Cellectis Presents Updated Clinical and Translational Data on
BALLI-01 at the European Hematology Association (EHA) 2023
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, presented today updated clinical and translational data
on its clinical trial BALLI-01 (evaluating UCART22) at the European
Hematology Association (EHA) 2023. The data presented support the
preliminary safety and efficacy of UCART22 in a heavily pretreated
relapsed/refractory B-cell acute lymphoblastic leukemia (r/r B-ALL)
population.
“These clinical data are very positive for
patients with r/r B-ALL who have failed multiple lines of treatment
options including chemotherapy, CD19 directed CAR T-cell therapy
and allogeneic stem cell transplant and encourage further
enrollment into the BALLI-01 clinical study” said Nicolas Boissel,
M.D., Ph.D., Head of Hematology Adolescent and Young Adult Unit at
Hôpital Saint-Louis, Paris, France.
The poster presentation at EHA
highlights the following data:
BALLI-01 is a Phase 1/2a open-label study,
evaluating the safety and clinical activity of UCART22 in patients
with relapsed/refractory B-cell acute lymphoblastic leukemia (r/r
B-ALL).
The poster presentation reviewed clinical and
translational data from patients who received UCART22 after
lymphodepletion (LD) with fludarabine and cyclophosphamide (FC)
(F : 30 mg/m2 × 3d, C : 1.0 g/m2 × 3d) or fludarabine,
cyclophosphamide and alemtuzumab (FCA) (F: 30 mg/m2 × 3d, C :
0.5g/m2 × 3d, A : 20 mg/d × 3d) in patients with r/r
B-ALL.
Compared to the clinical update on BALLI-01 at
ASH 2021, the poster presents data from six additional patients who
received UCART22 at dose level 3 (DL3), 5 x 106 cells/kg, as of the
December 31, 2022 data cutoff.
Preliminary safety data
UCART22 administered after FC or FCA LD regimen
was well tolerated. No dose limiting toxicities (DLTs) nor immune
effector cell-associated neurotoxicity syndrome (ICANS) were
observed; 61% of patients reported cytokine release syndrome (CRS)
(Grade 1 [N=9] or Grade 2 [N=2]). One serious adverse event of
special interest (AESI) of Grade 2 graft-versus-host disease (GvHD)
(skin) was reported in the setting of reactivation of prior
allogeneic hematopoietic stem cell transplantation (HSCT) donor
stem cells. Serious adverse events (SAEs) (G≥3) reported in 72% of
patients included infections (39%) and febrile neutropenia (28%),
and all were not related to UCART22.
Preliminary efficacy data
Responses were assessed beginning on Day 28.
Up to FC/FCA-Intermediate Dose Level 2 (DL2i): 3
complete remissions with incomplete count recovery (CRi) and 1
morphologic leukemia-free state (MLFS) were observed and previously
reported at the American Society of Hematology (ASH) 2021
conference.
For FCA-Dose Level 3 (DL3), 50% of the six
patients responded:
- 1 patient who failed 4 prior lines,
including multiagent chemotherapy, blinatumomab, inotuzumab,
autologous CAR19, and allogeneic HSCT, achieved a minimal residual
disease (MRD) negative complete response (CR) lasting over 90 days
after UCART22 infusion as of the December 31, 2022 data
cutoff.
- 1 patient who failed 4 prior lines,
including multiagent chemotherapy, venetoclax, autologous CAR19,
and allogeneic HSCT, achieved an MRD negative complete response
with incomplete count recovery (CRi) consolidated with donor
lymphocyte infusion (DLI) after Day 90 and remains in an MRD
negative CRi past 7 months as of the December 31, 2022 data
cutoff.
- 1 patient who failed 3 prior lines,
including multiagent chemotherapy, venetoclax, autologous CAR19,
and allogeneic HSCT, achieved an MRD negative MLFS up to Day
114.
Host lymphocytes remained suppressed (mean ALC
<0.1 x103 cells/mL) through Day 28 for all patients who received
FCA LD. Peak ferritin levels correlated with UCART22 cell expansion
and cytokine release syndrome (CRS). UCART22 continues to be safe
and tolerable, with no treatment emergent serious adverse events
(TEAEs) or DLTs reported. UCART22 cell expansion was detected in 9
of 13 patients in the FCA LD arm and associated with clinical
activity.
Overall, these data support the preliminary
safety and efficacy of UCART22 in this heavily pretreated r/r B-ALL
population.
The BALLI-01 study is currently enrolling
patients after FCA lymphodepletion. UCART22 is currently the most
advanced allogeneic CAR T-cell product in development for r/r
B-ALL. The next data set is expected to be released later this
year.
The poster presentation is available on
Cellectis’ website:
https://www.cellectis.com/en/investors/scientific-presentations/
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 over
23 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
“anticipate,” “believe,” “intend”, “expect,” “plan,” “scheduled,”
“could” and “will,” or the negative of these and similar
expressions. These forward-looking statements, which are based on
our management’s current expectations and assumptions and on
information currently available to management. Forward-looking
statements include statements about the preliminary results for the
BALLI-01 trial and the objectives of this trial, which remains
ongoing; the ability to progress our clinical trial and to present
any additional data from this trial; clinical outcomes from our
trial, which may materially change as more patient data becomes
available, potential benefits of our UCART product candidates, and
our manufacturing capabilities. 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. With respect to our cash runway, our
operating plans, including product development plans, may change as
a result of various factors, including factors currently unknown to
us. 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, 2022 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
contact: Pascalyne
Wilson, Director, Communications, +33 (0)7 76 99 14
33, media@cellectis.com
Investor Relations
contacts: Arthur Stril,
Chief Business Officer, +1 (347) 809 5980,
investors@cellectis.com Ashley
R. Robinson, LifeSci Advisors, +1 617 430 7577
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