Ziopharm Oncology, Inc. (Nasdaq:ZIOP), a biotechnology company
focused on development of next generation immunotherapies utilizing
gene- and cell-based therapies to treat patients with cancer, today
announced its financial results for the fourth quarter and year
ended December 31, 2017, and provided an update on the Company’s
recent activities.
“This is an exciting year for Ziopharm and the
clinical development of our two platform technologies, Controlled
IL-12 and Sleeping Beauty,” said Laurence Cooper, M.D., Ph.D.,
Chief Executive Officer of Ziopharm. “With Sleeping Beauty, we
achieved major milestones in 2017 and are looking forward to what
is ahead in 2018. We plan to move our point-of-care technology into
the clinic and, for the first time ever, we intend to infuse
patients with T cells genetically modified and manufactured without
virus using the Sleeping Beauty platform within two days from
harvesting the T cells from patients. Also,
Sleeping-Beauty-modified T-cell receptors targeting neoantigens –
the very mutations that cause solid tumors – will enter the clinic
with Dr. Steven Rosenberg’s team at the National Cancer
Institute.”
Dr. Cooper continued, “With Controlled IL-12, in
addition to investigating this cytokine as a monotherapy, we look
forward to seeing data from our first combination of IL-12 and an
immune checkpoint inhibitor, and exploring its impact on the care
options for the treatment of patients with brain cancer. We believe
Controlled IL-12 is a powerful platform that can turn cold tumors
hot and has the potential for broad applicability across
oncology.”
Program Updates
Controlled IL-12
Ziopharm is advancing Ad-RTS-hIL-12 plus
veledimex as a gene therapy product candidate to treat patients
with recurrent glioblastoma (rGBM). Ad-RTS-hIL-12 is an adenoviral
vector administered via a single injection into the tumor and
engineered to conditionally express human IL-12, a powerful
cytokine that has demonstrated 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 crosses the blood-brain
barrier.
Combination Trial with Checkpoint
Inhibitor in rGBM Initiated. As announced earlier this
year in January, Ziopharm has initiated a trial of adult patients
with rGBM to evaluate a single dose of Ad-RTS-hIL-12 plus veledimex
in combination with OPDIVO® (nivolumab), an immune checkpoint
inhibitor targeting programmed death-1 (PD-1).
Plans to Initiate Pivotal Trial in rGBM
in Second Half of 2018. Ziopharm plans to initiate a
pivotal trial for Ad-RTS-hIL-12 plus veledimex for the treatment of
patients with rGBM in the second half of 2018. As previously
disclosed, Ziopharm is currently in the process of completing
Chemistry Manufacturing and Control technical requirements for a
planned Phase 3 clinical trial, subject to regulatory approval.
Phase 1 Trial for Pediatric Brain Tumors
Ongoing. Ziopharm is enrolling pediatric patients in its
Phase 1 trial of Ad-RTS-hIL-12 with veledimex for the treatment of
brain tumors.
Fourth Quarter Highlights
In November 2017, Ziopharm presented updated
data from its Phase 1 trial of Ad-RTS-hIl-12 plus veledimex to
treat patients with rGBM that supports a survival benefit and the
underlying immune system mechanism at the 22nd Annual Meeting
and Education Day of the Society for Neuro-Oncology.
The data showed median overall survival (mOS) of
12.5 months sustained for patients treated with Ad-RTS-hIL-12 plus
20 mg of veledimex (n=15) at a mean follow-up time of 11.1 months
as of October 18, 2017. This mOS compares favorably to the 5 to 8
months survival established in historical controls for patients
with rGBM. Additional highlights observed included:
- An anti-tumor effect evident with centralized review of
magnetic resonance imaging showing decreasing size of brain tumor
lesions in patients;
- Immunohistochemistry analyses from three of three patient
biopsies greater than four months after completion of veledimex
demonstrated that IL-12 results in an extensive infiltration of
CD8+ T cells within the tumor;
- These same biopsies showed sustained production of
interferon-gamma, a cytokine crucial to arming an immune response
in the tumor microenvironment;
- Interferon-gamma was undetectable in the peripheral blood at
the time of biopsies providing further evidence of an on-target
T-cell response;
- These same biopsies demonstrated evidence of an anti-tumor
response;
- These same biopsies showed upregulation of both PD-1 and PD-L1,
which suggests added potential efficacy for combining Ad-RTS-hIL-12
plus veledimex with an immune checkpoint inhibitor;
- Ratio of circulating killer CD8+ T cells to suppressor
FOXP3+ T cells correlates with overall survival;
- Patients in the trial who received low-dose systemic
corticosteroids for peri-operative management have a much better
survival rate than those who received higher doses of
corticosteroids, as the latter presumably interferes with immune
activation;
- Ad-RTS-hIL-12 plus veledimex continues to be well tolerated, as
adverse events (AE) in the trial were predictable and reversible,
neurologic AEs were relatively mild and transient, and there were
no drug-related deaths.
Adoptive Cell TherapiesUsing Ziopharm’s
non-viral approach leveraging Sleeping Beauty to genetically modify
cells, the Company 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
Precigen Inc., a wholly-owned subsidiary of Intrexon Corporation,
and with MD Anderson Cancer Center, the National Cancer Institute
and Merck KGaA, Darmstadt, Germany. This non-viral approach
to genetically modifying T cells has the potential to reduce the
costs of and expand access to this immunotherapy based on very
rapid production and thus avoiding the need for centralized
manufacturing.
Initiation of First Point-of-Care
Clinical Trial Expected in 2018. Ziopharm is
advancing the Sleeping Beauty platform towards point-of-care
manufacturing for the very rapid manufacturing of genetically
modified CAR+ T cells, with Ziopharm’s first clinical trial
utilizing this approach expected to begin in the second half of
2018. Ziopharm’s third-generation point-of-care trial intends to
use the Sleeping Beauty platform to manufacture CAR+ T cells
co-expressing membrane-bound interleukin-15, or mbIL15, within two
days after harvesting T cells from the patient.
Phase 1 Trial of Sleeping
Beauty-Modified TCRs to Treat
Solid Tumors to Initiate in Second Half of 2018. The
NCI anticipates initiation of a Phase 1 trial in the second half of
2018 to evaluate adoptive cell transfer (ACT)-based immunotherapies
genetically modified using the Sleeping Beauty
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 Sleeping Beauty system to express TCRs that recognize specific
immunogenic mutations, or neoantigens, expressed within a patient's
cancer.
Phase 1 Trial of CD33-specific
CAR+ T Therapy for Acute Myeloid Leukemia
(AML). Enrollment is underway at MD Anderson Cancer
Center in the Phase 1 adoptive cellular therapy clinical trial of
CAR+ T-cell therapy in patients with refractory/recurrent AML
that express CD33. This trial infuses autologous T cells
genetically modified with lentivirus to express a CD33-specific CAR
and a cetuximab-activated kill switch for elimination of
genetically modified cells. Data from this trial are expected to
serve as the basis for evaluating CD33 as a potential target for
further development using non-viral manufacturing of T cells with
Ziopharm’s point-of-care technology.
Fourth Quarter Update
Data supporting the third-generation
point-of-care technology were presented at the 59th American
Society of Hematology (ASH) Annual Meeting in December 2017, where
first- and second-generation Sleeping Beauty clinical trial data
demonstrated tolerability, disease response including long-term
survival, and sustained persistence of infused CD19-specific
CAR+ T cells. Ziopharm also presented at ASH preclinical data
demonstrating that Sleeping Beauty can manufacture CAR+ T
cells co-expressing mbIL15 in less than two days. In addition,
preclinical data were also presented last month at the 2018
Keystone Symposia Emerging Cellular Therapies: T Cells and Beyond.
These data further demonstrated that T cells expressing
CD19-specific CAR with mbIL15 could be generated with the Sleeping
Beauty system in less than two days and did not require ex
vivo activation or propagation. Ziopharm observed in these
trials that T cells designed to express mbIL15 showed greater
persistence and more potent antitumor activity than comparator T
cells without mbIL15.
Graft-versus-Host Disease (GvHD)
UpdateFollowing an in-depth review of Ziopharm’s research
and development portfolio, management made the strategic decision
to focus resources on developing Controlled IL-12 and Sleeping
Beauty platforms for oncology indications and to stop development
of engineered cell therapies for the treatment of GvHD. Ziopharm
reverted its rights to the GvHD program to Precigen and is winding
down its final clinical activities.
Fourth Quarter 2017 Financial Results
- Net loss applicable to the common stockholders for the fourth
quarter of 2017 was $18.3 million, or $(0.13) per share, compared
to a net loss of $14.8 million, or $(0.11) per share, for the
fourth quarter of 2016. The increase is primarily due to an
increase in operating expenses of $2.3 million and an increase of
$1.5 million related to the value of preferred stock
dividends.
- Research and development expenses were $11.2 million for the
fourth quarter of 2017, compared to $9.4 million for the fourth
quarter of 2016. The increase in research and development expenses
for the three months ended December 31, 2017 is primarily due to
expanded development in Ziopharm’s gene and cell therapy
programs.
- General and administrative expenses were $3.9 million for the
fourth quarter of 2017, compared to $3.3 million for the fourth
quarter of 2016.
- Ziopharm ended the quarter with unrestricted cash resources of
approximately $70.9 million.
- As part of Ziopharm’s strategic co-development activities at MD
Anderson Cancer Center, a prepayment of approximately $31.9 million
remains available for programs to be conducted by the Company at MD
Anderson Cancer Center under the current Research and Development
Agreement.
- Ziopharm believes its current resources will be sufficient to
fund its currently planned operations into the fourth quarter of
2018.
ZIOPHARM Oncology, Inc. |
Statements of Operations |
(in thousands except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended |
Year Ended |
|
|
|
December 31, |
December 31, |
|
|
|
(unaudited) |
|
(audited) |
|
|
|
|
2017 |
|
|
|
2016 |
|
|
|
2017 |
|
|
|
2016 |
|
|
|
|
|
|
|
|
|
|
|
Collaboration
revenue |
|
|
$ |
1,597 |
|
|
$ |
1,597 |
|
|
$ |
6,389 |
|
|
$ |
6,861 |
|
|
|
|
|
|
|
|
|
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
|
11,181 |
|
|
|
9,389 |
|
|
|
45,084 |
|
|
|
157,791 |
|
General
and administrative |
|
|
|
3,852 |
|
|
|
3,319 |
|
|
|
14,798 |
|
|
|
14,377 |
|
Total
operating expenses |
|
|
|
15,033 |
|
|
|
12,708 |
|
|
|
59,882 |
|
|
|
172,168 |
|
|
|
|
|
|
|
|
|
|
|
Loss from
operations |
|
|
|
(13,436 |
) |
|
|
(11,111 |
) |
|
|
(53,493 |
) |
|
|
(165,307 |
) |
|
|
|
|
|
|
|
|
|
|
Other
income (expense), net |
|
|
|
166 |
|
|
|
32 |
|
|
|
465 |
|
|
|
134 |
|
Change in
fair value of derivative liabilities |
|
|
|
(3 |
) |
|
|
(145 |
) |
|
|
(1,295 |
) |
|
|
(124 |
) |
Net
loss |
|
|
|
(13,273 |
) |
|
|
(11,224 |
) |
|
|
(54,323 |
) |
|
|
(165,297 |
) |
Preferred
stock dividends |
|
|
|
(4,999 |
) |
|
|
(3,532 |
) |
|
|
(18,938 |
) |
|
|
(7,123 |
) |
Net loss applicable to common stockholders |
|
$ |
(18,272 |
) |
|
$ |
(14,756 |
) |
|
$ |
(73,261 |
) |
|
$ |
(172,420 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted net
loss per share |
|
|
$ |
(0.13 |
) |
|
$ |
(0.11 |
) |
|
$ |
(0.53 |
) |
|
$ |
(1.32 |
) |
|
|
|
|
|
|
|
|
|
|
Weighted
average common shares outstanding used |
|
|
|
|
|
|
|
|
to
compute basic and diluted net loss per share |
|
|
140,644,238 |
|
|
|
130,524,204 |
|
|
|
136,938,264 |
|
|
|
130,391,463 |
|
ZIOPHARM Oncology, Inc. |
Balance Sheet Data |
(in thousands) |
(audited) |
|
|
|
|
|
|
|
December 31, |
|
December 31, |
|
|
2017 |
|
2016 |
|
|
|
|
|
Cash and cash
equivalents |
|
70,946 |
|
|
81,053 |
|
Working capital |
|
69,927 |
|
|
89,075 |
|
Total assets |
|
105,606 |
|
|
106,348 |
|
Total stockholders'
(deficit) |
|
(96,806 |
) |
|
(77,298 |
) |
|
|
|
|
|
|
|
Conference Call and Slide
Webcast
Ziopharm will host a conference call and webcast
slide presentation today, March 1, at 4:30 p.m. ET. The call can be
accessed by dialing 1-844-309-0618 (U.S. and Canada) or
1-661-378-9465 (international). The passcode for the conference
call is 3782628. 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 Ziopharm's
website for two weeks.
About Ziopharm Oncology,
Inc.
Ziopharm Oncology is a Boston-based
biotechnology company focused on development of next-generation
immunotherapies utilizing gene- and cell-based therapies to treat
patients with cancer. In partnership with Precigen Inc., a
wholly-owned subsidiary of Intrexon Corporation (NYSE:XON),
Ziopharm is focused on the development of two platform technologies
designed to deliver safe, effective and scalable cell- and
viral-based therapies for the treatment of multiple cancer types:
Controlled IL-12 and Sleeping Beauty for genetically modifying T
cells. The Company’s lead gene therapy product candidate,
Ad-RTS-hIL-12 plus veledimex, has demonstrated in clinical trials
the potential to control interleukin-12, leading to an infiltration
of T cells that fight brain cancer. The Company also is advancing
therapies using Sleeping Beauty, a non-viral approach to
genetically modify chimeric antigen receptor (CAR+) and T-cell
receptor (TCR+) T cells, which target specific antigens in blood
cancers and neoantigens solid tumors. Sleeping Beauty is designed
using the Company’s point-of-care technology, a shortened
manufacturing process which potentially can be developed as a
decentralized manufacturing process based in hospitals. These
programs are being advanced in collaboration with Precigen and with
MD Anderson Cancer Center, the National Cancer Institute and Merck
KGaA, Darmstadt, Germany.
Forward-Looking Disclaimer This
press release contains certain forward-looking statements within
the meaning of the Private Securities Litigation Reform Act of
1995, as amended. Forward-looking statements are statements that
are not historical facts. The words “anticipate,” “believe,”
“estimate,” “expect,” “intend,” “may,” “plan,” “predict,”
“project,” “would,” “could,” “potential,” “possible,” “hope” and
similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain
these identifying words. These statements include, but are not
limited to, statements regarding the statements regarding the
Company’s financial condition and results of operations; the
advancement of, and anticipated development and regulatory
milestones and plans related to the Company’s product candidates
and clinical studies including the Company’s research, clinical
development and manufacturing plans for its Ad-RTS-hIL-12 plus
veledimex gene therapy product candidate; the application of the
Company’s platforms for the treatment of oncology and the progress
and timing of the development of its research and development
programs; plans related to the Company’s collaborations,
particularly with Precigen and National Cancer Institute; and the
Company’s ability to establish a commercially-viable manufacturing
approach and its expected outcomes. All 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: changes in the
Company’s financial condition and cash needs, funding or other
strategic opportunities that become available to the Company, 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 Annual Report on Form 10-K
for the fiscal quarter ended December 31, 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.
Contact:David ConnollyZIOPHARM
Oncology617-502-1881dconnolly@ziopharm.com
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