Cellectis Announces Successful cGMP Manufacturing for Second Product Candidate: UCART123

- November 16th, 2016

Cellectis, today announced that a series of production runs of UCART123, a Company’s wholly-owned TALEN® gene edited product candidate, was successfully performed in large scale.

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 that a series of production runs of UCART123, a Company’s wholly-owned TALEN® gene edited product candidate, was successfully performed in large scale, according to cGMP standards, for the purpose of conducting twoPhase 1 clinical trials in patients with acute myeloid leukemia (AML) and blastic plasmacytoid dendritic cell neoplasm (BPDCN).
The cGMP manufacturing of UCART123 clinical batches has been operated
with CELLforCURE, a LFB group company and the largest industrial
facility for clinical and commercial production of innovative cell and
gene therapies in Europe. CELLforCURE is in charge of
implementing cGMP manufacturing processes that are designed and
developed by Cellectis.
The manufacturing process for 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
UCART123 is an engineered T-cell product candidate that targets CD123,
an antigen that is located on CD123-expressing leukemic cells in AML as
well as leukemic and other tumoral cells in BPDCN. We are planning to
file an Investigational New Drug application (IND) with the United
States Food and Drug Administration (FDA) in order to initiate clinical
AML is a devastating clonal hematopoietic stem cell neoplasm
characterized by uncontrolled proliferation and accumulation of leukemic
blasts in the 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.
Preclinical and translational activities on UCART123 in AML are
performed in collaboration with Dr. Monica Guzman, Associate Professor
of Pharmacology in Medicine at Weill Cornell Medical College. 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 the bone marrow and blood cells, but also often affects
skin and lymph nodes.
Cellectis collaborates with the MD Anderson Cancer Center on the
preclinical development of UCART123 in BPDCN preliminary to the Phase I
clinical trial in BPDCN to be activated. The UCART123 clinical program
at MD Anderson will be led by Professor Hagop Kantarjian, MD, Department
Chair, Department of Leukemia, Division of Cancer Medicine.
“We are proud of achieving this important milestone for UCART123, our
first wholly owned product candidate. The successful translation of an
R&D concept into a cGMP clinical grade industrial product is one of the
key gating factors for us to move into clinical trials,” said Arjan
Roozen, Vice President, GMP Solutions and Manufacturing.
“With this work and Cellectis’ breakthrough TALEN®-based gene
editing technology, we continue building upon the Company’s milestones
as a pioneer in the field, strengthening our pipeline and bringing us
closer and closer to finding efficient and cost-effective products for
cancer patients across the globe,” added David J.D. Sourdive, Executive
Vice President, Corporate Development, Cellectis.
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.
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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