Full Press Release Details
Grants Cellectis IND Approval for UCART22 in B-ALL
TALEN -based Gene-Edited Allogeneic CAR T-Cell Product Candidate
Entering Clinical Development
NEW YORK--(BUSINESS WIRE)--June 4, 2018--Regulatory News:
Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Euronext Growth: ALCLS - Nasdaq:
CLLS), a clinical-stage biopharmaceutical company focused on developing
immunotherapies based on gene-edited allogeneic CAR T-cells (UCART),
announced today that the U.S. Food and Drug Administration (FDA) has
approved the Company's Investigational New Drug (IND) application to
initiate a Phase 1 clinical trial for UCART22, Cellectis' second wholly
controlled TALEN gene-edited product candidate, for the treatment of
B-cell acute lymphoblastic leukemia (B-ALL) in adult patients.
UCART22 is the 3rd allogeneic, off-the-shelf, gene-edited CAR
T-cell product candidate approved by the FDA for clinical trials in the
U.S., following UCART19 (exclusively licensed to Servier and under joint
development agreement between Servier and Allogene), and Cellectis'
UCART123. Cellectis intends to begin the UCART22 Phase 1 study in the
second half of 2018. The research for UCART22 will be led by Dr. Nitin
Jain, Assistant Professor, and Prof. Hagop Kantarjian, Chairman in the
Department of Leukemia and University Chair in Cancer Medicine, at The
University of Texas MD Anderson Cancer Center in Houston.
"The FDA's approval of Cellectis' UCART22 IND application for the
treatment of B-ALL puts us one step closer to providing patients in need
with a better access to an effective drug candidate for such a rare,
devastating disease," said Prof. St phane Depil, Senior Vice President,
Research & Development, and Chief Medical Officer, Cellectis. "With this
opportunity, Cellectis is well-positioned to further its work in the
off-the-shelf gene-editing space, in the hope of helping patients to
beat B-ALL in the near future."
"Given that Cellectis is leading the allogeneic CAR-T approach across
today's medical landscape - first, with the FDA's IND approval for
UCART123 last year and now, with approval of UCART22 IND - we are eager
to bring an innovative therapy to the market for patients who are
suffering from B-ALL everywhere," added Stephan Reynier, Chief
Regulatory and Compliance Officer, Cellectis. "The bottom line is that
patients are in dire need of effective, affordable and easily accessible
treatments across the board, and our off-the-shelf product candidates
aim to do just that."
UCART22 is an allogeneic, off-the-shelf gene-edited T-cell product
candidate designed for the treatment of B-ALL. Like CD19, CD22 is a cell
surface antigen expressed from the pre-B-cell stage of development
through mature B-cells. CD22 expression occurs in more than 90% of
patients with B-ALL.1
UCART22 clinical trial is a Phase 1, open label dose-escalation and
dose-expansion study to evaluate the safety, expansion, persistence and
clinical activity of UCART22 (allogeneic engineered T-cells expressing
anti-CD22 chimeric antigen receptor) in patients with relapsed or
refractory CD22+ B-cell acute lymphoblastic leukemia (B-ALL). Dose level
1 to be administered is 1x105 UCART22 cells per kilogram.
Dose levels 2 and 3 are respectively at 1x106 and 5x106.
ALL is a heterogeneous hematologic disease characterized by the
proliferation of immature lymphoid cells in the bone marrow, peripheral
blood, and other organs. It can start either with early B-cells or
T-cells at different stages of maturity. The American Cancer Society's
estimates for acute lymphocytic leukemia (ALL) in the United States for
2018 (including both children and adults) are about 5,960 new cases of
ALL and about 1,470 deaths from ALL. Approximately 85% of ALL cases
involve precursor B-cells (B-ALL).
The manufacturing process of Cellectis' allogeneic CAR T-cell product
line, Universal CARTs or UCARTs, yields frozen, off-the-shelf,
non-alloreactive engineered CAR T-cells. UCARTs are meant to be readily
available CAR T-cells for a large patient population. Their production
is industrialized with defined pharmaceutical release criteria.
Information about ongoing clinical trials is publicly available on
dedicated websites, such as:
www.clinicaltrials.gov in the U.S.
Cellectis is a clinical-stage biopharmaceutical company focused on
developing a new generation of cancer immunotherapies based on
gene-edited T-cells (UCART). By capitalizing on its 18 years of
expertise in gene editing - built on its flagship TALEN technology and
pioneering electroporation system PulseAgile - Cellectis uses the power
of the immune system to target and eradicate cancer 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 Euronext
Growth (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
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.
Further information on the risk factors that may affect company business
and financial performance is included in Cellectis' Annual Report on
Form 20-F and the financial report (including the management report) for
the year ended December 31, 2017 and subsequent filings Cellectis makes
with the Securities Exchange Commission from time to time. Except as
required by law, we assume no obligation to update these forward-looking
statements publicly, or to update the reasons why actual results could
differ materially from those anticipated in the forward-looking
statements, even if new information becomes available in the future.
VP of Communications
Strategic Communications
Caitlin Kasunich, 212-896-1241
Harnest, 646-385-9008
VP of Corporate Strategy and Finance