ZIOPHARM Announces First Patient Enrolled in Phase 1 Study of Second Generation Non-Viral CD19-Specific CAR T-Cell Therapy f...
February 09 2016 - 7:00AM
ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company
focused on new cancer immunotherapies, today announced that the
first patient has been enrolled in a Phase 1 clinical study of its
second generation non-viral CD19-specific chimeric antigen receptor
(CAR) modified T-cell therapy in patients with advance lymphoid
malignancies. The CD19-specific T cells were modified using the
Sleeping Beauty system to stably express the CAR in T cells.
The Sleeping Beauty transposon-transposase is a
unique non-viral system for introducing genes encoding CARs and
T-cell receptors (TCRs) into lymphocytes and is exclusively
licensed by Intrexon Corporation (NYSE:XON) through The University
of Texas MD Anderson Cancer Center and accessed as part of
ZIOPHARM’s collaboration. This non-viral approach may play an
important role in immunotherapy and has several potential
advantages over viral delivery systems, including:
- Lower cost of generating genetically modified T cells
- Generate T cells with minimal ex vivo processing
- Conduit to targeting solid tumor neo-antigens using TCRs
- Pathway to overcome regulatory hurdles
"The survival benefit seen in early clinical
results with our first generation CD19-specific CAR+ T cells were
highly encouraging, and preclinical results to date suggest that
our next generation CAR structure may improve upon these outcomes,"
said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of
ZIOPHARM. "These studies continue to strengthen our understanding
of the application and benefit of the Sleeping Beauty platform, the
only efficient non-viral gene transfer system in clinical
application. Sleeping Beauty offers the potential to significantly
reduce the expense and simplify the implementation of genetically
modified T cells, both of which are critical to the personalization
and broad application of immunotherapies based on CARs and
TCRs."
In two prior trials the first generation
CD19-specific CAR+ T cells, patient-derived (autologous) or
donor-derived (allogeneic) T cells were administered to recipients
with advanced CD19-expressing leukemias and lymphomas after
hematopoietic stem-cell transplantation (HSCT). Results
demonstrated an apparent doubling of survivals compared to
historical controls.
The second-generation Sleeping Beauty CAR+ T
cells employ a revised CAR construct designed to improve
persistence and anti-tumor response over the first generation
therapy. Additionally, this investigational treatment is
independent of HSCT. This trial is being conducted at 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. 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, 2014, and our Quarterly Reports on Form 10Q for the
quarters ended March 31, 2015, June 30, 2015 and September 30,
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.
Contact:
Lori Ann Occhiogrosso
ZIOPHARM Oncology, Inc.
617-259-1987
locchiogrosso@ziopharm.com
David Pitts
Argot Partners
212-600-1902
david@argotpartners.com
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