Cellectis Announces Recombinant DNA Advisory Committee’s (RAC) Unanimous Approval of UCART123 Phase 1 Study Protocols in AML and BPDCN

NEW YORK–(BUSINESS WIRE)– Regulatory News:

Cellectis
(Alternext: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on
developing immunotherapies based on gene edited CAR T-cells (UCART),
today announced the National Institute of Health’s Recombinant DNA
Advisory Committee (RAC)’s unanimous approval of two Phase 1 study
protocols for Cellectis’ 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).

Cellectis will host a conference call in the next coming days. The
details will be communicated shortly.

The RAC hearing was held on December 14, 2016 during a session dedicated
to UCART projects and TALEN^® based gene editing. This was the
first time that allogeneic CAR T-cell programs gene edited with TALEN^®
technology were presented during a RAC hearing.

Cellectis expects to file an Investigational New Drug (IND) application
with the U.S. Food and Drug Administration (FDA) by the end of 2016 and,
pending FDA clearance, plans to initiate Phase 1 clinical trials in the
first half of 2017. These programs will be the first therapeutic
applications of a gene edited allogeneic “off-the-shelf” product
candidate in the U.S.

UCART123 is a gene edited T-cell product candidate that targets CD123,
an antigen that is located on CD123-expressing leukemic cells in AML, as
well as in leukemic and other tumoral cells in BPDCN.

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 primarily
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 Professor
Hagop Kantarjian, MD, Department Chair, Department of Leukemia, Division
of Cancer Medicine and Professor Naveen Pemmaraju, MD, Assistant
Professor.

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.

About RAC

The Recombinant DNA Advisory Committee (RAC) is a federal advisory
committee that provides recommendations to the NIH Director related to
basic and clinical research involving recombinant or synthetic nucleic
acid molecules. The NIH, through the RAC, reviews the most innovative
clinical study protocols involving a gene therapy product.

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 16 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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