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Sarepta Therapeutics Inc

For the period ended Mar 31, 2025. Filed May 6, 2025.

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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 6, 2025

SRPT$17.82+1.4%Stored close · as of Nov 6, 2026

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Notes to Condensed Consolidated Financial Statements

(unaudited)

  1. ORGANIZATION AND NATURE OF BUSINESS

Sarepta Therapeutics, Inc. (together with its wholly-owned subsidiaries, “Sarepta” or the “Company”) is a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying its proprietary, highly-differentiated and innovative technologies, and through collaborations with its strategic partners, the Company has developed multiple approved products for the treatment of Duchenne muscular dystrophy (“Duchenne”) and is developing potential therapeutic candidates for a broad range of diseases and disorders, including Duchenne, Limb-girdle muscular dystrophies (“LGMDs”) and other neuromuscular and central nervous system (“CNS”) disorders.

The Company's products in the U.S., EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”) and AMONDYS 45 (casimersen) Injection (“AMONDYS 45”), were granted accelerated approval by the U.S. Food and Drug Administration (the “FDA”) in 2016, 2019 and 2021, respectively. Indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51, exon 53 and exon 45 skipping, respectively, EXONDYS 51, VYONDYS 53 and AMONDYS 45 (collectively, the “PMO Products”) use the Company’s phosphorodiamidate morpholino oligomer (“PMO”) chemistry and exon-skipping technology to skip exon 51, exon 53 and exon 45 of the dystrophin gene. Exon skipping is intended to promote the production of an internally truncated but functional dystrophin protein.

ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-(“AAV”) based gene therapy, was approved by the FDA on June 20, 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene, as well as for non-ambulatory patients under the accelerated approval pathway. ELEVIDYS was previously granted accelerated approval by the FDA in June 2023 for the treatment of ambulatory patients aged four through five years with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene.

As of March 31, 2025, the Company had $647.5 million of cash, cash equivalents, restricted cash and investments, consisting of $240.9 million of cash and cash equivalents, $391.0 million of investments and $15.6 million ofnon-current restricted cash. The Company believes that its balance of cash, cash equivalents and investments as of the date of the issuance of this report, along with cash inflows from operations and availability under the Company’s Revolving Credit Facility (defined below), is sufficient to fund its current operational plan for at least the next twelve months, though it may pursue additional cash resources through public or private debt and equity financings, seek funded research and development arrangements and additional government contracts and establish collaborations with or license its technology to other companies.

  1. SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES

Basis of Presentation

The accompanying unaudited condensed consolidated financial statements, which have been prepared in accordance with accounting principles generally accepted in the United States (“U.S. GAAP”), reflect the accounts of Sarepta and its wholly-owned subsidiaries. All intercompany transactions between and among its consolidated subsidiaries have been eliminated. Management has determined that the Company operates inone segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases.

In the opinion of the Company’s management, all adjustments of a normal recurring nature necessary for a fair presentation have been reflected. Certain financial information that is normally included in annual financial statements prepared in accordance with U.S. GAAP, but that is not required for interim reporting purposes, has been omitted. These unaudited condensed consolidated financial statements should be read in conjunction with the consolidated financial statements and related notes for the year ended December 31, 2024 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission on February 28, 2025. The results for the three months ended March 31, 2025 are not necessarily indicative of the results to be expected for the full year.

Estimates and Uncertainties

The preparation of the unaudited condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates and assumptions that affect the reported amounts of assets, liabilities, equity, revenues, expenses and the disclosure of contingent assets and liabilities. Actual results could differ from those estimates.

Concentration of Credit Risk

Financial instruments that potentially subject the Company to concentrations of credit risk consist of cash held at financial institutions, cash equivalents, investments and accounts receivable, net, from customers. As of March 31, 2025, the Company’s cash was concentrated at three financial institutions, which potentially exposes the Company to credit risks. However, the Company does not believe that there is significant risk of non-performance by the financial institutions. The Company also purchases commercial paper, government and government agency bonds, corporate bonds and certificates of deposit issued by highly rated corporations, financial institutions and governments and limits the amount of credit exposure to any one issuer. These amounts may at times exceed federally insured limits. The Company has not experienced any credit losses related to these financial instruments and does not believe to be exposed to any significant credit risk related to these instruments. As of March 31, 2025, three entities accounted for 56%,17% and 13% of accounts receivable, net, respectively. As of December 31, 2024, three entities accounted for 54%,21% and 13% of accounts receivable, net, respectively.

Significant Accounting Policies

For details about the Company's accounting policies, please read Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncementsof the Annual Report on Form 10-K for the year ended December 31, 2024.

Acquired in-process research and development

Acquired in-process research and development (“IPR&D”) is recognized at fair value and initially characterized as an indefinite-lived intangible asset, irrespective of whether the acquired IPR&D has an alternative future use. If the acquired net assets do not constitute a business under the acquisition method of accounting, the transaction is accounted for as an asset acquisition and no goodwill is recognized. In an asset acquisition, the amount allocated to acquired IPR&D with no alternative future use is recorded as an expense at the acquisition date.

There have not been any other material changes to the Company's accounting policies or recent accounting pronouncements that could have a material impact through March 31, 2025.

  1. LICENSE AND COLLABORATION AGREEMENTS

License Agreement with Arrowhead Pharmaceuticals, Inc.

On November 25, 2024, the Company and Arrowhead Pharmaceuticals, Inc. (“Arrowhead”) entered into an exclusive global license and collaboration agreement and a stock purchase agreement (collectively, the “Arrowhead Agreement”), which became effective as of February 7, 2025 (the “Effective Date”) upon the expiration or termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976.

The Arrowhead Agreement granted the Company an exclusive license under certain of Arrowhead’s intellectual property rights to develop, manufacture and commercialize lead candidate (and all backup candidates) for the following programs:

Development StageIndication
Arrowhead Clinical Programs(a) DUX4 for the treatment of facioscapulohumeral muscular dystrophy,(b) DMPK for the treatment of type 1 myotonic dystrophy,(c) ATXN2 for the treatment of ataxias, and (d) MMP7 for the treatment of idiopathic pulmonary fibrosis.
Arrowhead Pre-clinical Programs(a) ATXN1 for the treatment of ataxias, (b) ATXN3 for the treatment of ataxias, and (c) HTT for the treatment of Huntington’s Disease.

In addition, the parties will collaborate on the discovery and development of compounds that are directed to six targets to be selected by the Company during the term (each an “Arrowhead Discovery Program,” and together with the Arrowhead Clinical Programs and Pre-clinical Programs, the “Arrowhead Programs”).

The development and manufacturing activities under the Arrowhead Agreement are governed by a series of committees established to facilitate transition and collaboration between the two parties.

Under the terms of the Arrowhead Agreement, Arrowhead will conduct development activities with respect to the Arrowhead Programs pursuant to agreed-upon development plans. At pre-determined transition points for each Arrowhead Program, Arrowhead will transfer development responsibility to the Company, who will then perform all development activities necessary to obtain and maintain regulatory approvals throughout the world. The Company will have the sole worldwide right to commercialize licensed products.

The Company will reimburse Arrowhead for certain pre-determined development activities for the Arrowhead Clinical Programs. Each party is responsible for the costs and expenses of other development activities under the Arrowhead Agreement. Arrowhead will complete all manufacturing activities necessary for their development activities and provide clinical supply for all Arrowhead Programs and commercial supply for the Arrowhead Clinical Programs. The parties will determine at a later date whether Arrowhead will provide commercial supply for the Arrowhead Pre-clinical Programs and Arrowhead Discovery Programs. Upon the occurrence of certain conditions, Arrowhead will transfer control of manufacturing and supply to the Company.

In connection with the Arrowhead Agreement, Arrowhead appointed the Company’s Chief Executive Officer (“CEO”), Douglas Ingram, as a director, effective February 2025.

When the Arrowhead Agreement became effective, the Company paid Arrowhead an up-front payment of $500.0 million and invested $325.0 millionin approximately 11.9 million shares of Arrowhead's common stock at a premium to the valuation on the closing date. The Company is contractually restricted from selling its investment in Arrowhead common stock until August 2025. Based on the closing price of Arrowhead’s common stock traded on the Nasdaq Global Select Market (“Nasdaq”) on the Effective Date, $241.4 million was allocated to the equity investment in Arrowhead and recorded within strategic investments on the Company’s unaudited condensed consolidated balance sheets. For the three months ended March 31, 2025, the Company recorded $583.6 million in research and development expense, as the remainder of the up-front payments is allocated to the up-front license fee representing rights to potential future benefits associated with research and development activities that have no alternative future use. The Company concluded that it does not have a controlling financial interest in Arrowhead as the Company does not have the power to direct the activities that would most significantly impact the economic performance of Arrowhead. Additionally, the Company concluded that, as it does not have significant influence over Arrowhead, the Company’s investment in Arrowhead is not subject to the equity method of accounting. Accordingly, the investment in Arrowhead’s common stock is recorded at fair value and remeasured each reporting period with changes recorded to other (expense) income, net in the Company’s unaudited condensed consolidated statements of comprehensive (loss) income. For the three months ended March 31, 2025, the Company recognized a loss of $89.5 million related to the change in fair value of this strategic investment. The Arrowhead Agreement also provides the Company with the option to exchange its holding of Arrowhead’s common stock into pre-funded warrants (“Warrants”) with an exercise price of $0.001 per Warrant share. The Company currently does not have any plan to exercise this option. For the three months ended March 31, 2025, the Company recorded $2.3 million of research and development expense related to reimbursable development costs incurred by Arrowhead.

Additionally, the Company will pay Arrowhead an aggregate total of $250.0 million in annual installments of $50.0 million over five years (“Annual Fees”), with the first payment due on the first anniversary of the Effective Date. The Annual Fees are contingent upon the Company not terminating the Arrowhead Agreement. The Company may be obligated to make payments to Arrowhead totaling up to $10.3 billion upon the achievement of certain development, regulatory and sales milestones, inclusive of $300.0 million in near-term payments associated with the continued enrollment of certain cohorts of a Phase 1/2 study for an individual program. Furthermore, upon commercialization, the Company will be required to make tiered royalty payments based on net sales.

Collaboration Agreement with F. Hoffman-La Roche Ltd.

For the three months ended March 31, 2025 and March 31, 2024, the Company recognized $112.0 million and $48.0 million, respectively, of collaboration revenue associated with the license, collaboration and option agreement (the “Roche Collaboration Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). Under the Roche Collaboration Agreement, the Company has granted Roche options to acquire ex-U.S. rights to certain future Duchenne development programs (the “Options”) in exchange for separate option exercise payments, milestone and royalty considerations, and cost-sharing provisions. These Options were accounted for as material rights related to individual programs and recorded in deferred revenue at the inception of the Roche Collaboration Agreement. The value assigned to the material rights is included in deferred revenue until such rights expire or are exercised. In February 2025, an Option for a certain program expired, which resulted in the immediate recognition of $112.0 million of collaboration revenue for the three months ended March 31, 2025. In February 2024, Roche declined to exercise its option related to one external, early-stage development program, which resulted in the immediate recognition of $48.0 million of collaboration revenue for the three months ended March 31, 2024. As of March 31, 2025 and December 31, 2024, the Company had total deferred revenue of $369.9 million and $455.3 million, primarily related to the separate material rights for the Options associated with the Roche Collaboration Agreement, of which $44.9 million and $130.3 million are classified as current, respectively.

In accordance with the Roche Collaboration Agreement, the parties agreed to enter into a supply agreement in order to supply them with clinical and commercial batches of ELEVIDYS (the “Roche Supply Agreement”). Roche utilizes the supply for sales of ELEVIDYS in territories outside of the U.S. where Roche has received certain approvals for ELEVIDYS. While the Roche Supply Agreement is in the process of being negotiated at the issuance of this report, the Company delivered commercial ELEVIDYS supply to Roche that were agreed upon on a purchase order-by-purchase order basis. Contract manufacturing revenue and royalty revenue are included in collaboration and other revenues in the unaudited condensed consolidated statements of comprehensive (loss) income. The following table summarizes certain Roche activity for each of the periods indicated:

For the Three Months Ended March 31,
20252024
(in thousands)
Contract manufacturing revenue$17,379$5,807
Royalty revenue3,954173
Cost of sales (inventory costs related to products sold to Roche)(12,143)(1,654)

The costs associated with co-development activities performed under the Roche Collaboration Agreement are included in operating expenses, with any reimbursement of costs by Roche reflected as a reduction of such expenses when the related expense is incurred. For the three months ended March 31, 2025 and 2024, costs reimbursable by Roche and reflected as a reduction to operating expenses were $28.5 million and $21.7 million, respectively. As of March 31, 2025 and December 31, 2024, there was $55.2 million and $34.6 million of collaboration and other receivables included in other current assets on the unaudited condensed consolidated balance sheets, respectively.

Research and Option Agreements

The Company has research and option agreements with third parties in order to develop various technologies and biologics that may be used in the administration of the Company’s genetic therapeutics. The agreements generally provide for research services related to pre-clinical development programs and options to license the technology for clinical development. Prior to the options under these agreements being executed, the Company may be required to make up to $30.8 million in research milestone payments. Under these agreements, there are $111.8 million in potential option payments to be made by the Company upon the determination to exercise the options. Additionally, if the options for each agreement are executed, the Company would incur additional contingent obligations and may be required to make development, regulatory, and sales milestone payments and royalty payments based on the net sales of the developed products upon commercialization. During the three months ended March 31, 2025 and 2024, the Company did not exercise any options or have any additional research milestone payments become probable of occurring.

Milestone Obligations

Including the agreements discussed above, the Company has license and collaboration agreements in place for which it could be obligated to pay, in addition to the payment of up-front fees upon execution of the agreements, certain milestone payments as a product candidate proceeds from the submission of an investigational new drug application through approval for commercial sale and beyond. As of March 31, 2025, the Company may be obligated to make up to $12.6 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements. These obligations exclude potential future option and milestone payments for options that have yet to be exercised within agreements entered into by the Company as of March 31, 2025, which are discussed above. For the three months ended March 31, 2025, the Company recognized up-front and development milestone expenses of $583.8 million as research and development expense in the accompanying unaudited condensed consolidated statements of comprehensive (loss) income, withno similar activity for the three months ended March 31, 2024.

  1. FAIR VALUE MEASUREMENTS

The Company has certain financial assets and liabilities that are recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements.

Level 1 — quoted prices for identical instruments in active markets;

Level 2 — quoted prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active, and model-derived valuations in which all significant inputs and significant value drivers are observable in active markets; and

Level 3 — valuations derived from valuation techniques in which one or more significant value drivers are unobservable.

During the three months ended March 31, 2025, there were no transfers into or out of Level 3. The tables below present information about the Company’s financial assets and liabilities that are measured and carried at fair value and indicate the level within the fair value hierarchy of the valuation techniques it utilizes to determine such fair value:

TotalLevel 1Level 2Level 3
(in thousands)
Assets
Money market funds$41,671$41,671$—$—
Commercial paper5,475—5,475—
Government and government agency bonds328,550—328,550—
Corporate bonds68,667—68,667—
Strategic investments*154,370153,370—1,000
Certificates of deposit13,307—13,307—
Deferred compensation plan assets227227——
Total assets$612,267$195,268$415,999$1,000
Liabilities
Contingent consideration$47,400$—$—$47,400
Total liabilities$47,400$—$—$47,400
TotalLevel 1Level 2Level 3
(in thousands)
Assets
Money market funds$455,535$455,535$—$—
Government and government agency bonds279,899—279,899—
Corporate bonds93,727—93,727—
Strategic investments3,7102,710—1,000
Certificates of deposit11,319—11,319—
Total assets$844,190$458,245$384,945$1,000
Liabilities
Contingent consideration$47,400$—$—$47,400
Total liabilities$47,400$—$—$47,400

*The balance at March 31, 2025 includes investments with a fair value of $151.9 million, which are subject to a contractual sale restriction that expires in August 2025.

The Company’s assets with a fair value categorized as Level 1 within the fair value hierarchy include money market funds, the Company's strategic investments in Arrowhead and another biotechnology company, both listed on Nasdaq, and assets associated with the Company's deferred compensation plan that are held in a trust. For more information related to Arrowhead, please read Note 3, License and Collaboration Agreements. The Company's deferred compensation plan allows for certain employees and directors to defer the receipt of compensation until a later date based on the terms of the plan. The Company has recorded an asset within other non-current assets on the Company's unaudited condensed consolidated balance sheets which reflects the participants' investment elections and has been valued at daily quoted market prices.

The Company's assets with a fair value categorized as Level 2 within the fair value hierarchy consist of commercial paper, government and government agency bonds, corporate bonds and certificates of deposit. These assets have been initially valued at the transaction price and subsequently valued at the end of each reporting period. The Company uses observable market inputs to determine value, which primarily consist of reportable trades. Certain short-term investments with maturities of less than three months at the date of acquisition are presented as cash equivalents on the unaudited condensed consolidated balance sheets as of March 31, 2025 and December 31, 2024.

The Company’s assets with a fair value categorized as Level 3 within the fair value hierarchy consist of a strategic investment in a private biotechnology company whose fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. At the end of each reporting period, the fair value of the Company's strategic investments that are not listed securities are adjusted if the issuers were to issue similar or identical securities or when there is a triggering event for impairment. There were no valuation measurement events related to the fair value of the Company's Level 3 strategic investment during the three months ended March 31, 2025 or 2024, as no impairment indicators were identified nor were similar securities issued.

The Company’s contingent consideration liability with a fair value categorized as Level 3 within the fair value hierarchy relates to the regulatory-related contingent payments to Myonexus Therapeutics, Inc. (“Myonexus”) selling shareholders as well as to an academic institution under a license agreement that meets the definition of a derivative. For more information related to Myonexus, please read Note 3, License and Collaboration Agreementsto the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024. The contingent consideration liability was estimated using an income approach based on the probability-weighted expected cash flows that incorporated industry-based probability adjusted assumptions relating to the achievement of the milestone and thus the likelihood of making the payments. This fair value measurement was based upon significant inputs not observable in the market and therefore represented a Level 3 measurement. Significant changes which increase or decrease the probabilities of achieving the milestone, or shorten or lengthen the time required to achieve the milestone, would result in a corresponding increase or decrease in the fair value of the liability. At the end of each reporting period, the fair value is adjusted to reflect the most current valuation assumptions through earnings. For the three months ended March 31, 2024, the Company recorded an increase of $10.1 million to account for the change in fair value of the Company's existing contingent consideration liabilities, with no similar activity for the three months ended March 31, 2025. As of March 31, 2025, the contingent consideration was recorded as a non-current liability on the Company's unaudited condensed consolidated balance sheets.

The fair value of the Company's 1.25% convertible senior notes due on September 15, 2027 (the “2027 Notes”) is based on open market trades and is classified as Level 1 in the fair value hierarchy. As of March 31, 2025 and December 31, 2024, the fair value of the 2027 Notes was $1,068.5 million and $1,254.9 million, respectively.

The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents, accounts receivable, net and accounts payable approximated fair value because of the immediate or short-term maturity of these financial instruments.

  1. CASH, CASH EQUIVALENTS AND MARKETABLE SECURITIES

The following table summarizes the Company’s financial assets with maturities of less than 90 days from the date of purchase included in cash equivalents in the unaudited condensed consolidated balance sheets for each of the periods indicated:

As of March 31, 2025As of December 31, 2024
(in thousands)
Money market funds$41,671$455,535
Government and government agency bonds24,979—
Total$66,650$455,535

It is the Company’s policy to mitigate credit risk in its financial assets by maintaining a well-diversified portfolio that limits the amount of exposure as to maturity and investment type. The weighted average maturity of the Company's available-for-sale securities as of March 31, 2025 and December 31, 2024 was approximately eight andeleven months, respectively. All of the Company's non-current investments as of March 31, 2025 and December 31, 2024 had maturities betweenone and two years.

The following tables summarize the Company’s cash, cash equivalents, short-term investments and non-current investments for each of the periods indicated:

Amortized CostGross Unrealized GainsGross Unrealized LossesFair MarketValue
(in thousands)
Cash and money market funds$215,888$—$—$215,888
Commercial paper5,475——5,475
Government and government agency bonds328,468185(103)328,550
Corporate bonds68,67841(52)68,667
Certificates of deposit13,307——13,307
Total cash, cash equivalents and investments$631,816$226$(155)$631,887
As reported:
Cash and cash equivalents$240,867$—$—$240,867
Short-term investments281,811151(67)281,895
Non-current investments109,13875(88)109,125
Total cash, cash equivalents and investments$631,816$226$(155)$631,887
Amortized CostGross Unrealized GainsGross Unrealized LossesFair MarketValue
(in thousands)
Cash and money market funds$1,103,010$—$—$1,103,010
Government and government agency bonds280,001227(329)279,899
Corporate bonds93,81667(156)93,727
Certificates of deposit11,319——11,319
Total cash, cash equivalents and investments$1,488,146$294$(485)$1,487,955
As reported:
Cash and cash equivalents$1,103,010$—$—$1,103,010
Short-term investments251,598286(102)251,782
Non-current investments133,5388(383)133,163
Total cash, cash equivalents and investments$1,488,146$294$(485)$1,487,955
  1. PRODUCT REVENUES, NET, ACCOUNTS RECEIVABLE, NET, AND RESERVES FOR PRODUCT REVENUES

Net product revenues, which includes revenues associated with the PMO Products and ELEVIDYS, consisted of the following:

For the Three Months Ended March 31,
20252024
(in thousands)
PMO Products
United States$185,828$189,667
Rest of World50,71035,881
Total PMO product revenues, net$236,538$225,548
ELEVIDYS
United States374,985133,936
Total ELEVIDYS product revenue, net$374,985$133,936
Total product revenues, net$611,523$359,484

For the three months ended March 31, 2025 and 2024, no individual country outside the U.S. exceeded 10% of total net product revenues.

The following table summarizes the Company's net product revenues, by customer, for those customers that exceeded 10% for the periods indicated:

For the Three Months Ended March 31,
20252024
Product revenues, net
Customer 119%36%
Customer 212%20%

As of March 31, 2025 and December 31, 2024, the Company's accounts receivable, net were $659.4 million and $602.0 million, respectively, both of which were related to product sales, net of discounts and allowances. As of March 31, 2025, the majority of the Company’s accounts receivable arose from product sales in the U.S. and all customers have standard payment terms that generally require payment within 65 to 90 days. Outside of the U.S., the majority of the Company’s customers have payment terms ranging between 90 and 150 days.

The following tables summarize an analysis of the change in reserves for discounts and allowances for each of the periods indicated:

ChargebacksRebatesPrompt PayOther AccrualsTotal
(in thousands)
Balance, as of December 31, 2024$45,904$107,843$5,941$34,611$194,299
Provision73,44358,7985,55227,901165,694
Adjustments relating to prior periods—(5,416)—(625)(6,041)
Payments/credits(52,480)(44,155)(5,340)(16,401)(118,376)
Balance, as of March 31, 2025$66,867$117,070$6,153$45,486$235,576
ChargebacksRebatesPrompt PayOther AccrualsTotal
(in thousands)
Balance, as of December 31, 2023$27,486$98,194$3,831$35,261$164,772
Provision20,46635,9514,38118,96579,763
Adjustments relating to prior periods783(2,532)——(1,749)
Payments/credits(30,358)(36,623)(3,685)(16,220)(86,886)
Balance, as of March 31, 2024$18,377$94,990$4,527$38,006$155,900

The following table summarizes the total reserves above included in the Company’s unaudited condensed consolidated balance sheets for each of the periods indicated:

As of March 31, 2025As of December 31, 2024
(in thousands)
Reduction to accounts receivable, net$117,224$85,142
Component of accrued expenses118,352109,157
Total reserves$235,576$194,299
  1. INVENTORY

The following table summarizes the components of the Company’s inventory for each of the periods indicated:

As of March 31, 2025As of December 31, 2024
(in thousands)
Raw materials$298,984$280,045
Work in progress762,233610,692
Finished goods61,37347,209
Total inventory$1,122,590$937,946

No material inventory reserves existed as of March 31, 2025 or December 31, 2024. The Company classifies inventory associated with its PMO Products as non-current inventory when consumption of the inventory is expected beyond the Company's normal PMO Product inventory operating cycle of two years. Non-current inventory consists of raw materials and work in progress associated with the PMO Products.

The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated:

As of March 31, 2025As of December 31, 2024
(in thousands)
Balance sheet classification
Inventory$941,432$749,960
Non-current inventory181,158187,986
Total inventory$1,122,590$937,946
  1. OTHER ASSETS

The following table summarizes the Company’s other current assets for each of the periods indicated:

As of March 31, 2025As of December 31, 2024
(in thousands)
Collaboration and other receivables$55,216$34,608
Prepaid maintenance services16,62513,407
Prepaid clinical and pre-clinical expenses8,91410,220
Tax-related receivables and prepaids7,02613,132
Prepaid commercial expenses3,7663,371
Prepaid employee benefits3,0342,841
Interest receivable2,9111,970
Other7,98610,912
Total other current assets$105,478$90,461

The following table summarizes the Company’s other non-current assets for each of the periods indicated:

As of March 31, 2025As of December 31, 2024
(in thousands)
Manufacturing-related deposits and prepaids$27,600$25,964
Intangible assets, net26,19926,887
Restricted cash*15,57915,579
Prepaid maintenance services4,7464,381
Other6,4273,394
Total other non-current assets$80,551$76,205

*The Company had $15.6 million in restricted cash included in other non-current assets on the Company’s unaudited condensed consolidated balance sheets as of both March 31, 2025 and December 31, 2024. Restricted cash for both periods relates to (i) letters of credit established under the Company’s various property leases that serve as security for potential future default of lease payments, (ii) a letter of credit established under a certain commercial supply agreement and (iii) collateralized cash for the Company’s credit cards. The restricted cash is unavailable for withdrawal or use for general obligations.

  1. ACCRUED EXPENSES

The following table summarizes the Company’s accrued expenses for each of the periods indicated:

As of March 31, 2025As of December 31, 2024
(in thousands)
Product revenue related reserves$118,352$109,157
Accrued income taxes79,86417,391
Accrued contract manufacturing costs66,84777,842
Accrued employee compensation costs35,29691,119
Accrued clinical and pre-clinical costs27,46126,849
Accrued professional fees23,48917,691
Accrued royalties15,66316,625
Accrued fixed assets14,6605,305
Other10,01811,534
Total accrued expenses$391,650$373,513
  1. STOCK-BASED COMPENSATION

The following table summarizes the Company’s stock awards granted for each of the periods indicated:

For the Three Months Ended March 31,
2025 · 2024
Grants · WeightedAverageGrantDate FairValue · Grants · WeightedAverageGrantDate FairValue
Stock options466,176$48.49316,378$67.56
Restricted stock units1,498,184(1)$97.841,161,985(2)$128.58

(1) Included in restricted stock units (“RSUs”) for the three months ended March 31, 2025 are 120,378 shares with performance conditions which are related to the achievement of certain financial performance goals, regulatory development and approval of certain of the Company's product candidates and certain manufacturing achievements. As of March 31, 2025, none of the performance conditions were probable of being achieved. Stock options and the remaining RSUs granted during the three months ended March 31, 2025 have only service-based criteria and vest over four years.

(2) Included in RSUs for the three months ended March 31, 2024 are 88,600 shares with performance conditions(the “March 2024 PSUs”) which are related to the achievement of certain financial performance goals and regulatory approval of certain of the Company's product candidates. The expanded regulatory approval of ELEVIDYS in June 2024 resulted in the cliff-vesting of 44,300 of the shares during the year ended December 31, 2024. As of March 31, 2025, none of the remaining performance conditions associated with the March 2024 PSUs were probable of being achieved.

Stock-based Compensation Expense

For the three months ended March 31, 2025 and 2024, total stock-based compensation expense included in total expenses was $41.4 million and $40.7 million, respectively. The following table summarizes stock-based compensation expense by grant type and by function included within the unaudited condensed consolidated statements of comprehensive (loss) income:

For the Three Months Ended March 31,
20252024
(in thousands)
Stock options$12,889$16,281
Restricted stock units31,54327,527
Employee stock purchase plan2,1241,397
Subtotal$46,556$45,205
Capitalized stock-based compensation costs(5,128)(4,513)
Total stock-based compensation expense included in expenses$41,428$40,692
Research and development$17,317$16,273
Selling, general and administrative24,11124,419
Total stock-based compensation expense included in expenses$41,428$40,692
  1. OTHER (LOSS) INCOME, NET

The following table summarizes other (loss) income, net for each of the periods indicated:

For the Three Months Ended March 31,
20252024
(in thousands)
Interest income$10,190$7,093
Accretion of investment discount, net2,23812,804
(Loss) gain on strategic investments(90,728)931
Interest expense(4,503)(4,166)
Change in fair value of derivatives*—(10,100)
Other, net(329)(19)
Total other (loss) income, net$(83,132)$6,543
  • Related to the change in fair value of the contingent consideration derivative liabilities. For more information, please read Note 4, Fair Value Measurements.
  1. INCOME TAXES

During the three months ended March 31, 2025, the Company recorded an income tax provision of $64.0 million, representing an effective tax rate of(16.7)%. The income tax provision primarily relates to the current tax provision on taxable profits, including in certain states which restrict the amount of net operating loss carryforwards which may be utilized to offset taxable income and the requirement to capitalize research and development costs for tax purposes.

On a periodic basis, the Company reassesses the valuation allowance on its deferred tax assets, weighing positive and negative evidence to assess the recoverability of such deferred tax assets. In assessing the Company’s ability to realize its net deferred tax assets, the Company considered various factors, including future reversals of existing taxable temporary differences, projected future taxable income, potential carryback claims and tax planning strategies to determine whether it is more likely than not that some portion or all of its net deferred tax assets will not be realized. Based upon these factors, the Company has determined that the uncertainty regarding the realization of these assets is sufficient to warrant the need to maintain a full valuation allowance against its net deferred tax assets as of March 31, 2025. It is possible that with continued positive performance of the business in future periods, there may be sufficient positive evidence to release a portion or all of the Company’s valuation allowance. The release of the valuation allowance, as well as the exact timing and the amount of such release, continue to be subject to, among other things, the Company’s level of profitability, revenue growth and expectations regarding future profitability. Release of the valuation allowance would result in a benefit to income tax expense for the period the release is recorded, which could have a material impact on net earnings. The total deferred tax asset balance subject to the valuation allowance was approximately $989.6 million as of March 31, 2025. The Company will continue to assess the realizability of its deferred tax assets on a quarterly basis.

During the three months ended March 31, 2024, the Company recorded an income tax provision of $5.3 million representing an effective tax rate of 14.2%. For tax years beginning on or after January 1, 2022, the Tax Cuts and Jobs Act of 2017 eliminates the option to currently deduct research and development expenses and requires taxpayers to capitalize and amortize the costs over five years for research activities performed in the U.S. and 15 years for research activities performed outside the U.S. The requirement to capitalize research and development costs for tax purposes resulted in the Company having taxable profits and recording federal and state tax expense of $3.2 million and $2.1 million, respectively.

  1. REVOLVING CREDIT FACILITY

On February 13, 2025 (the “Closing Date”), the Company entered into a credit agreement (the “Credit Agreement”) with JPMorgan Chase Bank, N.A., as administrative agent (the “Administrative Agent”) and as collateral agent and the lenders party thereto. The Credit Agreement provides for a five-year, $600.0 million senior secured revolving credit facility (the “Revolving Credit Facility”). The Company's obligations under the Credit Agreement are secured by substantially all of the Company's assets and the assets of certain wholly-owned material subsidiaries, subject to certain customary exceptions and exclusions.

Interest rates under the Revolving Credit Facility are variable and equal to the Secured Overnight Financing Rate plus a credit spread adjustment of 0.10% per annum (“Adjusted SOFR”), plus a margin of 1.125% to 1.75% per annum, or, at the Company’s option, at a base reference rate equal to the highest of (a) the federal funds rate plus 0.50%, (b) the rate of interest last quoted by the Administrative Agent as its “base rate” and (c) the one-month Adjusted SOFR rate plus 1.00%, plus a margin of 0.125% to 0.75% per annum. The Company also will pay an unused commitment fee ranging from 0.20% to 0.35% per annum on the unused commitments.

The Credit Agreement contains customary representations and warranties, affirmative covenants, negative covenants and events of default. The Credit Agreement also contains financial covenants that are assessed on the last day of each of the Company’s fiscal quarters, including certain financial ratios such as a maximum secured net leverage ratio and minimum consolidated interest coverage ratio. The Company may voluntarily prepay the outstanding revolving loans under the Revolving Credit Facility in whole or in part without premium or penalty provided that the prepayment shall be in certain amounts as specified therein.

The Company paid $3.2 million in arrangement and up-front fees on the Closing Date, of which $2.8 million and $0.4 million were included in other non-current assets and other current assets, respectively, within the unaudited condensed consolidated balance sheets as of March 31, 2025 and are being amortized over thefive-year term of the Revolving Credit Facility. As of March 31, 2025, there were no amounts outstanding under the Revolving Credit Facility and the Company was in compliance with the covenants described above.

  1. (LOSS) EARNINGS PER SHARE

Basic (loss) earnings per share is computed by dividing net (loss) income by the weighted-average number of shares of common stock outstanding. Diluted earnings per share is computed based on the treasury stock method for stock awards and the if-converted method for convertible debt by dividing net income by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding. Given that the Company recorded a net loss for the three months ended March 31, 2025, there is no difference between basic and diluted net loss per share since the effect of common stock equivalents would be anti-dilutive and are, therefore, excluded from the diluted net loss per share calculation.

The following table sets forth the computation of basic and diluted (loss) earnings per common share:

For the Three Months Ended March 31,
20252024
(in thousands, except per share amounts)
Numerator:
Net (loss) income - basic$(447,508)$36,119
Add: interest expense, net of tax, on the Company's convertible debt—429
Net (loss) income - diluted$(447,508)$36,548
Denominator:
Weighted-average common shares outstanding, basic97,36293,991
Effect of dilutive securities:
Common stock issuable under the Company's equity incentive plans—3,681
Common stock issuable under the Company's convertible debt—1,442
Weighted-average common shares outstanding, diluted97,36299,114
(Loss) earnings per common share, basic$(4.60)$0.38
(Loss) earnings per common share, diluted$(4.60)$0.37

The following table summarizes potential shares of common stock that were excluded from the computation of diluted earnings per share as they were anti-dilutive:

For the Three Months Ended March 31,
2025 · 2024
(in thousands)
Common stock issuable under the Company's equity incentive plans12,353(1)4,270(2)
Common stock issuable under the Company's convertible debt8,1008,100
Total number of potentially issuable common stock20,45312,370

(1)As of March 31, 2025, the anti-dilutive common stock issuable under the Company's equity incentive plans includes 1.3 million shares that have performance or market conditions that were not met. These and all shares issued under the Company’s equity incentive plans were anti-dilutive as the Company was in a net loss position at the end of the period.

(2)As of March 31, 2024, the anti-dilutive common stock issuable under the Company's equity incentive plans excludes 1.2 million shares that are dilutive but have performance or market conditions that were not met as of the end of the period.

  1. SEGMENT INFORMATION

The measure of segment profit or loss that the CEO uses to allocate resources and assess performance as the chief operating decision-maker (“CODM”) is the Company’s consolidated net (loss) income. The table below includes information about the Company’s segment, including significant segment expenses, and a reconciliation to net (loss) income:

For the Three Months Ended March 31,
20252024
(in thousands)
Total revenues$744,856$413,464
Segment expenses and other segment items
Cost of sales (excluding amortization of in-licensed rights)137,56450,559
Compensation and other personnel expenses93,08483,587
Up-front and milestone expenses583,787—
Manufacturing expenses67,46569,700
Clinical trial expenses33,14744,147
Facility- and technology-related expenses (excluding depreciation and amortization)27,65724,978
Research and development- other (excluding non-cash items) (a)28,47128,848
Selling, general and administrative- other (excluding non-cash items) (b)51,14248,962
Roche collaboration reimbursement(28,481)(21,658)
Other segment items (c)(1,909)(2,685)
Loss (gain) on strategic investments90,728(931)
Interest expense4,5034,166
Interest income(10,190)(7,093)
Income tax expense63,9905,329
Depreciation and amortization expense9,9788,744
Stock-based compensation expense41,42840,692
Segment net (loss) income$(447,508)$36,119
Reconciliation of profit or loss
Adjustments and reconciling items——
Consolidated net (loss) income$(447,508)$36,119

(a)Research and development-other includes professional services, pre-clinical expenses and research and other expenses.

(b)Selling, general and administrative-other includes professional services and other expenses.

(c)Other segment items included in segment net (loss) income include accretion of investment discount, net, change in fair value of derivatives and other, net, as well as the items separately presented and not defined as significant expenses below.

Significant expense categories that are regularly provided to the CODM include cost of sales (excluding amortization of in-licensed rights), compensation and other personnel expenses, up-front and milestone expenses, manufacturing expenses, clinical trial expenses, facility- and technology-related expenses, research and development- other and selling, general and administrative- other. The other expense or income information are other segment items and include separate presentation of loss (gain) on strategic investments, interest expense, income tax expense, depreciation and amortization and stock-based compensation, which are included in the measure of segment net (loss) income, but are not significant segment expenses.

Assets provided to the CODM for the single segment are consistent with those reported on the unaudited condensed consolidated balance sheets.

  1. COMMITMENTS AND CONTINGENCIES

Manufacturing Obligations

The following table summarizes the aggregate non-cancelable contractual obligations arising from the Company’s manufacturing obligations:

(in thousands)
2025 (April-December)$846,649
2026168,167
202779,856
202872,395
Total manufacturing commitments*$1,167,067

*Total manufacturing commitments include the Catalent, Inc. (“Catalent”) manufacturing and supply agreement, for which the Company has ROU assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of March 31, 2025. For more information, please read Note 22, Commitments and Contingenciesto the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024.

Litigation

In the normal course of business, the Company from time to time is named as a party to various legal claims, actions and complaints, which have included and may include matters involving securities, employment, intellectual property, arising from the use of therapeutics utilizing its technology, or others. The Company records a loss contingency reserve for a legal proceeding when it considers the potential loss probable and it can reasonably estimate the amount of the loss or determine a probable range of loss. The Company provides disclosure when it considers a loss reasonably possible or when it determines that a loss in excess of a reserve is reasonably possible. The Company provides an estimate of such reasonably possible losses or an aggregate range of such reasonably possible losses, unless the Company believes that such an estimate cannot be made. The Company has not recorded any material accruals for loss contingencies, and in management's opinion, no material range of loss is estimable for the matters described below as of March 31, 2025.

On September 15, 2020, REGENXBIO INC. (“Regenx”) and the Trustees of the University of Pennsylvania (“U-Penn”) filed a lawsuit against the Company and Sarepta Therapeutics Three, LLC, in the U.S. District Court for the District of Delaware. The plaintiffs assert patent infringement of U.S. Patent No. 10,526,617 (“the ’617 Patent”) under 35 U.S.C.§§ 271(a)-(c) based on Sarepta’s alleged direct or indirect manufacture and use of the patented cultured host cell technology allegedly used to make AAV gene therapy products, including SRP-9001 (approved June 22, 2023 in the U.S. as ELEVIDYS®). Specifically, the Complaint essentially includes the allegation that Sarepta’s use, and the use by its contract manufacturers on its behalf, of a host cell containing a recombinant acid molecule that encodes a capsid protein having at least 95% amino acid identity to AAVrh10 infringes the ’617 Patent asserted by Regenx. Plaintiffs seek injunctive relief, a judgment of infringement and willful infringement, damages that are no less than a reasonable royalty (treble damages), attorneys’ fees and costs, and such other relief as the court deems just and proper. On January 5, 2024, the Court granted Sarepta’s motion for summary judgment on the grounds that the asserted claims of Regenx’s ’617 Patent are invalid because they cover patent ineligible subject matter under 35 U.S.C. § 101. On January 12, 2024, the Court entered judgment and closed the case. Plaintiffs have appealed to the U.S. Court of Appeals for the Federal Circuit.

On June 20, 2023, Regenx and U-Penn commenced a second patent infringement lawsuit against Sarepta and its contract manufacturer, Catalent asserting patent alleged infringement of U. S. Patent No. 11,680,274 (“the ’274 Patent”). In the second lawsuit, Regenx and U-Penn allege that Sarepta and Catalent’s manufacture, use and commercial launch of ELEVIDYS® (formerly/also known as SRP-9001) infringe the ’274 Patent. Sarepta answered the complaint on August 10, 2023, and a case schedule has been set with a trial commencing on November 17, 2025. On February 21, 2024, Sarepta submitted a petition for Inter Partes Review for filing with the Patent Trial and Appeal Board (“PTAB”) at the U.S. Patent and Trademark Office (“USPTO”). The petition seeks to invalidate the ’274 Patent. On August 22, 2024, the PTAB instituted inter partes review of all challenged claims of the ’274 Patent on all asserted grounds.

On July 13, 2021, Nippon Shinyaku Co., Ltd. (“Nippon Shinyaku” or “NS”) filed a lawsuit against the Company in the U.S. District Court for the District of Delaware. NS asserted a claim for breach of contract arising from Sarepta filing seven petitions for Inter Partes Review (“IPR Petitions”) with the PTAB at the USPTO in which Sarepta sought to invalidate certain NS patents concerning exon 53 skipping technology (U.S. Patent Nos. 9,708,361, 10,385,092, 10,407,461, 10,487,106, 10,647,741, 10,662,217, and 10,683,322, respectively, and collectively the “NS Patents”). In addition, NS asserted claims for patent infringement and willful infringement of each of the NS Patents allegedly arising from Sarepta’s activities, including the sale of, its exon 53 skipping product, VYONDYS 53 (golodirsen). NS further sought a determination of non-infringement by NS alleged to arise from NS’s activities, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and invalidity of certain patents licensed to the Company from UWA (U.S. Patent Nos. 9,994,851, 10,227,590, and 10,266,827, collectively the “UWA Patents”). In its complaint, NS sought legal fees and costs, an unspecified amount of monetary relief (treble damages) attributed to Sarepta’s alleged infringement, and such other relief as the court deems just and proper. In January 2022, the PTAB granted institution of all claims of all NS Patents in response to Sarepta’s IPR Petitions and determined that Sarepta demonstrated a reasonable likelihood of success in proving that the NS Patents are unpatentable. NS filed a motion for preliminary injunction solely seeking Sarepta’s withdrawal of the IPR Petitions, which was ultimately granted after the U.S. Court of Appeals for the Federal Circuit reversed and remanded to the district court on February 8, 2022. Sarepta subsequently withdrew the IPRs, which were terminated on June 14, 2022. On December 27, 2021, the district court partially granted and denied the motion to dismiss by Sarepta and ordered NS to file a Second Amended Complaint (“SAC”), which it did on January 14, 2022. In the SAC, NS maintained all claims of the original complaint of July 13, 2021, except a determination of non-infringement of the UWA Patents. On January 28, 2022, Sarepta filed its answer to the SAC, with defenses and counterclaims against NS and NS Pharma Inc. that include infringement of the UWA Patents allegedly arising from their activities concerning, including the sale of, its exon 53 skipping product, Viltepso (viltolarsen) and breach of contract. Sarepta also sought a determination of invalidity of the NS Patents. In its counterclaim complaint, Sarepta sought an award of relief in its defenses to NS’ allegations, a judgment of breach of contract, a determination of invalidity of the NS Patents, a judgment of infringement and willful infringement of the UWA Patents, legal fees and costs, an unspecified amount of monetary relief (treble damages) attributable to NS’ alleged infringement, and such other relief as the court deems just and proper. UWA has since been joined as a Plaintiff in Sarepta’s counterclaims against NS. On August 14, 2023, the Court granted cross motions to amend the pleadings, allowing Sarepta to add a counterclaim against NS for inequitable conduct, and NS to add counterclaims against Sarepta for inequitable conduct and Walker Process fraud. The parties have since stipulated to the dismissal of NS’s claim of infringement of its ’361 Patent and certain claims of the ’322 Patent, and NS’s breach of contract claim. The Court bifurcated the Walker Process fraud claim on April 18, 2024, and granted Sarepta's motion for summary judgment of infringement of the ’851 Patent and NS’s motion for partial summary judgment of infringement of certain NS patents on May 1, 2024. After a jury trial in December 2024, the jury found that NS’s ’092 Patent is invalid as obvious and Sarepta’s and UWA’s ’851 Patent is not invalid. The jury did not find that NS’s infringement was willful. The jury awarded Sarepta approximately $115.2 million in damages for NS’s infringement relating to its sales of Viltepso in the United States, and the parties stipulated to approximately $0.8 million in reasonable royalty damages for NS's sales of Viltepso outside of the United States, both through December 15, 2024. Judgment was entered on January 7, 2025. On February 14, 2025, Sarepta filed a motion seeking supplemental damages and NS filed post-trial motions challenging the jury's verdict, as well as briefing relating to its inequitable conduct claim, which was tried to the court in December 2024.

On July 26, 2024, Genzyme Corporation filed a lawsuit against Sarepta Therapeutics, Inc. and Sarepta Therapeutics Three, LLC, in the U.S. District Court for the District of Delaware. The complaint asserts infringement of United States Patent Nos. 9,051,542 (the “’542 Patent”) and 7,704,721 (the “’721 Patent”) arising from Sarepta’s alleged manufacture and sale of ELEVIDYS® (delandistrogene moxeparvovec-rokl). In its complaint, Genzyme seeks, inter alia, damages for the alleged infringement, including increased damages up to three times the amount found or assessed, together with prejudgment and post-judgment interest and costs. Following a partial motion to dismiss by Sarepta, Genzyme filed its First Amended Complaint on November 21, 2024. In its First Amended Complaint, Genzyme no longer alleges willfulness or indirect infringement before the filing of the complaint. Sarepta answered the First Amended Complaint on December 12, 2024. The Court entered a scheduling order, with a trial scheduled for January 25, 2027.

On December 20, 2024, Brammer Bio MA, LLC filed an arbitration demand against Sarepta relating to Sarepta’s termination of the Thermo Agreement. Brammer Bio MA, LLC alleges claims for breach of contract, breach of the implied covenant of good faith and fair dealing and a violation of MGL c. 93A, and seeks relief including damages, treble damages, and attorneys’ fees and costs. On January 24, 2025, Sarepta filed its answer and asserted counterclaims for declaratory judgment and breach of contract, seeking relief including damages and attorneys’ fees and costs.

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