Caribou Biosciences, Inc. — FY2022 Form 10-K
Reporting period: Fiscal year ended December 31, 2022; filed March 9, 2023. This is an annual filing, not a standalone fourth-quarter report.
Business context
Caribou is a clinical-stage genome-editing biotechnology company developing off-the-shelf CAR-T and CAR-NK cell therapies using its CRISPR chRDNA platform. It has no approved products and has generated no product-sale revenue. Its four wholly owned programs are CB-010, CB-011, CB-012 and CB-020; it is also developing two CAR-T programs for AbbVie.
Financial performance and liquidity
| Metric | FY2022 | FY2021 |
|---|---|---|
| Licensing and collaboration revenue | $13.9 million | $9.6 million |
| Research and development expense | $82.2 million | $52.3 million |
| General and administrative expense | $38.0 million | $24.3 million |
| Operating loss | $106.4 million | $67.0 million |
| Net loss | $99.4 million | $66.9 million |
| Net loss per share, basic and diluted | $1.64 | $2.11 |
| Cash used in operating activities | $91.0 million | $32.5 million |
| Cash, cash equivalents and marketable securities at year-end | $317.0 million | $413.5 million |
FY2022 revenue rose $4.3 million, principally from AbbVie collaboration revenue, which increased to $8.0 million from $4.0 million. Operating expenses increased $43.7 million, reflecting expanded clinical, manufacturing, research and staffing activity. Interest income increased, and a $2.4 million gain from remeasurement of the MSKCC success-payment liability contributed to other income. FY2021 also included a $1.6 million gain from forgiveness of the PPP loan.
At December 31, 2022, total assets were $373.8 million, total liabilities $72.9 million and stockholders’ equity $300.9 million. Current assets were $260.2 million and current liabilities $28.1 million. The balance sheet reports no outstanding debt; the PPP loan had been forgiven in 2021. The company reported no product gross margin; revenue was from licenses and collaborations.
Cash used in investing activities was $93.2 million, largely reflecting purchases of marketable securities and property and equipment, partly offset by security maturities. Financing activities provided $2.1 million, mainly from employee equity plans; FY2021 financing had included IPO and preferred-stock proceeds. The company reported $45.4 million of undiscounted operating lease payments, including $3.3 million due within 12 months. It said available cash, cash equivalents and marketable securities were expected to fund its current operating plan for at least 12 months from issuance of the financial statements.
Programs, outlook and material developments
- CB-010: In the ANTLER Phase 1 trial for relapsed/refractory B-cell non-Hodgkin lymphoma, six patients at dose level 1 had a complete response as best response; three maintained a response at six months and two at 12 months, based on data through November 30, 2022. The filing reports no graft-versus-host disease; five of six patients experienced Grade 3 or 4 treatment-emergent adverse events, and one had Grade 3 ICANS characterized as a dose-limiting toxicity. Three patients treated at dose level 2 had no dose-limiting toxicities. Dose level 3 enrollment was ongoing, with an update planned for the second half of 2023. CB-010 had FDA RMAT and Fast Track designations, and orphan drug designation for follicular lymphoma.
- CB-011: The CaMMouflage Phase 1 trial for relapsed/refractory multiple myeloma had begun recruiting at dose level 1. The filing describes the clinical program as early-stage; it does not report clinical efficacy results.
- CB-012: IND-enabling studies were underway for relapsed/refractory AML, with an IND submission planned for the second half of 2023.
- CB-020: The ROR1-targeted CAR-NK program remained preclinical. Reported supportive data were from preclinical models, not human trials.
- AbbVie: AbbVie reimburses collaboration work and may owe development, regulatory and sales milestones and royalties. It may terminate the agreement for any reason on 90 days’ notice; manufacturing activities under the agreed 2023 budget were scheduled no earlier than 2024. Collaboration revenue and future payments depend on work performed and AbbVie’s decisions and progress.
Management expects continuing losses and higher research and development spending as trials and preclinical programs advance. The company may need substantial additional financing; its $400 million shelf registration included a $100 million at-the-market program, under which no shares had been sold by year-end 2022. Management cautioned that runway estimates depend on assumptions and capital could be used sooner than expected.
Risks, contingencies and unusual items
- Clinical-stage, genome-edited therapies face substantial efficacy, safety, regulatory and manufacturing uncertainty. Early CB-010 results involve a small cohort and may change with longer follow-up; the filing specifically cautions that preliminary clinical data may not predict final outcomes.
- The company relies on third-party manufacturers, suppliers and clinical research organizations, including some single-source providers. Manufacturing delays, donor-cell variability, quality issues or enrollment challenges could delay programs.
- Competition is intense in genome editing and cell therapy. Intellectual-property exposure includes ongoing administrative proceedings involving foundational CRISPR-Cas9 patents, as well as potential third-party patent claims and license obligations.
- A putative securities class action was filed February 10, 2023, alleging misleading disclosures concerning CB-010’s therapeutic durability and clinical and commercial prospects. The complaint seeks unspecified damages; the company said it would vigorously defend the case and believed it was without merit. No litigation liability was recorded as probable or reasonably estimable in the filing.
- The MSKCC agreement provides for potential success and change-of-control payments capped at $35 million in aggregate; the recorded liability was $1.7 million at year-end. The company also has contingent license milestones and royalties whose timing and likelihood were not estimable.
- Marketable securities had $1.6 million of gross unrealized losses at year-end, all in positions outstanding for less than 12 months. The company reported no impairment and no intent or requirement to sell before maturity or recovery.
Investor verification priorities
- Review the full ANTLER patient-level data, response duration, safety events and subsequent dose-level results; distinguish best response from sustained response.
- Track CB-011 enrollment and safety data, and whether CB-012’s planned IND submission and CB-020 development remain on schedule.
- Reconcile quarterly cash burn and investment maturities against management’s stated runway, and monitor any use of the shelf or ATM financing capacity and resulting dilution.
- Verify AbbVie collaboration spending, reimbursements, program timing and termination or expansion decisions.
- Follow the securities class action and CRISPR patent proceedings, including possible effects on costs, licensing revenue and freedom to operate.