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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, 2021


☐	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 400 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.

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 5, 2021 was 37,289,504.

Table of Contents


		Page
PART I.	FINANCIAL INFORMATION	1

Item 1.	Financial Statements (Unaudited)	1
	Condensed Consolidated Balance Sheets as of September 30, 2021 and December 31, 2020	1
	Condensed Consolidated Statements of Operations and Comprehensive Loss for the Three and Nine Months Ended September 30, 2021 and 2020	2
	Condensed Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders’ Equity (Deficit) for the Three and Nine Months Ended September 30, 2021 and 2020	3
	Condensed Consolidated Statements of Cash Flows for the Nine Months Ended September 30, 2021 and 2020	5
	Notes to Unaudited Condensed Consolidated Financial Statements	6
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	13
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	20
Item 4.	Controls and Procedures	21

PART II.	OTHER INFORMATION	22

Item 1.	Legal Proceedings	22
Item 1A.	Risk Factors	22
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	72
Item 5.	Other Information	72
Item 6.	Exhibits	74
Signatures		75

Note Regarding Company References

Throughout this Quarterly Report on Form 10-Q, the “Company,” “Vor,” “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,” “intend,” “target,” “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, 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 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 our research and development programs;



the timing of clinical trials and timing of expected clinical results for our current and future product candidates;



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;



the COVID-19 pandemic, which could continue to adversely impact our business, including our preclinical studies and clinical trials;



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 before we can expect to generate any revenue from product sales;



our ability to establish or maintain collaborations or strategic relationships;



our ability to identify, recruit and retain key personnel;



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 investments 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;



our estimates regarding future revenue, expenses and needs for additional financing;



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.

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. We have included important factors in this Quarterly Report on Form 10-Q and our Annual Report on Form 10-K for the year ended December 31, 2020, particularly in the "Summary Risk Factors" and “Risk Factors” sections, that could cause actual results or events to differ materially from the forward-looking statements that we make.

Summary Risk Factors

Our business is subject to a number of risks that if realized could materially affect our business, financial condition, results of operations, cash flows and access to liquidity. These risks are discussed more fully in the “Risk Factors” section of this Quarterly Report on Form 10-Q. Our principal risks include the following:



We have incurred significant net losses since inception. We expect to incur net losses for the foreseeable future and may never achieve or maintain profitability.



We will need substantial additional funding. If we are unable to raise capital when needed, we would be forced to delay, reduce or eliminate our research and product development programs or future commercialization efforts.



We have a limited operating history, have not yet completed any clinical trials and have no history of commercializing products, which may make it difficult to evaluate the success of our business to date and to assess our future viability.



Engineered hematopoietic stem cells (“eHSCs”) are a novel technology that is not yet clinically validated for human use. The approaches we are taking to create eHSCs are unproven and may never lead to marketable products.



We are substantially dependent on the success of our two most advanced product candidates, VOR33 and VCAR33. If we are unable to complete development of, obtain approval for and commercialize VOR33 or VCAR33 in a timely manner, our business will be harmed.



We may not be successful in our efforts to identify, develop or commercialize additional product candidates. If these efforts are unsuccessful, we may never become a commercial stage company or generate any revenues.



We have not successfully tested our product candidates in clinical trials and any favorable preclinical results are not predictive of results that may be observed in clinical trials.



Development of a product candidate such as VOR33, which is intended for use in combination or in sequence with an already approved therapy, will present increased complexity and more or different challenges than development of a product candidate for use as a single agent.



If VOR33, VCAR33, VOR33 in combination or in sequence with VCAR33 as a targeted therapeutic, which we refer to as the VOR33/VCAR33 Treatment System, or any of the other product candidates we may develop, or the delivery modes we rely on to administer them, cause serious adverse events, undesirable side effects or unexpected characteristics, such events, side effects or characteristics could delay or prevent regulatory approval of the product candidates, limit their commercial potential or result in significant negative consequences following any potential marketing approval.



We face significant competition in an environment of rapid technological change, and there is a possibility that our competitors may achieve regulatory approval before us or develop therapies that are safer or more advanced or effective than ours, which may harm our financial condition and our ability to successfully market or commercialize VOR33, VCAR33, the VOR33/VCAR33 Treatment System or any other product candidates we may develop.



Adverse public perception of genetic medicines, and of genome engineering in particular, may negatively impact regulatory approval of, and/or demand for, our potential products.



Gene engineering technology is subject to a number of challenges and risks. Because genome engineering technology is novel and the regulatory landscape that will govern VOR33, VCAR33, the VOR33/VCAR33 Treatment System and any future product candidates we may develop is uncertain and may change, we cannot predict the time and cost of obtaining regulatory approval, if we receive it at all, for our product candidates.

iii



Because we are developing product candidates using new technologies, as well as potential mechanisms of action for which there are few precedents, there is increased risk that the U.S. Food and Drug Administration, the European Medicines Agency or other regulatory authorities may not consider the endpoints of our clinical trials to provide clinically meaningful results and that these results may be difficult to analyze.



We contract with third parties for the manufacture and supply of materials for development of our product candidates and advancement of our current clinical trial, as well as our research programs and preclinical studies, and we expect to continue to do so for future clinical trials and for commercialization of VOR33, VCAR33, the VOR33/VCAR33 Treatment System and any other product candidates we may develop. This reliance on third parties increases the risk that we will not have sufficient quantities of such materials, product candidates or any products that we may develop and commercialize, or that such supply will not be available to us at an acceptable cost, which could delay, prevent or impair our development or commercialization efforts.



We are highly dependent on intellectual property licensed from third parties and termination of any of these licenses could result in the loss of significant rights, which would harm our business.



We may not be successful in acquiring or in-licensing necessary rights to key technologies underlying VOR33, VCAR33, the VOR33/VCAR33 Treatment System or any future product candidates we may develop.



Third-party claims of intellectual property infringement, misappropriation or other violations may prevent or delay our product discovery and development efforts and have a material adverse effect on our business.



The COVID-19 pandemic has caused, and could continue to cause, severe disruptions in the United States, regional and global economies and could seriously harm our development efforts, increase our costs and expenses and have a material adverse effect on our business, financial condition and results of operations.

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 amounts)	2021			2020
Assets
Current assets:
Cash and cash equivalents	$	143,034		$	48,539
Investments		82,934			—
Prepaid expenses		5,355			467
Other current assets		749			100
Total current assets		232,072			49,106
Restricted cash equivalents		2,413			1,559
Property and equipment, net		5,990			4,728
Operating lease right-of-use assets		16,016			17,117
Other assets		2,141			3,398
Total assets	$	258,632		$	75,908
Liabilities, redeemable convertible preferred stock and stockholders’ equity (deficit)
Current liabilities:
Accounts payable	$	1,957		$	2,361
Accrued expenses		5,676			6,260
Operating lease liability		1,147			863
Other current liabilities		430			723
Total current liabilities		9,210			10,207
Long-term liabilities:
Operating lease liabilities—non-current		16,465			17,430
Total liabilities		25,675			27,637
Series A-1 redeemable convertible preferred stock, $0.0001 par value; 0 shares and 20,000,000 shares authorized, issued, and outstanding as of September 30, 2021 and December 31, 2020, respectively (liquidation preference of $4,000 as of December 31, 2020)		—			2
Series A-2 redeemable convertible preferred stock, $0.0001 par value; 0 and 107,194,866 shares authorized, issued, and outstanding as of September 30, 2021 and December 31, 2020, respectively (liquidation preference of $42,878 as of December 31, 2020)		—			42,786
Series B redeemable convertible preferred stock, $0.0001 par value; 0 shares and 211,778,825 shares authorized as of September 30, 2021 and December 31, 2020, respectively; 0 shares and 124,519,220 shares issued and outstanding as of September 30, 2021 and December 31, 2020, respectively (liquidation preference of $64,750 as of December 31, 2020)		—			64,548
Stockholders’ equity (deficit):
Preferred stock, $0.0001 par value; 10,000,000 and 0 shares authorized as of September 30, 2021 and December 31, 2020, respectively; 0 shares issued and outstanding as of September 30, 2021 and December 31, 2020		—			—
Common stock, $0.0001 par value; 400,000,000 and 420,000,000 shares authorized as of September 30, 2021 and December 31, 2020 respectively; 37,247,414 and 893,231 shares issued and 37,018,282 and 505,074 outstanding as of September 30, 2021 and December 31, 2020, respectively		4			1
Additional paid-in capital		344,824			2,158
Accumulated deficit		(111,871	)		(61,224	)
Total stockholders’ equity (deficit)		232,957			(59,065	)
Total liabilities, redeemable convertible preferred stock and stockholders’ equity (deficit)	$	258,632		$	75,908

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

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)	2021			2020			2021			2020
Operating expenses:
Research and development	$	12,925		$	8,142		$	34,836		$	20,288
General and administrative		5,677			3,643			15,876			7,415
Total operating expenses	$	18,602		$	11,785		$	50,712		$	27,703
Loss from operations	$	(18,602	)	$	(11,785	)	$	(50,712	)	$	(27,703	)
Other income:
Interest income		48			—			65			29
Total other income		48			—			65			29
Net loss and comprehensive loss	$	(18,554	)	$	(11,785	)	$	(50,647	)	$	(27,674	)
Cumulative dividends on redeemable convertible preferred stock		—			(2,185	)		(1,228	)		(3,755	)
Net loss attributable to common stockholders	$	(18,554	)	$	(13,970	)	$	(51,875	)	$	(31,429	)
Net loss per share attributable to common stockholders, basic and diluted	$	(0.50	)	$	(61.23	)	$	(1.62	)	$	(177.84	)
Weighted-average common shares outstanding, basic and diluted		36,934,311			228,144			32,067,535			176,726

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

Vor Biopharma Inc.

CONDENSED CONSOLIDATED Statements of Redeemable Convertible Preferred Stock and

Stockholders’ EQUITY (Deficit)

(Unaudited)


	Series A-1 Preferred Stock						Series A-2 Preferred Stock						Series B Preferred Stock						Common Stock				Additional Paid-In		Accumulated			Total Stockholders’
(in thousands, except share amounts)	Shares			Amount			Shares			Amount			Shares			Amount			Shares		Amount		Capital		Deficit			Equity (Deficit)
Balance at December 31, 2020		20,000,000		$	2			107,194,866		$	42,786			124,519,220		$	64,548			505,074	$	1	$	2,158	$	(61,224	)	$	(59,065	)
Issuance of Series B redeemable convertible preferred stock		—			—			—			—			87,259,605			45,375			—		—		—		—			—
Conversion of redeemable convertible preferred stock into common stock upon closing of initial public offering		(20,000,000	)		(2	)		(107,194,866	)		(42,786	)		(211,778,825	)		(109,923	)		24,924,501		2		152,709		—			152,711
Issuance of common shares upon closing of initial public offering, net of offering costs and underwriter fees of $17,132		—			—			—			—			—			—			11,302,219		1		186,307		—			186,308
Issuance of common stock upon vesting and exercise of stock options		—			—			—			—			—			—			38,216		—		65		—			65
Stock-based compensation expense		—			—			—			—			—			—			—		—		891		—			891
Net loss		—			—			—			—			—			—			—		—		—		(13,723	)		(13,723	)
Balance at March 31, 2021		—		$	—			—		$	—			—		$	—			36,770,010	$	4	$	342,130	$	(74,947	)	$	267,187
Issuance of common stock upon vesting and exercise of stock options		—			—			—			—			—			—			115,362		—		224		—			224
Stock-based compensation expense		—			—			—			—			—			—			—		—		1,067		—			1,067
Net loss		—			—			—			—			—			—			—		—		—		(18,370	)		(18,370	)
Balance at June 30, 2021		—		$	—			—		$	—			—		$	—			36,885,372	$	4	$	343,421	$	(93,317	)	$	250,108
Issuance of common stock upon vesting and exercise of stock options		—			—			—			—			—			—			132,910		—		249		—			249
Stock-based compensation expense		—			—			—			—			—			—			—		—		1,154		—			1,154
Net loss		—			—			—			—			—			—			—		—		—		(18,554	)		(18,554	)
Balance at September 30, 2021		—		$	—			—		$	—			—		$	—			37,018,282	$	4	$	344,824	$	(111,871	)	$	232,957


	Series A-1 Preferred Stock				Series A-2 Preferred Stock				Series B Preferred Stock				Common Stock				Additional Paid-In		Accumulated			Total Stockholders’
(in thousands, except share amounts)	Shares		Amount		Shares		Amount		Shares		Amount		Shares		Amount		Capital		Deficit			Equity (Deficit)
Balance at December 31, 2019		20,000,000	$	2		62,819,866	$	25,067		—	$	—		119,936	$	—	$	458	$	(17,887	)	$	(17,429	)
Issuance of Series A-2 Redeemable Convertible Preferred Stock		—		—		44,375,000		17,719		—		—		—		—		—		—			—
Issuance of common stock upon exercise of stock options		—		—		—		—		—		—		31,384		—		43		—			43
Stock-based compensation expense		—		—		—		—		—		—		—		—		112		—			112
Net loss		—		—		—		—		—		—		—		—		—		(7,849	)		(7,849	)
Balance at March 31, 2020		20,000,000	$	2		107,194,866	$	42,786		—	$	—		151,320	$	—	$	613	$	(25,736	)	$	(25,123	)
Issuance of Series B Redeemable Convertible Preferred Stock		—		—		—		—		124,519,220		64,548		—				—		—			—
Issuance of common stock upon exercise of stock options		—		—		—		—		—		—		13,990		—		17		—			17
Stock-based compensation expense		—		—		—		—		—		—		—		—		153		—			153
Net loss		—		—		—		—		—		—		—		—		—		(8,040	)		(8,040	)
Balance at June 30, 2020		20,000,000	$	2		107,194,866	$	42,786		124,519,220	$	64,548