Caribou Biosciences Announces the FDA Granted Regenerative Medicine Advanced Therapy (RMAT) and Fast Track Designations to CB-010, an Allogeneic Anti-CD19 CAR-T Cell Therapy
November 29 2022 - 9:00AM
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) has granted CB-010
Regenerative Medicine Advanced Therapy (RMAT) designation for
relapsed or refractory large B cell lymphoma (LBCL) and Fast Track
designation for relapsed or refractory B cell non-Hodgkin lymphoma
(r/r B-NHL). CB-010, an allogeneic anti-CD19 CAR-T cell therapy
with a PD-1 knockout, is being evaluated in the company’s ongoing
ANTLER Phase 1 clinical trial in patients with r/r B-NHL, which can
enroll three LBCL subtypes: diffuse large B cell lymphoma (DLBCL),
primary mediastinal large B cell lymphoma (PMBCL), and high-grade B
cell lymphoma (HGBL). CB-010 is the first allogeneic anti-CD19
CAR-T cell therapy in the clinic with a PD-1 knockout, a
genome-editing strategy designed to improve the persistence of
antitumor activity by limiting premature CAR-T cell exhaustion.
“RMAT and Fast Track designations for CB-010 are important
recognitions of the significant unmet patient need for an
off-the-shelf cell therapy in the treatment of aggressive B-NHL,”
said Rachel Haurwitz, Ph.D., Caribou’s president and chief
executive officer. “Through genome editing with our precision
CRISPR chRDNA genome-editing technology, CB-010 has been designed
with a PD-1 knockout strategy to improve the persistence of
antitumor activity by limiting premature CAR-T cell exhaustion. In
our ANTLER Phase 1 trial, 3 of 6 patients treated with CB-010 at
dose level 1 maintained a durable complete response at 6 months. We
are encouraged that CB-010 has demonstrated early potential as an
off-the-shelf cell therapy that may meaningfully rival autologous
cell therapies.”
Encouraging safety data and antitumor activity for CB-010 at
dose level 1 (40x106 CAR-T cells) have been reported from the
ANTLER trial. As presented at the European Hematology Association
(EHA) 2022 Congress, 6 of 6 patients (100%) achieved a complete
response (CR) as best response after treatment with CB-010 at dose
level 1 (40x106 CAR-T cells). Subsequently, at 6 months, 3 of 6
patients (50%) maintained a CR. Fifteen months is the longest CR
maintained to date, observed in the first patient dosed in the
ANTLER trial. CB-010 was generally well tolerated at dose level
1.
Both RMAT and Fast Track designations are dedicated programs
designed to expedite the development and review processes for
promising therapeutic candidates intended to address an unmet
medical need in patients with serious conditions. These
designations provide important benefits in the drug development
process and are designed to facilitate and expedite development and
regulatory review, including providing eligibility for Priority and
Rolling Reviews, and Accelerated Approval, if relevant criteria are
satisfied.
About CB-010CB-010 is the lead product
candidate from Caribou’s allogeneic CAR-T cell therapy platform and
is being evaluated in patients with relapsed or refractory B cell
non-Hodgkin lymphoma (r/r B-NHL) in the ongoing ANTLER Phase 1
trial. CB-010 is an allogeneic anti-CD19 CAR-T cell therapy
engineered using Cas9 CRISPR hybrid RNA-DNA (chRDNA) technology to
insert a CD19-specific CAR into the TRAC gene and knock out PD-1 to
boost the persistence of antitumor activity. CB-010 is the first
allogeneic CAR-T cell therapy in the clinic with a PD-1 knockout.
CB-010 has been granted Regenerative Medicine Advanced Therapy
(RMAT), Fast Track, and Orphan Drug designations. Additional
information on the ANTLER trial can be found at
https://clinicaltrials.gov/ using identifier NCT04637763.
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 its timing and expectations relating to the
release of patient data from its ongoing ANTLER Phase 1 clinical
trial for CB-010, as well as the timing of regulatory review and
approval. 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
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