corporation (NYSE:XON), and MD Anderson to expeditiously move promising treatments from the bench to the clinic.

“By targeting myeloid cells with a CD33-specific CAR, we have the opportunity to leverage T-cell mediated cytotoxicity in a highly directed manner,” said Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM. Dr. Lebel continued, “Following a recent review by the Recombinant DNA Advisory Committee, we anticipate progressing plans for this Phase I trial in the second half of 2016 to move this promising treatment closer to the clinic for patients with relapsed or refractory AML who have poor prognosis."

The American Cancer Society estimates that there will be approximately 20,000 new cases of AML and over 10,000 patient deaths from AML in the United States in 2016. AML is a rapidly progressing cancer of the blood and bone marrow characterized by uncontrolled proliferation of immature blast cells with multiple associated gene mutations. A majority of AML patients relapse or present with refractory disease and have overall poor prognosis.

Preclinical studies demonstrated that lentiviral transduced CAR-T cells targeting CD33 exhibit specific cytotoxic activity for CD33+ AML cells. A proof-of-concept study utilizing an in vivo mouse model for AML showed that these CAR-T cells were able to eliminate disease burden and significantly enhance survival as compared to control groups. These positive preliminary results indicate biological activity and are suggestive of potential therapeutic effect for the treatment of AML.

Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM Oncology commented, “As we progress through the remainder of 2016 with our Intrexon Corporation and MD Anderson collaborators, we believe our clinical stage assets and promising preclinical pipeline will position the company at the forefront of those utilizing the immune system in a controlled manner to combat cancer.” Dr. Cooper added, “We believe that our CD33 CAR-T cell approach has the potential to positively impact a disease area that has been largely unexplored with this type of immune-therapy and overall this malignancy has seen inadequate improvement in treatment options. We look forward to moving into the clinic as soon as possible to help patients with advanced AML urgently in need of new therapies."

This planned Phase I trial will add to ZIOPHARM’s three active trials at leading institutions, two employing controlled gene therapy to express a powerful anti-cancer effector in interleukin-12 and the other incorporating the innovative non-viral Sleeping Beauty gene delivery system in CAR-T cell therapy for advanced lymphoid malignancies. This new program targeting CD33 is part of the Exclusive Channel Collaboration with Intrexon and is the next CAR-T target the companies are pursuing in addition to the first two targets already selected by the biopharmaceutical division of Merck KGaA.

ZIOPHARM intends to further expand its clinical programs in the area of natural killer (NK) cells and a combination approach to its Ad-RTS-hlL-12 gene therapy using an immune checkpoint inhibitor through the remainder of 2016. Additionally, ZIOPHARM and Intrexon continue to advance preclinical efforts leveraging the Sleeping Beauty platform, the clinical-stage RheoSwitch® (RTS®), and improving cell manufacturing for the clinical applications of T cells engineered to express CAR and TCR, as well as off-the-shelf approaches.

About ZIOPHARM Oncology, Inc.
ZIOPHARM Oncology is a Boston, Massachusetts-based biotechnology company employing novel gene expression, control and cell technologies designed to deliver safe, effective and scalable cell- and viral-based therapies for the treatment of cancer. The Company's 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 working to advance programs in multiple stages of development together with Intrexon Corporation's RheoSwitch Therapeutic System® technology, a switch designed to turn on and off, and 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.

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, the progress of the Company's research and development programs, and the Company's expectations that a Phase I adoptive cellular therapy clinical trial utilizing autologous T cells transduced with lentivirus to express a CD33-specific CAR in patients with relapsed or refractory AML will be initiated by its collaborator, The University of Texas MD Anderson Cancer Center, in the second half of 2016, to expand its clinical programs in the area of NK cells and a combination approach to its Ad-RTS-hlL-12 gene therapy using checkpoint inhibitors through the remainder of 2016, to advance preclinical efforts leveraging the Sleeping Beauty platform, the clinical-stage RTS®, and to improve cell manufacturing for the clinical applications of T cells engineered to express CAR and TCR, as well as off-the-shelf approaches. 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-hIL-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-hIL-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 and our Quarterly Report for the quarter ended March 31, 2016. 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.


For more information, contact: ZIOPHARM: Corporate Contact: Lori Ann Occhiogrosso ZIOPHARM Oncology, Inc. 617-259-1987 locchiogrosso@ziopharm.com Investor Contact: David Pitts Argot Partners 212-600-1902 david@argotpartners.com

Source:ZIOPHARM Oncology, Inc.