Intellia Therapeutics, Inc. — 2022 Form 10-K
Reporting period: Fiscal year ended December 31, 2022. The filing is an annual report, not a standalone fourth-quarter report. Amounts below are in U.S. dollars; financial statement amounts are generally shown in millions unless noted.
Business context
Intellia is a clinical-stage genome-editing company developing CRISPR/Cas9-based in vivo therapies and ex vivo engineered cell therapies. It has no approved products and generated no product-sale revenue; reported revenue came from collaborations. Lead clinical programs were NTLA-2001 for ATTR amyloidosis and NTLA-2002 for hereditary angioedema (HAE).
Financial performance and liquidity
| Metric | 2022 | 2021 |
|---|---|---|
| Collaboration revenue | $52.1 million | $33.1 million |
| Research and development expense | $420.0 million | $229.8 million |
| General and administrative expense | $90.3 million | $71.1 million |
| Operating loss | $(458.2) million | $(267.9) million |
| Net loss | $(474.2) million | $(267.9) million |
| Basic and diluted loss per share | $(6.16) | $(3.78) |
| Net cash used in operating activities | $(333.3) million | $(225.0) million |
- Fourth-quarter 2022 net loss was $113.4 million. The filing text provided does not give a clear standalone fourth-quarter revenue, expense, or cash-flow breakdown.
- Cash, cash equivalents and marketable securities totaled $1.262 billion at year-end 2022, versus $1.086 billion at year-end 2021. Current assets were $1.217 billion and current liabilities $126.6 million.
- The balance sheet lists operating lease liabilities of $130.7 million, including $16.7 million current. No financial borrowings are shown in the balance sheet presented in the filing.
- Financing cash flow was $583.0 million, principally from a $337.9 million follow-on stock offering and $227.9 million net proceeds from at-the-market share sales. Common shares outstanding increased to 87.1 million from 74.5 million.
- Management expected year-end resources and collaboration cost reimbursements to fund operating and capital needs beyond 24 months, excluding potential milestones and strategic uses of capital. The financial statement note separately states funding for at least the 12 months following issuance.
Profitability and operating margins are not meaningful indicators for this loss-making development-stage company. Collaboration revenue rose, but expenses grew substantially faster; the operating loss widened by $190.3 million. R&D expense increased 83%, including $56.0 million of acquired in-process R&D from the Rewrite acquisition. Net loss also included a $13.5 million increase in the fair value of contingent consideration and an $11.1 million share of equity-method investment losses, partly offset by higher interest income.
Material changes and outlook
- NTLA-2001: Interim ATTR-CM Phase 1 data from 12 patients showed mean serum TTR reductions above 90% at day 28 for 0.7 mg/kg and 1.0 mg/kg, sustained through follow-up of four to six months. One Grade 3 infusion-related reaction was reported and resolved. The company planned an IND submission in mid-2023 and a pivotal ATTR-CM trial by year-end 2023, subject to regulatory feedback.
- NTLA-2002: Interim Phase 1/2 results showed dose-dependent plasma kallikrein reductions; at 25 mg, the reported mean HAE attack reduction was 91% through the 16-week observation period. The company selected 25 mg and 50 mg for Phase 2 evaluation and reported no serious adverse events or Grade 3-or-higher adverse events in the cited interim data.
- NTLA-3001 (AATD-associated lung disease) remained preclinical; the company planned an IND or equivalent filing in the second half of 2023. NTLA-2003 (AATD-associated liver disease) was also preclinical, with IND-enabling activities targeted for completion by year-end 2023.
- The company acquired Rewrite for approximately $45.0 million upfront and potential additional milestone payments. A $25.0 million research milestone was achieved in January 2023 and settled in February 2023 using cash and 567,045 shares.
- In November 2022, Intellia discontinued its AvenCell co-development and co-funding arrangement, leaving the program under AvenCell’s control. In February 2023, Novartis discontinued development of its CRISPR-edited HSC program targeting fetal hemoglobin for sickle cell disease.
- Management expected R&D expense to rise in 2023 as it expanded development teams, advanced pivotal trials and progressed other programs. The company has incurred losses since inception and stated it may never achieve profitability.
Risks, contingencies and unusual items
- Clinical evidence remains early-stage: interim results may change with longer follow-up and do not establish efficacy, long-term safety, regulatory approval or commercial success. Genome editing may produce unintended or irreversible effects; long-term monitoring may be required.
- CRISPR intellectual-property rights are subject to material disputes. In February 2022, the PTAB found the Broad Institute had priority over UC/Vienna/Charpentier in an interference concerning eukaryotic CRISPR/Cas9 subject matter; appeals were pending. Related ToolGen and Sigma-Aldrich proceedings also create uncertainty that could affect access to or use of key IP.
- Manufacturing and supply are complex, rely partly on third parties, and must satisfy stringent regulatory requirements. The planned Waltham GMP facility lease is expected to commence in 2024.
- Future funding needs, continued losses, share dilution, collaboration dependence, reimbursement and market acceptance, competition, cybersecurity and changing regulation are significant risks. The company reported no material new litigation in 2022, but ordinary-course legal matters and IP proceedings remain relevant.
- Lease commitments were $170.4 million for commenced leases at year-end 2022; the not-yet-commenced Waltham lease carries at least $146.0 million of expected rent over its initial 12-year term. Legally enforceable other commitments due within one year were $8.0 million.
- The auditor issued unqualified opinions on the financial statements and internal control over financial reporting. The Rewrite contingent-consideration valuation was identified as a critical audit matter because it depended on significant unobservable probability and timing assumptions.
Key facts for investors to verify
- Latest NTLA-2001 and NTLA-2002 safety, durability and clinical-endpoint data, and whether pivotal-trial timing and regulatory plans were achieved.
- Cash, marketable securities and operating cash burn against the stated runway; also reconcile management’s “beyond 24 months” estimate with the financial-statement note’s “at least 12 months” statement.
- Share-count growth and remaining capacity under the $400 million at-the-market program, as well as subsequent equity issuance and dilution.
- Progress and potential costs of the Rewrite milestone program, the Waltham facility and other lease commitments.
- Developments in CRISPR patent proceedings and the status, funding and economics of key collaborations, including Regeneron and AvenCell.