BOSTON, May 02, 2017 (GLOBE NEWSWIRE) -- ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new
immunotherapies, today announced that an investigator-initiated Investigational New Drug (IND) application to the U.S. Food and
Drug Administration (FDA) for a Phase 1 trial infusing the Company’s CD33-specific CAR+ T therapy for relapsed or
refractory acute myeloid leukemia (AML) is now active, with the first patient to be enrolled in the study expected to begin
treatment in the third quarter of 2017. The CD33-specific CAR+ T cells incorporate a kill switch designed to eliminate
the modified T cells under potential adverse safety conditions.
“Relapsed AML is an aggressive disease with very poor outcomes,” said William G. Wierda, M.D., Ph.D., Professor
and Center Medical Director, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center
and principal investigator for the CD33 study. “In vivo preclinical animal studies have demonstrated that these CAR-T
cells targeting CD33 exhibit specific killing of AML cells, eliminating disease burden, and significantly enhancing survival
compared to controls, and I look forward to evaluating the safety and effectiveness of this gene therapy for patients with
AML.”
Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM
added, “CAR-T cells expressing CD33 have shown promise in preclinical studies, but to-date, there has been limited experience in
humans, representing a significant white space for us in treating AML. We look forward to seeing the positive preclinical results
with our CD33-specific CAR-T cells translate to the clinic for relapsed/refractory AML patients who have far too few treatment
options. In parallel with this Phase 1 study, we have also begun preclinical studies to evaluate rapid non-viral manufacturing of
CAR+ T CD33-specific therapy for treatment of AML under point-of-care.”
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. The American Cancer Society estimates that there were approximately
20,000 new cases of AML and over 10,000 patient deaths from AML in the United States in 2016. A majority of AML patients relapse or
present with refractory disease and have overall poor prognosis.
This will be the second CAR target for genetically modified T cells to be studied at The University of Texas MD
Anderson Cancer Center under the research and development agreement among ZIOPHARM, Intrexon Corporation (NYSE:XON), and MD
Anderson.
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 and
graft-versus-host-disease. 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 advancing programs in multiple stages of development together
with Intrexon Corporation's RheoSwitch Therapeutic System® technology, a switch 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.
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 Company's plans and expectations regarding its securities offerings, fundraising activities and financial
strategy, 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: our ability to finance our operations and business initiatives and obtain funding for such activities, 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 preclinical 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 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, 2016 and our Quarterly Report on Form 10-Q for the quarter ended March
31, 2017. 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® and RTS® are registered trademarks of Intrexon Corporation.
Contact: Amy Trevvett Vice President, Corporate Communications and Investor Relations 617-502-1881 atrevvett@ziopharm.com David Pitts Argot Partners 212-600-1902 david@argotpartners.com
