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 SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________________ to __________________ Commission File Number: 001-39979 VOR BIOPHARMA INC. (Exact Name of Registrant as Specified in its Charter) (State or ot ...

[PART I. FINANCIAL INFORMATION](index=6&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) The company presents its unaudited condensed consolidated financial statements for Q1 2022 and Q1 2021 [Condensed Consolidated Balance Sheets](index=6&type=section&id=Condensed%20Consolidated%20Balance%20Sheets) Total assets decreased to $234.3 million while total liabilities increased to $39.8 million as of Q1 2022 Condensed Consolidated Balance Sheet Highlights (in thousands) | Balance Sheet Item | March 31, 2022 | December 31, 2021 | | :--- | :--- | :--- | | Cash and cash equivalents | $90,042 | $119,801 | | Marketable securities | $91,575 | $87,668 | | **Total Assets** | **$234,269** | **$242,590** | | Total current liabilities | $18,145 | $10,153 | | **Total Liabilities** | **$39,822** | **$26,327** | | **Total Stockholders' Equity** | **$194,447** | **$216,263** | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) The company's net loss increased to $22.7 million in Q1 2022, driven by higher R&D and G&A expenses Statement of Operations Highlights (in thousands, except per share data) | Operating Item | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | | :--- | :--- | :--- | | Research and development | $15,280 | $8,941 | | General and administrative | $7,520 | $4,789 | | **Total operating expenses** | **$22,800** | **$13,730** | | **Loss from operations** | **$(22,800)** | **$(13,730)** | | **Net loss** | **$(22,737)** | **$(13,723)** | | Net loss per share, basic and diluted | $(0.61) | $(0.67) | [Condensed Consolidated Statements of Cash Flows](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows) Net cash used in operations increased to $22.9 million in Q1 2022, reflecting a higher net loss Cash Flow Highlights (in thousands) | Cash Flow Activity | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | | :--- | :--- | :--- | | Net cash used in operating activities | $(22,904) | $(17,649) | | Net cash used in investing activities | $(7,055) | $(1,171) | | Net cash provided by financing activities | $200 | $232,848 | | **Net (decrease) increase in cash** | **$(29,759)** | **$214,028** | [Notes to Unaudited Condensed Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Unaudited%20Condensed%20Consolidated%20Financial%20Statements) Notes detail key accounting policies, cash sufficiency, stock compensation, and significant new lease agreements - The company is a clinical-stage cell and genome engineering company focused on treating hematological malignancies and anticipates incurring significant operating losses for the next several years[26](index=26&type=chunk)[28](index=28&type=chunk) - Management believes existing **cash, cash equivalents, and marketable securities of $181.6 million** are sufficient to fund operations for at least one year[28](index=28&type=chunk)[65](index=65&type=chunk) - The company entered into two lease amendments for its Cambridge facility, involving **$8.4 million and $21.9 million** in total fixed rent payments[49](index=49&type=chunk)[50](index=50&type=chunk)[51](index=51&type=chunk) Stock-Based Compensation Expense (in thousands) | Expense Category | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | | :--- | :--- | :--- | | Research and development | $796 | $449 | | General and administrative | $950 | $442 | | **Total** | **$1,746** | **$891** | [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=16&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) The company discusses its clinical programs, financial results, and capital resources, including its funding runway - The company is a clinical-stage cell and genome engineering company focused on treating blood cancers by engineering hematopoietic stem cells (HSCs)[59](index=59&type=chunk) - The lead product candidate, VOR33, is in a Phase 1/2a trial, with **initial clinical data anticipated in H2 2022**[60](index=60&type=chunk) - The company plans to submit an IND for its VCAR33 ALLO program in the **first half of 2023**[61](index=61&type=chunk) - As of March 31, 2022, the company had **$181.6 million in cash, cash equivalents, and marketable securities**, expected to fund operations into Q4 2023[65](index=65&type=chunk) - In March 2022, the company filed a **$350.0 million universal shelf registration** and a **$125.0 million at-the-market (ATM) facility**[86](index=86&type=chunk) [Results of Operations](index=19&type=section&id=Results%20of%20Operations) Operating expenses increased to $22.8 million in Q1 2022, driven by higher personnel and external R&D costs Comparison of Operating Results (in thousands) | Item | Three Months Ended March 31, 2022 | Three Months Ended March 31, 2021 | Change | | :--- | :--- | :--- | :--- | | Research and development | $15,280 | $8,941 | $6,339 | | General and administrative | $7,520 | $4,789 | $2,731 | | **Total operating expenses** | **$22,800** | **$13,730** | **$9,070** | | **Net loss** | **$(22,737)** | **$(13,723)** | **$(9,014)** | - The **$6.4 million increase in R&D expenses** was primarily due to a $2.5 million rise in external costs, a $2.7 million increase in personnel costs, and a $0.6 million increase in facilities expenses[82](index=82&type=chunk) - The **$2.7 million increase in G&A expenses** was driven by a $1.7 million rise in personnel costs, a $0.5 million increase in professional fees, and a $0.5 million increase in facilities expenses[83](index=83&type=chunk) [Liquidity and Capital Resources](index=20&type=section&id=Liquidity%20and%20Capital%20Resources) The company has funded operations primarily through equity sales and established new financing facilities in March 2022 - The company has funded operations with **$344.0 million in net proceeds** from equity and debt financings as of March 31, 2022[85](index=85&type=chunk) - **Net cash used in operating activities increased by $5.3 million** year-over-year, primarily due to higher operating expenses related to advancing VOR33 and increased personnel costs[96](index=96&type=chunk) - **Net cash used in investing activities was $7.1 million** in Q1 2022, consisting of $5.1 million for marketable securities and $2.0 million for property and equipment[97](index=97&type=chunk) - **Net cash provided by financing activities was only $0.2 million** in Q1 2022 from stock option exercises, compared to $232.8 million in Q1 2021 which included IPO proceeds[98](index=98&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=23&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity, which is considered immaterial - Primary market risk exposure is to interest rate changes affecting cash equivalents and marketable securities, but the **impact is considered immaterial** due to their short-term maturities[102](index=102&type=chunk) - The company has **no outstanding debt** as of March 31, 2022, and therefore no related interest rate risk[103](index=103&type=chunk) - Foreign currency exchange risk and inflation are **not considered to have a material effect** on the company's financial statements[104](index=104&type=chunk)[105](index=105&type=chunk) [Controls and Procedures](index=23&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and procedures were effective as of the end of the quarter - Based on an evaluation as of March 31, 2022, the CEO and CFO concluded that the company's **disclosure controls and procedures were effective** at the reasonable assurance level[107](index=107&type=chunk) - There were **no material changes in internal control** over financial reporting during the quarter ended March 31, 2022[108](index=108&type=chunk) [PART II. OTHER INFORMATION](index=24&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=24&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently a party to any material legal proceedings - As of the report date, the company is **not a party to any material legal proceedings**[110](index=110&type=chunk) [Risk Factors](index=24&type=section&id=Item%201A.%20Risk%20Factors) The company faces numerous risks related to its financial position, clinical development, and intellectual property - The company has a history of **significant net losses ($22.7M for Q1 2022)** and expects to incur losses for the foreseeable future[112](index=112&type=chunk) - The company is **substantially dependent on the success** of its two most advanced product candidates, VOR33 and VCAR33 ALLO[135](index=135&type=chunk) - Engineered hematopoietic stem cells (eHSCs) are a **novel, unproven technology** that is not yet clinically validated[131](index=131&type=chunk) - The company is **highly dependent on intellectual property licensed** from third parties like Columbia University and the National Cancer Institute (NCI)[255](index=255&type=chunk)[256](index=256&type=chunk) - The **COVID-19 pandemic has caused delays** in the VOR33 Phase 1/2a clinical trial and could continue to disrupt operations[342](index=342&type=chunk) [Risks Related to Financial Position and Capital Needs](index=24&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) The company has a history of net losses and will require substantial additional funding to continue operations - The company has incurred significant net losses since inception, with an **accumulated deficit of $152.9 million** as of March 31, 2022[112](index=112&type=chunk) - **Substantial additional funding is required**, as existing cash of $181.6 million is expected to fund operations only into Q4 2023[115](index=115&type=chunk)[116](index=116&type=chunk) - The company's **limited operating history** and lack of commercial products make it difficult to evaluate its future viability[124](index=124&type=chunk) [Risks Related to Discovery, Development, Manufacturing and Commercialization](index=28&type=section&id=Risks%20Related%20to%20Discovery%2C%20Development%2C%20Manufacturing%20and%20Commercialization) The company's novel technology, reliance on lead candidates, and complex manufacturing present significant risks - **eHSCs are a novel technology not yet clinically validated**, and the company's approach is unproven and faces numerous development challenges[131](index=131&type=chunk) - Product candidates could cause **serious adverse events**, such as off-target gene alterations, which could delay or prevent regulatory approval[141](index=141&type=chunk)[143](index=143&type=chunk) - Developing VOR33 for use with an approved therapy like Mylotarg presents complexities, and prospects could be harmed if **Mylotarg is withdrawn from the market**[157](index=157&type=chunk) - **Manufacturing of eHSCs and CAR-T therapies is complex**, susceptible to product loss, and difficult to scale[192](index=192&type=chunk)[193](index=193&type=chunk) [Risks Related to Regulatory Review](index=42&type=section&id=Risks%20Related%20to%20Regulatory%20Review) The company faces an uncertain regulatory landscape for its novel genome engineering technology - Clinical trials may **fail to demonstrate safety and efficacy**, and positive preclinical results are not predictive of clinical success[210](index=210&type=chunk)[212](index=212&type=chunk) - The **regulatory landscape for genome engineering technology is novel and uncertain**, which could lengthen the review process and increase costs[219](index=219&type=chunk)[220](index=220&type=chunk)[222](index=222&type=chunk) - Regulators may not consider the proposed **clinical trial endpoints to be clinically meaningful** for a novel therapy like VOR33[225](index=225&type=chunk) - **Difficulties in enrolling patients** in clinical trials for rare diseases could increase development costs and delay or prevent regulatory approvals[229](index=229&type=chunk) [Risks Related to Third-Party Relationships](index=48&type=section&id=Risks%20Related%20to%20Our%20Relationships%20with%20Third%20Parties) The business model relies heavily on third-party CROs and manufacturers, including single-source suppliers - The company **relies on third parties like CROs** to conduct clinical trials, reducing control over the process[236](index=236&type=chunk)[238](index=238&type=chunk) - The company **relies on third-party manufacturers**, including single-source suppliers, increasing the risk of supply shortages and cost issues[241](index=241&type=chunk)[242](index=242&type=chunk) - **Future collaborations are important** for development and commercialization but pose risks such as loss of control and potential disputes[246](index=246&type=chunk)[247](index=247&type=chunk) [Risks Related to Intellectual Property](index=51&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) The company depends on licensed IP and faces a complex landscape for technologies like CRISPR-Cas9 - The company is **highly dependent on exclusive licenses** from Columbia University and the National Cancer Institute; termination would severely harm the business[255](index=255&type=chunk)[256](index=256&type=chunk)[257](index=257&type=chunk) - The company will need to **acquire licenses for key technologies like CRISPR-Cas9**, for which the IP landscape is complex and unsettled[283](index=283&type=chunk)[284](index=284&type=chunk) - **Third parties may claim infringement**, which could lead to costly litigation and block commercialization[288](index=288&type=chunk)[289](index=289&type=chunk) - Some in-licensed IP was discovered via government funding and may be subject to federal regulations, including **"march-in" rights**[294](index=294&type=chunk) [Risks Related to Regulatory and Legal Compliance](index=60&type=section&id=Risks%20Related%20to%20Regulatory%20and%20Other%20Legal%20Compliance%20Matters) The company faces extensive compliance risks from healthcare laws, reform efforts, and international regulations - Even if approved, products will be subject to **continual and extensive regulation**, including potential marketing restrictions and post-marketing study requirements[311](index=311&type=chunk) - Relationships with healthcare providers are subject to complex **anti-kickback, fraud and abuse laws** with severe penalties for violations[322](index=322&type=chunk)[323](index=323&type=chunk) - **Healthcare reform efforts** could increase costs, affect pricing, and reduce demand for the company's products[327](index=327&type=chunk)[328](index=328&type=chunk)[329](index=329&type=chunk) - International operations are subject to laws like the **Foreign Corrupt Practices Act (FCPA)** and stringent data privacy laws[334](index=334&type=chunk)[338](index=338&type=chunk) [Risks Related to COVID-19, Operations, and IT](index=66&type=section&id=Risks%20Related%20to%20COVID-19%2C%20Employee%20Matters%2C%20Managing%20Growth%20and%20Information%20Technology) The COVID-19 pandemic, talent retention, growth management, and IT security pose operational challenges - The **COVID-19 pandemic has caused delays** in clinical trial enrollment for VOR33 and may continue to adversely affect operations[340](index=340&type=chunk)[342](index=342&type=chunk) - The company is **highly dependent on its key executives** and faces intense competition for qualified personnel[344](index=344&type=chunk)[345](index=345&type=chunk) - The company expects to **expand its operations**, which may strain resources and lead to operational mistakes if not managed effectively[347](index=347&type=chunk)[348](index=348&type=chunk) - Internal and third-party computer systems are **vulnerable to security breaches**, which could disrupt development and compromise sensitive data[350](index=350&type=chunk) [Risks Related to Common Stock Ownership](index=69&type=section&id=Risks%20Related%20to%20the%20Ownership%20of%20Our%20Common%20Stock) Common stock ownership risks include price volatility, insider control, and reduced disclosure requirements - The **market price of the common stock is likely to be volatile**, and an active trading market may not be sustained[353](index=353&type=chunk)[354](index=354&type=chunk) - **Insiders hold a substantial amount of common stock**, giving them significant influence and potentially preventing a change of control[360](index=360&type=chunk) - The company's status as an **"emerging growth company"** allows it to rely on reduced disclosure requirements[364](index=364&type=chunk)[366](index=366&type=chunk) - The company **does not expect to pay dividends** for the foreseeable future, so stockholders must rely on capital appreciation[371](index=371&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=73&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company details the use of its $186.3 million in net proceeds from its February 2021 IPO - The company closed its IPO on February 9, 2021, receiving **net proceeds of approximately $186.3 million**[379](index=379&type=chunk)[380](index=380&type=chunk) - As of March 31, 2022, **$4.0 million of the IPO net proceeds have been used** to fund development programs and for general corporate purposes[381](index=381&type=chunk) - In January 2022, the company **repurchased 7,660 shares of unvested common stock** from a former employee[384](index=384&type=chunk) [Exhibits](index=75&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the report, including an ATM agreement and required officer certifications - Lists exhibits filed with the report, including an **Open Market Sale Agreement with Jefferies LLC**, CEO/CFO certifications, and XBRL data files[386](index=386&type=chunk)

| --- | --- | --- | --- | --- | |----------------------------------------------------------|-------|-------|-------|-------| | | | | | | | | | | | | | | | | | | | | | | | | | Cure blood cancers through cell and genome engineering | | | | | | March 2022 | | | | | | Confidential | | | | | Disclaimer This presentation (the "Presentation") contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 about Vor Biopharma Inc. ("Vor," "Vor Bio" or the "Company") th ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number: 001-39979 VOR BIOPHARMA INC. (Exact name of registrant as specified in its charter) Delaware 81-1591163 (State or other jurisdiction of ...

| --- | --- | |---------------------------------------------|-------| | | | | | | | Cure blood cancers through cell and genome | | 1 Confidential Disclaimer This presentation (the "Presentation") contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995 about Vor Biopharma Inc. ("Vor" or the "Company") that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Presentation, are forward lo ...

Vor Biopharma's Platform and Vision - Vor Biopharma is focused on curing blood cancers through cell and genome engineering[11] - The company's paradigm involves engineering hematopoietic stem cells (eHSCs) to remove target expression on healthy cells, making cancer killing more specific[7] - Vor's platform combines genome engineering with hematopoietic stem cells and CAR-T-cell therapies to create treatment-resistant transplants[10] VOR33 and VCAR33 Programs - VOR33 aims to provide treatment-resistant HSC transplants[19] - VCAR33 is an allogeneic CAR-T therapy with potential advantages for healthy donor cell source and addressing tumor heterogeneity[24] - The VCAR33 construct is being studied in a Phase 1/2 clinical trial sponsored by the National Marrow Donor Program (NMDP)[26] Clinical Development and Milestones - Vor anticipates initial clinical data for VOR33 in the first half of 2022[25,73] - The company plans to file an IND for the VOR33 + VCAR33 treatment system in the second half of 2022, following initial VOR33 and NMDP clinical data[25,73] - A Phase 1/2a clinical trial (VBP101) is underway, evaluating VOR33 + Mylotarg in transplant-eligible AML patients at high risk of relapse[48,49] Market and Financial Position - Approximately 20,000 AML cases are diagnosed annually[13] - For patients who relapse post-transplant, 2-year survival is less than 20%[17] - Vor Biopharma secured $203 million in gross proceeds from its IPO in February 2021, providing a cash runway into mid-2023[73]

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 September 30, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________________ to __________________ Commission File Number: 001-39979 VOR BIOPHARMA INC. (Exact Name of Registrant as Specified in its Charter) | Delaw ...

Company Vision and Platform - Vor Biopharma's vision is to cure blood cancer through transformative cell engineering, utilizing a VOR platform that combines Hematopoietic Stem Cells (HSCs), genome engineering, and CAR-T cell therapies[6] - The company is developing engineered HSCs (eHSCs) as a solution to remove target expression on healthy cells, making cancer killing more cancer-specific[7, 8] - Vor's technology-driven platform vision includes VOR33 + VCAR33, multiplexed HSCs + dual-specific CAR-T, exploring targets beyond CD33, and exploring next-generation technologies like genome engineering and companion therapeutics[49] Pipeline and Clinical Development - The company's pipeline includes VOR33 (eHSC targeting CD33) for AML, MDS, and MPN, and VCAR33 (CAR-T targeting CD33) as a bridge-to-transplant for AML[13] - VOR33 is expected to have initial clinical data in the first half of 2022, and a development candidate selection for MDS and MPN[13] - The VOR33/VCAR33 treatment system is planned for an IND filing in the second half of 2022, following initial VOR33 and NMDP clinical data[13] VOR33 and CD33 Target - Preclinical validation of CD33 deletion in HSCs shows successful homing, engraftment, survival, and resistance to toxic therapy[18] - VOR33 shows no observed impact on cell populations or function, including B cells, myeloid cells, and phagocytosis[21, 23, 25] - In vitro studies show VOR33 provides resistance to CD33 therapy, with approximately 70-fold resistance to Mylotarg[33] Manufacturing and Clinical Trial - Vor is building a fully integrated manufacturing facility for eHSC and CAR-T drug products, with a streamlined cell manufacturing process for VOR33 that takes approximately 3 days, with a 7-10 day vein-to-vein time[35, 37] - VBP101 is a VOR33+Mylotarg Phase 1/2a clinical trial for transplant-eligible AML patients in remission but at high risk of relapse[38, 39]

[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements (Unaudited)](index=7&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Cash and investments reached **$244.6 million** by June 30, 2021, primarily from IPO and Series B financing, despite a **$32.1 million** net loss Condensed Consolidated Balance Sheet Data (in thousands) | Account | June 30, 2021 | December 31, 2020 | | :--- | :--- | :--- | | Cash and cash equivalents | $169,516 | $48,539 | | Investments | $75,118 | $0 | | Total assets | $275,143 | $75,908 | | Total liabilities | $25,035 | $27,637 | | Total stockholders' equity (deficit) | $250,108 | $(59,065) | Condensed Consolidated Statements of Operations (in thousands) | Metric | Three Months Ended June 30, 2021 | Three Months Ended June 30, 2020 | Six Months Ended June 30, 2021 | Six Months Ended June 30, 2020 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $12,970 | $5,975 | $21,911 | $12,146 | | General and administrative | $5,410 | $2,065 | $10,199 | $3,772 | | **Loss from operations** | **$(18,380)** | **$(8,040)** | **$(32,110)** | **$(15,918)** | | **Net loss** | **$(18,370)** | **$(8,040)** | **$(32,093)** | **$(15,889)** | | Net loss per share | $(0.50) | $(56.85) | $(1.13) | $(115.83) | Condensed Consolidated Statements of Cash Flows (in thousands) | Cash Flow Activity | For the Six Months Ended June 30, 2021 | For the Six Months Ended June 30, 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(34,214) | $(14,413) | | Net cash used in investing activities | $(77,212) | $(1,097) | | Net cash provided by financing activities | $232,403 | $81,954 | - On February 9, 2021, the company completed its IPO, selling 11,302,219 shares of common stock at $18.00 per share, resulting in net proceeds of approximately **$186.3 million** after deducting underwriting discounts and commissions[26](index=26&type=chunk) - Upon the IPO closing, all outstanding shares of redeemable convertible preferred stock automatically converted into an aggregate of 24,924,501 shares of common stock[27](index=27&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=20&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses strategic focus on VOR33 and VCAR33, increased operating expenses, and substantial IPO and Series B funding expected to last into Q1 2023 [Overview](index=20&type=section&id=Overview) Vor Biopharma develops VOR33 and VCAR33 for hematological malignancies, with VOR33's IND accepted and initial Phase 1/2a data expected in H1 2022 - The company is developing VOR33, an engineered hematopoietic stem cell (eHSC) product candidate, and VCAR33, a companion CAR-T therapy, to treat acute myeloid leukemia (AML) and other hematological malignancies[69](index=69&type=chunk)[70](index=70&type=chunk) - The Investigational New Drug (IND) application for VOR33 was accepted by the FDA in January 2021, and a no objection letter was received from Health Canada in April 2021 for the Phase 1/2a clinical trial[70](index=70&type=chunk) - Initial clinical data from the VOR33 trial is expected in the first half of 2022, and the company plans to submit an IND for the VOR33/VCAR33 Treatment System in the second half of 2022[70](index=70&type=chunk)[72](index=72&type=chunk) - The company has experienced delays in its Phase 1/2a trial for VOR33 in part due to the COVID-19 pandemic[76](index=76&type=chunk) [Results of Operations](index=24&type=section&id=Results%20of%20Operations) Operating expenses significantly increased in H1 2021, leading to a **$32.1 million** operating loss, primarily due to higher R&D and G&A costs Comparison of Operating Expenses (in thousands) | Expense Category | Six Months Ended June 30, 2021 | Six Months Ended June 30, 2020 | Change | | :--- | :--- | :--- | :--- | | Research and development | $21,911 | $12,146 | $9,765 | | General and administrative | $10,199 | $3,772 | $6,427 | | **Total operating expenses** | **$32,110** | **$15,918** | **$16,192** | - The **$9.8 million** increase in R&D expenses for the six months ended June 30, 2021, was primarily due to a **$6.0 million** increase in personnel costs from higher headcount, a **$2.2 million** increase in external clinical and preclinical study costs, and a **$1.6 million** increase in facilities and other expenses[91](index=91&type=chunk) - The **$6.4 million** increase in G&A expenses for the six months ended June 30, 2021, was mainly due to a **$3.0 million** increase in personnel costs, a **$2.6 million** increase in professional fees (consultants, insurance), and a **$0.8 million** increase in facilities and other expenses[93](index=93&type=chunk) [Liquidity and Capital Resources](index=26&type=section&id=Liquidity%20and%20Capital%20Resources) The company held **$244.6 million** in cash and investments by June 30, 2021, primarily from IPO and Series B financing, expected to fund operations into Q1 2023 - As of June 30, 2021, the company had cash, cash equivalents, and investments of **$244.6 million**[75](index=75&type=chunk)[102](index=102&type=chunk) - In the first half of 2021, the company raised approximately **$232.4 million** from financing activities, including **$45.4 million** from the final tranche of its Series B preferred stock financing and **$187.0 million** in net proceeds from its IPO[95](index=95&type=chunk)[101](index=101&type=chunk) - Existing cash is expected to fund operating expenses and capital expenditure requirements into at least the **first quarter of 2023**[75](index=75&type=chunk)[103](index=103&type=chunk) - In June 2021, the company entered into two lease amendments to expand its corporate office and laboratory space, resulting in future fixed payment commitments of **$31.3 million** not yet recorded on the balance sheet[105](index=105&type=chunk)[106](index=106&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=28&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) Primary market risk is immaterial interest rate sensitivity on short-term cash and investments, with no significant foreign currency or inflation risk - The primary market risk is interest rate sensitivity on cash equivalents and investments, but the impact is considered immaterial due to their short-term maturities[109](index=109&type=chunk) - The company is not currently exposed to significant foreign currency exchange risk as operations are located in the U.S. and expenses are denominated in U.S. dollars[111](index=111&type=chunk) [Item 4. Controls and Procedures](index=28&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of June 30, 2021, with no material changes to internal control over financial reporting during the quarter - Management concluded that as of June 30, 2021, the company's disclosure controls and procedures were effective at the reasonable assurance level[114](index=114&type=chunk) - There were no changes in internal control over financial reporting during the quarter ended June 30, 2021, that have materially affected, or are reasonably likely to materially affect, internal controls[115](index=115&type=chunk) [PART II. OTHER INFORMATION](index=30&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Item 1. Legal Proceedings](index=30&type=section&id=Item%201.%20Legal%20Proceedings) The company is not currently involved in any material legal proceedings, though ordinary course litigation may arise - The company is not currently a party to any material legal proceedings[118](index=118&type=chunk) [Item 1A. Risk Factors](index=30&type=section&id=Item%201A.%20Risk%20Factors) The company faces extensive risks including net losses, funding needs, unproven eHSC technology, manufacturing complexity, regulatory uncertainties, third-party reliance, and intellectual property challenges [Risks Related to Financial Position and Capital Needs](index=30&type=section&id=Risks%20Related%20to%20Our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) The company has significant net losses and accumulated deficit, expects continued losses, requires substantial additional funding, and its limited operating history complicates future viability assessment - The company has incurred significant net losses since inception, with a net loss of **$32.1 million** for the six months ended June 30, 2021, and an accumulated deficit of **$93.3 million** as of that date[120](index=120&type=chunk) - The company will need substantial additional funding and may be forced to delay, reduce, or eliminate research and development programs if unable to raise capital when needed[123](index=123&type=chunk) - The company has a limited operating history, has not completed any clinical trials, and has no history of commercializing products, making it difficult to assess its future viability[133](index=133&type=chunk) [Risks Related to Discovery, Development, and Commercialization](index=37&type=section&id=Risks%20Related%20to%20Discovery,%20Development,%20Manufacturing%20and%20Commercialization) Risks include unproven eHSC technology, dependence on VOR33 and VCAR33, complex manufacturing, intense competition, and potential market acceptance or reimbursement issues - Engineered hematopoietic stem cells (eHSCs) are a novel technology that is not yet clinically validated for human use, and the company's approach is unproven[142](index=142&type=chunk) - The company is substantially dependent on the success of its two most advanced product candidates, VOR33 and VCAR33[146](index=146&type=chunk) - The company's product candidates are complex and difficult to manufacture, which could lead to delays, limit supply, or otherwise harm the business[207](index=207&type=chunk) - The company faces significant competition, and competitors may develop safer, more effective, or less expensive therapies or achieve regulatory approval sooner[184](index=184&type=chunk) [Risks Related to Regulatory Review](index=54&type=section&id=Risks%20Related%20to%20Regulatory%20Review) Regulatory hurdles include potential clinical trial failures, an uncertain genome engineering landscape, and risk that trial endpoints may not be deemed clinically meaningful by authorities - Clinical trials may fail to demonstrate safety and efficacy, and the company may incur additional costs or be unable to complete development and commercialization[222](index=222&type=chunk) - Genome engineering technology is novel, and the regulatory landscape is uncertain and may change, making it difficult to predict the time and cost of obtaining regulatory approval[231](index=231&type=chunk) - There is a risk that regulatory authorities like the FDA may not consider the endpoints of the company's clinical trials to provide clinically meaningful results due to the novel technology and mechanisms of action[238](index=238&type=chunk) [Risks Related to Third-Party Relationships](index=63&type=section&id=Risks%20Related%20to%20Our%20Relationships%20with%20Third%20Parties) Heavy reliance on third parties for research, clinical trials, and manufacturing, including single-source suppliers, poses risks of performance issues, delays, and supply disruptions - The company relies on third parties like CROs to conduct clinical trials, and these parties may not perform satisfactorily, potentially delaying product development[249](index=249&type=chunk) - The company relies on third-party manufacturers, including single-source suppliers, for materials, which increases the risk of insufficient quantities or supply disruptions that could impair development[256](index=256&type=chunk) - Future collaborations for research, development, and commercialization are uncertain and, if unsuccessful, could prevent the company from capitalizing on market potential[262](index=262&type=chunk) [Risks Related to Intellectual Property](index=67&type=section&id=Risks%20Related%20to%20Our%20Intellectual%20Property) Success depends on in-licensed IP, with risks including license termination, challenges in acquiring key technology rights like CRISPR-Cas9, and potential third-party infringement claims - The company is highly dependent on intellectual property licensed from third parties, including Columbia University for VOR33 and the National Cancer Institute (NCI) for VCAR33, and termination of these licenses would significantly harm the business[270](index=270&type=chunk)[271](index=271&type=chunk)[272](index=272&type=chunk) - The company may not be successful in acquiring or in-licensing necessary rights to key technologies, such as CRISPR-Cas9, which are required for commercialization of its product candidates[297](index=297&type=chunk)[298](index=298&type=chunk) - Third-party claims of intellectual property infringement could prevent or delay product development, and the company is aware of third-party patents that may be construed to cover its eHSC technology[303](index=303&type=chunk)[304](index=304&type=chunk) [Risks Related to Regulatory and Other Legal Compliance](index=79&type=section&id=Risks%20Related%20to%20Regulatory%20and%20Other%20Legal%20Compliance%20Matters) Risks include challenges in foreign marketing approvals, ongoing regulatory compliance, exposure to complex healthcare laws, and potential impacts from healthcare reform on costs and pricing - Failure to obtain marketing approval in foreign jurisdictions would prevent product sales and impair the ability to generate revenue[326](index=326&type=chunk) - Relationships with healthcare providers and payors are subject to anti-kickback, fraud and abuse, and other healthcare laws, which could expose the company to criminal and civil penalties[339](index=339&type=chunk) - Healthcare reform legislation, such as the ACA, may increase the difficulty and cost of obtaining marketing approval and commercializing products, and could negatively affect prices[345](index=345&type=chunk)[346](index=346&type=chunk) [Risks Related to COVID-19, Personnel, Growth, and IT](index=87&type=section&id=Risks%20Related%20to%20COVID-19,%20Employee%20Matters,%20Managing%20Growth%20and%20Information%20Technology) Risks include COVID-19 disruptions, dependence on key personnel, challenges in managing growth, and vulnerability of IT systems to security breaches - The COVID-19 pandemic has caused delays in activating clinical sites for the VOR33 Phase 1/2a trial and could continue to disrupt clinical trials, supply chains, and regulatory reviews[356](index=356&type=chunk)[358](index=358&type=chunk) - The company is highly dependent on its key executives and its ability to attract and retain qualified scientific, clinical, and manufacturing personnel[359](index=359&type=chunk)[360](index=360&type=chunk) - Internal computer systems and those of third-party vendors are vulnerable to security breaches, which could result in material disruption of product development programs and business operations[365](index=365&type=chunk) [Risks Related to Common Stock Ownership](index=90&type=section&id=Risks%20Related%20to%20the%20Ownership%20of%20Our%20Common%20Stock) Common stock ownership risks include price volatility, lock-up expiration impact, substantial insider control, reduced disclosure as an 'emerging growth company,' and charter provisions hindering change of control - The market price of the company's common stock may be volatile due to factors such as clinical trial results, regulatory developments, and market conditions[370](index=370&type=chunk) - Insiders, including executive officers, directors, and 5%+ stockholders, have substantial control over the company, which could limit other stockholders' ability to influence key transactions[376](index=376&type=chunk) - The company is an "emerging growth company" and a "smaller reporting company," allowing it to rely on reduced disclosure requirements, which may make its stock less attractive to some investors[379](index=379&type=chunk)[382](index=382&type=chunk) [Item 2. 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's IPO closed February 9, 2021, raising **$186.3 million** in net proceeds, unused as of June 30, 2021, with no material change to planned use - The company closed its IPO on February 9, 2021, raising net proceeds of approximately **$186.3 million**[393](index=393&type=chunk)[394](index=394&type=chunk) - As of June 30, 2021, the company had not used any of the net proceeds from its IPO, and the planned use of proceeds has not materially changed[395](index=395&type=chunk) [Item 6. Exhibits](index=96&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed, including lease amendments and certifications from Principal Executive and Financial Officers - Exhibits filed include two amendments to the lease agreement with PPF Off 100 Cambridge Park Drive, LLC, dated June 15, 2021[397](index=397&type=chunk) - Certifications from the CEO and CFO pursuant to Sarbanes-Oxley Act Sections 302 and 906 are included as exhibits[397](index=397&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 March 31, 2021 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from __________________ to __________________ Commission File Number: 001-39979 VOR BIOPHARMA INC. (Exact Name of Registrant as Specified in its Charter) Delaware 81 ...