Ziopharm Oncology, Inc. (“Ziopharm” or the “Company”) (Nasdaq:
ZIOP), today provided corporate updates for the second quarter of
2021, ended June 30, 2021. The Company will host a conference call
and webcast today at 4:30 pm EDT.
“Since our last update, the Company has made
tremendous progress across multiple fronts,” said Heidi Hagen,
Interim Chief Executive Officer. “The Company has built significant
positive momentum in our TCR-T Library clinical trial enabling
activities and in our efforts to strengthen the balance sheet. This
quarterly update reflects the Company doing exactly what we said we
would do earlier in the year and this is very exciting to see. The
appointment of a permanent CEO is also something we look forward to
in the very near future. The strong science underlying our work
continues to provide us with confidence as we move forward. On
behalf of the Board and management team, I want to thank all our
dedicated researchers, scientists, partners, and the entire
organization for their commitment to our mission.”
Significant Progress in Company’s TCR-T
Library Clinical Program
Since the last quarterly update, the Company
achieved several key milestones in its TCR-T Program. Importantly,
the Company remains on track to begin dosing patients in its Phase
I/II TCR-T Library trial during the second half of 2021, and now
anticipates dosing the first patient during the fourth quarter of
this year. The trial initially targets six individual solid tumor
indications: cholangiocarcinoma, pancreatic cancer, ovarian cancer,
endometrial cancer, colorectal cancer, and lung cancer, which were
selected due to the frequency of KRAS and / or TP53 mutations. The
Company intends on expanding into additional indications in the
future.
The clinical trial will open for enrollment upon
establishing clinical manufacturing readiness. During 2020, the
Company successfully transferred the manufacturing process for its
TCR-T cell products to KBI Biopharma, a previously undisclosed
contract manufacturing organization with cGMP cell therapy
manufacturing facilities in The Woodlands, TX. TCR-T batch data
generated at both KBI and the Company’s own laboratory were the
basis of the Chemistry, Manufacturing and Controls portion of the
Investigational New Drug Application (IND) filed earlier this year.
KBI is now working to complete the process qualification and
aseptic process validation to facilitate clinical
manufacturing.
Additionally, the Company has been implementing
a strategy to build in-house cGMP clinical production capabilities
at the Company’s facility in Houston, TX. This is being done to
provide greater flexibility and control of this important aspect of
clinical development and the Company is moving forward rapidly to
establish these manufacturing capabilities. The commissioning of
the Company’s clinical production unit (CPU), as well as aseptic
process validation, were completed this past quarter, which are
meaningful steps in establishing the CPU’s capabilities. The team
is completing process qualification, which will support the opening
of the facility to manufacture TCR-T cells for the clinical trial.
The ability to use both its in-house facility and KBI’s facility
for manufacturing will provide the Company a strong degree of risk
mitigation and greater capacity as the Company scales up clinical
trial activities.
The Company continues to qualify TCRs in its
Library and plans to amend the IND during the second half of 2021
to include these additional TCRs. The Company expects that the
supplemental TCRs will expand the potential utility, applicable
patient population, and addressable commercial market for the
Library, and may include additional KRAS and / or TP53 mutations or
other genetic hotspots associated with solid tumors such as
EGFR.
During the second quarter, the Company presented
a poster at the annual American Association of Cancer Research
(AACR) meeting, entitled “Hotspot mutations in KRAS targeted by
TCR-T cells genetically modified with the Sleeping Beauty
transposon/transposase system”. The poster highlighted preclinical
work regarding the Company’s TCR-T program and demonstrated that
multiple TCRs with unique specificities targeting recurrent p53 and
KRAS substitutions in frequent HLA haplotypes could be stably
expressed using Sleeping Beauty transposition to re-direct
peripheral blood T-cells towards tumor cells. The Company plans on
providing additional preclinical data further demonstrating the
strong science behind the program later this year at a scientific
conference.
“Our TCR-T program continues to build momentum
scientifically and clinically, and we remain poised to begin dosing
during the fourth quarter of this year. We have begun pre-screening
patients and based on initial data from investigators, we are very
optimistic that we will consistently find mutation / HLA
combinations within the targeted patient populations that match
TCRs in our Library,” said Raffaele Baffa, MD, Ph.D., Chief Medical
Officer. “I thank the team for their tremendous efforts as they
work to ensure success.”
Closing of Venture Debt Financing with
Silicon Valley Bank
The Company today announced that it has closed a
venture debt facility with Silicon Valley Bank in the aggregate
amount of $50 million. The Company will draw down an initial $25
million tranche from this facility immediately. A second $25
million tranche is available contingent on the achievement of
certain clinical milestones and other conditions.
The initial $25 million tranche extends the
Company’s cash runway into the fourth quarter of 2022, well beyond
the time required to generate and assess the initial clinical data
from the Company’s Phase I/II TCR-T Library trial.
Holger Weis, member of the Board of Directors
and Chair of the Audit Committee, commented, “We have developed a
very strong relationship with Silicon Valley Bank and are happy to
have its partnership and support on this funding. This debt vehicle
will be used in a prudent and judicious manner and extends the
Company’s cash runway significantly. This is a clear demonstration
that the Company is being guided by strong consideration of
shareholder impact and a desire to support expanding shareholder
value through our capital strategy and tactics.”
Update on Phase I CD19 RPM CAR-T Trial
Being Conducted by Eden BioCell in Taiwan
As previously disclosed, in March 2021, Eden
BioCell, the Company’s Joint Venture in Taiwan with TriArm
Therapeutics, began treating patients in a clinical trial with the
Company’s investigational CD19 RPM CAR-T cell therapy, under the
Phase I IND cleared by the Taiwan Food and Drug Administration in
December 2020. The Company today provided an update on this
program.
Two patients have been treated in the trial. The
lead investigator at National Taiwan University in Taipei has
reported no serious adverse safety events in either of these
patients. Laboratory results continue to support, as previously
published, that non-viral Sleeping Beauty gene transfer is
effective in genetically modifying autologous T-cells. Patients
were infused two days after gene transfer, thus shortening the
turnaround time and providing a clear advantage over viral
methods.
However, based on laboratory data generated from
the first two patients between March and May 2021, the TriArm/Eden
BioCell team concluded, in concert with the lead investigator and
with the support from the team at Ziopharm, that further process
development work is required. This additional work is intended to
optimize and refine the manufacturing process in order to
manufacture cells more consistently in the desired clinical dose
range seeking to be studied.
Per the terms of the Joint Venture agreement,
the TriArm/Eden BioCell team will work towards the necessary
process development improvements before infusing additional
patients. The length of time to do so is unknown and maybe require
up to 12 months. The ongoing COVID-19 outbreak in Taiwan presents
added uncertainty, as the operational activities in the
manufacturing facility are currently limited due to employee
restrictions related to the pandemic. These restrictions are
impacting clinical trials broadly in Taiwan.
Additionally, consistent with its strategic
focus on TCR, the Company is seeking and considering broader
partnerships to enable further development of the investigational
CD19 RPM CAR-T cell therapy. Several parties have expressed
interest in such a partnership, including TriArm Therapeutics. The
Company will carefully consider all options regarding the future of
the Joint Venture, the technology, and the global development
pathway, in order to maximize shareholder value.
The Company noted the distinctions between the
CAR-T program and the TCR-T program. “As we have previously
described, the TCR and CAR-T processes are intrinsically different
and follow very separate process development pathways,” commented
Dr. Baffa. “While both involve Sleeping Beauty gene transfer, the
constructs, and manufacturing processes are very distinct. The
unique clinical presentations and challenges associated with the
treatment of blood cancers and solid tumors also reflect
differences and we believe the recent findings from CAR-T do not
read through to the TCR-T program.”
Update on Search for Permanent Chief
Executive Officer
The Company expects to make an announcement
regarding its permanent CEO position in the near future.
Robert Postma, member of the Board of Directors,
said, “We have seen and considered a number of very strong and
qualified candidates during the search, and the Board is in the
final stages of selecting our new CEO.”
Cash Position
- As of June 30, 2021, the Company had approximately $76.7
million of cash and cash equivalents. This amount is unaudited and
preliminary, and does not present all information necessary for an
understanding of our financial condition as of June 30, 2021, which
will be presented in our Quarterly Report on Form 10-Q for the
quarter ended June 30, 2021.
- Additionally, a prepayment of approximately $1.8
million remains for work to be conducted by the Company at MD
Anderson under the Company’s research and development
agreements.
- The $25 million drawdown of the Silicon Valley Bank debt
facility is not included in the above figure as it was closed
subsequent to June 30, 2021.
Conference Call and Webcast
Ziopharm will host a conference call and webcast
for the investment community today, August 9, 2021, at 4:30 p.m.
EDT. The conference call can be accessed by dialing 877-451-6152
(U.S. and Canada) or 201-389-0879 (International). The passcode for
the conference call is 13721210. A live webcast may be accessed
using the link here, or by visiting the “Investors” section of the
Ziopharm website at www.ziopharm.com. The call will be recorded and
available for replay on the Company’s website for approximately 90
days after the call.
About Ziopharm Oncology,
Inc.Ziopharm is developing non-viral and cytokine-driven
cell and gene therapies that weaponize the body’s immune system to
treat the millions of people globally diagnosed with cancer each
year. With its multiplatform approach, Ziopharm is at the forefront
of immuno-oncology. Ziopharm’s pipeline is built for commercially
scalable, cost-effective T-cell receptor (TCR) T-cell therapies
based on its non-viral Sleeping Beauty gene transfer platform, a
rapidly manufactured Sleeping Beauty-enabled CD19-specific CAR-T
program and a precisely controlled IL-12 gene therapy. The Company
has clinical and strategic collaborations with the National Cancer
Institute and The University of Texas MD Anderson Cancer Center.
For more information, please visit www.ziopharm.com.
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 wind down of the Company’s Controlled IL-12
clinical program, the timing of activities relating to the
Company’s GMP facility, the execution of potential future
partnerships or transactions, and the timing of the Company's
research and development programs, including the anticipated dates
for enrolling patients in the Company’s TCR-T clinical trial.
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 the Company’s
operating plans that may impact its 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 Quarterly Report in the most
recent Form 10-Q and 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.
Investor Relations
Contacts:Adam D. Levy, Ph.D., MBAExecutive Vice President,
Corporate Development and Investor RelationsT:
508.552.9255E:alevy@ziopharm.com
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