PepGen Inc
For the period ended Dec 31, 2024. Filed Feb 24, 2025.
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FY2024 · Filed Feb 24, 2025
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Notes to Consolidated Financial Statements
- Nature of Business and Basis of Presentation
PepGen Inc., hereinafter referred to as PepGen or the Company, is a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. The Company's principal offices are located in Boston, Massachusetts.
The Company was initially formed as PepGen Limited on January 25, 2018, in the United Kingdom, or the U.K. On November 9, 2020, PepGen Limited completed a corporate reorganization, or the Reorganization. As part of the Reorganization, PepGen Limited formed PepGen, a Delaware corporation with nominal assets and liabilities, for the purpose of consummating the Reorganization. In connection with the Reorganization, the existing stockholders of PepGen Limited exchanged each of its classes of shares of PepGen Limited for the same number and class of common stock of PepGen on a one-to-one basis. The newly issued stock of PepGen had substantially identical rights to the exchanged shares of PepGen Limited. As a result of the exchange, PepGen became the sole stockholder of PepGen Limited. Upon the completion of the Reorganization on November 23, 2020, the historical financial statements of PepGen Limited became the historical financial statements of PepGen as the Reorganization was deemed to be between entities under common control. PepGen Limited was dissolved on December 10, 2024.
Liquidity and Going Concern
Since inception, the Company has not generated any revenue from product sales or other sources and has incurred significant operating losses and negative cash flows from operations. The Company’s primary uses of cash, cash equivalents, and marketable securities to date have been funding research and development activities, business planning, establishing and maintaining the Company’s intellectual property portfolio, hiring personnel, leasing premises and associated capital expenditures, raising capital, and providing general and administrative support for these operations. As of December 31, 2024, the Company had an accumulated deficit of $271.5 million. To date, the Company has funded operations primarily through private placements of convertible preferred stock, the sale of shares of common stock in its initial public offering, or IPO, the sale of shares of common stock under its At-the-Market Equity Offering Sales Agreement, or Sales Agreement, with Stifel, Nicolaus & Company, Incorporated, or Stifel, and through the sale of shares of common stock in a follow-on public offering, referred to as the Follow-on Offering. As of December 31, 2024, the Company had cash, cash equivalents, and marketable securities of $120.2 million. The Company believes that its cash, cash equivalents, and marketable securities as of December 31, 2024, will be sufficient to fund its currently planned operations for at least the next 12 months from the issuance of these consolidated financial statements.
The Company filed a shelf registration statement on Form S-3 with the United States Securities and Exchange Commission, or SEC, which covers the offering, issuance and sale of an amount up to $300.0 million in the aggregate of shares of common stock, preferred stock, debt securities, warrants, and/or units or any combination thereof, which was declared effective on June 16, 2023. On August 8, 2023, the Company filed a prospectus supplement and entered into the Sales Agreement with Stifel, as sales agent, which provides for the issuance and sale by the Company of up to $100.0 million of shares of common stock from time to time in “at-the-market” offerings. The sales agent is entitled to receive a commission of up to 3.0% of gross proceeds from sales under the Sales Agreement. On February 5, 2024, the Company sold 1,000,000 shares of common stock at a price of $10.00 per share under the Sales Agreement, resulting in net proceeds of $9.9 million. On February 9, 2024, the Company sold 7,530,000 shares of common stock in the Follow-on Offering, pursuant to an underwriting agreement with Leerink Partners LLC dated as of February 6, 2024, at a price of $10.635 per share, resulting in net proceeds of $76.4 million after deducting underwriting fees and offering costs of $3.7 million. Net proceeds from sales under the Sales Agreement and Follow-on Offering, after deducting underwriters' fees and costs, were $86.3 million. On June 28, 2024, the Company filed a second shelf registration statement on Form S-3 with the SEC, which covers the offering, issuance and sale of an amount up to $250.0 million in the aggregate of shares of common stock, preferred stock, debt securities, warrants, and/or units or any combination thereof, which was declared effective on July 8, 2024.
As the Company continues to pursue its business plan to successfully develop and obtain regulatory approval for its product candidates, it expects to finance its operations through the sale of equity, debt financings or other capital resources, which could include income from collaborations, strategic partnerships or marketing, distribution, licensing or other strategic arrangements with third parties, or from grants. However, there can be no assurance that any additional financing or strategic transactions will be available to the Company on acceptable terms, if at all. If events or circumstances occur such that the Company does not obtain additional funding, it may need to delay, reduce or eliminate its product development or future commercialization efforts, which could have a material adverse effect on the Company’s business, results of operations or financial condition
Basis of Presentation and Consolidation
The accompanying consolidated financial statements have been prepared in accordance with accounting principles generally accepted in the United States, or U.S. GAAP. The consolidated financial statements include the accounts of PepGen Inc. (a U.S. corporation) and its wholly owned subsidiaries PepGen Limited (a U.K. corporation which was dissolved on December 10, 2024) and PepGen Securities Corp. (a U.S. corporation). All intercompany accounts and transactions have been eliminated in consolidation.
Reclassifications
Restricted cash on the consolidated balance sheet as of December 31, 2023, previously included in other assets, has been presented as a separate line item. The consolidated financial statements have otherwise been prepared on the same basis as the audited 2023 consolidated financial statements. The reclassification has no effect on net income, earnings per share, retained earnings, cash flows or total assets.
- Summary of Significant Accounting Policies
Use of Estimates
The preparation of consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the amounts reported in the consolidated financial statements and accompanying notes. Management bases its estimates and judgments on historical experience, knowledge of current conditions, and beliefs of what could occur in the future, given the available information. On an ongoing basis, management evaluates such estimates and assumptions for continued reasonableness. In particular, management makes estimates with respect to accruals for research and development activities, for the valuation of intellectual property, for the fair value of common stock and convertible preferred stock warrants and stock-based compensation expense. Appropriate adjustments, if any, to the estimates used are made prospectively based upon such periodic evaluation. Actual results could differ materially from those estimates and assumptions.
Segment Reporting
Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision-maker, or CODM. The Company’s CODM is its chief executive officer who reviews financial information together with certain operating metrics principally to make decisions about how to allocate resources and to measure the Company’s performance. The Company has determined that it operates as a single reportable segment. The Company’s CODM evaluates financial information on a consolidated basis.
Foreign Currency Remeasurement
The Company’s reporting currency is the U.S. Dollar. The functional currency of PepGen Limited is the British Pound. The assets and liabilities of PepGen Limited are translated into U.S. Dollars at the exchange rates in effect at each balance sheet date, and the results of operations are translated using the average exchange rates prevailing throughout the reporting period. Adjustments resulting from translating foreign functional currency financial statements into U.S. Dollars are included in the cumulative translation adjustment, a component of accumulated other comprehensive loss in stockholders’ equity.
Concentration of Credit Risk
Financial instruments, which potentially subject the Company to concentrations of credit risk, consist of cash and money market accounts. As of December 31, 2024, the Company’s cash, cash equivalents, and marketable securities were held by four financial institutions in the U.S. At times, the Company’s deposits held in the U.S. may exceed the respective insured limits of the Federal Depository Insurance Corporation and Financial Services Compensation Scheme.
Cash and Cash Equivalents
The Company considers all highly liquid investments with an original maturity of three months or less when purchased to be cash equivalents. Cash and cash equivalents are recorded at cost, which approximates fair value. As of December 31, 2024 and 2023, cash and cash equivalents consisted primarily of checking and money market funds composed of U.S. government obligations, and U.S. treasury notes.
Restricted Cash
The Company classifies all cash the use of which is limited by contractual provisions as restricted cash. Restricted cash arises from the requirement for the Company to maintain cash of $1.5 million as collateral under a lease agreement.
Marketable Securities
The Company’s marketable securities consist of U.S. treasury notes which are classified as available-for-sale and are reported at fair value. Unrealized gains and losses on available-for-sale debt securities are reported as a component of accumulated other comprehensive loss in stockholders’ equity. Realized gains and losses are based on the specific identification method and are included as a component of other income (expense), net in the consolidated statements of operations and comprehensive loss.
The Company evaluates its marketable securities with unrealized losses for other-than-temporary impairment. When assessing marketable securities for other-than-temporary declines in value, the Company considers such factors as, among other things, how significant the decline in value is as a percentage of the original cost, how long the market value of the investment has been less than its original cost, the Company’s ability and intent to retain the investment for a period of time sufficient to allow for any anticipated recovery in fair value, and market conditions in general. If any adjustment to fair value reflects a decline in the value of the investment that the Company considers to be “other-than-temporary,” the Company reduces the investment to fair value through a charge to the consolidated statement of operations and comprehensive loss. No such adjustments were necessary during the periods presented.
Fair Value Measurements
Certain assets and liabilities of the Company are carried at fair value under U.S. 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: Fair values are determined utilizing prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access.
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.
For certain financial instruments, including cash and cash equivalents, prepaid expenses, accounts payable, as well as certain accrued liabilities, the recorded amount approximates estimated fair value due to their relatively short maturity period.
Property and Equipment, Net
Property and equipment are stated at cost, net of accumulated depreciation. Depreciation of property and equipment is calculated using the straight-line method over the estimated useful lives of the respective assets. Maintenance and repairs that do not improve or extend the life of the assets are expensed when incurred.
The estimated useful lives of the Company’s property and equipment are as follows:
| Laboratory and computer equipment | 5 years |
|---|---|
| Furniture and fixtures | 3 years |
Deferred Offering Costs
The Company capitalizes within other long-term assets certain legal, accounting, and other third-party fees that are directly related to the Company’s in-process equity financings, until such financings are consummated. After consummation of the equity financing, these costs are recorded as a reduction of the proceeds received as a result of the offering. Should a planned equity financing be abandoned, terminated, or significantly delayed, the deferred offering costs are immediately written off to operating expenses. As of December 31, 2024, deferred offering costs of $0.4 million in relation to the Company's shelf registration statements on Form S-3 were recorded within other assets on the consolidated balance sheets.
Leases
Under ASU 2016-02, Leases (Topic 842), or ASC 842, at the inception of an arrangement, the Company determines whether the arrangement is or contains a lease based on the unique facts and circumstances presented in the arrangement, including whether the Company controls the use of identified assets. The Company classifies leases with a term greater than one year as either operating or finance leases at the lease commencement date and records a right-of-use asset and current and non-current lease liabilities, as applicable on the balance sheet. The Company has elected not to recognize on the balance sheet leases with terms of one year or less, but payments are recognized as expense on a straight-line basis over the lease term. If a lease includes options to extend the lease term, the Company does not assume the option will be exercised in its initial lease term assessment unless there is reasonable certainty that the Company will renew based on an assessment of economic factors present as of the lease commencement date. The Company monitors its plans to renew its material lease each reporting period. If a lease includes provisions for leasehold improvements for which the Company has an obligation to pay, the Company determines if the improvements should be considered lessor or lessee assets. If the improvements are considered lessor assets, the Company records the payments in the calculation of the lease liability and corresponding right-of-use asset.
Lease liabilities and the corresponding right-of-use assets are recorded based on the present value of lease payments over the remaining lease term. The present value of future lease payments are discounted using the interest rate implicit in lease contracts if that rate is readily determinable; otherwise, the Company utilizes information available at the commencement of the lease to calculate the incremental borrowing rate, or IBR, which reflects the fixed rate at which the Company could borrow on a collateralized basis over a similar term, the amount of the lease payments in a similar economic environment. In order to determine the appropriate IBR, the Company uses available third-party information, including comparable company collateralized borrowing information. After lease commencement and the establishment of a right-to-use asset and operating lease liability, lease expense is recorded on a straight-line basis over the lease term.
The Company enters into contracts that contain both lease and non-lease components. Non-lease components include costs that do not provide a right-to-use leased asset but instead provide a service, such as maintenance costs. The Company has elected to account for the lease and non-lease components together as a single component for all classes of underlying assets. Variable costs associated with the lease, such as maintenance and utilities, are not included in the measurement of right-to-use assets and lease liabilities but rather are expensed when the events determining the amount of variable consideration to be paid have occurred.
Impairment of Long-Lived Assets
The Company reviews long-lived assets for impairment whenever events or changes in circumstances indicate that the carrying amount of an asset or asset group may not be recoverable. The recoverability of assets to be held and used is measured by a comparison of the carrying amount of an asset or asset group to the estimated undiscounted future cash flows expected to be generated by the asset or asset group.
If the carrying amount of an asset or asset group exceeds its estimated undiscounted future cash flows, an impairment charge is recognized as the amount by which the carrying amount of the asset or asset group exceeds the estimated discounted future cash flows of the asset or asset group. There have beenno such impairments of long-lived assets for the years ended December 31, 2024 and 2023.
Commitment and Contingencies
The Company recognizes a liability with regard to loss contingencies when it believes it is probable a liability has been incurred, and the amount can be reasonably estimated. If some amount within a range of loss appears at the time to be a better estimate than any other amount within the range, the Company accrues that amount. When no amount within the range is a better estimate than any other amount, the Company accrues the minimum amount in the range. The Company has not recorded any such liabilities as of December 31, 2024 and 2023 that were material to the consolidated financial statements.
Research and Development
Research and development costs are expensed as incurred. Research and development costs consist of salaries, benefits, and other personnel-related costs, including stock-based compensation, laboratory supplies, process development costs, fees paid to other entities to conduct certain research and development activities on the Company’s behalf, including contract manufacturing organizations and contract research organizations, and allocated facility and other related costs. Non-refundable advance payments for goods or services that will be used or rendered for future research and development activities are deferred and capitalized as prepaid expenses until the related goods are delivered or services are performed. The Company hasnot recorded any research and development credits on the consolidated balance sheet at December 31, 2024 and 2023.
The Company records accrued liabilities for estimated costs of research and development activities conducted by third-party service providers. The Company accrues for these costs based on factors such as estimates of the work completed and in accordance with agreements established with its third-party service providers under the service agreements. The Company makes significant judgments and estimates in determining the accrued liabilities balance in each reporting period. As actual costs become known, the Company adjusts its accrued liabilities. The historical accrual estimates made by the Company have not been materially different from the actual costs.
Stock-Based Compensation
The Company accounts for its stock-based compensation in accordance with ASC Topic 718, Compensation-Stock Compensation, or ASC 718. ASC 718 requires all stock-based payments to employees, non-employees and directors, to be recognized as expense in the consolidated statements of operations based on their grant date fair values. The Company estimates the fair value of stock options granted using the Black-Scholes option pricing model, or Black-Scholes, for stock option grants to employees, non-employees and directors. The fair value of the Company’s common stock is used to determine the fair value of restricted stock awards.
The Company’s stock-based compensation awards are generally subject to service-based vesting conditions. Compensation expense related to awards to employees, non-employees and directors with service-based vesting conditions is recognized on a straight-line basis based on the grant date fair value over the associated service period of the award, which is generally the vesting term.
Black-Scholes requires inputs based on certain subjective assumptions, including (i) the expected stock price volatility, (ii) the expected term of the award, (iii) the risk-free interest rate and (iv) expected dividends. The Company determines the expected volatility using the historical volatility of a peer group of comparable publicly traded companies with product candidates in similar stages of development to the Company’s product candidates. The Company uses the simplified method as prescribed by the SEC Staff Accounting Bulletin No. 107, Share-Based Payment, to calculate the expected term for stock options granted to employees, non-employees and directors whereby, the expected term equals the arithmetic average of the vesting term and the original contractual term of the stock options due to its lack of sufficient historical data. The risk-free interest rate is based on U.S. Treasury securities with a maturity date commensurate with the expected term of the associated award. The expected dividend yield is assumed to be zero as the Company has never paid dividends and has no current plans to pay any dividends on its common stock. The Company recognizes forfeitures as they occur.
The fair value of the common stock underlying stock-based awards is the closing price of the Company’s common stock on the date of grant.
The Company classifies stock-based compensation expense in its consolidated statements of operations in the same manner in which the award recipient’s payroll costs are classified or in which the award recipient’s service payments are classified.
Income Taxes
Income taxes are accounted for using the asset and liability method. Deferred tax assets and liabilities are recognized for the future tax consequences attributable to differences between the financial statement carrying amounts or existing assets and liabilities and their respective tax bases. Deferred tax assets and liabilities are measured using the enacted tax rates expected to apply to taxable income in the years in which those temporary differences are expected to be recovered or settled. The effect on deferred tax assets and liabilities of a change in tax rates is recognized in income in the period of enactment. The Company records a valuation allowance to reduce deferred tax assets to an amount for which realization is more likely than not.
The Company recognizes the tax benefit from an uncertain tax position if it is more likely than not that the tax position will be sustained upon examination by the tax authorities, based on the merits of the position. The Company’s policy is to recognize interest and penalties related to the underpayment of income taxes as a component of income tax expense or benefit. To date, there have beenno interest or penalties charged in relation to the unrecognized tax benefits.
Comprehensive Loss
Comprehensive loss is composed of two components — net loss and other comprehensive loss. Other comprehensive loss refers to gains and losses that under U.S. GAAP are recorded as an element of stockholders’ equity but are excluded from net loss. The Company’s other comprehensive loss consists of foreign currency translation adjustments and unrealized gains and losses on available-for-sale marketable debt securities.
Net Loss Per Share
Basic net loss per share is calculated by dividing the net loss attributable to common stockholders by the weighted average number of shares of common stock outstanding for the period, without consideration of potential dilutive securities. Diluted net loss per share is computed by dividing the net loss attributable to common stockholders by the sum of the weighted average number of common shares plus the potential dilutive effects of potential dilutive securities outstanding during the period.
Potential dilutive securities are excluded from diluted earnings or loss per share if the effect of such inclusion is anti-dilutive. The Company’s potentially dilutive securities include unvested common stock under the Company’s equity incentive plan which have been excluded from the computation of diluted net loss per share as they would be anti-dilutive to the net loss per share. For all periods presented, there is no difference in the number of shares used to calculate basic and diluted shares outstanding due to the Company’s net loss position.
The following table sets forth the outstanding potentially dilutive securities that have been excluded in the calculation of diluted net loss per share because their inclusion would be anti-dilutive:
| As of December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Options to purchase common stock | 5,845,039 | 4,233,203 |
| Total | 5,845,039 | 4,233,203 |
Emerging Growth Company Status
The Company is an “emerging growth company,” as defined in the Jumpstart Our Business Startups Act of 2012, or JOBS Act, and may take advantage of certain exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies. The Company may take advantage of these exemptions until the Company is no longer an “emerging growth company.” Section 107 of the JOBS Act provides that an “emerging growth company” can take advantage of the extended transition period afforded by the JOBS Act for the implementation of new or revised accounting standards. The Company has elected to use the extended transition period for complying with new or revised accounting standards and as a result of this election, its consolidated financial statements may not be comparable to companies that comply with public company effective dates. The Company may take advantage of these exemptions up until the last day of the fiscal year following the fifth anniversary of the IPO or such earlier time that it is no longer an “emerging growth company.” As a result of the Company having elected the extended transition period for complying with new or revised accounting standards pursuant to Section 107(b) of the JOBS Act, 2016-13 and ASU 2019-11 are effective for the Company for fiscal years beginning after December 15, 2022, and interim periods within those fiscal years.
Recent Accounting Pronouncements
From time to time, new accounting pronouncements are issued by the Financial Accounting Standards Board, or FASB, or other standard-setting bodies that the Company adopts as of the specified effective date. The Company qualifies as an “emerging growth company” as defined in the JOBS Act and has elected not to “opt out” of the extended transition related to complying with new or revised accounting standards, which means that when a standard is issued or revised and it has different application dates for public and non-public companies, the Company can adopt the new or revised standard at the time non-public companies adopt the new or revised standard and can do so until such time that the Company either (i) irrevocably elects to “opt out” of such extended transition period or (ii) no longer qualifies as an emerging growth company. The Company may choose to early adopt any new or revised accounting standards whenever such early adoption is permitted for non-public companies. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on its consolidated financial statements and disclosures.
In November 2023, the FASB issued ASU No. 2023-07, Segment Reporting (Topic 280), or ASU 2023-07. This update expands segment disclosure requirements, including new segment disclosure requirements for entities with a single reportable segment among other disclosure requirements. This update is effective for the Company in the consolidated financial statements for the year ending December 31, 2024, and interim periods beginning on January 1, 2025. The Company adopted this standard for the year ended December 31, 2024. (See Note 12 - Segment Reporting).
In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvement to Income Tax Disclosures, or ASU 2023-07, to enhance the transparency and decision usefulness of income tax disclosures. ASU 2023-09 is effective for annual periods beginning after December 15, 2024 on a prospective basis. Early adoption and retrospective application is permitted. The Company is currently evaluating the potential impact of the adoption of ASU 2023-09 on its consolidated financial statements and related disclosures.
In November 2024, the FASB issued ASU No. 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures (Subtopic 220-40), or ASU 2024-03. ASU 2024-03 requires companies to disaggregate certain expenses presented on the face of the financial statements to enhance transparency and help investors better understand an entity's performance within the notes to the financial statements. The amendments in ASU 2024-03 are effective for fiscal years beginning after December 15, 2026, and interim periods within fiscal years beginning after December 15, 2027, with early adoption permitted. The Company will not be required to adopt ASU 2024-03 until January 1, 2027. The Company is currently evaluating the potential impact of the adoption of ASU 2024-03 on its consolidated financial statements and related disclosures.
- Fair Value Measurements
The following tables present information about the Company’s financial assets that have been measured at fair value as of December 31, 2024 and December 31, 2023, and indicate the fair value of the hierarchy of the valuation inputs utilized to determine such fair value. In general, fair values determined by Level 1 inputs utilize quoted prices (unadjusted) in active markets for identical assets or liabilities. Fair value determined by Level 2 inputs utilize observable inputs other than Level 1 prices, such as quoted prices for similar assets or liabilities, quoted market prices in markets that are not active or other inputs that are observable or can be corroborated by observable market data for substantially the full term of the related assets or liabilities. Fair values determined by Level 3 inputs are unobservable data points for the asset or liability, and include situations where there is little, if any, market activity for the asset or liability. The following tables set forth marketable securities as of December 31, 2024 and December 31, 2023 (in thousands):
| Amortized Cost | Unrealized Gains | Unrealized Losses | Total | |
|---|---|---|---|---|
| U.S. Treasury-backed money market funds | 70,731 | 61 | (22) | 70,770 |
| Total | $70,731 | $61 | $(22) | $70,770 |
| Amortized Cost | Unrealized Gains | Unrealized Losses | Total | |
|---|---|---|---|---|
| U.S. Treasury-backed money market funds | 29,622 | 11 | — | 29,633 |
| Total | $29,622 | $11 | $— | $29,633 |
The following tables set forth the fair value of the Company’s financial assets measured at fair value on a recurring basis and indicate the level within the fair value hierarchy utilized to determine such values (in thousands):
| Total | Level 1 | Level 2 | Level 3 | |
|---|---|---|---|---|
| Cash Equivalents: | ||||
| U.S. Treasury-backed money market funds | $45,570 | $45,570 | $— | $— |
| U.S. Treasury notes | — | — | — | — |
| Marketable Securities: | ||||
| U.S. Treasury notes | $70,770 | $70,770 | $— | $— |
| Total | $116,340 | $116,340 | $— | $— |
| Total | Level 1 | Level 2 | Level 3 | |
|---|---|---|---|---|
| Cash Equivalents: | ||||
| U.S. Treasury-backed money market funds | $64,397 | $64,397 | $— | $— |
| U.S. Treasury notes | 11,980 | 11,980 | — | — |
| Marketable Securities: | ||||
| U.S. Treasury notes | $29,633 | $29,633 | $— | $— |
| Total | $106,010 | $106,010 | $— | $— |
Money market funds are highly liquid investments that are valued based on quoted market prices in active markets, which represent a Level 1 measurement within the fair value hierarchy. These money market funds are classified on the consolidated balance sheets under cash and cash equivalents.
- Property and Equipment, Net
The cost and accumulated depreciation of property and equipment were as follows (in thousands):
| December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Lab equipment | $5,110 | $4,821 |
| Computer and office equipment | 1,556 | 1,446 |
| Construction in process | 96 | 69 |
| Total property and equipment | 6,762 | 6,336 |
| Less accumulated depreciation | (3,046) | (1,572) |
| Total property and equipment, net | $3,716 | $4,764 |
Depreciation expense was $1.5 million and $1.2 million for the years ended December 31, 2024 and 2023, respectively.
- Accrued Expenses
Accrued expenses consisted of the following (in thousands):
| December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Research and development | $6,903 | $9,521 |
| Employee-related expenses | 3,617 | 2,368 |
| Professional services | 1,138 | 715 |
| Other | 192 | 918 |
| Total accrued expenses | $11,850 | $13,522 |
- Leases
In December 2021, the Company entered into a non-cancellable operating lease agreement, or the Harrison Lease, pursuant to which the Company leases 31,668 square feet of office and laboratory space located in Boston, Massachusetts, or the Premises. The Harrison Lease commenced for accounting purposes when the Company gained access to the Premises on December 29, 2022, or the Lease Commencement Date. The Harrison Lease has a term ofnine years and two months from the Lease Commencement Date. The Company’s obligation for the payment of base rent for the Premises began in May 2023, and is $0.2 million per month, increasing up to $0.3 million per month during the term of the Lease. The Company has oneoption to extend the term of the Harrison Lease, for a period of an additional five years. This is not included in the lease term as it was not deemed probable that the Company would exercise this option. At December 31, 2024, the Harrison Lease was the only lease for which the Company recorded a lease liability and corresponding right-of-use asset.
The landlord completed significant leasehold improvements to the Premises, a portion of which the Company was obligated to pay per the terms of the Harrison Lease. The Company paid $2.0 million for the improvements prior to the Lease Commencement Date, and paid $3.4 million for the improvements in 2023, for total payments to the landlord for improvements of $5.4 million. The Company determined that the landlord is the accounting owner of the improvements, and payments by the Company for the improvements are included in the calculation of the right-of-use asset and lease liability.
During the first quarter of 2023, the Company leased office space in Cambridge, Massachusetts, the terms of which were month-to-month, with a 30-day written notice of cancellation. The Company terminated this lease in January 2023. The Company also leased laboratory space at the University of Massachusetts, Mount Ida Campus in Newton, Massachusetts, with an initial lease term from
February 1, 2022 to January 31, 2023, which the Company extended until March 2023. All of these leases had initial terms of under 12 months and are considered short-term lease costs and are not recognized on the consolidated balance sheets.
Summary of Lease Costs
The components of lease cost under ASC 842 for the leases were as follows (in thousands):
| December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Fixed lease cost | $3,748 | $3,799 |
| Variable lease cost | 1,965 | 993 |
| Short-term lease cost | — | 188 |
| Total lease cost | $5,713 | $4,980 |
Supplemental disclosure of cash flow information under ASC 842 for the leases was as follows (in thousands):
| Operating cash payments for operating leases | $3,004 |
|---|
Cash payments for operating leases during 2024 related to $3.0 million in rent payments under the Harrison Lease. Additionally, the Company made payments on variable lease costs throughout the year of $2.0 million. These payments related to parking, management fees, and other costs associated with maintaining our office and lab space.
The remaining lease term for the Harrison Lease is 7.4 years, and the discount rate is 8.0%.
Future minimum lease payments under the non-cancelable operating lease consisted of the following as of December 31, 2024:
| Year Ending December 31, · (in thousands) | ||
|---|---|---|
| 2025 | $3,094 | |
| 2026 | 3,187 | |
| 2027 | 3,282 | |
| 2028 | 3,381 | |
| 2029 | 3,483 | |
| Thereafter | 8,596 | |
| Total future minimum lease payments | $25,023 | |
| Less: imputed interest | (6,362) | |
| Present value of lease liability | $18,661 | |
| Included in the consolidated balance sheet (in thousands) | December 31, 2024 | |
| Lease liability | $3,094 | |
| Lease liability, net of current portion | 15,567 | |
| Total lease liability | $18,661 | |
- Related Party Transactions
Technology License Agreement with Oxford University Innovation Limited
In March 2018, the Company, Oxford University Innovation Limited, or OUI, and the Medical Research Council of United Kingdom Research and Innovation, or MRC, or collectively, the Licensors, entered into a license of technology agreement, which was subsequently amended in December 2018, further amended and restated in November 2020, and further amended in February 2022, referred to, as so amended, as the License Agreement. The Licensors and affiliates held shares of Series A-1 and Series A-2 preferred stock, Series B preferred stock and Class A common stock. The License Agreement provides the Company with an exclusive world wide license to licensed data and technology owned by OUI and MRC in respect of cell-penetrating peptides for the treatment of Duchenne muscular dystrophy, spinal muscular atrophy, and other conditions. The License Agreement provides the Company with the rights to grant and authorize sublicenses to make, use, sell, and import products and otherwise exploit the patent rights.
As consideration for the license, the Company made an initial upfront payment in 2018, as well as a Restatement Completion Fee and a License Data Fee (each as defined in the License Agreement) in 2020 totaling $0.1 million.
The Company could be required to make milestone payments to the Licensors upon achievement of certain patent and commercial milestones related to the patents and commercialization of certain of the Company’s product candidates. The aggregate potential milestone payments are $0.1 million. In 2024, the Company paid the Licensors a patent procurement milestone of £10,000, adjusted per the retail price index under the terms of the License Agreement. The Company also agreed to pay the Licensors low single digit royalties on net sales of any licensed products that are commercialized by the Company in excess of a threshold amount between £20 million and £30 million ($25.1 million and $37.7 million as of December 31, 2024), subject to certain adjustments. The term of the License Agreement continues until the later of (i) the date on which all the patents and patent applications covered thereunder have been abandoned or allowed to lapse or expired or been rejected or revoked or (ii)20 years from the date of the original license agreement.
Upon completion of the IPO, the Company became obligated to pay OUI an exit fee between 0.5% to 2% of the value determined in an acquisition or initial public offering, not to exceed £5 million ($6.2 million as of the IPO date). The exit fee due to OUI, based on the proceeds of the IPO, was $1.4 million, which was paid during the second quarter of 2022 and included in research and development expense on the condensed consolidated statement of operations.
During the year ended December 31, 2024, one member of the Company’s board of directors, Dr. Christopher Ashton, was employed by Oxford Science Enterprises, or OSE, which is an affiliate of OUI. On September 19, 2024, the Company announced that Dr. Ashton had resigned from the board of directors, effective as of September 30, 2024.
As of December 31, 2024, OSE owned 14.6% of the Company’s outstanding common stock. Pursuant to the terms of Dr. Ashton's employment agreement with OSE, he was obligated to transfer any cash compensation from the Company to OSE. Fees paid in cash for Dr. Ashton's service on the Company's board of directors were paid directly to OSE and were included within general and administrative expense on the consolidated statement of operations. The Company paid $36,875 and $47,500 to OSE for the years ended December 31, 2024 and 2023, respectively, as compensation for Dr. Ashton's service on the board of directors.
RA Capital Management, L.P.
Entities affiliated with RA Capital Management, L.P., or RA Capital, purchased common stock in the Company’s IPO in May 2022 and the Company’s Follow-on Offering in February 2024. As of December 31, 2024, entities affiliated with RA Capital owned 32.8% of the Company’s outstanding common stock.
Two members of the Company’s board of directors, Dr. Joshua Resnick and Habib Dable, are affiliated with RA Capital. Pursuant to the governing legal documents of RA Capital, Dr. Resnick is obligated to transfer any cash compensation from the Company to RA Capital. Fees paid in cash for Dr. Resnick's service on the Company's board of directors were paid directly to RA Capital and are included within general and administrative expense on the consolidated statement of operations. The Company paid $45,500 and $43,000 to RA Capital for the years ended December 31, 2024 and 2023, respectively, for Dr. Resnick’s service on the board of directors.
- Commitments and Contingencies
Legal proceedings
From time to time, the Company may have certain contingent liabilities that arise in the ordinary course of its business activities. The Company accrues a liability for such matters when it is probable that future expenditures will be made and that such expenditures can be reasonably estimated.
The Company is not party to any litigation and does not have contingency reserves established for any litigation liabilities.
Other
The Company enters into standard indemnification agreements in the ordinary course of business. Pursuant to the agreements, the Company agrees to indemnify, hold harmless, and to reimburse the indemnified party for losses suffered or incurred by the indemnified party, including in connection with any U.S. patent or any copyright or other intellectual property infringement claim by any third-party with respect to the Company’s products. Further, the Company indemnifies its directors and officers who are, or were, serving at the Company’s request in such capacities. The Company’s maximum exposure under these arrangements is unknown as of December 31, 2024. The Company does not anticipate recognizing any significant losses relating to these arrangements. The term of these indemnification agreements is generally perpetual any time after execution of the agreement. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements may be unlimited. The Company has never incurred costs to defend lawsuits or settle claims related to these indemnification agreements.
- Stockholders’ Equity
Under the Third Amended and Restated Certificate of Incorporation, dated May 10, 2022, as amended on June 20, 2024, as currently in effect, the Company has the authority to issue a total of 500,000,000 shares of common stock (par value of $0.0001 per share) and 10,000,000 shares of undesignated preferred stock (par value of $0.0001 per share). Each share of common stock has the right toone vote. The holders of common stock are also entitled to receive dividends whenever funds are legally available and when declared by the board of directors, subject to the prior rights of holders of all classes of stock outstanding having priority rights as to dividends. No cash dividends were declared by the board of directors during the years ended December 31, 2024 and December 31, 2023.
The Company has reserved shares of common stock for issuance, on an as-converted basis, as follows:
| December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Stock options issued and outstanding | 5,845,039 | 4,233,203 |
| Unvested restricted stock and performance stock units | 101,280 | — |
| Authorized for future stock awards or option grants under the 2022 Plan | 657,167 | 1,091,187 |
| Authorized for future stock awards or option grants under the Inducement Plan | 702,184 | — |
| Authorized for future issuance under the ESPP | 397,998 | 216,306 |
| Stock options issued and outstanding | 7,703,668 | 5,540,696 |
- Stock-Based Compensation
The Company maintains four equity compensation plans; the 2020 Stock Plan, or the 2020 Plan, the 2022 Stock Option and Incentive Plan, or the 2022 Plan, the 2022 Employee Stock Purchase Plan, or the ESPP, and the 2024 Inducement Plan, or the Inducement Plan. As of the Company's IPO in May 2022, the Company's board of directors determined that no further awards would be made under the 2020 Plan. The number of shares of common stock that may be issued under the 2022 Plan is subject to increase by the number of shares under any outstanding stock options forfeited and not exercised under the 2020 Plan. Additionally, the number of shares reserved for issuance under the 2022 Plan automatically increases on the first day of each fiscal year in an amount equal to the lower of (1)5% of the shares of common stock outstanding on such date and (2) an amount determined by the Company’s board of directors. The 2022 Plan allows the board of directors to grant incentive stock options, non-qualified stock options, stock appreciation rights, restricted stock awards, restricted stock units, unrestricted stock awards, cash-based awards, and dividend equivalent rights to the Company’s officers, employees, directors and other key persons. The number of shares reserved for issuance under the ESPP automatically increases on the first day of each fiscal year in an amount equal to the lower of (1)1% of the shares of common stock outstanding on such date and (2) an amount determined by the Company’s board of directors.
As of December 31, 2024,657,167 shares remained available for grant under the 2022 Plan and 397,998 shares remained available for issuance under the ESPP.
2024 Inducement Plan
In August 2024, the Company's board of directors adopted the Inducement Plan. The Inducement Plan provides for the grant of non-qualified stock options, stock appreciation rights, restricted stock awards, restricted stock units unrestricted stock awards, dividend equivalent rights and other stock-based awards with respect to an aggregate of 1,000,000 shares of common stock (subject to adjustment as provided in the Inducement Plan). Awards under the Inducement Plan may only be granted to new employees who were not previously employed by the Company or its affiliates in accordance with the requirements of Nasdaq Stock Market Rule 5635(c)(4).
As of December 31, 2024,702,184 shares remained available for issuance under the Inducement Plan.
Stock Option Valuation
In determining the fair value of the stock options granted, the Company uses Black-Scholes and the assumptions discussed below. Each of these inputs is subjective and generally requires significant judgment.
Expected Term—The Company’s expected term represents the period that the Company’s stock-based awards are expected to be outstanding. For stock options with service-based vesting periods, the expected term of the Company’s stock options has been determined utilizing the “simplified” method for awards that qualify as “plain-vanilla” options.
Expected Volatility—The Company has historically been a private company and lacks significant company-specific historical and implied volatility information. The expected volatility is estimated based on a blend of the Company's own volatility and the average historical volatilities 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 their similar size, stage in the life cycle and area of specialty. The Company will continue to apply this process until enough 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 option.
Expected 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 fair value of stock options was estimated using the following weighted average assumptions:
| Year EndedDecember 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Risk-free interest rate % | 4.15% | 4.19% |
| Expected volatility % | 81.33% | 65.13% |
| Expected term (in years) | 6.06 | 6.05 |
| Expected dividend yield | — | — |
Stock Option Activity
Stock option activity for the year ended December 31, 2024 was as follows:
| StockOptions | Weighted-AverageExercisePrice | Weighted-AverageRemainingContractualTerm(In Years) | AggregateIntrinsicValue(In Thousands) | |
|---|---|---|---|---|
| Outstanding as of December 31, 2023 | 4,233,203 | $10.83 | 8.4 | $2,147 |
| Granted | 2,175,133 | 14.13 | ||
| Exercised | (209,882) | 10.62 | ||
| Canceled/Forfeited | (353,415) | 13.23 | ||
| Outstanding as of December 31, 2024 | 5,845,039 | $11.92 | 8.0 | $578 |
| Vested and exercisable as of December 31, 2024 | 2,467,174 | $9.92 | 7.0 | $565 |
| Vested and expected to vest as of December 31, 2024 | 5,845,039 | $11.92 | 8.0 | $578 |
The aggregate intrinsic value of stock options is calculated as the difference between the exercise prices of the stock options and the fair value of the Company's common stock for those stock options that had exercises prices lower than the fair value of the Company's common stock. The total intrinsic value of the stock options exercised during the years ended December 31, 2024 and 2023 was $1.0 million and $1.4 million, respectively. The intrinsic value is the difference between the estimated fair value of the Company’s common stock at the time of exercise and the exercise price of the stock option. During the years ended December 31, 2024 and 2023, the total proceeds to the Company from stock option exercises was $2.2 million and $0.2 million, respectively.
The weighted-average grant date fair value of stock options granted during the years ended December 31, 2024 and 2023 was $10.19 and $8.56 per share, respectively.
Restricted Stock Units
A restricted stock unit, or RSU, represents the right to receive one share of common stock upon vesting of the RSU. In February 2024, the Company granted employees a one-time RSU award that vests fully on the one-year anniversary of the grant date, provided that the employee remains employed with the Company. Certain employees, including employees who are executive officers of the Company, received a one-time RSU award that vests upon the achievement of certain performance-based clinical development milestones, or PSUs. Such awards cannot vest in less than one year, regardless of when the performance milestone is achieved. The Company’s chief executive officer did not receive any RSU or PSU awards. A summary of the Company’s RSU and PSU activity and related information for the year ended December 31, 2024 was as follows:
| Time-Based RSUs | PSUs | Weighted-AverageGrant Date Fair Value | |
|---|---|---|---|
| Outstanding as of December 31, 2023 | — | — | $— |
| Granted | 77,645 | 28,555 | 10.64 |
| Vested | — | — | — |
| Forfeited | (4,920) | — | 10.64 |
| Issued and unvested as of December 31, 2024 | 72,725 | 28,555 | $10.64 |
The weighted average grant date fair value of the time-based RSUs and the PSUs granted during the year ended December 31, 2024 was $10.64. As of December 31, 2024, there was $0.1 million of unrecognized compensation costs related to unvested time-based RSUs, which are expected to be recognized over a weighted-average period of 0.1 years, and $0.3 million of unrecognized compensation costs related to unvested PSUs, which are dependent upon achievement of the aforementioned clinical development milestones
Stock-Based Compensation Expense
Stock-based compensation expense recognized for stock option grants included in the accompanying consolidated statements of operations and comprehensive loss is as follows (in thousands):
| Year EndedDecember 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Research and development | $5,012 | $2,966 |
| General and administrative | 6,445 | 4,082 |
| Total stock-based compensation expense | $11,457 | $7,048 |
The Company had 5,845,039 vested and unvested stock options outstanding as of December 31, 2024. As of December 31, 2024, total unrecognized compensation expense related to stock options was $29.7 million. This amount is expected to be recognized over a weighted average period of approximately 2.6 years.
- Income Taxes
The Company’s loss before income taxes for the years ended December 31, 2024 and 2023 were generated in the following jurisdictions (in thousands):
| Year EndedDecember 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Domestic | $(87,466) | $(78,195) |
| Foreign | (3,132) | (358) |
| Worldwide | $(90,598) | $(78,553) |
The components of net deferred income taxes consisted of the following as of December 31, 2024 and 2023 (in thousands):
| December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Deferred tax assets: | ||
| Net operating loss carryforwards | $17,039 | $8,038 |
| Research and development credits | 7,769 | 4,547 |
| Accrued expenses | 864 | 577 |
| Capitalized Section 174 R&D Costs | 41,821 | 29,470 |
| Stock compensation | 4,511 | 2,510 |
| Right-of-use liability | 5,086 | 5,485 |
| Intangible asset amortization | 6,847 | 7,425 |
| Other, net | 4 | 21 |
| Deferred tax assets | 83,941 | 58,073 |
| Deferred tax liabilities | ||
| Right-of-use-Asset | (5,842) | (6,444) |
| Fixed Assets | (1,316) | (1,474) |
| Other, net | — | — |
| Deferred tax liabilities | (7,158) | (7,918) |
| Net deferred tax assets | 76,783 | 50,155 |
| Valuation allowance | (76,783) | (50,155) |
| Net deferred tax assets | $— | $— |
The components of income tax expense were as follows for the years ended December 31, 2024 and 2023 (in thousands):
| Year EndedDecember 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Current expense: | ||
| U.S. Federal | $— | $— |
| State | 84 | 73 |
| Foreign | (701) | — |
| Total current expense | (617) | 73 |
| Deferred expense: | ||
| U.S. Federal | — | — |
| State | — | — |
| Foreign | — | — |
| Total deferred expense | — | — |
| Provision for income taxes | $(617) | $73 |
A reconciliation of income tax expense to the amount computed by applying the statutory federal income tax rate to the loss from operations is summarized for the years ended December 31, 2024 and 2023, as follows:
| Year EndedDecember 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Tax at statutory rate | 21.0% | 21.0% |
| State tax (net of federal benefit) | (0.1)% | (0.7)% |
| Permanent differences | (0.8)% | (0.4)% |
| Research and development credit | 3.4% | 3.2% |
| GILTI Inclusion | 0.0% | 0.0% |
| U.K. R&D credit | 0.0% | 0.0% |
| Foreign rate differential | 1.0% | 0.0% |
| Change in valuation allowance | (23.6)% | (23.9)% |
| Other | (0.2)% | 0.0% |
| Income tax expense (benefit) | 0.7% | (0.8)% |
On January 1, 2022, or the Transfer Date, the Company’s wholly owned subsidiary, PepGen Limited, transferred all IP assets to the parent company, PepGen, in an arm’s length transaction at fair value pursuant to an asset transfer agreement. The Company recognized a tax expense in 2022 of $3.7 million, inclusive of a $0.7 million unrecognized tax benefit. The Company’s income tax benefit and expense was a net benefit of $0.6 million for the year ended December 31, 2024 and expense of $0.1 million for the year ended December 31, 2023. The income tax benefit for the year ended December 31, 2024 was due to the derecognition of an unrecognized tax benefit of $0.7 million upon the dissolution of our U.K.-based entity, PepGen Limited on December 10, 2024. This is partially offset by $0.1 million instate taxes on interest income generated from the Company's cash equivalents and marketable securities. The income tax expense for the year ended December 31, 2023 consisted of state taxes on interest income generated from the Company's cash equivalents and marketable securities.
The Company has federal net operating losses, or NOLs, of $58.2 million and federal research and development, or R&D, credits of approximately $9.1 million as of December 31, 2024, before consideration of limitations under IRC Section 382 of the Internal Revenue Code of 1986, as amended, or Section 383, as further described below. The federal NOLs do not expire, but generally limit the NOLs deduction to the lesser of the NOL carryover or 80% of a corporation's taxable income. The tax credits will begin to expire in 2042. As of December 31, 2024, the Company has state NOLs of $76.6 million and state research and development credits of $1.8 million. The state NOLs will begin to expire in 2041, and the tax credits will begin to expire in 2037.
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 stockholders over athree-year period in excess of 50%, as defined under Sections 382 and 383 of the IRC, 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. An assessment of such ownership changes under Section 382 was not completed through December 31, 2024. To the extent that an assessment is completed in the future, the Company’s ability to utilize tax attributes could be restricted on a year-by-year basis and certain attributes could expire before they are utilized. The Company will examine the impact of any potential ownership changes in the future. A full valuation allowance has been provided against the deferred tax assets related to the Company's net operating loss and tax credits carryforwards and, if an adjustment is required, this adjustment would be offset by an adjustment to the valuation allowance.
The Company files tax returns as prescribed by the tax laws of the jurisdictions in which it operates. In a normal course of business, the Company is subject to examination by U.S. federal and state as well as foreign jurisdictions, where applicable. The Company’s tax years are still open since inception. To the extent that the Company has tax attribute carryforwards, the tax year in which the attributes were generated may still be adjusted upon examination by the U.S. Internal Revenue Services or state tax authorities to the extent utilized in a future period. The Company is not currently under examination by any tax authorities.
The following table summarizes the reconciliation of the beginning and ending amount of total unrecognized tax benefits recorded in the statement of operations for each of the periods indicated:
| December 31, | ||
|---|---|---|
| 2024 | 2023 | |
| Balance at beginning of the period | $713 | $673 |
| Increase related to current year tax positions | — | — |
| Increase related to prior year tax positions | — | — |
| Decrease related to prior year tax positions | (701) | — |
| Currency translation | (12) | 40 |
| Balance at the end of the period | $— | $713 |
The Company's policy is to recognize interest and/or penalties related to income tax matters in income tax expense. The Company released approximately $0.7 million in unrecognized tax benefits due to the liquidation of its UK subsidiary, PepGen Limited. As of December 31, 2024, the Company hadno unrecognized tax benefits or accrued interest or penalties.
- Segment Reporting
ASU 2023-07 establishes standards for reporting information about operating segments on a basis consistent with the Company’s internal organization structure as well as information about services categories, business segments and major customers in financial statements.
Segment reporting is prepared on the same basis that the chief executive officer, who is the Company’s chief operating decision maker and manages the business, makes business decisions and assesses performance. The Company hasone reportable segment and regularly reviews operating expenses and net loss on a consolidated basis. The Company operates in one location in Boston, Massachusetts focused on advancing oligonucleotide therapeutics with the goal of transforming the treatment of severe neuromuscular and neurologic diseases. The Company tracks external operating expenses on a program-by-program basis. Net loss is comprised of expenses related to our two lead product candidates and other personnel, facility, and general and administrative costs. Operating expenses and net loss are the basis the chief operating decision maker uses for the purpose of deciding how to allocate resources, specifically on a program basis as presented below.
The chief operating decision maker uses operating expenses and net loss to evaluate progress on research and development activities on the Company's two lead product candidates as well as when making strategic decisions regarding the Company's pipeline. Operating expenses and loss are also used to monitor budget versus actual results.
The following table outlines the Company’s functional operating expenses:
| Year Ended | ||
|---|---|---|
| 2024 | 2023 | |
| Research and Development Expenses | ||
| PGN-EDO51 direct research and development expenses | $19,195 | $21,369 |
| PGN-EDODM1 direct research and development expenses | 21,265 | 16,486 |
| Platform and external research and development expenses | 2,556 | 4,762 |
| Personnel-related (including stock-based compensation) | 23,433 | 16,143 |
| Facility related and other | 5,819 | 5,165 |
| Other research and development expenses | 4,210 | 4,201 |
| Total research and development expense | 76,478 | 68,126 |
| General and Administrative Expenses | ||
| Personnel-related (including stock-based compensation) | 12,449 | 8,198 |
| Facility related and other | 1,987 | 1,504 |
| Other general and administrative | 6,825 | 6,938 |
| Total general and administrative expense | 21,261 | 16,640 |
| Loss from operations | (97,739) | (84,766) |
| Other income (expense): | ||
| Interest income | 7,142 | 6,400 |
| Other (expense) income, net | (1) | (187) |
| Total other income (expense), net | 7,141 | 6,213 |
| Income tax expense | 617 | (73) |
| Net loss | $(89,981) | $(78,626) |
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.