Sarepta Therapeutics, Inc.
For the period ended Sep 30, 2026. Filed Nov 5, 2026.
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Sep 30, 2026 · Filed Nov 5, 2026
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Notes to Condensed Consolidated Financial Statements
(unaudited)
- 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, small interfering RNA ("siRNA") knockdown therapies, gene therapies and other genetic therapeutic modalities for the treatment of rare diseases. Applying its proprietary, 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, Myotonic Dystrophy type 1 (“DM1"), facioscapulohumeral muscular dystrophy (“FSHD”), Huntington's Disease ("HD"), limb girdle muscular dystrophy ("LGMD"), and other neuromuscular and central nervous system disorders.
The Company's RNA-targeted products EXONDYS 51 (eteplirsen) Injection (“EXONDYS 51”), VYONDYS 53 (golodirsen) Injection (“VYONDYS 53”) and AMONDYS 45 (casimersen) Injection (“AMONDYS 45”) (collectively, the “PMO Products”) 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 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.
The Company's gene therapy product, ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-(“AAV”) based gene therapy, was initially 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 subsequently received traditional approval by the FDA in June 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS was also approved for non-ambulatory patients under the accelerated approval pathway in June 2024. In response to safety events announced in March and June 2025, the Company suspended all shipments of ELEVIDYS to non-ambulatory patients in the U.S. in June 2025. In November 2025, the Company announced a boxed warning for acute liver injury and acute liver failure and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information. The Company is currently in the process of conducting various clinical trials for ELEVIDYS, including a study to evaluate the use of sirolimus as an enhanced immunosuppressive regimen as part of treatment with ELEVIDYS for non-ambulatory individuals living with Duchenne. The Company intends to discuss with the FDA the results of this study and a potential pathway forward to resume commercial dosing in the non-ambulatory population. Resumption of dosing in the non-ambulatory population will depend in part on the FDA's analysis of whether the sirolimus data positively changes ELEVIDYS' risk/benefit profile and on aligning with the FDA on the process for revising the label, both of which involve risks and uncertainties.
As of September 30, 2026, the Company had $998.7 million of cash, cash equivalents, restricted cash and investments, consisting of $472.8 million of cash and cash equivalents, $514.8 million of investments and $11.1 million of non-current restricted cash and investments. The Company believes that its balance of cash, cash equivalents and investments as of the date of the issuance of this report, along with future cash inflows from operations and availability under the Company's Revolving Credit Facility (please refer to Note 13, Revolving Credit Facilityfor further information) 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/or establish collaborations with or license its technology to other companies.
- SUMMARY OF SIGNIFICANT ACCOUNTING POLICIES AND RECENT ACCOUNTING PRONOUNCEMENTS
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, 2025 which are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the U.S. Securities and Exchange Commission on March 2, 2026. The results for the three and nine months ended September 30, 2026 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, revenue, 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 September 30, 2026, the Company’s cash was concentrated at seven 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, certificates of deposit and bank time deposits 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 September 30, 2026, three entities accounted for 35%,24% and 21% of accounts receivable, net, respectively. As of December 31, 2025, four entities accounted for 33%,26%,20% and 10% of accounts receivable, net, respectively.
Correction of Immaterial Error
As disclosed in Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncementsto the Company's Annual Report on Form 10-K for the year ended December 31, 2025, the Company recorded an immaterial error correction associated with the accounting for its partial refinancing of its convertible senior notes due on September 15, 2027 (the “2027 Notes”) that was completed on August 28, 2025 (the "August 2025 Exchange"). The Company concluded that the error related to the August 2025 Exchange was not material to any previously issued financial statements and the unaudited condensed consolidated financial statements for the three and nine months ended September 30, 2025 presented herein have been revised to reflect the correction.
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, 2025.
Recent Accounting Pronouncements
Recently adopted
Effective in the fourth quarter of 2025, the Company early adopted Accounting Standards Update ("ASU") 2025-07, Derivatives and Hedging (Topic 815) and Revenue from Contracts with Customers (Topic 606): Scope Refinements ("ASU 2025-07") using the modified retrospective transition method. The Company recorded the cumulative effect of this accounting change to remove the previously recognized derivative liabilities as of January 1, 2025, reducing the contingent consideration liability by $47.4 million, with an offsetting adjustment to accumulated deficit. Accordingly, the comparative stockholders' equity balance as of September 30, 2025 presented herein reflects this $47.4 million reduction to opening accumulated deficit and differs from the previously reported stockholders' equity balance in the Company's Quarterly Report on Form 10-Q for the quarterly period ended September 30, 2025. The elimination of this derivative liability increased the previously reported net loss for both the three and nine months ended September 30, 2025 by $11.1 million, or $0.11 per basic and diluted share. For additional information regarding the adoption of ASU 2025-07, please refer to Note 2, Summary of Significant Accounting Policies and Recent Accounting Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2025.
Recently issued but not yet adopted
In November 2024, the FASB issued ASU 2024-03, Income Statement - Reporting Comprehensive Income - Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses("ASU 2024-03"), which requires public entities to provide disaggregated disclosure of income statement expenses. Public entities are required to disaggregate, in a tabular presentation, each relevant expense caption on the face of the consolidated statements of comprehensive (loss) income such as the following expenses: purchases of inventory, employee compensation, intangible asset amortization, and depreciation. ASU 2024-03 is effective for annual reporting periods beginning after December 15, 2026 and interim periods beginning after December 15, 2027, with early adoption permitted. The Company is currently evaluating the impact of this guidance and does not expect its adoption to have a material impact on its consolidated financial statements and related disclosures.
- LICENSE AND COLLABORATION AGREEMENTS
Arrowhead Pharmaceuticals, Inc.
In November 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 Collaboration Agreement”) that subsequently became effective as of February 7, 2025 (the “Arrowhead Collaboration Agreement Effective Date”).
The Arrowhead Collaboration Agreement granted the Company an exclusive license under certain of Arrowhead’s intellectual property rights to develop, manufacture and commercialize the lead candidate (and all backup candidates) for the programs listed below. The following table summarizes the current development stage of each program:
| Development Stage | Indication |
|---|---|
| Arrowhead Clinical Programs | (a) DUX4 for the treatment of FSHD;(b) DM1 for the treatment of myotonic dystrophy type 1;(c) ATXN2 for the treatment of ataxias;(d) MMP7 for the treatment of idiopathic pulmonary fibrosis; and(e) HD for the treatment of Huntington’s Disease. |
| Arrowhead Pre-clinical Programs | (a) ATXN1 for the treatment of ataxias; and (b) ATXN3 for the treatment of ataxias. |
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”).
Under the terms of the Arrowhead Collaboration 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, which 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.
Upon the effectiveness of the Arrowhead Collaboration Agreement, the Company paid Arrowhead an up-front payment of $500.0 million and invested $325.0 million in approximately 11.9 million shares of Arrowhead's common stock at a premium to the valuation on the closing date. On the Arrowhead Collaboration Agreement 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, based on the closing price of Arrowhead’s common stock traded on the Nasdaq Global Select Market (“Nasdaq”), and
$583.6 million was 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 and recorded as research and development expense, on the Company’s unaudited condensed consolidated statement of comprehensive (loss) income.
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 Collaboration 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. For the three and nine months ended September 30, 2026, the Company recorded $5.7 million and $20.5 million, respectively, of research and development expense related toreimbursable development costs incurred by Arrowhead. For the three and nine months ended September 30, 2025, the Company recorded $7.0 million and $15.8 million, respectively, 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 Collaboration License Fees”), with the first payment due on the first anniversary of the Arrowhead Collaboration Agreement Effective Date. The Annual Collaboration License Fees are contingent upon the Company not terminating the Arrowhead Collaboration Agreement. For the nine months ended September 30, 2026, the Company paid and recorded the first $50.0 million Annual Collaboration License Fee in research and development expense in the unaudited condensed consolidated statements of comprehensive (loss) income.
Over the term of the Arrowhead Collaboration 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 two milestones associated with the continued enrollment of certain cohorts of a Phase 1/2 study for the DM1 program(the "Arrowhead DM1 Milestones"), totaling $300.0 million, both of which were achieved and recognized during the year ended December 31, 2025. In January 2026, the Company paid $200.0 million in cash related to the second Arrowhead DM1 Milestone, which was included in accounts payable as of December 31, 2025. For details about the Arrowhead DM1 Milestones, please read Note 3, License and Collaboration Agreements of the Annual Report on Form 10-K for the year ended December 31, 2025. Furthermore, upon commercialization, the Company will be required to make tiered royalty payments based on net sales.
F. Hoffmann-La Roche Ltd.
For the nine months ended September 30, 2026 and 2025, the Company recognized $365.0 million and $175.5 million, respectively, of collaboration revenue associated with the license, collaboration and option agreement (the “Roche Collaboration Agreement”) with F. Hoffmann-La Roche Ltd. (“Roche”), with no collaboration revenue recognized during the three months ended September 30, 2026 or 2025. Under the Roche Collaboration Agreement, the Company 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 deemed as material rights related to individual programs and, thus, 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 2026, Roche declined to exercise an Option for certain program rights, which resulted in the immediate recognition of $325.0 million of collaboration revenue duringthe nine months ended September 30, 2026. Additionally, Roche dosed the first commercial patient in Japan in March 2026, which resulted in the recognition of a $40.0 million milestone payment as collaboration revenue under the Roche Collaboration Agreement.
In June 2025, the Company received a milestone payment of $63.5 million in connection with the receipt of regulatory approval of ELEVIDYS in Japan for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon 8 and/or exon 9 in the Duchenne gene and who are negative for anti-AAVrh74 antibodies. The milestone payment was included in collaboration and other revenues for thenine months ended September 30, 2025. In February 2025, an Option for a certain program expired, which resulted in the immediate recognition of $112.0 million of collaboration revenue for thenine months ended September 30, 2025.
In accordance with the Roche Collaboration Agreement, the parties agreed to enter into a supply agreement in order to supply Roche 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, the Company has delivered commercial ELEVIDYS supply to Roche that is 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. During the nine months ended September 30, 2026, Roche product cost of sales exceeded Roche contract manufacturing revenue as a result of the following period costs being allocated to Roche product cost of sales:
write-offs of certain batches of ELEVIDYS that did not meet quality specifications
the Company's reserve for obsolete ELEVIDYS inventory on hand, and
scrapped and expired materials.
The following table summarizes certain Roche activity for each of the periods indicated:
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| (in thousands) | ||||
| Contract manufacturing revenue | $50,100 | $18,705 | $135,604 | $63,107 |
| Royalty revenue | 7,488 | 10,608 | 19,834 | 22,007 |
| Roche product cost of sales | (47,581) | (17,527) | (145,437) | (51,488) |
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 and nine months ended September 30, 2026, costs reimbursable by Roche and reflected as a reduction to operating expenses were $7.1 million and $22.5 million, respectively. For the three and nine months ended September 30, 2025, costs reimbursable by Roche and reflected as a reduction to operating expenses were $12.2 million and $66.4 million, respectively. As of September 30, 2026 and December 31, 2025, there was $41.5 million and $104.1 million of collaboration and other receivables included in other current assets on the unaudited condensed consolidated balance sheets, respectively.
As of September 30, 2026 and December 31, 2025, the Company had total deferred revenue of $188.8 million and $527.3 million, of which $104.9 million and $443.4 million are classified as current, respectively. As of September 30, 2026 and December 31, 2025, all deferred revenue was related to the Roche Collaboration Agreement and Roche Supply Agreement.
Milestone Obligations
Including the Arrowhead Collaboration Agreement, 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 September 30, 2026, the Company may be obligated to make up to $12.0 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements.
- 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 nine months ended September 30, 2026, 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:
| Total | Level 1 | Level 2 | Level 3 | |
|---|---|---|---|---|
| (in thousands) | ||||
| Assets | ||||
| Money market funds | $194,009 | $194,009 | $— | $— |
| Government and government agency bonds | 294,612 | — | 294,612 | — |
| Certificates of deposit | 142,898 | — | 142,898 | — |
| Corporate bonds | 92,191 | — | 92,191 | — |
| Strategic investments | 5,823 | 1,392 | — | 4,431 |
| Commercial paper | 4,538 | — | 4,538 | — |
| Deferred compensation plan assets | 1,345 | 1,345 | — | — |
| Total assets | $735,416 | $196,746 | $534,239 | $4,431 |
| Total | Level 1 | Level 2 | Level 3 | |
|---|---|---|---|---|
| (in thousands) | ||||
| Assets | ||||
| Money market funds | $474,914 | $474,914 | $— | $— |
| Government and government agency bonds | 179,765 | — | 179,765 | — |
| Corporate bonds | 42,583 | — | 42,583 | — |
| Strategic investments | 9,520 | 4,089 | — | 5,431 |
| Commercial paper | 3,614 | — | 3,614 | — |
| Certificates of deposit | 3,368 | — | 3,368 | — |
| Deferred compensation plan assets | 883 | 883 | — | — |
| Total assets | $714,647 | $479,886 | $229,330 | $5,431 |
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 investment in a biotechnology company listed on Nasdaq and assets associated with the Company's deferred compensation plan that are held in a trust.
The Company's assets with a fair value categorized as Level 2 within the fair value hierarchy consist of government and government agency bonds, corporate bonds, commercial paper 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 highly liquid 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 September 30, 2026 and December 31, 2025.
The Company’s assets with a fair value categorized as Level 3 within the fair value hierarchy consist of strategic investments in private biotechnology companies whose fair value measurement is 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. Impairments and other measurement events related to the Company's Level 3 strategic investments were not material during the nine months ended September 30, 2026 and 2025.
The fair values of the 2027 Notes and the Company’s 4.875% convertible senior notes due September 1, 2030 (the "2030 Notes") are based on open market trades and are classified as Level 1 in the fair value hierarchy. As of September 30, 2026, the carrying value of the 2027 Notes was classified within current liabilities on the unaudited condensed consolidated balance sheets as the 2027 Notes mature on September 15, 2027. The following table summarizes the Company's debt instruments by indenture for the periods indicated:
| As of September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Net carrying value of the 2027 Notes | $157,971 | $157,478 |
| Net carrying value of the 2030 Notes | 699,393 | 671,496 |
| Total carrying value of debt instruments | $857,364 | $828,974 |
| Fair value of 2027 Notes | $152,100 | $143,076 |
| Fair value of 2030 Notes | 752,014 | 721,791 |
| Total fair value of debt instruments | $904,114 | $864,867 |
The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents, accounts receivable, net, accounts payable and bank time deposits included in short-term investments approximated fair value because of the immediate or short-term maturity of these financial instruments.
- FINANCIAL INSTRUMENTS
The following table summarizes the Company’s financial assets with maturities of less than three months at the date of acquisition included in cash equivalents in the unaudited condensed consolidated balance sheets for each of the periods indicated:
| As of September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Money market funds | $194,009 | $474,914 |
| Government and government agency bonds | 21,914 | 89,914 |
| Total | $215,923 | $564,828 |
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 September 30, 2026 and December 31, 2025 was approximately eight andthree months, respectively. All of the Company's non-current investments as of September 30, 2026 and December 31, 2025 hadmaturities betweenone andtwo years.
The following tables summarize the Company’s financial instruments for each of the periods indicated:
| Amortized Cost | Gross Unrealized Gains | Gross Unrealized Losses | Fair MarketValue | |
|---|---|---|---|---|
| (in thousands) | ||||
| Cash and money market funds | $450,847 | $— | $— | $450,847 |
| Government and government agency bonds | 295,847 | — | (1,235) | 294,612 |
| Certificates of deposit | 142,898 | — | — | 142,898 |
| Corporate bonds | 92,664 | 75 | (548) | 92,191 |
| Commercial paper | 4,538 | — | — | 4,538 |
| Bank time deposits | 2,505 | — | — | 2,505 |
| Total cash, cash equivalents and investments | $989,299 | $75 | $(1,783) | $987,591 |
| As reported: | ||||
| Cash and cash equivalents | $472,761 | $— | $(1) | $472,760 |
| Short-term investments | 406,848 | 75 | (907) | 406,016 |
| Non-current investments | 109,690 | — | (875) | 108,815 |
| Total cash, cash equivalents and investments | $989,299 | $75 | $(1,783) | $987,591 |
| Amortized Cost | Gross Unrealized Gains | Gross Unrealized Losses | Fair MarketValue | |
|---|---|---|---|---|
| (in thousands) | ||||
| Cash and money market funds | $711,368 | $— | $— | $711,368 |
| Government and government agency bonds | 179,615 | 150 | — | 179,765 |
| Corporate bonds | 42,527 | 56 | — | 42,583 |
| Commercial paper | 3,614 | — | — | 3,614 |
| Certificates of deposit | 3,368 | — | — | 3,368 |
| Total cash, cash equivalents and investments | $940,492 | $206 | $— | $940,698 |
| As reported: | ||||
| Cash and cash equivalents | $801,269 | $13 | $— | $801,282 |
| Short-term investments | 138,175 | 193 | — | 138,368 |
| Non-current investments | 1,048 | — | — | 1,048 |
| Total cash, cash equivalents and investments | $940,492 | $206 | $— | $940,698 |
- 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 September 30, | For the Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| (in thousands) | ||||
| PMO Products | ||||
| United States | $204,331 | $209,132 | $591,725 | $597,882 |
| Rest of World | 35,982 | 29,413 | 107,702 | 108,473 |
| Total PMO product revenues, net | $240,313 | $238,545 | $699,427 | $706,355 |
| ELEVIDYS | ||||
| United States | $83,450 | $131,498 | $283,540 | $788,334 |
| Total ELEVIDYS product revenues, net | $83,450 | $131,498 | $283,540 | $788,334 |
| Total product revenues, net | $323,763 | $370,043 | $982,967 | $1,494,689 |
For the three and nine months ended September 30, 2026 and 2025, 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 September 30, | For the Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| Product revenues, net | ||||
| Customer 1 | 38% | 34% | 37% | 25% |
| Customer 2 | 24% | 22% | 22% | 15% |
As of September 30, 2026 and December 31, 2025, the Company's accounts receivable, net were $369.2 million and $398.2 million, respectively, both of which were related to product sales, net of discounts and allowances. As of September 30, 2026, 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:
| Chargebacks | Rebates | Prompt Pay | Other Accruals | Total | |
|---|---|---|---|---|---|
| (in thousands) | |||||
| Balance, as of December 31, 2025 | $25,563 | $116,585 | $3,831 | $54,057 | $200,036 |
| Provision | 66,296 | 115,320 | 11,900 | 64,511 | 258,027 |
| Adjustments relating to prior periods | — | (10,274) | (1,429) | (21) | (11,724) |
| Payments/credits | (76,682) | (113,427) | (10,158) | (76,403) | (276,670) |
| Balance, as of September 30, 2026 | $15,177 | $108,204 | $4,144 | $42,144 | $169,669 |
| Chargebacks | Rebates | Prompt Pay | Other Accruals | Total | |
|---|---|---|---|---|---|
| (in thousands) | |||||
| Balance, as of December 31, 2024 | $45,904 | $107,843 | $5,941 | $34,611 | $194,299 |
| Provision | 159,510 | 148,822 | 15,702 | 65,864 | 389,898 |
| Adjustments relating to prior periods | — | (10,327) | — | (625) | (10,952) |
| Payments/credits | (185,724) | (118,600) | (17,498) | (62,232) | (384,054) |
| Balance, as of September 30, 2025 | $19,690 | $127,738 | $4,145 | $37,618 | $189,191 |
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 September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Reduction to accounts receivable, net | $60,571 | $82,518 |
| Component of accrued expenses | 109,098 | 117,518 |
| Total reserves | $169,669 | $200,036 |
- INVENTORY
The following table summarizes the components of the Company’s inventory for each of the periods indicated:
| As of September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Raw materials | $132,256 | $141,278 |
| Work in progress | 883,808 | 892,689 |
| Finished goods | 60,633 | 65,320 |
| Total inventory | $1,076,697 | $1,099,287 |
For the nine months ended September 30, 2026, inventory losses for write-downs totaled $11.6 million related to obsolete ELEVIDYS inventory. There wereno inventory losses for write-downs of excess and obsolete inventory for thenine months ended September 30, 2025.
The Company classifies inventory associated with its PMO Products and ELEVIDYS as non-current inventory when consumption of the inventory is expected beyond the Company's normal inventory operating cycle oftwo years for the PMO Products and approximately 3.5 years for ELEVIDYS. Non-current inventory consists of raw materials and work in progress associated with the PMO Productsand raw materials associated with ELEVIDYS.
The following table summarizes the balance sheet classification of the Company's inventory for each of the periods indicated:
| As of September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Balance sheet classification: | ||
| Inventory | $876,118 | $914,744 |
| Non-current inventory | 200,579 | 184,543 |
| Total inventory | $1,076,697 | $1,099,287 |
- OTHER ASSETS
The following table summarizes the Company’s other current assets for each of the periods indicated:
| As of September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Collaboration and other receivables | $41,499 | $104,117 |
| Tax-related receivables and prepaids | 13,807 | 15,604 |
| Prepaid maintenance services | 11,643 | 12,639 |
| Prepaid clinical and pre-clinical expenses | 6,432 | 7,976 |
| Prepaid commercial expenses | 3,189 | 2,290 |
| Interest receivable | 2,994 | 1,656 |
| Prepaid insurance | 2,094 | 4,004 |
| Other | 4,953 | 23,570 |
| Total other current assets | $86,611 | $171,856 |
The following table summarizes the Company’s other non-current assets for each of the periods indicated:
| As of September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Manufacturing-related deposits and prepaids | $90,755 | $92,890 |
| Intangible assets, net | 29,834 | 28,949 |
| Restricted cash and investments* | 11,125 | 13,125 |
| Strategic investments | 5,823 | 9,520 |
| Prepaid maintenance services | 3,061 | 3,058 |
| Deferred tax asset | 2,854 | 2,822 |
| Other | 3,334 | 5,190 |
| Total other non-current assets | $146,786 | $155,554 |
- Restricted cash and investments 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 and investments are unavailable for withdrawal or use for general obligations.
- ACCRUED EXPENSES
The following table summarizes the Company’s accrued expenses for each of the periods indicated:
| As of September 30, 2026 | As of December 31, 2025 | |
|---|---|---|
| (in thousands) | ||
| Product revenue related reserves | $109,098 | $117,518 |
| Accrued contract manufacturing costs | 58,946 | 92,281 |
| Accrued employee compensation costs | 45,812 | 58,607 |
| Accrued litigation settlement charge | 39,000 | — |
| Accrued professional fees | 22,015 | 17,840 |
| Accrued clinical and pre-clinical costs | 14,303 | 18,894 |
| Accrued income taxes | 10,217 | 4,755 |
| Accrued royalties | 9,427 | 10,863 |
| Accrued interest expense | 4,575 | 20,714 |
| Accrued clinical collaboration costs | 3,336 | 10,222 |
| Other | 5,035 | 7,965 |
| Total accrued expenses | $321,764 | $359,659 |
- STOCK-BASED COMPENSATION
The following table summarizes the Company’s stock awards granted for each of the periods indicated:
| For the Three Months Ended September 30, · For the Nine Months Ended September 30, | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 2026 · 2025 · 2026 · 2025 | ||||||||||||
| Grants · WeightedAverageGrantDate FairValue · Grants · WeightedAverageGrantDate FairValue · Grants · WeightedAverageGrantDate FairValue · Grants · WeightedAverageGrantDate FairValue | ||||||||||||
| Stock options | 2,224,342 | (1) | $10.48 | — | $— | 2,224,342 | (1) | $10.48 | 488,192 | $46.93 | ||
| Restricted stock units | 967,174 | (1) | $16.04 | 3,496,155 | (2) | $18.95 | 1,755,359 | (1) | $17.00 | 5,191,278 | (2) | $39.73 |
| Performance stock units | — | $— | 225,000 | (3) | $18.95 | — | $— | 345,378 | (4) | $47.09 | ||
(1)On July 28, 2026 (the "Grant Date"), the Company granted its Chief Executive Officer, Dr. Michael Severino, approximately 2.2 million premium-priced stock options that vest 25% on the first anniversary of the Grant Date, with the remainder vesting monthly over the following three years and approximately 0.8 million restricted stock units(“RSUs”) that vest in equal annual installments over four years, in each case generally subject to continued employment.
(2) In September 2025, approximately 3.4 million RSUs were granted, which will vest overtwo years while the remaining vest over four years.
(3) In September 2025, RSUs with performance conditions (“PSUs”) were granted (the "September 2025 PSUs"). The performance condition is related to the achievement of a certain financial performance goal. The September 2025 PSUs have a three-year cliff vesting schedule. As of September 30, 2026, the performance condition associated with the September 2025 PSUs has been achieved.
(4) In addition to the September 2025 PSUs, included in PSUs for the nine months ended September 30, 2025 are approximately 0.1 million 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 (the “March 2025 PSUs”). As of September 30, 2026, none of the performance conditions for the March 2025 PSUs were probable of being achieved.
Stock-based Compensation Expense
For the three months ended September 30, 2026 and 2025, total stock-based compensation expense included in total expenses was $26.7 million and $14.9 million, respectively. For the nine months ended September 30, 2026 and 2025, total stock-based compensation expense included in total expenses was $80.6 million and $93.4 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 September 30, | For the Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| (in thousands) | ||||
| Stock options | $5,663 | $8,939 | $17,737 | $33,517 |
| Restricted and performance stock units | 24,145 | 10,724 | 73,774 | 70,381 |
| Employee Stock Purchase Plan | 479 | (3,267) | 479 | 672 |
| Subtotal | $30,287 | $16,396 | $91,990 | $104,570 |
| Capitalized stock-based compensation costs | (3,544) | (1,469) | (11,376) | (11,190) |
| Total stock-based compensation expense included in expenses | $26,743 | $14,927 | $80,614 | $93,380 |
| Research and development | 8,509 | 4,139 | 27,295 | 36,733 |
| Selling, general and administrative | 18,234 | 10,788 | 53,319 | 56,647 |
| Total stock-based compensation expense included in expenses | $26,743 | $14,927 | $80,614 | $93,380 |
- OTHER (LOSS) INCOME, NET
The following table summarizes other (loss) income, net for each of the periods indicated:
| For the Three Months Ended September 30, | For the Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| (in thousands) | ||||
| Interest income | $7,086 | $7,249 | $19,653 | $22,693 |
| Accretion of investment discount, net | 1,117 | 1,132 | 2,714 | 5,257 |
| Interest expense | (21,616) | (9,460) | (64,837) | (19,191) |
| (Loss) gain on strategic investments | (523) | 36,739 | (2,683) | (17,268) |
| Other, net | (2,790) | (702) | (1,872) | (1,604) |
| Other (expense) income, net | $(16,726) | $34,958 | $(47,025) | $(10,113) |
| Gain on debt extinguishment | — | 3,761 | — | 3,761 |
| Total other (loss) income, net | $(16,726) | $38,719 | $(47,025) | $(6,352) |
- INCOME TAXES
During the three and nine months ended September 30, 2026, the Company recorded an income tax expense of $2.7 million and $18.1 million, representing an effective tax rate of(10.5)% and 5.7%, respectively. The provision for income taxes primarily reflects current foreign and state income tax expense. State tax expense primarily relates to taxable income in certain states in which the Company operates that continue to require the capitalization of research and development costs or limit the utilization of net operating loss carryforwards.
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. Under the applicable accounting standards, management has considered the Company’s history of losses and concluded that it is more likely than not that the Company will not recognize the benefits of its net federal and state deferred tax assets. Accordingly, a full valuation allowance against the U.S. net deferred tax asset is maintained at September 30, 2026. It is possible the Company may release a portion or all of its valuation allowance in future periods. The Company will continue to assess the realizability of its deferred tax assets on a quarterly basis.
During the three and nine months ended September 30, 2025, the Company recorded an income tax (benefit) expense of $(14.1) million and $6.6 million, representing an effective tax rate of 21.8% and(2.3)%, respectively. The income tax expense, for the nine months ended September 30, 2025 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. The income tax (benefit), for the three months ended September 30, 2025, primarily relates to the tax (benefit) recorded on the quarter to date loss which offsets tax expense recorded on profits in prior interim periods.
- REVOLVING CREDIT FACILITY
The Company entered into a credit agreement (the “Credit Agreement”) on February 13, 2025, which was subsequently amended on December 18, 2025, 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.
During the nine months ended September 30, 2026, the Company drew down and repaid $425.0 million under the Revolving Credit Facility and paid approximately $0.8 million of interest expense. As of September 30, 2026, there were no amounts outstanding under the Revolving Credit Facility and the Company was in compliance with the covenants described above.
- (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. In periods in which the Company records a net loss, 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 September 30, · For the Nine Months Ended September 30, | |||||
|---|---|---|---|---|---|
| 2026 · 2025 · 2026 · 2025 | |||||
| (in thousands, except per share amounts) | |||||
| Numerator: | |||||
| Net (loss) income - basic | $(28,461) | $(50,568) | $297,608 | $(301,184) | |
| Add: interest expense, net of tax, on the Company's convertible debt | — | — | 1,865 | (1) | — |
| Net (loss) income - diluted | $(28,461) | $(50,568) | $299,473 | $(301,184) | |
| Denominator: | |||||
| Weighted-average common shares outstanding, basic | 105,930 | 100,237 | 105,453 | 98,545 | |
| Effect of dilutive securities: | |||||
| Common stock issuable under the Company's equity incentive plans | — | — | 1,076 | — | |
| Common stock issuable under the Company's convertible debt | — | — | 1,117 | (1) | — |
| Weighted-average common shares outstanding, diluted | 105,930 | 100,237 | 107,646 | 98,545 | |
| (Loss) earnings per common share, basic | $(0.27) | $(0.50) | $2.82 | $(3.06) | |
| (Loss) earnings per common share, diluted | $(0.27) | $(0.50) | $2.78 | $(3.06) | |
The following table summarizes potential shares of common stock that were excluded from the computation of diluted(loss) earnings per share as they were anti-dilutive:
| For the Three Months Ended September 30, · For the Nine Months Ended September 30, | ||||||||
|---|---|---|---|---|---|---|---|---|
| 2026 · 2025 · 2026 · 2025 | ||||||||
| (in thousands) | ||||||||
| Common stock issuable under the Company's equity incentive plans | 13,124 | (2)(4) | 12,842 | (3)(4) | 11,191 | (2) | 12,842 | (3)(4) |
| Common stock issuable under the Company's convertible debt | 16,007 | 13,203 | 14,890 | (1) | 13,203 | |||
| Total number of potentially issuable common stock | 29,131 | 26,045 | 26,081 | 26,045 | ||||
For thenine months ended September 30, 2026, the interest expense, net of tax, and potential share settlement of the 2030 Convertible Notes were excluded from diluted net income and diluted weighted-average common stock outstanding, respectively, as the inclusion of such shares was anti-dilutive during the period.
For thethree and nine months ended September 30, 2026, the anti-dilutive common stock issuable under the Company's equity incentive plans excludes 0.4 million shares that have performance conditions that were not met as of the end of the period.
For thethree and nine months ended September 30, 2025, the anti-dilutive common stock issuable under the Company's equity incentive plans excludes 0.3 million shares that have performance conditions that were not met as of the end of the period.
For thethree months ended September 30, 2026 and the three and nine months ended September 30, 2025, all shares issued under the Company’s equity incentive plans were anti-dilutive as the Company was in a net loss position during these periods.
- SEGMENT INFORMATION
The Company operates inone segment: discovering, developing, manufacturing and delivering therapies to patients with rare diseases. As of September 30, 2026, there have not been material changes in significant expense categories or assets provided to the Company’s CEO, the Company’s chief operating decision maker (the "CODM"). For further details regarding the Company's segment information, please read Note 21, Segment Information of the Annual Report on Form 10-K for the year ended December 31, 2025. 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 September 30, | For the Nine Months Ended September 30, | |||
|---|---|---|---|---|
| 2026 | 2025 | 2026 | 2025 | |
| (in thousands) | ||||
| Total revenues | $381,351 | $399,356 | $1,513,405 | $1,755,303 |
| Segment expenses and other segment items: | ||||
| Cost of sales (excluding amortization of in-licensed rights) | 188,512 | 150,775 | 446,665 | 440,897 |
| Compensation and other personnel expenses | 62,644 | 44,428 | 185,299 | 227,351 |
| Up-front and collaboration license fees | — | 100,000 | 50,000 | 683,787 |
| Manufacturing expenses | 2,251 | 33,897 | 23,846 | 182,047 |
| Clinical trial expenses | 20,511 | 28,613 | 61,917 | 95,271 |
| Facility- and technology-related expenses (excluding depreciation and amortization) | 22,940 | 20,577 | 69,772 | 76,814 |
| Restructuring charge | — | 40,510 | — | 40,510 |
| Research and development- other (excluding non-cash items) (1) | 17,241 | 20,883 | 55,759 | 81,197 |
| Selling, general and administrative- other (excluding non-cash items) (2) | 50,787 | 41,883 | 133,442 | 149,347 |
| Roche collaboration reimbursement | (7,138) | (12,198) | (22,496) | (66,368) |
| Other segment items (3) | 1,673 | (430) | (842) | (3,653) |
| Gain on debt extinguishment | — | (3,761) | — | (3,761) |
| Loss (gain) on strategic investments | 523 | (36,739) | 2,683 | 17,268 |
| Interest expense | 21,616 | 9,460 | 64,837 | 19,191 |
| Interest income | (7,086) | (7,249) | (19,653) | (22,693) |
| Income tax expense (benefit) | 2,695 | (14,102) | 18,051 | 6,634 |
| Depreciation and amortization expense | 5,900 | 18,450 | 26,903 | 39,268 |
| Stock-based compensation expense | 26,743 | 14,927 | 80,614 | 93,380 |
| Litigation settlement charge | — | — | 39,000 | — |
| Segment net (loss) income | $(28,461) | $(50,568) | $297,608 | $(301,184) |
| Reconciliation of profit or loss | ||||
| Adjustments and reconciling items | — | — | — | — |
| Consolidated net (loss) income | $(28,461) | $(50,568) | $297,608 | $(301,184) |
(1)
Research and development-other includes professional services, pre-clinical expenses and research and other expenses.
(2)
Selling, general and administrative-other includes professional services and other expenses.
(3)
Other segment items included in segmentnet (loss) income include accretion of investment discount, net and other, net, as well as 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 collaboration license fees, manufacturing expenses, clinical trial expenses, facility- and technology-related expenses, restructuring charge, 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, interest income, income tax expense (benefit), depreciation and amortization, stock-based compensation expense and litigation settlement charge, 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.
- COMMITMENTS AND CONTINGENCIES
Manufacturing Obligations
The following table summarizes the aggregate non-cancelable contractual obligations arising from the Company’s manufacturing obligations:
| As ofSeptember 30, 2026** | |
|---|---|
| (in thousands) | |
| 2026 (October-December) | $168,990 |
| 2027 | 145,452 |
| 2028 | 39,640 |
| Total manufacturing commitments* | $354,082 |
*Total manufacturing commitments include the Catalent Agreement (defined below), to which the Company has right of use assets and lease liabilities recorded on the unaudited condensed consolidated balance sheets as of September 30, 2026. For more information, please read Note 23, Commitments and Contingenciesto the financial statements included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025.
**Total manufacturing commitments include obligations related to the global supply of ELEVIDYS, including inventories necessary to supply Roche for sales of ELEVIDYS in territories outside of the U.S. where Roche has received certain approvals for ELEVIDYS.
Catalent, Inc.
Under the manufacturing and supply agreements with Catalent, Inc. (“Catalent”) (the agreements, the "Catalent Agreements") described in Note 23, Commitments and Contingencies, to the consolidated financial statements included in the Company's Annual Report on Form 10-K for the year ended December 31, 2025, the Company has certain non-cancelable manufacturing obligations. In September 2026, the Company elected not to manufacture certain inventories subject to these obligations. As a result, the Company will receive no other future economic benefit, and accordingly, recorded a take-or-paycharge of $62.0 million within cost of sales (excluding amortization of in-licensed rights) during the three and nine months ended September 30, 2026. As of September 30, 2026, $22.1 million of thetake-or-pay charge remained unpaid and is included within accrued expenses in the accompanying unaudited condensed consolidated balance sheets.
In October 2026, the Company amended its August 2025 supplemental letter agreement with Catalent in connection with the Catalent Agreements (the “Letter Agreement Amendment”). The Letter Agreement Amendment resolved the obligations underlying the charge described above and reduced the Company's committed manufacturing batch quantity. The Company's remaining non-cancelable manufacturing obligations under the Catalent Agreements as of September 30, 2026 are reflected in the manufacturing commitments table above.
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 accrual 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. Except as disclosed below, the Company has not recorded any material accruals or charges for loss contingencies or settlements, and in management's opinion, no material range of loss is reasonably estimable for the remaining matters described below as of September 30, 2026.
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 ("Sarepta Three" and, together with the Company, "Sarepta"), 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 (including 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 appealed to the U.S. Court of Appeals for the Federal Circuit (the “Federal Circuit”), which reversed the district court's decision on February 20, 2026, and remanded the case for further proceedings. On March 23, 2026, the Company filed a petition for rehearing en banc before the Federal Circuit, which the Federal Circuit denied on April 22, 2026. The Federal Circuit issued a mandate on April 29, 2026.
On June 20, 2023, Regenx and U-Penn commenced a second patent infringement lawsuit against Sarepta and its contract manufacturer, Catalent, asserting alleged patent 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. On February 21, 2024, Sarepta submitted a petition for inter partes review ("IPR") for filing with the Patent Trial and Appeal Board (“PTAB”) at the U.S. Patent and Trademark Office (“USPTO”). The petition sought to invalidate the ’274 Patent. On March 20, 2024, the court in the patent infringement litigation ordered that the case be stayed pending final resolution of the IPR proceedings. On August 22, 2024, the PTAB instituted review of all challenged claims of the ’274 Patent on all asserted grounds. U-Penn subsequently disclaimed five of the six challenged claims, and on August 20, 2025, the PTAB entered a final written decision in the IPR proceedings finding that the sole remaining claim was not unpatentable. On October 3, 2025, the parties submitted a joint status report to the court in the patent infringement litigation, in which they agreed that the stay of the patent litigation should remain in place until Sarepta has exhausted its right to review by the Federal Circuit. On October 20, 2025, Sarepta filed a notice of appeal of the PTAB's decision to the Federal Circuit.
On September 23, 2026 (the “Settlement Effective Date”), Sarepta, Regenx, U-Penn and Catalent entered into a settlement agreement (the “Settlement Agreement”) regarding, among other matters, the resolution of certain patent infringement litigations and an appeal from inter partes review proceedings relating to the '274 Patent and the '617 Patent (collectively, the “Litigations”).
Under the terms of the Settlement Agreement, Sarepta paid Regenx a lump sum of $39.0 million within ten days of the Settlement Effective Date of the Settlement Agreement. Regenx and U-Penn released Sarepta, Catalent and their affiliates from any claims relating to any Sarepta gene therapy product that uses the AAVrh74-based capsid, including ELEVIDYS, that are claims of infringement of the ‘274 Patent, the ‘617 Patent and U.S. Patent No. 9,198,984 and certain related patents (collectively, the “U-Penn Patents”), arising at any time up to and including the Effective Date.
In addition, Regenx and U-Penn agreed not to sue Sarepta, Catalent or their current affiliates, licensees, commercial partners, successors and permitted assigns with respect to any claims of patent infringement of the U-Penn Patents to the extent that infringement arises from or relates to any Sarepta gene therapy product that uses an AAVrh74-based capsid, including ELEVIDYS. Regenx further agreed not to sue Sarepta or its licensees, commercial partners, successors and permitted assigns with respect to claims of patent infringement of any Regenx-controlled or owned patents or patent applications that cover the existing form of ELEVIDYS or the AAVrh74 capsid sequence.
The Settlement Agreement provides for the dismissal, withdrawal or other disposal of the Litigations with prejudice. Each party will continue to bear its own costs, expenses and attorneys’ fees in connection with the Litigations and the negotiation and execution of the Settlement Agreement. The Settlement Agreement does not include any admission of liability or wrongdoing by any party. During the nine months ended September 30, 2026, the Company recorded a litigation settlement charge of $39.0 million related to the settlement of the Litigations.
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 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
Sareptaand 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. Those motions remain pending.
On July 26, 2024, Genzyme Corporation ("Genzyme") 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 treble damages, 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. Sarepta answered the First Amended Complaint on December 12, 2024. On May 27, 2025, the court subsequently granted Genzyme’s motion to amend the complaint to include new allegations of infringement related to five patents: United States Patent Nos. 12,031,894 (the “’894 Patent"), 12,013,326 (the “’326 Patent”), 11,698,377 (the “’377 Patent”), 12,123,880 (the “’880 Patent”), and 12,298,313 (the “’313 Patent”). The Court also entered an amended scheduling order, with trial scheduled for June 14, 2027. Sarepta answered the Second Amended Complaint on June 17, 2025. Sarepta submitted IPR petitions challenging the validity of the ’542 and ’721 Patents, for which the USPTO director discretionarily denied institution in November 2025. In November and December 2025, Sarepta submitted IPR petitions challenging the validity of the ’894 Patent, ’326 Patent, ’377 Patent, ’880 Patent, and ’313 Patent. The USPTO director instituted review of the ’894 Patent and denied institution of review of the ’326 Patent, ’377 Patent, ’880 Patent, and ’313 Patent. In June 2026, the USPTO director denied Genzyme's request for director review of the decision granting institution of review of the ’894 Patent, and oral argument in that proceeding is currently scheduled for February 4, 2027. Proceedings in the patent litigation remain ongoing.
On June 26, 2025, a putative securities class action complaint was filed against the Company, former Chief Executive Officer Douglas Ingram, former Chief Customer Officer Dallan Murray, and President of Research and Development and Technical Operations Louise Rodino-Klapac in the U.S. District Court for the Southern District of New York (the “Securities Action”). The complaint alleges violations of the Securities Exchange Act of 1934 (the “Exchange Act”) and Rule 10b-5 in connection with disclosures made regarding ELEVIDYS’s safety and efficacy and the Company’s financial statements and projections. The plaintiff sought to represent a class of shareholders who purchased or otherwise acquired the Company’s securities between June 22, 2023 and June 24, 2025. The complaint seeks unspecified damages. On October 17, 2025, the court appointed lead plaintiffs and lead counsel. On October 24, 2025, lead plaintiffs filed a motion to transfer venue to the U.S. District Court for the District of Massachusetts, which the court granted on November 6, 2025. On January 22, 2026, the lead plaintiffs filed an amended complaint including a new plaintiff, removing Dallan Murray as a defendant, adding President and Chief Operating Officer Ian Estepan as a defendant, and expanding the class period to shareholders who purchased or otherwise acquired the Company’s securities between June 22, 2023 and November 3, 2025 (the “Amended Complaint”). The Amended Complaint further alleges additional theories of liability, including that Sarepta and the named defendants made false or misleading public statements related to (i) the safety profile of ELEVIDYS and LGMD therapies that utilize the AAVrh74 viral vector; (ii) the status of the development of LGMD therapies; and (iii) the status of the ESSENCE trial. On March 9, 2026, Sarepta and the named defendants filed a motion to dismiss the Amended Complaint, and oral argument was held on August 4, 2026. On September 15, 2026, the court granted Sarepta and the named defendants' motion to dismiss and dismissed the case with prejudice. On October 13, 2026, the plaintiffs filed a notice of appeal with the U.S. Court of Appeals for the First Circuit.
On July 15, 2025, a shareholder derivative lawsuit concerning certain of the same disclosures underlying the first complaint in the Securities Action was filed in the U.S. District Court for the Southern District of New York, naming Company directors M.
Kathleen Behrens, Richard J. Barry, Kathryn Boor, Michael Chambers, Deirdre Connelly, Stephen L. Mayo, Claude Nicaise, and Hans Wigzell, as well as Douglas Ingram, Dallan Murray, and Louise Rodino-Klapac (the “Individual Defendants”). Two substantially similar shareholder derivative lawsuits were filed in the same court against the same defendants on August 20, 2025 and September 10, 2025, respectively (collectively, the “S.D.N.Y. Derivative Actions”). The Company is named as a nominal defendant in all three lawsuits. The S.D.N.Y. Derivative Actions allege, among other claims, breaches of the Individual Defendants’ fiduciary duties in connection with certain of the same disclosures at issue in the Securities Action and violations of Section 14(a) of the Exchange Act in connection with the Company’s 2024 and 2025 annual proxy statements. The S.D.N.Y. Derivative Actions seek unspecified damages, including costs incurred in defending the Company against the Securities Action. Two of the S.D.N.Y. Derivative Actions also seek an order that the Company take all necessary action to reform and improve its corporate governance and internal procedures. On October 10, 2025, the court consolidated the Derivative Actions and appointed co-lead counsel (the “S.D.N.Y. Consolidated Derivative Action”). On November 17, 2025, the court stayed the S.D.N.Y. Consolidated Derivative Action until the Securities Action is resolved.
On April 7, 2026, a separate shareholder derivative lawsuit was filed in the U.S. District Court for the District of Massachusetts, naming the same Individual Defendants as in the S.D.N.Y. Consolidated Derivative Action. On July 23, 2026, a similar lawsuit was filed in the same court against nearly all of the Individual Defendants, except it did not name Dallan Murray and added Ian Estepan as a defendant. The Company is named as a nominal defendant in both lawsuits (the “D. Mass. Derivative Actions”). The D. Mass. Derivative Actions assert claims based upon substantially similar statements and theories as the underlying Securities Action, along with allegations concerning statements regarding Company performance and compensation from the Company's 2025 annual proxy statement; the Company's November 2024 repurchase of Company common stock; and certain director stock sales. The D. Mass. Derivative Actions allege, among other claims, breaches of the Individual Defendants’ fiduciary duties and violations of Section 14(a) of the Exchange Act. The D. Mass. Derivative Actions further seek, among other relief, unspecified damages and an order directing the Company to take all necessary actions to reform and improve its corporate governance and internal procedures. On June 22, 2026, the court stayed the first D. Mass. Derivative Action until the Securities Action is resolved and ordered that the terms of the stay shall apply to stockholder derivative actions later filed in that court. On September 14, 2026, the court ordered that the second D. Mass. Derivative Action be consolidated with the first (the "D. Mass. Consolidated Derivative Action") and stayed pursuant to the same terms.
On September 3, 2026, a different shareholder derivative lawsuit was filed in the Supreme Court of the State of New York (the “N.Y. State Derivative Action”) against the same Individual Defendants named in the S.D.N.Y. Consolidated Derivative Action and the first D. Mass. Derivative Action. The Company is named as a nominal defendant. The N.Y. State Derivative Action asserts claims based on substantially similar statements and theories as the Securities Action, S.D.N.Y. Consolidated Derivative Action, and D. Mass. Consolidated Derivative Action. The N.Y. State Derivative Action seeks, among other relief, unspecified damages and an order directing the Individual Defendants to take all necessary actions to reform and improve the Company’s corporate governance and internal procedures.
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