FDA Grants Cellectis IND Approval to Proceed with the Clinical Development of UCART123, the First Gene Edited Off-the-Shelf CAR T-Cell Product Candidate developed in the U.S.

NEW YORK–(BUSINESS WIRE)– class= »bwalignl »>
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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