ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company
developing new gene and cell-based immunotherapies for cancer,
today announced that the first patient has been dosed in a new
Phase 1 study of Ad-RTS-hIL-12 with veledimex for the treatment of
pediatric brain tumors.
This open label study will assess the safety and
tolerability of a single intratumoral injection of Ad-RTS-hIL-12, a
gene therapy designed to control the expression of human
interleukin 12 (hIL-12), a critical protein for stimulating a
localized anti-cancer immune response. The study is conducted in
two groups: the first is comprised of pediatric patients with
recurrent or progressive brain tumors in the cortex, while the
second is comprised of pediatric patients with diffuse intrinsic
pontine glioma (DIPG).
“Studies in adults with recurrent glioblastoma
have shown that Ad-RTS-hIL-12 with veledimex is not only well
tolerated, but also have shown growing evidence that this treatment
elicits a targeted immune response against brain tumor cells that
gives rise to improvement in overall survival,” said Francois
Lebel, M.D., Executive Vice President, Research and Development,
Chief Medical Officer at ZIOPHARM. “We look forward to advancing
our studies in pediatric patients with brain tumors as these
patients have limited-to-no therapeutic options.”
This Phase 1 study is being conducted at leading
pediatric cancer centers across the United States, including Ann
& Robert H. Lurie Children’s Hospital in Chicago, Dana-Farber
Cancer Institute in Boston and the University of California, San
Francisco. The first pediatric patient to receive Ad-RTS-hIL-12
plus veledimex is receiving care at Lurie Children’s.
“Pediatric gliomas are a devastating diagnosis
for children and families, and DIPG, specifically, while rare, is
extremely aggressive and always a fatal disease with no viable
treatment options,” said Stewart Goldman, M.D., Division Head
Hematology-Oncology, Neuro-Oncology & Stem Cell Transplantation
at Lurie Children’s. “We look forward to evaluating the potential
of Ad-RTS-hIL-12 plus veledimex as a treatment option for children
with brain tumors.”
About Glioma
Glioblastoma (GBM) is a fast-growing, aggressive
type of central nervous system tumor, with an estimated 12,390 new
adult cases predicted in 2017 according to the American Brain Tumor
Association. Recurrence rates for this type of cancer are near 90
percent, and prognosis for adult patients is poor with treatment
often combining multiple approaches including surgery, radiation
and chemotherapy i. In children, the incidence of brain cancer is
approximately 4.84 per 100,000, according to the National Cancer
Institute. Glioma in the cortex (cerebrum) of children is unusual
and is treated along the same lines as in adults with occurrence
common and survival poor. Glioma in the pontine region of the
brain, or DIPG, accounts for approximately 15 percent of all cases
of pediatric brain tumors, with a median survival time of less than
one yearii. Because of where these tumors are situated, DIPG is
inaccessible to surgery and there are no curative options.
About Ad-RTS-hIL-12 plus Veledimex
ZIOPHARM is advancing Ad-RTS-hIL-12 plus
veledimex as a gene therapy for recurrent GBM (rGBM). Ad-RTS-hIL-12
is an adenoviral vector administered via a single injection into
the tumor and engineered to express hIL-12, a powerful cytokine
that has demonstrated the potential to stimulate a targeted,
anti-tumor immune response. The expression of hIL-12 is controlled
and 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. The Company has recently reported
that biopsies from three patients treated with Ad-RTS-hIL-12 plus
veledimex provided evidence of documented pseudo-progression rather
than tumor progression. Pseudo-progression may be seen in serial
post-treatment imaging studies of cancers where the tumor appears
larger compared to baseline, but these changes are due to
infiltration of immune cells, as evidenced by subsequent biopsies.
ZIOPHARM’s Phase 1 stereotactic study of Ad-RTS-hIL-12 with
veledimex for the treatment of patients with brain tumors is
underway. The Company also plans to initiate enrollment of adult
patients with rGBM who will receive a single dose of Ad-RTS-hIL-12
plus veledimex in combination with a checkpoint inhibitor targeting
programmed cell death protein 1 (PD-1) by the end of the year.
About ZIOPHARM Oncology,
Inc.
ZIOPHARM Oncology is a Boston-based
biotechnology company employing innovative 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® (RTS®)
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 June
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.
i Mrugala MM. Advances and challenges in the
treatment of glioblastoma: a clinician's perspective. Discov Med.
2013;15:221-230.
http://www.discoverymedicine.com/Maciej-M-Mrugala/2013/04/25/advances-and-challenges-in-the-treatment-of-glioblastoma-a-clinicians-perspective/
ii Hargrave D, Bartels U, Bouffet E. Diffuse brainstem glioma
in children: critical review of clinical trials. Lancet Oncol. 2006
Mar;7(3):241-8.
TrademarksRheoSwitch
Therapeutic System® and RTS® are registered trademarks of Intrexon
Corporation.
Contact: David ConnollyZIOPHARM
Oncology617-502-1881dconnolly@ziopharm.com
or David PittsArgot
Partners212-600-1902david@argotpartners.com
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