Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today announced
that it has received clearance of its Investigational New Drug
(IND) application from the U.S. Food and Drug Administration (FDA)
for CB-011, a genome-edited allogeneic anti-BCMA CAR-T cell therapy
with immune cloaking. The CaMMouflage Phase 1 clinical trial, a
multicenter, open-label study to evaluate the safety and efficacy
of a single dose of CB-011 in adult patients with relapsed or
refractory multiple myeloma (r/r MM), is expected to initiate
patient enrollment for treatment at dose level 1 (50x106 CAR-T
cells) in early 2023.
“Clearance of our second IND application represents another key
milestone for our pipeline of promising allogeneic cell therapies
designed to have enhanced persistence of antitumor activity,” said
Rachel Haurwitz, Ph.D., Caribou’s president and chief executive
officer. “CB-011 is designed with an immune cloaking strategy to
reduce rejection of the cell therapy by a patient’s T and NK cells
and we use Caribou’s highly precise and specific Cas12a chRDNA
genome-editing technology to manufacture this product candidate. We
are excited to develop CB-011 as an off-the-shelf cell therapy that
may reach a broader number of patients with multiple myeloma than
are currently being served, and we look forward to initiating
patient enrollment in the CaMMouflage trial in early 2023.”
CB-011 is the second allogeneic cell therapy advancing into
clinical development from Caribou’s CAR-T cell platform targeting
hematologic malignancies. Caribou’s first allogeneic cell therapy,
CB-010, an allogeneic anti-CD19 CAR-T cell therapy with a PD-1
knockout, is being evaluated in the ongoing ANTLER Phase 1 clinical
trial in patients with relapsed or refractory B cell non-Hodgkin
lymphoma (r/r B-NHL). Encouraging safety and antitumor activity for
CB-010 at dose level 1 have been reported from the ANTLER
trial.
“CAR-T cell therapies have shown great promise for treating
patients with relapsed or refractory multiple myeloma,” said Sundar
Jagannath, M.D., director of the Multiple Myeloma Center of
Excellence at Tisch Cancer Institute, Mount Sinai Hospital, New
York. “Allogeneic, or ‘off-the-shelf,’ CAR-T cell therapies would
provide a great option for patients with multiple myeloma, helping
to overcome the need for bridging therapies as well as variable
quality and manufacturing timelines of autologous CAR-T cells.”
CB-011 is the first allogeneic anti-BCMA CAR-T cell therapy, to
Caribou’s knowledge, that is engineered to improve persistence of
antitumor response through an immune cloaking genome-editing
approach that removes the B2M protein and inserts a B2M–HLA-E
fusion protein. CB-011 has four edits implemented via Caribou’s
Cas12a CRISPR hybrid RNA-DNA (chRDNA) technology:
- Edits 1 and 2 – A humanized anti-BCMA CAR is site-specifically
inserted into the TRAC gene to target cancer cells, thereby
knocking out expression of the T cell receptor to reduce the risk
of graft versus host disease (GvHD).
- Edits 3 and 4 – A B2M–HLA-E peptide fusion gene is
site-specifically inserted into the B2M gene of the CAR-T cells to
prevent recognition and rejection by patient T cells and blunt
rejection by NK cells. These edits knock out endogenous B2M
expression, eliminating endogenous HLA class I presentation and
reducing T cell-mediated rejection, while enabling expression of
B2M–HLA-E needed to inhibit NK cell-mediated rejection.
About the CaMMouflage Trial The CaMMouflage
Phase 1 trial is an open-label, multicenter clinical trial designed
to evaluate CB-011 in adults with relapsed or refractory multiple
myeloma (r/r MM). Part A, a 3+3 dose escalation design, will
evaluate the safety and tolerability of CB-011 at multiple dose
levels and will be utilized to determine the maximum tolerated dose
and/or the recommended Phase 2 dose. Part B is the dose expansion
portion with the primary objective of determining tumor response
after a single dose of CB-011. CaMMouflage will include patients
who have had 3 or more prior lines of therapy and will exclude
patients who have received a BCMA-targeted therapy within the last
3 months and/or any prior CAR-T cell therapy.
Caribou plans to initiate patient enrollment in the CaMMouflage
trial to treat patients with a single administration of CB-011 at
dose level 1 (50x106 CAR-T cells) in early 2023.
About Multiple MyelomaMultiple myeloma (MM) is
the second most common hematologic malignancy in the United States.
According to the National Cancer Institute, an estimated 160,000
people in 2019 were living with MM and an estimated 34,000
individuals are diagnosed with MM each year in the United States.
Approximately 50% of patients with MM relapse and have their cancer
return after first line treatment or are refractory and do not
respond to current treatments.
About
Caribou’s Novel Next-Generation CRISPR PlatformCRISPR
genome editing uses easily designed, modular biological tools to
make DNA changes in living cells. There are two basic components of
Class 2 CRISPR systems: the nuclease protein that cuts DNA and the
RNA molecule(s) that guide the nuclease to generate a
site-specific, double-stranded break, leading to an edit at the
targeted genomic site. CRISPR systems are capable of editing
unintended genomic sites, known as off-target editing, which may
lead to harmful effects on cellular function and phenotype. In
response to this challenge, Caribou has developed CRISPR hybrid
RNA-DNA guides (chRDNAs; pronounced “chardonnays”) that direct
substantially more precise genome editing compared to all-RNA
guides. Caribou is deploying the power of its Cas12a chRDNA
technology to carry out multiple edits at high efficiency,
including multiplex gene insertions, to develop CRISPR-edited
therapies.
About Caribou Biosciences, Inc.Caribou
Biosciences is a clinical-stage CRISPR genome-editing
biopharmaceutical company dedicated to developing transformative
therapies for patients with devastating diseases. The company’s
genome-editing platform, including its proprietary Cas12a chRDNA
technology, enables superior precision to develop cell therapies
that are specifically engineered for enhanced persistence. Caribou
is advancing a pipeline of off-the-shelf CAR-T and CAR-NK cell
therapies for the treatment of patients with hematologic
malignancies and solid tumors.
For more information about Caribou, visit www.cariboubio.com and
follow the company @CaribouBio.
“Caribou Biosciences” and the Caribou logo are registered
trademarks of Caribou Biosciences, Inc.
Forward-Looking StatementsThis press release
contains forward-looking statements, within the meaning of the
Private Securities Litigation Reform Act of 1995. These
forward-looking statements include, without limitation, statements
related to Caribou’s strategy, plans, and objectives, and
expectations regarding its clinical and preclinical development
programs, including the timing of the initiation of its CaMMouflage
Phase 1 trial for CB-011, the expected timing of patient enrollment
for the trial, as well as the results and duration of the trial and
expectations and timing regarding the release of clinical data from
Caribou’s ongoing ANTLER Phase 1 clinical trial for its CB-010
product candidate. Management believes that these forward-looking
statements are reasonable as and when made. However, such
forward-looking statements are subject to risks and uncertainties,
and actual results may differ materially from any future results
expressed or implied by the forward-looking statements. Risks and
uncertainties include, without limitation, risks inherent in
development of cell therapy products; uncertainties related to the
initiation, cost, timing, progress, and results of current and
future research and development programs, preclinical studies, and
clinical trials; and the risk that initial or interim clinical
trial data will not ultimately be predictive of the safety and
efficacy of Caribou’s product candidates or that clinical outcomes
may differ as more patient data becomes available; as well as other
risk factors described from time to time in Caribou’s filings with
the Securities and Exchange Commission, including its Annual Report
on Form 10-K for the year ended December 31, 2021 and subsequent
filings. In light of the significant uncertainties in these
forward-looking statements, you should not rely upon
forward-looking statements as predictions of future events. Except
as required by law, Caribou undertakes no obligation to update
publicly any forward-looking statements for any reason.
Caribou Biosciences, Inc. Contacts:
Investors:Amy Figueroa,
CFAafigueroa@cariboubio.com
Media:Peggy Vorwald,
Ph.D.pvorwald@cariboubio.com
Investors and Media: Elizabeth Wolffe, Ph.D.,
and Sylvia WheelerWheelhouse LSA
lwolffe@wheelhouselsa.comswheeler@wheelhouselsa.com
Photos accompanying this announcement are available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/5f227084-9845-4932-b673-81588114f336
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