Stoke Therapeutics, Inc.
For the period ended Mar 31, 2025. Filed May 13, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2025 · Filed May 13, 2025
Extracted filing text, with the paragraph anchors used by the comparison.
See the whole filing, with its images and exhibitsContents
Notes to unaudited consolidated financial statements
Notes to Consolidated financial statements—(unaudited)
- Nature of the business
Organization
Stoke Therapeutics, Inc. (the Company) was founded in June 2014 and was incorporated under the laws of the State of Delaware. The Company is an early-stage biopharmaceutical company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression.
Shelf Registration
In May 2022, the Company filed a universal Shelf Registration statement on Form S-3 (the “Registration Statement”) with the Securities and Exchange Commission (the “SEC”). The Registration Statement was declared effective by the SEC on May 31, 2022, and contained two prospectuses: a base prospectus, which covered the offering, issuance and sale by the Company of up to a maximum aggregate offering price of $400.0 million of its common stock, preferred stock, debt securities, warrants to purchase common stock, preferred stock or debt securities, subscription rights to purchase common stock, preferred stock or debt securities and/or units consisting of some or all of these securities; and a sales agreement prospectus covering the offering, issuance and sale by the Company of up to a maximum aggregate offering price of $150.0 million of its common stock that may be issued and sold under a Controlled Equity Offering Sales Agreement (“Sales Agreement”). The specific terms of any securities to be offered pursuant to the base prospectus were specified in a prospectus supplement to the base prospectus. The $150.0 million of common stock that could have been offered, issued and sold under the Sales Agreement prospectus was included in the $400.0 million of securities that could have been offered, issued and sold by the Company under the base prospectus. As of March 31, 2025, the Company had issued approximately 7.0 million shares of common stock pursuant to the Sales Agreement for net proceeds of $61.0 million.
In April 2024, the Company completed an underwritten public offering of its common stock and issued and sold 5,555,557 shares of common stock at a public offering price of $13.50 per share and issued pre-funded warrants to purchase 3,703,730 shares of common stock at a public offering price of $13.499 per share subject to an exercise price equal to $0.0001. The common stock and pre-funded warrants sold resulted in net proceeds of approximately $119.9 million after deducting underwriting discounts, commissions and offering costs.
In October 2024, the Company filed an automatic universal Shelf Registration statement on Form S-3 (the “2024 Registration Statement”) with the SEC. The 2024 Registration Statement contains two prospectuses: a base prospectus, which covers the offering, issuance and sale by the Company of its common stock, preferred stock, debt securities, warrants to purchase common stock, preferred stock or debt securities, subscription rights to purchase common stock, preferred stock or debt securities and/or units consisting of some or all of these securities; and a sales agreement prospectus covering the offering, issuance and sale by the Company of up to a maximum aggregate offering price of $150.0 million of its common stock that may be issued and sold under the Sales Agreement. The specific terms of any securities to be offered pursuant to the base prospectus will be specified in a prospectus supplement to the base prospectus. Following the filing of the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 in March 2025, the 2024 Registration Statement is no longer effective. In addition, as of March 31, 2025, the Company hadnot issued or sold any shares pursuant to the 2024 Registration Statement.
Uncertainties
The Company is subject to risks and uncertainties common to early-stage companies in the biotechnology industry including, but not limited to, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations and ability to secure additional capital to fund operations. Product candidates currently under development will require significant additional research and development efforts, including extensive preclinical and clinical testing and regulatory approval prior to commercialization. These efforts require significant amounts of additional capital, adequate personnel and infrastructure and extensive compliance-reporting capabilities. Even if the Company’s product development efforts are successful, it is uncertain when, if ever, the Company will realize significant revenue from product sales.
Liquidity
The Company expects that its operating losses and negative cash flows will continue for the foreseeable future. As of the issuance date of these unaudited consolidated financial statements, the Company expects that its cash, cash equivalents and marketable securities will be sufficient to fund its operating expenses and capital expenditure requirements through at least twelve months from the issuance date of these unaudited consolidated financial statements.
- Summary of significant accounting policies and recent accounting pronouncements
Basis of presentation and consolidation
The accompanying unaudited consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) and include the accounts of the Company and its wholly-owned subsidiary. Any reference in these notes to applicable guidance is meant to refer to GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Update (“ASU”) of the Financial Accounting Standards Board (“FASB”). All intercompany transactions between and among the Company and its consolidated subsidiary have been eliminated.
Unaudited interim financial information
The accompanying interim unaudited consolidated financial statements and related disclosures are unaudited and have been prepared in accordance with GAAP for interim financial information and the instructions to Form 10-Q and Article 10 of Regulation S-X. Accordingly, they do not include all the information and footnotes required by GAAP for complete financial statements and should be read in conjunction with the Company’s consolidated financial statements and related notes as of and for the year ended December 31, 2024, which was filed with the SEC on March 18, 2025. The Company’s financial information as of March 31, 2025 and for the three months ended March 31, 2025 and 2024 is unaudited, but in the opinion of management, all adjustments, consisting only of normal recurring adjustments, considered necessary for a fair presentation of the financial position, results of operations and cash flows at the dates and for the periods presented of the results of these interim periods have been included. The balance sheet information as of December 31, 2024 was derived from audited consolidated financial statements. The results of the Company’s operations for any interim period are not necessarily indicative of the results that may be expected for any other interim period or for a full fiscal year.
Use of estimates
The preparation of consolidated financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenue, expenses and disclosure of contingent assets and liabilities. Estimates are periodically reviewed in light of changes in circumstances, facts and experience. Actual results could differ from those estimates.
Cash, cash equivalents and restricted cash
The Company considers all highly liquid investments with an original maturity of three months or less at the date of purchase to be cash equivalents. The Company deposits its cash in checking, sweep and money market accounts.
At March 31, 2025, restricted cash consisted of money market accounts collateralizing letters of credit issued as security deposits in connection with the Company’s leases of its corporate facilities.
Cash and cash equivalents, and restricted cash in the consolidated statements of cash flows consists of the following (in thousands):
| As of March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Cash and cash equivalents | $274,817 | $178,581 |
| Restricted cash - current | 75 | — |
| Restricted cash - long-term | 721 | 569 |
| Total cash, cash equivalents and restricted cash | $275,613 | $179,150 |
Smaller reporting company status
The Company is a “smaller reporting company,” meaning that the market value of its stock held by non-affiliates was less than $700.0 million and its annual revenue is less than $100.0 million as of the last business day of its most recently completed fiscal year. The Company will continue to be a smaller reporting company for so long as either (i) the market value of its stock held by non-affiliates is less than $250.0 million or (ii) its annual revenue is less than $100.0 million during the most recently completed fiscal year and the market value of its stock held by non-affiliates is less than $700.0 million. In addition, as a smaller reporting company the Company may choose to present only the two most recent fiscal years of audited financial statements in its Annual Report on Form 10-K and smaller reporting companies have reduced disclosure obligations regarding executive compensation.
Revenue Recognition under ASC 606
For revenue arrangements accounted for under ASC 606, the Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the entity expects to receive in exchange for those goods or services. To determine the appropriate amount of revenue to be recognized for arrangements determined to be within the scope of ASC 606, the Company performs the following five steps: (i) identification of the contract; (ii) determination of whether the promised goods or services are performance obligations; (iii) measurement of the transaction price, including evaluating the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. The Company applies the five-step model to all contracts, including transactions with collaborators or partners, if they are a transaction with a customer.
Performance obligations are promises to transfer distinct goods or services to the customer. Promised goods or services are considered distinct when (i) the customer can benefit from the good or service on its own or together with other readily available resources and (ii) the promised good or service is separately identifiable from other promises in the contract. In assessing whether promised good or services are distinct, the Company considers factors such as the stage of development of the underlying intellectual property, the capabilities of the customer to develop the intellectual property on their own, or whether the required expertise is readily available.
The Company estimates the transaction price based on the amount expected to be received for transferring the promised goods or services in the contract. The consideration may include both fixed consideration and variable consideration. At the inception of an arrangement that includes variable consideration and at each reporting period, the Company evaluates the amount of potential payment and the likelihood that the payments will be received. The Company utilizes either the most likely amount method or expected amount method to estimate the amount to be received based on which method better predicts the amount expected to be received. If it is probable that a significant reversal of cumulative revenue would not occur, the variable consideration is included in the transaction price. The Company will assess its revenue generating arrangements in order to determine whether a significant financing component exists and conclude that a significant financing component does not exist in any of its arrangements if: (a) the promised consideration approximates the cash selling price of the promised goods and services or any significant difference is due to factors other than financing; and (b) timing of payment approximates the transfer of goods and services and performance is over a relatively short period of time within the context of the entire term of the contract.
Contracts may include pre-commercial milestone payments. At contract inception and at each reporting period, the Company assesses whether achievement of the milestone was most likely and whether it is probable that a significant reversal in the amount of cumulative revenue recognized would not occur. If it is probable that a significant reversal of cumulative revenue would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the Company’s control or the customer’s control are constrained and not included in the transaction price. At the end of each subsequent reporting period, the Company re-evaluates the probability of achievement of such milestones and any related constraint, and if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment.
For arrangements that may include sales-based royalties, including milestone payments based on the level of sales, and the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). To date, the Company has not recognized any royalty revenue resulting from any of the Company’s collaboration arrangements.
The Company allocates the transaction price based on the estimated standalone selling price of the underlying performance obligations or in the case of certain variable consideration to one or more performance obligations. The Company must develop assumptions that require judgment to determine the stand-alone selling price for each performance obligation identified in the contract. The Company utilizes a range of key assumptions to determine the stand-alone selling price, which may include prices observed in comparable market transactions, pricing considered in negotiating the transaction, the estimated costs, plus a reasonable margin, forecasted revenue, development timelines, reimbursement rates for personnel costs, discount rates and probabilities of success in achieving various pre-commercial and commercial milestones, to complete the respective performance obligation. Certain variable consideration is allocated specifically to one or more performance obligations in a contract when the terms of the variable consideration relate to the satisfaction of the performance obligation and the resulting amounts allocated to each performance obligation are consistent with the amounts the Company would expect to receive for each performance obligation.
For performance obligations consisting of licenses, research and development activities, and other promises, the Company utilizes judgment to assess the nature of the performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. If the license to the Company’s intellectual property is determined to be distinct from the other performance obligations identified in the arrangement, the Company will recognize revenue when the license is transferred to the customer and the customer is able to use and benefit from the license. Upfront payments and fees are recorded as deferred revenue upon receipt or when due until the Company performs its obligations under these arrangements. Amounts are recorded as accounts receivable when the right to consideration is unconditional, including unbilled amounts.
Payment terms and conditions vary by contract type, although terms typically require payment within 30 to 60 days. In instances where the timing of revenue recognition differs from that of invoicing, the Company has determined that its contracts generally do not include a significant financing component. The primary purpose of invoicing terms is to provide customers with simplified and predictable ways of purchasing the Company’s products and services, not to facilitate financing arrangements.
- Fair value measurements
The following tables present information about the Company’s financial assets measured at fair value on a recurring basis and indicate the level of the fair value hierarchy utilized to determine such fair values (in thousands):
| Level 1 | Level 2 | Level 3 | Total | |
|---|---|---|---|---|
| Cash equivalents: | ||||
| Money market funds | $270,159 | $— | $— | $270,159 |
| Total | $270,159 | $— | $— | $270,159 |
| Marketable Securities: | ||||
| Corporate bonds | $— | $24,446 | $— | $24,446 |
| Commercial paper | — | 14,775 | — | 14,775 |
| US Government debt securities | — | 66,275 | — | 66,275 |
| Total | $— | $105,496 | $— | $105,496 |
| Level 1 | Level 2 | Level 3 | Total | |
|---|---|---|---|---|
| Cash equivalents: | ||||
| Money market funds | $122,446 | $— | $— | $122,446 |
| Total | $122,446 | $— | $— | $122,446 |
| Marketable Securities: | ||||
| Corporate bonds | $— | $18,668 | $— | $18,668 |
| Commercial paper | — | 6,944 | — | 6,944 |
| US Government debt securities | — | 93,128 | — | 93,128 |
| Total | $— | $118,740 | $— | $118,740 |
The Company’s cash equivalents and marketable securities are carried at fair value, determined according to the fair value hierarchy. The carrying value of the Company’s accounts payable and accrued expenses approximate their fair values due to the short-term nature of these liabilities.
The Company’s assets with fair value categorized as Level 1 within the fair value hierarchy include money market funds. Money market funds are publicly traded mutual funds and are presented as cash equivalents on the consolidated balance sheets as of March 31, 2025 and December 31, 2024.
The Company measures its marketable securities at fair value on a recurring basis and classifies those instruments within Level 2 of the fair value hierarchy. Marketable securities are valued using models or other valuation methodologies that use Level 2 inputs. These models are primarily industry-standard models that consider various assumptions, including time value, yield curve, volatility factors, default rates, current market and contractual prices for the underlying financial instruments, as well as other economic measures. Substantially all of these assumptions are observable in the marketplace, can be derived from observable data or are supported by observable levels at which transactions are executed in the marketplace.
There wereno transfers to Level 3 in the periods presented.
- Marketable securities
The following table summarizes the Company’s marketable securities as of March 31, 2025 (in thousands):
| Amortized Cost | Unrealized Gains | Unrealized Loss | Fair Value | |
|---|---|---|---|---|
| Marketable securities: | ||||
| Corporate bonds | $24,493 | $5 | $(52) | $24,446 |
| Commercial paper | 14,776 | 2 | (3) | 14,775 |
| US Government debt securities | 66,330 | 16 | (71) | 66,275 |
| Total | $105,599 | $23 | $(126) | $105,496 |
The following table summarizes the Company’s marketable securities as of December 31, 2024 (in thousands):
| Amortized Cost | Unrealized Gains | Unrealized Loss | Fair Value | |
|---|---|---|---|---|
| Marketable securities: | ||||
| Corporate bonds | $18,786 | $— | $(118) | $18,668 |
| Commercial paper | 6,947 | — | (3) | 6,944 |
| US Government debt securities | 93,158 | 94 | (124) | 93,128 |
| Total | $118,891 | $94 | $(245) | $118,740 |
As of March 31, 2025, the weighted average maturity of the Company’s marketable securities is 0.63 years.
The Company didnot record an allowance for credit losses as of March 31, 2025 related to its marketable securities. Further, given the lack of significant change in the credit risk of these investments, the Company did not recognize any other-than-temporary impairment losses.
- Accrued and other current liabilities
Accrued and other current liabilities consisted of the following (in thousands):
| March 31, | December 31, | |
|---|---|---|
| 2025 | 2024 | |
| Accrued employee compensation costs | $2,938 | $7,686 |
| Accrued professional costs | 1,424 | 1,108 |
| Accrued research and development costs | 15,548 | 6,698 |
| Current portion of operating lease liabilities | 2,351 | 2,322 |
| Other current liabilities | 2,074 | 753 |
| $24,335 | $18,567 |
- Commitments and contingencies
Operating lease
The Company determines whether an arrangement is a lease at inception. The Company accounts for a lease when it has the right to control the leased asset for a period of time while obtaining substantially all of the assets’ economic benefits. Operating lease right-of-use assets and operating lease liabilities are recognized based on the present value of the future minimum lease payments over the lease term at the lease commencement date. The discount rate used to determine the present value of the lease payments is the Company’s incremental borrowing rate based on the information available at lease inception, as the Company did not have information to determine the rate implicit in the leases. Lease expense for operating leases is recognized on a straight-line basis over the reasonably assured lease term based on the total lease payments (which include initial direct costs and lease incentives). The expense is included in operating expenses in the consolidated statements of operations and comprehensive loss. The Company’s lease agreements also contain variable payments, primarily maintenance-related costs, which are expensed as incurred and not included in the measurement of the right-of-use assets and lease liabilities.
In August 2018, the Company entered into an agreement to lease approximately 23,000 square feet of space for a term ofthree years. Lease terms are triple net lease commencing at $0.9 million per year, then with 3% annual base rent increases plus operating expenses, real estate taxes, utilities and janitorial fees. The lease commencement date was December 10, 2018.
In September 2021, the Company entered into an agreement to extend the initial term of the 23,000 square foot lease for a period ofthree years commencing on December 15, 2021 and ending December 31, 2024. In addition, this lease provides for the lease of an additional 15,000 square feet of rentable space beginning on April 1, 2022 and ending on December 31, 2024. In December 2021, the Company recognized a right-of-use asset and operating lease liability of $3.5 million for the 23,000 square feet. On April 1, 2022, the Company recognized a right-of-use asset and operating lease liability of $1.8 million for the 15,000 square feet.
In December 2023, the Company entered into an agreement to extend the term of the 38,000 square foot lease for a period oftwo years commencing on January 1, 2025 and ending on December 31, 2026. In December 2023, the Company recognized a right-of-use asset and operating lease liability of $4.1 million.
In December 2018, the Company entered into an agreement to lease 2,485 square feet of space for an initial term ofthree years. The lease includesone renewal option for an additionaltwo years; however, any time after the initial term the landlord may relocate the Company from the premises to a space reasonably comparable in size and utility. As the Company does not have the right to control the use of the identified asset after the initial term, the renewal option was excluded from the lease liability calculation. Lease terms commence at $0.2 million per annum, with 2.5% annual base rent increases plus operating expenses, real estate taxes, utilities and janitorial fees. The lease commencement date was May 1, 2019.
In June 2021, the Company amended the agreement toextend the initial term of the 2,485 square foot lease for a period ofthree years commencing May 1, 2022 and ending April 30, 2025. In addition, the amendment provided for the lease of an additional 2,357 square feet of rentable space beginning on July 6, 2021 and ending on April 30, 2025. In March 2025, the Company further amended this lease to extend the term from April 30, 2025 to June 30, 2025. The amended lease provides the Company with the option to extend the term of the lease for an additionaltwo years. In 2021, the Company recognized a right-of-use asset and operating lease liabilities of $0.7 million for the extension of the lease to April 30, 2025 and a right-of-use asset and operating lease liabilities of $0.8 million for the additional 2,357 square feet of rentable space.
In January 2025, the Company entered into an agreement to lease 7,581 square feet of space for an initial term ofthree years and three months. The lease includestwo renewal options for an additional three years each. Lease terms commence at $0.7 million per annum, with 2.0% annual base rent increases plus operating expenses, real estate taxes, and utilities. The lease commencement date is expected to be July 1, 2025. As the Company did not have access to the space, a right-of-use asset wasnot recognized as of March 31, 2025.
Future minimum lease payments under non-cancellable leases as of March 31, 2025 were as follows (in thousands):
| 2025 | $2,167 |
| 2026 | 3,250 |
| 2027 | 699 |
| 2028 | 532 |
| Total lease payments | 6,648 |
| Less imputed interest | (698) |
| Present value of lease liabilities | $5,950 |
Lease balances as of March 31, 2025 were as follows (in thousands):
| Operating right-of-use assets | $3,831 |
|---|---|
| Current Portion of operating lease liabilities | $2,351 |
| Non-current portion of operating lease liabilities | 1,861 |
| Total operating lease liabilities | $4,212 |
The weighted average remaining lease term and weighted average discount rate of the Company’s operating leases as of March 31, 2025 were as follows:
| Weighted average remaining lease term in years | 2.3 |
|---|---|
| Weighted average discount rate | 10.25% |
Lease expense incurred under operating leases was $0.7 million for the three months ended March 31, 2025 and was $0.7 million for the three months ended March 31, 2024.
License and research agreements
In April 2016, the Company entered into an exclusive, worldwide license agreement with the University of Southampton (the “Southampton Agreement”), whereby the Company acquired rights to foundational technologies related to the Company’s TANGO technology. Under the Southampton Agreement, the Company receives an exclusive, worldwide license under certain licensed patents and applications relating to TANGO. Under the Southampton Agreement, the Company may be obligated to make additional payments that are contingent upon certain milestones being achieved, as well as royalties on future product sales. These royalty obligations survive until the latest of (i) the expiration of the last valid claim of a licensed patent covering a subject product or (ii) the expiration of any regulatory exclusivity for the subject product in a country. In addition, if the Company sublicenses its rights under the Southampton Agreement, the Company is required to pay a mid-single digit percentage of the sublicense revenue to the University of Southampton. As of March 31, 2025, the Company had paid $0.7 million under the Southampton Agreement as a result of entering into the Acadia Pharmaceuticals Inc. license and collaboration agreement in January 2022 and had recorded a liability of $8.2 million, for a sublicense fee expense, due to the Biogen license and collaboration agreement (see Note 7). Additionally, certain licenses under the Southampton Agreement require the Company to reimburse the University of Southampton for certain past and ongoing patent related expenses. For the three months ended March 31, 2025 these expenses were $0.05 million compared to $0.03 million for the three months ended March 31, 2024.
- License and collaboration agreements
Acadia Pharmaceuticals Inc.
In January 2022, the Company entered into a License and Collaboration Agreement (the “Acadia Agreement”) with Acadia Pharmaceuticals Inc. (“Acadia”) for the discovery, development and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system. The Acadia Agreement focuses on the targets SYNGAP1, MECP2 (Rett syndrome), and an undisclosed neurodevelopmental target of mutual interest. In connection with each target, the Company will collaborate with Acadia to identify potential treatments for further development and commercialization as licensed products. With respect to SYNGAP1, the Company has agreed with Acadia to co-develop and co-commercialize licensed products for such target globally, and in connection therewith the Company granted to Acadia worldwide, co-exclusive (with the Company) licenses for such licensed products. With respect to MECP2 and the neurodevelopmental target, the Company granted to Acadia worldwide, exclusive licenses to develop and commercialize licensed products for such targets.
Pursuant to the terms of the Acadia Agreement, the Company received an upfront payment of $60.0 million from Acadia. Acadia agreed to fund the research to identify potential licensed products for MECP2 and the neurodevelopmental target, and the Company will equally fund with Acadia the research to identify potential licensed products for SYNGAP1. The Company is eligible to receive up to $907.5 million in potential total milestone payments based upon the achievement of certain development, regulatory, first commercial sales and sales milestone events across the programs for the three targets, assuming each milestone were achieved at least once. With respect to licensed products for MECP2 and the neurodevelopmental target, the Company is also eligible to receive tiered royalties at percentages ranging from the mid-single digits to the mid-teens on future net sales by Acadia of licensed products worldwide. Royalties payable under the Acadia Agreement are subject to standard royalty reductions. For SYNGAP1 licensed products that the Company is co-developing and co-commercializing, the Company will be responsible for 50% of the development and commercialization costs and will receive 50% of the profits from global commercialization. The Company is provided with a co-development and co-commercialization opt out option relating to the SYNGAP1 target indication at the Company’s discretion. Such opt-out would reduce development and commercialization milestones but would provide the Company with royalties on an escalating basis attributable to net sales milestones.
Acadia Agreement accounting
At the commencement of the Acadia Agreement, the Company identified three performance obligations consisting of pre-clinical research activities for each of the three targets, SYNGAP1, MECP2, and the undisclosed neurodevelopmental target. The exclusive or co-exclusive licenses granted to Acadia to conduct pre-clinical research activities on each of the three targets, and participation on each of the respective joint research committees were identified as promised services. However, the licenses granted to Acadia and the research activities were determined to be not distinct from each other, and therefore are considered a combined performance obligation for each of the three targets. Participation on each of the joint research committees was determined to be quantitatively and qualitatively immaterial in the context of the arrangement with Acadia.
The Company is recognizing the transaction price for the pre-clinical research activities for each of the three targets over time as the research services are provided. The transfer of control to Acadia occurs over this time period, and in management’s judgment, is the best measure of progress towards satisfying the performance obligation. An input method is used that measures the cost incurred to date in satisfying each of the three research activities in relation to the estimated total projected cost of each of the research activities to fulfill the respective obligations. The cumulative effect of revisions to estimated costs and/or the transaction price to complete the research performance obligations will be recorded in the period in which changes are identified and amounts can be reasonably estimated.
Milestone payments that are not within the control of the Company, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. For other milestones, the Company evaluated factors such as the scientific, clinical, regulatory, commercial, and other risks that must be overcome to achieve the particular milestone in making this assessment. Milestones that are outside of the Company’s or Acadia’s control will not be recognized until such milestones are achieved. As to the other milestones, to date, no milestone payments have been included in the transaction price due to the uncertainty as to whether these milestones will be achieved. The Company will at the end of each reporting period reevaluate the probability of achievement of all milestones subject to constraint and, if necessary, adjust its estimate of the overall transaction price for each of the research activities on the three targets. Any such adjustments will be recorded on a cumulative catch-up basis.
As of March 31, 2025, the Company had $14.4 million in upfront consideration associated with the Acadia Agreement relating to performance obligations that are unsatisfied or partially unsatisfied.
Biogen International GmbH License and Collaboration Agreement
On February 14, 2025 (the “Effective Date”), the Company entered into a License and Collaboration Agreement (the “Biogen Agreement”) with Biogen International GmbH (“Biogen”) for the joint development and commercialization of zorevunersen and other compounds targeting the SCN1A gene (the “Licensed Product”). Under the terms of the Biogen Agreement, the Company granted Biogen an exclusive, royalty-bearing license to develop, manufacture, and export licensed products in all territories outside the U.S., Canada, and Mexico (the “Biogen Territory”). In addition, Biogen has the option to exercise an exclusive, royalty-bearing, sublicensable and transferable license for certain future follow-on ASOs directed to SCN1A controlled by the Company.
Under the Biogen Agreement, the parties are jointly developing zorevunersen, with the Company leading global development. The Company and Biogen share global development costs based on an agreed budget. The Company is responsible for 70.0% of these development costs and Biogen is responsible for the remaining 30.0% of these development costs.
In connection with the closing of this transaction in February 2025, the Company received an upfront payment of $165.0 million and is eligible to receive development and commercial milestone payments that could total up to approximately $50.0 million and $335.0 million, respectively, if all the specified milestones set forth in this collaboration are achieved. In addition, the Company is eligible to receive tiered, double-digit royalties ranging from the low double-digit to high teen percentages of future net sales on the licensed products. Royalties payable under the Agreement are subject to standard royalty reductions.
Biogen agreement accounting
At the commencement of the Agreement, the Company evaluated the Biogen Agreement and determined that it was reflective of a vendor-customer relationship and comprised of the following performance obligations within the scope of ASC 606.
As of the Effective Date, the Company identified the following performance obligations in the Biogen Agreement that were evaluated under the scope of ASC 606: (i) an exclusive royalty-bearing sublicensable, transferable license under the licensed intellectual property to exploit Licensed Products in the Biogen Territory, and non-exclusive, royalty-free, sublicensable, transferable license under the licensed intellectual property to develop, manufacture and export any Licensed Products in the Biogen Territory (collectively “IP License” and considered as one performance obligation), and (ii) development services, that are expected to generate data necessary for regulatory approval in the Biogen Territory for the licensed products (“Global Development Activities”).
The IP License is considered functional intellectual property and distinct from the Global Development Activities, as Biogen can benefit from the license on its own or together with other readily available resources. Global Development Activities are considered distinct as they are not expected to significantly modify or customize the initial intellectual property as the Licensed Product is in Phase 3 of clinical development at contract inception.
The Company determined that the initial transaction price under ASC 606 was $243.4 million and is comprised of the non-refundable upfront payment of $165 million and $78.4 million of estimated variable consideration attributed to cost reimbursement in reimbursement for Global Development Activities. The Company utilized the expected value method to estimate the variable consideration for the future reimbursement of the Global Development Activities, and any subsequent adjustments arising from changes in estimates will be recorded on a cumulative catch-up basis in the period of such adjustment.
The Company determined that royalties and sales milestones relate solely to the licenses of intellectual property and are therefore excluded from the transaction price under the sales- or usage-based royalty exception of ASC 606. In addition, the Company estimated the probability, likelihood, and magnitude of a reversal of revenue for the commercial milestone payments and determined that any variable consideration related to commercial milestones was deemed to be fully constrained at inception and therefore excluded from the initial transaction price due to high degree of uncertainty and risks associated with these potential payments as the Company can not assert that it is probable that a significant reversal in the amount of cumulative revenue recognized would not occur.
The Company developed the estimated standalone selling price, at inception, for each of the performance obligations with the objective of determining the price at which we would sell each item if it were to be sold regularly on a standalone basis. The Company developed the estimated standalone selling price for the IP License primarily based on the probability-weighted present value of expected future cash flows associated with the underlying license or activity. In developing such estimates, the Company applied judgment in determining the forecasted revenues, taking into consideration the applicable market conditions and relevant entity-specific factors, the probability of success and the discount rate. The estimated standalone selling price for the Global Development Activities is primarily based on the costs necessary to perform the service plus a reasonable margin. In management’s judgment the above inputs provide a faithful estimate and reflects a consideration that would be received to transfer the promised goods or services in an orderly transaction between unrelated market participants. As such, the estimated standalone selling price reflects the best estimate of the fair value that market participants would pay for each performance obligation on a standalone basis.
The Company allocated the transaction price to the performance obligations on a relative stand-alone selling price basis consistent with the allocation objective of ASC 606.
The IP License performance obligation was satisfied at a point in time upon transfer of the IP License to Biogen. Control of the IP License was transferred on the Effective Date as Biogen could begin to use and benefit from the IP License immediately. For the Global Development Activities performance obligation, the Company measures proportional performance over time using an input method based on costs incurred relative to the total estimated costs of the obligation by determining the proportion of costs incurred as a percentage of total costs the Company expects to expend. This ratio is applied to the transaction price allocated to this performance obligation to arrive at the cumulative revenue that should be recognized at each reporting date. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition.
The following table provides a summary of the transaction price allocated to each performance obligation, in addition to revenue activity during the period:
| Transaction Price Allocated | Revenue Recognized | |
|---|---|---|
| Performance obligations | ||
| IP License | $150,779 | $150,779 |
| Global Development Activity | 92,644 | 1,642 |
| $243,423 | $152,421 | |
As of March 31, 2025 the Company had $14.0 million in upfront consideration associated with the Biogen Agreement relating to performance obligations that are unsatisfied or partially unsatisfied.
- Equity incentive plans
In June 2019, the Company’s board of directors and stockholders approved the 2019 Equity Incentive Plan (the “2019 Plan”) which became effective on June 17, 2019 and replaced the Company’s 2014 Equity Incentive Plan (the “2014 Plan”). In addition to the shares of common stock reserved for future issuance under the 2014 Plan that were added to the 2019 Plan upon its effective date, the Company initially reserved 2,200,000 shares of common stock for issuance under the 2019 Plan. The number of shares reserved for issuance under the 2019 Plan will increase automatically on January 1 of each of 2020 through 2029 by the number of shares equal to 4% of the aggregate number of outstanding shares of the Company’s common stock as of the immediately preceding December 31, or a lesser number as may be determined by the Company’s board of directors.
In April 2023, the Company’s board of directors adopted the Stoke Therapeutics, Inc. 2023 Inducement Plan (the “2023 Plan”). As permitted by Nasdaq stock market rules, the Company’s stockholders were not required to approve the 2023 Plan. The 2023 Plan provides for up to 1,000,000 shares of the Company’s common stock under awards granted to newly hired employees. An “award” is any right to receive common stock of the Company through nonstatutory stock options or restricted stock units (“RSUs”).
As of March 31, 2025, there wereno shares available for future issuance under the 2014 Plan,1,875,367 shares were available under the 2019 Plan and 31,800 shares were available under the 2023 Plan.
During the three months ended March 31, 2025, the Company granted options to purchase 1,959,171 shares of common stock to certain of its employees. The options vest over a period of up tofour years and are exercisable at a per share price equal to the fair value of the common stock on the grant date. During the three months ended March 31, 2025, the Company granted 1,007,842 RSUs and didnot grant any performance stock units ("PSUs") to its employees. The RSUs vest over a period of up tofour years.
Stock-based compensation
As of March 31, 2025, there was $38.6 million of unrecognized compensation cost related to unvested stock options granted under the 2019 and 2023 Plans. Compensation expense is expected to be recognized over a weighted average period of 2.8 years as of March 31, 2025. As of March 31, 2025, there was $23.7 million of unrecognized stock-based compensation related to RSUs and $1.5 million of unrecognized stock-based compensation related to PSUs. The compensation expense for RSUs and PSUs is expected to be recognized over a weighted average period of 2.9 years.
Stock-based compensation expense recorded as research and development and general and administrative expenses in the accompanying consolidated statements of operations and comprehensive loss is as follows (in thousands):
| Three Months Ended March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Research and development | $2,799 | $2,091 |
| General and administrative | 3,954 | 3,319 |
| Total | $6,753 | $5,410 |
2019 Employee stock purchase plan
In June 2019, the Company adopted the 2019 Employee Stock Purchase Plan (the “ESPP”), which became effective on June 18, 2019. The Company initially reserved 315,000 shares of common stock for sale under the ESPP. At March 31, 2025, the Company had 2,131,930 shares available for issuance under the ESPP. The average grant date fair value per share under the ESPP was $11.27 for 2025. The total ESPP stock-based compensation expense for the three months ended March 31, 2025 was $0.2 million, and for the three months ended March 31, 2024 was $0.1 million. The number of shares reserved for issuance under the ESPP will increase automatically on January 1 of each of the first ten calendar years following the first offering date by the number of shares equal to the lesser of 1% of the total outstanding shares of the Company’s common stock as of the immediately preceding December 31 or a lower amount determined by the Company’s board of directors. The aggregate number of shares issued over the term of the ESPP will not exceed 3,150,000 shares of the Company’s common stock.
- Net income (loss) per share
The following table summarizes the computation of basic and diluted net loss per share of the Company (in thousands except share and per share amounts):
| Three Months Ended March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Numerator: | ||
| Net income (loss) | $112,879 | $(26,374) |
| Denominator: | ||
| Weighted-average number of common shares, basic | 57,862,674 | 46,246,889 |
| Weighted-average number of common shares, diluted | 59,398,600 | 46,246,889 |
| Net loss per share, basic | $1.95 | $(0.57) |
| Net loss per share, diluted | $1.90 | $(0.57) |
As the Company had net income during the three months ended March 31, 2025, the Company’s potential dilutive securities, which include stock options, restricted stock units and performance stock units, were included in the calculation of diluted net income per share attributable to common stockholders. For the three months ended March 31, 2024, the Company’s potential dilutive securities, which include stock options, restricted stock units and performance stock units have been excluded from the computation of diluted net loss per share as the effect would be to reduce the net loss per share. Therefore, the weighted-average number of common stock outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same for the three months ended March 31, 2024.
The Company excluded the following potential common stock, presented based on amounts outstanding at March 31, 2025 and 2024 from the computation of diluted net loss per share attributable to common stockholders because including them would have had an anti-dilutive effect.
| March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Outstanding options to purchase common stock | 7,214,876 | 7,439,396 |
| Restricted stock units | 589,219 | 2,410,752 |
| Performance stock units | 73,000 | — |
| Total | 7,877,095 | 9,850,148 |
- Income taxes
For the three months ended March 31, 2025, the Company recorded federal and state tax expense of $1.1 million and $0.1 million, respectively due to recognizing income from the Biogen collaboration, offset by net operating loss carryforwards (NOLs) that were able to be utilized to the extent allowable by tax law. The Company didnot record an income tax expense in its consolidated statements of operations and comprehensive loss for the three months ended March 31, 2024. The Company has provided a valuation allowance for the full amount of its net deferred tax assets as of March 31, 2025 and December 31, 2024, as management has determined it is more likely than not that any future benefit from deductible temporary differences and net operating loss and tax credit carryforwards would not be realized.
- Segment Information
The Company comprises ofone reportable segment, Stoke. As of March 31, 2025, this segment has not generated any product revenue since inception, as it does not yet have approved products for sale.
The Company primarily generates revenue in North America, with its long-lived assets also concentrated in this region, and manages its business activities on a consolidated basis. Decisions concerning the allocation of the Company’s resources are made by the Company’s Chief Operating Decision Maker (“CODM”), which is the Company’s Interim Chief Executive Officer. The CODM views the Company’s operations as a single operating segment which is the business of discovering and developing RNA-based medicines, using the Targeted Augmentation of Nuclear Gene Output (“TANGO”) approach and developing antisense oligonucleotides (“ASOs”) to selectively restore protein levels.
The CODM assesses performance for the Company's operations segment and decides how to allocate resources based on net income or loss that is also reported on the income statement as consolidated net income or loss and the measure of segment assets as reported on the balance sheet as total consolidated assets. Net income or loss is used to monitor budget vs actual results as well as assess the general performance of the segment in a given period.
The following table reconciles segment direct profit or loss to the Company’s consolidated results (in thousands):
| Three Months Ended March 31, | ||
|---|---|---|
| 2025 | 2024 | |
| Revenue | $158,569 | $4,216 |
| Zorevunersen(1) | 17,674 | 8,780 |
| ADOA(1) | 1,201 | 1,722 |
| SYNGAP1(1) | 342 | 284 |
| MECP2(1) | 147 | 242 |
| Other external program costs | 1,829 | 2,043 |
| Payroll and other personnel costs - R&D (2) | 11,012 | 8,163 |
| Payroll and other personnel costs - G&A (2) | 8,929 | 6,850 |
| Other non-program costs - R&D (3) | 1,407 | 1,460 |
| Other non-program costs - G&A (3) | 4,788 | 3,044 |
| Subtotal | (47,329) | (32,588) |
| Other segment items | ||
| Interest expense | (1) | (22) |
| Interest income | 2,890 | 2,448 |
| Other income (expense) | 28 | (428) |
| Provision for income taxes | (1,278) | — |
| Total other segment items | 1,639 | 1,998 |
| Segment net income (loss) | $112,879 | $(26,374) |
(1)Includes external research and development and general and administrative expenses associated with programs.
(2)Includes salaries, benefits and other personnel related expenses, including stock based compensation expense, of relevant personnel.
(3)Includes costs incurred for conducting non-program activities related to Company activities.
- Subsequent events
In May 2025, the Company received notice from Acadia that it had elected to discontinuetwo of thethree research programs under the Acadia Agreement. Under the Acadia Agreement, the licenses granted to Acadia for the discontinued programs, which are the MECP2 program for Rett syndrome and the program for an undisclosed neurodevelopmental target, will terminate and the parties will wind down Acadia’s funding for the two programs.
The text of this filing, as the company filed it with the SEC, without its images or exhibits. This is not investment advice.