Cellectis Submits IND Application for UCART123, an Allogeneic Gene Edited CAR T-Cell Product Candidate, in AML and BPDCN

NEW YORK–(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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