Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today reported
financial results for the third quarter of 2023 and reviewed recent
business updates.
“In 2023, we have advanced our programs to build
value across the pipeline and position Caribou as a leader in the
allogeneic CAR-T cell therapy space,” said Rachel Haurwitz, PhD,
Caribou’s president and chief executive officer. “We plan to meet
with the FDA to discuss a potential pivotal trial in second-line
LBCL patients for our lead program, CB-010, and we intend to share
the agency’s feedback by the end of this year. Patient enrollment
is ongoing in the ANTLER trial, and we expect to report initial
dose expansion data in the first half of 2024. Additionally, we are
enrolling patients in our CaMMouflage trial for CB-011 and plan to
initiate enrollment in the AMpLify Phase 1 clinical trial for
CB-012 by mid-2024. With two years of cash and continued financial
discipline, we are well positioned to execute on our current
programs and continue Caribou’s momentum.”
Accomplishments and highlights
Pipeline and technology
- CB-010: Caribou
continues to enroll second-line LBCL patients in the dose expansion
portion of the ongoing ANTLER Phase 1 clinical trial based on
positive data from the dose escalation portion of the trial. CB-010
is an allogeneic anti-CD19 CAR-T cell therapy in development for
relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL). The
dose expansion data will be used to determine the recommended Phase
2 dose (RP2D).
- CB-011: Caribou
continues to enroll patients at dose level 2 (150x106 CAR-T cells)
in the dose escalation portion of the ongoing CaMMouflage Phase 1
trial of CB-011, an allogeneic anti-BCMA CAR-T cell therapy, for
relapsed or refractory multiple myeloma (r/r MM). Caribou has
concluded dose level 1 (50x106 CAR-T cells, N=3) without any
dose-limiting toxicities (DLTs) and received Study Steering
Committee clearance to proceed with dosing patients at dose level
2.
- CB-012: Caribou
recently announced clearance of the company’s investigational new
drug (IND) application from the U.S. Food and Drug Administration
(FDA) for CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy, for
relapsed or refractory acute myeloid leukemia (r/r AML). Clinical
site activation is underway for the AMpLify Phase 1 clinical trial
and Caribou plans to initiate patient enrollment by mid-2024 to
evaluate CB-012 at ascending dose levels starting at dose level 1
(25x106 CAR-T cells).
Anticipated milestones
- CB-010: Caribou plans to meet with the FDA to
discuss a potential pivotal clinical trial in second-line LBCL
patients and plans to share FDA feedback by year-end 2023. The
Company also plans to report initial dose expansion data in
second-line LBCL patients from the ongoing ANTLER trial in H1
2024.
- CB-011: Caribou plans to provide updates on
dose escalation as the CaMMouflage Phase 1 clinical trial in r/r MM
advances.
- CB-012: Caribou plans
to initiate patient enrollment in the AMpLify Phase 1 clinical
trial in r/r AML by mid-2024.
Corporate updates
- Appointed two multiple myeloma experts to Caribou’s
scientific advisory board: Caribou appointed two experts
in multiple myeloma drug development to its scientific advisory
board in the third quarter of 2023.
- Sundar Jagannath, MD, director of the Center of Excellence for
Multiple Myeloma and professor of medicine at the Tisch Cancer
Institute of Mount Sinai.
- Sriram Krishnaswami, PhD, vice president and development head
for multiple myeloma at Pfizer Oncology’s Global Product
Development division.
- Appointed chief people officer: In September
2023, Caribou appointed Reigin Zawadzki to the newly created
position of chief people officer. Ms. Zawadzki brings over 20 years
of experience leading human resources in the biotechnology industry
and will lead Caribou’s people strategy.
- Completed successful $134.4 million follow-on
financing: In the third quarter of 2023, Caribou completed
an underwritten public offering of 22,115,384 shares of its common
stock, which included the full exercise of the underwriters’ option
to purchase additional shares. The net proceeds to Caribou were
$134.4 million.
- Received $25.0 million Pfizer investment: In
July 2023, Caribou announced that Pfizer invested $25.0 million in
Caribou common shares on June 30, 2023. Caribou will use the
proceeds from this investment to advance CB-011. Caribou maintains
full ownership and control of CB-011 and its other allogeneic CAR-T
and CAR-NK cell therapies.
Third quarter 2023 financial results
Cash, cash equivalents, and marketable
securities: Caribou had $396.7 million in cash, cash
equivalents, and marketable securities as of September 30, 2023,
compared to $317.0 million as of December 31, 2022. This amount
includes the approximately $134.4 million in net proceeds from the
Company’s underwritten public offering and the $25.0 million equity
investment from Pfizer. Caribou expects its cash, cash equivalents,
and marketable securities will be sufficient to fund its current
operating plan into Q4 2025.
Licensing and collaboration revenue: Revenue
from Caribou’s licensing and collaboration agreements was $23.7
million for the three months ended September 30, 2023, compared to
$3.3 million for the same period in 2022. The increase was
primarily due to $21.5 million in revenue recognized under the
AbbVie Collaboration and License Agreement, including $20.8 million
of 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 $28.6 million for the three months ended September
30, 2023, compared to $20.0 million for the same period in 2022.
The increase was primarily due to costs to advance pipeline
programs, including the CB-010 ANTLER and CB-011 CaMMouflage 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 September 30, 2023, compared to $9.8 million for the same
period in 2022. The decrease was primarily due to lower patent
prosecution and maintenance costs, and lower insurance, accounting,
and other service-related expenses. This decrease was partially
offset by an increase in personnel-related expenses, including
stock-based compensation, due to headcount increases.
Net loss: Caribou reported a net loss of $10.0
million for the three months ended September 30, 2023, compared to
$26.6 million for the same period 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. 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 designation 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 will be
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.
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 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.
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
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, plans for meeting with the FDA to discuss a
potential pivotal clinical trial of CB-010 in second-line LBCL
patients and plans for sharing feedback from such meeting,
expectations about product developments, and expectations regarding
the timing of initiating patient enrollment in the AMpLify Phase 1
clinical trial for CB-012, and Caribou’s 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; 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) |
|
|
|
|
|
September 30, 2023 |
|
December 31, 2022 |
Cash, cash equivalents, and marketable securities |
$ |
396,707 |
|
|
$ |
317,036 |
|
Total
assets |
|
457,521 |
|
|
|
373,765 |
|
Total
liabilities |
|
59,253 |
|
|
|
72,894 |
|
Total
stockholders' equity |
|
398,268 |
|
|
|
300,871 |
|
Total
liabilities and stockholders' equity |
$ |
457,521 |
|
|
$ |
373,765 |
|
Caribou Biosciences,
Inc.Condensed Consolidated Statement of Operations(in thousands,
except share and per share data)(unaudited) |
|
|
|
|
|
|
|
|
|
Three Months Ended, September 30, |
|
Nine Months Ended, September 30, |
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Licensing
and collaboration revenue |
$ |
23,662 |
|
|
$ |
3,303 |
|
|
$ |
30,919 |
|
|
$ |
10,159 |
|
Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
|
28,584 |
|
|
|
19,991 |
|
|
|
80,796 |
|
|
|
56,494 |
|
General and administrative |
|
9,711 |
|
|
|
9,849 |
|
|
|
28,740 |
|
|
|
29,486 |
|
Total operating expenses |
|
38,295 |
|
|
|
29,840 |
|
|
|
109,536 |
|
|
|
85,980 |
|
Loss from
operations |
|
(14,633 |
) |
|
|
(26,537 |
) |
|
|
(78,617 |
) |
|
|
(75,821 |
) |
Other income
(expense): |
|
|
|
|
|
|
|
Change in fair value of equity securities |
|
(4 |
) |
|
|
31 |
|
|
|
3 |
|
|
|
(73 |
) |
Change in fair value of the MSKCC success payments liability |
|
(139 |
) |
|
|
(1,607 |
) |
|
|
395 |
|
|
|
1,041 |
|
Other income, net |
|
4,774 |
|
|
|
1,466 |
|
|
|
10,654 |
|
|
|
2,421 |
|
Total other income (expense) |
|
4,631 |
|
|
|
(110 |
) |
|
|
11,052 |
|
|
|
3,389 |
|
Net
loss |
$ |
(10,002 |
) |
|
$ |
(26,647 |
) |
|
$ |
(67,565 |
) |
|
$ |
(72,432 |
) |
Other
comprehensive income (loss): |
|
|
|
|
|
|
|
Net unrealized gain (loss) on available-for-sale marketable
securities, net of tax |
|
155 |
|
|
|
(454 |
) |
|
|
537 |
|
|
|
(1,900 |
) |
Net
comprehensive loss |
$ |
(9,847 |
) |
|
$ |
(27,101 |
) |
|
$ |
(67,028 |
) |
|
$ |
(74,332 |
) |
Net loss per
share, basic and diluted |
$ |
(0.12 |
) |
|
$ |
(0.44 |
) |
|
$ |
(0.98 |
) |
|
$ |
(1.19 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
83,783,992 |
|
|
|
60,886,921 |
|
|
|
68,878,921 |
|
|
|
60,731,520 |
|
|
|
|
|
|
|
|
|
Caribou Biosciences, Inc.
contacts:Investors:Amy Figueroa,
CFAinvestor.relations@cariboubio.com
Media:Peggy Vorwald,
PhDmedia@cariboubio.com
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