- Controlled IL-12 advancing as drug platform as
monotherapy and in combination with OPDIVO® (nivolumab) --
Non-viral T-cell platform shows potential for efficacy, scalability
and cost reduction for multiple oncology targets -
Ziopharm Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company
developing gene- and cell-based immunotherapies for cancer, today
provided an update on the Company’s clinical programs and corporate
development activities during the 36th Annual J.P. Morgan
Healthcare Conference.
“Ziopharm’s immunotherapies in clinical trials
are based on two powerful platforms. First, we have shown that we
can super-charge a patient’s own immune system with controlled,
local production of IL-12 and second, we can provide a new immune
response where none existed, infusing genetically modified T cells
with a unique and advantageous manufacturing process,” said
Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm.
“Producing IL-12 in the tumor microenvironment under full control
is a new drug platform for oncology, which we will further exploit
by combining with checkpoint inhibitors. Likewise, we are
implementing a new paradigm for production of genetically modified
T cells with our Sleeping Beauty (SB) non-viral platform and
proprietary membrane bound IL-15 which enables very rapid
manufacture of CAR- and TCR-modified cells at low cost and
scale.”
The Company’s corporate presentation at the J.P.
Morgan conference is Thursday, Jan. 11, at 10 a.m. PST. To access a
live audio webcast of the presentation, please visit the Investor
Relations section at www.ziopharm.com. The webcast will be archived
for 90 days.
Program Updates
Controlled IL-12 for Gliomas
Ziopharm, in partnership with Intrexon
Corporation (NYSE:XON), is advancing Ad-RTS-hIL-12 plus veledimex,
or controlled interleukin-12 (IL-12), as a gene therapy for
recurrent glioblastoma (rGBM). Ad-RTS-hIL-12 is an adenoviral
vector administered via a single injection into the brain tumor and
engineered to conditionally express human interleukin-12 (hIL-12).
The expression of hIL-12 is modulated with the RheoSwitch
Therapeutic System® (RTS®) by the small molecule veledimex, an
activator ligand which has been shown to cross the blood-brain
barrier. A Phase 1 trial produced compelling data demonstrating the
safety of controlled local expression of IL-12 and that IL-12 can
turn previously cold tumors hot, which could have a profound impact
for oncology in general.
Combination Trial with OPDIVO
(nivolumab) Initiated - The Company announced the
initiation of a Phase I clinical trial to evaluate Ad-RTS-hIL-12
plus veledimex in combination with OPDIVO® (nivolumab), an immune
checkpoint, or PD-1, inhibitor, in adult patients with rGBM.
Pivotal Trial to Initiate in Second Half
of 2018 - The Company updated guidance on its planned
pivotal trial and announced that it will initiate in the second
half of 2018. Ziopharm has designed the randomized control trial to
evaluate controlled IL-12 for the treatment of patients with rGBM
and following meetings with U.S. and European regulators is
completing Chemistry Manufacturing and Control (CMC) technical
requirements. The Company continues to engage in partnership
discussions in this indication and this updated timeframe allows
for accumulation of additional clinical data from the open Phase 1
trials, including the combination trial.
“As the Phase 1 survival data matured over the
latter half of 2017, we saw compelling evidence from biopsies,
taken more than four months after administration of Ad-hIL-12 plus
veledimex, demonstrating that controlled IL-12 causes an influx of
killer T cells into brain tumors, and upregulated expression of
PD-1 biomarkers,” said Francois Lebel, M.D., Executive Vice
President, Research and Development, Chief Medical Officer at
Ziopharm. “The randomized control trial will allow us to confirm
the activity of our drug as a monotherapy and combining with
nivolumab allows us to advance a much needed therapeutic option by
exploring a potentially synergistic mechanism of action.”
Adoptive Cell Therapies
Ziopharm is developing chimeric antigen receptor
(CAR) T cell (CAR+ T) and T-cell receptor (TCR) T cell (TCR+ T)
therapies. These programs are being advanced in collaboration with
Intrexon and selectively with MD Anderson Cancer Center, the
National Cancer Institute (NCI) and Merck KGaA, Darmstadt, Germany.
Initiation of First point-of-care
(P-O-C) Clinical Trial Expected in 2018. The Company is
advancing its non-viral Sleeping Beauty (SB) platform towards P-O-C
for the very rapid manufacturing of genetically modified CAR+ T
cells, with the first clinical trial utilizing this approach
expected to begin in 2018. Data supporting P-O-C were presented at
the 59th American Society of Hematology Annual Meeting in December
2017, where first- and second-generation SB clinical trial data
demonstrated safety, tolerability, disease response including
long-term survival, and sustained persistence of infused
CD19-specific CAR+ T cells. The Company expects to initiate the
third-generation P-O-C study, which leverages SB to manufacture
CAR+ T cells co-expressing a membrane-bound interleukin-15, or
mbIL15, in less than two days. Manufacturing under P-O-C has the
potential to reduce the costs of T-cell therapy and broaden
application based on avoiding the need for centralized
manufacturing.
Phase 1 Trial of
SB-Modified TCRs to Treat Solid Tumors to Initiate in
Second Half of 2018. The Company updated guidance on the
anticipated start of the National Cancer Institute (NCI)-led Phase
1 trial to evaluate adoptive cell transfer (ACT)-based
immunotherapies genetically modified using the SB
transposon/transposase system to express TCRs for the treatment of
solid tumors. Ziopharm, Intrexon, and the NCI last year entered
into a Cooperative Research and Development Agreement to develop
and evaluate ACT for patients with advanced cancers using
autologous peripheral blood lymphocytes genetically modified using
the non-viral SB system to express TCRs that recognize specific
immunogenic mutations, or neoantigens, expressed within a patient’s
cancer.
“We have used the Sleeping Beauty platform to
generate neoantigen-specific T cells and look forward to initiating
a clinical trial infusing these genetically modified T cells to
target solid tumors,” said Steven A. Rosenberg, M.D., Ph.D., Chief
of the Surgery Branch at the NCI’s Center for Cancer Research, who
is leading this research. “The Sleeping Beauty system is able to
target the unique mutations that give rise to a patient’s
malignancy.”
Phase 1 Trial of CD33-specific CAR+ T
Therapy for Acute Myeloid Leukemia (AML). Enrollment is
underway in the Phase 1 adoptive cellular therapy clinical trial of
CD33-specific CAR+ T cell therapy in patients with
refractory/recurrent AML. This study infuses autologous T cells
genetically modified with lentivirus to express a CD33-specific CAR
and a cetuximab-activated (HER1t) kill switch for elimination of
genetically modified cells in the case of unmanageable severe
adverse events. The trial is enrolling at The University of Texas
MD Anderson Cancer Center. The data are expected to serve as the
basis for evaluating CD33 as a potential target for further
development using very rapid non-viral manufacturing of T cells
under P-O-C.
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 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 progress and timing of the
development 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: the Company's ability to finance its
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 other product candidates will advance further in the preclinical
research or clinical trial 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 the
Company's other therapeutic products it develops will be
successfully marketed if approved; the strength and enforceability
of the Company's intellectual property rights; competition from
other pharmaceutical and biotechnology companies; as well as other
risk factors contained in the Company's periodic and interim
reports filed from time to time with the Securities and Exchange
Commission, including but not limited to, the risks and
uncertainties set forth in the "Risk Factors" section of the
Company's Quarterly Report on Form 10-Q for the quarter ended
September 30, 2017 and subsequent reports that the Company may file
with the Securities and Exchange Commission. Readers are cautioned
not to place undue reliance on these forward-looking statements
that speak only as of the date hereof, and the Company does 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:David ConnollyZIOPHARM
Oncology617-502-1881dconnolly@ziopharm.com
David PittsArgot
Partners212-600-1902david@argotpartners.com
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