Ziopharm Oncology Reports on Status of Investigational New Drug Application for Phase 1 Trial to Evaluate CD19-targeted CAR T...
June 18 2018 - 7:30AM
Ziopharm Oncology, Inc. (Nasdaq:ZIOP) today announced the U.S. Food
and Drug Administration (FDA) placed on clinical hold a Phase 1
trial to evaluate CD19-specific CAR-T therapies manufactured under
point-of-care and requested additional information in support of
the investigational new drug (IND) application for the
trial.
Ziopharm, Precigen, Inc., a wholly-owned
subsidiary of Intrexon Corporation (NYSE:XON) and The University of
Texas MD Anderson Cancer Center, the IND sponsor, are seeking to
conduct a clinical trial to evaluate CAR+ T cells manufactured with
Sleeping Beauty technology as an investigational treatment for
patients with relapsed or refractory, CD19+ leukemias and
lymphomas. CAR+ T cells very-rapidly manufactured with the Sleeping
Beauty platform for this third-generation trial are designed to
co-express CD19-specific chimeric antigen receptor, or CAR,
membrane-bound interleukin 15 and a safety switch. The FDA has
requested additional information relative to Chemistry,
Manufacturing and Controls. Ziopharm and its partners will address
the FDA’s requests, and the initiation of this trial may be
delayed.
“We know what is needed to address the hold
issues and are looking forward to responding to the agency in a
timely manner,” said Laurence Cooper, M.D., Ph.D., Chief Executive
Officer of Ziopharm. “We are undertaking cutting-edge science and
are on the verge of a paradigm shift based on our approach to
very-rapidly manufacture CD19-specific T cells within two days
using our non-viral approach to CAR-T therapy based on the Sleeping
Beauty platform.”
Ziopharm believes this feedback from the FDA
does not affect timelines for the Company’s planned trial at the
National Cancer Institute using the Sleeping Beauty platform to
target solid tumors infusing TCR-modified T cells. The
second-generation clinical trial evaluating Sleeping
Beauty-manufactured CD19-specific CAR-T cells continues enrolling
and infusing patients at MD Anderson.
About Ziopharm Oncology,
Inc.Ziopharm Oncology is a Boston-based biotechnology
company focused on the 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 asset, 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 in solid tumors. Sleeping Beauty uses the
Company's so-called 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 DisclaimerThis
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, 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 progress and timing of the development of the
Company's research and development programs, including the timing
for initiating its first in-human trial using its very rapid
manufacturing process and other clinical trials using the Sleeping
Beauty technology. 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: the Company’s
ability to address FDA’s clinical hold concerns, the FDA’s review
of our other Sleeping Beauty programs, 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 year 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 Connolly Ziopharm
Oncology 617-502-1881dconnolly@ziopharm.com
Intrexon (NASDAQ:XON)
Historical Stock Chart
From Aug 2024 to Sep 2024
Intrexon (NASDAQ:XON)
Historical Stock Chart
From Sep 2023 to Sep 2024