Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage
CRISPR genome-editing biopharmaceutical company, today announced
the appointment of Tim Kelly to the newly created position of chief
technology officer. Mr. Kelly brings over 25 years of experience in
global clinical and commercial product development, manufacturing,
and supply chain operations with large and small biopharmaceutical
and cell and gene therapy companies in the US and Europe. In
addition, Caribou highlighted successful execution across its
allogeneic CAR-T cell therapy platform over the past year and
provided an outlook on multiple clinical catalysts planned for
2024.
“2023 was a year of exceptional progress and leadership for
Caribou as we advanced our three clinical programs and strengthened
our balance sheet with a public offering and investment from
Pfizer. We are excited to kick off 2024 by welcoming Tim Kelly as
Caribou’s chief technology officer,” said Rachel Haurwitz, PhD,
Caribou’s president and chief executive officer. “Tim’s extensive
experience in global pharmaceutical product development,
manufacturing, and commercialization, combined with his proven
leadership and strategic vision, will be instrumental as we advance
our pipeline and plan for the future. We are pleased to have Tim
join us on our mission of bringing transformative therapies to
patients with devastating diseases.”
Mr. Kelly will lead the company’s technical operations strategy
and execution and will report to Dr. Haurwitz. Tim joins Caribou
from Oxford Biomedica Solutions, a spin-out of Homology Medicines,
offering AAV product development and manufacturing services, where
he was chief executive officer and board chair. Prior to Oxford
Biomedica Solutions, he was chief operating officer at Homology
Medicines, Inc., where he led operations, process and platform
development strategy, and product manufacturing strategy for gene
therapy and gene editing technology. Earlier, he led technical
operations at Sarepta, Shire, UCB, and Biogen, and was a fighter
pilot in the US Air Force. Tim earned an MS/MBA from Troy State
University and a BS, with an emphasis in engineering mechanics,
from the United States Air Force Academy.
"I'm thrilled to join Caribou as the company prepares to
initiate its first pivotal trial. The potential of genome editing
to revolutionize medicine is undeniable, and Caribou's innovative
chRDNA technology has enabled development of armored allogeneic
CAR-T cell therapies that hold immense promise,” said Mr. Kelly. “I
look forward to leveraging my experience in building and scaling
global process and manufacturing operations to ensure provider
support and rapid patient access for commercialization and delivery
of these potentially transformative therapies."
2023 clinical highlights and corporate
accomplishments
Clinical highlightsCB-010, an
allogeneic anti-CD19 CAR-T cell therapy for B cell non-Hodgkin
lymphoma
- Caribou reported encouraging data from the dose escalation
portion of the ANTLER Phase 1 trial evaluating CB-010 in 16
patients with relapsed or refractory B cell non-Hodgkin lymphoma
(r/r B-NHL). Dose escalation data showed CB-010’s response rates
and safety profile have the potential to rival those of the
approved autologous CAR-T cell therapies.
- In the ongoing ANTLER Phase 1 trial, Caribou is enrolling
second-line relapsed or refractory large B cell lymphoma (r/r LBCL)
patients in the dose expansion portion. Currently, 22 U.S. and 3
international sites are active, with additional site activation
underway.
- Caribou provided a regulatory update based on feedback from the
U.S. Food and Drug Administration (FDA) following a Type B clinical
meeting. The company received the FDA’s feedback 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.
- 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.
“My enthusiasm for the potential of off-the-shelf cell therapies
combined with the encouraging CB-010 dose escalation data prompted
me to establish Westmead Hospital as the first international site
for the ANTLER trial,” said Kenneth Micklethwaite, MD, clinical
associate professor at the University of Sydney and hematology
staff specialist at the Westmead Hospital. “I look forward to
participating in the planned pivotal trial as patients in the
second-line setting have an urgent need for treatment given the
limitations of current treatment options."
CB-011, an allogeneic anti-BMCA CAR-T cell therapy for
multiple myeloma
- Caribou initiated the CaMMouflage Phase 1 trial evaluating
CB-011 in patients with relapsed or refractory multiple myeloma
(r/r MM).
- CB-011 was granted Fast Track and Orphan Drug designation by
the FDA, which are designed to expedite the development and review
processes for promising therapeutic candidates that may fill an
unmet medical need.
- CaMMouflage trial enrollment has concluded for dose level 1
(50x106 CAR-T cells, N=3) and dose level 2 (150x106 CAR-T cells,
N=3) without any dose-limiting toxicities (DLTs), and the trial is
enrolling patients at dose level 3 (450x106 CAR-T cells).
CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy for
acute myeloid leukemia
- The investigational new drug (IND) application for CB-012 was
cleared by the FDA for evaluation in relapsed or refractory acute
myeloid leukemia (r/r AML).
- The first clinical sites are active for the AMpLify Phase 1
trial for CB-012 in r/r AML with additional site activation
underway.
Corporate
accomplishmentsExpanded executive
leadership team and scientific
advisory board (SAB)
- Appointed Tim Kelly as Caribou’s chief technology officer,
leading the company’s process development and manufacturing
organizations.
- Appointed Reigin Zawadzki as Caribou’s chief people officer,
leading the company’s people strategy.
- Expanded Caribou’s SAB with a renowned lymphoma expert and two
leading multiple myeloma experts:
- Stephen Schuster, MD, Robert and Margarita Louis-Dreyfus
professor of chronic lymphocytic leukemia and lymphoma and the
director of the lymphoma program and lymphoma translational
research at the Perelman School of Medicine at the University of
Pennsylvania, with Penn Medicine’s Abramson Cancer Center.
- 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.
Armored balance sheet for cash runway into Q4
2025
- Completed successful $134.4 million follow-on financing.
- Received $25 million Pfizer investment with proceeds to advance
CB-011.
- Reported $396.7 million in cash, cash equivalents, and
marketable securities as of September 30, 2023, which is expected
to fund the current operating plan into Q4 2025.
2024 anticipated
milestonesCB-010: Caribou plans to report
initial dose expansion data from the ANTLER trial and share the
recommended Phase 2 dose (RP2D) for CB-010 in Q2 2024. The company
plans to initiate a Phase 3 pivotal trial evaluating CB-010 in
second-line LBCL by year-end 2024. CB-011: Caribou
plans to report initial dose escalation data by year-end 2024.
CB-012: Caribou plans to initiate patient
enrollment in the AMpLify Phase 1 clinical trial in r/r AML in H1
2024.
Caribou to present at the 42nd Annual J.P. Morgan
Healthcare ConferenceDr. Haurwitz is scheduled to present
a corporate update at the 42nd Annual J.P. Morgan Healthcare
Conference on Thursday, January 11, 2024 at 11:15 am PST.
A live webcast of the presentation will be accessible via
Caribou’s website on the Events page. The archived
webcast will be available on the Caribou website for 30 days after
the event.
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 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. 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 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
status and updates from its ANTLER Phase 1 clinical trial for
CB-010, including expectations regarding the timing of reporting
initial dose expansion data from the ANTLER trial and disclosure of
the recommended Phase 2 dose for CB-010, its planned Phase 3
pivotal trial for CB-010 in second-line LBCL patients, as well as
the status and updates from its CaMMouflage Phase 1 clinical trial
for CB-011 and expectations regarding the timing of reporting
initial dose escalation data, 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; 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, 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.
In addition, caution should be exercised when interpreting
results from separate trials involving separate product candidates.
Clinical trials of other companies’ CAR-T cell therapies referenced
in this press release were run independently of Caribou and Caribou
has only reviewed publicly available reports of those trials.
Caribou has not performed any head-to-head trials comparing any of
these other CAR-T cell therapies with CB-010. As such, the results
of these other clinical trials may not be comparable to clinical
results for CB-010. The design of these other trials vary in
material ways from the design of the clinical trials for CB-010,
including with respect to patient populations, follow-up times, the
clinical trial phase, and subject characteristics. As a result,
cross-trial comparisons may have no interpretive value on Caribou’s
existing or future results. For further information and to
understand these material differences, you should read the reports
for the other companies’ clinical trials.
Caribou Biosciences, Inc.
contacts:Investors:Amy Figueroa,
CFAinvestor.relations@cariboubio.com
Media:Peggy Vorwald,
PhDmedia@cariboubio.com
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/483a3c6c-d044-4558-b33a-d06cb0ad1327
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