Sarepta Therapeutics, Inc. 10-Q

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UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 10-Q

(Mark One)


☒	QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended March 31, 2026


☐	TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from to

Commission file number 001-14895

SAREPTA THERAPEUTICS, INC.

(Exact name of registrant as specified in its charter)


Delaware		93-0797222
(State or other jurisdiction of incorporation or organization)		(I.R.S. Employer Identification No.)

215 First Street, Suite 415 Cambridge, MA		02142
(Address of principal executive offices)		(Zip Code)

Registrant’s telephone number, including area code: (617) 274-4000

Securities registered pursuant to Section 12(b) of the Act:


Title of each class	Trading Symbol	Name of exchange on which registered
Common Stock, $0.0001 par value per share	SRPT	The Nasdaq Global Select Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒

Indicate the number of shares outstanding of each of the issuer’s classes of common stock, as of the latest practicable date.


Common Stock with $0.0001 par value		105,576,222
(Class)		(Outstanding as of May 1, 2026)

SAREPTA THERAPEUTICS, INC.

FORM 10-Q

INDEX


		Page
PART I — FINANCIAL INFORMATION

Item 1.	Financial Statements (unaudited)	3

	Condensed Consolidated Balance Sheets — As of March 31, 2026 and December 31, 2025	3

	Condensed Consolidated Statements of Comprehensive Income (Loss) — For the Three Months Ended March 31, 2026 and 2025	4

	Condensed Consolidated Statements of Stockholders’ Equity — For the Three Months Ended March 31, 2026 and 2025	5

	Condensed Consolidated Statements of Cash Flows — For the Three Months Ended March 31, 2026 and 2025	6

	Notes to Condensed Consolidated Financial Statements	7

Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	23

Item 3.	Quantitative and Qualitative Disclosures about Market Risk	36

Item 4.	Controls and Procedures	37

PART II — OTHER INFORMATION

Item 1.	Legal Proceedings	38

Item 1A.	Risk Factors	38

Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	79

Item 3.	Defaults Upon Senior Securities	79

Item 4.	Mine Safety Disclosures	79

Item 5.	Other Information	80

Item 6.	Exhibits	80

Exhibits		81

Signatures		82

PART I — FINANCIAL INFORMATION

Item 1. Financial Statements

SAREPTA THERAPEUTICS, INC.

CONDENSED CONSOLIDATED BALANCE SHEETS

(unaudited, in thousands, except share and per share amounts)


	As of March 31, 2026			As of December 31, 2025
Assets
Current assets:
Cash and cash equivalents	$	464,450		$	801,282
Short-term investments		188,739			138,368
Accounts receivable, net		394,817			398,233
Inventory		1,001,112			914,744
Manufacturing-related deposits and prepaids		72,785			113,455
Other current assets		187,973			171,856
Total current assets		2,309,876			2,537,938
Property and equipment, net		336,241			345,125
Right of use assets		124,059			125,495
Non-current inventory		174,865			184,543
Non-current investments		81,936			1,048
Other non-current assets		151,957			155,554
Total assets	$	3,178,934		$	3,349,703

Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	70,376		$	280,841
Accrued expenses		302,372			359,659
Deferred revenue, current portion		116,037			443,397
Other current liabilities		10,379			11,393
Total current liabilities		499,164			1,095,290
Long-term debt		838,162			828,974
Lease liabilities, net of current portion		198,505			199,378
Deferred revenue, net of current portion		136,354			83,910
Other non-current liabilities		1,647			1,529
Total liabilities		1,673,832			2,209,081
Commitments and contingencies (Note 16)
Stockholders’ equity:
Preferred stock, $0.0001 par value, 3,333,333 shares authorized; none issued and outstanding		—			—
Common stock, $0.0001 par value, 198,000,000 shares authorized; 106,226,788 and 105,571,146 issued and outstanding, respectively, at March 31, 2026 and 105,615,096 and 104,964,220 issued and outstanding, respectively, at December 31, 2025		11			11
Treasury stock, at cost, 655,642 and 650,876 shares at March 31, 2026 and December 31, 2025, respectively		(25,263	)		(25,263	)
Additional paid-in capital		6,076,500			6,042,586
Accumulated other comprehensive (loss) income, net of tax		(121	)		272
Accumulated deficit		(4,546,025	)		(4,876,984	)
Total stockholders’ equity		1,505,102			1,140,622
Total liabilities and stockholders’ equity	$	3,178,934		$	3,349,703

See accompanying notes to unaudited condensed consolidated financial statements.

SAREPTA THERAPEUTICS, INC.

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE INCOME (LOSS)

(unaudited, in thousands, except per share amounts)


	For the Three Months Ended March 31,
	2026			2025
Revenues:
Products, net	$	330,515		$	611,523
Collaboration and other		400,288			133,333
Total revenues		730,803			744,856

Cost and expenses:
Cost of sales (excluding amortization of in-licensed rights)		108,768			137,564
Research and development		153,960			773,448
Selling, general and administrative		108,951			133,629
Amortization of in-licensed rights		691			601
Total cost and expenses		372,370			1,045,242
Operating income (loss)		358,433			(300,386	)

Other loss, net:
Other expense, net		(15,259	)		(83,132	)

Income (loss) before income tax expense		343,174			(383,518	)
Income tax expense		12,215			63,990
Net income (loss)	$	330,959		$	(447,508	)

Other comprehensive income (loss):
Unrealized (losses) gains on investments, net of tax		(393	)		252
Total other comprehensive (loss) income		(393	)		252
Comprehensive income (loss)	$	330,566		$	(447,256	)

Earnings (loss) per share:
Basic	$	3.15		$	(4.60	)
Diluted	$	2.88		$	(4.60	)

Weighted average number of shares of common stock used in computing earnings (loss) per share:
Basic		104,988			97,362
Diluted		121,916			97,362

See accompanying notes to unaudited condensed consolidated financial statements.

SAREPTA THERAPEUTICS, INC.

CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY

(unaudited, in thousands)


													Accumulated
											Additional		Other						Total
	Common Stock				Treasury Stock						Paid-In		Comprehensive			Accumulated			Stockholders'
	Shares		Amount		Shares			Amount			Capital		Income (Loss)			Deficit			Equity
BALANCE AT DECEMBER 31, 2025		105,615	$	11		(651	)	$	(25,263	)	$	6,042,586	$	272		$	(4,876,984	)	$	1,140,622
Exercise of options for common stock		25		—		—			—			241		—			—			241
Vest of restricted stock units		587		—		(5	)		—			—		—			—			—
Stock-based compensation		—		—		—			—			33,673		—			—			33,673
Unrealized losses on investments, net of tax		—		—		—			—			—		(393	)		—			(393	)
Net income		—		—		—			—			—		—			330,959			330,959
BALANCE AT MARCH 31, 2026		106,227	$	11		(656	)	$	(25,263	)	$	6,076,500	$	(121	)	$	(4,546,025	)	$	1,505,102


													Accumulated
											Additional		Other						Total
	Common Stock				Treasury Stock						Paid-In		Comprehensive			Accumulated			Stockholders'
	Shares		Amount		Shares			Amount			Capital		(Loss) Income			Deficit			Equity
BALANCE AT DECEMBER 31, 2024		96,900	$	10		—		$	—		$	5,738,924	$	(218	)	$	(4,210,974	)	$	1,527,742
Exercise of options for common stock		146		—		—			—			9,036		—			—			9,036
Vest of restricted stock units		1,135		—		—			—			—		—			—			—
Issuance of common stock under employee stock purchase plan		74		—		—			—			6,645		—			—			6,645
Stock-based compensation		—		—		—			—			46,556		—			—			46,556
Unrealized gains on investments, net of tax		—		—		—			—			—		252			—			252
Net loss		—		—		—			—			—		—			(447,508	)		(447,508	)
BALANCE AT MARCH 31, 2025		98,255	$	10		—		$	—		$	5,801,161	$	34		$	(4,658,482	)	$	1,142,723

See accompanying notes to unaudited condensed consolidated financial statements.

SAREPTA THERAPEUTICS, INC.

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS

(unaudited, in thousands)


	For the Three Months Ended March 31,
	2026			2025
Cash flows from operating activities:
Net income (loss)	$	330,959		$	(447,508	)
Adjustments to reconcile net income (loss) to net cash flow from operating activities
Stock-based compensation		29,399			41,428
Depreciation and amortization		10,594			9,978
Non-cash interest expense		9,612			1,254
Reduction in the carrying amounts of the right of use assets		3,110			3,934
Loss on strategic investments		1,712			90,728
Accretion of investment discount, net		(530	)		(1,912	)
Other		(1,288	)		438
Changes in operating assets and liabilities, net:
Decrease (increase) in accounts receivable		3,416			(57,383	)
Decrease in manufacturing-related deposits and prepaids		43,487			71,136
Increase in inventory		(69,756	)		(177,359	)
Increase in other assets		(14,547	)		(15,067	)
Decrease in deferred revenue		(274,916	)		(85,397	)
Decrease in accounts payable		(210,465	)		(58,337	)
(Decrease) increase in accrued expenses		(60,026	)		31,652
(Decrease) increase in lease liabilities and other liabilities		(3,444	)		8,978
Net cash used in operating activities		(202,683	)		(583,437	)

Cash flows from investing activities:
Purchase of available-for-sale securities		(170,197	)		(44,658	)
Maturity and sales of available-for-sale securities		38,888			40,758
Purchase of property and equipment		(2,081	)		(43,651	)
Purchase of intangible assets		(1,000	)		(2,238	)
Acquisition of strategic investment		—			(241,388	)
Net cash used in investing activities		(134,390	)		(291,177	)

Cash flows from financing activities:
Proceeds from exercise of stock options and purchase of stock under the Employee Stock Purchase Program		241			15,681
Proceeds from borrowings under the Revolving Credit Facility		250,000			—
Repayments of borrowings under the Revolving Credit Facility		(250,000	)		—
Payments related to obtaining the Revolving Credit Facility		—			(3,210	)
Net cash provided by financing activities		241			12,471

Decrease in cash, cash equivalents and restricted cash		(336,832	)		(862,143	)

Cash, cash equivalents and restricted cash:
Beginning of period		814,407			1,118,589
End of period	$	477,575		$	256,446

Reconciliation of cash, cash equivalents and restricted cash:
Cash and cash equivalents	$	464,450		$	240,867
Restricted cash in other assets		13,125			15,579
Total cash, cash equivalents and restricted cash	$	477,575		$	256,446

Supplemental disclosure of cash flow information:
Cash paid during the period for income taxes	$	1,728		$	835
Cash paid during the period for interest	$	23,419		$	7,188
Supplemental schedule of non-cash activities:
Intangible assets and property and equipment included in accounts payable and accrued expenses	$	5,953		$	29,225
Lease liabilities arising from obtaining right of use assets	$	—		$	5,232
Capitalized stock-based compensation and depreciation as inventory	$	6,934		$	7,285

See accompanying notes to unaudited condensed consolidated financial statements.

SAREPTA THERAPEUTICS, INC.

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, small interfering RNA ("siRNA") knockdown therapies, gene therapy 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”), 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”), 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.

The Company's gene therapy product, ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-(“AAV”) based gene therapy, was initially granted accelerated approval from 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 response to a request from the FDA that we voluntarily stop all shipments of ELEVIDYS in the U.S., we temporarily suspended all shipments of ELEVIDYS in the U.S., effective July 22, 2025. On July 28, 2025, the FDA informed us that it recommended the removal of the voluntary hold for ambulatory patients. On July 31, 2025, we resumed shipments of ELEVIDYS for ambulatory patients in the U.S. 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-ambulant 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 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 March 31, 2026, the Company had $748.3 million of cash, cash equivalents, restricted cash and investments, consisting of $464.5 million of cash and cash equivalents, $270.7 million of investments and $13.1 million of non-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 future cash inflows from operations and availability under the Company's Revolving Credit Facility (please refer to Note 13 Revolving Credit Facility for 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 establish collaborations with or license its technology to other companies.

2. 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 in one 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 months ended March 31, 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 March 31, 2026, the Company’s cash was concentrated at five 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, 2026, three entities accounted for 33%, 28% and 20% of accounts receivable, net, respectively. As of December 31, 2025, four entities accounted for 33%, 26%, 20% and 10% 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 Pronouncements of the Annual Report on Form 10-K for the year ended December 31, 2025.

3. LICENSE AND COLLABORATION AGREEMENTS

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 Collaboration Agreement”) that subsequently became effective as of February 7, 2025 (the “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 following programs:


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; 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”).

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, 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.

During the three months ended March 31, 2025, 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. 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 on the Effective Date. 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 were 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 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 months ended March 31, 2026 and March 31, 2025, the Company recorded $8.5 million and $2.3 million of research and development expense, respectively, 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 Effective Date. The Annual Fees are contingent upon the Company not terminating the Arrowhead Collaboration Agreement. For the three months ended March 31, 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 income (loss). 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. During the three months ended March 31, 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. Hoffman-La Roche Ltd.

For the three months ended March 31, 2026 and 2025, the Company recognized $365.0 million and $112.0 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 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 2026, Roche declined to exercise an Option for certain program rights, which resulted in the immediate recognition of $325.0 million of collaboration revenue during the three months ended March 31, 2026. Additionally, Roche dosed the first commercial patient in Japan during the three months ended March 31, 2026, which resulted in the recognition of a $40.0 million milestone payment as collaboration revenue under the Roche Collaboration Agreement. 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 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, 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 income (loss). During the three months ended March 31, 2026, Roche product cost of sales exceeded Roche contract manufacturing revenue as a portion of scrapped and

expired materials as well as the write-offs of certain batches of ELEVIDYS that did not meet quality specifications in the period were allocated to Roche product cost of sales. The following table summarizes certain Roche activity for each of the periods indicated:


	For the Three Months Ended March 31,
	2026			2025
	(in thousands)
Contract manufacturing revenue	$	31,105		$	17,379
Royalty revenue		4,183			3,954
Roche product cost of sales		(39,653	)		(12,143	)

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, 2026 and 2025, costs reimbursable by Roche and reflected as a reduction to operating expenses were $8.2 million and $28.5 million, respectively. As of March 31, 2026 and December 31, 2025, there was $136.4 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 March 31, 2026 and December 31, 2025, the Company had total deferred revenue of $252.4 million and $527.3 million, of which $116.0 million and $443.4 million are classified as current, respectively. As of March 31, 2026, all of the deferred revenue related to the Roche Supply Agreement.

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, 2026, the Company may be obligated to make up to $12.1 billion of future development, regulatory, commercial and up-front royalty payments associated with its collaboration and license agreements.

4. 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, 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:


	Fair Value Measurement as of March 31, 2026
	Total		Level 1		Level 2		Level 3
	(in thousands)
Assets
Money market funds	$	216,673	$	216,673	$	—	$	—
Government and government agency bonds		188,232		—		188,232		—
Corporate bonds		79,696		—		79,696		—
Strategic investments		7,795		2,364		—		5,431
Certificates of deposit		2,898		—		2,898		—
Deferred compensation plan assets		995		995		—		—
Total assets	$	496,289	$	220,032	$	270,826	$	5,431


	Fair Value Measurement as of December 31, 2025
	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 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 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 March 31, 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 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 investments during the three months ended March 31, 2026 or 2025, as no impairment indicators were identified nor were similar securities issued.

The fair values of the Company's 1.25% senior convertible notes due September 15, 2027 (the “2027 Notes”) and the Company’s 4.875% senior convertible 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. The following table summarizes the Company's debt instruments by indenture for the periods indicated:


	As of March 31, 2026		As of December 31, 2025
	(in thousands)
Net carrying value of the 2027 Notes	$	157,642	$	157,478
Net carrying value of the 2030 Notes		680,520		671,496
Total carrying value of debt instruments	$	838,162	$	828,974

Fair value of 2027 Notes	$	146,002	$	143,076
Fair value of 2030 Notes		732,127		721,791
Total fair value of debt instruments	$	878,129	$	864,867

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.

5. 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 March 31, 2026		As of December 31, 2025
	(in thousands)
Money market funds	$	216,673	$	474,914
Corporate bonds		151		—
Government and government agency bonds		—		89,914
Total	$	216,824	$	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 March 31, 2026 and December 31, 2025 was approximately nine and three months, respectively. All of the Company's non-current investments as of March 31, 2026 and December 31, 2025 had maturities between one and two years.

The following tables summarize the Company’s financial instruments for each of the periods indicated:


	As of March 31, 2026
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses			Fair Market Value
	(in thousands)
Cash and money market funds	$	464,299	$	—	$	—		$	464,299
Government and government agency bonds		188,291		70		(129	)		188,232
Corporate bonds		79,745		10		(59	)		79,696
Certificates of deposit		2,898		—		—			2,898
Total cash, cash equivalents and investments	$	735,233	$	80	$	(188	)	$	735,125
As reported:
Cash and cash equivalents	$	464,450	$	—	$	—		$	464,450
Short-term investments		188,785		31		(77	)		188,739
Non-current investments		81,998		49		(111	)		81,936
Total cash, cash equivalents and investments	$	735,233	$	80	$	(188	)	$	735,125


	As of December 31, 2025
	Amortized Cost		Gross Unrealized Gains		Gross Unrealized Losses		Fair Market Value
	(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

6. 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,
	2026		2025
	(in thousands)
PMO Products
United States	$	188,789	$	185,828
Rest of World		39,761		50,710
Total PMO product revenues, net	$	228,550	$	236,538
ELEVIDYS
United States	$	101,965	$	374,985
Total ELEVIDYS product revenues, net	$	101,965	$	374,985
Total product revenues, net	$	330,515	$	611,523

For the three months ended March 31, 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:

As of March 31, 2026 and December 31, 2025, the Company's accounts receivable, net were $394.8 million and $398.2 million, respectively, both of which were related to product sales, net of discounts and allowances. As of March 31, 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		23,997			40,700			3,587			24,271			92,555
Adjustments relating to prior periods		—			(687	)		—			(21	)		(708	)
Payments/credits		(25,903	)		(43,191	)		(2,069	)		(31,751	)		(102,914	)
Balance, as of March 31, 2026	$	23,657		$	113,407		$	5,349		$	46,556		$	188,969


	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		73,443			58,798			5,552			27,901			165,694
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

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, 2026		As of December 31, 2025
	(in thousands)
Reduction to accounts receivable, net	$	74,684	$	82,518
Component of accrued expenses		114,285		117,518
Total reserves	$	188,969	$	200,036

7. INVENTORY

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


	As of March 31, 2026		As of December 31, 2025
	(in thousands)
Raw materials	$	136,958	$	141,278
Work in progress		981,044		892,689
Finished goods		57,975		65,320
Total inventory	$	1,175,977	$	1,099,287

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. The Company classifies inventory associated with ELEVIDYS as non-current inventory when consumption of the inventory is expected beyond the Company's normal ELEVIDYS inventory operating cycle of approximately 3.5 years. Non-current inventory consists of raw materials and work in progress associated with the PMO Products and 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 March 31, 2026		As of December 31, 2025
	(in thousands)
Balance sheet classification:
Inventory	$	1,001,112	$	914,744
Non-current inventory		174,865		184,543
Total inventory	$	1,175,977	$	1,099,287

For the three months ended March 31, 2026 and 2025, there were no material inventory losses for write-downs of excess and obsolete inventory.

8. OTHER ASSETS

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


	As of March 31, 2026		As of December 31, 2025
	(in thousands)
Collaboration and other receivables	$	136,446	$	104,117
Prepaid maintenance services		14,584		12,639
Tax-related receivables and prepaids		14,033		15,604
Prepaid clinical and pre-clinical expenses		7,859		7,976
Interest receivable		2,616		1,656
Prepaid insurance		2,217		4,004
Prepaid commercial expenses		2,155		2,290
Prepaid regulatory		1,799		2,335
Prepaid employee benefits		1,037		12,034
Other		5,227		9,201
Total other current assets	$	187,973	$	171,856

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


	As of March 31, 2026		As of December 31, 2025
	(in thousands)
Manufacturing-related deposits and prepaids	$	90,073	$	92,890
Intangible assets, net		31,384		28,949
Restricted cash*		13,125		13,125
Strategic investments		7,795		9,520
Deferred tax asset		2,808		2,822
Prepaid maintenance services		2,652		3,058
Other		4,120		5,190
Total other non-current assets	$	151,957	$	155,554

* 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.

9. ACCRUED EXPENSES

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


	As of March 31, 2026		As of December 31, 2025
	(in thousands)
Product revenue related reserves	$	114,285	$	117,518
Accrued contract manufacturing costs		76,607

Large accelerated filer

Accelerated filer

Non-accelerated filer

Smaller reporting company

Emerging growth company

For the Three Months Ended
March 31,

Product revenues, net

Customer 1

Customer 2