Cellectis Submits IND Application for UCART123, an Allogeneic Gene Edited CAR T-Cell Product Candidate, in AML and BPDCN
January 03 2017 - 5:00PM
Business Wire
Regulatory News:
Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Alternext: ALCLS;
Nasdaq: CLLS), a biopharmaceutical company focused on developing
immunotherapies based on gene edited CAR T-cells (UCART), today
announced the submission of an Investigational New Drug (IND)
application to the U.S. Food and Drug Administration (FDA)
requesting approval to initiate Phase 1 clinical trials of UCART123
the Company’s most advanced, wholly owned TALEN® gene edited
product candidate in patients with acute myeloid leukemia (AML) and
blastic plasmacytoid dendritic cell neoplasm (BPDCN).
Pending regulatory clearance, Cellectis plans to initiate Phase
1 clinical trials in the first half of 2017. This is the first IND
filing for human clinical applications of a gene edited allogeneic
“off-the-shelf” product candidate in the U.S.
UCART123 is a gene edited T-cell investigational drug that
targets CD123, an antigen expressed at the surface of leukemic
cells in AML, as well as on leukemic and other tumoral cells in
BPDCN.
The UCART123 program was subject to a public hearing by the
National Institutes of Health's Recombinant DNA Advisory Committee
(RAC) in December 2016, where it received the unanimous approval of
the RAC committee members.
AML is a devastating clonal hematopoietic stem cell neoplasm
that is characterized by uncontrolled proliferation and
accumulation of leukemic blasts in bone marrow, peripheral blood
and, occasionally, in other tissues. These cells disrupt normal
hematopoiesis and rapidly cause bone marrow failure and death. In
the U.S. alone, there are an estimated 19,950 new AML cases per
year, with 10,430 estimated deaths per year.
The clinical research at Weill Cornell will be led by principal
investigator Dr. Gail J. Roboz, Director of the Clinical and
Translational Leukemia Programs and Professor of Medicine.
BPDCN is a very rare and aggressive hematological malignancy
that is derived from plasmacytoid dendritic cell precursors. BPDCN
is a disease of bone marrow and blood cells but also often affects
skin and lymph nodes.
The UCART123 clinical program at MD Anderson will be led by Dr
Naveen Pemmaraju, MD, Assistant Professor, and Professor Hagop
Kantarjian, MD, Department Chair, Department of Leukemia, Division
of Cancer Medicine.
The manufacturing process of Cellectis’ allogeneic CAR T-cell
product line, Universal CARTs or UCARTs, yields frozen,
off-the-shelf, engineered CAR T-cells. UCARTs are meant to be
readily available CAR T-cells for a large patient population. Their
production can be industrialized and standardized with defined
pharmaceutical release criteria.
“Following a Pre-IND meeting with the FDA in August 2016 and a
NIH-RAC public hearing in December 2016, filing this IND is an
important regulatory milestone for the Company. It represents many
years of research and development by a dedicated team focused on
developing highly innovative UCART products for the benefit of
patients”, stated Stephan Reynier, Chief Regulatory and Compliance
Officer, Cellectis.
Chief Medical Officer, Dr. Loan Hoang-Sayag, commented:
“UCART123 represents a unique therapeutic approach for patients
with unmet medical needs such as relapsed or refractory AML, high
risk AML and BPDCN and we are excited to move this experimental
product into clinical development. We have designed robust Phase 1
clinical trials to better understand the potential of UCART123 to
address the needs of different patient populations.”
Information about ongoing clinical trials are publically
available on dedicated websites such as:
www.clinicaltrials.gov in the U.S.www.clinicaltrialsregister.eu
in Europe
About Cellectis
Cellectis is a biopharmaceutical company focused on developing
immunotherapies based on gene edited CAR T-cells (UCART). The
company’s mission is to develop a new generation of cancer
therapies based on engineered T-cells. Cellectis capitalizes on its
17 years of expertise in genome engineering - based on its flagship
TALEN® products and meganucleases and pioneering electroporation
PulseAgile technology - to create a new generation of
immunotherapies. CAR technologies are designed to target surface
antigens expressed on cells. Using its life-science-focused,
pioneering genome-engineering technologies, Cellectis’ goal is to
create innovative products in multiple fields and with various
target markets. Cellectis is listed on the Nasdaq market (ticker:
CLLS) and on the NYSE Alternext market (ticker: ALCLS). To find out
more about us, visit our website: www.cellectis.com
Talking about gene editing? We do it. TALEN® is a registered
trademark owned by the Cellectis Group.
Disclaimer
This press release contains “forward-looking” statements that
are based on our management’s current expectations and assumptions
and on information currently available to management.
Forward-looking statements involve known and unknown risks,
uncertainties and other factors that may cause our actual results,
performance or achievements to be materially different from any
future results, performance or achievements expressed or implied by
the forward-looking statements. The risks and uncertainties
include, but are not limited to, the risk that the preliminary
results from our product candidates will not continue or be
repeated, the risk of not obtaining regulatory approval to commence
clinical trials on the UCART product candidates, the risk that any
one or more of our product candidates will not be successfully
developed and commercialized. Further information on the risks
factors that may affect company business and financial performance,
is included in filings Cellectis makes with the Security Exchange
Commission from time to time and its financial reports. Except as
required by law, we assume no obligation to update these
forward-looking statements publicly, or to update the reasons
actual results could differ materially from those anticipated in
the forward-looking statements, even if new information becomes
available in the future.
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version on businesswire.com: http://www.businesswire.com/news/home/20170103006318/en/
Media:Jennifer Moore, 917-580-1088VP of
Communicationsmedia@cellectis.comorKCSA Strategic
CommunicationsCaitlin Kasunich,
212-896-1241ckasunich@kcsa.comorIR:Simon Harnest,
646-385-9008VP of Corporate Strategy and
Financesimon.harnest@cellectis.com
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