On November 8, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported financial results for the third quarter of 2022 and provided a business update (Press release, Caribou Biosciences, NOV 8, 2022, View Source [SID1234623513]).
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"We have seen highly promising results from our lead allogeneic cell therapy, CB-010, at the lowest starting dose in the ANTLER clinical trial in patients with relapsed or refractory B cell non-Hodgkin lymphoma," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "The safety and antitumor activity for CB-010 at dose level 1 are encouraging, and we look forward to generating additional efficacy and durability data from the dose escalation phase of the ANTLER trial. In addition, progress continues across our pipeline as we submitted our second IND, for CB-011, in the fourth quarter and plan to submit our third IND, for CB-012, in 2023. Later this year, we look forward to sharing the target selection for CB-020, the lead program in our CAR-NK platform for solid tumors."
Recent Business Highlights
Pipeline and Technology
•For CB-011, an investigational new drug (IND) application was submitted to the U.S. Food and Drug Administration (FDA) in Q4 2022 for relapsed or refractory multiple myeloma (r/r MM).
•CB-010 and the ANTLER Phase 1 trial:
◦In October 2022, a poster for a case report on long-term follow up for the first patient dosed in the ANTLER Phase 1 trial was presented at the Lymphoma, Leukemia, & Myeloma (LL&M) Congress. This patient achieved a complete response (CR) at day 28 and maintained a long-term CR at 15 months following a single dose of CB-010 at dose level 1 (40×106 CAR-T cells). Before joining the ANTLER trial, the patient had received eight prior lines of systemic anti-cancer therapy for relapsed aggressive B cell non-Hodgkin lymphoma (r/r B-NHL).
◦Based on the promising initial safety data and response rates at dose level 1, the ANTLER trial is currently enrolling patients at dose level 2 (80×106 CAR-T cells).
◦In September 2022, CB-010 was granted Orphan Drug Designation (ODD) for follicular lymphoma (FL) by the FDA. Patients with aggressively behaving FL are 1 of 7 subtypes of r/r B-NHL patients eligible for enrollment in the dose escalation portion of the ANTLER Phase 1 trial.
◦As previously reported:
▪6 of 6 patients (100%) achieved a CR as their best response
▪3 of 6 patients (50%) maintained a CR at 6 months
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▪15 months has been the longest CR, achieved by the first patient dosed with CB-010
▪CB-010 at dose level 1 was generally well tolerated in the ANTLER trial
Anticipated Milestones for Fourth Quarter 2022 and Beyond
•CB-010: Caribou plans to share additional data from cohort 1 of the ongoing ANTLER Phase 1 trial for CB-010, an allogeneic anti-CD19 CAR-T cell therapy for r/r B-NHL, by YE 2022.
•CB-011: Caribou anticipates the FDA’s response to Caribou’s IND application for CB-011, an allogeneic anti-BCMA CAR-T cell therapy for r/r MM, by YE 2022.
•CB-020: Caribou expects to announce target selection for CB-020, an iPSC-derived CAR-NK cell therapy for solid tumors, in Q4 2022. Additionally, Caribou expects to disclose armoring strategies under development for its CAR-NK cell platform in Q4 2022.
•CB-012: Caribou expects to submit an IND application for CB-012, an allogeneic anti-CLL-1 CAR-T cell therapy for relapsed or refractory acute myeloid leukemia (r/r AML), in 2023.
Upcoming Meetings
•American Society of Hematology (ASH) (Free ASH Whitepaper) 64th Annual Meeting, December 10-13, 2022
◦Trial-in-progress poster presentation for the ANTLER Phase 1 trial of CB-010 in r/r B-NHL
•Caribou management plans to participate in the following upcoming investor conferences:
◦November 14: Barclays 2022 Gene Editing & Gene Therapy Summit
◦November 17: Jefferies Global Healthcare Conference
◦November 30: 5th Annual Evercore ISI HealthCONx Conference
◦December 7: BofA Securities Biotech SMID Cap Conference
Third Quarter 2022 Financial Results
Cash, cash equivalents, and marketable securities: Caribou had $342.6 million in cash, cash equivalents, and marketable securities as of September 30, 2022, compared to $413.5 million as of December 31, 2021.
Licensing and collaboration revenue: Revenue from Caribou’s licensing and collaboration agreements was $3.3 million for the three months ended September 30, 2022, compared to $4.0 million for the same period in 2021. The decrease was primarily due to a decrease in revenue under the AbbVie collaboration and license agreement.
R&D expenses: Research and development expenses were $20.0 million for the three months ended September 30, 2022, compared to $15.8 million for the same period in 2021. The increase was primarily due to costs to advance pipeline programs; increased headcount, including stock-based compensation; and facilities and other expenses; partially offset by a decrease related to the timing of external manufacturing activities.
G&A expenses: General and administrative expenses were $9.8 million for the three months ended September 30, 2022, compared to $6.8 million for the same period in 2021. The increase was
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primarily due to costs for increased headcount, including stock-based compensation; facilities and other expenses; and legal, accounting, insurance, and other expenses necessary to support the growth and operation of a clinical-stage public company.
Net loss: Caribou reported a net loss of $26.6 million for the three months ended September 30, 2022, compared to a net loss of $21.0 million for the same period 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.