10-Q 1 vor-20240331.htm 10-Q 10-Q
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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, 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 May 3, 2024 was 68,259,602.

 


 

Table of Contents

 

Page

PART I.

FINANCIAL INFORMATION

1

 

 

 

Item 1.

Financial Statements (Unaudited)

1

Condensed Consolidated Balance Sheets as of March 31, 2024 and December 31, 2023

1

Condensed Consolidated Statements of Operations and Comprehensive Loss for the Three Months Ended March 31, 2024 and 2023

2

 

Condensed Consolidated Statements of Stockholders’ Equity for the Three Months Ended March 31, 2024 and 2023

3

Condensed Consolidated Statements of Cash Flows for the Three Months Ended March 31, 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

11

Item 3.

Quantitative and Qualitative Disclosures About Market Risk

17

Item 4.

Controls and Procedures

17

 

 

 

PART II.

OTHER INFORMATION

19

 

 

 

Item 1.

Legal Proceedings

19

Item 1A.

Risk Factors

19

Item 2.

Unregistered Sales of Equity Securities and Use of Proceeds

19

Item 6.

Exhibits

20

 

Signatures

21

 

 

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 and our need for or ability to obtain additional funding;
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 “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)

 

 

 

March 31,

 

 

December 31,

 

(in thousands, except share and per share amounts)

 

2024

 

 

2023

 

Assets

 

 

 

 

 

 

Current assets:

 

 

 

 

 

 

Cash and cash equivalents

 

$

44,964

 

 

$

31,360

 

Marketable securities

 

 

62,515

 

 

 

105,815

 

Prepaid expenses

 

 

3,831

 

 

 

3,153

 

Other current assets

 

 

507

 

 

 

475

 

Total current assets

 

 

111,817

 

 

 

140,803

 

Restricted cash equivalents

 

 

2,413

 

 

 

2,413

 

Property and equipment, net

 

 

9,165

 

 

 

10,050

 

Operating lease right-of-use assets

 

 

38,816

 

 

 

40,048

 

Other assets

 

 

4,819

 

 

 

4,812

 

Total assets

 

$

167,030

 

 

$

198,126

 

Liabilities and stockholders’ equity

 

 

 

 

 

 

Current liabilities:

 

 

 

 

 

 

Accounts payable

 

$

2,325

 

 

$

815

 

Accrued liabilities

 

 

6,797

 

 

 

10,877

 

Operating lease liabilities

 

 

3,957

 

 

 

3,830

 

Other current liabilities

 

 

134

 

 

 

50

 

Total current liabilities

 

 

13,213

 

 

 

15,572

 

Long-term liabilities:

 

 

 

 

 

 

Operating lease liabilities—non-current

 

 

30,782

 

 

 

31,830

 

Total liabilities

 

 

43,995

 

 

 

47,402

 

Stockholders’ equity:

 

 

 

 

 

 

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

 

 

 

 

 

 

Common stock, $0.0001 par value; 400,000,000 shares authorized as of March 31, 2024 and December 31, 2023; 68,223,496 and 67,901,610 shares issued and 68,215,771 and 67,891,311 outstanding as of March 31, 2024 and December 31, 2023, respectively

 

 

7

 

 

 

7

 

Additional paid-in capital

 

 

493,999

 

 

 

490,874

 

Accumulated other comprehensive loss

 

 

(87

)

 

 

(77

)

Accumulated deficit

 

 

(370,884

)

 

 

(340,080

)

Total stockholders’ equity

 

 

123,035

 

 

 

150,724

 

Total liabilities and stockholders’ equity

 

$

167,030

 

 

$

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 March 31,

 

(in thousands, except share and per share amounts)

 

2024

 

 

2023

 

Operating expenses:

 

 

 

 

 

 

Research and development

 

$

24,322

 

 

$

21,915

 

General and administrative

 

 

8,004

 

 

 

8,507

 

Total operating expenses

 

$

32,326

 

 

$

30,422

 

Loss from operations

 

$

(32,326

)

 

$

(30,422

)

Other income:

 

 

 

 

 

 

Interest income

 

 

1,522

 

 

 

1,989

 

Total other income

 

 

1,522

 

 

 

1,989

 

Net loss

 

$

(30,804

)

 

$

(28,433

)

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

 

$

(0.45

)

 

$

(0.43

)

Weighted-average common shares outstanding,
   basic and diluted

 

 

68,030,966

 

 

 

66,265,703

 

 

 

 

 

 

 

 

Other comprehensive income (loss):

 

 

 

 

 

 

Unrealized (loss) gain on available for sale marketable securities

 

 

(10

)

 

 

596

 

Total other comprehensive (loss) income

 

 

(10

)

 

 

596

 

 

 

$

(30,814

)

 

$

(27,837

)

 

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

 

 

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 and vesting
    and exercise of stock options, net of shares withheld for taxes

 

 

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

 

 

 

 

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 and exercise of stock options, net of shares withheld for taxes

 

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

 

 

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

3


 

Vor Biopharma Inc.

condensed CONSOLIDATED StatementS of Cash Flows

(unaudited)

 

 

 

Three Months Ended March 31,

 

(in thousands)

 

2024

 

 

2023

 

Cash flows from operating activities

 

 

 

 

 

 

Net loss

 

$

(30,804

)

 

$

(28,433

)

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

 

 

 

 

 

 

Depreciation expense

 

 

943

 

 

 

837

 

Non-cash lease expense

 

 

1,232

 

 

 

1,139

 

Stock-based compensation

 

 

3,081

 

 

 

4,068

 

Interest amortization on marketable securities

 

 

(797

)

 

 

(1,202

)

Changes in operating assets and liabilities:

 

 

 

 

 

 

Operating lease liabilities

 

 

(921

)

 

 

(730

)

Prepaid expenses and other current assets

 

 

(710

)

 

 

7

 

Accounts payable and accrued liabilities

 

 

(2,482

)

 

 

(88

)

Other assets

 

 

(7

)

 

 

134

 

Net cash used in operating activities

 

 

(30,465

)

 

 

(24,268

)

Cash flow from investing activities

 

 

 

 

 

 

Purchases of marketable securities

 

 

(9,913

)

 

 

(23,408

)

Proceeds from maturities of marketable securities

 

 

54,000

 

 

 

30,000

 

Purchases of property and equipment

 

 

(114

)

 

 

(94

)

Net cash provided by investing activities

 

 

43,973

 

 

 

6,498

 

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

 

 

278

 

 

 

3,622

 

Shares repurchased for tax withholdings upon vesting of restricted stock unit awards

 

 

(182

)

 

 

(400

)

Proceeds from stock option exercises

 

 

 

 

 

14

 

Net cash provided by financing activities

 

 

96

 

 

 

2,519

 

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

 

 

13,604

 

 

 

(15,251

)

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

 

$

33,773

 

 

$

60,119

 

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

 

$

47,377

 

 

$

44,868

 

Supplemental disclosure of non-cash activities

 

 

 

 

 

 

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

 

$

 

 

$

270

 

Purchases of property and equipment in accounts payable and accrued liabilities

 

$

14

 

 

$

3

 

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

 

$

92

 

 

$

 

 

A reconciliation of the cash, cash equivalents and restricted cash 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 Three Months Ended March 31,

 

(in thousands)

 

2024

 

 

2023

 

Cash and cash equivalents

 

$

44,964

 

 

$

42,455

 

Restricted cash equivalents

 

 

2,413

 

 

 

2,413

 

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

 

$

47,377

 

 

$

44,868

 

 

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

4


 

 

VOR BIOPHARMA INC.

NOTES TO CONDENSED CONSOLIDATED 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. The Company believes that its existing cash, cash equivalents and marketable securities at March 31, 2024 will be sufficient to allow the Company to fund its current operations through at least a period of one year after the date the financial statements are issued. 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.

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”).

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

5


 

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 three months ended March 31, 2024, there have been no changes to the Company’s significant accounting policies as described in the 2023 Annual Report.

3. Marketable Securities

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

 

 

March 31, 2024

 

(in thousands)

 

Amortized Cost

 

 

Gross Unrealized Holding Gains

 

 

Gross Unrealized Holding Losses

 

 

Fair Value

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Maturing in one year or less

 

 

 

 

 

 

 

 

 

 

 

 

U.S. Treasury Bill

 

$

8,924

 

 

$

 

 

$

(1

)

 

$

8,923

 

U.S. Treasuries

 

 

43,758

 

 

 

 

 

 

(35

)

 

 

43,723

 

Maturing after one year through five years

 

 

 

 

 

 

 

 

 

 

 

 

U.S. Treasuries

 

 

9,920

 

 

 

 

 

 

(51

)

 

 

9,869

 

Total

 

$

62,602

 

 

$

 

 

$

(87

)

 

$

62,515

 

 

 

 

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 following tables summarize the fair value and gross unrealized losses aggregated by category and the length of time that individual securities have been in an unrealized loss position:

 

 

March 31, 2024

 

 

 

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. Treasury Bill

 

$

8,923

 

 

$

(1

)

 

$

 

 

$

 

 

$

8,923

 

 

$

(1

)

U.S. Treasuries

 

 

26,762

 

 

 

(53

)

 

 

26,830

 

 

 

(33

)

 

 

53,592

 

 

 

(86

)

Total

 

$

35,685

 

 

$

(54

)

 

$

26,830

 

 

$

(33

)

 

$

62,515

 

 

$

(87

)

 

 

 

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 holds investment grade marketable securities considered to be in an unrealized loss position. Although these marketable securities are held at an unrealized loss position at March 31, 2024, the Company does not intend to sell the marketable securities prior to the value of the securities being recovered, and the Company has concluded that it is more likely than not that the marketable securities cost basis values will be recovered prior to sale of the securities and that there are no conditions or events that might require the Company to sell the securities before recovery of the cost basis occurs. Further, 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.

6


 

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:

 

 

March 31, 2024

 

(in thousands)

 

Level 1

 

 

Level 2

 

 

Level 3

 

 

Total

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Cash equivalents

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

$

44,868

 

 

$

 

 

$

 

 

$

44,868

 

Marketable securities

 

 

 

 

 

 

 

 

 

 

 

 

U.S. Treasury Bill

 

 

8,923

 

 

 

 

 

 

 

 

 

8,923

 

U.S. Treasuries

 

 

 

 

 

53,592

 

 

 

 

 

 

53,592

 

Total marketable securities

 

 

8,923

 

 

 

53,592

 

 

 

 

 

 

62,515

 

Restricted cash equivalents

 

 

 

 

 

 

 

 

 

 

 

 

Money market funds

 

 

2,413

 

 

 

 

 

 

 

 

 

2,413

 

Total

 

$

56,204

 

 

$

53,592

 

 

$

 

 

$

109,796

 

 

 

 

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 no transfers between levels during the three months ended March 31, 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.

5. Property and Equipment, Net

Property and equipment, net consisted of the following:

 

 

 

March 31,

 

 

December 31,

 

(in thousands)

 

2024

 

 

2023

 

Laboratory equipment

 

$

10,090

 

 

$

10,028

 

Manufacturing equipment

 

 

7,028

 

 

 

6,936

 

Computer equipment

 

 

432

 

 

 

432

 

Furniture, fixtures and other

 

 

620

 

 

 

599

 

Construction in progress

 

 

29

 

 

 

146

 

Total

 

 

18,199

 

 

 

18,141

 

Less: Accumulated depreciation

 

 

(9,034

)

 

 

(8,091

)

Property and equipment, net

 

$

9,165

 

 

$

10,050

 

Depreciation expense for the three months ended March 31, 2024 and 2023 was $0.9 million and $0.8 million, respectively.

7


 

6. Accrued Liabilities

Accrued liabilities consisted of the following:

 

 

 

March 31,

 

 

December 31,

 

(in thousands)

 

2024

 

 

2023

 

Employee-related expenses

 

$

1,908

 

 

$

5,962

 

Professional fees

 

 

1,192

 

 

 

1,245

 

Clinical expenses

 

 

1,748

 

 

 

1,495

 

Manufacturing expenses

 

 

1,138

 

 

 

842

 

Research and development expenses

 

 

592

 

 

 

1,059

 

Other

 

 

219

 

 

 

274

 

Total accrued liabilities

 

$

6,797

 

 

$

10,877

 

 

7. Stock-Based Compensation

2023 Inducement Plan

As of March 31, 2024, the Company had 3,127,811 shares of its common stock available for future issuance under the 2023 Inducement Plan.

2021 Equity Incentive Plan

As of March 31, 2024, the Company had 1,845,382 shares of its common stock available for future issuance under its 2021 Equity Incentive Plan.

Stock Options

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

 

 

Three Months Ended March 31,

 

 

 

2024

 

 

2023

 

Expected term (in years)

 

 

6.0

 

 

 

6.0

 

Expected volatility

 

 

90.8

%

 

 

82.1

%

Risk-free interest rate

 

 

4.0

%

 

 

3.8

%

Dividend yield

 

 

 

 

 

 

During the three months ended March 31, 2024 and 2023, the Company granted stock options to purchase 1,449,975 shares and 1,543,219 shares of its common stock, respectively, with a weighted-average grant-date fair value of $1.84 and $3.98 per share, respectively. As of March 31, 2024, total unrecognized compensation expense related to stock options was $14.0 million, which is expected to be recognized over a weighted-average period of 2.6 years.

As of March 31, 2024, options for 7,725 shares of Company common stock with a weighted-average exercise price of $4.90 were exercised and unvested. The underlying proceeds from the unvested exercises of less than $0.1 million is recorded in other current liabilities on the condensed consolidated balance sheet.

Restricted Stock Units

During the three months ended March 31, 2024 and 2023, the Company granted 1,070,025 restricted stock units and 645,360 restricted stock units, respectively, with a weighted-average grant date fair value of $2.41 and $5.55 per share, respectively. As of March 31, 2024, total unrecognized compensation expense related to restricted stock units was $6.0 million, which is expected to be recognized over a weighted-average period of 2.2 years.

Employee Stock Purchase Plan

As of March 31, 2024, the Company had 1,968,620 shares of its common stock available for issuance under its Employee Stock Purchase Plan (“ESPP”).

8


 

The Company did not issue any shares of common stock under the ESPP during the three months ended March 31, 2024 and 2023.

Stock-Based Compensation

Stock-based compensation expense was allocated as follows:

 

 

Three Months Ended March 31,

 

(in thousands)

 

2024

 

 

2023

 

Research and development

 

$

1,405

 

 

$

2,354

 

General and administrative

 

 

1,676

 

 

 

1,714

 

Total stock-based compensation expense

 

$

3,081

 

 

$

4,068

 

 

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, 2022 and April 29, 2022, respectively. The terms of the Lease Amendments are through September 2030 for approximately $8.4 million and $22.3 million in fixed payments for the First Lease Amendment and Second Lease Amendment, respectively. There are no options to 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 $2.4 million for the benefit of the Landlord, which has been secured by money market investments and is presented as restricted cash equivalents.

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 March 31,

 

(in thousands)

 

2024

 

 

2023

 

Operating lease cost

 

$

1,951

 

 

$

1,953

 

Variable lease cost

 

 

530

 

 

 

597

 

Total lease cost

 

$

2,481

 

 

$

2,550

 

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)

 

March 31, 2024

 

 

December 31, 2023

 

Assets

 

 

 

 

 

 

Operating lease right-of-use assets

 

$

38,816

 

 

$

40,048

 

Liabilities

 

 

 

 

 

 

Operating lease liabilities, current

 

$

3,957

 

 

$

3,830

 

Operating lease liabilities, non-current

 

 

30,782

 

 

 

31,830

 

Total lease liabilities

 

$

34,739

 

 

$

35,660

 

Weighted-Average Lease Term and Discount Rate

 

 

 

 

 

 

Weighted-average remaining lease term (years)

 

6.5

 

 

6.7

 

Weighted-average discount rate

 

 

8.2

%

 

 

8.2

%

 

9


 

The following table represents other lease activity:

 

 

Three Months Ended March 31,

 

(in thousands)

 

2024

 

 

2023

 

Other Information

 

 

 

 

 

 

Cash paid for amounts included in the measurement of lease liabilities

 

 

 

 

 

 

Operating cash flows for operating leases

 

$

1,639

 

 

$

1,544

 

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

 

$

 

 

$

270

 

 

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 2023 Annual 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 months ended March 31, 2024 and 2023:

 

 

Three Months Ended March 31,

 

(in thousands, except share and per share amounts)

 

2024

 

 

2023

 

Numerator:

 

 

 

 

 

 

Net loss attributable to common stockholders

 

$

(30,804

)

 

$

(28,433

)

Denominator:

 

 

 

 

 

 

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

 

 

68,030,966

 

 

 

66,265,703

 

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

 

$

(0.45

)

 

$

(0.43

)

The Company’s potentially dilutive securities were stock options, unvested restricted stock and restricted stock units. Based on the amounts outstanding as of March 31, 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 March 31,

 

 

 

2024

 

 

2023

 

Options to purchase common stock

 

 

9,617,839

 

 

 

7,872,497

 

Unvested restricted stock

 

 

7,725

 

 

 

56,162

 

Restricted stock units

 

 

1,870,070

 

 

 

1,792,185

 

 

10


 

Item 2. Management’s Discussion and Analysis of Financial 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 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. 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 for this trial most recently in December 2023. The data showed that primary neutrophil engraftment occurred in all eight patients treated with trem-cel. Three out of three patients treated with Mylotarg experienced hematologic protection from deep cytopenias through repeat doses, suggesting that trem-cel transplants shielded patients’ healthy cells from the on-target toxicity typically seen with Mylotarg treatment. We expect to release additional engraftment and hematologic protection data in the second half of 2024. If successful, this trial will provide important validating evidence of the potential of trem-cel and our broader approach.

VCAR33ALLO is 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 VCAR33ALLO in VBP301 and expect to treat multiple additional patients in the first half of 2024. We expect to report initial data in the second half of 2024.

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 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.

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

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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 March 31, 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 $464.3 million.

We have incurred significant operating losses since inception, including net losses of $30.8 million for the three months ended March 31, 2024 and $117.9 million for the year ended December 31, 2023. As of March 31, 2024, we had an accumulated deficit of $370.9 million.

As of March 31, 2024, we had cash, cash equivalents and marketable securities of $107.5 million. We expect that our cash, cash equivalents and marketable securities at March 31, 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.

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.

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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;
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.

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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 and marketable securities held in financial institutions.

Results of Operations

Comparison of Three Months Ended March 31, 2024 and 2023

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

 

 

Three Months Ended
March 31,

 

 

 

 

 

 

2024

 

 

2023

 

 

Change

 

Operating expenses:

 

 

 

 

 

 

 

 

 

Research and development

 

$

24,322

 

 

$

21,915

 

 

$

2,407

 

General and administrative

 

 

8,004

 

 

 

8,507

 

 

 

(503

)

Total operating expenses

 

 

32,326

 

 

 

30,422

 

 

 

1,904