PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20%28Unaudited%29) Unaudited consolidated financial statements for Q3 2023 show increased collaboration revenue, rising expenses, and a net loss [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) | Balance Sheet Items (in thousands) | September 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $78,370 | $115,412 | | Marketable securities | $406,856 | $439,396 | | Total current assets | $498,261 | $563,159 | | Total assets | $612,407 | $679,223 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $34,575 | $35,095 | | Deferred revenue, non-current | $48,554 | $20,014 | | Total liabilities | $152,038 | $128,291 | | Total stockholders' equity | $460,369 | $550,932 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) | Statement of Operations (in thousands) | Nine months ended Sep 30, 2023 | Nine months ended Sep 30, 2022 | | :--- | :--- | :--- | | Collaboration revenue | $6,614 | $929 | | Research and development expenses | $138,135 | $92,811 | | General and administrative expenses | $37,655 | $26,095 | | Loss from operations | $(169,176) | $(117,977) | | Net loss | $(151,716) | $(116,302) | | Net loss per common share | $(2.43) | $(2.26) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) | Cash Flow Activities (in thousands) | Nine months ended Sep 30, 2023 | Nine months ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | $(108,869) | $(89,440) | | Net cash provided by investing activities | $36,583 | $12,358 | | Net cash provided by financing activities | $35,194 | $289,358 | | **Decrease in cash, cash equivalents and restricted cash** | **$(37,092)** | **$212,276** | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) - The company expects its cash, cash equivalents, and marketable securities of **$485.2 million** as of September 30, 2023, will be sufficient to fund operations and capital expenditures beyond the next 12 months[28](index=28&type=chunk) - Under the Vertex Agreement, the company recognized **$2.4 million** and **$5.9 million** of revenue for the three and nine months ended September 30, 2023, respectively. As of September 30, 2023, **$20.0 million** was recorded as non-current deferred revenue[69](index=69&type=chunk) - The Lilly Agreement became effective in July 2023. The company received a **$30.0 million** upfront payment in August 2023 and recognized **$0.7 million** of revenue for the three and nine months ended September 30, 2023. As of September 30, 2023, **$28.5 million** was recorded as non-current deferred revenue[70](index=70&type=chunk)[72](index=72&type=chunk)[82](index=82&type=chunk) - On October 27, 2023, Eli Lilly and Company acquired certain rights from Beam Therapeutics under the Amended Beam Agreement, including opt-in rights for Verve's PCSK9 and ANGPTL3 programs[102](index=102&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses financial condition and operations, detailing pipeline progress, collaboration impacts, and increased R&D expenses [Overview](index=24&type=section&id=Overview) - Verve is a clinical-stage genetic medicines company focused on single-course gene editing treatments for cardiovascular disease (CVD), targeting pathways like LDL, triglycerides, and Lp(a)[104](index=104&type=chunk) - The company incurred net losses of **$45.8 million** for Q3 2023 and **$151.7 million** for the nine months ended September 30, 2023, with an accumulated deficit of **$496.0 million**[111](index=111&type=chunk)[192](index=192&type=chunk) - As of September 30, 2023, the company had **$485.2 million** in cash, cash equivalents, and marketable securities, which is expected to fund operations into 2026[115](index=115&type=chunk) [Clinical and Development Programs](index=25&type=section&id=Clinical%20and%20Development%20Programs) - **VERVE-101:** The heart-1 Phase 1b clinical trial is ongoing in New Zealand and the UK. In October 2023, the FDA lifted the clinical hold on the IND, allowing for the activation of U.S. trial sites. Initial data is expected to be reported on November 12, 2023[118](index=118&type=chunk)[121](index=121&type=chunk)[122](index=122&type=chunk) - **VERVE-102:** A second PCSK9-targeting candidate using GalNAc-LNP delivery. A Phase 1b clinical trial is expected to begin in the first half of 2024[123](index=123&type=chunk) - **VERVE-201:** An ANGPTL3-targeting candidate for HoFH and refractory hypercholesterolemia. A Phase 1b clinical trial is expected to begin in the second half of 2024[124](index=124&type=chunk) [Results of Operations](index=29&type=section&id=Results%20of%20Operations) | Financials (in thousands) | Q3 2023 | Q3 2022 | Change | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $3,117 | $929 | $2,188 | | Research & Development | $43,765 | $35,197 | $8,568 | | General & Administrative | $11,686 | $9,592 | $2,094 | | **Net Loss** | **$(45,758)** | **$(45,190)** | **$(568)** | | Financials (in thousands) | Nine Months 2023 | Nine Months 2022 | Change | | :--- | :--- | :--- | :--- | | Collaboration Revenue | $6,614 | $929 | $5,685 | | Research & Development | $138,135 | $92,811 | $45,324 | | General & Administrative | $37,655 | $26,095 | $11,560 | | **Net Loss** | **$(151,716)** | **$(116,302)** | **$(35,414)** | - The increase in R&D expenses for the nine months ended Sep 30, 2023 was primarily driven by an **$18.6 million** increase in employee-related costs, a **$15.4 million** increase in manufacturing costs, and an **$8.6 million** increase in facility-related costs[152](index=152&type=chunk)[153](index=153&type=chunk) [Liquidity and Capital Resources](index=33&type=section&id=Liquidity%20and%20Capital%20Resources) - As of September 30, 2023, the company had **$485.2 million** in cash, cash equivalents, and marketable securities[157](index=157&type=chunk) - Recent financing activities in 2023 include receiving a **$30.0 million** upfront payment from Lilly and raising **$30.0 million** from a private placement with Lilly[157](index=157&type=chunk) - The company believes its existing capital will fund operating expenses and capital expenditure requirements into 2026, but this is based on assumptions that may prove wrong[169](index=169&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) The company's primary market risk is interest rate exposure on cash and marketable securities, with other market risks not significant - The company's main market risk is interest rate risk on its **$78.4 million** in cash and cash equivalents and **$406.9 million** in marketable securities[183](index=183&type=chunk) - Foreign currency exchange risk is not currently significant, though the company contracts with some vendors outside the U.S[184](index=184&type=chunk) - Inflation is not believed to have had a material effect on the business during the reported period[185](index=185&type=chunk) [Item 4. Controls and Procedures](index=37&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of September 30, 2023, with no material changes to internal controls - Management concluded that as of September 30, 2023, the company's disclosure controls and procedures were effective at the reasonable assurance level[187](index=187&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended September 30, 2023, that have materially affected, or are reasonably likely to materially affect, internal controls[188](index=188&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=38&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings but may face future litigation - As of the filing date, the company is not a party to any material legal proceedings[190](index=190&type=chunk) [Item 1A. Risk Factors](index=38&type=section&id=Item%201A.%20Risk%20Factors) This section details substantial risks including financial, development, third-party, intellectual property, commercialization, regulatory, and operational challenges [Risks related to our financial position and need for additional capital](index=38&type=section&id=Risks%20related%20to%20our%20financial%20position%20and%20need%20for%20additional%20capital) - The company has a history of significant losses, with an accumulated deficit of **$496.0 million** as of September 30, 2023, and expects to incur losses for the foreseeable future[192](index=192&type=chunk) - Substantial additional funding will be required to advance clinical trials and commercialization efforts. Failure to raise capital when needed could force delays or elimination of programs[198](index=198&type=chunk) - The company's limited operating history since its 2018 inception makes it difficult to evaluate its business success and future viability[206](index=206&type=chunk) [Risks related to discovery and development](index=42&type=section&id=Risks%20related%20to%20discovery%20and%20development) - Gene editing, particularly base editing, is a novel and largely unproven technology that has not yet been clinically validated as safe and efficacious for human therapeutic use[218](index=218&type=chunk) - The FDA placed the IND for VERVE-101 on clinical hold in November 2022, which was subsequently lifted in October 2023. There is no certainty that future holds will not occur or that planned trials for VERVE-102 and VERVE-201 will be permitted to start on schedule[213](index=213&type=chunk)[214](index=214&type=chunk) - Significant risks in gene editing include "off-target" edits and potential adverse events from the LNP delivery system, which could delay or halt development[258](index=258&type=chunk)[259](index=259&type=chunk) [Risks related to our dependence on third parties](index=54&type=section&id=Risks%20related%20to%20our%20dependence%20on%20third%20parties) - The company relies on third-party Contract Manufacturing Organizations (CMOs) and Contract Research Organizations (CROs) for manufacturing and clinical testing, reducing control over these critical activities[279](index=279&type=chunk) - Manufacturing biologic products is complex and susceptible to product loss, contamination, and challenges in scaling up, which could delay development and commercialization[283](index=283&type=chunk)[285](index=285&type=chunk) - Success of collaborations (e.g., with Vertex and Lilly) depends on the efforts of partners, who may have different priorities or may terminate agreements, potentially delaying programs[289](index=289&type=chunk)[290](index=290&type=chunk) [Risks related to our intellectual property](index=59&type=section&id=Risks%20related%20to%20our%20intellectual%20property) - The company's success depends on obtaining and defending patent protection for its gene editing technology, which is uncertain and costly, especially in the highly litigious field of gene editing[303](index=303&type=chunk)[306](index=306&type=chunk) - The company relies on licenses from third parties like Beam, Harvard, and Broad. Failure to comply with license obligations could result in the loss of critical intellectual property rights[312](index=312&type=chunk)[313](index=313&type=chunk) - The intellectual property landscape for CRISPR-based technologies is subject to ongoing interference proceedings and disputes, the outcomes of which are uncertain and could adversely affect the company's licensed rights[322](index=322&type=chunk)[324](index=324&type=chunk)[329](index=329&type=chunk) [Risks related to commercialization](index=73&type=section&id=Risks%20related%20to%20commercialization) - Even if approved, product candidates may fail to gain market acceptance from physicians, patients, and payors due to competition from well-established treatments like statins[384](index=384&type=chunk)[385](index=385&type=chunk) - The company faces substantial competition from major pharmaceutical and biotech companies with approved products (e.g., Repatha®, Leqvio®) and investigational therapies targeting PCSK9, ANGPTL3, and Lp(a)[389](index=389&type=chunk)[391](index=391&type=chunk) - The company's ability to commercialize products depends on securing coverage and adequate reimbursement from government and private payors, which is a time-consuming and uncertain process[408](index=408&type=chunk) [Risks related to regulatory approval and other legal compliance matters](index=79&type=section&id=Risks%20related%20to%20regulatory%20approval%20and%20other%20legal%20compliance%20matters) - The regulatory landscape for novel gene editing therapies is uncertain and evolving, which could lead to longer and more expensive approval processes[420](index=420&type=chunk) - Healthcare reform legislation, such as the Inflation Reduction Act (IRA), could result in more rigorous coverage criteria and downward pressure on pricing for approved products[457](index=457&type=chunk)[465](index=465&type=chunk) - The company is subject to complex global privacy and data security laws (e.g., GDPR, HIPAA, CCPA), and failure to comply could result in significant fines and penalties[471](index=471&type=chunk)[475](index=475&type=chunk)[477](index=477&type=chunk) [Risks related to employee matters and managing growth](index=92&type=section&id=Risks%20related%20to%20employee%20matters%20and%20managing%20growth) - The company is highly dependent on its key executives and scientific team, and the loss of their services could impede research and development objectives[495](index=495&type=chunk)[496](index=496&type=chunk) - Expected expansion of operations may lead to difficulties in managing growth, which could disrupt operations and divert management resources[498](index=498&type=chunk) - Internal IT systems are vulnerable to cybersecurity threats, which could result in a material disruption of development programs, compromise of sensitive information, and potential liability[501](index=501&type=chunk)[503](index=503&type=chunk) [Risks related to ownership of our common stock and our status as a public company](index=95&type=section&id=Risks%20related%20to%20ownership%20of%20our%20common%20stock%20and%20our%20status%20as%20a%20public%20company) - Executive officers, directors, and their affiliates beneficially owned approximately **21.3%** of common stock as of October 31, 2023, giving them significant influence over stockholder matters[510](index=510&type=chunk) - The company's stock price has been and is likely to continue to be volatile, influenced by factors such as clinical trial results, regulatory decisions, and market conditions[516](index=516&type=chunk) - The company will cease to qualify as an "emerging growth company" and a "smaller reporting company" as of December 31, 2023, which will result in increased legal and financial compliance costs[522](index=522&type=chunk)[527](index=527&type=chunk) [Item 2. Unregistered Sales of Equity Securities, Use of Proceeds and Issuer Purchases of Equity Securities](index=101&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%2C%20Use%20of%20Proceeds%20and%20Issuer%20Purchases%20of%20Equity%20Securities) No unregistered equity securities were issued during the quarter, and $281.6 million in IPO proceeds remain unused and invested - No unregistered equity securities were issued during the period covered by the report, other than those previously disclosed[540](index=540&type=chunk) - As of September 30, 2023, the **$281.6 million** in net proceeds from the company's IPO in June 2021 had not been used and remain invested in money market funds and marketable securities[542](index=542&type=chunk) [Item 6. Exhibits](index=102&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including corporate governance documents, key agreements, and officer certifications - The report includes key corporate governance documents, significant agreements such as the one with Eli Lilly, and required officer certifications as exhibits[544](index=544&type=chunk)

1 | --- | --- | --- | |-------|---------------------------------------------------------------------------------------------|-------| | | | | | | | | | | | | | | | | | | | | | | Verve Therapeutics | | | | Disrupting the Care of Cardiovascular Disease Through Single-course Gene Editing Medicines | | | | August 2023 | | Forward looking statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and ...

Part I [FINANCIAL INFORMATION](index=7&type=section&id=Part%20I.%20FINANCIAL%20INFORMATION) This section presents the company's unaudited condensed consolidated financial statements and related disclosures [Item 1. Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) The unaudited condensed consolidated financial statements for the period ended June 30, 2023, show a net loss of $54.0 million for the second quarter and $106.0 million for the six-month period, with total assets decreasing to $589.1 million [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) The company's total assets decreased to $589.1 million as of June 30, 2023, from $679.2 million at December 31, 2022, primarily due to a reduction in cash and marketable securities Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2023 | December 31, 2022 | | :--- | :--- | :--- | | Cash and cash equivalents | $70,042 | $115,412 | | Marketable securities | $392,434 | $439,396 | | **Total current assets** | **$473,066** | **$563,159** | | **Total assets** | **$589,131** | **$679,223** | | Total current liabilities | $33,031 | $35,095 | | **Total liabilities** | **$123,909** | **$128,291** | | **Total stockholders' equity** | **$465,222** | **$550,932** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For the three months ended June 30, 2023, the company reported collaboration revenue of $2.1 million and a net loss of $54.0 million, driven by higher research and development expenses Statement of Operations Summary (in thousands, except per share data) | Metric | Three Months Ended June 30, 2023 | Three Months Ended June 30, 2022 | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Collaboration revenue | $2,093 | $— | $3,497 | $— | | Research and development | $47,260 | $33,125 | $94,370 | $57,614 | | General and administrative | $13,416 | $9,067 | $25,969 | $16,503 | | Loss from operations | ($58,583) | ($42,192) | ($116,842) | ($74,117) | | **Net loss** | **($53,983)** | **($40,946)** | **($105,958)** | **($71,112)** | | Net loss per share | ($0.87) | ($0.84) | ($1.71) | ($1.46) | [Condensed Consolidated Statements of Cash Flows](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2023, net cash used in operating activities was $97.1 million, primarily due to a higher net loss, while investing activities provided $48.4 million Cash Flow Summary (in thousands) | Activity | Six Months Ended June 30, 2023 | Six Months Ended June 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($97,058) | ($60,265) | | Net cash provided by investing activities | $48,417 | $67,874 | | Net cash provided by financing activities | $3,271 | $950 | | **(Decrease) / Increase in cash, cash equivalents and restricted cash** | **($45,370)** | **$8,559** | [Notes to Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) The notes detail significant events and accounting policies, including a new research and collaboration agreement with Eli Lilly and the company's cash runway projection - In June 2023, the company entered into a research collaboration and stock purchase agreement with Eli Lilly, which became effective in July 2023, providing a **$30.0 million upfront payment** and a **$30.0 million equity investment** in August 2023, with eligibility for up to **$465 million in future milestones**[28](index=28&type=chunk)[29](index=29&type=chunk)[91](index=91&type=chunk) - The company expects its cash, cash equivalents, and marketable securities of **$462.5 million** as of June 30, 2023, will be sufficient to fund operations and capital expenditures beyond the next 12 months[30](index=30&type=chunk) - During the three and six months ended June 30, 2023, the company recognized **$2.1 million** and **$3.5 million** of revenue, respectively, from research services performed under its collaboration agreement with Vertex[72](index=72&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=23&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's focus on developing single-course gene editing medicines for cardiovascular disease, progress in clinical programs, increased net losses, and sufficient capital to fund operations into 2026 [Clinical and Development Programs](index=24&type=section&id=Clinical%20and%20Development%20Programs) The company is advancing its lead candidate, VERVE-101, in the heart-1 Phase 1b clinical trial, with initial data expected in Q4 2023, while preclinical development continues for VERVE-102 and VERVE-201 - Initial safety, pharmacodynamic, PCSK9, and LDL-C data for the dose-escalation portion of the heart-1 clinical trial of VERVE-101 are expected in the **fourth quarter of 2023**[118](index=118&type=chunk) - The Investigational New Drug (IND) application for VERVE-101 in the United States remains on hold by the FDA, and the company expects to complete trial enrollment outside the U.S[119](index=119&type=chunk) - The company plans to initiate a Phase 1b clinical trial for VERVE-102 in **H1 2024** and for VERVE-201 in **H2 2024**, subject to regulatory approval[120](index=120&type=chunk)[121](index=121&type=chunk) [Results of Operations](index=28&type=section&id=Results%20of%20Operations) For Q2 2023, collaboration revenue was $2.1 million, while R&D expenses increased by $14.1 million year-over-year to $47.3 million, leading to a widened net loss of $54.0 million Comparison of Results for Three Months Ended June 30 (in thousands) | Item | 2023 | 2022 | Change | | :--- | :--- | :--- | :--- | | Collaboration revenue | $2,093 | $0 | $2,093 | | Research and development | $47,260 | $33,125 | $14,135 | | General and administrative | $13,416 | $9,067 | $4,349 | | **Net loss** | **($53,983)** | **($40,946)** | **($13,037)** | - The **$14.1 million increase** in Q2 2023 R&D expenses was primarily driven by a **$6.3 million rise** in employee-related costs, a **$6.6 million increase** in manufacturing activities, and a **$2.4 million increase** in lab supplies[139](index=139&type=chunk)[145](index=145&type=chunk) [Liquidity and Capital Resources](index=31&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2023, the company had $462.5 million in cash and marketable securities, supplemented by an additional $60 million from Eli Lilly, projecting sufficient capital into 2026 - The company had **$462.5 million** in cash, cash equivalents, and marketable securities as of June 30, 2023[153](index=153&type=chunk) - Including the **$60 million** received from Lilly in July and August 2023, the company projects its cash runway will extend into **2026**[113](index=113&type=chunk)[166](index=166&type=chunk) Cash Flow Summary for Six Months Ended June 30 (in thousands) | Activity | 2023 | 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($97,058) | ($60,265) | | Net cash provided by investing activities | $48,417 | $67,874 | | Net cash provided by financing activities | $3,271 | $950 | [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=35&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk exposure is to interest rate changes affecting its $462.4 million in cash and marketable securities, with no significant foreign currency or inflation risk - The primary market risk is interest rate risk on cash, cash equivalents, and marketable securities totaling **$462.4 million**, where an immediate **10% change** in interest rates is not expected to have a material effect[179](index=179&type=chunk) - The company is not currently exposed to significant foreign currency exchange risk or inflation risk[180](index=180&type=chunk)[181](index=181&type=chunk) [Item 4. Controls and Procedures](index=35&type=section&id=Item%204.%20Controls%20and%20Procedures) As of June 30, 2023, the company's disclosure controls and procedures were deemed effective, with no material changes to internal control over financial reporting during the quarter - Management concluded that disclosure controls and procedures were effective as of June 30, 2023[183](index=183&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended June 30, 2023, that have materially affected, or are reasonably likely to materially affect, internal controls[184](index=184&type=chunk) Part II [OTHER INFORMATION](index=36&type=section&id=Part%20II.%20OTHER%20INFORMATION) This section provides other important information, including legal proceedings, risk factors, and equity sales [Item 1. Legal Proceedings](index=36&type=section&id=Item%201.%20Legal%20Proceedings) The company reports that it is not currently a party to any material legal proceedings - As of the filing date, Verve Therapeutics is not a party to any material legal proceedings[186](index=186&type=chunk) [Item 1A. Risk Factors](index=36&type=section&id=Item%201A.%20Risk%20Factors) This section outlines significant risks to the company's business, including financial position, early-stage development, reliance on third parties, intellectual property, and commercialization challenges [Risks related to our financial position and need for additional capital](index=36&type=section&id=Risks%20related%20to%20our%20financial%20position%20and%20need%20for%20additional%20capital) The company has a history of significant operating losses and will require substantial additional funding to advance its product candidates, with an inability to raise capital potentially forcing delays or elimination of development programs - The company has incurred significant losses since inception, with a net loss of **$106.0 million** for the six months ended June 30, 2023, and an accumulated deficit of **$450.2 million**[188](index=188&type=chunk) - Substantial additional funding is required to continue operations, and failure to raise capital could force the company to delay, reduce, or eliminate product development programs[194](index=194&type=chunk) [Risks related to discovery and development](index=40&type=section&id=Risks%20related%20to%20discovery%20and%20development) The company is in the very early stages of clinical development, with its first trial for VERVE-101 initiated in 2022, facing an FDA clinical hold and the unproven nature of gene editing technology - The company is in early-stage development, having only initiated its first clinical trial for VERVE-101 in July 2022, and its other programs are preclinical[208](index=208&type=chunk) - The FDA has placed the IND for VERVE-101 on hold in the U.S., requesting additional preclinical data on potency, germline editing risks, and off-target analyses, which may delay or prevent the trial from starting in the U.S[231](index=231&type=chunk) - Gene editing is a novel technology that is not yet clinically validated, with risks including potential off-target edits that could cause serious adverse events, and the permanence of edits means therapy cannot be withdrawn if side effects occur[214](index=214&type=chunk)[255](index=255&type=chunk) [Risks related to our dependence on third parties](index=52&type=section&id=Risks%20related%20to%20our%20dependence%20on%20third%20parties) Verve relies heavily on third parties for critical functions, including contract manufacturing organizations and contract research organizations, which introduces risks of delays, quality issues, and supply chain disruptions, while the success of collaborations is not guaranteed - The company relies on third-party CMOs for manufacturing, which increases the risk of insufficient quantities, unacceptable cost or quality, and potential development delays[279](index=279&type=chunk) - The company depends on collaborations with partners like Beam, Acuitas, Novartis, Vertex, and Lilly, and the success of these collaborations is not guaranteed and depends on the collaborators' performance and strategic focus[286](index=286&type=chunk) [Risks related to our intellectual property](index=57&type=section&id=Risks%20related%20to%20our%20intellectual%20property) The company's success hinges on its ability to obtain, maintain, and defend its intellectual property, which is subject to in-licensed technology and a highly dynamic, litigious gene editing IP landscape - The company's rights to develop its technology are subject to licenses from third parties, including Beam, Harvard, and Broad, and failure to comply with license obligations could result in termination of these critical agreements[309](index=309&type=chunk)[310](index=310&type=chunk) - The intellectual property landscape for CRISPR-based gene editing is highly dynamic and subject to ongoing interference proceedings and litigation, which could impact the validity and scope of the company's in-licensed patents[318](index=318&type=chunk)[319](index=319&type=chunk)[340](index=340&type=chunk) [Risks related to commercialization](index=71&type=section&id=Risks%20related%20to%20commercialization) Even if approved, the company's product candidates may not achieve market acceptance due to competition from well-established treatments and other novel therapies, and the company currently lacks sales, marketing, and distribution capabilities - The company faces substantial competition from established CVD treatments and other companies developing therapies targeting PCSK9 and ANGPTL3, including major pharmaceutical firms like Amgen, Sanofi, Novartis, and Regeneron[382](index=382&type=chunk)[384](index=384&type=chunk)[386](index=386&type=chunk) - The company has no sales, marketing, or distribution infrastructure and will need to build these capabilities or partner with third parties to commercialize any approved products[393](index=393&type=chunk) - Successful commercialization depends on obtaining favorable pricing, coverage, and reimbursement from government and private payors, which is uncertain and subject to downward pressure from healthcare reform initiatives[400](index=400&type=chunk)[401](index=401&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=99&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities during the period and confirmed that the $281.6 million net proceeds from its June 2021 IPO remain unused and invested - There were no unregistered sales of equity securities during the period[531](index=531&type=chunk) - As of June 30, 2023, the company had not used any of the **$281.6 million** in net proceeds from its June 2021 IPO, with the funds invested in money market funds and marketable securities[533](index=533&type=chunk) [Item 6. Exhibits](index=100&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including key agreements with Eli Lilly and Company and officer certifications - Key exhibits filed include the Research and Collaboration Agreement and the Stock Purchase Agreement with Eli Lilly and Company[535](index=535&type=chunk)

1 | --- | --- | |-------|----------------------------------------------------------------------------------------------| | | | | | Developing 'once-and-done' | | | gene editing medicines to treat cardiovascular disease | | | Sekar Kathiresan, MD CEO, Verve Therapeutics Lecturer in Medicine, Harvard Medical School | | | May 8, 2023 TIDES USA: Oligonucleotide and peptide therapeutics | Atherosclerotic cardiovascular disease (ASCVD): #1 cause of death worldwide despite available treatments One person dies ever ...

[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Unaudited Q1 2023 financial statements report a **$52.0 million** net loss, increased operating expenses, and **$1.4 million** in collaboration revenue Condensed Consolidated Balance Sheet Data (in thousands USD) | Account | March 31, 2023 | December 31, 2022 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $83,746 | $115,412 | | Marketable securities | $424,941 | $439,396 | | Total current assets | $517,787 | $563,159 | | Total assets | $634,376 | $679,223 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $33,552 | $35,095 | | Total liabilities | $124,900 | $128,291 | | Total stockholders' equity | $509,476 | $550,932 | Condensed Consolidated Statements of Operations (in thousands USD) | Account | Three months ended March 31, 2023 | Three months ended March 31, 2022 | | :--- | :--- | :--- | | Collaboration revenue | $1,404 | $0 | | Research and development | $47,110 | $24,490 | | General and administrative | $12,553 | $7,435 | | Loss from operations | ($58,259) | ($31,925) | | Net loss | ($51,975) | ($30,166) | | Net loss per common share, basic and diluted | ($0.84) | ($0.62) | Condensed Consolidated Statements of Cash Flows (in thousands USD) | Activity | Three months ended March 31, 2023 | Three months ended March 31, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | ($49,096) | ($33,507) | | Net cash provided by investing activities | $15,392 | $60,092 | | Net cash provided by financing activities | $2,038 | $505 | [Notes to Unaudited Condensed Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes detail financial position, accounting policies, **$508.7 million** cash sufficiency, **$1.4 million** Vertex revenue, and **$8.0 million** stock-based compensation - The company expects its cash, cash equivalents, and marketable securities of **$508.7 million** as of March 31, 2023, to be sufficient to fund operations and capital expenditures beyond the next 12 months[29](index=29&type=chunk) - In July 2022, the company entered into a collaboration agreement with Vertex, receiving a **$25 million** upfront payment. For Q1 2023, Verve recognized **$1.4 million** in revenue from this agreement and has **$20.0 million** in non-current deferred revenue[57](index=57&type=chunk)[68](index=68&type=chunk) - The company has a success payment liability to Harvard and Broad, valued at **$2.1 million** as of March 31, 2023. The fair value decreased by **$0.7 million** during the quarter, which was recorded as other income[43](index=43&type=chunk)[45](index=45&type=chunk) - Stock-based compensation expense for Q1 2023 was **$8.0 million**, up from **$4.2 million** in Q1 2022. As of March 31, 2023, there was **$91.5 million** of unrecognized expense related to stock options and **$18.6 million** related to restricted stock units[75](index=75&type=chunk)[77](index=77&type=chunk)[78](index=78&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=21&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's focus on gene editing medicines for CVD, with lead candidate VERVE-101 in trials despite a U.S. FDA hold, and a Q1 2023 net loss of **$52.0 million** - Verve is a clinical-stage company focused on single-course gene editing medicines for CVD, with a pipeline targeting PCSK9 (VERVE-101, VERVE-102) and ANGPTL3 (VERVE-201)[86](index=86&type=chunk)[87](index=87&type=chunk) - The heart-1 clinical trial for VERVE-101 is ongoing in New Zealand and the United Kingdom, with initial data expected in the second half of 2023. However, the U.S. IND application for this trial was placed on hold by the FDA in October 2022[99](index=99&type=chunk)[101](index=101&type=chunk) Financial and Operational Summary (in millions USD) | Metric | Q1 2023 | Q1 2022 | | :--- | :--- | :--- | | Net Loss | $52.0 | $30.2 | | R&D Expenses | $47.1 | $24.5 | | G&A Expenses | $12.6 | $7.4 | | Collaboration Revenue | $1.4 | $0 | | Cash, Cash Equivalents & Marketable Securities | $508.7 | N/A | - The company believes its existing cash, cash equivalents, and marketable securities of **$508.7 million** will fund operating expenses and capital expenditure requirements into the second half of 2025[95](index=95&type=chunk)[140](index=140&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=31&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is exposed to market risk from changes in interest rates affecting its cash, cash equivalents, and marketable securities, which totaled **$508.6 million** as of March 31, 2023, but expects no material effect from a 10% rate change - The company's primary market risk is interest rate risk on its **$508.6 million** in cash, cash equivalents, and marketable securities. However, due to the short-term, low-risk profile of these holdings, the impact of interest rate changes is not considered material[154](index=154&type=chunk) - Verve is not currently exposed to significant foreign currency exchange risk but may be in the future due to contracts with vendors outside the U.S.[155](index=155&type=chunk) - Inflation is not believed to have had a material effect on the company's business, financial condition, or results of operations during Q1 2023[156](index=156&type=chunk) [Controls and Procedures](index=31&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of March 31, 2023, with no material changes to internal control over financial reporting during the quarter - As of March 31, 2023, the Principal Executive Officer and Principal Financial Officer concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level[158](index=158&type=chunk) - No material changes to the company's internal control over financial reporting occurred during the quarter ended March 31, 2023[159](index=159&type=chunk) [PART II. OTHER INFORMATION](index=33&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=33&type=section&id=Item%201.%20Legal%20Proceedings) As of the report date, Verve Therapeutics is not a party to any material legal proceedings, though future involvement in litigation is possible - The company is currently not a party to any material legal proceedings[161](index=161&type=chunk) [Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) The company faces extensive risks including financial losses, need for funding, unproven gene editing technology, FDA holds, reliance on third parties, IP litigation, and commercialization challenges [Risks related to financial position and need for additional capital](index=33&type=section&id=Risks%20related%20to%20our%20financial%20position%20and%20need%20for%20additional%20capital) The company has a history of significant losses, expects them to continue, and requires substantial additional funding to advance programs, with no guarantee of profitability - The company has a history of significant operating losses, with a net loss of **$52.0 million** for Q1 2023 and an accumulated deficit of **$396.2 million** as of March 31, 2023[163](index=163&type=chunk) - Substantial additional funding is needed. If unable to raise capital, the company may be forced to delay, reduce, or eliminate product development or commercialization efforts[171](index=171&type=chunk) - The company's limited operating history, having commenced operations in 2018 and only recently initiated its first clinical trial, makes it difficult to evaluate its success and future viability[178](index=178&type=chunk) [Risks related to discovery and development](index=37&type=section&id=Risks%20related%20to%20discovery%20and%20development) This section highlights the high-risk nature of the company's early-stage clinical development, including the novel and unproven base editing technology and an FDA hold on VERVE-101's IND - The company is in the very early stages of development, having only recently initiated its first clinical trial for VERVE-101. The FDA has placed the IND application for this trial on hold in the United States[185](index=185&type=chunk)[188](index=188&type=chunk) - Gene editing, particularly base editing, is a novel and unproven technology. There is no guarantee it will lead to safe and effective marketable products[191](index=191&type=chunk) - Product candidates or their LNP delivery systems could cause serious adverse events, including off-target edits, liver injury, or immune reactions, which could delay or halt development[230](index=230&type=chunk)[232](index=232&type=chunk)[233](index=233&type=chunk) [Risks related to dependence on third parties](index=49&type=section&id=Risks%20related%20to%20our%20dependence%20on%20third%20parties) Verve relies heavily on third parties for manufacturing, clinical trials, and key collaborations, introducing risks of performance failures, delays, and loss of control - The company relies on third-party CMOs for manufacturing and CROs for research and clinical testing, which reduces control over these activities and introduces risks of unsatisfactory performance or delays[251](index=251&type=chunk) - Manufacturing of biologic products is complex and the company has no manufacturing facilities of its own, making it dependent on third parties who must comply with cGMP regulations[256](index=256&type=chunk)[258](index=258&type=chunk) - The company has entered into key collaborations (e.g., with Beam, Acuitas, Novartis, Vertex) and may not have full control over the resources dedicated by collaborators, posing risks to development and commercialization[263](index=263&type=chunk) [Risks related to our intellectual property](index=54&type=section&id=Risks%20related%20to%20our%20intellectual%20property) Success depends on obtaining and defending IP in the litigious gene editing field, with risks from patent interference proceedings and compliance with third-party license agreements - The company's ability to commercialize its products depends on obtaining and enforcing patent protection for its gene editing technology, which is uncertain and complex[276](index=276&type=chunk)[277](index=277&type=chunk) - The company is subject to the outcomes of ongoing patent interference proceedings involving its licensors (the Boston Licensing Parties) and other entities like CVC and Toolgen, which could result in the loss of key patent rights for CRISPR-Cas9 technology[295](index=295&type=chunk)[296](index=296&type=chunk)[297](index=297&type=chunk) - The company relies on licenses from third parties like Beam and Harvard/Broad. Failure to comply with the terms of these licenses could result in losing rights to essential technology for its product candidates[286](index=286&type=chunk)[287](index=287&type=chunk) [Risks related to commercialization](index=68&type=section&id=Risks%20related%20to%20commercialization) Approved products face significant commercialization hurdles including market acceptance, competition from established treatments, and securing favorable pricing and reimbursement - Approved products may fail to gain market acceptance from physicians, patients, and payors due to competition from established treatments and the novel nature of gene editing[357](index=357&type=chunk)[358](index=358&type=chunk) - The company faces substantial competition from major pharmaceutical companies with existing products for lowering LDL-C, including mAbs (Repatha®, PRALUENT®) and siRNA (Leqvio®), as well as other gene editing programs in development[362](index=362&type=chunk)[363](index=363&type=chunk) - The company has no sales, marketing, or distribution infrastructure and will need to build these capabilities or rely on third parties, which carries its own risks[370](index=370&type=chunk) - Successfully commercializing products depends on obtaining adequate coverage and reimbursement from government and private payors, which is uncertain and subject to cost-containment pressures[377](index=377&type=chunk)[378](index=378&type=chunk) [Risks related to regulatory approval and other legal compliance matters](index=73&type=section&id=Risks%20related%20to%20regulatory%20approval%20and%20other%20legal%20compliance%20matters) The company faces risks from an uncertain and evolving regulatory landscape for gene editing, ongoing compliance with healthcare and data privacy laws, and potential impacts from new legislation - The regulatory landscape for gene editing is novel and uncertain, which could lead to unpredictable timelines, costs, and requirements for obtaining marketing approval[388](index=388&type=chunk) - Even if approved, products will be subject to continual and extensive regulatory oversight, and any failure to comply could result in restrictions, withdrawal from the market, or penalties[416](index=416&type=chunk)[418](index=418&type=chunk) - The business is subject to numerous healthcare laws, including anti-kickback and false claims statutes, as well as global data privacy laws like GDPR and HIPAA, violations of which can lead to severe penalties[424](index=424&type=chunk)[425](index=425&type=chunk)[441](index=441&type=chunk) - Recent legislation, such as the Inflation Reduction Act (IRA), could impact pharmaceutical pricing and reimbursement, potentially affecting the profitability of future products[435](index=435&type=chunk)[436](index=436&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=95&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reported no unregistered sales of equity securities and has not used any of the **$281.6 million** net proceeds from its June 2021 IPO - There were no unregistered sales of equity securities in the reported period[507](index=507&type=chunk) - As of March 31, 2023, the company had not used any of the **$281.6 million** in net proceeds from its June 2021 IPO. The funds are invested in money market funds and marketable securities[509](index=509&type=chunk) [Exhibits](index=96&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including corporate documents and officer certifications

PART I [Business](index=7&type=section&id=Item%201.%20Business) Verve Therapeutics is a clinical-stage genetic medicines company focused on developing single-course gene editing treatments for cardiovascular disease (CVD), with lead candidates VERVE-101 and VERVE-201 targeting PCSK9 and ANGPTL3 genes respectively, leveraging base editing and LNP delivery systems for a stepwise clinical development plan - Verve Therapeutics is pioneering single-course in vivo gene editing treatments to transform cardiovascular disease (CVD) care from chronic management to a one-time therapy, addressing the root causes of the disease[20](index=20&type=chunk) - The company's initial programs, VERVE-101 and VERVE-201, target the validated genes PCSK9 and ANGPTL3, respectively, to durably lower blood lipids like LDL-C and reduce the risk of atherosclerotic cardiovascular disease (ASCVD)[20](index=20&type=chunk) - The lead product candidate, VERVE-101, is currently in a Phase 1b clinical trial (heart-1) for patients with Heterozygous Familial Hypercholesterolemia (HeFH), with the first patient dosed in **July 2022**[31](index=31&type=chunk)[87](index=87&type=chunk) - In **October 2022**, the U.S. FDA placed a clinical hold on the Investigational New Drug (IND) application for VERVE-101, requesting additional preclinical data on potency, germline editing risks, and off-target analyses[32](index=32&type=chunk)[148](index=148&type=chunk) - The second program, VERVE-201, targets ANGPTL3 for Homozygous Familial Hypercholesterolemia (HoFH) and utilizes a proprietary GalNAc-LNP delivery system to bypass the deficient LDL receptor in these patients, with a Phase 1b trial anticipated to start in **2024**[33](index=33&type=chunk)[35](index=35&type=chunk)[90](index=90&type=chunk) [Our Approach](index=15&type=section&id=Our%20Approach) The company employs a tailored approach using precise base editing delivered via non-viral Lipid Nanoparticles (LNPs) to target the liver, prioritizing validated liver-cardiovascular axis genes and leveraging NHP models for rapid preclinical validation to create potent, durable, and safe single-course therapies with scalable manufacturing potential - Verve utilizes base editing, a next-generation gene editing approach that enables precise single-base changes in the genome without making double-stranded DNA breaks, akin to a "pencil" that erases and rewrites a single letter in a gene[61](index=61&type=chunk)[64](index=64&type=chunk) - The company uses non-viral Lipid Nanoparticle (LNP) delivery for its gene editors due to their natural tropism to the liver, transient expression of editing proteins, and a potentially superior safety profile compared to viral vectors[70](index=70&type=chunk)[73](index=73&type=chunk) - Target selection is based on strong human genetic evidence, where loss-of-function mutations confer disease resistance without adverse effects, and there is existing clinical proof-of-concept from other therapeutic modalities[67](index=67&type=chunk)[72](index=72&type=chunk) - A key part of the development strategy is the early and rapid iteration of product candidates in non-human primate (NHP) preclinical models, which are considered powerful predictors of efficacy for liver-directed gene editing and LNP delivery in humans[62](index=62&type=chunk) [Our Gene Editing Programs](index=19&type=section&id=Our%20Gene%20Editing%20Programs) Verve is advancing a pipeline of in vivo gene editing programs targeting liver-expressed genes implicated in CVD, with lead program VERVE-101 in Phase 1b for HeFH and VERVE-201 in preclinical development for HoFH, employing a stepwise development approach from rare genetic disorders to broader ASCVD populations Verve Therapeutics Pipeline Overview | Target | Indication | Technology | Development Status | | :--- | :--- | :--- | :--- | | **PCSK9 (VERVE-101)** | Heterozygous familial hypercholesterolemia, ASCVD | Base Editor | Clinical (Phase 1b) | | **ANGPTL3 (VERVE-201)** | Homozygous familial hypercholesterolemia, Refractory Hypercholesterolemia | Base Editor | IND-enabling | | **LPA** | ASCVD patients with high blood Lp(a) | Novel Editor | Research | | **Undisclosed** | Undisclosed ASCVD | Base Editor | Research | | **Undisclosed** | Undisclosed liver disease (with Vertex) | Novel Editor | Research | - VERVE-101 is in the heart-1 Phase 1b clinical trial in New Zealand and the United Kingdom for patients with HeFH, with initial safety and pharmacodynamic data from the dose-escalation portion expected in the second half of **2023**[87](index=87&type=chunk)[146](index=146&type=chunk) - VERVE-201 is being developed for HoFH and refractory hypercholesterolemia, and the company anticipates initiating a Phase 1b clinical trial in **2024**[89](index=89&type=chunk)[90](index=90&type=chunk) [Intellectual Property and Collaborations](index=41&type=section&id=Intellectual%20Property%20and%20Collaborations) Verve's intellectual property strategy combines owned patent applications and in-licensed technologies for gene editing, base editing, CRISPR nucleases, and LNP delivery, supported by key agreements with Beam Therapeutics, Acuitas, Novartis, and a recent strategic collaboration with Vertex for an undisclosed liver disease program - The company has an exclusive, worldwide license with Beam Therapeutics for base editing technology directed at PCSK9, ANGPTL3, and a third cardiovascular target, with Beam retaining opt-in rights for co-development and co-commercialization[201](index=201&type=chunk)[202](index=202&type=chunk)[204](index=204&type=chunk) - Verve has a non-exclusive, worldwide license from Acuitas Therapeutics for the LNP technology used in its lead product candidate, VERVE-101[214](index=214&type=chunk) - A non-exclusive license agreement with Novartis provides access to lipid technology used in the research and development of certain product candidates, including VERVE-201[221](index=221&type=chunk) - In **July 2022**, Verve entered into a **four-year** global research collaboration with Vertex Pharmaceuticals to discover and develop an in vivo gene editing program for a single undisclosed liver disease, receiving a **$25 million** upfront payment[59](index=59&type=chunk)[243](index=243&type=chunk)[245](index=245&type=chunk) [Government Regulation](index=49&type=section&id=Government%20Regulation) The company's product candidates are subject to extensive regulation as biologics and gene therapies by the FDA in the U.S. and comparable authorities like the EMA in Europe, involving rigorous preclinical testing, multi-phase clinical trials, and marketing approval processes, with ongoing post-approval requirements and evolving regulatory guidance for gene editing technologies - Verve's product candidates are regulated as biologics in the U.S., requiring a Biologics License Application (BLA) for marketing approval, which involves extensive preclinical and clinical data to establish safety, potency, and purity[251](index=251&type=chunk)[252](index=252&type=chunk) - Gene therapy products are subject to special regulations and guidance from the FDA's Center for Biologics Evaluation and Research (CBER), which may require long-term follow-up of patients for up to **15 years** to monitor for potential delayed adverse effects[282](index=282&type=chunk)[283](index=283&type=chunk)[285](index=285&type=chunk) - In the European Union, gene therapy products are classified as advanced therapy medicinal products (ATMPs) and are reviewed under a centralized procedure by the EMA, involving both the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP)[346](index=346&type=chunk)[351](index=351&type=chunk) - The company may seek expedited review programs such as Fast Track, Breakthrough Therapy, and Priority Review in the U.S., and PRIME designation in the E.U., to potentially accelerate development and review timelines for its product candidates[303](index=303&type=chunk)[342](index=342&type=chunk)[663](index=663&type=chunk) [Risk Factors](index=76&type=section&id=Item%201A.%20Risk%20Factors) The company faces substantial risks including the need for significant additional funding due to a history of losses and no commercial products, high development risks from novel gene editing technologies with potential adverse events and regulatory hurdles like the FDA clinical hold on VERVE-101, reliance on third-party collaborators for technology and manufacturing, a dynamic intellectual property landscape, and commercialization challenges from competition, market acceptance, and pricing - The company has a history of significant losses (**$157.4 million** in **2022**) and expects to incur losses for the foreseeable future, requiring substantial additional funding to advance its product pipeline[406](index=406&type=chunk)[413](index=413&type=chunk) - The FDA has placed the IND application for VERVE-101 on clinical hold in the U.S., and there is no certainty that the hold will be lifted, which could delay or prevent the initiation of U.S. clinical trials[431](index=431&type=chunk)[454](index=454&type=chunk) - Gene editing, particularly base editing, is a novel and unproven technology in humans, with risks of serious adverse events, undesirable side effects, or off-target edits that could delay or halt development[435](index=435&type=chunk)[480](index=480&type=chunk)[482](index=482&type=chunk) - The company relies on third parties for critical aspects of its business, including manufacturing (CMOs), research (CROs), and technology licenses (e.g., Beam, Acuitas), which introduces risks related to performance, supply, and compliance[503](index=503&type=chunk)[508](index=508&type=chunk)[515](index=515&type=chunk) - The intellectual property landscape for genome editing is highly dynamic and litigious, and the company faces risks of infringement claims and challenges to its in-licensed patents, which could interfere with product development[541](index=541&type=chunk)[574](index=574&type=chunk) [Properties](index=142&type=section&id=Item%202.%20Properties) The company leases a **105,182 square foot** office and laboratory space in Boston, Massachusetts, under a lease expiring in **December 2032**, which management deems sufficient for current needs - The company's primary facility is a **105,182 square foot** leased office and laboratory space in Boston, MA, with the lease expiring in **December 2032**[776](index=776&type=chunk) [Legal Proceedings](index=142&type=section&id=Item%203.%20Legal%20Proceedings) As of the report date, Verve Therapeutics is not a party to any material legal proceedings, though it may become involved in litigation in the ordinary course of business in the future - The company is not currently a party to any material legal proceedings[778](index=778&type=chunk) PART II [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=143&type=section&id=Item%205.%20Market%20for%20Registrant%27s%20Common%20Equity%2C%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) Verve's common stock has traded on the Nasdaq Global Select Market under "VERV" since **June 16, 2021**, with approximately **17 holders** of record as of **February 27, 2023**, and the company has never paid cash dividends, intending to retain earnings for growth, with no equity security purchases made in **2022** - The company's common stock trades on the Nasdaq Global Select Market under the symbol "VERV" since its IPO on **June 16, 2021**[781](index=781&type=chunk) - The company has never declared or paid cash dividends and intends to retain any future earnings to finance business operations and expansion[783](index=783&type=chunk) - Net proceeds from the **June 2021** IPO were **$281.6 million**, none of which had been used as of **December 31, 2022**, and are invested in money market funds and short-term investments[789](index=789&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=146&type=section&id=Item%207.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) For the year ended **December 31, 2022**, Verve Therapeutics reported a net loss of **$157.4 million**, an increase from **$120.3 million** in **2021**, driven by an **89%** increase in operating expenses to **$167.6 million** primarily due to a **$61.9 million** rise in R&D costs and **$18.7 million** in G&A expenses, while recognizing its first collaboration revenue of **$1.9 million** from Vertex, ending **2022** with **$554.8 million** in cash, cash equivalents, and marketable securities projected to fund operations into the second half of **2025** Results of Operations (2022 vs. 2021) | (in millions) | 2022 | 2021 | Change | | :--- | :--- | :--- | :--- | | **Collaboration revenue** | **$1.9** | **$0.0** | **$1.9** | | Research and development | **$130.1** | **$68.2** | **$61.9** | | General and administrative | **$37.5** | **$18.9** | **$18.7** | | **Total operating expenses** | **$167.6** | **$87.1** | **$80.6** | | **Net loss** | **($157.4)** | **($120.3)** | **($37.1)** | - Research and development expenses increased by **$61.9 million** (**91%**) in **2022**, driven by a **$26.6 million** increase in personnel costs from higher headcount, a **$9.3 million** increase in preclinical study costs, and a **$3.0 million** increase in clinical trial costs for the heart-1 trial[822](index=822&type=chunk)[823](index=823&type=chunk) - General and administrative expenses rose by **$18.7 million** (**99%**) in **2022**, primarily due to a **$12.9 million** increase in personnel and facility costs to support growth and a **$3.8 million** increase in legal and professional fees[824](index=824&type=chunk)[830](index=830&type=chunk) - The company ended **2022** with **$554.8 million** in cash, cash equivalents, and marketable securities, which is projected to fund operations and capital expenditures into the second half of **2025**[802](index=802&type=chunk)[829](index=829&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=159&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk stems from interest rate changes affecting its **$554.8 million** in cash, cash equivalents, and marketable securities, though a **10%** rate change is not expected to be material due to their short-term nature, with limited but potentially increasing exposure to foreign currency exchange risk and no material effect from inflation in **2022** - The primary market risk is interest rate risk on cash, cash equivalents, and marketable securities (**$554.8 million** total as of **Dec 31, 2022**), however, due to short-term maturities, the impact of a **10%** rate change is not considered material[875](index=875&type=chunk) - Exposure to foreign currency exchange risk is not currently significant but may increase as the company contracts with more vendors located outside the United States[876](index=876&type=chunk) [Controls and Procedures](index=160&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures and internal control over financial reporting were effective as of **December 31, 2022**, with no material changes in the fourth quarter of **2022**, and as an emerging growth company, this report does not include an attestation report from the registered public accounting firm - Management concluded that the company's disclosure controls and procedures were effective as of **December 31, 2022**[882](index=882&type=chunk) - Management concluded that the company's internal control over financial reporting was effective as of **December 31, 2022**[883](index=883&type=chunk) - As an emerging growth company, the annual report does not include an auditor's attestation report on internal control over financial reporting[884](index=884&type=chunk) PART III [Directors, Executive Officers and Corporate Governance](index=162&type=section&id=Item%2010.%20Directors%2C%20Executive%20Officers%20and%20Corporate%20Governance) Information regarding directors, executive officers, and corporate governance will be incorporated by reference from the company's definitive proxy statement for its **2023** Annual Meeting of Stockholders - Information regarding directors, executive officers, and corporate governance is incorporated by reference from the definitive proxy statement for the **2023** Annual Meeting of Stockholders[889](index=889&type=chunk) [Executive Compensation](index=162&type=section&id=Item%2011.%20Executive%20Compensation) Information regarding executive compensation will be incorporated by reference from the company's definitive proxy statement for its **2023** Annual Meeting of Stockholders - Information regarding executive compensation is incorporated by reference from the definitive proxy statement for the **2023** Annual Meeting of Stockholders[891](index=891&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=162&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Information regarding security ownership of certain beneficial owners and management will be incorporated by reference from the company's definitive proxy statement for its **2023** Annual Meeting of Stockholders - Information regarding security ownership is incorporated by reference from the definitive proxy statement for the **2023** Annual Meeting of Stockholders[892](index=892&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=162&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions%2C%20and%20Director%20Independence) Information concerning related party transactions and director independence will be incorporated by reference from the company's definitive proxy statement for its **2023** Annual Meeting of Stockholders - Information regarding certain relationships, related transactions, and director independence is incorporated by reference from the definitive proxy statement for the **2023** Annual Meeting of Stockholders[893](index=893&type=chunk) [Principal Accounting Fees and Services](index=162&type=section&id=Item%2014.%20Principal%20Accounting%20Fees%20and%20Services) Information regarding principal accounting fees and services will be incorporated by reference from the company's definitive proxy statement for its **2023** Annual Meeting of Stockholders - Information regarding principal accounting fees and services is incorporated by reference from the definitive proxy statement for the **2023** Annual Meeting of Stockholders[894](index=894&type=chunk) PART IV [Exhibits, Financial Statement Schedules](index=163&type=section&id=Item%2015.%20Exhibits%2C%20Financial%20Statement%20Schedules) This section details the financial statements of Verve Therapeutics, Inc., including the report from Ernst & Young LLP, along with a comprehensive list of exhibits filed as part of the Annual Report on Form 10-K, while financial statement schedules have been omitted as not required - This item contains the financial statements of Verve Therapeutics, Inc. and the report from its independent registered public accounting firm, Ernst & Young LLP[896](index=896&type=chunk) - A detailed list of exhibits filed with the report is provided, including the Restated Certificate of Incorporation, Bylaws, material contracts such as license agreements with Beam and Vertex, and executive employment agreements[898](index=898&type=chunk)[899](index=899&type=chunk)

1 Verve Therapeutics Disrupting the Care of Cardiovascular Disease Through Single-course Gene Editing Medicines January 2023 Forward looking statements This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regarding the timing and availability of clinical data from the Company's heart-1 clinical trial, the timing of initiation of clinical trials of VERVE-201, th ...

1 | --- | --- | |-------|-------------------------------------------------------------------------------| | | | | | VERVE-101 – an investigational singlecourse gene editing medicine targeting | | | PCSK9 – durably and potently lowers | | | PCSK9 and LDL-C concentrations in | | | non-human primates | | | Amit V. Khera MD, MSc Verve Therapeutics | | | Paul Dudley White International Scholar Awardee | Forward looking statements This press release contains "forward-looking statements" within the meaning of the ...

PART I. FINANCIAL INFORMATION [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section presents Verve Therapeutics' unaudited condensed consolidated financial statements, including balance sheets, statements of operations, and cash flows, along with notes detailing key accounting policies and recent financial activities [Condensed Consolidated Balance Sheets](index=7&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | Sep 30, 2022 | Dec 31, 2021 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $277,019 | $64,330 | | Marketable securities | $273,691 | $296,112 | | Total current assets | $561,630 | $367,128 | | Total assets | $673,355 | $384,124 | | **Liabilities & Stockholders' Equity** | | | | Total current liabilities | $29,018 | $22,024 | | Total liabilities | $125,412 | $26,772 | | Total stockholders' equity | $547,943 | $357,352 | | Total liabilities and stockholders' equity | $673,355 | $384,124 | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) Statement of Operations Highlights (in thousands) | Metric | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | | :--- | :--- | :--- | | Collaboration revenue | $929 | $0 | | Research and development | $92,811 | $42,263 | | General and administrative | $26,095 | $12,264 | | Loss from operations | ($117,977) | ($54,527) | | Net loss | ($116,302) | ($88,977) | | Net loss per common share | ($2.26) | ($4.52) | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Cash Flow Summary for Nine Months Ended September 30 (in thousands) | Cash Flow Activity | 2022 | 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | ($89,440) | ($49,837) | | Net cash provided by (used in) investing activities | $12,358 | ($182,113) | | Net cash provided by financing activities | $289,358 | $375,865 | | **Increase in cash, cash equivalents and restricted cash** | **$212,276** | **$143,915** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) These notes detail the company's accounting policies and financial activities, including the Vertex collaboration, recent financing through a follow-on offering and ATM program, and ongoing license and lease obligations - In July 2022, Verve entered into a strategic collaboration with Vertex Pharmaceuticals, receiving a **$25.0 million** upfront payment and a **$35.0 million** equity investment through a private placement[31](index=31&type=chunk)[32](index=32&type=chunk)[102](index=102&type=chunk) - The company completed a follow-on public offering in July 2022, raising net proceeds of approximately **$242.9 million**[33](index=33&type=chunk)[118](index=118&type=chunk) - An Open Market Sale Agreement (ATM offering) was established in July 2022, and as of September 30, 2022, the company had sold shares for aggregate net proceeds of **$7.3 million**[34](index=34&type=chunk)[35](index=35&type=chunk) - As of September 30, 2022, the company's cash, cash equivalents, and marketable securities of **$550.7 million** are expected to be sufficient to fund operations beyond the next 12 months[36](index=36&type=chunk) - The company recognized its first-ever revenue of **$0.9 million** during the three and nine months ended September 30, 2022, related to research services performed under the Vertex Agreement[111](index=111&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=28&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) The MD&A section provides management's perspective on the company's financial condition and results of operations, detailing business developments, including the heart-1 clinical trial and FDA clinical hold, analyzing increased R&D and G&A expenses, and reviewing liquidity and capital resources [Recent Developments](index=30&type=section&id=Recent%20Developments) This section outlines key recent developments, including the initiation of the heart-1 clinical trial for VERVE-101, the subsequent FDA clinical hold in the U.S., and progress on the VERVE-201 program - The first patient was dosed with VERVE-101 in the heart-1 clinical trial in July 2022, with enrollment ongoing in New Zealand and the United Kingdom[154](index=154&type=chunk) - On November 4, 2022, the FDA placed the company's Investigational New Drug (IND) application for VERVE-101 on a clinical hold in the United States[155](index=155&type=chunk) - The company plans to report initial safety and pharmacodynamic data for the dose-escalation portion of the heart-1 study in the second half of 2023[157](index=157&type=chunk) - VERVE-201, targeting the ANGPTL3 gene, has initiated IND-enabling studies and will utilize a proprietary GalNAc-LNP delivery technology to enable uptake in the liver of HoFH patients[158](index=158&type=chunk)[159](index=159&type=chunk)[160](index=160&type=chunk) [Results of Operations](index=34&type=section&id=Results%20of%20Operations) Comparison of Operating Expenses (in thousands) | Expense Category | Three Months Ended Sep 30, 2022 | Three Months Ended Sep 30, 2021 | Change | | :--- | :--- | :--- | :--- | | Research and development | $35,197 | $17,495 | $17,702 | | General and administrative | $9,592 | $6,007 | $3,585 | | **Total operating expenses** | **$44,789** | **$23,502** | **$21,287** | - The **$17.7 million** increase in R&D expenses for Q3 2022 compared to Q3 2021 was primarily driven by a **$6.8 million** increase in personnel-related costs due to higher headcount, a **$2.8 million** increase in preclinical study costs, and a **$0.9 million** increase in clinical trial costs for the heart-1 trial[189](index=189&type=chunk)[191](index=191&type=chunk) Comparison of Nine-Month Operating Results (in thousands) | Metric | Nine Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2021 | Change | | :--- | :--- | :--- | :--- | | Collaboration revenue | $929 | $0 | $929 | | Research and development | $92,811 | $42,263 | $50,548 | | General and administrative | $26,095 | $12,264 | $13,831 | | **Net loss** | **($116,302)** | **($88,977)** | **($27,325)** | [Liquidity and Capital Resources](index=38&type=section&id=Liquidity%20and%20Capital%20Resources) - As of September 30, 2022, the company had **$550.7 million** in cash, cash equivalents, and marketable securities[203](index=203&type=chunk) - Management believes that existing cash, cash equivalents, and marketable securities will be sufficient to fund operating expenses and capital expenditure requirements into the second half of 2025[217](index=217&type=chunk) - For the nine months ended September 30, 2022, net cash used in operating activities was **$89.4 million**, while net cash provided by financing activities was **$289.4 million**, primarily from stock offerings and the Vertex private placement[209](index=209&type=chunk)[210](index=210&type=chunk)[214](index=214&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=43&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section discusses the company's exposure to market risks, primarily interest rate risk on its cash and marketable securities, and notes no significant foreign currency or inflation risk - The company's primary market risk is related to changes in interest rates on its cash, cash equivalents (**$277.0 million**), and marketable securities (**$273.7 million**)[240](index=240&type=chunk) - Due to the short-term maturities and low-risk profile of its investments, management believes a 10% change in interest rates would not materially affect their fair market value[240](index=240&type=chunk) - The company is not currently exposed to significant foreign currency exchange risk or inflation risk[241](index=241&type=chunk)[242](index=242&type=chunk) [Item 4. Controls and Procedures](index=43&type=section&id=Item%204.%20Controls%20and%20Procedures) This section addresses the company's disclosure controls and internal control over financial reporting, concluding their effectiveness and noting no material changes during the quarter - Based on an evaluation as of September 30, 2022, the principal executive officer and principal financial officer concluded that the company's disclosure controls and procedures were effective[244](index=244&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended September 30, 2022, that have materially affected, or are reasonably likely to materially affect, internal controls[246](index=246&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=45&type=section&id=Item%201.%20Legal%20Proceedings) The company states that it is not currently a party to any material legal proceedings - As of the filing date, Verve Therapeutics is not a party to any material legal proceedings[248](index=248&type=chunk) [Item 1A. Risk Factors](index=45&type=section&id=Item%201A.%20Risk%20Factors) This section comprehensively outlines the company's risks, including a history of losses, the early stage of development, the FDA clinical hold on VERVE-101, the unproven nature of gene editing technology, reliance on third parties, and intense competition - The company has a history of significant losses, with a net loss of **$116.3 million** for the nine months ended September 30, 2022, and expects to incur losses for the foreseeable future, requiring substantial additional funding[250](index=250&type=chunk)[257](index=257&type=chunk) - A significant risk is the FDA's clinical hold on the IND application for VERVE-101 in the United States, which could delay or prevent the initiation of the U.S. clinical trial and impair the ability to obtain FDA approval[293](index=293&type=chunk) - The company's approach using gene editing, particularly base editing, is a novel and largely unproven technology that is not yet clinically validated as safe and efficacious for human therapeutic use[277](index=277&type=chunk) - The company relies on third parties for manufacturing, research, and clinical testing, which reduces control and introduces risks of delays, non-performance, and supply chain disruptions[335](index=335&type=chunk)[340](index=340&type=chunk) - Verve faces substantial competition from major pharmaceutical and biotech companies with approved products or candidates in development for cardiovascular disease, including those targeting PCSK9 and ANGPTL3[442](index=442&type=chunk)[444](index=444&type=chunk)[445](index=445&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=106&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section reports no unregistered equity sales during the period and confirms that the net proceeds from the June 2021 IPO remain unused and invested in short-term instruments - The company received net proceeds of **$281.6 million** from its Initial Public Offering (IPO) in June 2021[586](index=586&type=chunk) - As of September 30, 2022, the company had not used any of the net proceeds from the IPO, which are invested in money market funds and short-term investments[586](index=586&type=chunk) [Item 6. Exhibits](index=107&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including corporate governance documents, material agreements like the Amended Beam Agreement and Vertex Agreement, and CEO/CFO certifications - Key agreements filed as exhibits include the Amended and Restated Collaboration and License Agreement with Beam Therapeutics, Inc. and the Strategic Collaboration and License Agreement with Vertex Pharmaceuticals Incorporated[588](index=588&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2022 OR ☐ TRANSITION REPORT PURSUANT TO 2SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from ____________ to ____________ Commission File Number: 001-40489 VERVE THERAPEUTICS , INC. (Exact Name of Registrant as Specified in its Charter) Delaware 82-4800 ...