Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today announced
that the U.S. Food and Drug Administration (FDA) granted Fast Track
designations to CB-010 for refractory systemic lupus erythematosus
(SLE) and to CB-012 for relapsed or refractory acute myeloid
leukemia (r/r AML). CB-010, an allogeneic anti-CD19 CAR-T cell
therapy, will be evaluated in the GALLOP Phase 1 clinical trial in
patients with lupus nephritis (LN) and extrarenal lupus (ERL),
subcategories of SLE. The GALLOP clinical trial is on track to
initiate by year-end 2024. CB-012, an allogeneic anti-CLL-1 CAR-T
cell therapy, is being evaluated in the company’s ongoing AMpLify
Phase 1 clinical trial in patients with r/r AML.
“We are pleased to receive Fast Track designations from the FDA.
This is a testament to the significant unmet need and the potential
of CB-010 and CB-012 as readily available, off-the-shelf CAR-T cell
therapies,” said Tina Albertson, MD, PhD, Caribou’s chief medical
officer. “Based on previously reported data in our ongoing trial
for patients with relapsed or refractory B cell non-Hodgkin
lymphoma, CB-010 has shown encouraging safety, efficacy, and
prolonged B cell aplasia following a single dose. Based on these
data, we are eager to expand the clinical development program for
CB-010 into LN and ERL, prevalent and severe autoimmune
diseases.”
Dr. Albertson continued, “Most patients with acute AML are
refractory or relapse quickly after currently available therapeutic
options, and allogeneic hematopoietic stem cell transplant is the
only potentially curative option after salvage chemotherapy
regimens. As we advance CB-012 and enroll patients at dose level 2,
we are focused on our goal to deliver an effective, off-the-shelf
treatment option for patients living with this disease.”
Fast Track designation is intended to help rapidly advance the
development and review processes for promising therapeutic
candidates for serious conditions that may fill an unmet medical
need. Clinical programs with Fast Track designation may benefit
from early and frequent communication with the FDA throughout the
regulatory review process and may also be eligible for Accelerated
Approval and Priority Review when relevant criteria are met.
About CB-010CB-010 is the lead clinical-stage
product candidate from Caribou’s allogeneic CAR-T cell therapy
platform, and it is being evaluated in patients with relapsed or
refractory B cell non-Hodgkin lymphoma (r/r B-NHL) in the ongoing
ANTLER Phase 1 clinical trial and will be evaluated in patients
with lupus nephritis (LN) and extrarenal lupus (ERL) in the GALLOP
Phase 1 clinical trial. In the ANTLER clinical trial, Caribou is
enrolling second-line (2L) patients with large B cell lymphoma
(LBCL) comprised of different subtypes of aggressive r/r B-NHL
(DLBCL NOS, PMBCL, HGBL, tFL, and tMZL) who have never received
prior CD19-targeted therapy as well as LBCL patients who have
relapsed after a prior CD19-targeted therapy. To Caribou’s
knowledge, CB-010 is the first allogeneic CAR-T cell therapy in the
clinic with a PD-1 knockout, a genome-editing strategy designed to
improve activity against diseases by limiting premature CAR-T cell
exhaustion. CB-010 is also, to Caribou’s knowledge, the first
anti-CD19 allogeneic CAR-T cell therapy to be evaluated in the 2L
LBCL setting. The FDA granted CB-010 Regenerative Medicine Advanced
Therapy (RMAT) and Orphan Drug designations for B-NHL and Fast
Track designations for both B-NHL and refractory systemic lupus
erythematosus (SLE). Additional information on the ANTLER trial
(NCT04637763) can be found at clinicaltrials.gov.
About CB-012CB-012 is a product candidate from
Caribou’s allogeneic CAR-T cell therapy platform and is being
evaluated in the AMpLify Phase 1 clinical trial in patients with
relapsed or refractory acute myeloid leukemia (r/r AML). CB-012 is
an anti-CLL-1 CAR-T cell therapy engineered with five genome edits,
enabled by Caribou’s patented next-generation CRISPR technology
platform, which uses Cas12a chRDNA genome editing to significantly
improve the specificity of genome edits. To Caribou’s knowledge,
CB-012 is the first allogeneic CAR-T cell therapy with both
checkpoint disruption, through a PD-1 knockout, and immune
cloaking, through a B2M knockout and B2M–HLA-E fusion protein
insertion; both armoring strategies are designed to improve
antitumor activity. Caribou has exclusively in-licensed from
Memorial Sloan Kettering Cancer Center (MSKCC) in the field of
allogeneic CLL-1-targeted cell therapy a panel of fully human scFvs
targeting CLL-1, from which the company has selected a scFv for the
generation of the company’s CAR. CB-012 was granted Fast Track
designation by the FDA. Additional information on the AMpLify trial
(NCT06128044) can be found at clinicaltrials.gov.
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 chRDNA
technology to carry out high efficiency multiple edits, 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 Cas12a chRDNA technology,
enables superior precision to develop cell therapies that are
armored to potentially improve activity against disease. Caribou is
advancing a pipeline of off-the-shelf cell therapies from its CAR-T
platform as readily available treatments for patients with
hematologic malignancies and autoimmune diseases. Follow us
@CaribouBio and visit www.cariboubio.com.
Forward-Looking Statements This press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. In some cases,
you can identify forward-looking statements by terms such as “may,”
“will,” “should,” “expect,” “plan,” “anticipate,” “could,”
“intend,” “target,” “project,” “contemplate,” “believe,”
“estimate,” “predict,” “potential,” or “continue,” or the negative
of these terms or other similar expressions, although not all
forward-looking statements contain these words. These
forward-looking statements include, without limitation, statements
related to Caribou’s strategy, plans, and objectives, and
expectations regarding its clinical development programs, including
its expectations relating to development, regulatory approval,
results, and the timing of and updates from its ANTLER Phase 1
clinical trial for CB-010, its GALLOP Phase 1 clinical trial for
CB-010, and its AMpLify Phase 1 clinical trial for CB-012.
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 the development of
cell therapy products; uncertainties related to the initiation,
cost, timing, progress, and results of Caribou’s current and future
research and development programs, preclinical studies, and
clinical trials; and the risk that initial, preliminary, 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 patient enrollment continues and as more
patient data becomes available; the risk that preclinical study
results observed will not be borne out in human patients or
different conclusions or considerations are reached once additional
data have been received and fully evaluated; the ability to obtain
key regulatory input and approvals; 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, 2023 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,
CFAinvestor.relations@cariboubio.com
Media:Peggy Vorwald,
PhDmedia@cariboubio.com
Caribou Biosciences (NASDAQ:CRBU)
Historical Stock Chart
From Sep 2024 to Oct 2024
Caribou Biosciences (NASDAQ:CRBU)
Historical Stock Chart
From Oct 2023 to Oct 2024