Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today reported
financial results for the second quarter of 2023 and reviewed
recent business updates.
“In 2023, we have advanced our programs to build value across
the pipeline and position Caribou for continued momentum ahead,”
said Rachel Haurwitz, PhD, Caribou’s president and chief executive
officer. “For our lead program, we are excited by the positive
CB-010 dose escalation data demonstrating response rates that rival
those from the approved autologous CAR-T cell therapies. As we
develop CB-010 for the larger second-line LCBL patient population,
we continue to enroll patients in dose expansion and anticipate
reporting initial dose expansion data in the first half of 2024. We
also look forward to meeting with the FDA later this year to
discuss a potential pivotal clinical trial in second-line LBCL
patients. Additionally, we continue to enroll patients in our
CaMMouflage trial for CB-011 and plan for an IND submission for
CB-012 in the second half of this year.”
Accomplishments and highlights
Pipeline and technology
- CB-010: Caribou reported long-term follow-up
data from all 16 patients treated in dose escalation of the ongoing
ANTLER Phase 1 clinical trial of CB-010, an allogeneic anti-CD19
CAR-T cell therapy. In ANTLER dose escalation, three dose levels of
CB-010 were evaluated (40x106, 80x106, and 120x106 CAR-T cells) in
patients with multiple subtypes of aggressive relapsed or
refractory B cell non-Hodgkin lymphoma (r/r B-NHL). As of the June
20, 2023 data cutoff date, results demonstrated:
- CB-010 was generally well tolerated with adverse events
consistent with autologous and allogeneic anti-CD19 CAR-T cell
therapies.
- 94% overall response rate (ORR; 15 of 16 patients) was observed
following a single dose of CB-010.
- 69% of patients (11 of 16) achieved a complete response
(CR).
- 44% of patients (7 of 16) had a CR at ≥6 months; 24 months is
the longest CR maintained to date.
- For the subset of patients with large B cell lymphoma (LBCL)
(N=10):
- A 90% ORR (9 of 10) was observed.
- 70% (7 of 10) achieved a CR.
- 50% (5 of 10) had a CR at ≥6 months; 18 months is the longest
CR maintained to date.
- Based on these positive data, Caribou is enrolling second-line
patients with LBCL in the ongoing dose expansion portion of the
ANTLER clinical trial. In expansion, the mid dose and the high dose
from escalation (80x106 and 120x106 CAR-T cells) are being
evaluated in approximately 30 second-line patients (approximately
15 patients per dose level) to determine the recommended Phase 2
dose (RP2D). Once the RP2D is determined, Caribou may enroll
additional patients in ANTLER.
- CB-011: Caribou is enrolling patients at dose
level 1 (50x106 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).
- CB-012: Caribou is advancing IND-enabling
activities for CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy,
for relapsed or refractory acute myeloid leukemia (r/r AML).
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 YE 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 submit an IND application for r/r AML in H2 2023.
Corporate updates
- $25.0 million Pfizer investment: On June 30,
2023, Pfizer invested $25.0 million in Caribou common shares. In
conjunction with the investment, Sriram Krishnaswami, PhD, joined
Caribou’s scientific advisory board. 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.
- Completed successful $134.6 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 approximate net proceeds to
Caribou were $134.6 million.
Second quarter 2023 financial results
Cash, cash equivalents, and marketable
securities: Caribou had $292.5 million in cash, cash
equivalents, and marketable securities as of June 30, 2023, which
included the $25.0 million proceeds from the Pfizer investment,
compared to $317.0 million as of December 31, 2022. This amount
does not include the approximately $134.6 million in net proceeds
from the Company’s underwritten public offering completed in the
third quarter of 2023. Caribou expects its cash, cash equivalents,
marketable securities, and net proceeds from the recent public
offering 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 $3.8
million for the three months ended June 30, 2023, compared to $4.2
million for the same period in 2022. The decrease was primarily due
to a reduction in revenue recognized under the AbbVie Collaboration
and License Agreement, partially offset by a revenue increase
related to a veterinary therapeutics licensing agreement.
R&D expenses: Research and development
expenses were $26.5 million for the three months ended June 30,
2023, compared to $22.6 million for the same period in 2022. The
increase was primarily due to personnel-related expenses, including
stock-based compensation; costs to advance pipeline programs,
including the ANTLER and CaMMouflage Phase 1 trials; and facilities
and other allocated expenses.
G&A expenses: General and
administrative expenses were $10.1 million for the three months
ended June 30, 2023, compared to $10.0 million for the same period
in 2022. The increase was primarily due to facilities and other
allocated expenses; patent prosecution and maintenance costs; and
personnel-related expenses, including stock-based compensation, due
to headcount increases. The increase was partially offset by lower
insurance and legal expenses.
Net loss: Caribou reported a net loss of $29.5
million for the three months ended June 30, 2023, compared to $26.7
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) comprising four different subtypes of aggressive
r/r B-NHL (DLBCL NOS, PMBCL, HGBL, and tFL). CB-010 is an
allogeneic anti-CD19 CAR-T cell therapy engineered using Cas9
CRISPR hybrid RNA-DNA (chRDNA) 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 also 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 the second 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 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 the third product
candidate from Caribou’s allogeneic CAR-T cell therapy platform and
is being evaluated in investigational new drug (IND)-enabling
studies. 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. CB-012 is 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.
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, expectations about product developments in 2023, and
expectations regarding the submission of an IND application 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) |
|
|
|
|
|
|
|
|
June 30, 2023 |
|
December 31, 2022 |
|
Cash, cash equivalents, and marketable securities |
|
$ |
292,521 |
|
$ |
317,036 |
|
Total assets |
|
|
349,647 |
|
|
373,765 |
|
Total liabilities |
|
|
80,189 |
|
|
72,894 |
|
Total stockholders' equity |
|
|
269,458 |
|
|
300,871 |
|
Total liabilities and stockholders' equity |
|
$ |
349,647 |
|
$ |
373,765 |
|
|
|
|
|
|
|
Caribou Biosciences, Inc. |
Condensed Consolidated Statement of Operations |
(in thousands, except share and per share data) |
(unaudited) |
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended, June 30, |
|
Six Months Ended, June 30, |
|
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
|
Licensing and collaboration revenue |
|
$ |
3,755 |
|
|
$ |
4,192 |
|
|
$ |
7,257 |
|
|
$ |
6,856 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
26,503 |
|
|
|
22,579 |
|
|
|
52,212 |
|
|
|
36,503 |
|
|
General and administrative |
|
|
10,120 |
|
|
|
10,044 |
|
|
|
19,029 |
|
|
|
19,637 |
|
|
Total operating expenses |
|
|
36,623 |
|
|
|
32,623 |
|
|
|
71,241 |
|
|
|
56,140 |
|
|
Loss from operations |
|
|
(32,868 |
) |
|
|
(28,431 |
) |
|
|
(63,984 |
) |
|
|
(49,284 |
) |
|
Other income (expense): |
|
|
|
|
|
|
|
|
|
Change in fair value of equity securities |
|
|
22 |
|
|
|
(16 |
) |
|
|
7 |
|
|
|
(104 |
) |
|
Change in fair value of the MSKCC success payments liability |
|
|
279 |
|
|
|
1,052 |
|
|
|
534 |
|
|
|
2,648 |
|
|
Other income, net |
|
|
3,048 |
|
|
|
698 |
|
|
|
5,880 |
|
|
|
955 |
|
|
Total other income |
|
|
3,349 |
|
|
|
1,734 |
|
|
|
6,421 |
|
|
|
3,499 |
|
|
Net loss |
|
$ |
(29,519 |
) |
|
$ |
(26,697 |
) |
|
$ |
(57,563 |
) |
|
$ |
(45,785 |
) |
|
Other comprehensive income (loss): |
|
|
|
|
|
|
|
|
|
Net unrealized gain (loss) on available-for-sale marketable
securities, net of tax |
|
|
(406 |
) |
|
|
(492 |
) |
|
|
382 |
|
|
|
(1,446 |
) |
|
Net comprehensive loss |
|
$ |
(29,925 |
) |
|
$ |
(27,189 |
) |
|
$ |
(57,181 |
) |
|
$ |
(47,231 |
) |
|
Net loss per share, basic and diluted |
|
$ |
(0.48 |
) |
|
$ |
(0.44 |
) |
|
$ |
(0.94 |
) |
|
$ |
(0.75 |
) |
|
Weighted-average common shares outstanding, basic and diluted |
|
|
61,417,934 |
|
|
|
60,757,689 |
|
|
|
61,302,863 |
|
|
|
60,652,532 |
|
|
|
|
|
|
|
|
|
|
|
|
Caribou Biosciences, Inc.
contacts:Investors:Amy Figueroa,
CFAinvestor.relations@cariboubio.com
Media:Peggy Vorwald,
PhDmedia@cariboubio.com
Caribou Biosciences (NASDAQ:CRBU)
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Caribou Biosciences (NASDAQ:CRBU)
과거 데이터 주식 차트
부터 5월(5) 2023 으로 5월(5) 2024