ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company
focused on new immunotherapies, today announced the initiation of
enrollment in the stereotactic arm of its Phase 1 multicenter study
of Ad-RTS-hIL-12 + veledimex, a gene therapy for controlled
expression of IL-12, in patients with recurrent glioblastoma
multiforme (rGBM). The stereotactic cohort will include rGBM
patients that are not scheduled to undergo surgical resection to
assess the safety and tolerability of a single dose of
Ad-RTS-hIL-12 administered via injection and activated with
orally-administered veledimex (20 mg QD, days 1-14).
“Supported by strong clinical data highlighting
the potential for controlled immune activation to affect survival
outcomes in recurrent GBM, we are expanding our innovative
Ad-RTS-hIL-12 + veledimex gene therapy into new treatment settings
and combinations,” said Francois Lebel, M.D., Executive Vice
President, Research and Development, Chief Medical Officer at
ZIOPHARM. “By introducing stereotactic administration, we broaden
the population of patients who may be eligible for treatment,
including pediatric patients with diffuse intrinsic pontine glioma,
a rapidly fatal and inoperable form of brain cancer.
Additionally, preclinical studies of this tunable IL-12 gene
therapy in combination with an immune checkpoint inhibitor have
yielded promising results, notably a 100 percent survival rate of
treated mice, and we look forward to bringing this approach into
the clinic.”
Following an observation period, the Company
expects to immediately:
- Initiate enrollment in a study of adult patients with rGBM who
will receive a single dose of Ad-RTS-hIL-12 + veledimex in
combination with a checkpoint inhibitor targeting programmed cell
death protein 1 (PD-1);
- Initiate enrollment in a study of pediatric patients with
recurrent/progressive glioma who will receive a single dose of
Ad-RTS-hIL-12 + veledimex. The Company anticipates children with
diffuse intrinsic pontine glioma (DIPG) will be eligible for
enrollment.
At the 2017 American Society of Clinical
Oncology Annual Meeting held in June, the Company reported
encouraging results from patients receiving intratumoral
Ad-RTS-hIL-12 with 20 mg of orally-administered veledimex (n = 15)
following craniotomy, with a median overall survival (mOS) of 12.5
months comparing favorably to historical controls. Based on the
observed ratio of CD8+/FOXP3+ (effector/suppressor) T cells,
overall survival appears directly correlated with IL-12-mediated
cellular immune activation. Furthermore, patients who received low
dose steroids have a much better survival rate than those who
received elevated systemic steroids, as the latter presumably
interferes with immune activation. The Company continues to
progress towards a registration study for Ad-RTS-hIL-12 + veledimex
for rGBM to start in 2017 and is also evaluating partnership
opportunities for this promising treatment candidate.
“Stereotactic treatment serves as a runway into
our next phase of development for Ad-RTS-hIL-12 + veledimex
including combination therapy and pediatric studies," said Laurence
Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM Oncology.
“We anticipate translating the combination approach to patients
imminently and further leveraging initial observations on the
survival benefit of IL-12-driven immune activation. As a pediatric
oncologist, I know full well the devastating impact of DIPG and am
also looking forward to seeing the effect of controlling IL-12 in
this difficult-to-treat disease.”
Brain tumors
Recurrent GBM represents approximately 15% of
all primary adult brain tumors and remains a high unmet clinical
need that affects roughly 74,000 people worldwide annually.i,ii GBM
is an aggressive form of brain cancer with recurrence rates near
90%, and prognosis for patients is poor with treatment often
combining multiple approaches including surgery, radiation, and
chemotherapy. Patients with recurrent GBM typically have a mOS of
6-7 months, and overall survival in patients who have failed
temozolomide, bevacizumab or equivalent salvage chemotherapy, is
approximately 3-5 months.iii,iv DIPG is a rapidly progressive brain
tumor of children with tragically near universal fatal outcomes and
a median survival less than 12 months.v
About ZIOPHARM Oncology,
Inc.:
ZIOPHARM Oncology is a Boston,
Massachusetts-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 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.
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/.
Accessed March 24, 2015. ii McCubrey JA, LaHair MM, Franklin RA.
OSU—0312 in the treatment of glioblastoma. Mol Pharmacol.
2006;70:437-439. iii Omuro, A. Glioblastoma and Other
Malignant Gliomas. A Clinical Review JAMA. 2013 Nov
6;310(17):1842-50. iv Iwamoto et al. Patterns or relapse and
prognosis after bevacizumab failure in recurrent glioblastoma.
Neurology 2009; 73(15):1200-1206v 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.
David Pitts
Argot Partners
212-600-1902
david@argotpartners.com
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