On August 9, 2022 Caribou Biosciences, Inc. (Nasdaq: CRBU), a leading clinical-stage CRISPR genome-editing biopharmaceutical company, reported financial results for the second quarter of 2022 and provided a business update (Press release, Caribou Biosciences, AUG 9, 2022, View Source [SID1234617911]).

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"During the first half of this year, we made significant progress advancing our pipeline of genome-edited allogeneic CAR-T and CAR-NK cell therapies," said Rachel Haurwitz, Ph.D., Caribou’s president and chief executive officer. "We recently presented encouraging initial clinical data at EHA (Free EHA Whitepaper) from our Phase 1 ANTLER trial for CB-010, demonstrating a 100% complete response rate as the best response at dose level 1 in six patients with relapsed or refractory B cell non-Hodgkin lymphoma. Based on the promising initial safety and efficacy data at dose level 1, we are enrolling patients at dose level 2. The ANTLER data have exceeded our expectations and are an important step toward validating our chRDNA genome-editing platform. We are excited to advance our plans for future development of CB-010 and our broader pipeline, including CB-011 for relapsed or refractory multiple myeloma and CB-020, the first solid tumor-targeted program from our CAR-NK platform."

Recent Business Highlights

Pipeline and Technology

ANTLER trial:
Encouraging clinical data from the ANTLER Phase 1 trial of CB-010 in patients with relapsed or refractory B cell non-Hodgkin lymphoma (r/r B-NHL) were presented in a poster at the European Hematology Association (EHA) (Free EHA Whitepaper) 2022 Hybrid Congress. A 100% complete response (CR) rate (n=6) was observed as best response following a single dose of CB-010 at dose level 1 (40×106 CAR-T cells). CB-010 was generally well tolerated.
Following the EHA (Free EHA Whitepaper) poster presentation, 1 additional patient had their 6-month evaluation, which showed they maintained a CR at 6 months, resulting in an overall 50% 6-month CR rate (n=6) for cohort 1 following a single, starting dose of CB-010.
Additionally, as disclosed concurrently with the EHA (Free EHA Whitepaper) poster, the first patient treated with CB-010 maintained a CR at 12 months.
Based on the promising initial safety data and response rate at dose level 1, the ANTLER trial is currently enrolling patients at dose level 2 (80×106 CAR-T cells) and the company plans to share additional cohort 1 ANTLER data by YE 2022.
chRDNA technology:
Presentation of data on Caribou’s CRISPR hybrid RNA-DNA (chRDNA) technology at the 25th Annual Meeting of the American Society for Gene and Cell Therapy (ASGCT) (Free ASGCT Whitepaper). The preclinical studies highlighted the mechanism underlying the superior specificity of Caribou’s chRDNA guides for genome editing of primary human T cells.
Corporate

In May 2022, Caribou appointed David Johnson to the board of directors. Mr. Johnson, who currently serves as chief commercial officer at Global Blood Therapeutics, is a seasoned executive with 30 years of commercial and operational experience in the biopharmaceutical industry and has an impressive record of successfully building commercial infrastructure and launching new medicines for patients. Previously, Mr. Johnson worked at Gilead Sciences, Inc. for 15 years and at GlaxoSmithKline for 11 years.
Caribou recently hired several professionals with significant biotechnology and pharmaceutical industry experience:
Tonia Nesheiwat, Pharm.D., vice president of medical affairs
Daniel Poon, vice president of operations and information technology
Socorro Portella, M.D., vice president of clinical development
Saeid Yazdani, vice president of portfolio management
In July 2022, Caribou joined the American Society for Transplantation and Cellular Therapy (ASTCT) Corporate Council to engage in joint problem-solving and collaborative opportunities that will advance the cause and culture of blood and marrow transplantation and cellular therapy.
Anticipated Milestones for 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 anti-CD19 CAR-T cell therapy for r/r B-NHL, by YE 2022.
CB-011: Caribou expects to submit an IND application for CB-011, an anti-BCMA CAR-T cell therapy for relapsed or refractory multiple myeloma (r/r MM), in Q4 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 anti-CLL-1 CAR-T cell therapy for r/r acute myeloid leukemia (AML), in 2023.
Upcoming 2022 Meetings

September 19-22: 7th Annual CAR-TCR Summit
Syed Rizvi, M.D., Caribou’s chief medical officer, to present an encore of initial ANTLER clinical data
Justin Skoble, Ph.D., Caribou’s vice president of technical operations, to present on how Caribou’s chRDNA genome-editing technology is being applied to increase persistence and antitumor activity in preclinical models
In September and October, Caribou management plans to participate in the following investor conferences:
September 7-8: Citi 17th Annual BioPharma Conference 2022
September 12-14: Morgan Stanley 20th Annual Global Healthcare Conference
September 13: H.C. Wainwright 24th Annual Global Investment Conference
September 29-30: Jefferies Cell and Genetic Medicine Summit
October 6: BMO BioPharma Spotlight Series – Gene Editing & Therapy
Second Quarter 2022 Financial Results

Cash, cash equivalents, and marketable securities: Caribou had $366.1 million in cash, cash equivalents, and marketable securities as of June 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 $4.2 million for the three months ended June 30, 2022, compared to $1.5 million for the same period in 2021. The increase was primarily due to an increase in revenue recognized pursuant to the AbbVie collaboration and license agreement.

R&D expenses: Research and development expenses were $22.6 million for the three months ended June 30, 2022, compared to $12.3 million for the same period in 2021. The increase was primarily due to external activities related to the ANTLER Phase 1 clinical trial and contract manufacturing for CB-010 and additional product candidates; other research and development expenses to advance IND-enabling studies for CB-011 and preclinical research for additional programs; personnel-related expenses, including stock-based compensation, attributable to increased headcount; and facility expenses; partially offset by a decrease in expenses relating to licensing, sublicensing revenue, and milestones.

G&A expenses: General and administrative expenses were $10.0 million for the three months ended June 30, 2022, compared to $5.1 million for the same period in 2021. The increase was primarily due to personnel-related expenses, including stock-based compensation, attributable to increased headcount; facilities and other expenses; and legal, accounting, insurance, and other expenses associated with operating as a public company; partially offset by a decrease in patent cost reimbursements.

Net loss: Caribou reported a net loss of $26.7 million for the three months ended June 30, 2022, compared to $14.3 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.