FDA Grants Orphan Drug Designation to Cellectis’ CLLS52 (alemtuzumab) For ALL Treatment
August 01 2024 - 3:30PM
UK Regulatory
FDA Grants Orphan Drug Designation to Cellectis’ CLLS52
(alemtuzumab) For ALL Treatment
NEW YORK, Aug. 01, 2024 (GLOBE NEWSWIRE) --
Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ:
CLLS), a clinical-stage biotechnology company using its pioneering
gene-editing platform to develop life-saving cell and gene
therapies, today announced that the U.S. Food and Drug
Administration (FDA) has granted Orphan Drug Designation (ODD) to
Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal
Product (IMP) used as part of the lymphodepletion regimen
associated with UCART22, evaluated in the BALLI-01 clinical trial
in relapsed/refractory B-cell acute lymphoblastic leukemia
(ALL).
“We are excited that the FDA granted CLLS52
(alemtuzumab) ODD designation status. The importance of adding
alemtuzumab to the lymphodepletion regimen has been demonstrated in
Cellectis’ BALLI-01 study, where the addition of this
lymphodepletion agent to the fludarabine and cyclophosphamide
regimen was associated with sustained lymphodepletion and
significantly higher UCART22 cell expansion allowing for greater
clinical activity,” said Mark Frattini, M.D., Ph.D. Chief Medical
Officer at Cellectis.
Cellectis is the inventor of the combination of
CD52 knockout UCART cells with a lymphodepleting regimen
containing an anti-CD52 antibody such as alemtuzumab. The
CD52 knockout aims to render the UCART product candidates
resistant to alemtuzumab as part of the lymphodepleting regimen.
Cellectis’ UCART22 product candidate has the CD52 gene
inactivated by TALEN® gene editing technology.
The FDA grants ODD status to medicines intended
for the treatment, diagnosis or prevention of rare diseases or
disorders that affect fewer than 200,000 people in the US.
Receiving ODD may help to expedite and reduce the cost of
development, approval, and commercialization of a therapeutic
agent.
About Cellectis
Cellectis is a clinical-stage biotechnology company using its
pioneering gene-editing platform to develop life-saving cell and
gene therapies. Cellectis utilizes an allogeneic approach for CAR-T
immunotherapies in oncology, pioneering the concept of
off-the-shelf and ready-to-use gene-edited CAR T-cells to treat
cancer patients, and a platform to make therapeutic gene editing in
hemopoietic stem cells for various diseases. As a clinical-stage
biopharmaceutical company with 25 years of experience and expertise
in gene editing, Cellectis is developing life-changing product
candidates utilizing TALEN®, its gene editing technology, and
PulseAgile, its pioneering electroporation system to harness the
power of the immune system in order to treat diseases with unmet
medical needs. Cellectis’ headquarters are in Paris, France, with
locations in New York, New York and Raleigh, North Carolina.
Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and
on Euronext Growth (ticker: ALCLS).
Forward-looking Statements
This press release contains “forward-looking”
statements within the meaning of applicable securities laws,
including the Private Securities Litigation Reform Act of 1995.
Forward-looking statements may be identified by words such as
“may,” “aim,” and “intent,”, or the negative of these and similar
expressions. These forward-looking statements are based on our
management’s current expectations and assumptions and on
information currently available to management. Forward-looking
statements include statements about the potential of
CLLS52. These forward-looking statements are made in
light of information currently available to us and are subject to
numerous risks and uncertainties, including with respect to the
numerous risks associated with biopharmaceutical product candidate
development, including the risk of losing the orphan drug
designation if it is established that the product no longer meets
the orphan drug criteria before market authorization is granted (if
any).The priority review voucher may also not be granted at the
time of marketing authorization. Furthermore, many other important
factors, including those described in our Annual Report on Form
20-F and the financial report (including the management report) for
the year ended December 31, 2023 and subsequent filings Cellectis
makes with the Securities Exchange Commission from time to time, as
well as other known and unknown risks and uncertainties may
adversely affect such forward-looking statements and cause our
actual results, performance or achievements to be materially
different from those expressed or implied by the forward-looking
statements. 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.
For further information on Cellectis, please
contact:
Media contacts:
Pascalyne Wilson, Director, Communications, +33 (0)7 76 99 14
33, media@cellectis.com
Patricia Sosa Navarro, Chief of Staff to the CEO, +33 (0)7 76 77 46
93
Investor Relations
contact:
Arthur Stril, Interim Chief Financial Officer, +1 (347) 809 5980,
investors@cellectis.com
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