Vor Biopharma Inc.

11/07/2024 | Press release | Distributed by Public on 11/07/2024 15:02

Quarterly Report for Quarter Ending September 30, 2024 (Form 10-Q)

10-Q

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, 2024

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

incorporation or organization)

(I.R.S. Employer

Identification No.)

100 Cambridgepark Drive, Suite 101

Cambridge, Massachusetts

02140

(Address of principal executive offices)

(Zip Code)

Registrant's telephone number, including area code: (617) 655-6580

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading

Symbol(s)

Name of each exchange on which registered

Common Stock, $0.0001 par value per share

VOR

Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company, or an emerging growth company. See the definitions of "large accelerated filer," "accelerated filer," "smaller reporting company," and "emerging growth company" in Rule 12b-2 of the Exchange Act.

Large accelerated filer

☐

Accelerated filer

☐

Non-accelerated filer

☒

Smaller reporting company

☒

Emerging growth company

☒

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

Number of shares of the registrant's Common Stock outstanding as of November 1, 2024 was 68,673,612.

Table of Contents

Page

PART I.

FINANCIAL INFORMATION

1

Item 1.

Financial Statements (Unaudited)

1

Condensed Consolidated Balance Sheets as ofSeptember 30, 2024 and December 31, 2023

1

Condensed Consolidated Statements of Operations and Comprehensive Loss for the Three and Nine Months Ended September 30, 2024 and 2023

2

Condensed Consolidated Statements of Stockholders' Equity for the Three and Nine Months Ended September 30, 2024 and 2023

3

Condensed Consolidated Statements of Cash Flows for the Nine Months Ended September 30, 2024 and 2023

4

Notes to Condensed Consolidated Financial Statements (Unaudited)

5

Item 2.

Management's Discussion and Analysis of Financial Condition and Results of Operations

12

Item 3.

Quantitative and Qualitative Disclosures About Market Risk

19

Item 4.

Controls and Procedures

19

PART II.

OTHER INFORMATION

21

Item 1.

Legal Proceedings

21

Item 1A.

Risk Factors

21

Item 5.

Other Information

21

Item 6.

Exhibits

23

Signatures

24

i

Note Regarding Company References

Throughout this Quarterly Report on Form 10-Q, the "Company," "Vor," "Vor Bio," "Vor Biopharma Inc.," "we," "us," and "our," except where the context requires otherwise, refer to Vor Biopharma Inc. and its consolidated subsidiary, and "our board of directors" refers to the board of directors of Vor Biopharma Inc.

Special Note Regarding Forward-Looking Statements

This Quarterly Report on Form 10-Q contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, contained in this Quarterly Report on Form 10-Q, including statements regarding our strategy, future operations, future financial position, future revenue, projected costs, prospects, plans and objectives of management, are forward-looking statements. In some cases, you can identify forward-looking statements by terms such as "may," "will," "should," "expect," "plan," "anticipate," "could," "might," "intend," "target," "ongoing," "project," "estimate," "believe," "estimate," "predict," "potential" or "continue" or the negative of these terms or other similar expressions intended to identify statements about the future. These statements speak only as of the date of this Quarterly Report on Form 10-Q and involve known and unknown risks, uncertainties and other important factors that may cause our actual results, levels of activity, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.

We have based these forward-looking statements largely on our current expectations and projections about future events and financial trends that we believe may affect our business, financial condition and results of operations. These forward-looking statements include, without limitation, statements about:

•
the timing, progress and results of our preclinical studies and clinical trials of our product candidates, including statements regarding the timing and pace of initiation, enrollment and completion of studies or trials and related preparatory work, the period during which the results of the trials will become available and plans with respect to our research and development programs;
•
the timing and success of our in-house or third-party clinical manufacturing capabilities and efforts;
•
the timing of any submission of filings for regulatory approval of, and our ability to obtain and maintain regulatory approvals for, our product candidates for any indication;
•
our ability to identify patients with the diseases treated by our product candidates, and to enroll patients in trials;
•
our expectations regarding the market acceptance and opportunity for and clinical utility of our product candidates, if approved for commercial use;
•
our expectations regarding the scope of any approved indication for any product candidate;
•
our ability to successfully commercialize our product candidates;
•
our estimates of our expenses, ongoing losses, future revenue, capital requirements, our need for or ability to obtain additional funding and our ability to continue as a going concern;
•
our ability to establish or maintain collaborations or strategic relationships;
•
our ability to identify, recruit and retain key personnel, including executive officers and members of management;
•
our reliance upon intellectual property licensed from third parties and our ability to obtain such licenses on commercially reasonable terms or at all;
•
our ability to protect and enforce our intellectual property position for our product candidates, and the scope of such protection;
•
our financial performance;
•
the period over which we estimate our existing cash, cash equivalents and marketable securities will be sufficient to fund our future operating expenses and capital expenditure requirements;
•
our competitive position and the development of and projections relating to our competitors or our industry;
•
the impact of laws and regulations; and
•
our expectations regarding the time during which we will be an emerging growth company under the Jumpstart Our Business Startups Act of 2012.

ii

You should read this Quarterly Report on Form 10-Q and the documents that we have filed as exhibits to this Quarterly Report on Form 10-Q completely and with the understanding that our actual future results may be materially different from what we expect. The forward-looking statements contained in this Quarterly Report on Form 10-Q are made as of the date of this Quarterly Report on Form 10-Q, and we do not assume any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by applicable law. You should refer to the "Risk Factors" section in this Quarterly Report and the "Summary Risk Factors" and "Risk Factors" sections in our Annual Report on Form 10-K for the year ended December 31, 2023 for a discussion of material factors that could cause actual results or events to differ materially from the forward-looking statements that we make.

This Quarterly Report on Form 10-Q includes statistical and other industry and market data, which we obtained from our own internal estimates and research, as well as from industry and general publications and research, surveys, and studies conducted by third parties. Industry publications, studies, and surveys generally state that they have been obtained from sources believed to be reliable, although they do not guarantee the accuracy or completeness of such information. While we believe that each of these studies and publications is reliable, we have not independently verified market and industry data from third-party sources. While we believe our internal company research is reliable and the market definitions are appropriate, neither such research nor these definitions have been verified by any independent source.

All brand names or trademarks appearing in this Quarterly Report on Form 10-Q, including Mylotarg, are the property of their respective owners.

iii

PART I-FINANCIAL INFORMATION

Item 1. Financial Statements (Unaudited).

VOR BIOPHARMA INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(UNAUDITED)

September 30,

December 31,

(in thousands, except share and per share amounts)

2024

2023

Assets

Current assets:

Cash and cash equivalents

$

52,798

$

31,360

Marketable securities

10,011

105,815

Prepaid expenses

4,202

3,153

Other current assets

285

475

Total current assets

67,296

140,803

Restricted cash equivalents

2,413

2,413

Property and equipment, net

7,474

10,050

Operating lease right-of-use assets

36,296

40,048

Other assets

2,512

4,812

Total assets

$

115,991

$

198,126

Liabilities and stockholders' equity

Current liabilities:

Accounts payable

$

1,316

$

815

Accrued liabilities

8,853

10,877

Operating lease liabilities

4,163

3,830

Other current liabilities

13

50

Total current liabilities

14,345

15,572

Long-term liabilities:

Operating lease liabilities-non-current

28,691

31,830

Total liabilities

43,036

47,402

Stockholders' equity:

Preferred stock, $0.0001 par value; 10,000,000 shares authorized as of September 30, 2024 and December 31, 2023; 0 shares issued and outstanding as of September 30, 2024 and December 31, 2023

-

-

Common stock, $0.0001 par value; 400,000,000 shares authorized as of September 30, 2024 and December 31, 2023; 68,665,640 and 67,901,610 shares issued and 68,663,065 and 67,891,311 shares outstanding as of September 30, 2024 and December 31, 2023, respectively

7

7

Additional paid-in capital

499,162

490,874

Accumulated other comprehensive income (loss)

68

(77

)

Accumulated deficit

(426,282

)

(340,080

)

Total stockholders' equity

72,955

150,724

Total liabilities and stockholders' equity

$

115,991

$

198,126

The accompanying notes are an integral part of these condensed consolidated financial statements.

1

VOR BIOPHARMA INC.

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS

(UNAUDITED)

Three Months Ended
September 30,

Nine Months Ended
September 30,

(in thousands, except share and per share amounts)

2024

2023

2024

2023

Operating expenses:

Research and development

$

21,817

$

27,606

$

67,962

$

73,418

General and administrative

6,696

7,710

21,912

24,494

Total operating expenses

$

28,513

$

35,316

$

89,874

$

97,912

Loss from operations

$

(28,513

)

$

(35,316

)

$

(89,874

)

$

(97,912

)

Other income:

Interest income

954

2,126

3,672

6,310

Total other income

954

2,126

3,672

6,310

Net loss

$

(27,559

)

$

(33,190

)

$

(86,202

)

$

(91,602

)

Net loss per share attributable to common stockholders,
basic and diluted

$

(0.40

)

$

(0.49

)

$

(1.26

)

$

(1.37

)

Weighted-average common shares outstanding,
basic and diluted

68,465,801

67,607,713

68,266,044

66,973,771

Other comprehensive income:

Unrealized gain on available for sale marketable securities

138

156

145

490

Total other comprehensive income

138

156

145

490

Comprehensive loss

$

(27,421

)

$

(33,034

)

$

(86,057

)

$

(91,112

)

The accompanying notes are an integral part of these condensed consolidated financial statements.

2

Vor Biopharma Inc.

CONDENSED CONSOLIDATED Statements of stockholders' EQUITY

(Unaudited)

Common
Stock

Additional
Paid-In

Accumulated other
comprehensive

Accumulated

Total
Stockholders'

(in thousands, except share amounts)

Shares

Amount

Capital

(loss) income

Deficit

Equity

Balance at December 31, 2023

67,891,311

$

7

$

490,874

$

(77

)

$

(340,080

)

$

150,724

Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, and exercise of stock options

184,998

-

(169

)

-

-

(169

)

Issuance of common stock from at-the-market sales agreement

139,462

-

213

-

-

213

Stock-based compensation expense

-

-

3,081

-

-

3,081

Other comprehensive loss

-

-

-

(10

)

-

(10

)

Net loss

-

-

-

-

(30,804

)

(30,804

)

Balance at March 31, 2024

68,215,771

$

7

$

493,999

$

(87

)

$

(370,884

)

$

123,035

Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, exercise of stock options, and issuance of common stock under ESPP

142,113

-

74

-

-

74

Stock-based compensation expense

-

-

2,793

-

-

2,793

Other comprehensive income

-

-

-

17

-

17

Net loss

-

-

-

-

(27,839

)

(27,839

)

Balance at June 30, 2024

68,357,884

$

7

$

496,866

$

(70

)

$

(398,723

)

$

98,080

Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, and exercise of stock options

195,547

-

(57

)

-

-

(57

)

Issuance of common stock from at-the-market sales agreement

109,634

-

89

-

-

89

Stock-based compensation expense

-

-

2,264

-

-

2,264

Other comprehensive income

-

-

-

138

-

138

Net loss

-

-

-

-

(27,559

)

(27,559

)

Balance at September 30, 2024

68,663,065

$

7

$

499,162

$

68

$

(426,282

)

$

72,955

Common
Stock

Additional
Paid-In

Accumulated other comprehensive

Accumulated

Total
Stockholders'

(in thousands, except share amounts)

Shares

Amount

Capital

loss

Deficit

Equity

Balance at December 31, 2022

65,996,138

$

7

$

473,587

$

(770

)

$

(222,217

)

$

250,607

Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, and exercise of stock options

205,485

-

(340

)

-

-

(340

)

Issuance of common stock from at-the-market sales agreement

733,274

-

3,717

-

-

3,717

Stock-based compensation expense

-

-

4,068

-

-

4,068

Other comprehensive income

-

-

-

596

-

596

Net loss

-

-

-

-

(28,433

)

(28,433

)

Balance at March 31, 2023

66,934,897

$

7

$

481,032

$

(174

)

$

(250,650

)

$

230,215

Issuance of common stock upon vesting of RSUs, net of shares withheld for taxes, exercise of stock options, and issuance of common stock under ESPP

381,775

-

(317

)

-

-

(317

)

Issuance of common stock from at-the-market sales agreement

116,888

-

555

-

-

555

Stock-based compensation expense

-

-

4,238

-

-

4,238

Other comprehensive loss

-

-

-

(262

)

-

(262

)

Net loss

-

-

-

-

(29,979

)

(29,979

)

Balance at June 30, 2023

67,433,560

$

7

$

485,508

$

(436

)

$

(280,629

)

$

204,450

Issuance of common stock upon vesting of RSUs, vesting and exercise of stock options, net of shares withheld for taxes

219,409

-

(183

)

-

-

(183

)

Issuance of common stock from at-the-market sales agreement

132,500

-

386

-

-

386

Stock-based compensation expense

-

-

3,162

-

-

3,162

Other comprehensive income

-

-

-

156

-

156

Net loss

-

-

-

-

(33,190

)

(33,190

)

Balance at September 30, 2023

67,785,469

$

7

$

488,873

$

(280

)

$

(313,819

)

$

174,781

The accompanying notes are an integral part of these condensed consolidated financial statements.

3

Vor Biopharma Inc.

condensed CONSOLIDATED StatementS of Cash Flows

(unaudited)

Nine Months Ended September 30,

(in thousands)

2024

2023

Cash flows from operating activities

Net loss

$

(86,202

)

$

(91,602

)

Adjustments to reconcile net loss to net cash used in operations:

Depreciation expense

2,663

2,600

Non-cash lease expense

3,752

3,513

Stock-based compensation

8,138

11,468

Interest amortization on marketable securities

(1,137

)

(3,674

)

Changes in operating assets and liabilities:

Operating lease liabilities

(2,806

)

(2,646

)

Prepaid expenses and other current assets

(859

)

2,558

Accounts payable and accrued liabilities

(1,446

)

2,678

Other assets

2,300

(1,161

)

Net cash used in operating activities

(75,597

)

(76,266

)

Cash flow from investing activities

Purchases of marketable securities

(9,914

)

(58,369

)

Proceeds from maturities of marketable securities

107,000

120,000

Purchases of property and equipment

(157

)

(958

)

Net cash provided by investing activities

96,929

60,673

Cash flow from financing activities

Payment of issuance costs related to underwritten public offering and concurrent private placement

-

(717

)

Proceeds from the issuance of common stock from at-the-market sales agreement, net of issuance costs

297

4,593

Repurchases of shares for tax withholdings upon vesting of restricted stock unit awards

(303

)

(1,165

)

Proceeds from stock option exercises and the issuance of shares under ESPP

112

202

Net cash provided by financing activities

106

2,913

Net increase (decrease) in cash, cash equivalents and restricted cash equivalents

21,438

(12,680

)

Cash, cash equivalents and restricted cash equivalents,
beginning of period

$

33,773

$

60,119

Cash, cash equivalents and restricted cash equivalents, end of period

$

55,211

$

47,439

Supplemental disclosure of non-cash activities

Right-of-use assets obtained in exchange for lease obligations

$

-

$

37

Purchases of property and equipment in accounts payable and accrued liabilities

$

-

$

63

Financing costs associated with the sale of common stock included in accounts payable and accrued expenses

$

22

$

30

A reconciliation of the cash, cash equivalents and restricted cash equivalents reported within the condensed consolidated balance sheets that sum to the total of the same amounts shown in the statements of cash flows is as follows:

For the Nine Months Ended September 30,

(in thousands)

2024

2023

Cash and cash equivalents

$

52,798

$

45,026

Restricted cash equivalents

2,413

2,413

Total cash, cash equivalents and restricted cash equivalents as shown on the
statements of cash flows

$

55,211

$

47,439

The accompanying notes are an integral part of these condensed consolidated financial statements.

4

VOR BIOPHARMA INC.

NOTES TO CONDENSEDCONSOLIDATED FINANCIAL STATEMENTS

(UNAUDITED)

1. Nature of the Business

Vor Biopharma Inc. (the "Company") is a clinical-stage cell and genome engineering company that combines a novel patient engineering approach with targeted therapies to provide a single company solution for patients suffering from hematological malignancies. The Company's proprietary platform leverages its expertise in hematopoietic stem cell ("HSC") biology, genome engineering and targeted therapy development to genetically modify HSCs to remove surface targets expressed by cancer cells. The Company is headquartered in Cambridge, Massachusetts. The Company was incorporated on December 30, 2015.

Risks and Uncertainties

The Company is subject to a number of risks common to development stage companies in the biotechnology industry, including, but not limited to, risks of failure of preclinical studies and clinical trials, dependence on key personnel, protection of proprietary technology, reliance on third party organizations, risks of obtaining regulatory approval for any product candidate that it may develop, development by competitors of technological innovations, compliance with government regulations, adverse macroeconomic conditions and the need to obtain additional financing.

The Company anticipates that it will continue to incur significant operating losses for the next several years as it continues to develop its product candidates. As a result, the Company's continued operations are dependent on its ability to raise additional funding. If the Company is unable to obtain additional funding on a timely basis, it may be forced to significantly curtail, delay, or discontinue one or more of its planned research or development programs or be unable to expand its operations.

Liquidity and Capital Resources

As of September 30, 2024, the Company had $62.8million of cash, cash equivalents and marketable securities and an accumulated deficit of $426.3million. The Company anticipates that it will continue to incur significant operating losses for the next several years as it continues to develop its product candidates. As a result, the Company's continued operations are dependent on its ability to raise additional funding. Based on its current business plan and current capital resources, combined with the need to raise additional funding and the uncertainty regarding the availability of such additional funding, management has concluded that there is substantial doubt regarding the Company's ability to continue as a going concern within one year after the date these condensed consolidated financial statements are issued. Management's plans to alleviate the conditions that raise substantial doubt regarding the Company's ability to continue as a going concern include obtaining additional funding through equity or debt offerings and/or pursuant to collaboration or licensing arrangements. However, additional funding may not be available on terms acceptable to the Company or at all. The Company may also seek to reduce current spending requirements where necessary. Management has concluded the likelihood that its plans to successfully obtain sufficient additional funding from one or more of these sources, or adequately reduce expenditures, while reasonably possible, is less than probable.

If, for any reason, the Company utilizes its capital resources more quickly than anticipated or is unable to obtain additional funding on a timely basis, it may be required to revise its business plan and strategy. This may result in the Company significantly curtailing, delaying or discontinuing one or more of its research and development programs. As a result, the Company's business, financial condition, and results of operations could be materially affected. The accompanying condensed consolidated financial statements do not include any adjustments to the carrying amounts and classification of assets and liabilities that may be necessary if the Company were unable to continue as a going concern.

2. Summary of Significant Accounting Policies

Basis of Presentation

The accompanying condensed consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America ("GAAP"). Certain comparative amounts have been reclassified to conform to the current period presentation, including the presentation of non-cash lease expense in the condensed consolidated statements of cash flows. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification ("ASC") and Accounting Standards Update ("ASU") issued by the Financial Accounting Standards Board ("FASB").

5

Use of Estimates

The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company's management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the condensed consolidated financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the condensed consolidated financial statements. Management must apply significant judgment in this process. Management's estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: accrued expenses and research and development expenses.

Unaudited Interim Financial Information

The condensed consolidated financial statements of the Company included herein have been prepared pursuant to the rules and regulations of the Securities and Exchange Commission (the "SEC"). The unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiary. All intercompany transactions and balances have been eliminated in consolidation. In the opinion of management, the information furnished reflects all adjustments, all of which are of a normal and recurring nature, necessary for a fair presentation of the results for the reported interim periods. The results of operations for interim periods are not necessarily indicative of results to be expected for the full year or any other interim period.

The accompanying condensed consolidated balance sheet as of December 31, 2023 has been derived from the Company's audited consolidated financial statements for the year ended December 31, 2023. Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted pursuant to rules and regulations. However, the Company believes that the disclosures are adequate to make the information presented not misleading. These unaudited interim condensed consolidated financial statements should be read in conjunction with the audited annual consolidated financial statements and related notes included in the Company's Annual Report on Form 10-K for the year ended December 31, 2023 (the "2023 Annual Report").

During the nine months ended September 30, 2024, there have been no changes to the Company's significant accounting policies as described in the 2023Annual Report.

3. Marketable Securities

The amortized cost and estimated fair value of marketable securities, by remaining contractual maturity, are as follows:

September 30, 2024

(in thousands)

Amortized Cost

Gross Unrealized Holding Gains

Gross Unrealized Holding Losses

Fair Value

Maturing after one year through five years

U.S. Treasuries

9,943

68

-

10,011

Total

$

9,943

$

68

$

-

$

10,011

December 31, 2023

(in thousands)

Amortized Cost

Gross Unrealized Holding Gains

Gross Unrealized Holding Losses

Fair Value

Maturing in one year or less

U.S. Treasury Bill

$

8,806

$

6

$

-

$

8,812

U.S. Treasuries

97,086

12

(95

)

97,003

Total

$

105,892

$

18

$

(95

)

$

105,815

The Company did not have any individual securities in an unrealized loss position as of September 30, 2024. The following table summarizes the fair value and gross unrealized losses aggregated by category and the length of time that individual securities were in an unrealized loss position as of December 31, 2023:

6

December 31, 2023

Less than twelve months

Greater than twelve months

Total

(in thousands)

Fair value

Unrealized loss

Fair value

Unrealized loss

Fair value

Unrealized loss

U.S. Treasuries

$

-

$

-

$

53,447

$

(95

)

$

53,447

$

(95

)

Total

$

-

$

-

$

53,447

$

(95

)

$

53,447

$

(95

)

The Company did not record any impairments to marketable securities or reserves for credit losses related to its marketable debt securities during the periods presented.

4. Fair Value Measurements

The following tables present information about the Company's financial assets and liabilities measured at fair value on a recurring basis:

September 30, 2024

(in thousands)

Level 1

Level 2

Level 3

Total

Cash equivalents

Money market funds

$

52,682

$

-

$

-

$

52,682

Marketable securities

U.S. Treasuries

-

10,011

-

10,011

Total marketable securities

-

10,011

-

10,011

Restricted cash equivalents

Money market funds

2,413

-

-

2,413

Total

$

55,095

$

10,011

$

-

$

65,106

December 31, 2023

(in thousands)

Level 1

Level 2

Level 3

Total

Cash equivalents

Money market funds

$

31,164

$

-

$

-

$

31,164

Marketable securities

U.S. Treasury Bill

8,812

-

-

8,812

U.S. Treasuries

-

97,003

-

97,003

Total marketable securities

8,812

97,003

-

105,815

Restricted cash equivalents

Money market funds

2,413

-

-

2,413

Total

$

42,389

$

97,003

$

-

$

139,392

The fair value of the Company's cash equivalents and restricted cash equivalents is based on quoted market prices in active markets with no valuation adjustment. The fair value of marketable securities was determined based on observable market inputs. There were notransfers between levels during the nine months ended September 30, 2024.

Prepaid expenses, accounts payable and accrued expenses are stated at their respective historical carrying values, which approximate fair value due to their short-term nature.

7

5. Property and Equipment, Net

Property and equipment, net consisted of the following:

September 30,

December 31,

(in thousands)

2024

2023

Laboratory equipment

$

10,089

$

10,028

Manufacturing equipment

7,065

6,936

Computer equipment

432

432

Furniture, fixtures and other

606

599

Construction in progress

35

146

Total

18,227

18,141

Less: Accumulated depreciation

(10,753

)

(8,091

)

Property and equipment, net

$

7,474

$

10,050

Depreciation expense for the three and nine months ended September 30, 2024 was $0.9million and $2.7million, respectively, and for the three and nine months ended September 30, 2023 was $0.9million and $2.6million, respectively.

6. Accrued Liabilities

Accrued liabilities consisted of the following:

September 30,

December 31,

(in thousands)

2024

2023

Employee-related expenses

$

4,138

$

5,962

Professional fees

1,326

1,245

Clinical expenses

2,299

1,495

Manufacturing expenses

512

842

Research and development expenses

272

1,059

Other

306

274

Total accrued liabilities

$

8,853

$

10,877

7. Stock-Based Compensation

2023 Inducement Plan

As of September 30, 2024, the Company had 2,635,312shares of its common stock available for future issuance under the 2023 Inducement Plan.

2021 Equity Incentive Plan

As of September 30, 2024, the Company had 2,457,705shares of its common stock available for future issuance under its 2021 Equity Incentive Plan.

Stock Options

The Company's stock options generally vest over 48months with 25% vesting after one yearfollowed by ratable monthly vesting over the remaining three yearsand have a contractual term of 10years. The weighted-average assumptions used principally in determining the fair value of options granted were as follows:

Nine Months Ended September 30,

2024

2023

Expected term (in years)

6.0

6.0

Expected volatility

88.9

%

81.7

%

Risk-free interest rate

4.1

%

3.8

%

Dividend yield

-

-

During the nine months ended September 30, 2024 and 2023, the Company granted stock options to purchase 2,174,100shares and 2,247,844shares of its common stock, respectively, with a weighted-average grant-date fair value of $1.49and $3.65per share,

8

respectively. As of September 30, 2024, total unrecognized compensation expense related to stock options was $9.3million, which is expected to be recognized over a weighted-average period of 2.5years.

As of September 30, 2024, options for 2,575shares of Company common stock with a weighted-average exercise price of $4.90were exercised and unvested. An immaterial amount of underlying proceeds from the unvested exercises is recorded in other current liabilities on the condensed consolidated balance sheet.

Restricted Stock Units

During the nine months ended September 30, 2024 and 2023, the Company granted 1,196,217restricted stock units and 659,672restricted stock units, respectively, with a weighted-average grant date fair value of $2.29and $5.49per share, respectively. As of September 30, 2024, total unrecognized compensation expense related to restricted stock units was $3.9million, which is expected to be recognized over a weighted-average period of 2.4years.

Employee Stock Purchase Plan

As of September 30, 2024, the Company had 1,894,294shares of its common stock available for issuance under its Employee Stock Purchase Plan ("ESPP").

During the nine months ended September 30, 2024, the Company issued 74,326shares of common stock with a weighted-average purchase price of $1.48under the ESPP, which resulted in an immaterial amount of compensation expense. During the nine months ended September 30, 2023, the Company issued 44,977shares of common stock with a weighted-average purchase price of $3.76under the ESPP, which resulted in an immaterial amount of compensation expense.

Stock-Based Compensation

Stock-based compensation expense was allocated as follows:

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

2024

2023

2024

2023

Research and development

$

1,148

$

1,527

$

3,825

$

6,345

General and administrative

1,116

1,635

4,313

5,123

Total stock-based compensation expense

$

2,264

$

3,162

$

8,138

$

11,468

8. Leases

Cambridgepark Lease Amendments

On June 15, 2021, the Company entered into the first lease amendment ("First Lease Amendment") and second lease amendment ("Second Lease Amendment" and, together with the First Lease Amendment, the "Lease Amendments") with PPF Off 100 Cambridge Park Drive, LLC (the "Landlord"). The Lease Amendments amended the Company's lease agreement for its corporate office and laboratory facilities with the Landlord in Cambridge, Massachusetts to add additional leased space in the same building (the "Amended Cambridgepark Lease").

The First Lease Amendment and Second Lease Amendment commenced for accounting purposes on January 28, 2022and April 29, 2022, respectively. The terms of the Lease Amendments are through September 2030 for approximately $8.4million and $22.3millionin fixed payments for the First Lease Amendment and Second Lease Amendment, respectively. There are no optionsto extend the Lease Amendments.

Payments due associated with the Lease Amendments include fixed and variable payments. Variable payments relate to the Company's share of the Landlord's operating costs associated with the underlying assets and are recognized when the event on which those payments are assessed occurs. The Amended Cambridgepark Lease does not contain a residual value guarantee. The Lease Amendments term end dates are coterminous with the existing lease agreement. In conjunction with the Lease Amendments, the Company was required to increase its irrevocable standby letter of credit to $2.4million for the benefit of the Landlord, which has been secured by money market investments and is presented as restricted cash equivalents.

9

For further information regarding the Company's Cambridgepark lease, please see Note 9 to the consolidated financial statements included in the 2023 Annual Report.

The elements of lease expense were as follows:

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands)

2024

2023

2024

2023

Operating lease cost

$

1,951

$

1,950

$

5,853

$

5,851

Variable lease cost

681

663

1,890

1,921

Total lease cost

$

2,632

$

2,613

$

7,743

$

7,772

Amounts reported in the condensed consolidated balance sheets and the weighted-average lease term and discount rate information were as follows:

(in thousands except weighted-average amounts)

September 30, 2024

December 31, 2023

Assets

Operating lease right-of-use assets

$

36,296

$

40,048

Liabilities

Operating lease liabilities, current

$

4,163

$

3,830

Operating lease liabilities, non-current

28,691

31,830

Total lease liabilities

$

32,854

$

35,660

Weighted-Average Lease Term and Discount Rate

Weighted-average remaining lease term (years)

6.0

6.7

Weighted-average discount rate

8.2

%

8.2

%

The following table represents other lease activity:

Nine Months Ended September 30,

(in thousands)

2024

2023

Other Information

Cash paid for amounts included in the measurement of lease liabilities

Operating cash flows for operating leases

$

4,906

$

4,263

Right-of-use assets obtained in exchange for lease obligations

$

-

$

37

9. Significant Agreements

Since December 31, 2023, there have been no material changes to the key terms of the Company's license agreements. For further information regarding the Company's existing license agreements, please see Note 10 to the consolidated financial statements included in the 2023Annual Report.

10. Net Loss Per Share

The following table sets forth the computation of the Company's basic and diluted net loss per share for the three and nine months ended September 30, 2024 and 2023:

Three Months Ended September 30,

Nine Months Ended September 30,

(in thousands, except share and per share amounts)

2024

2023

2024

2023

Numerator:

Net loss attributable to common stockholders

$

(27,559

)

$

(33,190

)

$

(86,202

)

$

(91,602

)

Denominator:

Weighted-average number of common shares outstanding, basic and diluted

68,465,801

67,607,713

68,266,044

66,973,771

Net loss per share attributable to common stockholders, basic and diluted

$

(0.40

)

$

(0.49

)

$

(1.26

)

$

(1.37

)

10

The Company's potentially dilutive securities were stock options, unvested restricted stock and restricted stock units. Based on the amounts outstanding as of September 30, 2024 and 2023, the Company excluded the following potential common shares from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect:

As of September 30,

2024

2023

Options to purchase common stock

9,703,165

8,434,200

Unvested restricted stock

2,575

12,874

Restricted stock units

1,406,736

1,051,206

11

Item 2. Management's Discussion and Analysis ofFinancial Condition and Results of Operations.

You should read the following discussion and analysis of our financial condition and results of operations together with the unaudited condensed consolidated financial statements and related notes included elsewhere in this Quarterly Report on Form 10-Q and with the consolidated financial statements and related notes included in our Annual Report on Form 10-K for the year ended December 31, 2023 (the "2023 Annual Report"). Some of the information contained in this discussion and analysis or set forth elsewhere in this Quarterly Report, including information with respect to our plans and strategy for our business, includes forward-looking statements that involve risks and uncertainties. As a result of many factors, including those factors set forth in the section titled "Risk Factors" in our 2023 Annual Report and in other reports we have filed or may file with the SEC, our actual results could differ materially from the results described in or implied by the forward-looking statements contained in the following discussion and analysis.

Overview

Vor Bio is a clinical-stage company harnessing the power of cell and genome engineering to develop potentially transformative therapies in acute myeloid leukemia ("AML"), a devastating disease with few treatment options. AML is the most common type of acute leukemia in adults and one of the deadliest and most aggressive blood cancers, affecting approximately 20,000 newly diagnosed patients each year in the United States.

Leveraging our expertise in HSC biology and genome engineering, we genetically modify HSCs to remove surface targets and then provide these cells as hematopoietic cell transplants ("HCTs") to patients. Once these cells engraft into bone marrow, the patient's healthy cells are shielded because they no longer express the surface target, leaving only the cancerous cells exposed. We believe this will unlock the potential of targeted therapies to selectively destroy cancerous cells while shielding healthy cells. As a result, our shielded transplants are designed to limit the on-target toxicities associated with these targeted therapies, thereby enhancing their utility, and broadening their applicability. We intend to pair our shielded transplants with targeted therapeutics such as antibody drug conjugates ("ADCs") or VCAR33ALLO, a chimeric antigen receptor ("CAR")-T therapy designed to target CD33, to bring potentially transformative outcomes to patients and establish a new standard of care Treatment System in AML.

We are developing trem-cel, a shielded transplant, which we believe has the potential to transform the treatment for AML and other blood cancers. Trem-cel is created by genetically modifying healthy donor HSCs in order to remove the CD33 surface target. We intend to develop trem-cel as a HCT product candidate to replace the standard of care in transplant settings. We are actively enrolling and treating patients in VBP101, our first-in-human Phase 1/2a trial of trem-cel in combination with Mylotarg. We released clinical data on 18 patients treated in this trial in September 2024. The data demonstrated:

•
Reliable engraftment, robust platelet recovery, and high CD33 editing efficiency with full myeloid chimerism at Day 28;
•
Shielding of the blood system, with maintained neutrophil and platelet counts across multiple Mylotarg doses of 0.5, 1, and 2 mg/m2;
•
Broadened therapeutic index for Mylotarg with drug exposure represented by AUC which is related to efficacy, consistent with labeled Mylotarg doses, and with maximal concentrations, measured by Cmaxand related to veno-occlusive disease, well below known toxic range; and
•
Early evidence suggesting patient benefit as measured by relapse-free survival when compared to published high-risk AML comparators, with only four patients relapsing, two of which occurred before Mylotarg treatment, and two occurring following Mylotarg, both with TP53 mutation.

We expect to provide our next clinical data update at the American Society of Hematology (ASH) 2024 annual meeting in December 2024.

VCAR33ALLOis manufactured from lymphocytes collected from the patient's original transplant donor, generating a CAR-T cell product that is exactly matched to the recipient's engrafted blood system. By using healthy transplant donor cells as the starting material to produce VCAR33ALLO, the CAR-T cells have a more stem-like phenotype, leading to greater potential for expansion, persistence, and anti-leukemia activity compared to a product derived from a patient's own lymphocytes. In January 2024 we dosed the first patient with VCAR33ALLOin VBP301 and are continuing to dose patients in this study. In September 2024, we announced encouraging in vivoCAR-T expansion data from the first three patients treated, all at the lowest dose of 1 x 106CAR+ cells/kg.

We believe that the combination of trem-cel followed by treatment with VCAR33ALLO in the post-transplant setting, which we refer to as the trem-cel + VCAR33 Treatment System, may transform patient outcomes and offer the potential for cures for patients that have limited treatment options. The trem-cel + VCAR33 Treatment System would utilize the same healthy donor allogenic cell source for both trem-cel and VCAR33ALLO. We plan to collect initial data on trem-cel from the VBP101 clinical trial and initial

12

clinical data from the VCAR33ALLO program prior to IND submission for the trem-cel + VCAR33 Treatment System. However, the VBP301 protocol allows for patients who have received a trem-cel transplant on the VBP101 study to enroll in VBP301 and receive VCAR33ALLO. This may provide valuable early insights into the potential of the trem-cel + VCAR33 Treatment System to enable a more potent therapy and durable responses post-transplant.

In September 2024, we announced a new preclinical asset, VADC45, which has a number of potential opportunities in oncology, gene therapy, and autoimmune disorders. VADC45 is an ADC designed to target the CD45 protein. CD45 is a well-validated target for a wide variety of blood cancers with clinical proof of concept. The linker-payload used in VADC45 is also clinically validated. VADC45 has the potential to treat a number of diseases, including treatment of hematologic malignancies, as a targeted conditioning agent for gene therapies such as for sickle cell disease, holistic immune reset for autoimmune disorders, and for our approach of combining this asset with epitope modification of CD45 to shield healthy stem cells. We already have robust preclinical data for VADC45 and are progressing IND-enabling studies to enable future Phase 1 studies.

We operate an in-house clinical manufacturing facility in Cambridge, Massachusetts to support development of our shielded transplants and CAR-T therapeutic candidates for patients with blood cancers. While this facility is now operational, we continue to rely on third-party contract manufacturers for our required raw materials, manufacturing devices, active pharmaceutical ingredients and finished product for our research and clinical manufacturing. Since our inception in December 2015, we have devoted substantially all of our resources to raising capital, organizing and staffing our company, business and scientific planning, conducting discovery and research activities, acquiring or discovering product candidates, establishing and protecting our intellectual property portfolio, developing and progressing our product candidates and preparing for clinical trials, establishing arrangements with third parties for the manufacture of our product candidates and component materials, building out our internal clinical manufacturing facility, and providing general and administrative support for these operations. We do not have any product candidates approved for sale and have not generated any revenue from product sales. Through September 30, 2024, we funded our operations primarily through the sale of equity securities and debt financings and have received aggregate net proceeds from these transactions of approximately $464.4 million.

We have incurred significant operating losses since inception, including net losses of $27.6 million and $86.2 million, respectively, for the three and nine months ended September 30, 2024, and $117.9 million for the year ended December 31, 2023. As of September 30, 2024, we had an accumulated deficit of $426.3 million.

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $62.8 million. We expect that our cash, cash equivalents and marketable securities at September 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025.

Critical Accounting Estimates

Our management's discussion and analysis of our financial condition and results of operations is based on our condensed consolidated financial statements, which have been prepared in accordance with United States generally accepted accounting principles. The preparation of our condensed consolidated financial statements requires us to make judgments and estimates that affect the reported amounts of assets, liabilities, costs, and expenses, and the disclosure of contingent assets and liabilities in our condensed consolidated financial statements. We base our estimates on historical experience, known trends and events, and various other factors that we believe to be reasonable under the circumstances. Actual results may differ from these estimates under different assumptions or conditions. On an ongoing basis, we evaluate our judgments and estimates in light of changes in circumstances, facts, and experience. The effects of material revisions in estimates, if any, will be reflected in the condensed consolidated financial statements prospectively from the date of change in estimates. There have been no material changes to our critical accounting estimates from those described in "Management's Discussion and Analysis of Financial Condition and Results of Operations" in our 2023 Annual Report.

Financial Operations Overview

Revenue

We have not generated any revenue since our inception and do not expect to generate any revenue from the sale of products in the near future, if at all. If our development efforts for our product candidates are successful and result in marketing approval, or if we enter into collaboration or license agreements with third parties, we may generate revenue in the future from a combination of product sales or payments from such agreements.

13

Expenses

Research and Development Expenses

Research and development expenses consist primarily of external and internal expenses incurred in connection with our research and development activities, including our drug discovery efforts and the development of our product candidates. External expenses include:

•
research and development expenses incurred under agreements with CROs and other scientific development services;
•
costs of consultants, including their fees and related travel expenses;
•
costs related to compliance with quality and regulatory requirements;
•
costs of laboratory supplies and acquiring and developing preclinical and clinical trial materials, including expenses associated with our CMOs; and
•
payments made under third party licensing agreements.

Internal expenses include:

•
personnel-related expenses, including salaries, bonuses, benefits and stock-based compensation expenses, for employees involved in research and development activities; and
•
facilities, depreciation and other allocated expenses, which include direct and allocated expenses for rent, insurance, and other internal operating costs, and internal manufacturing expenses.

We expense research and development costs as incurred. We recognize external development costs based on an evaluation of the progress to completion of specific tasks using information provided to us by our vendors. Payments for these activities are based on the terms of the individual agreements, which may differ from the pattern of costs incurred, and are reflected in our condensed consolidated financial statements as prepaid expenses or accrued research and development expenses. Nonrefundable advance payments for goods or services to be received in the future for use in research and development activities are deferred and capitalized, even when there is no alternative future use for the research and development. The capitalized amounts are expensed as the related goods are delivered or the services are performed.

A significant portion of our research and development costs have been external costs, which we track by stage of development, preclinical or clinical. However we do not track our internal research and development expenses on a program specific basis because these costs are deployed across multiple projects and, as such, are not separately classified.

Research and development activities are central to our business model. We expect that our research and development expenses will increase significantly for the foreseeable future as we continue to identify and develop product candidates, particularly as more of our product candidates move into clinical development and later stages of clinical development.

The successful development of our product candidates in the future is highly uncertain. Therefore, we cannot reasonably estimate or know the nature, timing and estimated costs of the efforts that will be necessary to complete the development and commercialization of any of our product candidates. We are also unable to predict when, if ever, material net cash inflows will commence from the sale of our product candidates, if approved. This is due to the numerous risks and uncertainties associated with developing product candidates, many of which are outside of our control, including the uncertainty of:

•
the timing and progress of preclinical and clinical development activities;
•
the number and scope of preclinical and clinical programs we decide to pursue;
•
our ability to maintain our current research and development programs and to establish new ones;
•
establishing an appropriate safety profile with IND-enabling studies;
•
the number of sites and patients included in the clinical trials;
•
the countries in which the clinical trials are conducted;
•
per patient trial costs;
•
successful patient enrollment in, and the initiation of, clinical trials, as well as drop out or discontinuation rates or complications with donors;
•
the successful completion of clinical trials with safety, tolerability and efficacy profiles that are satisfactory to the FDA or any comparable foreign regulatory authority;
•
the number of trials required for regulatory approval;

14

•
the timing, receipt and terms of any regulatory approvals from applicable regulatory authorities;
•
our ability to establish new licensing or collaboration arrangements;
•
the performance of our current and future collaborators, if any;
•
establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers;
•
significant and changing government regulation and regulatory guidance;
•
the impact of any business interruptions to our operations or to those of the third parties with whom we work;
•
obtaining, maintaining, defending and enforcing patent claims and other intellectual property rights;
•
launching commercial sales of our product candidates, if approved, whether alone or in collaboration with others; and
•
maintaining a continued acceptable safety profile of the product candidates following approval.

Any changes in the outcome of any of these variables could mean a significant change in the costs and timing associated with the development of our product candidates.

General and Administrative Expenses

General and administrative expenses consist primarily of personnel-related costs, including salaries, bonuses, benefits and stock-based compensation expenses for employees involved in our executive, finance, corporate, business development and administrative functions, as well as expenses for outside professional services, including legal, audit, accounting and tax-related services and other consulting fees, facility-related expenses, which include depreciation costs and other allocated expenses for rent and maintenance of facilities, insurance costs, recruiting costs, travel expenses and other general administrative expenses.

We expect that our general and administrative expenses will increase as our business expands and we hire additional personnel to support our continued research and development activities, including our clinical programs.

Other Income

Interest Income

Interest income consists of interest income earned on our cash, cash equivalents, restricted cash and marketable securities held in financial institutions.

Results of Operations

Comparison of Three and Nine Months Ended September 30, 2024 and 2023

The following table summarizes our results of operations for the periods indicated (amounts in thousands):

Three Months Ended
September 30,

2024

2023

Change

Operating expenses:

Research and development

$

21,817

$

27,606

$

(5,789

)

General and administrative

6,696

7,710

(1,014

)

Total operating expenses

28,513

35,316

(6,803

)

Loss from operations

(28,513

)

(35,316

)

6,803

Other income:

Interest income

954

2,126

(1,172

)

Total other income

954

2,126

(1,172

)

Net loss

$

(27,559

)

$

(33,190

)

$

5,631

15

Nine Months Ended
September 30,

2024

2023

Change

Operating expenses:

Research and development

$

67,962

$

73,418

$

(5,456

)

General and administrative

21,912

24,494

(2,582

)

Total operating expenses

89,874

97,912

(8,038

)

Loss from operations

(89,874

)

(97,912

)

8,038

Other income:

Interest income

3,672

6,310

(2,638

)

Total other income

3,672

6,310

(2,638

)

Net loss

$

(86,202

)

$

(91,602

)

$

5,400

Research and Development Expenses

The following table summarizes our research and development expenses incurred for the periods indicated (amounts in thousands):

Three Months Ended
September 30,

2024

2023

Change

External expenses

$

7,856

$

14,550

$

(6,694

)

Internal expenses:

Personnel expenses (including stock-based compensation)

9,409

9,143

266

Manufacturing, facilities, and other expenses

4,552

3,913

639

Total research and development expenses

$

21,817

$

27,606

$

(5,789

)

Nine Months Ended
September 30,

2024

2023

Change

External expenses

$

24,449

$

34,568

$

(10,119

)

Internal expenses:

Personnel expenses (including stock-based compensation)

29,202

27,566

1,636

Manufacturing, facilities, and other expenses

14,311

11,284

3,027

Total research and development expenses

$

67,962

$

73,418

$

(5,456

)

Research and development expenses were $21.8 million for the three months ended September 30, 2024, compared to $27.6 million for the three months ended September 30, 2023. The decrease of $5.8 million was due to a decrease in license payments, preclinical expenses, and manufacturing starting materials, offset in part by an increase in clinical trial costs to support our trem-cel and VCAR33ALLO programs.

Research and development expenses were $68.0 million for the nine months ended September 30, 2024, compared to $73.4 million for the nine months ended September 30, 2023. The decrease of $5.5 million was primarily due to a decrease in license payments, preclinical expenses, and stock-based compensation expense, partially offset by an increase in personnel-related costs driven by additional headcount to support our clinical and manufacturing activities and an increase in clinical trial costs to support our trem-cel and VCAR33ALLO programs.

General and Administrative Expenses

General and administrative expenses were $6.7 million for the three months ended September 30, 2024, compared to $7.7 million for the three months ended September 30, 2023. The decrease of $1.0 million was primarily due to a decrease in professional fees and stock-based compensation expense.

General and administrative expenses were $21.9 million for the nine months ended September 30, 2024, compared to $24.5 million for the nine months ended September 30, 2023. The decrease of $2.6 million was primarily due to a decrease in professional fees and stock-based compensation expense, partially offset by an increase in personnel costs.

16

Other Income

Other income decreased by $1.2 million during the three months ended September 30, 2024, compared to the three months ended September 30, 2023. Other income decreased by $2.6 million for the nine months ended September 30, 2024, compared to the nine months ended September 30, 2023. The decrease in other income in both periods was due to decreases in interest earned from our cash, cash equivalents, restricted cash and marketable securities as the Company held lower balances of cash, cash equivalents and marketable securities.

Liquidity and Capital Resources

Sources of Liquidity

Since our inception, we have not recognized any revenue and have incurred operating losses and negative cash flows from our operations. We have not yet commercialized any product and we do not expect to generate revenue from sales of any products for several years, if at all. We have funded our operations primarily through the sale of equity securities and have received aggregate net proceeds from financing transactions of approximately $464.4 million as of September 30, 2024.

In order to fund our future operations, including our planned clinical trials, on March 14, 2022, we filed a universal shelf registration statement (the "Shelf Registration Statement"), to provide for aggregate offerings of up to $350.0 million of common stock, preferred stock, debt securities, warrants or any combination thereof. As of September 30, 2024, $274.4 million remains available under this Shelf Registration Statement, including $119.7 million reserved for at-the market offerings discussed below.

At-the-Market Sales Agreement

In December 2022, we entered into a Sales Agreement with Stifel, Nicolaus & Company, Incorporated ("Stifel") as the agent (the "Stifel ATM Facility"). Pursuant to the Stifel ATM Facility, we may offer and sell shares of common stock with an aggregate value of up to $125.0 million. We will pay Stifel a commission of up to 3.0% of the gross proceeds of any common stock sold through Stifel. We sold 249,096 shares of our common stock under the Stifel ATM Facility during the nine months ended September 30, 2024 at a weighted-average price per share of $1.64 for aggregate net proceeds of $0.4 million, after deducting commissions. As of September 30, 2024, $119.7 million remained available to be sold under the Stifel ATM Facility.

Cash Requirements

As of September 30, 2024, there were no material changes in our short-term and long-term cash requirements from those disclosed in the "Management's Discussion and Analysis of Financial Condition and Results of Operations" included in our 2023 Annual Report.

As of September 30, 2024, we had cash, cash equivalents and marketable securities of $62.8 million. We will need to raise additional capital to fund our planned future operations. However, we cannot guarantee that we will be able to obtain sufficient additional funding or that if we do obtain additional funding, that such funding will be obtainable on terms satisfactory to us.

Based on our current business plan and current capital resources, management has concluded that there is substantial doubt regarding our ability to continue as a going concern. We expect that our existing cash, cash equivalents and marketable securities at September 30, 2024 will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2025. We have based this estimate on assumptions that may prove to be wrong and we could exhaust our capital resources sooner than we expect.

We expect our expenses to increase substantially if, and as, we:

•
continue research and preclinical and clinical development of our product candidates, including in particular the expenses associated with our clinical trials;
•
incur both internal and third party manufacturing costs to support our preclinical studies and clinical trials of our product candidates and, if approved, their commercialization;
•
seek to identify and develop additional product candidates;
•
make investments in our platform, including the continuing costs of developing and maintaining our internal manufacturing capabilities;
•
seek regulatory and marketing approvals for our product candidates;
•
establish a sales, marketing and distribution infrastructure to commercialize any approved product candidates;
•
adapt our regulatory compliance efforts to incorporate requirements to applicable marketed products;

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•
acquire or in-license products, product candidates, or technologies;
•
maintain, expand, enforce, defend and protect our intellectual property;
•
hire additional clinical, quality control, manufacturing and other scientific personnel;
•
add operational, financial and management information systems and personnel;
•
expand our office, laboratory and manufacturing facility; and
•
experience any delays or encounter any issues with any of the above.

Developing pharmaceutical products, including conducting preclinical studies and clinical trials, is a time-consuming, expensive and uncertain process that takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval for any product candidates or generate revenue from the sale of any product candidate for which we may obtain marketing approval. In addition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales of products that we do not expect to be commercially available for at least several years, if ever.

As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through the public or private sale of our equity, government or private party grants, debt financings or other capital sources, including potential collaborations with other companies or other strategic transactions. To the extent that we raise additional capital through the sale of our equity or convertible debt securities, including through the use of the Stifel ATM Facility, the ownership interest of our shareholders will be or could be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders. Debt financing and equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures or declaring dividends. If we are unable to obtain additional funding, we could be forced to delay, reduce or eliminate some or all of our research and development programs, product portfolio expansion or any commercialization efforts, which could adversely affect our business prospects, or we may be unable to continue operations. If we raise funds through strategic collaborations or other similar arrangements with third parties, we may have to relinquish valuable rights to our platform technology, future revenue streams, research programs or product candidates or may have to grant licenses on terms that may not be favorable to us and/or may reduce the value of our common stock. Our ability to raise additional funds may be adversely impacted by worsening global economic conditions and disruptions to and volatility in the credit and financial markets in the United States and worldwide resulting from geopolitical tensions and adverse macroeconomic conditions or otherwise. Because of the numerous risks and uncertainties associated with product development, we cannot predict the timing or amount of increased expenses, and there is no assurance that we will ever be profitable or generate positive cash flow from operating activities.

We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements that have or are reasonably likely to have a material current or future effect on our financial condition, revenues or expenses, results of operations, liquidity, cash requirements or capital resources.

Cash Flows

The following table provides information regarding our cash flows for the periods presented (in thousands):

Nine Months Ended September 30,

2024

2023

Net cash used in operating activities

$

(75,597

)

$

(76,266

)

Net cash provided by investing activities

96,929

60,673

Net cash provided by financing activities

106

2,913

Net increase (decrease) in cash, cash equivalents and restricted cash equivalents

$

21,438

$

(12,680

)

Operating Activities

Net cash used in operating activities was $75.6 million for the nine months ended September 30, 2024, reflecting a net loss of $86.2 million and net cash used of $2.8 million for operating assets and liabilities, which were offset by non-cash charges of $13.4 million. The non-cash charges primarily consisted of stock-based compensation expense of $8.1 million, non-cash lease expense of $3.8 million and depreciation expense of $2.7 million, offset by $1.1 million of non-cash interest earned on marketable securities.

Net cash used in operating activities was $76.3 million for the nine months ended September 30, 2023, reflecting a net loss of $91.6 million, offset by non-cash adjustments to operating assets and liabilities of $1.4 million and non-cash charges of $13.9 million. The non-cash charges primarily consisted of stock-based compensation expense of $11.5 million, non-cash lease expense of $3.5 million and depreciation expense of $2.6 million, offset by $3.7 million of non-cash interest earned on marketable securities.

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The $0.7 million decrease in net cash used in operating activities for the nine months ended September 30, 2024 compared to the nine months ended September 30, 2023 was primarily due to a decrease in research and development expenses offset by differences in the timing of payments for costs incurred during each respective period.

Investing Activities

Net cash provided by investing activities was $96.9 million for the nine months ended September 30, 2024, which consisted of proceeds of $107.0 million from the maturity of marketable securities, offset by purchases of $9.9 million of marketable securities and $0.2 million of property and equipment. Net cash provided by investing activities was $60.7 million for the nine months ended September 30, 2023, which consisted of proceeds of $120.0 million from the maturity of marketable securities, offset by purchases of $58.4 million of marketable securities and $1.0 million of property and equipment.

Financing Activities

Net cash provided by financing activities was $0.1 million for the nine months ended September 30, 2024, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $0.3 million and proceeds from exercise of stock options and issuance of shares under the ESPP of $0.1 million, offset by $0.3 million of taxes paid related to net share settlement of equity awards. Net cash provided by financing activities was $2.9 million for the nine months ended September 30, 2023, which consisted of proceeds from the issuance of common stock under the Stifel ATM Facility of $4.6 million, and proceeds from stock option exercises and purchases of common stock under the ESPP of $0.2 million, offset by the payment of $0.7 million of issuance costs related to the underwritten public offering under our Shelf Registration Statement and concurrent private placement that closed in December 2022, and $1.2 million of taxes paid related to net share settlement of equity awards.

Contractual Obligations and Other Commitments

Contractual obligations relate to future minimum lease payments for existing non-cancellable leases primarily relating to corporate office and laboratory real estate, with terms expiring through February 2030. During the nine months ended September 30, 2024, there were no significant changes in contractual obligations and commitments from that described under the heading "Management's Discussion and Analysis of Financial Condition and Results of Operations-Contractual Obligations and Other Commitments" in our 2023 Annual Report.

Other commitments include license and collaboration agreements we have entered into with certain parties. Such arrangements require ongoing payments, including payments upon the achievement of certain development, regulatory and commercial milestones, receipt of sublicense income, as well as royalties on commercial sales. Payments under these arrangements are expensed as incurred.

We also have agreements with certain vendors for various services, including services related to clinical operations and support, which we are not contractually able to terminate for convenience and avoid any and all future obligations to the vendors. Under such agreements, we are contractually obligated to make certain payments to vendors to reimburse them for their unrecoverable outlays incurred prior to cancellation. The exact amounts of such obligations are dependent on the timing of termination and the exact terms of the relevant agreement and cannot be reasonably estimated. We do not include these payments in this summary as they are not fixed and estimable.

Recent Accounting Pronouncements

There are no new significant recent accounting pronouncements which may materially impact our financial statements.

Item 3. Quantitative and Qualitative Disclosures About Market Risk.

We are a smaller reporting company as defined by Item 10 of Regulation S-K and are not required to provide the information otherwise required under this item.

Item 4. Controls and Procedures.

Evaluation of Disclosure Controls and Procedures

Our management, with the participation of our Chief Executive Officer and our Chief Financial Officer, evaluated, as of the end of the period covered by this Quarterly Report on Form 10-Q, the effectiveness of our disclosure controls and procedures. The term "disclosure controls and procedures," as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended (the "Exchange Act"), means controls and other procedures of a company that are designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC's rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports

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that it files or submits under the Exchange Act is accumulated and communicated to the company's management, including its principal executive and principal financial officers, or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure. Our management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives, and our management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures.

Based on the evaluation of our disclosure controls and procedures as of September 30, 2024, our Chief Executive Officer and our Chief Financial Officer concluded that our disclosure controls and procedures as of such date were effective at the reasonable assurance level.

Changes in Internal Control over Financial Reporting

There were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) during the quarter ended September 30, 2024 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.

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PART II-OTHERINFORMATION

Item 1. LegalProceedings.

We are not currently a party to any material legal proceedings. From time to time, we may become involved in other litigation or legal proceedings relating to claims arising from the ordinary course of business.

Item 1A. Risk Factors.

Our business is subject to risks and events that, if they occur, could adversely affect our financial condition and results of operations and the trading price of our securities. Except as set forth below, there have been no material changes to the risk factors disclosed in "Part I. Item 1A. Risk Factors" in our 2023 Annual Report.

If we fail to satisfy all applicable requirements of Nasdaq and it determines to delist our common stock, the delisting could adversely affect the market liquidity of our common stock and the market price of our common stock could decrease.

To maintain the listing of our common stock on Nasdaq, we are required to meet certain listing requirements, including, a minimum closing bid price of $1.00 per share. On August 29, 2024, we received notice from Nasdaq that we are no longer in compliance with Nasdaq's Listing Rule 5450(a)(1) because the closing bid price of our common stock had fallen below $1.00 per share for 31 consecutive days. In accordance with Nasdaq Listing Rule 5810(c)(3)(A), we have an initial period of 180 calendar days, or until February 25, 2025 (the "Compliance Date"), to regain compliance with the minimum bid price requirement. To regain compliance, the closing bid price of our common stock must be at least $1.00 per share for a minimum of 10 consecutive business days before the Compliance Date.

If our common stock does not achieve compliance by the Compliance Date, we may be eligible for an additional 180-day period to regain compliance if we apply to transfer the listing of our common stock to the Nasdaq Capital Market. To qualify, we would be required to meet the continued listing requirement for market value of publicly held shares and all other initial listing standards, with the exception of the bid price requirement, and provide written notice to Nasdaq of our intention to cure the deficiency during the second compliance period by effecting a reverse stock split, if necessary.

There can be no assurance that we will maintain compliance with the requirements for listing our common stock on Nasdaq. If we are unable to satisfy the Nasdaq criteria for continued listing, our common stock would be subject to delisting. A delisting of our common stock could negatively impact us by, among other things, (i) reducing the liquidity and market price of our common stock; (ii) reducing the number of investors willing to hold or acquire our common stock, which could negatively impact our ability to raise equity financing; (iii) decreasing the amount of news and analyst coverage of us; (iv) limiting our ability to issue additional securities or obtain additional financing in the future; (v) limiting our ability to use a registration statement to offer and sell freely tradable securities, thereby preventing us from accessing the public capital markets; and (vi) impairing our ability to provide equity incentives to our employees. In addition, delisting from Nasdaq may negatively impact our reputation and, consequently, our business.

Item 5. Other Information

During the quarter ended September 30, 2024, no director or officer, as defined in Rule 16a-1(f), adoptedor terminateda "Rule 10b5-1 trading arrangement" or a "non-Rule 10b5-1 trading arrangement," each as defined in Regulation S-K Item 408.

Appointment of Principal Accounting Officer

As previously disclosed, on June 8, 2024, our board of directors designated Amy Quinlan, our Vice President of Finance, as our interim principal accounting officer, effective as of September 30, 2024. On November 7, 2024, our board of directors removed Ms. Quinlan as interim principal accounting officer and designated Han Choi, M.D., LL.M., our Chief Financial Officer, as our principal accounting officer.

Information regarding Dr. Choi's background and business experience is set forth under Item 5.02 of our Current Report on Form 8-K (File No. 001-39979) filed with the Securities and Exchange Commission on September 30, 2024, and is incorporated herein by reference. There are no arrangements or understandings between Dr. Choi and any other persons pursuant to which he was selected as an officer of Vor Bio. There are also no family relationships between Dr. Choi and any director or executive officer of Vor Bio, and Dr. Choi has no direct or indirect material interest in any related party transaction required to be disclosed pursuant to Item 404(a) of Regulation S-K.

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We did not enter into, or materially amend, any material plan, contract or arrangement to which Dr. Choi is a party or in which he participates in connection with Dr. Choi's designation as principal accounting officer, or make or modify any grant or award to Dr. Choi under any such plan, contract or arrangement.

22

Item 6. Exhibits.

Incorporated by Reference

Exhibit

Number

Description

Form

File No.

Exhibit Number

Filing Date

Filed Herewith

3.1

Amended and Restated Certificate of Incorporation of the Registrant

8-K

001-39979

3.1

February 9, 2021

3.2

Amended and Restated Bylaws of the Registrant

8-K

001-39979

3.2

February 9, 2021

4.1

Form of Common Stock Certificate of the Registrant

S-1/A

333-252175

4.1

February 1, 2021

4.2

Amended and Restated Investors' Rights Agreement, by and among the Registrant and certain of its stockholders, dated June 30, 2020

S-1/A

333-252175

4.2

February 1, 2021

10.1+

Offer Letter, by and between the Registrant and Han Choi, dated September 19, 2024

X

31.1

Certification of Principal Executive and Financial Officer pursuant to Rules 13a-14(a)/15d-14(a) under the Securities Exchange Act of 1934, as amended, as adopted pursuant to Section 302 of the Sarbanes-Oxley Act of 2002

X

32.1†

Certification of Principal Executive and Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002

X

101.INS

Inline XBRL Instance Document

X

101.SCH

Inline XBRL Taxonomy Extension Schema Document

X

101.CAL

Inline XBRL Taxonomy Extension Calculation Linkbase Document

X

101.DEF

Inline XBRL Taxonomy Extension Definition Linkbase Document

X

101.LAB

Inline XBRL Taxonomy Extension Label Linkbase Document

X

101.PRE

Inline XBRL Taxonomy Extension Presentation Linkbase Document

X

104

The cover page from the Company's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, formatted in Inline XBRL.

+ Indicates management contract or compensatory plan.

† The certifications furnished in Exhibit 32.1 hereto are deemed to be furnished with this Quarterly Report on Form 10-Q and will not be deemed to be "filed" for purposes of Section 18 of the Exchange Act. Such certifications will not be deemed to be incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Exchange Act, except to the extent that the Registrant specifically incorporates it by reference.

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SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

VOR BIOPHARMA INC.

Date: November 7, 2024

By:

/s/ Robert Ang

Robert Ang

President and Chief Executive Officer (Principal Executive Officer)

Date: November 7, 2024

By:

/s/ Han Choi

Han Choi

Chief Financial Officer (Principal Financial Officer and Principal Accounting Officer)

24