Intellia Therapeutics, Inc. (NASDAQ:NTLA) BofA Securities 2025 Healthcare Conference May 13, 2025 7:20 PM ET Company Participants Ed Dulac - CFO Conference Call Participants Alec Stranahan - BofA Securities Alec Stranahan Hello, everyone. Thanks for joining the session with Intellia Therapeutics on day 1 of the 2025 Bank of America Healthcare Conference. My name is Alec Stranahan. I'm senior biotech analyst covering Intellia here at BofA. And I have the pleasure of being joined by Ed Dulac, the Chief Financ ...

Summary of Intellia Therapeutics Conference Call Company Overview - **Company**: Intellia Therapeutics - **Industry**: Gene Editing and Biotechnology - **Focus**: In vivo applications of gene editing with a focus on late-stage programs and clinical studies [4][5] Key Assets and Programs - **Phase III Studies**: Two assets with three Phase III studies in progress, targeting different indications [4][5] - **ATTR Program**: Focused on TTR (transthyretin) amyloidosis with two Phase III studies, one for Polyneuropathy (PN) and one for Centimeters [6][8] - **HAE Program**: A pivotal Phase III program for Hereditary Angioedema (HAE) with aggressive enrollment and a timeline for updates [33][36] Market Dynamics and Competitive Landscape - **ATTR Market**: Increasing diagnosis rates and a high level of unmet need despite existing therapies. The company believes its approach to significantly reduce TTR levels will provide a competitive edge [6][7][10] - **Centimeters Opportunity**: The company is optimistic about the trajectory of the Centimeters opportunity, with strong enrollment momentum and investigator enthusiasm [8][10] - **HAE Market**: Aiming for a paradigm shift from chronic therapy to a one-time treatment, targeting zero attacks and no ongoing therapy for patients [37][38] Clinical Data and Efficacy - **TTR Knockdown**: The company has demonstrated consistent TTR knockdown in Phase I studies, with a nadir reached in 28 days [16][22] - **Durability of Response**: Emphasis on the importance of durability in treatment response, with ongoing data collection to support long-term efficacy [11][13][48] - **Functional Endpoints**: The studies are tracking various functional endpoints, including mortality, hospitalization, and quality of life metrics [18][19] Financial and Operational Strategy - **Cash Position**: The company has a cash balance exceeding $700 million, which is expected to support the execution of Phase III programs and commercial infrastructure development [52][54] - **Restructuring**: A recent restructuring aimed at focusing on key research areas while ensuring financial stability and operational efficiency [53][55] - **Market Strategy**: Plans to build the company through successful launches in smaller markets before tackling larger opportunities [25][27] Regulatory and Market Engagement - **FDA Interactions**: The company maintains a positive relationship with the FDA, running gold standard randomized placebo-controlled studies, which are expected to align with regulatory expectations [58][59] - **Market Research**: Ongoing market research to understand patient and physician perspectives, which will inform commercial strategies [35][36] Future Outlook - **Upcoming Milestones**: Anticipated data releases and updates on clinical trials over the next 12 to 18 months, with a focus on building a robust safety database and demonstrating treatment efficacy [30][56] - **Long-term Vision**: The company aims to leverage its gene editing technology to provide significant benefits to patients and the healthcare system, with a focus on sustainable growth and value creation [11][31][39]

Core Insights - The article discusses the author's transition to independent investment research after over 43 years in the industry, emphasizing a focus on actionable investment insights rather than adhering to external agendas [1] - The author specializes in rules and factor-based equity investing strategies, combining quantitative analysis with fundamental analysis to derive investment stories that predict future performance [1] - The author has extensive experience across various market segments, including large cap, small cap, micro cap, value, growth, and income stocks, as well as managing a high-yield fixed-income fund [1] Group 1 - The author aims to provide the best actionable investment insights without being influenced by any product agenda [1] - The approach taken is to use numbers as a tool to inspire human intelligence-driven investment narratives rather than relying solely on statistical studies [1] - The author has a background in investor education, having conducted seminars and authored books on stock selection and analysis [1] Group 2 - The author has developed and worked with various quantitative models and has experience in quantitative asset allocation strategies, which are foundational to modern Robo Advising [1] - The article highlights the author's previous roles, including editing stock newsletters and serving as an assistant research director at Value Line [1] - The author expresses openness to feedback and criticism from readers, indicating a willingness to engage with the investment community [1]

Core Viewpoint - Intellia Therapeutics reported a narrower loss per share in Q1 2025 compared to estimates, despite a significant decline in revenues year-over-year, primarily due to lower collaboration revenues from AvenCell [1][2]. Financial Performance - The company incurred a loss of $1.10 per share in Q1 2025, which was better than the Zacks Consensus Estimate of a loss of $1.26 and a slight improvement from a loss of $1.12 per share in the same quarter last year [1]. - Total revenues for Q1 2025 were $16.6 million, reflecting a 42.6% decline year-over-year, but exceeded the Zacks Consensus Estimate of $15 million [2]. - Research and development expenses were $108.4 million, down 3% from the previous year, attributed to lower employee-related expenses and stock-based compensation [4]. - General and administrative expenses decreased by approximately 6.5% year-over-year to $29 million, also due to reduced stock-based compensation and workforce [4]. - As of March 31, 2025, the company had cash, cash equivalents, and marketable securities totaling $707.1 million, down from $861.7 million at the end of 2024, which is expected to fund operations into the first half of 2027 [5]. Stock Performance - Shares of Intellia rose by 13.6% on May 8 following the better-than-expected results, although the stock has declined 28.9% year-to-date compared to an 8% decline in the industry [3]. Pipeline Developments - Intellia is collaborating with Regeneron Pharmaceuticals on the investigational in vivo genome-editing candidate, nexiguran ziclumeran (nex-z), which is being studied for ATTR amyloidosis indications [8]. - The phase III MAGNITUDE study for nex-z in patients with ATTR-CM is ongoing, with the FDA granting RMAT designation for this treatment in March [9]. - The first patient was dosed in the phase III MAGNITUDE 2 study for ATTRv-PN in April, with enrollment expected to complete in 2026 [10]. - Another candidate, NTLA-2002, is in the pivotal phase III HAELO study for hereditary angioedema, with the first patient dosed in January 2025 and expected enrollment completion in Q3 2025 [11]. Company Ranking - Intellia currently holds a Zacks Rank 2 (Buy), indicating a favorable outlook compared to other biotech stocks [12].

```markdown PART I - FINANCIAL INFORMATION [Item 1. Financial Statements (unaudited)](index=3&type=section&id=Item%201.%20Financial%20Statements%20(unaudited)) This section presents Intellia Therapeutics' unaudited condensed consolidated financial statements for Q1 2025 and 2024, covering balance sheets, statements of operations, stockholders' equity, cash flows, and detailed notes on accounting policies, investments, and other financial aspects [Condensed Consolidated Balance Sheets](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheets%20as%20of%20March%2031%2C%202025%20and%20December%2031%2C%202024) The Condensed Consolidated Balance Sheets show a decrease in total assets and stockholders' equity from December 31, 2024, to March 31, 2025, primarily driven by a reduction in cash, cash equivalents, and marketable securities, alongside a decrease in total liabilities Condensed Consolidated Balance Sheet Highlights (Amounts in thousands) | Metric | March 31, 2025 | December 31, 2024 | Change (2025 vs 2024) | Percentage Change | | :-------------------------------- | :------------- | :---------------- | :-------------------- | :------------------ | | Cash and cash equivalents | $126,880 | $189,182 | $(62,302) | -32.93% | | Marketable securities | $376,867 | $412,333 | $(35,466) | -8.60% | | Total current assets | $551,392 | $639,863 | $(88,471) | -13.83% | | Total assets | $986,163 | $1,191,015 | $(204,852) | -17.20% | | Total current liabilities | $112,593 | $110,851 | $1,742 | 1.57% | | Total liabilities | $206,244 | $319,059 | $(112,815) | -35.36% | | Total stockholders' equity | $779,919 | $871,956 | $(92,037) | -10.56% | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=5&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss%20for%20the%20Three%20Months%20Ended%20March%2031%2C%202025%20and%202024) For the three months ended March 31, 2025, Intellia Therapeutics reported a net loss of **$114.3 million**, an increase from **$107.4 million** in the prior year period, primarily due to decreased collaboration revenue and interest income, partially offset by reduced operating expenses and a favorable change in investment fair value Condensed Consolidated Statements of Operations and Comprehensive Loss (Amounts in thousands, except per share data) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | Change (2025 vs 2024) | Percentage Change | | :---------------------------------- | :-------------------------------- | :-------------------------------- | :-------------------- | :------------------ | | Collaboration revenue | $16,627 | $28,935 | $(12,308) | -42.54% | | Research and development | $108,427 | $111,847 | $(3,420) | -3.06% | | General and administrative | $29,007 | $31,091 | $(2,084) | -6.70% | | Total operating expenses | $137,434 | $142,938 | $(5,504) | -3.85% | | Operating loss | $(120,807) | $(114,003) | $(6,804) | 5.97% | | Interest income | $8,603 | $12,632 | $(4,029) | -31.90% | | Change in fair value of investments | $(2,125) | $(6,065) | $3,940 | -64.96% | | Net loss | $(114,329) | $(107,436) | $(6,893) | 6.42% | | Net loss per share, basic and diluted | $(1.10) | $(1.12) | $0.02 | -1.79% | | Weighted average shares outstanding | 103,500 | 95,502 | 7,998 | 8.37% | [Condensed Consolidated Statements of Stockholders' Equity](index=6&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity%20for%20the%20Three%20Months%20Ended%20March%2031%2C%202025%20and%202024) Total stockholders' equity decreased from **$871.9 million** at December 31, 2024, to **$779.9 million** at March 31, 2025, primarily due to the net loss incurred, partially offset by stock-based compensation and unrealized gains on marketable securities Key Changes in Stockholders' Equity (Amounts in thousands) | Metric | Balance at Dec 31, 2024 | Stock-based Compensation | Other Comprehensive Income | Net Loss | Balance at Mar 31, 2025 | | :----------------------- | :---------------------- | :----------------------- | :------------------------- | :------- | :---------------------- | | Additional Paid-In Capital | $3,048,741 | $21,840 | - | - | $3,070,581 | | Accumulated Other Comprehensive Income | $605 | - | $452 | - | $1,057 | | Accumulated Deficit | $(2,177,400) | - | - | $(114,329) | $(2,291,729) | | Total Stockholders' Equity | $871,956 | $21,840 | $452 | $(114,329) | $779,919 | [Condensed Consolidated Statements of Cash Flows](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20for%20the%20Three%20Months%20Ended%20March%2031%2C%202025%20and%202024) Net cash used in operating activities increased to **$148.9 million** in Q1 2025 from **$120.7 million** in Q1 2024, while investing activities provided **$94.3 million**, and financing activities provided no cash in 2025 Condensed Consolidated Statements of Cash Flows (Amounts in thousands) | Cash Flow Activity | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | Change (2025 vs 2024) | | :-------------------------------- | :-------------------------------- | :-------------------------------- | :-------------------- | | Net cash used in operating activities | $(148,930) | $(120,662) | $(28,268) | | Net cash provided by investing activities | $94,328 | $5,901 | $88,427 | | Net cash provided by financing activities | $- | $59,992 | $(59,992) | | Net decrease in cash, cash equivalents and restricted cash equivalents | $(54,602) | $(54,769) | $167 | | Cash, cash equivalents and restricted cash equivalents, end of period | $148,185 | $185,584 | $(37,399) | [Notes to Condensed Consolidated Financial Statements](index=8&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) These notes detail Intellia's financial reporting, covering its business overview, accounting policies, marketable securities, fair value measurements, accrued expenses, legal proceedings, collaborations, investments, leases, stock-based compensation, loss per share, stockholders' equity, and segment information [1. Overview and Basis of Presentation](index=8&type=section&id=1.%20Overview%20and%20Basis%20of%20Presentation) Intellia Therapeutics is a clinical-stage gene editing company focused on CRISPR-based therapies, expecting its cash, cash equivalents, and marketable securities as of March 31, 2025, to fund operations for at least the next twelve months - Intellia Therapeutics is a clinical-stage gene editing company leveraging CRISPR-based therapies for in vivo (genetic diseases) and ex vivo (immuno-oncology and autoimmune diseases) applications[19](index=19&type=chunk) - The company expects its cash, cash equivalents, and marketable securities as of March 31, 2025, to fund operations for at least the twelve-month period following the issuance of these financial statements[25](index=25&type=chunk) [2. Summary of Significant Accounting Policies](index=9&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) No material changes occurred to significant accounting policies during Q1 2025, with the Company evaluating the impact of new accounting pronouncements ASU No. 2023-09 and ASU No. 2024-03 - No material changes to significant accounting policies during the three months ended March 31, 2025[26](index=26&type=chunk) - The Company is evaluating the impact of ASU No. 2023-09 (Income Taxes) effective January 1, 2025, and ASU No. 2024-03 (Expense Disaggregation Disclosures) effective January 1, 2027[27](index=27&type=chunk)[28](index=28&type=chunk) [3. Marketable Securities](index=9&type=section&id=3.%20Marketable%20Securities) Available-for-sale marketable securities decreased from **$672.5 million** at December 31, 2024, to **$580.2 million** at March 31, 2025, primarily due to a reduction in U.S. Treasury and financial institution debt securities Available-for-Sale Marketable Securities (Amounts in thousands) | Category | March 31, 2025 (Estimated Fair Value) | December 31, 2024 (Estimated Fair Value) | Change | | :---------------------------------- | :------------------------------------ | :------------------------------------- | :------- | | U.S. Treasury and other government-backed securities | $293,711 | $352,616 | $(58,905) | | Financial institution debt securities | $186,243 | $217,827 | $(31,584) | | Corporate debt securities | $100,266 | $94,927 | $5,339 | | Other asset-backed securities | $- | $7,178 | $(7,178) | | Total | $580,220 | $672,548 | $(92,328) | - The Company generally does not intend to sell investments prior to recovery of their amortized cost basis for any investment in an unrealized loss position, classifying these losses as temporary[29](index=29&type=chunk) [4. Fair Value Measurements](index=11&type=section&id=4.%20Fair%20Value%20Measurements) As of March 31, 2025, total financial assets recognized at fair value on a recurring basis were **$658.5 million**, primarily marketable securities and cash equivalents, with a significant portion classified as Level 2 inputs Financial Assets Recognized at Fair Value (March 31, 2025, in thousands) | Asset Category | Total | Level 1 | Level 2 | Level 3 | | :---------------------------------- | :------ | :------ | :------ | :------ | | Cash equivalents and restricted cash equivalents | $76,058 | $76,058 | $- | $- | | Marketable securities: | | | | | | U.S. Treasury and other government-backed securities | $293,711 | $153,244 | $140,467 | $- | | Financial institution debt securities | $186,243 | $- | $186,243 | $- | | Corporate debt securities | $100,266 | $- | $100,266 | $- | | Total marketable securities | $580,220 | $153,244 | $426,976 | $- | | Investment in Kyverna Therapeutics, Inc. | $2,265 | $2,265 | $- | $- | | Total assets | $658,543 | $231,567 | $426,976 | $- | - The investment in Kyverna Therapeutics, Inc. is classified as Level 1, valued at a quoted market price in an active market[35](index=35&type=chunk) - Investments in SparingVision SAS and AvenCell Therapeutics, Inc. are accounted for using the measurement alternative at cost minus impairment, adjusted for changes in observable prices, with carrying values of **$14.6 million** and **$7.9 million** respectively as of March 31, 2025[36](index=36&type=chunk)[37](index=37&type=chunk) [5. Accrued Expenses](index=12&type=section&id=5.%20Accrued%20Expenses) Accrued expenses decreased to **$40.8 million** at March 31, 2025, from **$55.4 million** at December 31, 2024, primarily due to reduced employee compensation and benefits following a January 2025 strategic restructuring Accrued Expenses (Amounts in thousands) | Category | March 31, 2025 | December 31, 2024 | Change | | :------------------------------ | :------------- | :---------------- | :------- | | Accrued research and development | $24,732 | $26,362 | $(1,630) | | Employee compensation and benefits | $9,845 | $24,075 | $(14,230) | | Accrued legal and professional expenses | $3,201 | $2,845 | $356 | | Accrued other | $3,027 | $2,073 | $954 | | Total accrued expenses | $40,805 | $55,355 | $(14,550) | - In January 2025, the Company implemented a strategic restructuring, reducing its employee headcount by approximately **27%** and recognizing **$6.5 million** in restructuring charges for severance and termination-related costs[38](index=38&type=chunk) [6. Commitments and Contingencies](index=12&type=section&id=6.%20Commitments%20and%20Contingencies) The Company is involved in ongoing legal proceedings, including patent infringement and a stockholder lawsuit, and has contingent payment obligations of up to **$130.0 million** related to Rewrite Therapeutics, Inc. - BlueAllele Corp. filed a patent infringement complaint against Intellia on July 8, 2024, alleging infringement of bi-directional insertion template technology; the court denied Intellia's motion to dismiss, and discovery has begun[40](index=40&type=chunk) - A stockholder lawsuit, Gonzalez v. Intellia Therapeutics, Inc., was filed on February 11, 2025, alleging claims under the Securities Exchange Act of 1934 related to the NTLA-3001 program; the Company intends to defend vigorously[41](index=41&type=chunk) - Contingent payments of up to **$130.0 million** are related to Rewrite Therapeutics, Inc., including **$100.0 million** upon regulatory approval and **$30.0 million** upon pre-specified research milestones; the satisfaction and timing of these payments are uncertain[43](index=43&type=chunk) [7. Collaborations and Other Arrangements](index=13&type=section&id=7.%20Collaborations%20and%20Other%20Arrangements) Collaboration revenue decreased by **$12.3 million** in Q1 2025 compared to Q1 2024, primarily due to reduced revenue from the AvenCell license and collaboration agreement, with key collaborations including Regeneron and SparingVision Collaboration Accounts Receivable and Contract Liabilities (Amounts in thousands) | Metric | Balance at Beginning of Period (Dec 31, 2024) | Additions | Deductions | Balance at End of Period (Mar 31, 2025) | | :-------------------------------- | :------------------------------------------ | :-------- | :--------- | :-------------------------------------- | | Accounts receivable | $8,517 | $10,203 | $(8,216) | $10,504 | | Contract liabilities - deferred revenue | $38,917 | $- | $(5,095) | $33,822 | - Collaboration revenue decreased by **$12.3 million**, primarily due to a reduction in revenue under the AvenCell license and collaboration agreement[135](index=135&type=chunk) - Regeneron exercised its option to extend the technology collaboration term until April 2026, with a **$30.0 million** nonrefundable payment received in April 2024; a development milestone for the hemophilia B program was achieved in March 2025, recognizing **$1.8 million** in revenue[47](index=47&type=chunk)[48](index=48&type=chunk) - The Company recognized **$7.0 million** in collaboration revenue from the ATTR Co/Co agreement with Regeneron in Q1 2025, up from **$4.7 million** in Q1 2024[51](index=51&type=chunk) - Revenue from SparingVision LCA was **$0** for Q1 2025, down from **$0.5 million** in Q1 2024; deferred revenue for SparingVision remains at **$12.5 million**[57](index=57&type=chunk) - The Company recognized **$1.0 million** in revenue related to material shipments under the AvenCell LCA during Q1 2025[61](index=61&type=chunk) [8. Investments and Other Assets](index=15&type=section&id=8.%20Investments%20and%20Other%20Assets) Total investments and other assets increased to **$68.3 million** at March 31, 2025, from **$44.3 million** at December 31, 2024, primarily due to a **$26.8 million** increase in prepaid rent for the Tech Square Lease Investments and Other Assets (Amounts in thousands) | Category | March 31, 2025 | December 31, 2024 | Change | | :-------------------------------- | :------------- | :---------------- | :------- | | Investment in Kyverna | $2,265 | $4,390 | $(2,125) | | Prepaid rent Tech Square Lease, long-term | $26,804 | $- | $26,804 | | Other investments | $22,481 | $22,481 | $- | | Restricted cash equivalents, long-term | $13,776 | $13,605 | $171 | | Prepaid expenses and other assets, long-term | $2,990 | $3,788 | $(798) | | Total investments and other assets | $68,316 | $44,264 | $24,052 | - The Company's equity interest in AvenCell Therapeutics, Inc. is now accounted for under ASC 321 (Investments in Equity Securities) due to loss of significant influence, with a carrying value of **$7.9 million** as of March 31, 2025[64](index=64&type=chunk)[65](index=65&type=chunk) [9. Leases](index=16&type=section&id=9.%20Leases) The Company amended its 840 Winter Lease, incurring **$78.0 million** in modification payments, and entered a new Tech Square Lease for 101,000 square feet, with total consideration for the combined contract at **$244.4 million** - The 840 Winter Lease was amended to terminate by June 30, 2028, with lease modification payments totaling **$78.0 million**[66](index=66&type=chunk) - A new Tech Square Lease was signed for approximately 101,000 square feet, with an initial term of twelve years and three months, and base rent expected to be **$108.00** per square foot per year[67](index=67&type=chunk) - The total consideration for the combined Winter Street Amendment and Tech Square Lease contract is **$244.4 million**, with **$52.1 million** allocated to 840 Winter and **$192.3 million** to 400 Tech Square[68](index=68&type=chunk)[69](index=69&type=chunk) - As of March 31, 2025, the Company recorded **$26.8 million** of prepaid rent related to the Tech Square component within 'Investments and other assets'[71](index=71&type=chunk) Future Operating Lease Payments (in thousands) | Year Ending December 31, | Amount | | :----------------------- | :----- | | Remainder of 2025 | $31,489 | | 2026 | $32,219 | | 2027 | $24,125 | | 2028 | $15,637 | | 2029 | $12,666 | | Thereafter | $26,593 | | Total lease payments | $142,729 | | Less: imputed interest | $(23,476) | | Total operating lease liabilities at March 31, 2025 | $119,253 | [10. Stock-Based Compensation](index=17&type=section&id=10.%20Stock-Based%20Compensation) Stock-based compensation expense decreased by **$12.3 million** to **$21.8 million** in Q1 2025, primarily due to a reduced workforce and lower stock price, with 2,240,207 RSUs and 640,751 stock options granted Stock-Based Compensation Expense (Amounts in thousands) | Category | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | Change | | :------------------------ | :-------------------------------- | :-------------------------------- | :------- | | Research and development | $12,626 | $20,161 | $(7,535) | | General and administrative | $9,214 | $14,015 | $(4,801) | | Total | $21,840 | $34,176 | $(12,336) | Restricted Stock Unit (RSU) Activity (Three Months Ended March 31, 2025) | Metric | Number of Shares | Weighted Average Grant Date Fair Value per Share | | :---------------------------------- | :--------------- | :----------------------------------------------- | | Unvested RSUs as of Dec 31, 2024 | 5,547,530 | $39.13 | | Granted | 2,240,207 | $10.67 | | Vested | (1,510,091) | $42.85 | | Cancelled | (752,172) | $43.45 | | Unvested RSUs as of Mar 31, 2025 | 5,525,474 | $25.99 | Stock Option Activity (Three Months Ended March 31, 2025) | Metric | Number of Options | Weighted Average Exercise Price per Share | | :-------------------------- | :---------------- | :---------------------------------------- | | Outstanding at Dec 31, 2024 | 5,412,786 | $47.27 | | Granted | 640,751 | $10.09 | | Forfeited | (150,340) | $86.88 | | Outstanding at Mar 32, 2025 | 5,903,197 | $42.23 | | Exercisable at Mar 31, 2025 | 4,504,464 | $47.42 | - As of March 31, 2025, there was **$90.0 million** of unrecognized stock-based compensation expense related to RSUs, expected to be recognized over a weighted average remaining vesting period of **1.69 years**[87](index=87&type=chunk) - As of March 31, 2025, there was **$16.6 million** of unrecognized compensation cost related to stock options, expected to be recognized over a weighted average remaining vesting period of **1.23 years**[93](index=93&type=chunk) [11. Loss Per Share](index=21&type=section&id=11.%20Loss%20Per%20Share) Basic and diluted net loss per share for Q1 2025 was **$(1.10)**, an improvement from **$(1.12)** in the prior year, despite an increased net loss, due to a higher weighted average number of shares outstanding Loss Per Share Calculation (Amounts in thousands) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :---------------------------------- | :-------------------------------- | :-------------------------------- | | Net loss | $(114,329) | $(107,436) | | Weighted average shares outstanding, basic and diluted | 103,500 | 95,502 | | Net loss per share, basic and diluted | $(1.10) | $(1.12) | Anti-Dilutive Common Stock Equivalents (in thousands) | Category | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------ | :-------------------------------- | :-------------------------------- | | Unvested restricted stock units | 5,525 | 5,695 | | Stock options | 5,903 | 5,910 | | Total | 11,428 | 11,605 | [12. Stockholders' Equity](index=23&type=section&id=12.%20Stockholders'%20Equity) As of March 31, 2025, the Company had 103,583,394 shares of common stock outstanding, with **$249.1 million** in shares remaining eligible for sale under the amended 2022 Sale Agreement - The number of shares outstanding of the registrant's common stock as of April 30, 2025: **103,583,394 shares**[4](index=4&type=chunk) - The 2022 Sale Agreement was amended in February 2024 to increase the at-the-market offering program size from **$400.0 million** to **$750.0 million**; no shares were issued under this program during Q1 2025[100](index=100&type=chunk) - As of March 31, 2025, **$249.1 million** in shares of common stock remain eligible for sale under the 2022 Sale Agreement, as amended[100](index=100&type=chunk) [13. Segment Information](index=23&type=section&id=13.%20Segment%20Information) The Company operates as a single segment focused on gene editing-based therapies, with collaboration revenue decreasing to **$16.6 million** in Q1 2025 from **$28.9 million** in Q1 2024, and total R&D expenses decreasing to **$108.4 million** - The Company has identified one operating and reportable segment: the development of gene editing-based therapies[101](index=101&type=chunk) Reportable Segment Financial Information (Amounts in thousands) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :------------------------ | :-------------------------------- | :-------------------------------- | | Collaboration revenue | $16,627 | $28,935 | | Total research and development | $108,427 | $111,847 | | General and administrative | $29,007 | $31,091 | | Interest income | $(8,603) | $(12,632) | | Other segment information (Change in fair value of investments) | $2,125 | $6,065 | | Segment and consolidated net loss | $(114,329) | $(107,436) | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=25&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses Intellia Therapeutics' financial condition and results for Q1 2025, focusing on CRISPR-based therapies, pipeline progress, decreased collaboration revenue, net loss, and liquidity, projecting funding into H1 2027 [Management Overview](index=26&type=section&id=Management%20Overview) Intellia is a clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, with all revenue derived from collaboration agreements - Intellia is a leading clinical-stage gene editing company focused on CRISPR-based therapies, developing in vivo applications for genetic diseases and ex vivo applications for immuno-oncology and autoimmune diseases[108](index=108&type=chunk) - The company's strategy is to develop and commercialize product candidates and further its gene editing technology, with all revenue currently derived from collaboration agreements[113](index=113&type=chunk) [Our Pipeline](index=27&type=section&id=Our%20Pipeline) Intellia's pipeline features lead in vivo programs, nex-z for ATTR amyloidosis and NTLA-2002 for HAE, both in Phase 3 clinical trials, with key milestones achieved and BLA submission targeted for H2 2026 for NTLA-2002 - NTLA-2002, a wholly owned in vivo CRISPR-based therapy for HAE, initiated its global Phase 3 HAELO study in October 2024, with the first patient dosed in January 2025; enrollment is expected to complete in Q3 2025, with BLA submission targeted for H2 2026[117](index=117&type=chunk)[118](index=118&type=chunk) - Nex-z, an investigational CRISPR-based therapy for ATTR amyloidosis, is in pivotal Phase 3 MAGNITUDE trials for ATTR-CM and ATTRv-PN; the ATTR-CM trial is enrolling, expecting over 550 patients by end of 2025; the ATTRv-PN trial received FDA IND clearance in November 2024, with the first patient dosed in April 2025, and enrollment expected to complete in 2026[121](index=121&type=chunk)[123](index=123&type=chunk)[125](index=125&type=chunk) - Nex-z received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. FDA for the treatment of ATTR-CM in March 2025[124](index=124&type=chunk) [Collaborations and Other Arrangements](index=29&type=section&id=Collaborations%20and%20Other%20Arrangements) Intellia maintains strategic alliances with collaborators like Regeneron, AvenCell, and SparingVision to accelerate the development and commercialization of CRISPR/Cas9-based products - The Company has formed strategic alliances with collaborators including Regeneron, AvenCell Therapeutics, Inc., SparingVision SAS, Kyverna Therapeutics, Inc., ONK Therapeutics, Ltd., and ReCode Therapeutics, Inc. to accelerate gene editing product development[128](index=128&type=chunk) [Financial Overview](index=29&type=section&id=Financial%20Overview) The Company's revenue is primarily collaboration-based, with research and development expenses covering compensation, facility costs, and clinical trials, while general and administrative expenses include executive compensation and professional fees - Collaboration revenue includes upfront technology access payments, fees, research funding, and milestone payments[129](index=129&type=chunk) - Research and development expenses cover compensation, facility costs, license/milestone fees, and contract services for R&D activities[130](index=130&type=chunk) - General and administrative expenses primarily consist of compensation for executive, finance, legal, HR, and business development functions, along with professional fees[131](index=131&type=chunk) - Other income (expense), net, includes interest income from cash, cash equivalents, and marketable securities, and changes in the fair value of investments[132](index=132&type=chunk) [Results of Operations](index=31&type=section&id=Results%20of%20Operations) For Q1 2025, Intellia reported a net loss of **$114.3 million**, an increase from **$107.4 million** in the prior year, driven by decreased collaboration revenue and interest income, partially offset by reduced operating expenses Summary of Results of Operations (Amounts in thousands) | Metric | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | Period-to-Period Change | | :---------------------------------- | :-------------------------------- | :-------------------------------- | :---------------------- | | Collaboration revenue | $16,627 | $28,935 | $(12,308) | | Research and development | $108,427 | $111,847 | $(3,420) |\ | General and administrative | $29,007 | $31,091 | $(2,084) | | Total operating expenses | $137,434 | $142,938 | $(5,504) | | Operating loss | $(120,807) | $(114,003) | $(6,804) | | Interest income | $8,603 | $12,632 | $(4,029) | | Change in fair value of investments, net | $(2,125) | $(6,065) | $3,940 | | Total other income, net | $6,478 | $6,567 | $(89) | | Net loss | $(114,329) | $(107,436) | $(6,893) | - Collaboration revenue decreased by **$12.3 million**, primarily due to a reduction in revenue under the AvenCell license and collaboration agreement[135](index=135&type=chunk) - Research and development expenses decreased by **$3.4 million**, driven by lower employee-related expenses and stock-based compensation due to a workforce reduction, partially offset by increased external costs for nex-z and NTLA-2002 development[136](index=136&type=chunk)[142](index=142&type=chunk) - General and administrative expenses decreased by **$2.1 million**, mainly due to lower employee-related expenses and stock-based compensation from the January 2025 workforce reduction, partially offset by severance expenses[138](index=138&type=chunk) - Other income, net, decreased by **$0.1 million**, primarily due to a **$4.0 million** decrease in interest income, partially offset by a **$3.9 million** decrease in expense from the change in fair value of investments[139](index=139&type=chunk) [Liquidity and Capital Resources](index=32&type=section&id=Liquidity%20and%20Capital%20Resources) As of March 31, 2025, Intellia had **$707.1 million** in cash, cash equivalents, and marketable securities, expected to fund operations into the first half of 2027, with substantial future funding requirements anticipated - As of March 31, 2025, the Company had **$707.1 million** in cash, cash equivalents, and marketable securities[141](index=141&type=chunk) - The Company expects its cash, cash equivalents, and marketable securities as of March 31, 2025, along with collaboration funding, to fund operating expenses and capital expenditure requirements into the first half of 2027[146](index=146&type=chunk) - Primary uses of capital include R&D research materials, clinical trial costs, compensation, and facility expenses; expenses are expected to decrease in 2025 due to a strategic reorganization focusing on high-value programs like NTLA-2002 and nex-z[144](index=144&type=chunk) - The Company may need to raise additional capital through equity financings, collaboration arrangements, or other sources, as it has no committed external source of liquidity[145](index=145&type=chunk) [Cash Flows](index=36&type=section&id=Cash%20Flows) Net cash used in operating activities increased to **$148.9 million** in Q1 2025, while net cash provided by investing activities significantly increased to **$94.3 million**, and financing activities provided no cash Summary of Cash Flows (Amounts in thousands) | Cash Flow Activity | Three Months Ended March 31, 2025 | Three Months Ended March 31, 2024 | | :-------------------------------- | :-------------------------------- | :-------------------------------- | | Net cash used in operating activities | $(148,930) | $(120,662) | | Net cash provided by investing activities | $94,328 | $5,901 | | Net cash provided by financing activities | $- | $59,992 | - Net cash used in operating activities in Q1 2025 included approximately **$51.0 million** of non-recurring cash payments associated with portfolio prioritization, workforce reduction, and real estate consolidation[149](index=149&type=chunk) - Net cash provided by investing activities in Q1 2025 was primarily due to **$95.1 million** in maturities of marketable securities (net of purchases)[151](index=151&type=chunk) - Net cash provided by financing activities in Q1 2024 included **$58.0 million** from at-the-market offerings and **$2.0 million** from stock option exercises[153](index=153&type=chunk) [Critical Accounting Policies](index=36&type=section&id=Critical%20Accounting%20Policies) The Company's critical accounting policies, primarily related to revenue recognition, accrued R&D expenses, and stock-based compensation, remain unchanged from its 2024 Annual Report - No changes to critical accounting policies from the Annual Report for the year ended December 31, 2024[154](index=154&type=chunk) - Most critical accounting policies relate to revenue recognition, accrued research and development expenses, and stock-based compensation[154](index=154&type=chunk) [Recent Accounting Pronouncements](index=36&type=section&id=Recent%20Accounting%20Pronouncements) For information on recent accounting pronouncements applicable to the Company's business, refer to Note 2, 'Summary of Significant Accounting Policies,' in the condensed consolidated financial statements - Refer to Note 2 for a description of recent accounting pronouncements applicable to the business[155](index=155&type=chunk) [Contractual Obligations](index=37&type=section&id=Contractual%20Obligations) No material changes occurred to the Company's contractual obligations during Q1 2025, other than those described in Note 9, 'Leases,' in the notes to condensed consolidated financial statements - No material changes to contractual obligations during Q1 2025, except as described in Note 9, 'Leases'[156](index=156&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=37&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Intellia's primary market risk exposure is interest rate sensitivity, affecting its **$656.3 million** in cash equivalents and marketable securities, with no material impact expected from a 100 basis point interest rate change - As of March 31, 2025, the Company had **$656.3 million** in cash equivalents, restricted cash equivalents, and marketable securities, primarily exposed to interest rate sensitivity[157](index=157&type=chunk) - Due to the short-term duration and low-risk profile of investments, a 100 basis point change in interest rates is not expected to materially affect the fair market value of the investment portfolio[157](index=157&type=chunk) - The Company does not have foreign currency or derivative financial instruments and believes inflation did not materially affect Q1 2025 results[158](index=158&type=chunk) [Item 4. Controls and Procedures.](index=37&type=section&id=Item%204.%20Controls%20and%20Procedures.) Management concluded that Intellia's disclosure controls and procedures were effective at a reasonable assurance level as of March 31, 2025, with no material changes in internal control over financial reporting during the quarter - Disclosure controls and procedures were evaluated as effective at the reasonable assurance level as of March 31, 2025[160](index=160&type=chunk) - No material changes in internal control over financial reporting occurred during the three months ended March 31, 2025[161](index=161&type=chunk) PART II - OTHER INFORMATION [Item 1. Legal Proceedings](index=38&type=section&id=Item%201.%20Legal%20Proceedings) Intellia Therapeutics is involved in ongoing legal proceedings, including a patent infringement lawsuit by BlueAllele Corp. and a stockholder lawsuit by Gonzalez, both of which the company intends to vigorously defend - BlueAllele Corp. filed a patent infringement complaint against Intellia on July 8, 2024, alleging infringement of bi-directional insertion template technology; Intellia's motion to dismiss was denied, and discovery has begun[163](index=163&type=chunk) - A stockholder lawsuit, Gonzalez v. Intellia Therapeutics, Inc., was filed on February 11, 2025, alleging claims under Sections 10(b) and 20(a) and Rule 10b-5 of the Securities Exchange Act of 1934 related to the NTLA-3001 program[164](index=164&type=chunk) [Item 1A. Risk Factors](index=38&type=page&id=Item%201A.%20Risk%20Factors) Investing in Intellia's common stock involves significant risks, including the unproven nature of CRISPR/Cas9 technology, lengthy clinical development, market non-acceptance, intense competition, intellectual property disputes, and substantial funding requirements [Summary of the Material Risks Associated with Our Business](index=38&type=section&id=Summary%20of%20the%20Material%20Risks%20Associated%20with%20Our%20Business) Key risks include the nascent clinical validation of CRISPR/Cas9, unproven therapeutic approaches, potential failure to develop marketable products, lengthy and expensive clinical development, and intense competition - CRISPR/Cas9 genome editing technology has only recently been clinically validated for human therapeutic use, and the company's approaches are unproven[167](index=167&type=chunk)[170](index=170&type=chunk)[172](index=172&type=chunk) - The company has never generated revenue from product sales and its ability to become profitable depends on successful development, regulatory approval, and market acceptance of product candidates[173](index=173&type=chunk)[250](index=250&type=chunk) - Clinical development is lengthy, expensive, and uncertain, with risks of delays, additional costs, and failure to complete development or commercialization[175](index=175&type=chunk)[177](index=177&type=chunk) - Significant competition exists in an environment of rapid technological change, with competitors potentially achieving regulatory approval sooner or developing more effective therapies[171](index=171&type=chunk)[341](index=341&type=chunk) [Risks Related to Preclinical and Clinical Development](index=40&type=section&id=Risks%20Related%20to%20Preclinical%20and%20Clinical%20Development) The company faces substantial risks in preclinical and clinical development due to the novel nature of CRISPR/Cas9 technology, including regulatory uncertainty, potential for unforeseen events, and delays from patient enrollment or manufacturing issues - CRISPR/Cas9 genome editing technology is relatively new, and its therapeutic utility, especially for in vivo applications, is largely unproven, making regulatory approval uncertain[170](index=170&type=chunk)[172](index=172&type=chunk) - Clinical development is expensive, lengthy, and uncertain, with risks of delays or inability to complete trials due to regulatory authorization challenges, patient enrollment issues, manufacturing problems, or unforeseen safety concerns[175](index=175&type=chunk)[177](index=177&type=chunk)[178](index=178&type=chunk) - Interim clinical trial data may not be predictive of final results, and the high failure rate in biopharmaceutical development means positive results are not guaranteed to be replicated or lead to regulatory approval[187](index=187&type=chunk)[188](index=188&type=chunk) - Negative public opinion and increased regulatory scrutiny of genome editing or gene therapy, including ethical concerns, could damage public perception and adversely affect business operations or regulatory approvals[189](index=189&type=chunk) [Risks Related to the Industry](index=46&type=section&id=Risks%20Related%20to%20the%20Industry) Inconclusive results, lack of efficacy, or adverse events in clinical trials could impede regulatory approval and market acceptance, with the company potentially expending resources on unsuccessful programs - Inconclusive results, lack of efficacy, or adverse events in clinical trials could prevent or delay marketing approval, lead to narrower indications, or result in significant use restrictions[190](index=190&type=chunk)[195](index=195&type=chunk) - Even if approved, product candidates may not gain market acceptance among physicians, patients, hospitals, and third-party payors due to factors like perceived advantages over alternatives, side effects, cost, and competition[196](index=196&type=chunk)[198](index=198&type=chunk) - The company may not successfully use its genome editing technology to create a pipeline of product candidates or establish manufacturing capabilities, potentially expending resources on unsuccessful programs[192](index=192&type=chunk)[193](index=193&type=chunk) [Risks Related to Intellectual Property](index=49&type=section&id=Risks%20Related%20to%20Intellectual%20Property) Intellia faces significant intellectual property risks, including third-party infringement claims, reliance on licensed IP, and challenges in maintaining adequate patent protection globally - Third parties may assert patent infringement claims against Intellia, its licensors, or collaborators, potentially leading to injunctions, significant costs, or the need to obtain licenses on unfavorable terms[199](index=199&type=chunk)[201](index=201&type=chunk) - The company is dependent on licensed intellectual property, and termination or adverse modification of these licenses, or disputes over their scope and ownership (e.g., UC/Vienna/Charpentier patent family), could result in loss of significant rights[206](index=206&type=chunk)[207](index=207&type=chunk)[214](index=214&type=chunk)[215](index=215&type=chunk) - Obtaining and maintaining adequate patent protection globally is challenging due to varying laws, high costs, and the risk of patents being challenged, invalidated, or circumvented through litigation or administrative proceedings[226](index=226&type=chunk)[229](index=229&type=chunk)[231](index=231&type=chunk) - Trade secret protection and confidentiality agreements may not prevent unauthorized disclosure of proprietary information, and competitors could independently develop equivalent techniques or gain access through cybersecurity attacks[248](index=248&type=chunk) [Risks Related to Our Financial Position and Need for Additional Capital](index=66&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) Intellia has never generated revenue from product sales, has an accumulated deficit of **$2.3 billion**, and will require substantial additional funding, with failure to obtain financing potentially leading to delays or dilution of stockholders - The company has never generated revenue from product sales and has incurred net losses in each period since inception, with an accumulated deficit of **$2,291.7 million** as of March 31, 2025[255](index=255&type=chunk) - Substantial additional funding will be required for future operations, development, and commercialization of product candidates, and the company expects to rely on additional financing[257](index=257&type=chunk)[259](index=259&type=chunk) - Failure to obtain additional financing on acceptable terms could force delays, scaling back, or discontinuation of development efforts, or require relinquishing valuable rights to product candidates[261](index=261&type=chunk) - Raising additional capital through equity or convertible debt securities may dilute existing stockholders, and debt financing could impose restrictive covenants on operations[263](index=263&type=chunk) - Unfavorable national or global economic conditions or geopolitical developments could adversely affect the business, including demand for products, ability to raise capital, and supply chain disruptions[264](index=264&type=chunk)[265](index=265&type=chunk) [Risks Related to Manufacturing and Supply](index=70&type=section&id=Risks%20Related%20to%20Manufacturing%20and%20Supply) Manufacturing CRISPR/Cas9-based products is complex, with risks of production interruptions, raw material shortages, and reliance on third-party CMOs, which could harm the business - Manufacturing CRISPR/Cas9-based in vivo genome editing products and ex vivo engineered cell therapies is novel, complex, and difficult, with potential for production interruptions, raw material issues, and inability to meet regulatory requirements[266](index=266&type=chunk)[267](index=267&type=chunk) - The company relies on third-party CMOs for clinical product supplies and may face challenges in developing its own manufacturing capabilities, including hiring and retaining experienced personnel[268](index=268&type=chunk)[269](index=269&type=chunk) - Manufacturing and supply issues or delays could restrict the ability to meet clinical or market demand, be costly, and harm the business, financial condition, and prospects[270](index=270&type=chunk) [Risks Related to Government Regulation](index=70&type=section&id=Risks%20Related%20to%20Government%20Regulation) Intellia faces unpredictable and lengthy regulatory approval processes for its novel genome editing products, with risks of delays, changes in policies, and ongoing compliance obligations post-approval - The regulatory approval pathway for genome editing products is uncertain, complex, expensive, and lengthy, with limited precedent for in vivo gene editing therapies[271](index=271&type=chunk)[273](index=273&type=chunk) - Patient enrollment is a significant factor in clinical trial timing, affected by physician willingness, existing treatments, and regulatory/ethics committee approvals, with potential for delays or termination[275](index=275&type=chunk) - Obtaining regulatory approval in one jurisdiction does not guarantee approval in others, and foreign regulatory authorities may not accept data from trials conducted outside their jurisdiction[276](index=276&type=chunk)[285](index=285&type=chunk) - Orphan drug designation for nex-z and NTLA-2002 provides incentives but does not guarantee faster approval or market exclusivity, which can be lost under certain conditions[279](index=279&type=chunk)[280](index=280&type=chunk)[281](index=281&type=chunk) - RMAT designation for nex-z and NTLA-2002 may facilitate development and expedite review but does not assure faster approval or ultimate FDA approval[283](index=283&type=chunk)[284](index=284&type=chunk) - Existing regulatory policies may change, and new government regulations could prevent, limit, or delay product approval, increasing compliance burdens and costs[287](index=287&type=chunk)[288](index=288&type=chunk) - Even if approved, products will be subject to ongoing regulatory obligations (manufacturing, labeling, post-market studies) and continued review, with potential for significant expense and penalties for non-compliance[289](index=289&type=chunk)[291](index=291&type=chunk)[292](index=292&type=chunk) [Risks Related to Our Reliance on Third Parties](index=82&type=section&id=Risks%20Related%20to%20Our%20Reliance%20on%20Third%20Parties) Intellia's business heavily relies on collaborations and third-party CMOs/CROs, with risks of agreement termination, insufficient resource commitment, and failure to perform, impacting development and financial prospects - Reliance on collaborations, such as with Regeneron, means that termination or adverse alteration of agreements could harm business, financial condition, and prospects[309](index=309&type=chunk) - Collaborators may have discretion in resource allocation, may not perform as expected, or may develop competing products, impacting Intellia's programs and funding[313](index=313&type=chunk) - The company relies on third parties to manufacture clinical product supplies and for portions of the manufacturing process, facing risks if these parties fail to provide sufficient quantities, quality, or meet regulatory requirements[316](index=316&type=chunk)[318](index=318&type=chunk) - Reliance on third parties to conduct preclinical studies and clinical trials means their failure to carry out duties, meet deadlines, or comply with regulations could prevent regulatory approval or commercialization[321](index=321&type=chunk)[322](index=322&type=chunk) [Risks Related to Data and Privacy](index=88&type=section&id=Risks%20Related%20to%20Data%20and%20Privacy) Intellia's operations depend on secure IT systems, vulnerable to breaches, and the increasing use of social media/AI introduces new risks of data disclosure, non-compliance, and legal liabilities - Internal computer systems and those of collaborators/contractors are vulnerable to security breaches, incidents, or compromises, risking disruption of operations, loss of confidential information, and financial/reputational harm[328](index=328&type=chunk)[329](index=329&type=chunk) - Interruptions in server systems, internet/cloud services, or failure to maintain data security could harm the business, reputation, and incur substantial costs and liabilities[332](index=332&type=chunk) - Social media and AI platforms present new risks, including unauthorized disclosure of non-public information, negative publicity, and challenges in complying with evolving regulations, potentially leading to liability and reputational damage[334](index=334&type=chunk)[335](index=335&type=chunk)[336](index=336&type=chunk) [Risks Related to Competition](index=92&type=section&id=Risks%20Related%20to%20Competition) Intellia operates in a highly competitive biotechnology and pharmaceutical industry, facing competitors with greater resources, requiring first-to-market or product superiority for commercial success and profitability - The biotechnology and pharmaceutical industries are highly competitive, with Intellia facing competition from companies using genome editing, gene therapy, and traditional therapeutic modalities[341](index=341&type=chunk) - Competitors include large and specialty pharmaceutical/biotechnology companies, academic institutions, and government agencies, many with substantially greater resources[341](index=341&type=chunk)[348](index=348&type=chunk) - Specific competitors exist for nex-z (ATTR amyloidosis) and NTLA-2002 (HAE) programs, as well as other CRISPR-based and genome editing technologies[342](index=342&type=chunk)[343](index=343&type=chunk)[344](index=344&type=chunk)[345](index=345&type=chunk)[346](index=346&type=chunk) - To compete successfully, Intellia may need to be first-to-market or demonstrate product superiority, and failure to do so could limit commercial success and profitability[349](index=349&type=chunk)[350](index=350&type=chunk) [Risks Related to Commercialization](index=94&type=section&id=Risks%20Related%20to%20Commercialization) Intellia lacks established sales, marketing, and distribution infrastructure, requiring it to build these capabilities or partner with third parties, facing challenges in recruiting personnel and gaining physician acceptance - The company lacks sales, marketing, and distribution infrastructure and experience, requiring it to build these capabilities or partner with third parties for commercialization[351](index=351&type=chunk) - Challenges to commercial success include inability to recruit/train sales personnel, difficulty in gaining physician access/prescriptions, lack of complementary treatments, and unforeseen costs[351](index=351&type=chunk) - Reliance on third parties for sales and marketing may result in lower product revenue or profitability, and there's no assurance of favorable partnership terms or sufficient resource commitment[352](index=352&type=chunk) [Risks Related to Employee Matters and Managing Our Workforce](index=96&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Our%20Workforce) Future success depends on retaining key executives and attracting qualified personnel in a competitive market, with sustained stock price declines potentially impacting equity-based award retention value - Future success depends on retaining key executives and attracting, retaining, and motivating qualified personnel with specialized skills in research, development, manufacturing, and commercialization[353](index=353&type=chunk)[354](index=354&type=chunk) - The market for qualified personnel in genome editing and gene therapy, especially in Cambridge, Massachusetts, is highly competitive[354](index=354&type=chunk) - Sustained declines in stock price or lower stock price performance relative to competitors can reduce the retention value of equity-based awards, impacting compensation competitiveness[355](index=355&type=chunk) [Risks Related to Healthcare](index=96&type=section&id=Risks%20Related%20to%20Healthcare) Intellia's profitability depends on adequate coverage and reimbursement for its product candidates, subject to government and private payor decisions, and compliance with complex healthcare fraud and abuse laws - Coverage and reimbursement for product candidates, if approved, may be limited or unavailable, making profitable sales difficult, especially for novel genome editing and engineered cell products[356](index=356&type=chunk)[357](index=357&type=chunk) - Government authorities and third-party payors determine drug coverage and reimbursement, and obtaining approval is a time-consuming and costly process with no guarantee of adequate rates[359](index=359&type=chunk)[360](index=360&type=chunk) - The company is subject to federal and state healthcare fraud and abuse laws, false claims laws, physician payment transparency laws, health information privacy and security laws, and anti-corruption laws, with non-compliance risking substantial penalties[362](index=362&type=chunk)[365](index=365&type=chunk) - Healthcare cost control initiatives and legislative/regulatory reforms could prevent or delay marketing approval, affect profitability, and restrict post-approval activities[370](index=370&type=chunk)[371](index=371&type=chunk) [Risks Related to Our Common Stock](index=103&type=section&id=Risks%20Related%20to%20Our%20Common%20Stock) The market price of Intellia's common stock is highly volatile, subject to fluctuations, and future sales could dilute existing ownership, with management having broad discretion over capital use - The market price for Intellia's common stock has been highly volatile and is subject to wide fluctuations based on factors like product success, clinical trial results, regulatory developments, and market conditions[375](index=375&type=chunk)[377](index=377&type=chunk) - An active trading market for common stock may not be sustained, potentially making it difficult for stockholders to sell shares[374](index=374&type=chunk) - Future sales and issuances of common stock or rights to purchase common stock, including through equity incentive plans and at-the-market offerings, could result in additional dilution of stockholders' percentage ownership and cause the stock price to fall[387](index=387&type=chunk)[388](index=388&type=chunk) - Management has broad discretion over the use of cash, cash equivalents, and marketable securities, which may not be used effectively and could expose the company to liquidity issues at financial institutions[382](index=382&type=chunk)[383](index=383&type=chunk) - Operating as a public company incurs significant costs, and management must devote substantial time to compliance initiatives and corporate governance practices, including Section 404 of the Sarbanes-Oxley Act[385](index=385&type=chunk)[386](index=386&type=chunk) [Risks Related to our Charter and Bylaws](index=108&type=section&id=Risks%20Related%20to%20our%20Charter%20and%20Bylaws) Anti-takeover provisions in Intellia's charter documents and Delaware law could make an acquisition difficult and limit stockholder influence, potentially affecting the stock price - Anti-takeover provisions in the charter documents and under Delaware law could make an acquisition difficult and limit stockholder influence over management, potentially affecting the stock price[389](index=389&type=chunk) - Provisions include the board's ability to issue preferred stock, a classified board, requirements for stockholder meetings, and limitations on director removal[392](index=392&type=chunk) - The certificate of incorporation and by-laws designate specific courts (Delaware Court of Chancery and U.S. District Court for the District of Massachusetts) as exclusive forums for certain disputes, potentially limiting stockholders' choice of judicial forum[391](index=391&type=chunk)[393](index=393&type=chunk) [Risks Related to Tax Matters](index=110&type=section&id=Risks%20Related%20to%20Tax%20Matters) Changes in tax law could adversely affect Intellia's business, and the use of substantial net operating loss (NOL) carryforwards may be limited by an "ownership change" under the Internal Revenue Code - Changes in U.S. federal, state, and local tax laws could adversely affect the business and financial condition, increasing tax liability[395](index=395&type=chunk) - The company's ability to use its net operating loss (NOL) carryforwards and certain other tax attributes may be limited if an "ownership change" occurs under Sections 382 and 383 of the Internal Revenue Code[396](index=396&type=chunk) Tax Attributes as of December 31, 2024 (Amounts in millions) | Attribute | Amount | | :-------------------------------- | :------- | | Federal NOLs | $1,088.4 | | State NOLs | $1,079.2 | | Federal R&D and other credit carryforwards | $139.3 | | State R&D and other credit carryforwards | $77.9 | [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=110&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) No unregistered sales of equity securities or use of proceeds to report - No unregistered sales of equity securities or use of proceeds to report[397](index=397&type=chunk) [Item 5. Other Information](index=110&type=section&id=Item%205.%20Other%20Information) This section details Rule 10b5-1 trading plans adopted, modified, or terminated by directors or officers, including Director Jesse Goodman's plan for 3,094 securities Rule 10b5-1 Trading Plans (Three Months Ended March 31, 2025) | Name (Title) | Action Taken (Date of Action) | Type of Trading Arrangement | Nature of Trading Arrangement | Duration of Trading Arrangement | Aggregate Number of Securities | | :------------- | :---------------------------- | :-------------------------- | :---------------------------- | :------------------------------ | :----------------------------- | | Jesse Goodman (Director) | Adoption (March 31, 2025) | Rule 10b5-1 trading arrangement | Sale | Until the earlier of (a) August 5, 2025; (b) the first date on which all trades have been executed or all trading orders related to such trades have expired; and (c) the date on which the plan holder gives notice to terminate the plan. | 3,094 | [Item 6. Exhibits](index=111&type=section&id=Item%206.%20Exhibits) This section lists exhibits incorporated by reference or filed, including corporate governance documents, lease agreements, and various certifications - Exhibits include the Second Amended and Restated Certificate of Incorporation, Third Amended and Restated By-laws, Lease Agreements, and certifications from the CEO, CFO, and pursuant to Section 906 of Sarbanes-Oxley Act[404](index=404&type=chunk) [Signatures](index=112&type=section&id=Signatures) The report is signed by John M. Leonard, M.D. (President and CEO), Edward J. Dulac III (EVP, CFO), and Michael P. Dube (VP, Chief Accounting Officer) on May 8, 2025 - The report is signed by John M. Leonard, M.D. (President and CEO), Edward J. Dulac III (EVP, CFO), and Michael P. Dube (VP, Chief Accounting Officer) on May 8, 2025[407](index=407&type=chunk)[408](index=408&type=chunk) ```

Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities were approximately $707.1 million, down from $861.7 million as of December 31, 2024, reflecting normal operational expenses and nonrecurring costs associated with portfolio prioritization and workforce reduction [23][24] - Collaboration revenue for Q1 2025 was $16.6 million, compared to $28.9 million in Q1 2024, primarily due to a decrease in revenue under the Avansel license agreement [24] - R&D expenses were $108.4 million in Q1 2025, down from $111.8 million in Q1 2024, driven by lower employee-related expenses and stock-based compensation [25] Business Line Data and Key Metrics Changes - The company has dosed the first patient in its Phase 3 studies for hereditary angioedema (HAE) and hereditary ATTR with polyneuropathy, indicating significant progress in its clinical pipeline [9][10] - Enrollment in the global Phase 3 HALO study for HAE is progressing rapidly, with expectations to complete enrollment by the end of Q3 2025 [17][18] - The global Phase 3 MAGNETUDE study for ATTR with cardiomyopathy is also ahead of schedule, with over 90 sites actively enrolling patients [10][21] Market Data and Key Metrics Changes - The company is observing significant interest from both investigators and patients across its programs, indicating a high unmet need in the market for effective therapies [9][10] - The FDA granted RMAT designation for NexSee for the treatment of ATTR with cardiomyopathy, which will facilitate earlier engagement with the FDA [10][21] Company Strategy and Development Direction - The company is focused on operational excellence and aims to offer life-changing benefits through one-time therapies for severe diseases [8] - Intellia is building critical commercial foundations to bring its therapies to market quickly, with plans to file its first BLA in 2026 [14] - The company is monitoring the regulatory environment closely but has not experienced any tangible changes in its interactions with the FDA [13][14] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in meeting or exceeding regulatory timelines and objectives, with a strong relationship with the FDA [13][14] - The company anticipates a year-over-year decline in GAAP operating expenses of 5% to 10% and expects its cash balance to fund operations into the first half of 2027 [26][42] - Management highlighted the potential for significant value creation from its lead programs and the importance of maintaining a solid balance sheet [23][24] Other Important Information - The company is restructuring its real estate portfolio to reduce costs and simplify operations, expecting nearly $50 million in cash savings from a smaller footprint [39][41] - The company is actively considering non-dilutive financing options as it approaches commercialization, including collaborations and royalty transactions [50][51] Q&A Session Summary Question: Can you provide updated metrics regarding the patient baseline characteristics in the MAGNETUDE Phase 3 trial? - Management expects more than 50% of patients to be on tafamidis in the study, which is important for demonstrating a benefit over existing treatments [30] Question: What should we expect in terms of cash burn in the next 12 to 24 months? - The average cash use is estimated to be about $95 million per quarter, with a focus on efficient operations and significant milestones ahead [35][42] Question: How should we think about the launch dynamics for the first in vivo gene editing therapy? - The company is well-positioned for an efficient market entry post-approval, with a straightforward outpatient infusion process [54][56] Question: How are you thinking about pricing for your molecules given the competitive landscape? - The company aims to price its therapies competitively while providing significant value to patients and the healthcare system [60][89] Question: Can you discuss the impact of recent drug approvals on enrollment in the MAGNETUDE study? - Enrollment remains brisk despite the introduction of new drugs, as the company’s therapy offers distinct benefits [96][100]

Financial Data and Key Metrics Changes - As of March 31, 2025, the company's cash, cash equivalents, and marketable securities were approximately $707.1 million, down from $861.7 million as of December 31, 2024, reflecting normal operational expenses and nonrecurring costs associated with portfolio prioritization and workforce reduction [22][23] - Collaboration revenue for Q1 2025 was $16.6 million, compared to $28.9 million in Q1 2024, primarily due to a decrease in revenue under the Avansel license agreement [23] - R&D expenses were $108.4 million in Q1 2025, down from $111.8 million in Q1 2024, driven by lower employee-related expenses and stock-based compensation [24] Business Line Data and Key Metrics Changes - The company has dosed the first patient in its Phase 3 studies for hereditary angioedema (HAE) and hereditary ATTR with polyneuropathy, indicating significant progress in its clinical pipeline [7][14] - Enrollment in the global Phase 3 HALO study for HAE is progressing rapidly, confirming high unmet needs despite existing treatment options [8][15] - The FDA granted RMAT designation for NexSee for the treatment of ATTR with cardiomyopathy, which is expected to enhance engagement with the FDA [8][20] Market Data and Key Metrics Changes - The company is observing significant interest from both investigators and patients across its programs, with over 90 sites actively enrolling in the Phase 3 magnitude study for ATTR with cardiomyopathy [8][20] - The market for treatments for hereditary angioedema and ATTR amyloidosis is expanding, with increasing recognition of the diseases and improved diagnostic procedures [60] Company Strategy and Development Direction - The company is focused on operational excellence and aims to provide life-changing benefits through one-time therapies for severe diseases [6][7] - Intellia is building critical commercial foundations to bring its therapies to patients quickly, with a strong emphasis on evolving into a commercially ready company [9][10] - The company plans to file its first BLA in 2026 and is monitoring the regulatory environment closely, with no current changes affecting its timelines [12][13] Management's Comments on Operating Environment and Future Outlook - Management remains confident in meeting or exceeding regulatory timelines and objectives, with a strong relationship with the FDA [12][13] - The company is closely monitoring potential implications of pending pharmaceutical tariffs but is confident in its manufacturing and distribution capabilities [13] - Management expressed optimism about the potential of its therapies to reset treatment standards for patients with HAE and ATTR [14] Other Important Information - The company expects a year-over-year decline in GAAP operating expenses of 5% to 10% in 2025, with sufficient cash to fund operations into the first half of 2027 [25][36] - The company is undergoing a restructuring process aimed at reducing its real estate footprint and workforce, which is expected to yield significant cost savings [22][39] Q&A Session Summary Question: Update on magnitude Phase 3 trial enrollment and patient baseline characteristics - Management indicated that over 50% of patients in the study are expected to be on tafamidis, which is important for demonstrating the drug's benefit over existing treatments [30] Question: Cash burn and operating expenses expectations - The average cash use is estimated at $95 million per quarter for 2025 and 2026, with a focus on efficient operations and significant cost savings from real estate transactions [36][42] Question: Launch dynamics for the first in vivo gene editing therapy - The company is building a commercial organization with insights from prior one-time therapies, expecting efficient patient access post-approval [54][56] Question: Pricing strategy in light of competitive landscape - The company is focused on providing a competitive price for its one-time treatment, considering the significant cost savings for payers compared to chronic therapies [88][89] Question: Enrollment impact from new drugs in the market - Despite the approval of new drugs, the company has maintained brisk enrollment in its studies, with physicians seeing the potential for significant benefits from its therapies [95][96]

Exhibit 99.1 Intellia Therapeutics Announces First Quarter 2025 Financial Results and Highlights Recent Company Progress CAMBRIDGE, Mass., May 8, 2025 – Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading clinical-stage gene editing company focused on revolutionizing medicine with CRISPR-based therapies, today reported operational highlights and financial results for the first quarter ended March 31, 2025. "Intellia is full steam ahead and making excellent progress across its clinical programs," said Intel ...

Core Insights - Intellia Therapeutics reported significant progress in its clinical programs, including the initiation of Phase 3 studies for hereditary angioedema (HAE) and hereditary ATTR amyloidosis with polyneuropathy [2][5] - The company ended Q1 2025 with approximately $707.1 million in cash, expected to fund operations into the first half of 2027 [5][10] - Collaboration revenue decreased to $16.6 million in Q1 2025 from $28.9 million in Q1 2024, primarily due to a reduction in revenue from the AvenCell license [10][16] Operational Highlights - The first patients were dosed in the Phase 3 HAELO study for hereditary angioedema and the MAGNITUDE-2 study for hereditary ATTR amyloidosis with polyneuropathy [2][5] - Enrollment in the global Phase 3 MAGNITUDE trial for ATTR with cardiomyopathy is progressing ahead of projections, with expectations to exceed 550 patients by year-end [9][10] - Upcoming presentations include longer-term data from the Phase 1 study of NTLA-2002 at the EAACI Congress in June 2025 [2][6] Financial Results - Cash, cash equivalents, and marketable securities decreased from $861.7 million as of December 31, 2024, to $707.1 million as of March 31, 2025 [10][18] - Research and development expenses were $108.4 million in Q1 2025, a slight decrease from $111.8 million in Q1 2024 [10][16] - The net loss for Q1 2025 was $114.3 million, compared to a net loss of $107.4 million in Q1 2024 [10][16] Upcoming Events - Intellia will participate in several conferences, including the Bank of America Securities Health Care Conference on May 13, 2025, and the EAACI Congress on June 15, 2025 [10][12]

Core Viewpoint - Intellia Therapeutics, Inc. (NTLA) is showing potential for a bullish trend following a breakout above the 50-day moving average, indicating a key level of support [1][2]. Technical Analysis - NTLA has experienced a rally of 21.5% over the past four weeks, suggesting positive momentum [2]. - The 50-day simple moving average is a critical indicator for traders, marking the initial point of an up or down trend [2]. Earnings Estimates - There have been three upward revisions in NTLA's earnings estimates for the current fiscal year, with no downward revisions, indicating strong investor confidence [3]. - The consensus estimate for NTLA has also increased, further supporting the bullish outlook [3].