Vor Biopharma Inc.
For the period ended Dec 31, 2024. Filed Mar 20, 2025.
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FY2024 · Filed Mar 20, 2025
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Notes to Consolidated Financial Statements
- 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, and the need to obtain additional financing.
Liquidity and Capital Resources
As of December 31, 2024, the Company had$91.9 million of cash, cash equivalents and marketable securities and an accumulated deficit of$457.0 million. 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 for a period of one year after the date these 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 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.
- Summary of Significant Accounting Policies
Basis of Presentation
The accompanying consolidated financial statements have been prepared in conformity with accounting principles generally accepted in the United States of America (“GAAP”). Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the Accounting Standards Codification (“ASC”) or an Accounting Standards Update (“ASU”) issued by the Financial Accounting Standards Board (“FASB”).
Use of Estimates
The preparation of the 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 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 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 related research and development expenses.
Segments
Operating segments are defined as components of an enterprise for which separate and discrete information is available for evaluation by the chief operating decision-maker in deciding how to allocate resources and assess performance. The Company hasone operating segment. The Company’s chief operating decision maker, its Chief Executive Officer, manages the Company’s operations on an aggregate basis for the purpose of allocating resources. Refer to Note 15 for more information.
Cash and Cash Equivalents
The Company considers highly-liquid investments purchased with an original maturity date of ninety days or less from the date of purchase to be cash equivalents. Cash and cash equivalents include cash held in banks and amounts held in money market funds. Cash equivalents are stated at cost, which approximates market value.
Marketable Securities
Investments in marketable debt securities are classified as available-for-sale. Marketable securities with maturities beyond one year may be classified as short-term based on their highly liquid nature and because such securities represent an investment of cash that is available for current operations.
Available-for-sale marketable securities are reported at fair value at each balance sheet date. Unrealized gains or losses are reported as a component of accumulated other comprehensive income (loss), a component of stockholders’ equity. Amortization and accretion of premiums and discounts are recorded in interest income. Realized gains and losses are included as a component of other income, net in the consolidated statements of operations.
The Company evaluates its marketable securities with unrealized losses for impairment. When assessing marketable securities for unrealized declines in value, the Company considers whether the decline in value is related to a credit loss or non-credit loss. For credit losses, the Company reduces the marketable security to fair value through an allowance for credit losses recorded to the balance sheet and corresponding charge to the statement of operations. The allowance for credit losses and corresponding impairment charge is adjusted each period for changes in fair value. For non-credit losses, the Company reduces the marketable security to fair value through a charge to the statement of operations and comprehensive loss, reported as a component of accumulated other comprehensive income (loss) in stockholders’ equity. No credit losses were recorded during the periods presented.
Restricted Cash Equivalents
The Company had$2.4 million of restricted cash equivalents in the form of a letter of credit related to a lease at December 31, 2024 and 2023.
Comprehensive income (loss)
Comprehensive income (loss) includes net loss, as well as other changes in stockholders’ equity that result from transactions and economic events other than those with stockholders.
Concentrations of Credit Risk
Financial instruments that potentially expose the Company to concentrations of credit risk consist primarily of cash and cash equivalents, restricted cash equivalents and marketable securities. The Company maintains deposits in accredited financial institutions in excess of federally insured limits. The Company deposits its cash in financial institutions that it believes have high credit quality and has not experienced any losses on such accounts and does not believe it is exposed to any unusual credit risk beyond the normal credit risk associated with commercial banking relationships.
The Company’s marketable securities may consistof money market funds and marketable debt securities, including corporate bonds and U.S. Treasury securities. The Company’s investment policy limits instruments to investment grade securities with high credit quality issuers with the objective to preserve capital and to maintain liquidity until the funds can be used in business operations.
Fair Value Measurements
Certain assets and liabilities are carried at fair value under GAAP. Fair value is defined as the exchange price that would be received for an asset or paid to transfer a liability (an exit price) in the principal or most advantageous market for the asset or liability in an orderly transaction between market participants on the measurement date. Valuation techniques used to measure fair value must maximize the use of observable inputs and minimize the use of unobservable inputs. Financial assets and liabilities carried at fair value are to be classified and disclosed in one of the following three levels of the fair value hierarchy, of which the first two are considered observable and the last is considered unobservable:
Level 1—Quoted prices in active markets for identical assets or liabilities.
Level 2—Observable inputs (other than Level 1 quoted prices), such as quoted prices in active markets for similar assets or liabilities, quoted prices in markets that are not active for identical or similar assets or liabilities, or other inputs that are observable or can be corroborated by observable market data.
Level 3—Unobservable inputs that are supported by little or no market activity that are significant to determining the fair value of the assets or liabilities, including pricing models, discounted cash flow methodologies and similar techniques.
To the extent that the valuation is based on models or inputs that are less observable or unobservable in the market, the determination of fair value requires more judgment. Accordingly, the degree of judgment exercised by the Company in determining fair value is greatest for instruments categorized in Level 3. A financial instrument’s level within the fair value hierarchy is based on the lowest level of any input that is significant to the fair value measurement.
Cloud Computing Arrangements
The Company capitalizes implementation costs for cloud computing arrangement service contracts. The Company’s cloud computing arrangements relate to its enterprise resource planning and manufacturing software. For such cloud computing service contracts, the Company capitalizes certain implementation costs as prepaid expenses in the consolidated balance sheets. The Company amortizes these capitalized cloud computing implementation costs into general and administrative expenses using the straight-line method over the fixed, non-cancellable term of the associated hosting arrangement, plus any reasonably certain renewal periods.
Property and Equipment, Net
Property and equipment, net is recorded at cost less accumulated depreciation. Depreciation expense is recorded using the straight-line method over the estimated useful life of the related asset, which are as follows:
| Estimated Useful Life | |
| Computer equipment | 3 years |
|---|---|
| Manufacturing equipment | 5 years |
| Furniture and equipment | 5 years |
| Laboratory equipment | 5 years |
| Leasehold improvements | Shorter of remaining lease term or useful life |
Purchased assets that are not yet in service are recorded to construction-in-process and no depreciation expense is recorded. Once they are placed in service, they are reclassified to the appropriate asset class. When assets are retired or otherwise disposed of, the assets and related accumulated depreciation are eliminated from the accounts and any resulting gain or loss is reflected in the Company’s consolidated statements of operation and comprehensive loss. Expenditures for maintenance and repairs are expensed as incurred.
Impairment of Long-Lived Assets
Long-lived assets consist of property, equipment and right-of-use assets. The Company continually evaluates whether events or circumstances have occurred that indicate that the estimated remaining useful life of its long-lived assets may warrant revision or that the carrying value of these assets may not be recoverable. If circumstances require that a long-lived asset or asset group be tested for impairment, the Company first compares the estimated undiscounted future cash flows expected to result from the use or disposition of that asset or asset group to its carrying amount. If the carrying amount of the long-lived asset or asset group is not recoverable on an undiscounted cash flow basis, an impairment loss would be recognized to the extent the carrying value exceeds its fair value. Fair value is determined through various valuation techniques including discounted cash flow models, quoted market prices and third-party independent appraisals, as considered necessary. No impairments occurred in the years ending December 31, 2024 and 2023.
Leases
At the inception of an arrangement, the Company determines whether the arrangement is or contains a lease. Leases with a term greater than one year are recognized on the consolidated balance sheet as a right-of-use (“ROU”) asset and current and non-current lease liabilities, as applicable. The Company has made an accounting policy election, known as the short-term lease recognition exemption, which allows the Company to not recognize ROU assets and lease liabilities that arise from short-term leases (12 months or less); The Company has applied this election to all classes of underlying assets. The Company typically only includes an initial lease term in its assessment of a lease arrangement. Options to renew or options to cancel a lease are not included in the Company’s assessment unless there is reasonable certainty that the Company will renew or will not cancel, respectively. The Company monitors its material leases on a quarterly basis.
Operating lease liabilities and their corresponding ROU assets are recorded based on the present value of future lease payments over the expected remaining lease term. Lease cost for operating leases is recognized on a straight-line basis over the lease term as an operating expense. Certain adjustments to the ROU asset may be required for items such as lease prepayments or incentives received. The interest rate implicit in lease contracts is typically not readily determinable. As a result, the Company utilizes its incremental borrowing rate, which reflects the fixed rate at which the Company could borrow on a collateralized basis the amount of the lease payments in the same currency, for a similar term, in a similar economic environment.
The Company has elected to account for the lease and non-lease components together for office, laboratory, and manufacturing real estate leases.
Research and Development
Research and development expenses include costs directly attributable to the conduct of the Company’s research and development programs.
Expenditures relating to research and development are expensed in the period incurred. Nonrefundable advance payments for goods and services that will be used in future research and development activities are expensed when the activity has been performed or when the goods have been received rather than when the payment is made. The cost of materials for a research and development activity that have an alternative future use is capitalized when the materials are acquired and recognized as expense as consumed. The costs of materials that were acquired for a particular research and development activity and have no alternative future use are expensed in the period acquired.
Costs incurred in obtaining licenses are recognized as research and development expense as incurred if the license has no alternative use.
Accrued Research and Development Expenses
The Company has entered into various research and development related contracts, including contracts with third-party contract research organizations and contract manufacturing organizations. These agreements are cancelable, and related costs are recognized as research and development expenses as incurred. The Company records accrued liabilities for estimated ongoing research costs. When evaluating the adequacy of the accrued liabilities, the Company analyzes the progress of the studies, including the phase or completion of events, invoices received and contracted costs. Significant judgments and estimates may be required to determine the accrued balances at the end of any reporting period. Actual results could differ from the Company’s estimates. To date, the Company’s historical accrual estimates have not been materially different from the actual costs.
Classification of Warrants
The Company accounts for warrants to purchase its common stock as either equity-classified or liability-classified instruments based on an assessment of the warrant’s specific terms and applicable authoritative guidance in ASC Topic 480, Distinguishing Liabilities from Equity("ASC 480") and ASC Topic 815, Derivatives and Hedging(“ASC 815”). The assessment considers whether warrants are freestanding financial instruments pursuant to ASC 480, meet the liability classification requirements pursuant to ASC 480, and whether the warrants meet all of the requirements for equity classification under ASC 815, including whether the warrants are indexed to the Company’s own stock, among other conditions for equity classification. This assessment, which requires the use of professional judgment, is conducted at the time of warrant issuance and as of each subsequent period end date while the warrants are outstanding.
Stock-Based Compensation Expense
The Company accounts for stock-based compensation under the provisions of ASC 718-10, Compensation—Stock Compensation (“ASC 718-10”), which requires all stock-based payments to employees, non-employees and directors, including grants of stock options and restricted stock, to be recognized in the consolidated statements of operations and comprehensive loss based on their fair values on the date of grant over the requisite service period, which is generally the vesting period of the respective award. Forfeitures are accounted for as they occur. Generally, the Company issues awards with only service-based vesting conditions and records the expense for these awards using the ratable method. The Company classifies stock-based compensation expense in the same manner in which the award recipient’s payroll or service provider’s costs are classified. Stock-based payments that contain performance conditions are recognized when such conditions are probable of being achieved.
The fair value of each restricted common stock award and each restricted stock unit award is estimated on the date of grant based on the fair value of the Company’s common stock on that same date.
The fair value of each option grant is estimated on the date of grant using the Black-Scholes option pricing model which requires inputs based on certain subjective assumptions, including the following:
Fair Value of Common Stock— See the discussion below.
Expected Term—The expected term represents the period that the stock-based awards are expected to be outstanding. The Company uses the simplified method to determine the expected term, which is based on the average of the time-to-vesting and the contractual life of the options.
Expected Volatility—Because the Company does not have sufficient trading history for its common stock as of December 31, 2024, the expected volatility was estimated based on the average volatility for comparable publicly traded biotechnology companies over a period equal to the expected term of the stock option grants. The comparable companies were chosen based on the similar size, stage in life cycle or area of specialty. The Company will continue to apply this process until a sufficient amount of historical information regarding the volatility of its own stock price becomes available.
Risk-Free Interest Rate—The risk-free interest rate is based on the U.S. Treasury zero coupon issues in effect at the time of grant for periods corresponding with the expected term of the awards.
Dividend Yield—The Company has never paid dividends on its common stock and has no plans to pay dividends on its common stock. Therefore, the Company used an expected dividend yield ofzero.
The Company began to use the closing common stock price as reported on the Nasdaq Global Select Market exchange as the fair value of common stock on the date of a grant subsequent to the Company's initial public offering of its common stock (“IPO”). Prior to the Company's IPO the estimated fair value of common stock was determined by the Company’s board of directors as of the date of each option grant, with input from management, considering third-party valuations of its common stock as well as the Company’s board of directors’ assessment of additional objective and subjective factors that it believed were relevant and which may have changed from the date of the most recent third-party valuation through the date of the grant. These objective and subjective factors include: (i) prices paid for the Company’s redeemable convertible preferred stock, and the rights, preferences, and privileges of the Company’s redeemable convertible preferred stock and common stock; (ii) the Company’s stage of development; (iii) the fact that the grants of stock-based awards related to illiquid securities in a private company; and (iv) the likelihood of achieving a liquidity event for the common stock underlying the stock-based awards, such as an initial public offering or sale of the Company, given prevailing market conditions. These third-party valuations were performed in accordance with the guidance outlined in the American Institute of Certified Public Accountants’ Accounting and Valuation Guide, Valuation of Privately Held Company Equity Securities Issued as Compensation. Each valuation methodology includes estimates and assumptions that require the Company’s judgment. The methodology utilized to estimate the fair value of the Company’s common stock was the option-pricing method (“OPM”) to back-solve the estimated value of the Company’s equity and corresponding value of the Company’s common stock.
Income Taxes
The Company accounts for income taxes using the asset and liability approach. Deferred tax assets and liabilities represent future tax consequences of temporary differences between the financial statement carrying amounts and the tax basis of assets and liabilities and for loss carryforwards using enacted tax rates expected to be in effect in the years in which the differences reverse. A valuation allowance is established to reduce deferred tax assets to the amounts expected to be realized. The Company also recognizes a tax benefit from uncertain tax positions only if it is “more likely than not” that the position is sustainable based on its technical merits. The Company accounts for interest or penalties related to uncertain tax positions as part of its provision for income taxes. To date, the Company hasnot incurred interest and penalties related to uncertain tax positions. Should such costs be incurred, they would be classified as a component of provision for income taxes.
Net Loss Per Share
Basic net loss per share is computed by dividing net loss by the weighted-average number of common shares outstanding during the reporting period, without consideration for potentially dilutive securities. Diluted net loss per share is computed by dividing net loss by the weighted-average number of common shares and potentially dilutive securities outstanding during the periods. For purposes of the diluted net loss per share calculation, restricted stock, warrants, restricted stock units and stock options considered to be potentially dilutive securities were excluded from the calculation of diluted net loss per share because their effect would be anti-dilutive and therefore, basic and diluted net loss per share were the same for all reporting periods presented.
Recent Accounting Pronouncements
From time to time, new accounting pronouncements are issued and adopted by the Company as of the specified effective date. Unless otherwise discussed, the impact of recently issued standards that are not yet effective will not have a material impact on the Company’s consolidated financial statements upon adoption. Under the Jumpstart Our Business Startups Act of 2012, as amended (the “JOBS Act”), the Company meets the definition of an emerging growth company and has elected to take advantage of the extended transition period for complying with certain new or revised accounting standards pursuant to Section 107(b) of the JOBS Act.
In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures (“ASU 2023-07”). ASU 2023-07 updates reportable segment disclosure requirements, primarily through enhanced disclosures about significant segment expenses and information used to assess segment performance. The Company adopted this ASU for the annual period ended December 31, 2024 and the amendments have been applied retrospectively toall prior periods presented in the financial statements by expanding the disclosure of expenses included in its segment measure of profitability. Refer to Note 15 for more information.
Recently Issued Accounting Pronouncements Not Yet Adopted
In October 2023, the FASB issued ASU 2023-06, Disclosure Improvements: Codification Amendments in Response to the SEC’s Disclosure Update and Simplification Initiative (“ASU 2023-06”), which modifies certain disclosure and presentation requirements of a variety of Topics in the Codification and is intended to both clarify or improve such requirements and align the requirements with the SEC’s regulations. The effective date for each amendment is the effective date of the removal of the related disclosure from Regulation S-X or Regulation S-K, with early adoption prohibited. The Company will apply the provisions prospectively as such provisions become effective, and does not expect ASU 2023-06 to have a material impact on the consolidated financial statements.
In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures (“ASU 2023-09”). ASU 2023-09 requires more detailed income tax disclosures, requiring entities to disclose disaggregated information about their effective tax rate reconciliation as well as expanded information on income taxes paid by jurisdiction. The disclosure requirements will be applied on a prospective basis, with the option to apply them retrospectively. This update is effective beginning with the Company’s 2025 fiscal year annual reporting period, with early adoption permitted. The Company is currently evaluating the impact that the adoption of this standard will have on its consolidated financial statements and disclosures.
In November 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses ("ASU 2024-03"). ASU 2024-03 improves disclosures surrounding a public business entity’s expenses by requiring more detailed information about the types of expenses included within commonly presented income statement captions. The update is effective beginning with the Company's 2027 fiscal year annual reporting period, with early adoption permitted. The Company is currently evaluating the impact that the adoption of this standard will have on its consolidated financial statements and disclosures.
- Marketable Securities
The amortized cost and estimated fair value of marketable securities, by contractual maturity are as follows:
| (in thousands) | Amortized Cost | Gross Unrealized Holding Gains | Gross Unrealized Holding Losses | Fair Value |
|---|---|---|---|---|
| Maturing in one year or less | ||||
| U.S. Treasuries | $4,981 | $12 | $— | $4,993 |
| Maturing after one year through five years | ||||
| U.S. Treasuries | 4,974 | 10 | — | 4,984 |
| Total | $9,955 | $22 | $— | $9,977 |
| (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 December 31, 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:
| Less than twelve months · Greater than twelve months · Total | ||||||
|---|---|---|---|---|---|---|
| (in thousands) | Fair value | Unrealized gain (loss) | Fair value | Unrealized gain (loss) | Fair value | Unrealized loss |
| U.S. Treasuries | $— | $— | $53,447 | $(95) | $53,447 | $(95) |
| Total | $— | $— | $53,447 | $(95) | $53,447 | $(95) |
The Company didnot record any impairments to marketable securities or reserves for credit losses related to its marketable debt securities during the periods ended December 31, 2024 and December 31, 2023.
- Fair Value Measurements
The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis:
| (in thousands) | Level 1 | Level 2 | Level 3 | Total |
|---|---|---|---|---|
| Cash equivalents | ||||
| Money market funds | $81,718 | $— | $— | $81,718 |
| Marketable securities | ||||
| U.S. Treasuries | — | 9,977 | — | 9,977 |
| Total marketable securities | — | 9,977 | — | 9,977 |
| Restricted cash equivalents | ||||
| Money market funds | 2,413 | — | — | 2,413 |
| Total | $84,131 | $9,977 | $— | $94,108 |
| (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 withno valuation adjustment. The fair value of marketable securities was determined based on observable market inputs. During the years ended December 31, 2024 and 2023, there wereno transfers between levels.
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.
- Property and Equipment, Net
Property and equipment, net consisted of the following:
| December 31, | December 31, | |
|---|---|---|
| (in thousands) | 2024 | 2023 |
| Laboratory equipment | $9,625 | $10,028 |
| Manufacturing equipment | 7,082 | 6,936 |
| Computer equipment | 446 | 432 |
| Furniture, fixtures and other | 606 | 599 |
| Construction in progress | 36 | 146 |
| Total | 17,795 | 18,141 |
| Less: Accumulated depreciation | (11,214) | (8,091) |
| Property and equipment, net | $6,581 | $10,050 |
Depreciation expense for each of the years ended December 31, 2024 and 2023 was approximately$3.5 million.
- Accrued Expenses and Other Current Liabilities
Accrued expenses and other current liabilities consisted of the following:
| December 31, | December 31, | |
|---|---|---|
| (in thousands) | 2024 | 2023 |
| Employee-related expenses | $5,852 | $5,962 |
| Professional fees | 1,461 | 1,245 |
| Clinical expenses | 3,835 | 1,495 |
| Manufacturing expenses | 516 | 842 |
| Research and development expenses | 872 | 1,059 |
| Other | 356 | 274 |
| Total accrued liabilities | $12,892 | $10,877 |
- Stockholders' Equity
Common Stock
As of December 31, 2024 and 2023, the Company’s authorized capital stock included 400,000,000 shares of its$0.0001 par value common stock.
Each share of common stock entitles the holder toone vote on all matters submitted to a vote of the Company’s stockholders provided, however, that, except as otherwise required by law, holders of common stock shall not be entitled to vote on any amendment to the Company’s Certificate of Incorporation, as amended (the “Certificate of Incorporation”), that relates solely to the terms of one or more outstanding series of preferred stock if the holders of such affected series are entitled, either separately or together with the holders of one or more other such series, to vote thereon pursuant to the Certificate of Incorporation or pursuant to the Delaware General Corporation Law. Common stockholders are entitled to receive dividends, as may be declared by the Company’s board of directors, if any, subject to the preferential dividend rights of the preferred stock. No dividends have been declared or paid as of and for either of the years ended December 31, 2024 and 2023.
At-the-Market Sales
During the years ended December 31, 2024 and, 2023, the Company sold 249,096 and 1,016,662 shares of common stock, respectively, in at-the-market offerings at a weighted average price per share of $1.64 and $4.75, respectively, for aggregate net proceeds of $0.4 million and $4.7 million respectively after deducting commissions.
Private Placement
On December 27, 2024, the Company entered into a purchase agreement with certain institutional investors (collectively, the "Purchasers"), pursuant to which the Company issued and sold to the Purchasers in a private placement an aggregate of (i)55,871,260 shares of the Company's common stock and (ii) warrants to purchase up to 69,839,075 shares of the Company's common stock (the "Warrants") at the closing of the private placement on December 30, 2024. Net proceeds from the Private Placement were $52.7 million, after deducting placement fees and issuance costs payable by the Company.
The Warrants have an exercise price of $0.838 per share and are immediately exercisable, subject to certain limitations on exercise set forth in the Warrants. The Warrants will terminate inseven years on December 30, 2031.
The Company also entered into a registration rights agreement and filed with the Securities and Exchange Commission a registration statement to register for resale the shares of the common stock issued in the private placement and those issuable upon the exercise of the Warrants. The registration rights agreement became effective on January 29, 2025.
The Company determined that the Warrants are freestanding instruments that do not meet the definition of a liability or derivative. The Warrants are indexed to the Company’s common stock and meet all other conditions for equity classification. Accordingly, the Warrants are classified as equity and accounted for as a component of additional paid-in capital at the time issued. The Company also determined that the Warrants should be included in the determination of diluted net loss per share if their impact is dilutive. However, they are not included within diluted net loss per share for the year ended December 31, 2024 as the effect would be antidilutive.
- Stock-Based Compensation
2023 Inducement Plan
On August 25, 2023, the Company’s board of directors adopted the Company’s 2023 Inducement Plan (the “2023 Inducement Plan”) pursuant to which the Company reserved 3,500,000 shares of common stock for issuance under the 2023 Inducement Plan. The 2023 Inducement Plan provides for the grant of non-statutory stock options, restricted stock awards, restricted stock unit awards, stock appreciation rights, performance awards and other forms of stock-based compensation to eligible individuals. In accordance with Nasdaq Marketplace Rule 5635(c)(4), awards under the 2023 Inducement Plan may only be made to individuals not previously employees or directors of the Company (or following such individuals’ bona fide period of non-employment with the Company), as an inducement material to the individuals’ entry into employment with the Company. Awards granted under the 2023 Inducement Plan must be approved by either a majority of the Company’s independent directors or the compensation committee of the Company’s board of directors. As of December 31, 2024, the Company had 2,645,757 shares of its common stock available for future issuance under the 2023 Inducement Plan.
Stock Incentive Plans
In December 2015, the Company’s board of directors adopted and approved the 2015 Stock Incentive Plan (as amended to date, the “2015 Plan”). The 2015 Plan provided for the granting of incentive stock options, non-statutory stock options, restricted stock awards and other stock-based awards to eligible employees, officers, directors, consultants and advisors as determined by the Company’s board of directors.
In February 2021, the Company’s board of directors adopted and stockholders approved the 2021 Equity Incentive Plan (the “2021 Plan”). The 2021 Plan became effective on February 5, 2021, following whichno further grants were or will be made under the 2015 Plan. In March 2024, the Company’s board of directors adopted an amendment and restatement of the 2021 Plan, which the Company’s stockholders approved in May 2024. The 2021 Plan provides for the grant of stock options, non-statutory stock options, restricted stock awards, restricted stock unit awards, stock appreciation rights, performance stock awards and other forms of stock compensation to the Company's employees, consultants and directors.
The number of shares of the Company's common stock reserved for issuance under the 2021 Plan will automatically increase on January 1 of each year through January 1, 2034, by 5.0% of the total number of shares of common stock outstanding on December 31 of the preceding calendar year. Any grants that expire or are canceled, terminated, forfeited, fail to vest, or are withheldto satisfy a tax withholding obligation are allowed to be reissued under 2021 Plan. As of December 31, 2024, the Company had 3,017,555 shares of its common stock available for future issuance under the 2021 Plan.
Option Repricing
On February 3, 2025, the Company's board of directors approved a stock option repricing whereby the exercise price of certain outstanding options to purchase shares of the Company’s common stock was reduced to $1.34 per share. The repricing applied to options to purchase shares of the Company’s common stock held by continuing employees as of February 3, 2025 that had an exercise price per share greater than $1.34; provided that holders of repriced options must remain in continuous service with the Company through February 3, 2026 or, if earlier, a change in control of the Company or 30 days prior to the applicable repriced option’s original expiration date (the “Retention Period”). If any such repriced option is exercised prior to the end of the Retention Period, the exercise price per share shall be the original exercise price per share, and not the reduced exercise price. The total number of shares underlying all repriced options is approximately 6.76 million shares. The repriced options previously had exercise prices ranging from $1.36 to $44.96 per share. The repricing had no impact on the financial results for the period ended December 31, 2024.
Stock Options
The Company’s stock options generally vest over 48 months with 25% vesting afterone year followed by ratable monthly vesting over 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:
| Year Ended December 31, | ||
|---|---|---|
| 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 | — | — |
The following table summarizes the Company’s stock option activity for the year ended December 31, 2024:
| Shares | Weighted-AverageExercise Price | Weighted-AverageRemainingContractual Term(in years) | AggregateIntrinsicValue(in thousands) | |
|---|---|---|---|---|
| Outstanding at December 31, 2023 | 8,290,077 | $6.17 | 7.71 | $999 |
| Granted | 2,192,350 | 1.96 | ||
| Vested and exercised | (10,299) | 4.90 | ||
| Forfeited | (613,876) | 5.26 | ||
| Expired | (713,103) | 6.16 | ||
| Outstanding at December 31, 2024, | 9,145,149 | 5.22 | 6.99 | $211 |
| Exercisable at December 31, 2024 | 5,908,767 | $6.09 | 6.13 | $33 |
The aggregate intrinsic value of options is calculated as the difference between the exercise price of the stock options and the fair value of the Company’s common stock for those stock options that had exercise prices lower than the fair value of the common stock as of the respective date.
The weighted-average grant-date fair value of stock options granted during the years ended December 31, 2024 and 2023 was$1.48 and $3.56 per share, respectively. As of December 31, 2024, total unrecognized compensation expense related to stock options was$8.0 million which is expected to be recognized over a weighted-average period of 2.6 years. The wereno stock options exercised during the year ended December 31, 2024.
As of December 31, 2024, there wereno exercised and unvested shares. As of December 31, 2023, options for 10,299 shares with a weighted average exercise price of$4.90 were exercised and unvested. As of December 31, 2023, the underlying proceeds from the unvested exercises of less than$0.1 million were recorded in other current liabilities on the consolidated balance sheet.
Restricted Stock Units
As of December 31, 2024, there were 1,308,723 restricted stock units outstanding under the 2021 Plan and the 2023 Inducement Plan. The following table summarizes the Company's unvested restricted stock unit activity for the year ended December 31, 2024:
| Shares | Weighted-Average Grant Date Fair Value | |
|---|---|---|
| Unvested at December 31, 2023 | 1,113,381 | $4.94 |
| Granted | 1,223,592 | 2.24 |
| Vested | (752,558) | 4.49 |
| Forfeited | (275,692) | 3.94 |
| Unvested at December 31, 2024 | 1,308,723 | $2.89 |
As of December 31, 2024, total unrecognized compensation expense related to the unvested restricted stock units was$3.5 million, which is expected to be recognized over a weighted average period of 2.6 years.
2021 Employee Stock Purchase Plan
In February 2021, the Company’s board of directors adopted and stockholders approved the 2021 Employee Stock Purchase Plan (the “ESPP”). The ESPP became effective on February 5, 2021. The number of shares of common stock reserved for issuance under the ESPP will automatically increase on January 1 of each year through January 1, 2031, by the lesser of (i)1% of the total number of shares of our common stock outstanding on December 31 of the preceding calendar year, and (ii)1,800,000 shares. If purchase rights granted under the ESPP terminate without having been exercised, the shares of common stock not purchased under such purchase rights will again become available for issuance under the ESPP. As of December 31, 2024, the Company had 1,740,776 shares of its common stock available for future issuance under the ESPP.
The ESPP permits eligible employees to purchase common stock through accumulated payroll deductions at a purchase price equal to 85% of the lesser of the market value of the common stock at the beginning of the 6-month offering period or on the purchase date. During the years ended December 31, 2024 and 2023, the Company issued 227,844 and 82,703 shares, respectively, with a weighted average purchase price per share of$0.98 and $2.73, respectively, which resulted in an immaterial amount of compensation expense.
Stock-Based Compensation
Stock-based compensation expense was allocated as follows:
| Year Ended December 31, | ||
|---|---|---|
| (in thousands) | 2024 | 2023 |
| Research and development | $4,728 | $6,831 |
| General and administrative | 5,119 | 6,531 |
| Total stock-based compensation expense | $9,847 | $13,362 |
- Leases
Cambridgepark Lease
In December 2019, the Company entered into a lease agreement for its new corporate office and laboratory facility (the “Cambridgepark Lease”) in Cambridge, Massachusetts. The Cambridgepark Lease has a term of 10 years, beginning on the rent commencement date which is two months after the lease commencement date. There areno options to extend the lease. The lease commencement date, for accounting purposes, was deemed to be reached as of June 30, 2020.
On June 15, 2021, the Company entered into the first lease amendment (“First Lease Amendment”) and the 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 Cambridgepark Lease with the Landlord in Cambridge, Massachusetts to add additional leased space in the same building (the “Amended Cambridgepark Lease”).
The First Lease Amendment expanded the amount of space leased by the Company by an additional 10,262 square feet in exchange for aggregate total fixed rent payments of approximately $8.4 million with the annual fixed rental payments escalating from $0.8 million to $1.1 million during the term. The First Lease Amendment commenced for accounting purposes on January 28, 2022.
The Second Lease Amendment expands the amount of space leased by the Company by an additional 30,175 square feet in exchange for aggregate total fixed rent payments of approximately $22.3 million with the annual fixed rental payments escalating from $1.1 million to $3.0 million during the term. The Second Lease Amendment’s term commenced for accounting purposes on April 29, 2022.
Payments associated with the Amended Cambridgepark Lease 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. The Amended Cambridgepark Lease does not contain a residual value guarantee. The Lease Amendments term end dates are coterminous with the Cambridgepark
Lease. In conjunction with the Amended Cambridgepark Lease, the Company was required to establish a$2.4 million irrevocable standby letter of credit for the benefit of the Landlord, which has been secured by money market investments and is presented as restricted cash equivalents.
The elements of lease expense were as follows:
| Year Ended December 31, | ||
|---|---|---|
| (in thousands) | 2024 | 2023 |
| Operating lease cost | $7,804 | $7,802 |
| Variable lease cost | 2,653 | 2,618 |
| Total lease cost | $10,457 | $10,420 |
Amounts reported in the consolidated balance sheets and the weight-average lease term and discount rate information were as follows:
| (in thousands except weighted-average amounts) | December 31, 2024 | December 31, 2023 |
|---|---|---|
| Assets | ||
| Operating lease right-of-use assets | $35,007 | $40,048 |
| Liabilities | ||
| Operating lease liabilities, current | $4,215 | $3,830 |
| Operating lease liabilities, non-current | 27,615 | 31,830 |
| Total lease liabilities | $31,830 | $35,660 |
| Weighted-Average Lease Term and Discount Rate | ||
| Weighted-average remaining lease term (years) | 5.7 | 6.7 |
| Weighted-average discount rate | 8.2% | 8.2% |
The following table represents other lease activity:
| Year Ended December 31, | ||
|---|---|---|
| (in thousands) | 2024 | 2023 |
| Other Information | ||
| Cash paid for amounts included in the measurement of lease liabilities | ||
| Operating cash flows for operating leases | $6,591 | $6,658 |
Future lease payments for noncancelable leases as of December 31, 2024 were as follows:
| (in thousands) | December 31, 2024 |
|---|---|
| 2025 | $6,652 |
| 2026 | 6,681 |
| 2027 | 6,882 |
| 2028 | 7,088 |
| 2029 | 7,301 |
| Thereafter | 5,602 |
| Total lease payments | $40,206 |
| Less: imputed interest | (8,376) |
| Present value of lease liabilities | $31,830 |
- Significant Agreements
The Company has agreements with third parties in the normal course of business under which it has obtained licenses for certain developed technologies. Such arrangements may require ongoing payments including annual fees and payments upon the achievement of various development, clinical, regulatory, and commercial milestones. In addition, if any products related to these agreements are approved for sale, the Company may be required to pay significant milestones upon approval and milestones and/or royalties on future sales. The payment of these amounts, however, is contingent upon the occurrence of various future events, which have a high degree of uncertainty of occurrence.
- Commitments and Contingencies
Legal Proceedings
The Company is not currently a party to any material legal proceedings. At each reporting date, the Company evaluates whether or not a potential loss amount or potential range of loss is probable and reasonably estimated under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company recognizes expenses for its costs related to its legal proceedings, as incurred.
- Defined Contribution Benefit Plan
The Company maintains a defined contribution plan under Section 401(k) (the “401(k) Plan”) of the Internal Revenue Code, as amended (the “Code”). The 401(k) Plan covers all employees who meet defined minimum age and service requirements and allows participants to defer a portion of their annual compensation on a pretax basis, as well as Roth post tax deferrals. The Company matches 100% of compensation amounts deferred up to the first 1% of an employee's compensation plus 50% of compensation amounts deferred between 1% and 6% of an employee's compensation. All matching contributions are immediately vested. Expense recognized by the Company for matching contributions made in accordance with the 401(k) Plan was$1.2 million and$1.0 million for the years ended December 31, 2024 and December 31, 2023, respectively.
- Income Taxes
The Company accounts for income taxes under FASB ASC 740 (“ASC 740”). For the years ended December 31, 2024 and 2023, the Company did not record a current or deferred income tax expense or benefit. The following table reconciles the federal statutory income rate to the Company’s effective income tax rate:
| Year Ended December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Federal income tax rate | 21.0 % | 21.0 % |
| State income tax benefit | 5.6 % | 6.1 % |
| Permanent items | (1.6) % | (0.9) % |
| Research tax credits | 4.8 % | 5.7 % |
| Other | — | (0.4) % |
| Valuation allowance | (29.8) % | (31.5) % |
| Effective income tax rate | — | — |
Deferred tax assets and liabilities reflect the net tax effects of net operating loss and tax credit carryforwards and temporary differences between the carrying amount of assets and liabilities for financial reporting and the amounts used for tax purposes.
Significant components of the Company’s deferred tax assets and liabilities were as follows:
| December 31, | ||
|---|---|---|
| (in thousands) | 2024 | 2023 |
| Deferred tax assets: | ||
| Accrued expenses | $1,651 | $1,428 |
| Federal net operating loss carryforwards | 51,217 | 39,934 |
| State net operating loss carryforwards | 14,828 | 11,423 |
| Tax credits | 22,725 | 17,062 |
| Stock compensation | 1,733 | 1,545 |
| R&D capitalization | 46,973 | 33,503 |
| Amortization | 1,582 | 1,464 |
| Lease liability | 8,634 | 9,746 |
| Total deferred tax assets | 149,343 | 116,105 |
| Valuation allowance | (139,216) | (104,403) |
| Net total deferred tax assets | $10,127 | $11,702 |
| Deferred tax liabilities: | ||
| Lease right of use asset | (9,495) | (10,945) |
| Depreciation and amortization | (632) | (757) |
| Total deferred tax liabilities | $(10,127) | $(11,702) |
| Net deferred tax assets | $— | $— |
The Company has weighed the positive and negative evidence to assess the recoverability of its deferred tax assets. Realization of future tax benefits is dependent on many factors, including the Company’s ability to generate taxable income. After this assessment, the Company determined it was more likely than not that the Company will not realize the benefit of its deferred tax assets. As a result, the Company has provided a full valuation allowance against its net deferred tax assets. The valuation allowance for deferred tax assets as of December 31, 2024 and 2023 was$139.2 million and$104.4 million, respectively. For the years ended December 31, 2024 and 2023, the Company recorded an increase in the valuation allowance of$34.8 million and $36.1 million, respectively, primarily related to net operating losses incurred by the Company.
As of December 31, 2024, the Company had gross U.S. federal net operating loss carryforwards of$243.9 million including$242.0 million that had an indefinite carryforward period and$1.9 million that were subject to expiration at various dates through 2037. As of December 31, 2024, the Company had state net operating loss carryforwards of$234.7 million which will expire at various dates through 2044. As of December 31, 2024, the Company had U.S. federal research and development tax credit carryforwards of$16.8 million which will expire at various dates through 2044 and state research and credit carryforwards of$7.5 million which will expire at various dates through 2039. The net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service and state tax authorities.
Net operating loss and tax credit carryforwards may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest of significant shareholders over athree-year period in excess of 50%, as defined under Sections 382 and 383 of the Code, respectively, as well as similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. The amount of the annual limitation is determined based on the value of the Company immediately prior to the ownership change. Subsequent ownership changes may further affect the limitation in future years. The Company has not determined whether an ownership change has occurred and as such, the Company’s net operating losses may be limited. Any limitation may result in expiration of a portion of the net operating loss carryforwards or research development credit carryforwards before utilization.
The Company has not, as yet, conducted a study of research and development credit carryforwards. Such a study, once undertaken by the Company, may result in an adjustment to the research and development credit carryforwards. However, a full valuation allowance has been provided against the Company’s research and development credits and, if any adjustment is required, such adjustment would be offset by an adjustment to the valuation allowance. Thus, there would be no impact to the balance sheet or statement of operations if any adjustment is required.
As of December 31, 2024 and 2023, the Company didnot have any unrecognized tax benefits. Any future interest and penalties related to income tax matters would be recognized in the provision for income tax. As of December 31, 2024 and 2023, the Company didnot have a balance of accrued interest and penalties related to uncertain tax positions.
The Company files income tax returns in the United States and various states. As of December 31, 2024, there were no income tax examinations in progress.
The tax years 2020 through present remain open to examination by major taxing jurisdictions to which the Company is subject, which are primarily in the United States. In addition, tax years prior to 2019 resulted in losses and the Company also generated research and development tax credits during those years. Since carryforward attributes generated in these years may be utilized in future years, years prior to 2019 may still be adjusted upon examination by the Internal Revenue Service or state tax authorities if they have or will be used in a future period.
- Net Loss Per Share
The following table sets forth the computation of the Company’s basic and diluted net loss per share for the years ended December 31, 2024 and 2023:
| Year Ended December 31, | ||
|---|---|---|
| (in thousands, except share and per share amounts) | 2024 | 2023 |
| Numerator: | ||
| Net loss | $(116,914) | $(117,863) |
| Denominator: | ||
| Weighted-average number of common shares outstanding, basic and diluted | 68,705,639 | 67,191,973 |
| Net loss per share, basic and diluted | $(1.70) | $(1.75) |
The Company’s potentially dilutive securities were stock options, unvested restricted stock, restricted stock units, and warrants. Based on the amounts outstanding at December 31, 2024 and 2023, the Company excluded the following potential common shares from the computation of diluted net loss per share because including them would have had an anti-dilutive effect:
| As of December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Options to purchase common stock | 9,145,149 | 8,290,077 |
| Unvested restricted stock | — | 10,299 |
| Restricted stock units | 1,308,723 | 1,113,381 |
| Warrants | 69,839,075 | — |
- Segments
The Company operates and manages its business asone reportable and operating segment, centered around the commercial development of its product candidates. The Company’s chief operating decision maker (“CODM”) is the Chief Executive Officer (“CEO”).
The Company’s CODM reviews consolidated operating results, manages the business on a consolidated basis and utilizes consolidated net loss from the consolidated statements of operations and comprehensive loss to make decisions about allocating resources and assessing performance for the entire Company. Consolidated net loss is also used to monitor budget to actual results. The CODM is additionally regularly provided with more detailed expense information at the program level.
The following table is a summary of the segment profit or loss, including significant segment expenses (in thousands):
| Year ended December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Segment expenses: | ||
| Trem-cel(a) | $18,905 | $13,477 |
| VCAR33(a) | 8,930 | 8,434 |
| Other research and development(a) | 12,608 | 24,427 |
| Salaries and benefits | 44,165 | 38,193 |
| General corporate activities | 19,856 | 23,123 |
| Other segment items(b) | 12,450 | 10,209 |
| Segment expenses: | 116,914 | 117,863 |
| Segment net loss | (116,914) | (117,863) |
(a) Includes only external research and development expenditures.
(b) Other segment items are primarily comprised of taxes, interest income on marketable securities and certain non-cash expenses such as stock-based compensation, depreciation expense, and non-cash lease expense.
The measure of segment assets is reported on the consolidated balance sheet as total assets. The CODM additionally reviews cash, cash equivalents and marketable securities when reviewing segment assets. As of December 31, 2024, the Company’s cash, cash equivalents and marketable securities were$91.9 million. The Company does not provide its CODM with any more detailed segment asset information than what is included on the Company’s consolidated balance sheets.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.