FDA Grants Cellectis IND Approval to Proceed with the Clinical Development of UCART123, the First Gene Edited Off-the-Shelf C...
February 06 2017 - 4:30PM
Business Wire
Cellectis’ UCART123 Product Candidate Targets
AML and BPDCN
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), has
received an Investigational New Drug (IND) approval from the U.S.
Food and Drug Administration (FDA) to conduct Phase 1 clinical
trials with 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). This marks the first allogeneic, “off-the-shelf”
gene-edited CAR T-cell product candidate that the FDA has approved
for clinical trials. Cellectis intends to initiate Phase 1 trials
in the first half of 2017.
UCART123 is a gene-edited T-cell investigational drug that
targets CD123, an antigen expressed at the surface of leukemic
cells in AML, tumoral cells in BPDCN. The clinical research for AML
will be led, at Weill Cornell, by principal investigator Dr. Gail
J. Roboz, Director of the Clinical and Translational Leukemia
Programs and Professor of Medicine. The UCART123 clinical program
for BPDCN will be led, at the MD Anderson Cancer Center, by Dr.
Naveen Pemmaraju, MD, Assistant Professor, and Professor Hagop
Kantarjian, MD, Department Chair, Department of Leukemia, Division
of Cancer Medicine.
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.
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 FDA’s approval of Cellectis’ UCART123 – the first
“off-the-shelf” CAR T-cell product candidate to enter clinical
trials in the U.S. – is a major milestone not only for the Company
but also for the medical community, global biotech and
pharmaceutical industries at large,” said Dr. Loan Hoang-Sayag,
Cellectis Chief Medical Officer. “Cellectis’ allogeneic UCART
products have the potential to create an important shift with
regard to availability, and cost-effectiveness, to make these
therapies widely accessible to patient population across the
world.”
“After the National Institutes of Health's Recombinant DNA
Advisory Committee (RAC)’s unanimous approval of two Phase 1 study
protocols for Cellectis’ UCART123 in December 2016, the FDA’s
approval of Cellectis’ IND is a new major regulatory milestone
achieved, for having UCART123 proceed into clinical development and
reaching cancer patients in need,” added Stephan Reynier, Chief
Regulatory and Compliance Officer, Cellectis.
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 as well as its 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/20170206006061/en/
Media:Jennifer Moore, 917-580-1088VP of
Communicationsmedia@cellectis.comorCaitlin Kasunich,
212-896-1241KCSA Strategic
Communicationsckasunich@kcsa.comorIR:Simon Harnest,
646-385-9008VP of Corporate Strategy and
Financesimon.harnest@cellectis.com
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