FDA Clears the IND for UCARTCS1, the First Allogeneic CAR-T to Treat Multiple Myeloma Patients
Business Wire 2-Apr-2019 4:30 PM
Successful manufacturing and release of GMP vials of UCARTCS1
Regulatory News:
Cellectis
(NASDAQ:CLLS), a biopharmaceutical company
focused on developing immunotherapies based on gene-edited 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 UCARTCS1, in
patients with multiple myeloma (MM). The IND for UCARTCS1 was filed on
December 28, 2018 and approved by the FDA within a month, on January 25,
2019. Cellectis is the sponsor of the UCARTCS1 clinical study (MUNDI-01)
and successfully ensured the manufacturing and release of UCARTCS1 GMP
batches, as well as an IRB approval.
UCARTCS1 is based on a tailored manufacturing process developed by
Cellectis, which removes both the CS1 antigen and TCR from the T-cell
surface using TALEN® gene editing technology, before adding
the CS1 CAR construct. This approach has both clinical and operational
benefits: the UCART is designed to have a lymphodepleting effect, and
the CAR T-cell cross reaction is suppressed, allowing for successful
manufacturing.
UCARTCS1 is the first allogeneic CAR-T therapy for MM to enter clinical
development. This milestone reinforces Cellectis' leadership in the
space, as it represents the fourth TALEN® gene-edited
allogeneic CAR-T product candidate developed by Cellectis to be approved
for clinical trials following UCART191 for ALL patients,
UCART123 for AML patients and UCART22 for B-ALL patients. The Phase 1 of
the MUNDI-01 study is designed to assess the safety and tolerability at
increasing dose levels of UCARTCS1 in patients living with MM.
"The last quarters have been very productive for Cellectis' UCARTCS1
product candidate. We successfully manufactured and released GMP batches
of UCARTCS1, filed an IND and secured approval from the FDA to start the
MUNDI-01 Phase 1 clinical study," said Dr. André Choulika, Chairman and
CEO of Cellectis. "This is the 4th time in 4 years that
Cellectis demonstrates excellence with an allogeneic product candidate.
It further demonstrates the strength of our innovation, our
manufacturing process and our execution, as we are eager to bring the
first allogeneic multiple myeloma CAR T-cell treatment to patients."
We anticipate the clinical research to be led by Dr. Krina Patel,
Principal Investigator, Assistant Professor, Department of
Lymphoma/Myeloma, Division of Cancer Medicine at the MD Anderson Cancer
Center in Houston, Texas. We plan to have two additional sites enrolling
patients for this clinical study: Weill Cornell Medicine under the
leadership of Dr. Ruben Niesvizky, Director of the Multiple Myeloma
Center at New York Presbyterian Hospital-Cornell Medical Center and
Hackensack Meridian under the supervision of Dr. Andre Goy, Chairman and
Director of John Theurer Cancer Center (JTCC) at Hackensack University
Medical Center.
About UCARTCS1
UCARTCS1 is an allogeneic, off-the-shelf, gene-edited T-cell product
candidate designed for the treatment of multiple myeloma (MM). CS1
(SLAMF7) is highly expressed on MM tumor cells and is an attractive
target because there is strong evidence of tumor response to monoclonal
antibody treatment targeting it. The limitation so far has been the
presence of the CS1 target on the surface of T-cells, which has hindered
the access to CAR-Ts and bispecific antibodies. As an example, the
introduction of a CAR construct in T-cells induces cross T-cell reaction
and leads to their self-destruction during manufacturing. Cellectis
solved this issue by using TALEN® gene editing to knock-out
the CS1 gene from T-cells before introducing the CS1 CAR construct.
The UCARTCS1 MUNDI-01 clinical trial is a Phase 1 dose-escalation and
dose-expansion study to evaluate the safety, expansion, persistence and
clinical activity of UCARTCS1 (allogeneic engineered T-cells) in
patients with MM. Dose level 1 will be administered at 1x106
UCARTCS1 cells per kilogram, and dose levels 2 and 3 will be
administered at 3x106 and 9x106, respectively. The
Dose Limiting Toxicity (DLT) period is 28 days in concordance with a
28-day staggering for the first 2 patients at each dose level.
MM is a cancer that forms in a type of white blood cell called a plasma
cell, which helps the body to fight infections by making antibodies that
recognize and attack germs. MM causes cancer cells to accumulate in bone
marrow, where they crowd out healthy blood cells. The American Cancer
Society estimates that 32,110 new cases of MM will be diagnosed and
12,960 deaths are expected to occur in the U.S. in 2019.
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
(U.S.) and www.clinicaltrialsregister.eu
(Europe).
About Cellectis
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 19 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 (NASDAQ: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 owned by Cellectis.
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.
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, 2018 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.
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1 Developed by Cellectis, exclusively licensed to Servier and
now under a joint development agreement between Servier and Allogene.
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