Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today reported new
12-month clinical data from cohort 1 in the ongoing ANTLER Phase 1
trial, which show long-term durability following a single infusion
of CB-010 at the initial dose level 1 (40x106 CAR-T cells). Cohort
1 results show:
- 6 of 6 patients achieved a complete response (CR) as best
response
- 3 of 6 patients maintained a durable CR at 6 months
- 2 of 6 patients maintain a long-term CR at the 12 month scan
and remain on the trial
- 18 months is the longest CR maintained to date in ANTLER,
achieved by the first patient dosed with CB-010
- CB-010 was generally well tolerated with adverse events
consistent with autologous or allogeneic anti-CD19 CAR-T cell
therapies
Based on promising initial data, the U.S. Food and Drug
Administration (FDA) granted CB-010 both Regenerative Medicine
Advanced Therapy (RMAT) and Fast Track designations. In addition,
Caribou has observed an encouraging safety profile for CB-010 at
dose level 2 (80x106 CAR-T cells) with no dose-limiting toxicities
(DLTs) in the 3 patients treated and is currently enrolling
patients at dose level 3 (120x106 CAR-T cells). Caribou expects to
provide an ANTLER trial update in 2023.
A photo accompanying this announcement is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/c8122817-d16a-4469-921f-2b0b7994b2d6ANTLER
Phase 1 trial of CB-010 at dose level 1
“With next-generation CRISPR genome-editing technology, the
promise of allogeneic cell therapies has advanced significantly,
and the early results seen in the ANTLER trial to date are a
reflection of that potential,” said Rachel Haurwitz, Ph.D.,
Caribou’s president and chief executive officer. “The long-term
durability at dose level 1 is comparable to autologous cell
therapies and we believe CB-010 has the potential to set a new
therapeutic bar for what allogeneic anti-CD19 CAR-T cell therapies
can achieve. We are further encouraged by receiving RMAT and Fast
Track designations for CB-010 from the FDA, which is a testament to
both the encouraging initial ANTLER data and the need for novel
therapies for patients with relapsed or refractory B-NHL.”
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.
“Patients with relapsed or refractory B cell non-Hodgkin
lymphoma are in need of treatments that are immediately available
and do not require burdensome or ineffective bridging therapies,”
said Susan O’Brien, M.D., professor of medicine, Chao Family
Comprehensive Cancer Center at University of California, Irvine,
CA, and presenting investigator on the ANTLER clinical trial. “The
early results from the ANTLER trial are promising and I look
forward to enrolling additional patients in this trial to learn
more about the potential of CB-010 as an off-the-shelf treatment
option for patients with aggressive B-NHL who have a high unmet
medical need.”
ANTLER is a Phase 1, open-label, multicenter clinical trial
(NCT04637763) evaluating the safety and efficacy of the company’s
lead allogeneic cell therapy, CB-010, in patients with r/r B-NHL.
The trial includes Part A, a 3+3 dose escalation phase designed to
evaluate safety of CB-010 at multiple dose levels and establish the
recommended Phase 2 dose, and Part B, a dose expansion phase with
the primary objective to determine tumor response after a single
dose of CB-010. As permitted by the protocol, backfilling patients
has begun at doses deemed well tolerated to increase the
understanding of CB-010’s safety profile and antitumor activity and
provide additional data for establishing a recommended Phase 2
dose.
ANTLER Trial-in-Progress Poster at ASH
2022Today at the 64th Annual ASH meeting, a
trial-in-progress poster is being presented to provide details of
the design and objectives of the ANTLER Phase 1 trial for CB-010 in
r/r B-NHL. Details of the poster presentation are as follows:
Title: A First-in-Human Phase 1, Multicenter,
Open-Label Study of CB-010, a Next-Generation CRISPR-Edited
Allogeneic Anti-CD19 CAR-T Cell Therapy with a PD-1 Knockout, in
Patients with Relapsed/Refractory B Cell Non-Hodgkin Lymphoma
(ANTLER Study)Presenter: Susan O’Brien, M.D.,
professor of medicine, Chao Family Comprehensive Cancer Center at
University of California, Irvine, CASession Name:
626. Aggressive Lymphomas: Prospective Therapeutic Trials: Poster
IIISession Date: Monday, December 12,
2022Presentation Time: 6:00 pm - 8:00 pm
CSTLocation: Ernest N. Morial Convention Center,
Hall DAbstract number: 4257
The poster presentation will be available for registered
attendees on the ASH website and on Caribou’s website under
Scientific Publications on Monday, December 12, 2022 at 9:00 am
CST.
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 knock out.
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
www.clinicaltrials.gov using identifier NCT04637763.
About Caribou’s Novel Next-Generation CRISPR
Platform CRISPR 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 high efficiency multiple edits, 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.
Follow us @CaribouBio and visit www.cariboubio.com.
“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 expectations relating to the timing of the
release of additional patient data from its ANTLER Phase 1 clinical
trial for CB-010. 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 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: |
InvestorsAmy
Figueroa, CFA |
afigueroa@cariboubio.comMediaPeggy Vorwald,
Ph.D.pvorwald@cariboubio.com |
|
Investors and
Media: |
Elizabeth Wolffe, Ph.D., and
Sylvia Wheeler |
Wheelhouse LSA |
lwolffe@wheelhouselsa.com |
swheeler@wheelhouselsa.com |
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