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 2022 and
reviewed recent pipeline progress.
“We successfully demonstrated the potential of our chRDNA
genome-editing technology with promising clinical data from CB-010,
our lead allogeneic cell therapy,” said Rachel Haurwitz, PhD,
Caribou’s president and chief executive officer. “The initial dose
level of CB-010 demonstrated 6-month complete response rates that
have the potential to rival the responses seen with approved
autologous CAR-T cell therapies. We are excited that the FDA
granted the CB-010 program RMAT and Fast Track designations last
year. Our team drove additional pipeline progress with an IND
clearance for CB-011, enabling us to activate clinical sites for
our CaMMouflage Phase 1 trial. In 2023, Caribou plans to maintain
this momentum by advancing two ongoing clinical trials for our
off-the-shelf cell therapies in patients with hematologic
malignancies and preparing an IND submission for our third program,
CB-012.”
Accomplishments and Highlights
Pipeline and Technology
- CB-010: Caribou reported promising data at
dose level 1 (40x106 CAR-T cells) from its ongoing ANTLER Phase 1
clinical trial of CB-010 in patients with relapsed or refractory B
cell non-Hodgkin lymphoma (r/r B-NHL).
- Following a single infusion of CB-010 at dose level 1, all 6
patients in cohort 1 achieved complete responses as their best
response. 3 of 6 patients maintained complete responses at 6
months, with 2 of 6 maintaining complete responses at 12 months.
Caribou plans to provide an update from the ongoing ANTLER Phase 1
trial for CB-010 in H2 2023.
- Clinical data presentations are available on Caribou’s website
under Scientific Publications.
- Following demonstration of an encouraging safety profile at
dose level 2 (80x106 CAR-T cells), with no dose-limiting toxicities
(DLTs) in the 3 patients treated, Caribou continues to enroll
patients at dose level 3 (120x106 CAR-T cells).
- The U.S. Food and Drug Administration (FDA) has granted CB-010
Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and
Orphan Drug designations. 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.
- CB-010 is the first allogeneic anti-CD19 CAR-T cell therapy in
the clinic, to Caribou’s knowledge, with a PD-1 knockout (KO), a
genome-editing strategy designed to improve antitumor activity by
limiting premature CAR-T cell exhaustion.
- Additional information on the ANTLER trial (NCT04637763) can be
found at clinicaltrials.gov.
- CB-011: Caribou recently activated clinical
sites for the recruitment of patients at dose level 1 (50x106 CAR-T
cells) of CB-011 in the CaMMouflage Phase 1 trial for relapsed or
refractory multiple myeloma (r/r MM).
- CB-011 is the first allogeneic CAR-T cell therapy in the
clinic, to Caribou’s knowledge, that is engineered to improve
antitumor activity through an immune cloaking strategy with a B2M
KO and insertion of a B2M–HLA-E fusion protein to blunt
immune-mediated rejection.
- Preclinical data for CB-011 were presented in a poster at the
2023 Tandem Meeting: Transplantation & Cellular Therapy
Meetings of ASTCT and CIBMTR, February 15-19, 2023, in Orlando,
Florida. The poster presentation is available on Caribou’s website
under Scientific Publications.
- Additional information on the CaMMouflage trial (NCT05722418)
can be found at clinicaltrials.gov.
- CB-012: Caribou has initiated IND-enabling
studies for CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy, to
support a planned IND application submission for relapsed or
refractory acute myeloid leukemia (r/r AML).
- CB-012 is the first allogeneic CAR-T cell therapy, to Caribou’s
knowledge, with both checkpoint disruption, through a PD-1 KO, and
immune cloaking, through a B2M KO and B2M–HLA-E fusion protein
insertion; both armoring strategies are designed to improve
antitumor activity. CB-012 is engineered with 5 genome edits,
enabled by Caribou’s next-generation CRISPR technology platform,
which uses Cas12a chRDNA genome editing to significantly improve
the specificity of genome edits.
- In preclinical AML models, CB-012 significantly reduced tumor
burden and increased overall survival compared to controls.
- CB-020: Caribou’s first induced pluripotent
stem cell (iPSC)-derived allogeneic CAR-NK cell therapy, CB-020, is
designed to target solid tumors expressing the tumor antigen ROR1.
- Preclinical data supporting the selection of the ROR1 CAR
construct and armoring strategies for the company’s CAR-NK cell
platform were presented at the 12th American Association for Cancer
Research and Japanese Cancer Association (AACR-JCA) Joint
Conference in December 2022. The poster presentation is available
on Caribou’s website under Scientific Publications.
Anticipated 2023 Milestones
- CB-010: Caribou plans to provide an update
from the ongoing ANTLER Phase 1 trial for CB-010 in H2 2023.
- CB-011: Caribou recently activated clinical
sites for the recruitment of patients at dose level 1 and plans to
provide an update on the clearance of dose levels as appropriate
from the CaMMouflage Phase 1 trial for CB-011.
- CB-012: Caribou plans to submit an IND
application for CB-012 in H2 2023.
Upcoming Investor Conferences
- Caribou management plans to participate in the following
investor conferences:
- March 15: Oppenheimer’s 33rd Annual Healthcare Investor
Conference, virtual
- May 9-11: BofA Securities 2023 Healthcare Conference, Las
Vegas
Fourth Quarter and Full Year 2022 Financial
Results
Cash, cash equivalents, and marketable
securities: Caribou had $317.0 million in cash, cash
equivalents, and marketable securities as of December 31, 2022,
compared to $413.5 million as of December 31, 2021. Caribou expects
these cash, cash equivalents, and marketable securities will be
sufficient to fund its current operating plan into 2025.
Licensing and collaboration revenue: Revenue
from Caribou’s licensing and collaboration agreements was $3.7
million for the three months ended December 31, 2022 and $13.9
million for the full year 2022, compared to $2.6 and $9.6 million,
respectively, for the same periods in 2021. The increases were
primarily due to revenue recognized under the AbbVie Agreement.
R&D expenses: Research and development
expenses were $25.7 million for the three months ended December 31,
2022 and $82.2 million for the full year 2022, compared to $15.1
and $52.3 million, respectively, for the same periods in 2021. The
increases were primarily due to costs to advance pipeline programs;
increased headcount, including stock-based compensation; facilities
and other allocated expenses; and increased external manufacturing
and clinical activities.
G&A expenses: General and
administrative expenses were $8.5 million for the three months
ended December 31, 2022 and $38.0 million for the full year 2022,
compared to $7.9 and $24.3 million, respectively, for the same
periods in 2021. The increases were primarily due to increased
headcount, including stock-based compensation; legal, accounting,
insurance, and other expenses necessary to support the growth and
operation of a clinical-stage public company; and facilities and
other allocated expenses.
Net loss: Caribou reported a net loss of $27.0
million for the three months ended December 31, 2022 and $99.4
million for the full year 2022, compared to $18.5 and $66.9
million, respectively, for the same periods in 2021.
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 armored to potentially improve antitumor activity. Caribou
is advancing a pipeline of off-the-shelf cell therapies from its
CAR-T and CAR-NK platforms as readily available treatments for
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
updates from its ANTLER Phase 1 clinical trial for CB-010 as well
as the status and updates from its CaMMouflage Phase 1 clinical
trial for CB-011, expectations about product developments in 2023,
and the submission of an IND application 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 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; the risk that
preclinical study results we observed will not be borne out in
human patients; 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, 2022 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.Condensed Consolidated
Balance Sheet Data(in thousands) (unaudited)
|
December 31,2022 |
|
December 31,2021 |
Cash, cash equivalents, and marketable securities |
$ |
317,036 |
|
|
$ |
413,508 |
|
Total assets |
|
373,765 |
|
|
|
442,356 |
|
Total liabilities |
|
72,894 |
|
|
|
54,531 |
|
Total stockholders' equity |
|
300,871 |
|
|
|
387,825 |
|
Total liabilities and
stockholders' equity |
$ |
373,765 |
|
|
$ |
442,356 |
|
Caribou Biosciences, Inc.Condensed Consolidated
Statement of Operations(in thousands, except share and per share
data)(unaudited)
|
Three Months EndedDecember
31, |
|
Year EndedDecember 31, |
|
|
2022 |
|
|
|
2021 |
|
|
|
2022 |
|
|
|
2021 |
|
Licensing and collaboration
revenue |
$ |
3,692 |
|
|
$ |
2,559 |
|
|
$ |
13,851 |
|
|
$ |
9,598 |
|
Operating expenses: |
|
|
|
|
|
|
|
Research and development |
|
25,736 |
|
|
|
15,111 |
|
|
|
82,230 |
|
|
|
52,255 |
|
General and administrative |
|
8,534 |
|
|
|
7,853 |
|
|
|
38,020 |
|
|
|
24,322 |
|
Total operating expenses |
|
34,270 |
|
|
|
22,964 |
|
|
|
120,250 |
|
|
|
76,577 |
|
Loss from operations |
|
(30,578 |
) |
|
|
(20,405 |
) |
|
|
(106,399 |
) |
|
|
(66,979 |
) |
Other income (expense): |
|
|
|
|
|
|
|
Change in fair value of equity securities |
|
(60 |
) |
|
|
— |
|
|
|
(133 |
) |
|
|
— |
|
Change in fair value of the MSKCC success payments liability |
|
1,388 |
|
|
|
2,158 |
|
|
|
2,429 |
|
|
|
(1,426 |
) |
Gain on extinguishment of PPP Loan |
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
1,584 |
|
Other income, net |
|
2,331 |
|
|
|
89 |
|
|
|
4,752 |
|
|
|
219 |
|
Total other income (expense) |
|
3,659 |
|
|
|
2,247 |
|
|
|
7,048 |
|
|
|
377 |
|
Net loss before provision for
income taxes |
$ |
(26,919 |
) |
|
$ |
(18,158 |
) |
|
$ |
(99,351 |
) |
|
$ |
(66,602 |
) |
Provision for income
taxes |
|
70 |
|
|
|
321 |
|
|
|
70 |
|
|
|
321 |
|
Net loss |
$ |
(26,989 |
) |
|
$ |
(18,479 |
) |
|
$ |
(99,421 |
) |
|
$ |
(66,923 |
) |
Other comprehensive loss: |
|
|
|
|
|
|
|
Net unrealized loss on available-for-sale marketable
securities |
|
517 |
|
|
|
(135 |
) |
|
|
(1,383 |
) |
|
|
(135 |
) |
Net comprehensive loss |
$ |
(26,472 |
) |
|
$ |
(18,614 |
) |
|
$ |
(100,804 |
) |
|
$ |
(67,058 |
) |
Net loss per share, basic and
diluted |
$ |
(0.44 |
) |
|
$ |
(0.31 |
) |
|
$ |
(1.64 |
) |
|
$ |
(2.11 |
) |
Weighted-average common shares
outstanding, basic and diluted |
|
61,001,150 |
|
|
|
60,180,759 |
|
|
|
60,801,133 |
|
|
|
31,663,243 |
|
Caribou Biosciences Contacts:
Investors:Amy Figueroa,
CFAafigueroa@cariboubio.com
Media:Peggy Vorwald,
PhDpvorwald@cariboubio.com
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