- Phase 1 Trial of Sleeping Beauty-TCR-T cell
therapy for patients with solid tumors to begin at National Cancer
Institute in mid-2019 –- Third-generation Sleeping Beauty
CD19-specific CAR-T U.S. trial to begin in 2H2019 – -
Eden BioCell to advance third-generation Sleeping Beauty CAR-T for
Greater China – - Third cohort enrolling patients in
Controlled IL-12 combination trial with OPDIVO® for recurrent
glioblastoma (rGBM) – - Phase 2 trial of Controlled IL-12 in
combination with Regeneron’s Libtayo® for patients with rGBM
expected to open 2Q2019 – - Company to host conference call
today at 4:30 p.m. ET –
Ziopharm Oncology, Inc. (Nasdaq:ZIOP) today announced its financial
results for the fourth quarter and year ended December 31, 2018,
and provided an update on the Company’s recent activities.
“We are executing on our strategy to focus on
solid tumors with our Sleeping Beauty TCR-T program and our
Controlled IL-12 platform and advancing a solution that addresses
the cost and complexity of CAR-T therapies,” said Laurence Cooper,
M.D., Ph.D., CEO of Ziopharm. “Under our collaboration at the
National Cancer Institute (NCI), we expect to begin treating
patients with solid tumors mid-year with the first non-viral,
neoantigen-specific TCR-T cell therapy designed to attack the very
mutations that cause cancer. In addition, we are advancing - in
both the United States and greater China - our non-viral CAR-T
therapy to solve the issues standing in the way of commercial
success for approved CD19-specific CAR-T therapies. And, with
maturing data showing a positive effect on overall survival for
patients with recurrent glioblastoma, there is growing excitement
for our Controlled IL-12 platform among treating physicians.”
David Mauney, M.D., President of Ziopharm,
added, “We have significant momentum following our transformational
fourth quarter 2018 when we established a new license agreement
that provides us with clinical development autonomy. We secured two
new business development deals and strengthened our balance sheet
by eliminating $157 million in preferred stock and raising $50
million in a private placement. Thus, we are now well positioned to
achieve multiple milestones and to be in the clinic in 2019 with
each of our pillar programs: TCR-T, CAR-T and Controlled
IL-12.”
Program Updates
Sleeping Beauty TCR-T Therapies
The Company is using its Sleeping Beauty
platform to develop a personalized T-cell therapy targeting solid
tumors with T-cell receptors or TCRs. Under a Cooperative Research
and Development Agreement (CRADA), the NCI is expected to initiate
a Phase 1 clinical trial to treat patients who may have one of a
variety of solid tumors using the Sleeping Beauty platform to
genetically modify T-cells to target patient-specific
neoantigens.
- Phase 1 trial for TCR-T cell therapy expected to begin
in mid-2019: This trial is on track to begin treating
patients in mid-2019 under the direction of Steven A. Rosenberg,
M.D., Ph.D., Chief of the Surgery Branch at the NCI.
- CRADA extended: Ziopharm announces that the
Company and the NCI agreed to extend the CRADA governing this
program to evaluate genetically modified T cells targeting solid
tumors, which was established in January 2017. This agreement has
been extended through January 2022.
Sleeping Beauty CAR-T
Therapies
Ziopharm is advancing the Sleeping Beauty
platform towards the very rapid manufacturing of genetically
modified CAR+ T cells, co-expressing membrane-bound interleukin-15,
or mbIL15, with a safety switch, within two days after genetically
modifying T cells from the patient. This work is being done
in collaboration with the University of Texas MD Anderson
Cancer Center in the United States and will be done in Greater
China through a joint venture, Eden BioCell.
- Third-generation Phase 1 trial for very rapid
manufacturing of Sleeping Beauty CD19-specific CAR-T with mbIL15
expected to begin 2H2019: The Company affirms guidance on
beginning this trial and treating patients at MD Anderson Cancer
Center in the second half of this year. Ziopharm announced in June
2018 that the FDA placed this investigator-led IND on clinical hold
and requested additional information relating to chemistry,
manufacturing and controls, specifically requesting that the
product meet a minimum threshold for T-cell viability. The Company,
in partnership with MD Anderson Cancer Center, has made progress
toward achieving this threshold in manufacturing through improved
engineering and cell processing.
- Eden BioCell to advance third-generation Sleeping
Beauty CAR-T for Greater China: In December 2018, Ziopharm
in conjunction with TriArm Therapeutics announced Eden BioCell will
be launched to develop and commercialize Sleeping Beauty-generated
CD19-specific CAR-T therapies in the People’s Republic of China
(including Macau and Hong Kong), Taiwan and Korea. The teams have
begun meeting to prepare for technology transfer and launch the new
company. Ziopharm and TriArm each will own 50 percent of the joint
venture. Eden BioCell will be funded with up to $35 million from
TriArm, a privately-owned cell therapy company that was formed by
Panacea Venture Healthcare, a fund co-founded and managed by James
Huang, Managing Partner of Kleiner Perkins Caufield & Byers
China. Ziopharm and TriArm expect to close on this joint venture in
the first half of 2019. Ziopharm’s CEO Laurence Cooper, and Panacea
Venture Healthcare co-founder James Huang will serve on
Eden BioCell’s Board of Directors with each party sharing
decision-making authority. Ziopharm looks forward to providing an
update on clinical development plans for Eden BioCell later in the
year.
Controlled IL-12 Ziopharm is
developing its Controlled IL-12 platform, or Ad-RTS-hIL-12 plus
veledimex, as a drug to control the production of human interleukin
12 (hIL-12) which activates the immune system to recruit
cancer-fighting T cells into tumors. In the setting for the
treatment of recurrent glioblastoma (rGBM), Ziopharm is advancing
Ad-RTS-hIL-12 plus veledimex as a monotherapy and in combination
with immune checkpoint inhibitors.
- Enrollment completed in Phase 1 monotherapy expansion
substudy: Ziopharm announced that it rapidly completed
enrollment and treated a total of 36 patients in less than six
months in a substudy to expand a Phase 1 trial evaluating its
Controlled IL-12 platform as a monotherapy for the treatment of
rGBM. The trial was over enrolled by eleven patients more than the
target goal of 25, which the Company attributes to enthusiasm
stemming from encouraging survival and tumor biopsy data.
Preliminary data from this substudy is expected to be presented in
2019.
- Third cohort has begun in combination substudy
with OPDIVO® (nivolumab): Ziopharm today
announced that it has completed two dosing cohorts in its Phase 1
substudy of adult patients with rGBM to evaluate a single dose of
Ad-RTS-hIL-12 plus daily veledimex in combination with OPDIVO®
(nivolumab), an immune checkpoint inhibitor targeting programmed
death-1 (PD-1). The Company has begun the third cohort for this
study to evaluate the safety and tolerability of this combination
regimen, establish optimal dosing of veledimex and nivolumab, and
measure overall patient survival. The Company expects to complete
enrollment in the third cohort in the second quarter this year and
looks forward to presenting preliminary data from this trial in
2019.
- Phase 2 combination trial with Regeneron’s Libtayo®
(cemiplimab-rwlc) expected to open 2Q2019: The Company, in
collaboration with Regeneron Pharmaceuticals, expects to open a
Phase 2 trial to evaluate Ad-RTS-hIL-12 plus veledimex in
combination with Regeneron’s PD-1 antibody Libtayo®
(cemiplimab-rwlc) for treating patients with rGBM. The Company
expects to enroll approximately 30 patients in this trial.
- Phase 1 for pediatric 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 at
multiple U.S. sites.
Corporate Update
In addition to the clinical collaboration with
Regeneron and the execution of an agreement to launch Eden BioCell
during the fourth quarter of last year, Ziopharm on October 9,
2018, announced that it entered into a new licensing agreement that
replaced all existing agreements with Intrexon Corp. and its
subsidiary Precigen, Inc. Under the new license agreement, Ziopharm
has full developmental control and exclusivity utilizing Sleeping
Beauty for TCRs for the treatment of cancer. The CRADA with the NCI
related to Sleeping Beauty-generated T cells expressing TCRs to
target neoantigens within solid tumors was transferred to Ziopharm
and the Company will maintain this program. Ziopharm will build on
its Controlled IL-12 platform with exclusive access to Precigen’s
RheoSwitch Therapeutic System® gene switch with adenovirus for the
treatment of cancer. Using the Sleeping Beauty system, Ziopharm
will continue to exclusively advance its CD19-specific chimeric
antigen receptor (CAR) program leveraging membrane-bound
interleukin 15, while retaining rights to a second, unnamed CAR
target. Ziopharm has sole oversight for the relationship with MD
Anderson Cancer Center, the Company’s initial development partner
for the Sleeping Beauty platform.
As part of the new licensing agreement, Ziopharm
successfully negotiated the complete elimination of preferred stock
that had been issued to Intrexon that was valued at approximately
$157 million at that time.
In December, David Mauney, M.D., was promoted to
President. Dr. Mauney had joined the Company in September 2017 as
Executive Vice President and Chief Business Officer.
Fourth-Quarter 2018 Financial
Results
- Net income (loss) applicable to the common shareholders for the
fourth quarter of 2018 was $194.5 million,
or $1.29 per share, basic and diluted, compared to a net
loss of $18.3 million, or $(0.13) per share, basic
and diluted, for the fourth quarter of 2017. The increased income
attributable to common shareholders resulted primarily from the
forfeiture and return of all of the Company’s Series 1 preferred
stock held by Intrexon Corporation and the relinquishment of
Ziopharm’s obligations under the Ares Trading Agreement.
- Research and development expenses were $8.2
million for the fourth quarter of 2018, compared to $11.2
million for the fourth quarter of 2017. The decrease in
research and development expenses for the three months
ended Dec. 31, 2018 is primarily due to decreased
preclinical activity related to our cell and gene therapy
programs.
- General and administrative expenses were $4.6
million for the fourth quarter of 2018, compared to $3.9
million for the fourth quarter of 2017. The increase in
general and administrative expenses for the three months
ended Dec. 31, 2018 is primarily due to contracted
outside service costs.
Full Year 2018 Financial
Results
- Net income (loss) applicable to the common shareholders for the
year ended December 31, 2018 was $137.2 million,
or $0.96 per share, basic and diluted, compared to a net
loss applicable to the common shareholders of $73.3 million,
or $(0.53) per share, basic and diluted, for the year
ended December 31, 2017. The increased income attributable to
common shareholders resulted primarily from the forfeiture and
return of all of the Company’s Series 1 preferred stock held by
Intrexon Corporation and the relinquishment of Ziopharm’s
obligations under the Ares Trading Agreement.
- Research and development expenses were $34.1
million for the year ended December 31, 2018, compared
to $45.1 million for the year ended December 31, 2017.
The decrease in research and development expenses for the year
ended December 31, 2018, is primarily due to decreased
preclinical activity related to our cell and gene therapy
programs.
- General and administrative expenses were $19.9
million for the year ended December 31, 2018, compared
to $14.8 million for the Year ended December 31, 2017.
The increase in general and administrative expenses for the year
ended December 31, 2018, is primarily due to contracted
outside service costs.
The Company ended the year with unrestricted
cash resources of approximately $61.7 million.
In addition, a prepayment of
approximately $27.8 million remains for programs to be
conducted by the Company 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 second
quarter of 2020.
ZIOPHARM Oncology, Inc. |
|
Statements of Operations |
|
(in thousands except share and per share
data) |
|
(unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended
|
Year Ended |
|
|
|
|
December 31,
|
December 31, |
|
|
|
|
(unaudited) |
|
(audited) |
|
|
|
|
|
|
2018 |
|
|
|
2017 |
|
|
|
|
2018 |
|
|
|
2017 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration revenue |
|
|
|
$ |
- |
|
|
$ |
1,597 |
|
|
|
$ |
146 |
|
|
$ |
6,389 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
|
|
8,199 |
|
|
|
11,181 |
|
|
|
|
34,134 |
|
|
|
45,084 |
|
|
General
and administrative |
|
|
|
|
4,563 |
|
|
|
3,852 |
|
|
|
|
19,918 |
|
|
|
14,798 |
|
|
Total
operating expenses |
|
|
|
|
12,762 |
|
|
|
15,033 |
|
|
|
|
54,052 |
|
|
|
59,882 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Loss from
operations |
|
|
|
|
(12,762 |
) |
|
|
(13,436 |
) |
|
|
|
(53,906 |
) |
|
|
(53,493 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
Other
income (expense), net |
|
|
|
|
168 |
|
|
|
166 |
|
|
|
|
631 |
|
|
|
465 |
|
|
Change in
fair value of derivative liabilities |
|
|
|
|
113 |
|
|
|
(3 |
) |
|
|
|
158 |
|
|
|
(1,295 |
) |
|
Net
loss |
|
|
|
|
(12,481 |
) |
|
|
(13,273 |
) |
|
|
|
(53,117 |
) |
|
|
(54,323 |
) |
|
Preferred
stock dividends |
|
|
|
|
(342 |
) |
|
|
(4,999 |
) |
|
|
|
(16,998 |
) |
|
|
(18,938 |
) |
|
Settlement of a related party relationship |
|
|
|
|
207,361 |
|
|
|
- |
|
|
|
|
207,361 |
|
|
|
- |
|
|
Net Income (loss) applicable to common stockholders |
|
|
$ |
194,538 |
|
|
$ |
(18,272 |
) |
|
|
$ |
137,246 |
|
|
$ |
(73,261 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net income (loss) per
share - basic |
|
|
|
$ |
1.29 |
|
|
$ |
(0.13 |
) |
|
|
$ |
0.96 |
|
|
$ |
(0.53 |
) |
|
Net income (loss) per
share - diluted |
|
|
|
$ |
1.29 |
|
|
$ |
(0.13 |
) |
|
|
$ |
0.96 |
|
|
$ |
(0.53 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
Weighted average common
shares outstanding used |
|
|
|
|
|
|
|
|
|
|
to compute basic
net income (loss) per share |
|
|
|
|
150,893,470 |
|
|
|
140,644,238 |
|
|
|
|
143,508,674 |
|
|
|
136,938,264 |
|
|
Weighted average common
shares outstanding used |
|
|
|
|
|
|
|
|
|
|
to compute
diluted net income (loss) per share |
|
|
|
|
151,094,956 |
|
|
|
140,644,238 |
|
|
|
|
143,710,160 |
|
|
|
136,938,264 |
|
|
|
|
|
|
|
|
|
|
|
|
|
ZIOPHARM Oncology, Inc. |
|
Balance Sheet Data |
|
(in thousands) |
|
(unaudited) |
|
|
|
|
|
|
|
|
|
December 31, |
|
December 31, |
|
|
|
2018 |
|
2017 |
|
|
|
|
|
|
|
Cash and cash
equivalents |
|
61,729 |
|
70,946 |
|
Working capital |
|
74,802 |
|
69,927 |
|
Total assets |
|
95,051 |
|
105,606 |
|
Total stockholders'
equity (deficit) |
|
85,564 |
|
(96,806 |
) |
|
|
|
|
|
|
Conference Call and Slide
Webcast
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 3091306. To access the
slides and live 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 an immuno-oncology company focused on
developing end-to-end cost-effective solutions using its non-viral
Sleeping Beauty platform for TCR and CAR T-cell therapies and
immune-stimulating gene therapy with Controlled interleukin 12
(IL-12). The Sleeping Beauty platform genetically modifies T cells
with DNA plasmids to express T-cell receptors (TCRs) to target
specific antigens in solid tumors and chimeric antigen receptors
(CARs) to target CD19 in blood cancers with the Company’s very
rapid (3rd generation) T-cell manufacturing process. The Sleeping
Beauty platform is being advanced in collaboration with the
National Cancer Institute, The University of Texas MD Anderson
Cancer Center and Eden BioCell. The Company also is developing its
Controlled IL-12 platform or Ad-RTS-hIL-12 plus veledimex as
monotherapy and in combination with immune checkpoint inhibitors to
treat brain cancer, including in collaboration with Regeneron
Pharmaceuticals.
Forward-Looking Statements
DisclaimerThis press release contains forward-looking
statements as defined in 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 business and
strategic plans, the availability of cash resources, the progress
and timing of the Company's research and development programs,
including the anticipated dates for the initiation, completion and
readouts of its clinical trials, the expected timing and closing of
the joint venture with TriArm and the future funding of Eden
BioCell by TriArm. Although Ziopharm’s management team believes
that the expectations reflected in such forward-looking statements
are reasonable, investors are cautioned that forward-looking
information and statements are subject to various risks and
uncertainties, many of which are difficult to predict and generally
beyond the control of Ziopharm, that could cause actual results and
developments to differ materially from those expressed in, or
implied or projected by, the forward-looking information and
statements. These risks and uncertainties include among other
things, changes in our operating plans that may impact our cash
expenditures, the uncertainties inherent in research and
development, future clinical data and analysis, including whether
any of Ziopharm’s product candidates will advance further in the
preclinical research or clinical trial process, including receiving
clearance from the U.S. Food and Drug Administration or equivalent
foreign regulatory agencies to conduct clinical trials and whether
and when, if at all, they will receive final approval from the U.S.
FDA or equivalent foreign regulatory agencies and for which
indication; the strength and enforceability of Ziopharm’s
intellectual property rights; competition from other pharmaceutical
and biotechnology companies as well as risk factors discussed or
identified in the public filings with the Securities and Exchange
Commission made by Ziopharm, including those risks and
uncertainties listed in Ziopharm’s Annual Report on Form 10-K filed
by Ziopharm with the Securities and Exchange Commission. We are
providing this information as of the date of this press release,
and Ziopharm does not undertake any obligation to update or revise
the information contained in this press release whether as a result
of new information, future events or any other reason.
Company contacts:
David Connolly Vice
President, Corporate Communications and Investor Relations
617-502-1881 dconnolly@ziopharm.com |
Mike MoyerVice President,
Portfolio Strategy617-765-3770mmoyer@ziopharm.com |
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