ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company
focused on new immunotherapies, today announced its financial
results for the second quarter ended June 30, 2017, and provided an
update on the Company's recent activities.
“ZIOPHARM is unique in the breadth and stage of
technologies delivering cell- and gene-based therapies that are
impactful, controllable and, most critically, scalable,” said
Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM
Oncology. “We have seen ongoing validation of RheoSwitch® gene
switch technology through the now expanded Ad-RTS-hIL-12 +
veledimex study in recurrent glioblastoma. We are addressing the
complexities of commercializing CAR-T, T-cell receptor, and other
cell-based therapies with our rapid manufacture of patient-derived
T cells under point-of-care and our off-the-shelf natural killer
cells. The Sleeping Beauty system enables us to generate CAR-T
cells co-expressing important biological effectors such as membrane
bound IL-15 and uniquely positions us to express TCRs to
efficiently target multiple neoantigens in solid tumors. This
non-viral approach to gene therapy not only allows us to customize
immunotherapies, but enables controllable and cost-effective T
cells with curative potential in both solid and hematologic
malignancies.”
“During the quarter, we presented updated data
at ASCO that linked immune response to overall survival in patients
with recurrent glioblastoma when treated with Ad-RTS-hIL-12 +
veledimex. Additionally, we initiated a stereotactic arm in this
study as a lead-in to both the pediatric and immune checkpoint
combination trials. We continue to make progress toward finalizing
a registration path for Ad-RTS-hIL-12 + veledimex while evaluating
partnership opportunities for the program,” added Francois Lebel,
M.D., Executive Vice President, Research and Development, Chief
Medical Officer of ZIOPHARM Oncology. “In our cell therapy
programs, we continue to progress towards clinical evaluation of
Sleeping Beauty engineered peripheral blood lymphocytes to express
TCRs targeting solid tumors, we are advancing our non-viral
point-of-care approach for CAR-T therapies, and we also anticipate
the imminent initiation of the Phase 1 trial to evaluate CD33 as a
new target for CAR+ T cells in patients with relapsed or refractory
acute myeloid leukemia (AML), a highly aggressive and underserved
disease. Additionally, we look forward to the initiation of a Phase
1 trial evaluating our off-the-shelf primary NK Cells, which have
demonstrated the ability to eliminate AML in preclinical
models.”
Recent Updates
Adoptive Cell Therapies
ZIOPHARM is developing chimeric antigen receptor
(CAR) T cell (CAR+ T), T-cell receptor (TCR) T cell (TCR+ T), and
natural killer (NK) adoptive cell-based therapies. These programs
are being advanced in collaboration with Intrexon Corporation
(NYSE:XON) and selectively with MD Anderson Cancer Center, the
National Cancer Institute (NCI) and/or Merck Serono, the
biopharmaceutical business of Merck KGaA.
Announced FDA Acceptance of IND for
CD33-Specific CAR-T Cell Therapy Targeting Relapsed/Refractory
Acute Myeloid Leukemia. In May 2017, ZIOPHARM 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 cells for
relapsed or refractory AML is active, with the first patient
expected to begin treatment in the third quarter of 2017. The
genetically modified T cells incorporate a kill switch to eliminate
the infused product if there are serious adverse safety events.
Announced Advancement of Next-Generation
Non-Viral CAR-T Cell Platform
Empowered by Membrane-Bound IL-15 Under RTS® Gene Switch
Control. In April 2017, ZIOPHARM and its partners,
Intrexon and Merck KGaA, Darmstadt, Germany, announced the
advancement of a unique approach to develop therapeutic candidates
for two CAR targets expressed on hematologic malignancies and solid
tumors. The distinctive methodology centers on the proprietary RTS®
platform to regulate production of membrane-bound interleukin-15
(mbIL15) co-expressed with CARs and Sleeping Beauty, a non-viral
genetic modification system to genetically modify clinical-grade T
cells. The companies expect to advance this innovative approach
towards the clinic in 2018.
The IL-15 cytokine is increasingly recognized as
a key driver of therapeutic effect in CAR+ T therapy, including in
a recent Journal of Clinical Oncology publication which correlated
lymphoma remissions in patients whose IL-15 levels were elevated
after lymphodepleting chemotherapy.i Through the RTS® gene switch,
the expression of mbIL15 can be regulated to help CARs target
cancers in a controlled manner, thus potentiating a new paradigm in
T-cell therapy.
Additionally, the non-viral Sleeping Beauty
transposon-transposase system is an exceptional therapeutic tool
that holds multiple advantages over viral-based delivery systems
for stably introducing genes encoding CARs and TCRs into T cells.
It simplifies genetic modification, and when coupled with reduced
ex vivo processing, offers a pathway to shortened manufacturing and
personalized T-cell therapies.
Continued to Advance Research under the
Cooperative Research and Development Agreement with the National
Cancer Institute Utilizing the Sleeping Beauty System to Generate
TCR-modified T cells Targeting Neoantigens. ZIOPHARM
continued to advance research being carried out under the
Cooperative Research and Development Agreement (CRADA) that the
Company maintains with the NCI. Announced in January 2017 by
ZIOPHARM and partner Intrexon, research conducted under the CRADA
will be carried out under the direction of Steven A. Rosenberg,
M.D., Ph.D., Chief of the Surgery Branch at the NCI's Center for
Cancer Research. Dr. Rosenberg and his team are advancing the
Sleeping Beauty system into clinical trials to target neoantigens
in solid tumors on a patient-by-patient basis. These efforts are
based on published works in which the Sleeping Beauty system is
described as the most clinically advanced non-viral gene transfer
tool to reprogram T cells for personalized immunotherapy.ii,iii
Ad-RTS-hIL-12 + veledimex
Ad-RTS-hIL-12 + veledimex is ZIOPHARM’s gene
therapy candidate for the controlled expression of interleukin-12
(IL-12), a critical protein for stimulating an anti-cancer immune
response, using an RTS® inducible gene switch that enables
controlled in vivo expression of therapeutic proteins.
Announced Initiation of Stereotactic
Treatment Cohort in Phase 1 Study of Ad-RTS-hIL-12 + Veledimex in
Recurrent Glioblastoma. In June 2017, ZIOPHARM 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). Stereotactic
treatment will serve as a lead-in to the Company’s next phase of
development for Ad-RTS-hIL-12 + veledimex in brain tumors,
including planned anti‑PD‑1 combination therapy and pediatric
studies.
Announced Positive Updated Results of
Ad-RTS-hIL-12 + Veledimex in Recurrent Glioblastoma at the 2017
ASCO Annual Meeting. In June 2017, ZIOPHARM announced
updated results from its Phase 1 multicenter study of Ad-RTS-hIL-12
+ veledimex, including the 20 mg expansion cohort in patients with
recurrent or progressive GBM at the 2017 American Society of
Clinical Oncology (ASCO) Annual Meeting June 2-6 in Chicago. As of
May 24, 2017, the median overall survival of all patients receiving
intratumoral Ad-RTS-hIL-12 with 20 mg of orally-administered
veledimex was 12.5 months, with a mean follow-up time of 9.2
months. The majority of the 15 patients in the 20 mg cohort had two
or more recurrences prior to entry in the study, indicating very
advanced disease.
Based on the increased ratio of CD8+/FOXP3+
(effector/suppressor) T cells measured in peripheral blood 14 to 28
days after viral injection, overall survival appears correlated
with IL-12-mediated cellular immune activation. Furthermore,
patients who received low dose or no steroids showed a much better
survival rate than those who received elevated systemic steroids,
as the latter presumably interferes with immune activation.
As previously reported, a strong, dose-dependent
correlation between veledimex dose, veledimex blood-brain-barrier
penetration, and IL-12 and IFN-gamma production was observed.
Drug-related toxicities, which were primarily non-neurologic,
showed a dose response to veledimex, were consistent with those
previously reported, and importantly, continue to be reversed upon
cessation of the activator ligand, with no drug-related deaths.
Anticipated and Achieved 2017
Milestones
- Intra-tumoral IL-12 RheoSwitch® programs:- Updated
clinical data from Phase 1 of Ad-RTS-hIL-12 + veledimex for rGBM
presented at ASCO- Initiate pivotal clinical trial for rGBM-
Initiate stereotactic administration of Ad-RTS-hIL-12 +
veledimex for rGBM: - Initiate combination study
of Ad-RTS-hIL-12 + veledimex with anti-PD-1 for rGBM -
Initiate Phase 1 study in the treatment of brain tumors in
children- Update program at the Society of Neuro-Oncology
Annual Meeting
- CAR+ T programs:- Continue CD19-specific CAR+ T
2nd-generation clinical study, enrolling patients under shortened
manufacturing protocol- Advance mbIL15 CD19-specific CAR+ T
3rd generation toward a Phase 1 clinical study evaluating
point-of-care- Initiate a CD33-specific CAR+ T clinical study
in adults and children for relapsed or refractory AML-
Advance CAR+ T preclinical studies for at least one
hematological malignancy under a shortened manufacturing process
towards point-of-care
- TCR programs- Execute CRADA with NCI utilizing Sleeping
Beauty to generate T cells targeting neoantigens for treatment of
patients with solid tumor malignancies- Advance development
of process for delivering personalized gene-modified T-cell
products against neoantigens
- NK cell programs- Initiate a Phase 1 study of
off-the-shelf NK cells for elderly patients with AML not eligible
for standard intensive chemotherapy
- GvHD programs- Advance preclinical studies
Second-Quarter 2017 Financial Results
- Net loss applicable to the common shareholders for the second
quarter of 2017 was $17.7 million, or $(0.13) per share, compared
to a net loss of $131.2 million, or $(1.01) per share, for the
second quarter of 2016. The decrease is primarily due to the
commitment to issue Series 1 preferred stock at fair value under
the 2016 ECP Amendment and 2016 GvHD Amendment with Intrexon
resulting in a $119.1 million non-cash charge during the three
months ended June 30, 2016 that was not incurred during the three
months ended June 30, 2017.
- Research and development expenses were $10.8 million for the
second quarter of 2017, compared to $129.2 million for the second
quarter of 2016. The decrease in research and development expenses
for the three months ended June 30, 2017 is primarily due to the
Series 1 preferred stock issuance previously noted.
- General and administrative expenses were $3.8 million for the
second quarter of 2017, compared to $3.7 million for the second
quarter of 2016.
- The Company ended the quarter with unrestricted cash resources
of approximately $97.2 million, plus approximately $27.3M in cash
on hand at MD Anderson Cancer Center for programs to be conducted
at MD Anderson Cancer Center under the current Research and
Development Agreement. The Company believes its current resources
will be sufficient to fund its currently planned operations into
the fourth quarter of 2018.
Conference Call and Slide
Webcast
ZIOPHARM will host a conference call and webcast
slide presentation today, Monday, July 31, 2017, at 4:30 p.m. ET.
The call can be accessed by dialing (844) 309-0618 (U.S. and
Canada) or (661) 378-9465 (international). The passcode for the
conference call is 53023904. To access the slides and live audio
webcast, or the subsequent archived recording, visit the "Investors
& Media" section of the ZIOPHARM website at www.ziopharm.com.
The webcast will be recorded and available for replay on the
Company's website for two weeks.
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 June 30, 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.
TrademarksRheoSwitch
Therapeutic System® and RTS® are registered trademarks of Intrexon
Corporation.
i Kochenderfer et. al. (2017). Lymphoma Remissions Caused by
Anti-CD19 Chimeric Antigen Receptor T Cells Are Associated With
High Serum Interleukin-15 Levels. Journal of Clinical Oncology,
35(16), 1803-1813. ii Deniger et. al. (2016). Stable, Nonviral
Expression of Mutated Tumor Neoantigen-specific T-cell Receptors
Using the Sleeping Beauty Transposon/Transposase
System. Molecular Therapy, 24(6), 1078-1089. iii
Klebanoff, Rosenberg, & Restifo (2016). Prospects for
gene-engineered T cell immunotherapy for solid cancers. Nature
Medicine, 22(1), 26-36.
(Tables follow)
|
|
ZIOPHARM Oncology, Inc. |
Statements of Operations |
(in thousands except share and per share
data) |
(unaudited) |
|
|
|
|
|
|
|
Three Months Ended |
|
|
June 30, |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
|
Collaboration revenue |
|
$ |
1,597 |
|
|
$ |
1,697 |
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
Research
and development, including cost of contracts |
|
|
10,831 |
|
|
|
129,228 |
|
General
and administrative |
|
|
3,780 |
|
|
|
3,711 |
|
Total
operating expenses |
|
|
14,611 |
|
|
|
132,939 |
|
|
|
|
|
|
Loss from
operations |
|
|
(13,014 |
) |
|
|
(131,242 |
) |
|
|
|
|
|
Other
income (expense), net |
|
|
86 |
|
|
|
42 |
|
Change in
derivative liabilities |
|
|
66 |
|
|
|
- |
|
Net
loss |
|
|
(12,862 |
) |
|
|
(131,200 |
) |
Preferred
stock dividends |
|
|
(4,865 |
) |
|
|
- |
|
Net loss applicable to common stockholders |
$ |
(17,727 |
) |
|
$ |
(131,200 |
) |
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share |
|
$ |
(0.13 |
) |
|
$ |
(1.01 |
) |
|
|
|
|
|
Weighted average common
shares outstanding used |
|
|
|
|
to compute basic
and diluted net loss per share |
|
|
135,630,210 |
|
|
|
130,385,077 |
|
|
|
|
|
|
ZIOPHARM Oncology, Inc. |
Balance Sheet Data |
(in thousands) |
(unaudited) |
|
|
|
|
|
|
|
June 30, |
|
December 31, |
|
|
2017 |
|
2016 |
|
|
|
|
|
Cash and cash
equivalents |
|
97,194 |
|
|
81,053 |
|
Working capital |
|
109,142 |
|
|
89,075 |
|
Total assets |
|
126,813 |
|
|
106,348 |
|
Total stockholders'
(deficit) |
|
(64,274 |
) |
|
(77,298 |
) |
|
|
|
|
|
Contact:
David Pitts
Argot Partners
212-600-1902
david@argotpartners.com
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