Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading
clinical-stage CRISPR genome-editing biopharmaceutical company,
today reported financial results for the fourth quarter and full
year 2023 and reviewed recent pipeline progress.
“Following our execution in 2023, we enter 2024 with momentum
advancing three clinical-stage off-the-shelf CAR-T cell therapy
programs for patients with hematologic malignancies while we plan
for two clinical data releases this year,” said Rachel Haurwitz,
PhD, Caribou’s president and chief executive officer. “For our lead
program, CB-010, we plan to present initial dose expansion data and
the RP2D in the second quarter of 2024. For our second program,
CB-011, we continue to enroll patients in the CaMMouflage trial and
plan to report initial dose escalation data by year-end 2024. For
our third program, CB-012, we are thrilled to have recently dosed
the first patient in the AMpLify trial. Our team is focused on
clinical execution to inform two clinical datasets this year as
part of our mission of bringing transformative therapies to
patients with devastating diseases.”
Clinical highlightsCB-010, a
clinical-stage allogeneic anti-CD19 CAR-T cell therapy for B cell
non-Hodgkin lymphoma
- Caribou has dosed the 30th patient in the dose expansion
portion of the ongoing ANTLER Phase 1 clinical trial in second-line
relapsed or refractory large B cell lymphoma (r/r LBCL) patients.
Previously, 16 patients were dosed in the dose escalation portion
of ANTLER. The company will continue enrolling additional
second-line r/r LBCL patients in ANTLER to collect additional
clinical data.
- In December 2023, Caribou shared regulatory feedback from the
U.S. Food and Drug Administration (FDA) following a Type B clinical
meeting. The company received the FDA’s input on a Phase 3
randomized pivotal trial for CB-010 in second-line r/r LBCL,
stating that Caribou’s proposed comparator arm of platinum-based
immunochemotherapy followed by high dose chemotherapy (HDCT) and
autologous stem cell transplantation (ASCT) is acceptable.
- As previously reported, CB-010 demonstrated encouraging data
from the dose escalation portion of the ANTLER Phase 1 clinical
trial in 16 patients with relapsed or refractory B cell non-Hodgkin
lymphoma (r/r B-NHL). Dose escalation data showed CB-010 has the
potential to rival the efficacy and safety profile of approved
autologous CAR-T cell therapies.
- To Caribou’s knowledge, CB-010 is the first anti-CD19
allogeneic CAR-T cell therapy to be evaluated in the second-line
LBCL setting, and it was granted Regenerative Medicine Advanced
Therapy (RMAT), Fast Track, and Orphan Drug designations by the FDA
for specific indications in 2022.
CB-011, a clinical-stage allogeneic anti-BCMA CAR-T cell
therapy for multiple myeloma
- Caribou is enrolling patients with relapsed or refractory
multiple myeloma (r/r MM) in the dose escalation portion of the
ongoing CaMMouflage Phase 1 clinical trial. Patients are currently
being enrolled at dose level 3 (450x106 CAR-T cells).
- Preclinical data for CB-011 were published in Cancer Immunology
Research in February 2024. The manuscript is available on Caribou’s
website under Scientific Publications.
CB-012, a clinical-stage allogeneic anti-CLL-1 CAR-T
cell therapy for acute myeloid leukemia
- The first patient has been dosed in the AMpLify Phase 1
clinical trial, which is evaluating CB-012 in patients with
relapsed or refractory acute myeloid leukemia (r/r AML). Additional
site activation is underway.
- Preclinical data for CB-012 highlighting the investigational
new drug (IND)-enabling studies will be presented as a poster
presentation at the American Association for Cancer Research (AACR)
Annual Meeting 2024, held April 5-10, 2024 in San Diego.
CB-020, a preclinical allogeneic anti-ROR1 CAR-NK cell
therapy
- As part of a regular portfolio prioritization process, Caribou
has paused the development of CB-020, a preclinical allogeneic
anti-ROR1 CAR-NK cell therapy. Caribou continues to develop its
CAR-NK cell therapy platform as these therapies may have potential
for the treatment of multiple diseases.
2024 anticipated milestones
- CB-010: In Q2 2024, Caribou plans to present
initial dose expansion data, the recommended Phase 2 dose (RP2D),
and emerging translational data from the ANTLER Phase 1 clinical
trial, as well as an updated timeline for the pivotal Phase 3 trial
initiation.
- CB-011: Caribou plans to present initial dose
escalation data from the CaMMouflage Phase 1 clinical trial by
year-end 2024.
- CB-012: Caribou plans to provide updates on
dose escalation as the AMpLify Phase 1 clinical trial in r/r AML
advances.
Fourth quarter and full year 2023 financial
resultsCash, cash equivalents, and marketable
securities: Caribou had $372.4 million in cash, cash
equivalents, and marketable securities as of December 31, 2023,
compared to $317.0 million as of December 31, 2022. The 2023 amount
includes the approximately $134.4 million in net proceeds from the
Company’s underwritten public offering in July and August 2023 and
the $25.0 million equity investment in June 2023 from Pfizer.
Caribou expects these cash, cash equivalents, and marketable
securities will be sufficient to fund its current operating plan
into Q1 2026.
Licensing and collaboration
revenue: Revenue from Caribou’s licensing and
collaboration agreements was $3.6 million for the three months
ended December 31, 2023, and $34.5 million for the full year 2023,
compared to $3.7 million and $13.9 million, respectively, for the
same periods in 2022. The increase for the year ended December 31,
2023 was primarily due to $24.8 million in revenue recognized under
the now-terminated AbbVie Collaboration and License Agreement,
including $20.8 million of deferred revenue recognized upon
termination of this agreement as previously disclosed, which was
the remaining deferred revenue balance from AbbVie’s $30 million
upfront payment in February 2021.
R&D expenses: Research and development
expenses were $31.3 million for the three months ended December 31,
2023, and $112.1 million for the full year 2023, compared to $25.7
million and $82.2 million, respectively, for the same periods in
2022. The increase for the year ended December 31, 2023 was
primarily due to costs to advance pipeline programs, including the
CB-010 ANTLER, CB-011 CaMMouflage, and CB-012 AMpLify Phase 1
clinical trials; personnel-related expenses, including stock-based
compensation, due to headcount increases; and facilities and other
allocated expenses.
G&A expenses: General and
administrative expenses were $9.7 million for the three months
ended December 31, 2023, and $38.5 million for the full year 2023,
compared to $8.5 million and $38.0 million, respectively, for the
same periods in 2022. The increase for the year ended December 31,
2023 was primarily due to personnel-related expenses, including
stock-based compensation, due to headcount increases, and other
facilities and allocated expenses. These increases were partially
offset by decreases in insurance and other service-related
expenses, and patent prosecution and maintenance costs.
Net loss: Caribou reported a net loss of
$34.5 million for the three months ended December 31, 2023, and
$102.1 million for the full year 2023, compared to $27.0 million
and $99.4 million, respectively, for the same periods in 2022.
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, Caribou is enrolling second-line patients with large B cell
lymphoma (LBCL) comprised of different subtypes of aggressive r/r
B-NHL (DLBCL NOS, PMBCL, HGBL, tFL, and tMZL). CB-010 is an
allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9
CRISPR hybrid RNA-DNA (chRDNA) genome-editing technology. 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 antitumor activity by limiting
premature CAR-T cell exhaustion. To Caribou’s knowledge, CB-010 is
the first anti-CD19 allogeneic CAR-T cell therapy to be evaluated
in the second-line LBCL setting and it has been granted
Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and
Orphan Drug designations by the FDA for specific indications.
Additional information on the ANTLER trial (NCT04637763) can be
found at clinicaltrials.gov.
About CB-011CB-011 is a product candidate from
Caribou’s allogeneic CAR-T cell therapy platform and is being
evaluated in patients with relapsed or refractory multiple myeloma
(r/r MM) in the CaMMouflage Phase 1 trial. CB-011 is an allogeneic
anti-BCMA CAR-T cell therapy engineered using Cas12a chRDNA
genome-editing technology. To Caribou’s knowledge, CB-011 is the
first allogeneic CAR-T cell therapy in the clinic that is
engineered to improve antitumor activity through an immune cloaking
strategy with a B2M knockout and insertion of a B2M–HLA-E fusion
protein to blunt immune-mediated rejection. CB-011 has been granted
Fast Track and orphan drug designations by the FDA. Additional
information on the CaMMouflage trial (NCT05722418) 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 an appropriate
scFv for the generation of the company’s CAR. Additional
information on the AMpLify trial (NCT06128044) can be found at
clinicaltrials.gov.
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 Cas12a chRDNA technology,
enables superior precision to develop cell therapies that are
armored to potentially improve antitumor activity. Caribou is
advancing a pipeline of clinical-stage off-the-shelf cell therapies
from its CAR-T platform as readily available treatments for
patients with hematologic malignancies. 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. 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
status and updates from its ANTLER Phase 1 clinical trial for
CB-010, including (i) the timing of reporting initial dose
expansion data, emerging translational data, follow-up dose
escalation data from the ANTLER trial, disclosure of the
recommended Phase 2 dose for CB-010, and updated timeline for its
planned Phase 3 pivotal trial for CB-010 in second-line LBCL
patients; (ii) the timing of status and updates from its
CaMMouflage Phase 1 clinical trial for CB-011 and expectations
regarding the timing of presenting the initial dose escalation
data; (iii) the timing of status and updates from its AMpLify Phase
1 clinical trial for CB-012; and (iv) its expected cash runway.
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.
In addition, caution should be exercised when interpreting
results from separate trials of other companies’ CAR-T cell
therapies referenced in this press release. Caribou has not
performed any head-to-head trials comparing any of these other
CAR-T cell therapies with CB-010 and the design and patient
populations of those other trials may vary in material ways from
those of CB-010. As such, the results of these other clinical
trials may not be comparable to clinical results for CB-010.
Cross-trial comparisons may have no interpretive value on Caribou’s
existing or future results.
Caribou Biosciences,
Inc.Condensed Consolidated Balance Sheet Data(in thousands)
(unaudited) |
|
|
|
|
|
|
|
|
|
December 31, 2023 |
|
December 31, 2022 |
|
Cash, cash equivalents, and marketable securities |
|
$ |
372,404 |
|
$ |
317,036 |
|
Total
assets |
|
|
432,209 |
|
|
373,765 |
|
Total
liabilities |
|
|
63,808 |
|
|
72,894 |
|
Total
stockholders' equity |
|
|
368,401 |
|
|
300,871 |
|
Total
liabilities and stockholders' equity |
|
$ |
432,209 |
|
$ |
373,765 |
|
|
|
|
|
|
|
Caribou Biosciences,
Inc.Condensed Consolidated Statement of Operations(in thousands,
except share and per share data)(unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended, December 31, |
|
Year Ended, December 31, |
|
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
|
Licensing
and collaboration revenue |
|
$ |
3,558 |
|
|
$ |
3,692 |
|
|
$ |
34,477 |
|
|
$ |
13,851 |
|
|
Operating
expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
31,279 |
|
|
|
25,736 |
|
|
|
112,075 |
|
|
|
82,230 |
|
|
General and administrative |
|
|
9,721 |
|
|
|
8,534 |
|
|
|
38,461 |
|
|
|
38,020 |
|
|
Total operating expenses |
|
|
41,000 |
|
|
|
34,270 |
|
|
|
150,536 |
|
|
|
120,250 |
|
|
Loss from
operations |
|
|
(37,442 |
) |
|
|
(30,578 |
) |
|
|
(116,059 |
) |
|
|
(106,399 |
) |
|
Other income
(expense): |
|
|
|
|
|
|
|
|
|
Change in fair value of equity securities |
|
|
(9 |
) |
|
|
(60 |
) |
|
|
(6 |
) |
|
|
(133 |
) |
|
Change in fair value of the MSKCC success payments liability |
|
|
(1,683 |
) |
|
|
1,388 |
|
|
|
(1,288 |
) |
|
|
2,429 |
|
|
Other income, net |
|
|
4,822 |
|
|
|
2,331 |
|
|
|
15,476 |
|
|
|
4,752 |
|
|
Total other income |
|
|
3,130 |
|
|
|
3,659 |
|
|
|
14,182 |
|
|
|
7,048 |
|
|
Net loss
before provision for income taxes |
|
|
(34,312 |
) |
|
|
(26,919 |
) |
|
|
(101,877 |
) |
|
|
(99,351 |
) |
|
Provision for income taxes |
|
|
193 |
|
|
|
70 |
|
|
|
193 |
|
|
|
70 |
|
|
Net
loss |
|
$ |
(34,505 |
) |
|
$ |
(26,989 |
) |
|
$ |
(102,070 |
) |
|
$ |
(99,421 |
) |
|
Other
comprehensive income (loss): |
|
|
|
|
|
|
|
|
|
Net unrealized gain (loss) on available-for-sale marketable
securities, net of tax |
|
|
1,011 |
|
|
|
517 |
|
|
|
1,548 |
|
|
|
(1,383 |
) |
|
Net
comprehensive loss |
|
$ |
(33,494 |
) |
|
$ |
(26,472 |
) |
|
$ |
(100,522 |
) |
|
$ |
(100,804 |
) |
|
Net loss per
share, basic and diluted |
|
$ |
(0.39 |
) |
|
$ |
(0.44 |
) |
|
$ |
(1.38 |
) |
|
$ |
(1.64 |
) |
|
Weighted-average common shares outstanding, basic and diluted |
|
|
88,432,905 |
|
|
|
61,001,150 |
|
|
|
73,807,597 |
|
|
|
60,801,133 |
|
|
|
|
|
|
|
|
|
|
|
|
Caribou Biosciences, Inc.
contacts:Investors:Amy Figueroa,
CFAinvestor.relations@cariboubio.com
Media:Peggy Vorwald,
PhDmedia@cariboubio.com
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