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Item 2 — Management's Discussion and Analysis
Sarepta Therapeutics, Inc. · 10-Q · Q2 FY2026 · Period ended Jun 30, 2026
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The purpose of Management's Discussion and Analysis of Financial Condition and Results of Operations is to provide an understanding of the financial condition, changes in financial condition and results of operations of Sarepta Therapeutics, Inc. This section should be read in conjunction with our unaudited condensed consolidated financial statements and related notes included in Part I, Item 1 of this Quarterly Report on Form 10-Q and the section contained in our Annual Report on Form 10-K for the year ended December 31, 2025 under the caption “Part II-Item 7 — Management’s Discussion and Analysis of Financial Condition and Results of Operations”. This Quarterly Report on Form 10-Q contains certain forward-looking statements, which are often identified by words such as “believe,” “anticipate,” “expect,” “intend,” “plan,” “will,” “may,” “estimate,” “could,” “continue,” “ongoing,” “predict,” “potential,” “likely,” “seek” and other similar expressions, as well as variations or negatives of these words. These statements relate to our future plans, objectives, expectations, intentions and financial performance and the assumptions that underlie these statements. These forward-looking statements include, but are not limited to:
•our belief that our proprietary technology, technology platforms and collaborations can be used to develop potential therapeutic candidates to treat a broad range of diseases;
•our expectation that our partnerships with manufacturers will support our clinical and commercial manufacturing capacity for our products and product candidates, including our phosphorodiamidate morpholino oligomer (“PMO”), gene therapy, SRP-9003 Limb-girdle muscular dystrophy (“LGMD”), and small interfering RNA (“siRNA”) programs, while also acting as a manufacturing platform for potential future programs, and our belief that our current network of manufacturing partners is able to fulfill the requirements of our commercial plan;
•the possible impacts of the clinical hold the U.S. Food and Drug Administration (the “FDA") has placed on our investigational use gene therapy clinical trials for LGMD in July 2025 and the revocation of the platform technology designation for our AAVrh74 platform technology previously granted on June 2, 2025;
•the possible impacts of the results of our ESSENCE confirmatory trial for VYONDYS 53 and AMONDYS 45, including the timing and outcome of additional results, potential regulatory actions from the FDA, including directives to remove these products from the market or alter labels, patient demand for these products and changes to reimbursement and coverage by insurance companies;
•the possible impacts of the ELEVIDYS Suspension (as defined herein);
•the estimated and potential impacts of the strategic restructuring plan announced in July 2025;
•our expectation that our partnership with Catalent, Inc. (“Catalent”) will support our clinical and commercial manufacturing demand for our Duchenne gene therapy program and SRP-9003 LGMD program, while also acting as a manufacturing platform for any potential future gene therapy programs;
•our expectation that Aldevron LLC (“Aldevron”) will provide Good Manufacturing Processes (“GMP”)-grade plasmid for our Duchenne muscular dystrophy (“Duchenne”) gene therapy program and our SRP-9003 LGMD program, as well as plasmid source material for future gene therapy programs;
•the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business, including the addition of a boxed warning for acute liver injury ("ALI") and acute liver failure (“ALF”) and removal of the non-ambulatory population from the Indication and Usage section of the Prescribing Information for ELEVIDYS, as well as the development of our product candidates and our financial and contractual obligations;
•estimated timelines and milestones for the remainder of 2026 and beyond, including discussions with the FDA regarding ELEVIDYS, VYONDYS 53, AMONDYS 45 and SRP-9003, and sharing data for certain of our siRNA product candidates, SRP-1001 and SRP-1003;
•our expectations regarding the ongoing study to evaluate the use of sirolimus as an enhanced immunosuppression regimen as part of treatment with ELEVIDYS for non-ambulant individuals living with Duchenne, including discussions with the FDA and possible impacts on the resumption of dosing in the non-ambulatory population;
•our engagement with regulatory authorities outside of the U.S. including the European Medicines Agency (the “EMA”);
•our ability to maintain, and our plan to continue building out our distribution network for in jurisdictions in which our products are approved or in which we are seeking to make our products available, commercially or through our EAP (as defined herein);
•our plan to expand our pipeline through internal research and development and through strategic transactions;
•the timely completion and satisfactory outcome of our post-marketing requirements and commitments, including verification of a clinical benefit for our products in confirmatory trials;
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•our ability to further secure long-term supply of our commercial products and our product candidates to satisfy our planned commercial, early access programs (“EAP”) and clinical needs;
•the possible impact of any executive, legislative or regulatory action and competing products on the commercial success of our products and our product candidates and our ability to compete against such products;
•our ability to enter into research, development or commercialization alliances with universities, hospitals, independent research centers, non-profit organizations, pharmaceutical and biotechnology companies and other entities for specific molecular targets or selected disease indications and our ability to selectively pursue opportunities to access certain intellectual property rights that complement our internal portfolio through license agreements or other arrangements;
•our expectation regarding the potential benefits of the partnership, licensing and/or collaboration arrangements and other strategic arrangements and transactions we have entered into or may enter into in the future;
•our plans and ability to file and progress to issue additional patent applications to enhance and protect our new and existing technologies and programs;
•the potential benefits of our technologies and programs, including those with strategic partners;
•our estimates regarding how long our currently available cash and cash equivalents will be sufficient to finance our operations and business plans and statements about our future capital needs;
•our estimates regarding future revenues, research and development expenses, other expenses, capital requirements and payments to third parties;
•our expectation regarding the impact of environmental laws and regulations on our business;
•our expectation regarding the outcomes or impacts of our ongoing litigations that we are currently, or may in the future become, party to;
•our expectation regarding our ability to satisfy the conditions to borrow under our Credit Agreement (defined below); and
•our beliefs and expectations regarding milestone, royalty or other payments that could be due to third parties under existing agreements.
We undertake no obligation to update any of the forward-looking statements contained in this Quarterly Report on Form 10-Q after the date of this report, except as required by law or the rules and regulations of the U.S. Securities and Exchange Commission (the “SEC”). We caution readers not to place undue reliance on forward-looking statements. Our actual results could differ materially from those discussed in this Quarterly Report on Form 10-Q. The forward-looking statements contained in this Quarterly Report on Form 10-Q, and other written and oral forward-looking statements made by us from time to time, are subject to certain risks and uncertainties that could cause actual results to differ materially from those anticipated in the forward-looking statements, including the risks, uncertainties and assumptions identified under the heading “Risk Factors” in this Quarterly Report on Form 10-Q.
Overview
We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, siRNA knockdown therapies, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying our proprietary, differentiated and innovative technologies, and through collaborations with our strategic partners, we have developed multiple approved products for the treatment of Duchenne and are developing potential therapeutic candidates for a broad range of diseases and disorders, including LGMD. We are also developing potential therapeutic candidates through our partnered program with Arrowhead Pharmaceuticals, Inc. ("Arrowhead"), including Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia ("SCA"), and Huntington's disease ("HTT").
To date, we have developed and commercialized four products that have been approved by the FDA:
•The PMO Products:
oEXONDYS 51 (eteplirsen) Injection ("EXONDYS 51"), granted accelerated approval by the FDA in 2016, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping. EXONDYS 51 uses our phosphorodiamidate morpholino oligomer ("PMO") chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.
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oVYONDYS 53 (golodirsen) Injection ("VYONDYS 53"), granted accelerated approval by the FDA in 2019, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping. VYONDYS 53 uses our PMO chemistry and exon-skipping technology to skip exon 53 of the dystrophin gene.
oAMONDYS 45 (casimersen) Injection ("AMONDYS 45"), granted accelerated approval by the FDA in 2021, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 45 skipping. AMONDYS 45 uses our PMO chemistry and exon-skipping technology to skip exon 45 of the dystrophin gene.
•ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-("AAV") based gene therapy, 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. ELEVIDYS was previously granted accelerated approval by the FDA in June 2023 for the treatment of ambulatory patients aged four through five years with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene. In response to safety events announced in March and June 2025, we 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, to allow us the necessary time to respond to the FDA's requests for information and complete a labeling supplement process (the "ELEVIDYS Suspension"). 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, we announced a boxed warning for ALI and ALF and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information for ELEVIDYS.
We are in the process of conducting various clinical trials for our approved products, including studies that are required to comply with our post-marketing FDA requirements/commitments to verify and describe the clinical benefit of these products. In November 2025, we announced topline results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of two of our PMO Products: AMONDYS 45 and VYONDYS 53. The topline results did not show statistical significance on the study's primary endpoint. We intend to discuss with FDA the potential pathway forward and submitted supplemental new drug applications ("sNDAs") related to these products in April 2026, which the FDA accepted for filing in June 2026. We are also in the process of conducting various clinical trials for ELEVIDYS, including Cohort 8 of Study 9001-103 (ENDEAVOR), a study to evaluate the use of sirolimus as an enhanced immunosuppression regimen as part of treatment with ELEVIDYS for non-ambulant individuals living with Duchenne. We intend 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. Below, when referring to manufactured inventory for commercial sale in the U.S. and ex-U.S. territories, we use the designation of ELEVIDYS, which is recognized as inventory in our unaudited condensed consolidated balance sheets. Separately, when referring to manufactured product for clinical use, such as clinical trials, we use the designation of SRP-9001, which is recognized as research and development expense in our unaudited condensed consolidated statements of comprehensive (loss) income.
Our pipeline includes programs at various stages of discovery, pre-clinical and clinical development. Through our collaborations with our strategic partners, we are expanding into adjacent therapeutic areas. Our pipeline reflects our aspiration to apply our multifaceted approach and expertise in precision genetic medicine to make a profound difference in the lives of patients suffering from rare diseases. In July 2025, we announced a strategic restructuring plan designed to reduce operating expenses and align our cost structure with strategic priorities, aiming to enhance financial flexibility and meet our 2027 financial obligations (the "Restructuring"). The Restructuring suspended all development of our LGMD programs with the exception of SRP-9003.
Set forth below are our key clinical stage programs, including those in collaboration with our strategic partners, listed in the order of stage of development:
•SRP-9003 (LGMD, gene therapy program). SRP-9003, aims to treat LGMD2E, also known as beta-sarcoglycanopathy, a severe and debilitating form of LGMD characterized by progressive muscle fiber loss, inflammation and muscle fiber replacement with fat and fibrotic tissue. SRP-9003 is designed to transfect a gene that codes for and restores beta-sarcoglycan protein with the goal of restoring the dystrophin associated protein complex. SRP-9003 has generated positive pre-clinical safety and efficacy data utilizing the AAVrh.74 vector, the same vector used in our SRP-9001 gene therapy program. A Phase 1/2a trial of SRP-9003 commenced in the fourth quarter of 2018. In June 2020, we announced safety and expression results from three clinical trial participants in the high-dose cohort measured at 60 days, and one-year functional data from three clinical trial participants in the low-dose cohort. In March 2022, we announced 36-month functional data from three clinical trial participants in the low-dose cohort and 24-month functional data from two clinical trial participants in the high-dose cohort. In December 2024, we announced that we
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had completed enrollment and dosing in EMERGENE (Study SRP-9003-301), a Phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec). In October 2025, we announced safety and expression results from EMERGENE.
On July 21, 2025, we announced that the FDA placed a clinical hold on our investigational use gene therapy trials for LGMD, including our trials for product candidates SRP-9003 (LGMD2E/R4/bidridistrogene xeboparvovec), SRP-9004 (LGMD2D/patidistrogene bexoparvovec), SRP-6004 (LGMD2B/R2) and SRP-9005 (LGMD2C/R5 g-sarcoglycan), following the death of a patient in our Phase 1 LGMD clinical trial for SRP-9004. We previously announced on July 16, 2025 that we had suspended each of the LGMD programs mentioned above as part of the Restructuring, with the exception of SRP-9003. In December 2025, the FDA confirmed that we remain on clinical hold and informed us that it requires data from the study of sirolimus as an immunosuppressant before accepting a biologic license application ("BLA") for SRP-9003.We anticipate re-engaging with the agency on next steps for the program after we receive data from Cohort 8 of Study 9001-103 (ENDEAVOR).
•SRP-1003 (DM1). DM1 is an autosomal dominant, debilitating, chronic progressive multisystem disorder characterized by an expansion of a highly unstable CUGexp in the dystrophia myotonica protein kinase ("DMPK") gene. Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often have cardiac conduction abnormalities, and may become physically disabled and have a shortened life span. SRP-1003 is designed to reduce expression of the DMPK gene. There is currently no approved disease-modifying therapy for DM1. We are currently investigating SRP-1003 in a Phase 1/2 clinical trial, which our strategic partner Arrowhead has conducted to date. We shared early results from this trial in March 2026, and expect to share further data in the second half of 2026. We are in the process of transitioning sponsorship of this study to Sarepta.
•SRP-1001 (FSHD). FSHD is a rare genetic disease in which the body is unable to maintain complete epigenetic suppression of DUX4 expression in differentiated skeletal muscle, leading to overexpression of DUX4, which is myotoxic and can lead to muscle degeneration. SRP-1001 is designed to selectively target and knockdown DUX4 using RNAi, with the goal of preventing or reversing downstream myotoxicity and lead to muscle repair and improvement in muscle function in patients. There are currently no cures or approved disease-modifying treatments for FSHD. We are currently investigating SRP-1001 in a Phase 1/2 clinical trial, which our strategic partner Arrowhead has conducted to date. We shared early results from this trial in March 2026, and expect to share further data in the second half of 2026. We are in the process of transitioning sponsorship of this study to Sarepta.
•SRP-1005 (HTT). HTT is a rare and ultimately fatal inherited neurodegenerative disorder which is passed down from generation to generation within affected families. HTT is caused by a mutation in the gene for a protein called huntingtin, which leads to progressive deterioration of nerve cells in the brain, affecting cognition, movement, and behavior. Study SRP-1005-101 (INSIGHTT) is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants. SRP-1005 leverages an advanced TfR1 (transferrin receptor protein 1) approach that uses monovalent fragment antigen binding (fAb) designed for efficient delivery to the central nervous system. Subcutaneous administration is intended to avoid saturating the transferrin receptor and achieve constant and robust penetration across the blood brain barrier. During the second quarter of 2026, we enrolled the first patient in the study.
Manufacturing, Supply and Distribution
We have developed proprietary state-of-the-art Chemistry, Manufacturing and Controls ("CMC") capabilities that allow manufacturing and testing of our products and product candidates to support both clinical development and commercialization. We continue to refine and optimize our manufacturing processes and test methods. We have entered into certain manufacturing and supply arrangements with third-party suppliers (specialized contract manufacturing organizations, or "CMOs"), which will in part utilize these capabilities to support production of certain of our products and product candidates and their components. Specifically, we have entered into agreements with CMOs to produce custom starting materials, active pharmaceutical ingredients ("APIs"), drug product and finished goods for our products and product candidates for both commercial and clinical use. We also have opened facilities over the past several years to further enhance our internal research and development capabilities. However, we currently do not have internal GMP manufacturing capabilities to produce our products and product candidates for commercial and/or clinical use.
All of our CMO partners have extensive technical expertise, GMP experience and experience manufacturing medicinal products and, for commercial products, significant experience utilizing our specific technologies. Manufacturers and suppliers of our commercial products and product candidates are subject to the current GMP ("cGMP") requirements and other rules and regulations prescribed by the FDA and applicable foreign regulatory authorities. We depend on our third-party partners for continued compliance with cGMP requirements and applicable foreign standards.
We believe our current network of CMOs is able to fulfill our requirements for quantity, quality and purity of our commercial products and product candidates, and is capable of expanding capacity as needed. Additionally, we have evaluated and will continue to
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evaluate further relationships with additional suppliers as appropriate for specific products, taking into account production volume, logistics, business continuity and other routine business considerations.
Our gene therapy manufacturing capabilities continue to benefit from partnerships with Aldevron and Catalent. We utilize a hybrid development and manufacturing strategy in which we rely on experienced contract manufacturing partners to develop, manufacture and commercialize our gene therapy programs in partnership with our internal expertise relative to AAV-based development and manufacturing. Catalent supports our clinical and commercial manufacturing demand for ELEVIDYS and our SRP-9003 LGMD program, while also acting as a potential manufacturing partner for potential future gene therapy programs. Aldevron provides plasmid for ELEVIDYS and is expected to provide plasmid source material for any future gene therapy programs. The collaboration integrates process development, clinical and commercial production and testing.
Our PMO commercial products are distributed in the U.S. through a limited network of home infusion specialty pharmacy providers that deliver the medication to patients and a specialty distributor that distributes our products to hospitals and hospital outpatient clinics. With respect to the precommercial distribution of our products to patients outside of the U.S., we have contracted with third-party distributors and service providers to distribute our products in certain countries through our EAPs. We plan to continue building out our network for commercial distribution in jurisdictions in which our products are approved or in which we are seeking approval for our products.
The U.S. distribution model for ELEVIDYS employs multiple distribution partners that include third-party logistics providers as well as a limited network of specialty pharmacy providers that provide the medication to hospitals for infusion.
With respect to the siRNA programs in our portfolio, we collaborate with our partner Arrowhead to maintain continuity of manufacturing and testing services for ongoing and future clinical trials. Arrowhead’s cGMP manufacturing facility has the capability to manufacture drug substance at multiple scales available and can expand capacity as needed to support future clinical and potential commercial-scale needs. We plan to leverage existing contract partners, which have technical expertise and experience working with siRNA therapies, for the manufacture of drug product and finished goods as well as for distribution. As appropriate, we will evaluate additional relationships with supply partners for specific products, taking into account production volume, logistics, business continuity and other routine business considerations.
Cash, Cash Equivalents, Restricted Cash and Investments
As of June 30, 2026, we had $945.0 million of cash, cash equivalents, restricted cash and investments, consisting of $571.1 million of cash and cash equivalents, $362.7 million of investments and $11.2 million of non-current restricted cash and investments. We believe that our balance of cash, cash equivalents and investments, along with cash inflows from operations and availability under our Revolving Credit Facility (defined below), is sufficient to fund our current operational plan for at least the next twelve months.
Critical Accounting Policies and Estimates
The discussion and analysis of our financial condition and results of operations is based upon our unaudited condensed consolidated financial statements included elsewhere in this report. The preparation of our unaudited condensed consolidated financial statements in accordance with accounting principles generally accepted in the U.S. requires us to make estimates and judgments that affect the reported amounts of assets, liabilities, revenues and expenses and related disclosure of contingent assets and liabilities for the periods presented. Some of these judgments can be subjective and complex and, consequently, actual results may differ from these estimates. We believe that the estimates and judgments upon which we rely are reasonable based upon historical experience and information available to us at the time that we make these estimates and judgments. To the extent there are material differences between these estimates and actual results, our unaudited condensed consolidated financial statements will be affected. Although we believe that our judgments and estimates are appropriate, actual results may differ from these estimates. We believe the following accounting policies to be most critical to the judgments and estimates used in the preparation of our unaudited condensed consolidated financial statements:
•inventory; and
•income tax.
There have been no changes to our critical accounting policies and significant estimates as detailed in our Annual Report on Form 10-K for the year ended December 31, 2025.
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Results of Operations for the Three and Six Months Ended June 30, 2026 and 2025
The following tables set forth selected unaudited condensed consolidated statements of (loss) income data for each of the periods indicated:
For the Three Months Ended June 30,
2026 2025 Change Change
(in thousands, except per share amounts) $ %
Revenues:
Products, net $ 328,689 $ 513,123 $ (184,434 ) (36 )%
Collaboration and other 72,562 97,968 (25,406 ) (26 )%
Total revenues 401,251 611,091 (209,840 ) (34 )%
Cost and expenses:
Cost of sales (excluding amortization of in-licensed rights) 149,385 152,558 (3,173 ) (2 )%
Research and development 91,258 204,392 (113,134 ) (55 )%
Selling, general and administrative 107,600 137,897 (30,297 ) (22 )%
Litigation contingency charge 39,000 — 39,000 *
Amortization of in-licensed rights 717 667 50 7 %
Total cost and expenses 387,960 495,514 (107,554 ) (22 )%
Operating income 13,291 115,577 (102,286 ) (89 )%
Other (loss) income, net:
Other (expense) income, net (15,040 ) 38,061 (53,101 ) *
(Loss) income before income tax expense (benefit) (1,749 ) 153,638 (155,387 ) *
Income tax expense (benefit) 3,141 (43,254 ) 46,395 *
Net (loss) income $ (4,890 ) $ 196,892 $ (201,782 ) *
(Loss) earnings per share
Basic $ (0.05 ) $ 2.01 $ (2.06 ) *
Diluted $ (0.05 ) $ 1.89 $ (1.94 ) *
*Not meaningful
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For the Six Months Ended June 30,
2026 2025 Change Change
(in thousands, except per share amounts) $ %
Revenues:
Products, net $ 659,204 $ 1,124,646 $ (465,442 ) (41 )%
Collaboration and other 472,850 231,301 241,549 104 %
Total revenues 1,132,054 1,355,947 (223,893 ) (17 )%
Cost and expenses:
Cost of sales (excluding amortization of in-licensed rights) 258,153 290,122 (31,969 ) (11 )%
Research and development 245,218 977,840 (732,622 ) (75 )%
Selling, general and administrative 216,551 271,526 (54,975 ) (20 )%
Litigation contingency charge 39,000 — 39,000 *
Amortization of in-licensed rights 1,408 1,268 140 11 %
Total cost and expenses 760,330 1,540,756 (780,426 ) (51 )%
Operating income (loss) 371,724 (184,809 ) 556,533 *
Other loss, net
Other expense, net (30,299 ) (45,071 ) 14,772 (33 )%
Income (loss) before income tax expense 341,425 (229,880 ) 571,305 *
Income tax expense 15,356 20,736 (5,380 ) (26 )%
Net income (loss) $ 326,069 $ (250,616 ) $ 576,685 *
Earnings (loss) per share
Basic $ 3.10 $ (2.57 ) $ 5.67 *
Diluted $ 2.99 $ (2.57 ) $ 5.56 *
*Not meaningful
Revenues
Revenues from product sales are recorded at the time of sale at the net sales price (transaction price), which includes estimates of variable consideration for which reserves are established and which result from rebates, governmental chargebacks including Public Health Services ("PHS") chargebacks, prompt pay discounts, patient assistance programs and distribution fees. These reserves are based on the amounts earned or to be claimed on the related sales and are classified as reductions of accounts receivable (if no payments are required of us) or a current liability (if a payment is required of us). Our estimates take into consideration current contractual and statutory requirements. Actual amounts of consideration ultimately received or paid may differ from our estimates.
The following tables summarize the components of our net product revenues, by product group, for each of the periods indicated:
For the Three Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
PMO Products $ 230,564 $ 231,272 $ (708 ) (— )%
ELEVIDYS 98,125 281,851 (183,726 ) (65 )%
Products, net $ 328,689 $ 513,123 $ (184,434 ) (36 )%
For the Six Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
PMO Products $ 459,114 $ 467,810 $ (8,696 ) (2 )%
ELEVIDYS 200,090 656,836 (456,746 ) (70 )%
Products, net $ 659,204 $ 1,124,646 $ (465,442 ) (41 )%
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Net product revenues for the three and six months ended June 30, 2026 decreased by $184.4 million and $465.4 million compared with the same periods in 2025. The decreases were primarily driven by lower ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment.
Collaboration and other revenues primarily relate to our collaboration agreement (the “Roche Collaboration Agreement”) with F. Hoffmann-La Roche Ltd. (“Roche”). In addition, in accordance with the Roche Collaboration Agreement, the parties agreed to enter into a supply agreement in order for us 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. We are eligible to receive royalties on these sales. While the Roche Supply Agreement is in the process of being negotiated, we delivered commercial ELEVIDYS supply to Roche that was agreed upon on a purchase order-by-purchase order basis. The transaction price of the contract manufacturing revenue is estimated at contract inception and reassessed annually and on an as-needed basis when additional information becomes available.
The following tables summarize the components of our collaboration and other revenues for the periods indicated:
For the Three Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Contract manufacturing $ 54,399 $ 27,023 $ 27,376 101 %
License revenue 10,000 — 10,000 *
Royalty revenue 8,163 7,445 718 10 %
Collaboration revenue — 63,500 (63,500 ) (100 )%
Total collaboration and other $ 72,562 $ 97,968 $ (25,406 ) (26 )%
*Not meaningful
Collaboration and other revenues for the three months ended June 30, 2026 decreased by $25.4 million, or 26%, compared with the three months ended June 30, 2025. The decrease was primarily driven by the following:
•$27.4 million increase in contract manufacturing revenue primarily driven by increased deliveries of ELEVIDYS to Roche and increases in our manufacturing costs driven by greater-than-expected write-offs of batches of our products not meeting our quality specifications;
•$10.0 million increase in license revenue related to the grant of intellectual property rights under a certain license agreement executed during the three months ended June 30, 2026, with no similar activity during the same period in 2025; and
•$63.5 million decrease in collaboration revenue due to the recognition of milestone payments during the three months ended June 30, 2025 under the Roche Collaboration Agreement for the regulatory approval of ELEVIDYS in Japan (the “Japan Approval Milestone”) with no similar activity during the three months ended June 30, 2026. Please refer to Note 3, License and Collaboration Agreements for further discussion of the Japan Approval Milestone.
For the Six Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Collaboration revenue $ 365,000 $ 175,500 $ 189,500 108 %
Contract manufacturing 85,504 44,402 41,102 93 %
Royalty revenue 12,346 11,399 947 8 %
License revenue 10,000 — 10,000 *
Total collaboration and other $ 472,850 $ 231,301 $ 241,549 104 %
*Not meaningful
Collaboration and other revenues for the six months ended June 30, 2026 increased by $241.5 million, or 104%, compared with the six months ended June 30, 2025. The increase was primarily driven by the following:
•$189.5 million increase in collaboration revenue due to $365.0 million of revenue recognized in the six months ended June 30, 2026 related to (1) $325.0 million for Roche's declined option to acquire certain program rights previously recorded as deferred revenue, and (2) $40.0 million from a milestone recognized under the Roche Collaboration Agreement for the first commercial dosing of ELEVIDYS in Japan, as compared to $175.5 million of collaboration
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revenue recognized in the same period of 2025 related to the expiration of an option for a certain program and the Japan Approval Milestone payment of $63.5 million under the Roche Collaboration Agreement;
•$41.1 million increase in contract manufacturing revenue primarily driven by increased deliveries of ELEVIDYS to Roche and increases in our manufacturing costs driven by greater-than-expected write-offs of batches of our products not meeting our quality specifications; and
•$10.0 million increase in license revenue related to the grant of intellectual property rights under a certain license agreement executed during the six months ended June 30, 2026, with no similar activity during the same period in 2025.
Cost of sales (excluding amortization of in-licensed rights)
Our cost of sales (excluding amortization of in-licensed rights) consists of inventory costs that relate to sales of our products and the related overhead costs and royalty payments primarily to University of Western Australia ("UWA") for our PMO Products and to Nationwide Children's Hospital ("Nationwide") for ELEVIDYS. Cost of sales also include charges for inventory valuation for excess or obsolete inventory on hand and write-offs of batches of our products not meeting our quality specifications, including any associated costs expected to be reimbursed by Roche. Prior to receiving regulatory approval for our products, we expensed manufacturing and material costs as research and development expenses.
For the PMO Products, all previously expensed manufacturing costs had been fully consumed by December 2022. For ELEVIDYS sold in the three and six months ended June 30, 2026 and June 30, 2025, a portion of related manufacturing costs incurred had previously been expensed as research and development expenses. If certain product related costs had not previously been expensed as research and development expenses prior to receiving FDA approval, the incremental inventory costs related to ELEVIDYS sold, including products sold to Roche under the Roche Collaboration Agreement, would have been $2.8 million and $5.4 million higher for the three and six months ended June 30, 2026, respectively, as compared to $4.5 million and $18.2 million for the three and six months ended June 30, 2025, respectively.
The following tables summarize the components of our cost of sales (excluding amortization of in-licensed rights) for each of the periods indicated:
For the Three Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Product cost of sales (excluding Roche) $ 80,982 $ 116,743 $ (35,761 ) (31 )%
Roche product cost of sales** 58,203 21,818 36,385 167 %
Royalty payments 10,200 13,997 (3,797 ) (27 )%
Total cost of sales (excluding amortization of in-licensed rights) $ 149,385 $ 152,558 $ (3,173 ) (2 )%
**See above for further details regarding product supply sold to Roche via contract manufacturing under the Roche Collaboration Agreement.
The cost of sales (excluding amortization of in-licensed rights) for the three months ended June 30, 2026 decreased by $3.2 million, or 2%, compared with the same period in 2025. The decrease was primarily driven by the following:
•$35.8 million decrease in product cost of sales (excluding Roche) primarily due to decreased ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment as well as a lower allocation of costs to us associated with batches of ELEVIDYS not meeting our quality specifications, partially offset by an increase in batches of our PMO Products not meeting our quality specifications;
•$36.4 million increase in Roche product cost of sales primarily due to the timing of allocation of costs to Roche associated with write-offs of certain batches of ELEVIDYS not meeting our quality specifications, scrapped and expired materials and the volume of ELEVIDYS shipments under the Roche Collaboration Agreement; and
•$3.8 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.
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For the Six Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Product cost of sales (excluding Roche) $ 141,878 $ 226,501 $ (84,623 ) (37 )%
Roche product cost of sales** 97,856 33,961 63,895 188 %
Royalty payments 18,419 29,660 (11,241 ) (38 )%
Total cost of sales (excluding amortization of in-licensed rights) $ 258,153 $ 290,122 $ (31,969 ) (11 )%
**See above for further details regarding product supply sold to Roche via contract manufacturing under the Roche Collaboration Agreement.
The cost of sales (excluding amortization of in-licensed rights) for the six months ended June 30, 2026 decreased by $32.0 million, or 11%, compared with the same period in 2025. The decrease was primarily driven by the following:
•$84.6 million decrease in product cost of sales (excluding Roche) primarily due to decreased ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment as well as a lower allocation of costs to us associated with batches of ELEVIDYS not meeting our quality specifications, partially offset by an increase in batches of our PMO Products not meeting our quality specifications;
•$63.9 million increase in Roche product cost of sales primarily due to the timing of allocation of costs to Roche associated with write-offs of certain batches of ELEVIDYS not meeting our quality specifications, scrapped and expired materials and the volume of ELEVIDYS shipments under the Roche Collaboration Agreement; and
•$11.2 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.
Research and development expenses
Research and development expenses consist of costs associated with research activities as well as those associated with our product development efforts, conducting pre-clinical trials, clinical trials and manufacturing activities. Direct research and development expenses associated with our programs include clinical trial site costs, clinical manufacturing costs, costs incurred for consultants, up-front and collaboration license fees and milestones paid to third parties in connection with technologies that have not reached technological feasibility and do not have an alternative future use, and other external services, such as data management and statistical analysis support, and materials and supplies used in support of clinical programs. Indirect costs of our programs include salaries, stock-based compensation and allocation of our facility- and technology-related costs.
Research and development expenses represent a substantial percentage of our total operating expenses. We do not maintain or evaluate and, therefore, do not allocate internal research and development costs on a project-by-project basis. As a result, a significant portion of our research and development expenses are not tracked on a project-by-project basis, as the costs may benefit multiple projects.
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The following tables summarize our research and development expenses by project for each of the periods indicated:
For the Three Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
siRNA platform $ 13,789 $ 6,512 $ 7,277 112 %
SRP-9001 12,825 63,232 (50,407 ) (80 )%
Eteplirsen (exon 51) 5,896 11,103 (5,207 ) (47 )%
Other gene therapies 3,358 11,065 (7,707 ) (70 )%
LGMD platform 2,921 21,494 (18,573 ) (86 )%
Casimersen (exon 45) 274 1,388 (1,114 ) (80 )%
Golodirsen (exon 53) 131 1,577 (1,446 ) (92 )%
Other projects 904 9,885 (8,981 ) (91 )%
Internal research and development expenses 58,137 103,687 (45,550 ) (44 )%
Roche collaboration reimbursement (6,977 ) (25,551 ) 18,574 (73 )%
Total research and development expenses $ 91,258 $ 204,392 $ (113,134 ) (55 )%
For the Six Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Up-front and collaboration license fees $ 50,000 $ 583,787 $ (533,787 ) (91 )%
SRP-9001 36,266 132,089 (95,823 ) (73 )%
siRNA platform 29,849 8,805 21,044 239 %
Eteplirsen (exon 51) 10,413 21,443 (11,030 ) (51 )%
LGMD platform 8,752 49,149 (40,397 ) (82 )%
Other gene therapies 7,639 23,446 (15,807 ) (67 )%
Casimersen (exon 45) 1,168 5,554 (4,386 ) (79 )%
Golodirsen (exon 53) 887 3,797 (2,910 ) (77 )%
Other projects 1,705 13,718 (12,013 ) (88 )%
Internal research and development expenses 113,436 189,863 (76,427 ) (40 )%
Roche collaboration reimbursement (14,897 ) (53,811 ) 38,914 (72 )%
Total research and development expenses $ 245,218 $ 977,840 $ (732,622 ) (75 )%
The following tables summarize our research and development expenses by category for each of the periods indicated:
For the Three Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Compensation and other personnel expenses $ 25,775 $ 43,582 $ (17,807 ) (41 )%
Clinical trial expenses 23,102 33,511 (10,409 ) (31 )%
Facility- and technology-related expenses 19,165 25,045 (5,880 ) (23 )%
Stock-based compensation 8,509 15,277 (6,768 ) (44 )%
Professional services 5,379 9,044 (3,665 ) (41 )%
Manufacturing expenses 1,837 80,685 (78,848 ) (98 )%
Pre-clinical expenses 1,107 1,431 (324 ) (23 )%
Research and other 13,361 21,368 (8,007 ) (37 )%
Roche collaboration reimbursement (6,977 ) (25,551 ) 18,574 (73 )%
Total research and development expenses $ 91,258 $ 204,392 $ (113,134 ) (55 )%
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Research and development expenses for the three months ended June 30, 2026 decreased by $113.1 million, or 55%, compared with the three months ended June 30, 2025. The decrease was primarily driven by the following:
•$17.8 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
•$10.4 million decrease in clinical trial expenses primarily related to the pause of several studies for our LGMD programs as a result of our decision to reprioritize our pipeline announced in July 2025 (the "Pipeline Reprioritization") and the completion of certain SRP-9001 and PMO studies in 2025, partially offset by increased activity in our siRNA programs;
•$5.9 million decrease in facility- and technology-related expenses primarily as a result of the Restructuring;
•$6.8 million decrease in stock-based compensation primarily due to reduced headcount pursuant to the Restructuring and the completion of vesting for certain restricted stock units with performance conditions ("PSUs") in March 2026 with no similar award activity during the three months ended June 30, 2026;
•$3.7 million decrease in professional services primarily related to fewer third-party contractors used during the three months ended June 30, 2026 due to the Pipeline Reprioritization and the completion of certain SRP-9001 studies in 2025;
•$78.8 million decrease in manufacturing expenses primarily due to fewer clinical batches released for our SRP-9001 and LGMD programs as a result of our Pipeline Reprioritization, as well as our settlement agreement with Brammer Bio MA, LLC (“Brammer”) to resolve outstanding claims related to the termination of the Thermo Agreement (the “Brammer Settlement”) during the three months ended June 30, 2025, with no similar activity in 2026;
•$8.0 million decrease in research and other expenses primarily due to ongoing cost reduction efforts and the Pipeline Reprioritization; and
•$18.6 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to reduced SRP-9001 clinical supply as well as reduced headcount pursuant to the Restructuring resulting in lower reimbursable costs.
For the Six Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Compensation and other personnel expenses $ 51,909 $ 90,881 $ (38,972 ) (43 )%
Up-front and collaboration license fees 50,000 583,787 (533,787 ) (91 )%
Clinical trial expenses 41,406 66,658 (25,252 ) (38 )%
Facility- and technology-related expenses 37,901 49,267 (11,366 ) (23 )%
Manufacturing expenses 21,595 148,150 (126,555 ) (85 )%
Stock-based compensation 18,786 32,594 (13,808 ) (42 )%
Professional services 10,460 18,614 (8,154 ) (44 )%
Pre-clinical expenses 1,637 3,193 (1,556 ) (49 )%
Research and other 26,421 38,507 (12,086 ) (31 )%
Roche collaboration reimbursement (14,897 ) (53,811 ) 38,914 (72 )%
Total research and development expenses $ 245,218 $ 977,840 $ (732,622 ) (75 )%
Research and development expenses for the six months ended June 30, 2026 decreased by $732.6 million, or 75%, compared with the six months ended June 30, 2025. The decrease was primarily driven by the following:
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•$39.0 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
•$533.8 million decrease in up-front and collaboration license fees primarily due to the $583.6 million in payments allocated to the up-front license fee associated with our exclusive global licensing and collaboration agreement and stock purchase agreement (collectively, the “Arrowhead Collaboration Agreement”) with Arrowhead recognized during the six months ended June 30, 2025, partially offset by the $50.0 million annual collaboration license fee incurred and paid to Arrowhead pursuant to the Arrowhead Collaboration Agreement recognized during the six months ended June 30, 2026;
•$25.3 million decrease in clinical trial expenses primarily related to the pause of several studies for our LGMD programs as a result of our Pipeline Reprioritization and the completion of certain SRP-9001 and PMO studies in 2025, partially offset by increased activity in our siRNA programs;
•$11.4 million decrease in facility- and technology-related expenses primarily as a result of the Restructuring;
•$126.6 million decrease in manufacturing expenses primarily due to fewer clinical batches released for our SRP-9001 and LGMD programs as a result of the Pipeline Reprioritization, as well as the Brammer Settlement during the six months ended June 30, 2025, with no similar activity in 2026;
•$13.8 million decrease in stock-based compensation primarily related to reduced headcount pursuant to the Restructuring and the vesting of certain PSUs in March 2025 and March 2026;
•$8.2 million decrease in professional services primarily related to fewer third-party contractors used during the six months ended June 30, 2026 due to the completion of certain SRP-9001 and PMO studies in 2025 and the Pipeline Reprioritization;
•$1.6 million decrease in pre-clinical expenses primarily due to the Pipeline Reprioritization;
•$12.1 million decrease in research and other expenses primarily due to ongoing cost reduction efforts and the Pipeline Reprioritization; and
•$38.9 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to reduced SRP-9001 clinical supply as well as reduced headcount pursuant to the Restructuring resulting in lower reimbursable costs.
Selling, general and administrative expenses
Selling, general and administrative expenses consist of salaries, benefits, stock-based compensation and related costs for personnel in our executive, finance, legal, information technology, business development, human resources, commercial and other general and administrative functions. Other general and administrative expenses include an allocation of our facility- and technology-related costs and professional fees for legal, consulting and accounting services.
The following tables summarize our selling, general and administrative expenses by category for each of the periods indicated:
For the Three Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Professional services $ 38,232 $ 50,515 $ (12,283 ) (24 )%
Compensation and other personnel expenses 34,950 46,257 (11,307 ) (24 )%
Stock-based compensation 15,963 21,748 (5,785 ) (27 )%
Facility- and technology-related expenses 14,417 13,708 709 5 %
Other 4,230 5,807 (1,577 ) (27 )%
Roche collaboration reimbursement (192 ) (138 ) (54 ) 39 %
Total selling, general and administrative expenses $ 107,600 $ 137,897 $ (30,297 ) (22 )%
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Selling, general and administrative expenses for the three months ended June 30, 2026 decreased by $30.3 million, or 22% compared with the three months ended June 30, 2025. This decrease was primarily driven by the following:
•$12.3 million decrease in professional service expenses primarily related to reduced ELEVIDYS commercialization spending following the label update for ELEVIDYS which included only the ambulatory patient population for treatment and reduced legal spending driven by the settlement of a certain matter in 2025;
•$11.3 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
•$5.8 million decrease in stock-based compensation primarily related to reduced headcount pursuant to the Restructuring and the vesting of certain PSUs in March 2026 with no similar award activity during the three months ended June 30, 2026, partially offset by expense recognized for RSUs granted to our CEO in December 2025; and
•$1.6 million decrease in other expenses primarily related to the timing of charitable donations.
For the Six Months Ended June 30,
2026 2025 Change Change
(in thousands) $ %
Professional services $ 72,046 $ 92,745 $ (20,699 ) (22 )%
Compensation and other personnel expenses 70,746 92,042 (21,296 ) (23 )%
Stock-based compensation 35,085 45,859 (10,774 ) (23 )%
Facility- and technology-related expenses 28,526 26,520 2,006 8 %
Other 10,609 14,719 (4,110 ) (28 )%
Roche collaboration reimbursement (461 ) (359 ) (102 ) 28 %
Total selling, general and administrative expenses $ 216,551 $ 271,526 $ (54,975 ) (20 )%
Selling, general and administrative expenses for the six months ended June 30, 2026 decreased by $55.0 million, or 20%, compared with the six months ended June 30, 2025. This decrease was primarily driven by the following:
•$20.7 million decrease in professional service expenses primarily related to reduced ELEVIDYS commercialization spending following the label update for ELEVIDYS which included only the ambulatory patient population for treatment, reduced legal spending driven by the settlement of a certain matter in 2025 and reduced medical affairs spending related to our Pipeline Reprioritization;
•$21.3 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
•$10.8 million decrease in stock-based compensation primarily related to reduced headcount pursuant to the Restructuring and the vesting of certain PSUs in March 2025 and March 2026, partially offset by expense recognized for RSUs granted to our CEO in December 2025;
•$2.0 million increase in facility- and technology-related expenses primarily related to the utilization of our Bedford, Massachusetts facility, which was placed into service in June 2025; and
•$4.1 million decrease in other expenses primarily related to the timing of charitable donations.
Litigation contingency charge
During the three and six months ended June 30, 2026, we recorded a litigation contingency charge of $39.0 million related to the potential resolution of certain patent litigations. Following the parties' agreement in principle and based on management's assessment of the available information, we determined that a loss was probable and estimable as of June 30, 2026 and recognized our best estimate of the liability. The potential settlement remains outstanding subject to further negotiation and execution of definitive documentation as of the issuance of these unaudited condensed consolidated financial statements.
Amortization of in-licensed rights
Amortization of in-licensed rights relates to the agreements we entered into with UWA, Nationwide, BioMarin Pharmaceutical Inc. ("BioMarin") and Parent Project Muscular Dystrophy in April 2013, December 2016, July 2017 and May 2018, respectively. Each in-licensed right is being amortized on a straight-line basis over the remaining life of the relevant patent from the
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date the related fee was incurred, either the regulatory approval or the first commercial sale of the applicable product. For the three and six months ended June 30, 2026, we recorded amortization of in-licensed rights of approximately $0.7 million and $1.4 million, respectively. For the three and six months ended June 30, 2025, we recorded amortization of in-licensed rights of approximately $0.7 million and $1.3 million, respectively.
Other (expense) income, net
Other (expense) income, net primarily consists of the unrealized gain or loss from our investments in our strategic equity investments, interest expense on our 1.25% convertible senior notes due September 15, 2027 (the “2027 Notes”) and our 4.875% convertible senior notes due September 1, 2030 (the “2030 Notes”), interest income on our cash, cash equivalents and investments and accretion of investment discount. Our cash equivalents and investments consist of money market funds, government and government agency bonds, corporate bonds, commercial paper and certificates of deposit.
For the three months ended June 30, 2026, other expense, net increased by $53.1 million compared with the three months ended June 30, 2025. This was primarily due to a $37.2 million decrease in the gain on strategic investments primarily related to our investment in Arrowhead during the three months ended June 30, 2025, which we sold in August 2025, and a $16.3 million increase in interest expense due to the 2030 Notes carrying a higher interest rate than the 2027 Notes.
For the six months ended June 30, 2026, other expense, net decreased by $14.8 million compared with the six months ended June 30, 2025. The change was primarily due to a $51.8 million decrease in the loss on our strategic investments, primarily related to our investment in Arrowhead during the six months ended June 30, 2025. This was partially offset by a $33.5 million increase in interest expense due to the 2030 Notes carrying a higher interest rate than the 2027 Notes as well as a decrease in interest income as a result of reduced investment balances during the six months ended June 30, 2026, as compared to the six months ended June 30, 2025.
Income tax expense (benefit)
Income tax expense for the three and six months ended June 30, 2026 was $3.1 million and $15.4 million, respectively. Income tax (benefit) expense for the three and six months ended June 30, 2025 was $(43.3) million and $20.7 million, respectively. Income tax expense for all periods presented primarily relates to state income taxes as a result of taxable profits in certain states requiring the capitalization of research and development costs and states which have suspended or limited the utilization of net operating loss carryforwards. As of June 30, 2026, we continued to maintain a full valuation allowance against our deferred tax assets, with the exception of deferred tax assets in certain foreign jurisdictions. We continue to monitor the available evidence relative to recovery of our deferred tax assets and whether such evidence would be sufficient to conclude that it is more likely than not that such deferred tax assets may be partially or fully recoverable. If we were to remove our valuation allowance in part or full, any such adjustment could have a material impact on our effective tax rate in the applicable period.
Liquidity and Capital Resources
There have been no material changes to our obligations under lease or debt arrangements as reported in our Annual Report on Form 10-K for the year ended December 31, 2025, except for the partial draw downs and repayments of the five-year $600.0 million senior secured revolving credit facility entered into in February 2025 (the “Revolving Credit Facility”), as discussed in Note 13, Revolving Credit Facility.
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The following table summarizes our financial condition for each of the periods indicated:
As of June 30, 2026 As of December 31, 2025 Change Change
(in thousands) $ %
Financial assets:
Cash and cash equivalents $ 571,148 $ 801,282 $ (230,134 ) (29 )%
Short-term investments 217,341 138,368 78,973 57 %
Non-current investments 145,372 1,048 144,324 *
Restricted cash and investments 11,125 13,125 (2,000 ) (15 )%
Total cash, cash equivalents, restricted cash and investments $ 944,986 $ 953,823 $ (8,837 ) (1 )%
Borrowings:
Convertible debt $ 847,623 $ 828,974 $ 18,649 2 %
Total borrowings $ 847,623 $ 828,974 $ 18,649 2 %
Working capital:
Current assets $ 2,223,824 $ 2,537,938 $ (314,114 ) (12 )%
Current liabilities 502,908 1,095,290 (592,382 ) (54 )%
Total working capital $ 1,720,916 $ 1,442,648 $ 278,268 19 %
*Not meaningful
For the six months ended June 30, 2026, our principal sources of liquidity were primarily derived from the sales of our products, our collaboration arrangement with Roche, borrowings from the Revolving Credit Facility and the maturities and sales of investments. Please refer to Note 13, Revolving Credit Facility, for further discussion of our borrowing activity. Our principal uses of cash for the six months ended June 30, 2026 were our repayments of borrowings from the Revolving Credit Facility, the payment of $200.0 million for the second DM1 Milestone to Arrowhead, the $50.0 million annual license fee payment to Arrowhead, inventory commitments, research and development expenses, manufacturing costs, selling, general and administrative expenses, investments, capital expenditures and other working capital requirements.
For the year ended December 31, 2025, our principal sources of liquidity were primarily derived from the sales of our products, our collaboration arrangement with Roche, the maturities and sales of investments and proceeds from the exercise of stock options. Our principal uses of cash for 2025 were our $583.6 million up-front payment to Arrowhead, $241.4 million equity investment in Arrowhead's common stock and the $50.0 million cash component of the $100.0 million of the first DM1 Milestone, costs incurred as a result of the partial refinancings of the 2027 Notes in August and December 2025, inventory commitments, research and development expenses, manufacturing costs, selling, general and administrative expenses, investments, capital expenditures, share repurchases under our $500.0 million share repurchase program approved by the Board of Directors in November 2024 (the “2024 Repurchase Program”) and other working capital requirements. Please refer to our Annual Report on Form 10-K for the year ended December 31, 2025 for further discussion of share repurchases under the 2024 Repurchase Program.
Beyond June 30, 2027, our cash requirements will depend extensively on our ability to advance our research, development and commercialization of product candidates. We may seek additional financings primarily from, but not limited to, the sale and issuance of equity and debt securities, the licensing or sale of our technologies, and entering into additional government contracts and/or funded research and development agreements. Our future expenditures and long-term capital requirements may be substantial and will depend on many factors, including but not limited to the following:
•our ability to continue to generate revenues from sales of commercial products and potential future products;
•our ability to resume commercial shipments of ELEVIDYS for non-ambulatory patients in the U.S.;
•our ability to realize the benefits of the Restructuring;
•the risk that the Restructuring and Pipeline Reprioritization may not generate their intended benefits to the extent or as quickly as anticipated;
•the impact of potential regulatory actions from the FDA including changes to our drug labels or revocation of accelerated approvals and directives to remove products from the market relating to the topline results of our ESSENCE trial that failed to meet statistical significance on its primary endpoint;
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•the timing and costs associated with repurchases of our common stock under our 2024 Repurchase Program;
•the timing of payments related to our future inventory commitments and manufacturing obligations;
•the timing and costs associated with our existing lease obligations and new obligations expected to be entered into in future years;
•the timing and costs associated with our pre-clinical and clinical trials;
•the attainment of milestones and our obligations to make milestone payments to Arrowhead, Myonexus Therapeutics, Inc.'s selling shareholders, BioMarin, Nationwide, UWA and other institutions;
•the timing and repayment of future borrowings on our Revolving Credit Facility;
•the obligations to holders of our 2027 Notes and 2030 Notes; and
•the costs of filing, prosecuting, defending and enforcing patent claims and our other intellectual property rights.
We cannot provide assurances that financing will be available when and as needed or that, if available, the financings will be on favorable or acceptable terms. If we are unable to obtain additional financing when and if we require, this would have a material adverse effect on our business and results of operations. To the extent we issue additional equity securities, our existing stockholders could experience substantial dilution. We believe that existing cash and cash equivalents, along with future cash generated from operations and availability under our Revolving Credit Facility will be sufficient to meet the capital requirements of our operations for the next 12 months and foreseeable future. Additional information regarding our Revolving Credit Facility is provided in Note 13, Revolving Credit Facility to the unaudited condensed consolidated financial statements contained in Item 1.
We have entered into long-term contractual arrangements from time to time for our facilities, the provision of goods and services, and issuance of debt securities, among others. Additional information regarding our obligations under manufacturing arrangements is provided in Note 16, Commitments and Contingencies to the unaudited condensed consolidated financial statements contained in Item 1. There have been no material changes to our obligations under debt or leasing arrangements as reported in our Annual Report on Form 10-K for the year ended December 31, 2025, except for the partial draw downs and subsequent repayments of our Revolving Credit Facility, as discussed in Note 13, Revolving Credit Facility.
For products and product candidates that are currently approved or are in various research and development stages, we may be obligated to make up to $12.1 billion of future development, regulatory, up-front royalty and sales milestone payments associated with our license and collaboration agreements. Payments under these agreements generally become due and payable upon achievement of certain development, regulatory or commercial milestones. Because the achievement of these milestones is not probable, and payment is not required as of June 30, 2026, such contingencies have not been recorded in our unaudited condensed consolidated financial statements. Amounts related to contingent milestone payments are not yet considered contractual obligations as they are contingent on the successful achievement of certain development, regulatory approval and commercial milestones.
Cash Flows
The following table summarizes our cash flow activity for each of the periods indicated:
For the Six Months Ended
June 30,
2026 2025 Change Change
(in thousands) $ %
Cash (used in) provided by
Operating activities $ (5,613 ) $ (322,100 ) $ 316,487 (98 )%
Investing activities (227,762 ) (258,966 ) 31,204 (12 )%
Financing activities 241 (11,346 ) 11,587 (102 )%
Decrease in cash and cash equivalents $ (233,134 ) $ (592,412 ) $ 359,278 (61 )%
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Operating Activities
Cash used in operating activities, which consists of our net income (loss) adjusted for non-cash items and changes in net operating assets and liabilities, totaled $5.6 million and $322.1 million for the six months ended June 30, 2026 and 2025, respectively. Cash used in operating activities for the six months ended June 30, 2026 was primarily driven by the net income of $326.1 million, adjusted for the following:
•$53.9 million in stock-based compensation expense;
•$21.0 million in depreciation and amortization expense;
•$19.0 million in non-cash interest expense;
•$11.6 million in write-downs for obsolete inventory; and
•$7.2 million in other non-cash items.
These non-cash charges were partially offset by $1.4 million in accretion of investment discount, net.
The net cash outflow from changes in our operating assets and liabilities was primarily driven by the following:
•$307.0 million decrease in deferred revenue primarily related to the recognition of $325.0 million associated with Roche's declined option to acquire certain program rights previously recorded as deferred revenue, partially offset by new orders placed by Roche;
•$229.9 million decrease in accounts payable primarily related to timing of payments, including the payment of the second DM1 Milestone to Arrowhead during the six months ended June 30, 2026;
•$37.3 million decrease in accrued expenses primarily due to timing of payments to our CMOs related to manufacturing commitments and payment of accrued compensation, partially offset by the litigation contingency charge;
•$29.8 million increase in inventory primarily due to a continued manufacturing of ELEVIDYS to meet contractual commitments with contract manufacturing organizations, partially offset by sales of our products and batches of our products not meeting our quality specifications;
•$21.4 million decrease in accounts receivable primarily due to a decrease in demand for our products from year-end;
•$67.8 million decrease in other assets primarily driven by the timing of payment on receivables from Roche; and
•$69.3 million decrease in manufacturing-related deposits and prepaids primarily due to timing of prepaid raw materials with Catalent.
Cash used in operating activities for the six months ended June 30, 2025 was primarily driven by the net loss of $250.6 million, adjusted for the following:
•$78.5 million in stock-based compensation expense;
•$54.0 million loss on strategic investments;
•$20.8 million in depreciation and amortization expense; and
•$11.3 million in other non-cash items.
These non-cash charges were partially offset by the $3.8 million in accretion of investment discount, net.
The net cash outflow from changes in our operating assets and liabilities was primarily driven by the following:
•$236.5 million increase in inventory primarily due to a continuing build-up of ELEVIDYS inventory as a result of label expansion of the therapy in June 2024;
•$77.7 million decrease in accounts payable primarily due to the timing of invoices and payments;
•$59.8 million decrease in deferred revenue primarily related to our collaboration with Roche, partially offset by our contract manufacturing activities with Roche;
•$37.3 million increase in other assets primarily due to an increase in receivables from Roche associated with contract manufacturing revenue;
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•$15.4 million increase in lease liabilities and other liabilities primarily to reimbursements received for build-out of our Bedford facility;
•$20.2 million increase in accrued expenses primarily due to increased revenue-related accruals related to increased product revenue, partially offset by payments on accrued employee compensation costs and timing of payments to our CMOs related to manufacturing commitments;
•$68.9 million decrease in manufacturing-related deposits and prepaids primarily due to timing of payments to Catalent; and
•$74.7 million decrease in accounts receivable, net primarily due to a decrease in demand for ELEVIDYS from year-end.
Investing Activities
Cash used in investing activities was $227.8 million and $259.0 million for the six months ended June 30, 2026 and 2025, respectively. Cash used in investing activities for the six months ended June 30, 2026 primarily consisted of $351.0 million of purchases of investments and $2.6 million of purchases of property and equipment, partially offset by $126.9 million from the maturity of investments.
Cash used in investing activities for the six months ended June 30, 2025 primarily consisted of $245.8 million in the acquisition of strategic investments primarily related to Arrowhead, $75.4 million of purchases of property and equipment and $45.1 million of purchases of investments, partially offset by $109.4 million from the maturity and sale of investments.
Financing Activities
Cash provided by financing activities was $0.2 million for the six months ended June 30, 2026, compared to $11.3 million of cash used in financing activities for the six months ended June 30, 2025. Cash provided by financing activities for the six months ended June 30, 2026 consisted of $0.2 million in proceeds from exercise of options. During the six months ended June 30, 2026, we also drew down, and subsequently repaid, $425.0 million on our Revolving Credit Facility.
Cash used in financing activities for the six months ended June 30, 2025 consisted of $25.0 million of purchases of our common stock under our 2024 Repurchase Program and $3.5 million in arrangement, up-front and commitment fees related to the Revolving Credit Facility, partially offset by $17.2 million in proceeds from exercise of options and purchase of stock under our Employee Stock Purchase Program.