Full Press Release Details
of UCART123 Amendment in AML to Accelerate Clinical Development
of current tested dose levels from 6.25x104/kg
interval shortens between patients from 42 days to 28 days, then to 14
days for subsequent patients
Anderson Cancer Center added as new clinical site for the AML study
NEW YORK--(BUSINESS WIRE)--May 22, 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 the approval of an amendment to the protocol for the
Phase 1 clinical trial of Cellectis' UCART123 product candidate in
patients with acute myeloid leukemia (AML).
The main changes to the protocol include:
Dose levels to be administered increase 400 percent from 6.25x104
to 2.5x105 UCART123 cells per kilogram, with a capping at
80kg equivalent. The product's safety and tolerability profile allowed
Cellectis to increase the dose level.
The dose limiting toxicities (DLT) observation period decreases from
42 to 28 days post-UCART123 infusion, except for patients with
aplastic bone marrow at Day 28 for whom the DLT observation period
The time interval between the first and the second patient for
UCART123 infusion at each new dose level tested shortens from 42 days
to 28 days (42 days in case of aplastic anemia) then to 14 days for
subsequent patients.
A potential second UCART123 infusion is implemented.
In addition, a new AML clinical center has been opened at MD Anderson
Cancer Center in Houston, Texas, aiming at increasing the patient
enrollment pace. The study is led by Prof. Hagop Kantarjian, MD,
Department Chair, Department of Leukemia, Division of Cancer Medicine,
and Dr. Naveen Pemmaraju, MD, Assistant Professor, being Principal
"This amendment approval for Cellectis' UCART123 protocol is an
important step in the progression of our study, and opening another
clinical site at MD Anderson - one of the world's most premier cancer
centers - puts the Company on solid ground to help as many AML patients
as possible with this innovative new therapy," said Prof. St phane
Depil, Senior Vice President, R&D, and Chief Medical Officer at
Cellectis. "Off-the-shelf gene editing immunotherapy is continuing to
revolutionize the landscape of modern medicine, and we hope that this
approach leads to a lifesaving treatment for AML patients in the near
"As Cellectis has been working very closely with the concerned parties
to review the details of UCART123 study to date, we are eager to hit the
ground running with the new protocol in an effort to find a truly
effective treatment for AML patients with high unmet medical needs,"
added St phan Reynier, Chief Regulatory and Compliance Officer at
Cellectis. "We look forward to obtaining additional data so that we can
address such a rare and devastating disease."
The FDA review period for this protocol amendment has passed and
Cellectis obtained IRB's approval.
More information about this trial is available at ClinicalTrials.gov.
About UCART123 clinical trial
Our first wholly controlled product candidate, UCART123, is a gene
edited T-cell investigational drug that targets CD123, an antigen
expressed at the surface of leukemic cells in AML. Cellectis received in
February 2017 an Investigational New Drug (IND) approval from the U.S.
Food and Drug Administration (FDA) to conduct Phase 1 clinical trial
with UCART123 in patients with AML. This marks the first allogeneic,
"off-the-shelf" gene-edited CAR T-cell product candidate that the FDA
has approved for clinical trial.
UCART123 clinical trial in AML is a
Phase 1, open label dose-escalation and dose-expansion study to evaluate
the safety, expansion, persistence and clinical activity of UCART123
(allogeneic engineered T-cells expressing anti-CD123 chimeric antigen
receptor), administered in patients with relapsed/refractory AML, and
patients with newly diagnosed high-risk AML.
The clinical research is
coordinated by principal investigator Prof. Gail J. Roboz, MD, at Weill
Cornell, Professor of Medicine at Weill Cornell Medicine and Director of
the Clinical and Translational Leukemia Programs at Weill Cornell
Medicine and New York-Presbyterian.
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 in 2017 an
estimated 21,000 new AML cases per year, with 10,000 estimated deaths
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.
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