Ziopharm Oncology Announces FDA Clearance of IND for Rapid Personalized Manufacture of CD19-specific CAR-T
October 01 2019 - 8:00AM
Ziopharm Oncology, Inc. (“Ziopharm” or “the Company”)
(Nasdaq:ZIOP), today announced that the U.S. Food and Drug
Administration (FDA) has cleared an investigational new drug
application (IND) for a phase 1 clinical trial to evaluate
CD19-specific CAR-T, produced using a process termed rapid
personalized manufacture (RPM), as an investigational treatment for
patients with relapsed CD19+ leukemias and lymphomas.
The IND clearance builds upon the Company’s
experience with two prior generations of immunotherapy trials using
the Sleeping Beauty platform, which it believes is the most
clinically-advanced non-viral approach to the genetic modification
of T cells. With this third-generation trial, DNA from the Sleeping
Beauty system is stably inserted into the genome of resting T cells
to co-express a chimeric antigen receptor (CAR), membrane-bound
IL-15 (mbIL15) and a safety switch, which is designed to reduce
cost, simplify production, and preserve the therapeutic potential
of the T cells.
“There are currently no effective treatment
options for patients who relapse soon after allogeneic bone marrow
transplantation (BMT), as evidenced by their low rate of remission
and poor long-term survival. This trial expands the range of
patients with CD19-expressing malignancies that can be treated
using the RPM technology,” said Laurence Cooper, M.D., Ph.D., Chief
Executive Officer of Ziopharm. “We believe RPM is the fastest
approach to manufacturing and releasing CD19-specific CAR-T, as T
cells from the blood stream are genetically reprogramed with the
Sleeping Beauty system and then infused within two days of gene
transfer. Existing commercial T-cell products using viral-based
manufacturing are costly, time consuming to make and complex to
deliver. We are now positioned to not only address those issues,
but also to treat a patient group that remains underserved by
existing therapies.”
Up to 24 patients will be enrolled to evaluate
infusion of donor-derived RPM CAR-T in patients with CD19+
leukemias and lymphomas who have relapsed after allogeneic BMT.
This study will be conducted at The University of Texas MD Anderson
Cancer Center under an investigator-initiated trial expected to
begin later this year.
Research reveals three-year survival for adults
with CD19+ acute lymphoblastic leukemia after allogeneic BMT ranges
from 30% to 65%.1 For patients with other CD19+ cancers, allogeneic
BMT can provide a three-year survival rates between 30% to 75%.1
Few patients experience a durable remission who relapse in the
months following allogeneic BMT, regardless of the treatment
modality, with some having a median survival of only 2 to 3
months.2
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 to target neoantigens
inside and outside hotspots for solid tumors and CAR to target CD19
for blood cancers using the Company’s rapid personalized
manufacturing to produce and release CAR-T within two days of gene
transfer. 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 progress and timing of the
Company's research and development programs, including the
anticipated dates for the initiation of its clinical trials and the
Company’s expectations regarding the number of patients in its
clinical trials. 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
maintaining 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 on Form 10-Q
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.
References:1 D'Souza A, Fretham C. Current Uses
and Outcomes of Hematopoietic Cell Transplantation (HCT): CIBMTR
Summary Slides, 2018. Available at
https://www.cibmtr.org 2 Keil F, Prinz E, Kalhs P, et
al. Treatment of leukemic relapse after allogeneic stem cell
transplantation with cytotoreductive chemotherapy and/or
immunotherapy or second transplants. Leukemia 2001; 15:355-361.
Ziopharm Oncology Contact:
Chris Taylor VP, Investor Relations and Corporate Communications
617-502-1881 ctaylor@ziopharm.com
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