Intellia Therapeutics, Inc. — 2021 Form 10-K
Reporting period: Fiscal year ended December 31, 2021. This is an annual report, not a standalone fourth-quarter filing; it reports a fourth-quarter net loss of $81.2 million. Intellia is a clinical-stage genome-editing company developing CRISPR/Cas9-based in vivo and ex vivo therapies. It had no approved products or product-sale revenue.
Financial performance and liquidity
| Metric | 2021 | 2020 |
|---|---|---|
| Collaboration revenue | $33.1 million | $58.0 million |
| Research and development expense | $229.8 million | $150.4 million |
| General and administrative expense | $71.1 million | $44.2 million |
| Operating loss | $267.9 million | $136.6 million |
| Net loss | $267.9 million | $134.2 million |
| Basic and diluted loss per share | $3.78 | $2.40 |
| Net cash used in operating activities | $225.0 million | $49.9 million |
- Revenue was entirely collaboration-related; the filing reports no product sales or product royalties.
- At December 31, 2021, cash and cash equivalents were $123.4 million and marketable securities were $962.6 million, totaling approximately $1.086 billion. Total assets were $1.294 billion; stockholders’ equity was $1.040 billion.
- The balance sheet reports no borrowings or debt. Current assets were $769.3 million versus current liabilities of $125.8 million. Deferred revenue totaled $127.2 million.
- Cash used in investing activities was $550.8 million, primarily reflecting purchases of marketable securities; financing provided $736.7 million, including $648.3 million net proceeds from a July 2021 stock offering and $45.3 million from at-the-market sales.
- No profitability or product-revenue margin is reported. The company has an accumulated deficit of $703.0 million and does not pay dividends.
Changes versus 2020
- Collaboration revenue fell $24.9 million, chiefly because 2020 included a $15.3 million hemophilia A license transfer, an $8.4 million cumulative catch-up adjustment under the Regeneron agreement, and a $5.0 million Novartis milestone. AvenCell contributed $6.1 million of revenue in 2021.
- Operating expenses rose $106.3 million, or 55%, as R&D increased 53% and general and administrative expense increased 61%. The company cited expanded staffing, clinical and manufacturing preparation, facilities, and higher stock-based compensation.
- Net loss increased to $267.9 million from $134.2 million; operating cash use rose to $225.0 million from $49.9 million. The 2020 comparison included substantial upfront and other collaboration receipts.
- Common shares outstanding increased to 74.5 million from 66.2 million. The 2021 equity issuance included the follow-on offering and at-the-market sales.
Outlook, commentary and key risks
- Management expects R&D expense to rise in 2022 as it advances NTLA-2001, NTLA-2002 and NTLA-5001 and moves additional candidates forward. It expects year-end 2021 cash, cash equivalents and marketable securities, plus specified collaboration funding and reimbursements, to cover operating and capital needs beyond 24 months; this estimate excludes potential milestones and strategic uses of capital and depends on assumptions. The financial-statement note separately states liquidity is expected to cover at least 12 months.
- NTLA-2001: interim Phase 1 data from six ATTRv-PN patients showed mean serum TTR reductions of 52% and 87% at the 0.1 mg/kg and 0.3 mg/kg dose levels, respectively; no serious adverse events were reported by day 28 in those six patients. The study remained early-stage, with additional cohorts and cardiomyopathy enrollment underway.
- NTLA-2002’s first patient was dosed in December 2021; NTLA-5001’s Phase 1/2a study began patient screening. NTLA-3001 was nominated for AATD-associated lung disease, with an IND or equivalent filing planned for 2023. These are development plans, not assured outcomes.
- Material uncertainties include unproven CRISPR-based therapies, clinical safety and efficacy, manufacturing and supply, regulatory review, patient enrollment, competition, reimbursement, and reliance on collaborators. Intellectual-property disputes and proceedings involving foundational CRISPR patents could constrain development or commercialization.
- Unusual and strategic items included a $10.0 million Novartis payment recorded as R&D expense; equity received for licenses and services that generated deferred revenue, including AvenCell, SparingVision and Kyverna arrangements; and settlement of the Caribou arbitration through a license/leaseback arrangement with an upfront payment and potential future milestones and royalties.
- After year-end, Intellia acquired Rewrite for approximately $45.0 million upfront, with up to $155.0 million in contingent milestones, and entered into substantial additional facility leases, including a planned GMP manufacturing facility. These are post-period developments, not 2021 operating results.
Important facts for investors to verify
- Review the full NTLA-2001 safety, dose-response and durability data as cohorts mature; interim results from six patients are not proof of clinical benefit or approval.
- Track enrollment, dosing, safety updates and data timing for NTLA-2001, NTLA-2002 and NTLA-5001, as well as progress toward NTLA-3001’s planned regulatory filing.
- Assess cash burn and runway against expanding R&D costs, lease commitments, the Rewrite acquisition and any future financing or dilution.
- Check collaboration economics, deferred-revenue recognition, equity-investment valuations and the timing and conditions of potential milestones and royalties.
- Monitor CRISPR patent proceedings and license rights, manufacturing readiness, regulatory developments, and any material changes to management’s liquidity outlook.
The independent auditor issued an unqualified opinion on the financial statements and internal control over financial reporting; management and the auditor concluded internal controls were effective as of December 31, 2021.