Sarepta Therapeutics, Inc.
For the period ended Jun 30, 2025. Filed Aug 6, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Jun 30, 2025 · Filed Aug 6, 2025
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-03-31; filed 2025-05-06.
0000950170-25-064412
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-06-30; filed 2025-08-06.
0000950170-25-103971
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 149 previous paragraphs and 183 current paragraphs.
72 unchanged; 66 reworded; 39 added; 9 removed; 2 previous and 6 current paragraphs rearranged.
Tables are not compared: 9 previous and 16 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (39)
Current report
the possible impacts of the ELEVIDYS Suspension (as defined herein);
Current report
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;
Current report
the estimated impacts of the strategic restructuring plan announced in July 2025;
Current report
On July 21, 2025, we announced that the FDA placed a clinical hold on our investigational use gene therapy clinical 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 1/2 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. We intend to seek to discuss with FDA in 2025 the timing of a BLA submission for SRP-9003.
Current report
SRP-1003 (DM1). DM1 is an autosomal dominant, debilitating, chronic progressive multisystem disorder characterized by an expansion of a highly unstable CUGexp in the 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 dystrophia myotonica protein kinase (DMPK) gene. There is currently no approved disease-modifying therapy for DM1. We are currently investigating SRP-1003 in a Phase 1/2a clinical trial.
Current report
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 1/2a clinical trial.
Current report
With respect to our siRNA programs, we are relying on our partner Arrowhead to supply drug substance manufacturing and testing services for ongoing and future clinical trials. Arrowhead’s cGMP manufacturing facility has a variety of manufacturing lines at multiple scales available and is capable of potential future commercial-scale manufacture and capacity expansion as needed. In addition, we plan to leverage existing CMOs, which have technical expertise in manufacturing RNA therapies, for the manufacture of drug product.
Current report
Leadership Transition
Current report
On July 14, 2025, the Board appointed Ian Estepan as President and Chief Operating Officer, Louise Rodino-Klapac, Ph.D. as President, Research and Development and Technical Operations and Ryan Wong as Executive Vice President, Chief Financial Officer, each effective as of July 16, 2025. Douglas Ingram, who previously served as President and Chief Executive Officer, continues to serve as Chief Executive Officer. Mr. Wong replaced Mr. Estepan as principal financial officer and principal accounting officer.
Current report
Research and development expenses for the three months ended June 30, 2025 increased by $24.7 million compared with the three months ended June 30, 2024. The increase was primarily driven by the following:
Current report
$30.0 million increase in manufacturing expenses primarily due to ELEVIDYS clinical batch releases of $27.0 million as certain commercial batches being utilized for clinical activity and $13.0 million of costs associated with our settlement agreement with Brammer Bio MA, LLC (“Brammer”) to resolve outstanding claims related to the termination of the Thermo Agreement (the “Brammer Settlement”). Please refer to Note 17, Commitments and Contingenciesfor further discussion of the Brammer Settlement;
Current report
$6.2 million increase in compensation and other personnel expenses primarily due to changes in headcount;
Current report
Research and development expenses for the six months ended June 30, 2025 increased by $597.8 million compared with the six months ended June 30, 2024. The increase was primarily driven by the following:
Current report
$27.8 million increase in manufacturing expenses primarily due to ELEVIDYS clinical batch releases of $23.0 million due to certain commercial batches being utilized for clinical activity and $13.0 million of costs associated with the Brammer Settlement, partially offset by a decrease in PMO manufacturing activity of $10.7 million;
Current report
$11.9 million increase in compensation and other personnel expenses primarily due to changes in headcount;
Current report
$3.5 million decrease in stock-based compensation expense primarily due to the achievement of performance conditions related to certain PSUs during the three months ended June 30, 2024 and fulfillment of remaining service conditions associated with certain PSUs in March 2025, partially offset by changes in headcount;
Current report
$6.5 million increase in professional service expenses primarily due to marketing and consulting related to our PMO programs, LGMD site readiness and our continuing efforts to commercialize ELEVIDYS;
Current report
$6.1 million increase in compensation and other personnel expenses primarily due to annual merit-based salary increases and an increase in certain employer-related payroll taxes;
Current report
$8.9 million decrease in stock-based compensation primarily due to the achievement of performance conditions related to certain PSUs during the three months ended June 30, 2024 and fulfillment of remaining service conditions associated with certain PSUs in March 2025; and
Current report
$5.6 million decrease in other expenses primarily due to timing of charitable contribution activity.
Current report
Selling, general and administrative expenses for the six months ended June 30, 2025 increased by $5.7 million compared with the six months ended June 30, 2024. This increase was primarily driven by the following:
Current report
$9.9 million increase in compensation and other personnel expenses primarily due to annual merit-based salary increases and an increase in certain employer-related payroll taxes;
Current report
$9.2 million decrease in stock-based compensation primarily due to the achievement of performance conditions related to certain PSUs during the six months ended June 30, 2024 and fulfillment of remaining service conditions associated with certain PSUs in March 2025;
Current report
$2.0 million increase in facility- and technology-related expenses primarily due to our expansion efforts; and
Current report
$6.0 million decrease in other expenses primarily due to timing of charitable contribution activity.
Current report
Other income (expense), net
Current report
For the six months ended June 30, 2025, other income (expense), net changed by $65.9 million compared with the six months ended June 30, 2024. The unfavorable change is primarily due to a $55.1 million increase in the loss on our strategic investments and a $19.4 million decrease in accretion of investment discount, net due to changes in the investment mix of our investment portfolio. These decreases were partially offset by a loss of $10.1 million associated with the change in fair value of contingent consideration for the six months ended June 30, 2024 with no similar activity during the six months ended June 30, 2025.
Current report
Income tax (benefit) expense
Current report
On July 4, 2025, President Trump signed into law the One Big Beautiful Bill Act ("OBBBA"). The OBBBA includes provisions that could have a significant impact on us, such as the permanent extension of certain expiring provisions of the Tax Cuts and Jobs Act, modifications to the international tax framework and the restoration of favorable tax treatment for certain business provisions. We are currently evaluating the impact of the OBBBA and expect the results of such evaluations to be reflected in our future filings.
Current report
In response to two reported cases of ALF resulting in death of non-ambulatory patients, we suspended all commercial shipments of ELEVIDYS to non-ambulatory patients in June 2025. In July 2025, we also disclosed a recent death from ALF of a non-ambulatory LGMD patient participating in a Phase 1/2 trial for SRP-9004, who was not treated with ELEVIDYS. Thereafter, in response to a request from the FDA that we voluntarily stop all shipments of ELEVIDYS in the U.S., including to ambulatory patients, we temporarily suspended all shipments of ELEVIDYS in the U.S., effective July 22, 2025, to allow us the necessary time to respond to FDA's requests for information and complete a labeling supplement process. Although the FDA informed the Company on July 28, 2025 that it recommended the removal of the voluntary hold for ambulatory patients, the ELEVIDYS Suspension could materially impact our near-term revenue generation. The suspension of shipments for non-ambulatory patients remains in effect as of the date of issuance of this Quarterly Report. It is currently unclear whether or when we might resume shipments of ELEVIDYS for non-ambulatory patients, or whether the FDA will pursue further actions relating to the use of ELEVIDYS in ambulatory or non-ambulatory patients, such as additional studies, additional product modifications or controls, or withdrawal in the future. We considered the ELEVIDYS Suspension and the partial resumption of shipments when concluding that our cash, cash equivalents and investments as of the date of issuance of this report, along with future cash inflows from operations, are sufficient to fund our current operational plan for at least the next twelve months.
Current report
Further, in July 2025, we announced a strategic restructuring plan designed to enhance financial flexibility and meet our financial obligations associated with our 2027 Notes (the “Restructuring”). This plan includes a revised cost structure, program portfolio and a reduction in force. The workforce reduction represented approximately 36% of our workforce. As a result of this reduction in force, we estimate that we will record a one-time charge in the third quarter of 2025 related to employee termination benefits, including severance, between approximately $32.0 million and $37.0 million, all of which is anticipated to result in cash expenditures. While we anticipate that the Restructuring will result in a reduction in our future expenditures, actual savings realized may vary and there can be no assurance that the Restructuring will achieve our anticipated cost savings or operational efficiencies.
Current report
our ability to resume shipments of shipments of ELEVIDYS for non-ambulatory patients in the U.S.;
Current report
our ability to implement the Restructuring;
Current report
the risk that our Restructuring and pipeline reprioritization efforts may not generate their intended benefits to the extent or as quickly as anticipated;
Current report
$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;
Current report
$37.3 million increase in other assets primarily due to an increase in receivables from Roche associated with contract manufacturing revenue;
Current report
$10.1 million change in the fair value of contingent consideration; and
Current report
These amounts were partially offset by the following:
Current report
$6.6 million decrease in other assets primarily due to a decrease in the collaboration receivable related to Roche; and
Removed paragraphs (9)
Previous report
The cost of sales (excluding amortization of in-licensed rights) for the three months ended March 31, 2025 increased by $87.0 million, or 172%, compared with the same period in 2024. The change primarily reflects an increase in cost of sales related to
Previous report
Research and development expenses for the three months ended March 31, 2025 increased by $573.1 million compared with the three months ended March 31, 2024. The increase was primarily driven by the following:
Previous report
$2.2 million decrease in manufacturing expenses primarily due to timing of production activity across our programs;
Previous report
$5.8 million increase in compensation and other personnel expenses primarily due to changes in headcount;
Previous report
$3.7 million increase in compensation and other personnel expenses primarily due to changes in headcount;
Previous report
Other (expense) income, net
Previous report
Income tax expense
Previous report
$177.4 million increase in inventory primarily due to a ramp-up of manufacturing corresponding to an increase in demand for ELEVIDYS;
Previous report
$10.1 million non-cash change in the fair value of contingent consideration;
Reworded paragraphs (66)
Previous wording
our expectation that Aldevron LLC (“Aldevron”) will provide Good Manufacturing Processes (“GMP”)-grade plasmid for our Duchenne gene therapy program and LGMD programs, as well as plasmid source material for future gene therapy programs;
Current wording
our expectation that Aldevron LLC (“Aldevron”) will provide Good Manufacturing Processes (“GMP”)-grade plasmid for our Duchenne gene therapy program and our SRP-9003 LGMD program, as well as plasmid source material for any future gene therapy programs;
Previous wording
the possible impact of regulations and regulatory decisions by the Food and Drug Administration (the “FDA”) and other regulatory agencies on our business as well as the development of our product candidates and our financial and contractual obligations;
Current wording
the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business as well as the development of our product candidates and our financial and contractual obligations;
Previous wording
estimated timelines and milestones for the remainder of 2025 and beyond, including submitting a SRP-9003 BLA in the second half of 2025;
Current wording
estimated timelines and milestones for the remainder of 2025 and beyond, including seeking to discuss with the FDA in 2025 a SRP-9003 biologics license application (“BLA");
Previous wording
the possible impact of any legislation and competing products on the commercial success of our products and our product candidates and our ability to compete against such products;
Current wording
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;
Previous wording
our expectations 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;
Current wording
our expectations 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;
Previous wording
We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying our proprietary, highly 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 Duchenne, LGMDs, and other neuromuscular and central nervous system related disorders.
Current wording
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, highly 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 Duchenne and LGMD, as well as those through our partnered programs with Arrowhead Therapeutics Inc. (“Arrowhead”), including Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia type 2 ("SCA2"), Idiopathic Pulmonary Fibrosis ("IPF"), Huntington's disease and other neuromuscular and skeletal diseases.
Previous wording
ELEVIDYS (delandistrogene moxeparvovec-rokl), an AAV-based gene therapy, was approved by the FDA on June 20, 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene as well as non-ambulatory patients under the accelerated approval pathway. ELEVIDYS was previously granted accelerated approval by the FDA on June 22, 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.
Current wording
ELEVIDYS (delandistrogene moxeparvovec-rokl), an AAV-based gene therapy, was approved by the FDA on June 20, 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene as well as non-ambulatory patients under the accelerated approval pathway. ELEVIDYS was previously granted accelerated approval by the FDA on June 22, 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, in June 2025, we suspended all shipments of ELEVIDYS to non-ambulatory patients in the U.S. In response to a request from 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 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. As of the date of this Quarterly Report, we have resumed shipments of ELEVIDYS for ambulatory patients in the U.S. We remain in ongoing discussions with FDA regarding the labeling supplement process for ELEVIDYS, including a boxed warning for acute liver injury ("ALI") and acute liver failure ("ALF").
Previous wording
A summary description of our key product candidates, including those in collaboration with our strategic partners, is as follows:
Current wording
In July 2025, we announced a strategic restructuring plan designed to enhance financial flexibility and meet our 2027 financial obligations, including a revised program portfolio that prioritizes our siRNA platform assets. A summary description of our key product candidates, including those in collaboration with our strategic partners, is as follows:
Previous wording
SRP-9003 (LGMD, gene therapy program). We are developing gene therapy programs for various forms of LGMD. The most advanced of our LGMD product candidates, 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 had completed enrollment and dosing in EMERGENE (Study SRP-9003-301), a Phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec). We expect to file a BLA in the second half of 2025.
Current wording
SRP-9003 (LGMD, gene therapy program). We are developing gene therapy programs for various forms of LGMD. The most advanced of our LGMD product candidates, 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 had completed enrollment and dosing in EMERGENE (Study SRP-9003-301), a Phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec).
Previous wording
Our gene therapy manufacturing capabilities have been greatly enhanced through partnerships with Aldevron and Catalent Inc. (“Catalent”). We have adopted a hybrid development and manufacturing strategy in which we have built internal expertise relative to all aspects of AAV-based manufacturing, including gene therapy and gene editing, while closely partnering with experienced manufacturing partners to expedite development and commercialization of our gene therapy programs. We have secured manufacturing capacity at Catalent to support our clinical and commercial manufacturing demand for ELEVIDYS and our LGMD programs, while also acting as a manufacturing platform for potential future gene therapy programs. The collaboration integrates process development, clinical and commercial production and testing. Aldevron provides GMP-grade plasmid for ELEVIDYS and is expected to provide plasmid source material for our LGMD programs and future gene therapy programs.
Current wording
Our gene therapy manufacturing capabilities have been greatly enhanced through partnerships with Aldevron and Catalent. We have adopted a hybrid development and manufacturing strategy in which we have built internal expertise relative to all aspects of AAV-based manufacturing, including gene therapy and gene editing, while closely partnering with experienced manufacturing partners to expedite development and commercialization of our gene therapy programs. We have secured manufacturing capacity at Catalent to support our clinical and commercial manufacturing demand for ELEVIDYS and our LGMD programs, while also acting as a manufacturing platform for potential future gene therapy programs. The collaboration integrates process development, clinical and commercial production and testing. Aldevron provides GMP-grade plasmid for ELEVIDYS and is expected to provide plasmid source material for our LGMD programs and future gene therapy programs.
Previous wording
As of March 31, 2025, we had $647.5 million of cash, cash equivalents, restricted cash and investments, consisting of $240.9 million of cash and cash equivalents, $391.0 million of investments and $15.6 million of non-current restricted cash. 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.
Current wording
As of June 30, 2025, we had $850.3 million of cash, cash equivalents, restricted cash and investments, consisting of $510.6 million of cash and cash equivalents, $324.1 million of investments and $15.6 million of non-current restricted cash. We believe that our balance of cash, cash equivalents and investments, along with cash inflows from operations is sufficient to fund our current operational plan for at least the next twelve months.
Previous wording
Results of Operations for the Three Months Ended March 31, 2025 and 2024
Current wording
Results of Operations for the Three and Six Months Ended June 30, 2025 and 2024
Previous wording
The following tables set forth selected unaudited condensed consolidated statements of (loss) income data for each of the periods indicated:
Current wording
The following tables set forth selected unaudited condensed consolidated statements of income (loss) data for each of the periods indicated:
Previous wording
The following table summarizes the components of our net product revenues, by product, for each of the periods indicated:
Current wording
The following tables summarize the components of our net product revenues, by product, for each of the periods indicated:
Previous wording
Net product revenues for our products for the three months ended March 31, 2025 increased by $252.0 million compared with the three months ended March 31, 2024. The increase primarily reflects an increase in net product revenues of ELEVIDYS of $241.0 million in the three months ended March 31, 2025 as a result of its expanded label approval in June 2024.
Current wording
Net product revenues for our products for the three and six months ended June 30, 2025 increased by $152.6 million and $404.6 million compared with the three and six months ended June 30, 2024. The increase primarily reflects an increase in net product revenues of ELEVIDYS of $160.1 million and $401.2 million in the three and six months ended June 30, 2025 as a result of its expanded label approval in June 2024.
Previous wording
The following table summarizes the components of our collaboration and other revenues for the periods indicated:
Current wording
The following tables summarize the components of our collaboration and other revenues for the periods indicated:
Previous wording
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. For the three months ended March 31, 2025 and 2024, we recognized $17.4 million and $5.8 million of contract manufacturing revenue, respectively, which is related to these Roche shipments. In addition, we recognized $4.0 million and approximately $0.2 million of royalty revenue from sales of ELEVIDYS by Roche for the three months ended March 31, 2025 and 2024, respectively.
Current wording
For the three months ended June 30, 2025 and 2024, we recognized $98.0 million and $2.4 million of collaboration and other revenues, respectively. The increase is primarily due to $63.5 million of collaboration revenue recognized in the three months ended June 30, 2025 related to milestone payments received under the Roche Collaboration Agreement from the regulatory approval of ELEVIDYS in Japan for individuals ages 3- to less than 8-years-old, who do not have any deletions in exon 8 and/or exon 9 in the Duchenne gene and who are negative for anti-AAVrh74 antibodies (the “Japan Approval Milestone”). Please refer to Note 3, License and Collaboration Agreements for further discussion of the Roche Collaboration Agreement. While the Roche Supply Agreement is in the process of being negotiated, we delivered batches of commercial ELEVIDYS supply to Roche that were agreed upon on a purchase order-by-purchase order basis. For the three months ended June 30, 2025, we recognized $27.0 million of contract manufacturing revenue, which is related to these shipments, with no similar activity for the three months ended June 30, 2024. In addition, we recognized $7.4 million and $2.4 million of royalty revenue from sales of ELEVIDYS by Roche outside of the U.S. for the three months ended June 30, 2025 and 2024, respectively.
Previous wording
Collaboration and other revenues primarily relate to our collaboration arrangement (the “Roche Collaboration Agreement”) with F. Hoffman-La Roche Ltd. (“Roche”). For the three months ended March 31, 2025 and 2024, we recognized $133.3 million and $54.0 million of collaboration and other revenues, respectively. The increase is primarily due to the $112.0 million of collaboration revenue recognized in the three months ended March 31, 2025 related to the expiration of an option for a certain program, as compared to $48.0 million collaboration revenue in 2024 related to Roche’s declined option to acquire the ex-US rights to a certain external, early stage Duchenne development program. Please refer to Note 3, License and Collaboration Agreementsfor further discussion of the Roche Collaboration Agreement.
Current wording
For the six months ended June 30, 2025 and 2024, we recognized $231.3 million and $56.4 million of collaboration and other revenues, respectively. The increase is primarily due to $112.0 million of collaboration revenue recognized in the six months ended June 30, 2025 related to the expiration of an option for a certain program previously recorded as deferred revenue and the Japan Approval Milestone payment of $63.5 million under the Roche Collaboration Agreement, as compared to $48.0 million of collaboration revenue recognized in the six months ended June 30, 2024 related to Roche’s declined option to acquire the rights to a certain external, early stage Duchenne development program previously recorded as deferred revenue. For the six months ended June 30, 2025 and 2024, we recognized $44.4 million and $5.8 million of contract manufacturing revenue related to shipments of ELEVIDYS to Roche, respectively. In addition, we recognized $11.4 million and $2.6 million of royalty revenue from sales of ELEVIDYS by Roche outside of the U.S. for the six months ended June 30, 2025 and 2024, respectively.
Previous wording
Our cost of sales (excluding amortization of in-licensed rights) consists of inventory costs that relate to sales of our products and royalty payments primarily to BioMarin Pharmaceuticals, Inc. (“BioMarin”) and the University of Western Australia (“UWA”) for our PMO Products and to Nationwide Children’s Hospital (“Nationwide”) for ELEVIDYS. 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 prior to the three months ended March 31, 2025 and 2024. For ELEVIDYS sold in the three months ended March 31, 2025, a portion of related manufacturing costs incurred had previously been expensed as research and development expenses. For ELEVIDYS sold in the three months ended March 31, 2024, the majority of related manufacturing costs incurred had previously been expensed as research and development expenses, as such costs were incurred prior to FDA approval of the product. If 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 $13.7 million and $23.8 million higher for the three months ended March 31, 2025 and 2024, respectively.
Current wording
Our cost of sales (excluding amortization of in-licensed rights) consists of inventory costs that relate to sales of our products and royalty payments primarily to BioMarin Pharmaceuticals, Inc. (“BioMarin”) and the University of Western Australia (“UWA”) for our PMO Products and to Nationwide Children’s Hospital (“Nationwide”) for ELEVIDYS. 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 prior to the three and six months ended June 30, 2025 and 2024. For ELEVIDYS sold in the three and six months ended June 30, 2025, a portion of related manufacturing costs incurred had previously been expensed as research and development expenses. For ELEVIDYS sold in the three and six months ended June 30, 2024, the majority of related manufacturing costs incurred had previously been expensed as research and development expenses, as such costs were incurred prior to FDA approval of the product. If 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 $4.5 million and $18.2 million higher for the three and six months ended June 30, 2025, respectively, as compared to $15.9 million and $39.7 million for the three and six months ended June 30, 2024, respectively.
Previous wording
The following table summarizes the components of our cost of sales (excluding amortization of in-licensed rights) for each of the periods indicated:
Current wording
The following tables summarize the components of our cost of sales (excluding amortization of in-licensed rights) for each of the periods indicated:
Previous wording
ELEVIDYS as we consume previously expensed inventory, an increase in demand following ELEVIDYS’s expanded label approval in June 2024 as well as an increase in cost of sales related to products sold to Roche under the Roche Collaboration Agreement, partially offset by a decrease in the write-offs of certain batches of our products not meeting our quality specifications for the three months ended March 31, 2025, as compared to the same period of 2024.
Current wording
The cost of sales (excluding amortization of in-licensed rights) for the three months ended June 30, 2025 increased by $108.0 million, or 242%, compared with the same period in 2024. The cost of sales (excluding amortization of in-licensed rights) for the six months ended June 30, 2025 increased by $195.0 million, or 205%, compared with the same period in 2024. For both comparative periods, the changes primarily reflects (1) depletion of previously expensed ELEVIDYS inventory (2) increased demand following expanded label approval of ELEVIDYS in June 2024 (3) an increase in products sold to Roche under the Roche Collaboration Agreement and (4) an increase in the write-offs of certain batches of our products not meeting our quality specifications.
Previous wording
The following table summarizes our research and development expenses by project for each of the periods indicated:
Current wording
The following tables summarize our research and development expenses by project for each of the periods indicated:
Previous wording
The following table summarizes our research and development expenses by category for each of the periods indicated:
Current wording
The following tables summarize our research and development expenses by category for each of the periods indicated:
Previous wording
$11.0 million decrease in clinical trial expenses primarily due to our decision to discontinue our PPMO programs;
Current wording
$6.4 million decrease in clinical trial expenses primarily due to our decision to discontinue our PPMO programs, partially offset by increased activity in our LGMD programs;
Previous wording
$2.9 million increase in facility- and technology-related expenses primarily due to our continuing expansion efforts;
Current wording
$3.0 million increase in facility- and technology-related expenses primarily due to our continuing expansion efforts;
Previous wording
$1.0 million increase in stock-based compensation expense primarily due to changes in headcount;
Current wording
$4.5 million decrease in stock-based compensation expense primarily due to the achievement of performance conditions related to certain PSUs during the three months ended June 30, 2024 and fulfillment of remaining service conditions associated with certain PSUs in March 2025;
Previous wording
$3.0 million increase in professional services primarily due to an increase in reliance on third-party research and development contractors for clinical programs;
Current wording
$1.3 million increase in professional services primarily due to an increase in reliance on third-party research and development contractors for clinical programs;
Previous wording
$1.7 million decrease in research and other expenses primarily due to a decrease in collaboration cost-sharing expenses related to our license and collaboration agreement with Genethon, partially offset by $2.3 million of reimbursable development costs incurred by Arrowhead during the three months ended March 31, 2025; and
Current wording
$2.9 million increase in research and other expenses primary due to reimbursements to Arrowhead, with no similar activity for the three months ended June 30, 2024, partially offset by a decrease in collaboration cost-sharing expenses related to our license and collaboration agreement with Genethon; and
Previous wording
$6.8 million increase in the offset to expense associated with a collaboration reimbursement from Roche due to continuing development of our SRP-9001 gene therapy program.
Current wording
$7.8 million increase in the offset to expense associated with a collaboration reimbursement from Roche primarily due to an increase in ELEVIDYS clinical supply batch releases.
Previous wording
$583.8 million increase in up-front and milestone expenses 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 Agreement”) with Arrowhead Pharmaceuticals, Inc. (“Arrowhead”);
Current wording
$583.8 million increase in up-front and milestone expenses primarily due to the $583.6 million in acquired in-process research and development expense associated with our exclusive global licensing and collaboration agreement and stock purchase agreement with Arrowhead (the "Arrowhead Agreement");
Previous wording
the timing and costs associated with our expansion efforts;
Current wording
$5.9 million increase in facility- and technology-related expenses primarily due to our expansion efforts;
Previous wording
$1.7 million decrease in pre-clinical expenses primarily due to decreased activity across our gene therapy programs;
Current wording
$1.7 million decrease in pre-clinical expenses primarily due to a decrease in capsid and toxicology studies across our gene therapy programs; and
Previous wording
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.
Current wording
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 professional fees for legal, consulting and accounting services and an allocation of our facility- and technology-related costs.
Previous wording
The following table summarizes our selling, general and administrative expenses by category for each of the periods indicated:
Current wording
The following tables summarize our selling, general and administrative expenses by category for each of the periods indicated:
Previous wording
Selling, general and administrative expenses for the three months ended March 31, 2025 increased by $6.6 million, or 5%, compared with the three months ended March 31, 2024. This increase was primarily driven by the following:
Current wording
Selling, general and administrative expenses for the three months ended June 30, 2025 decreased by $0.9 million compared with the three months ended June 30, 2024. This decrease was primarily driven by the following:
Previous wording
$2.6 million increase in professional service expenses primarily related to our continuing efforts to commercialize ELEVIDYS, partially offset by a decrease in expenses related to ongoing litigation matters; and
Current wording
$9.0 million increase in professional service expenses primarily related to our continuing efforts to commercialize ELEVIDYS, partially offset by a decrease in expenses related to ongoing litigation matters;
Previous wording
Amortization of in-licensed rights relates to the agreements we entered into with UWA, Nationwide, 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 date the related fee was incurred, either the regulatory approval or the first commercial sale of the applicable product. For both the three months ended March 31, 2025 and 2024, we recorded amortization of in-licensed rights of approximately $0.6 million.
Current wording
Amortization of in-licensed rights relates to the agreements we entered into with UWA, Nationwide, 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 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, 2025, we recorded amortization of in-licensed rights of approximately $0.7 million and $1.3 million, respectively. For the three and six months ended June 30, 2024, we recorded amortization of in-licensed rights of approximately $0.6 million and $1.2 million, respectively.
Previous wording
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 on September 15, 2027 (the “2027 Notes”), interest income on our cash, cash equivalents and investments, accretion of investment discount and the change in the fair value of contingent consideration related to regulatory-related contingent payments meeting the definition of a derivative liability. Our cash equivalents and investments consist of money market funds, corporate bonds, commercial paper, government and government agency debt securities and certificates of deposit.
Current wording
Other income (expense), 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 on September 15, 2027 (the “2027 Notes”), interest income on our cash, cash equivalents and investments, accretion of investment discount and the change in the fair value of contingent consideration related to regulatory-related contingent payments meeting the definition of a derivative liability. Our cash equivalents and investments consist of money market funds, corporate bonds, commercial paper, government and government agency debt securities and certificates of deposit.
Previous wording
For the three months ended March 31, 2025, other (expense) income, net decreased by $89.7 million compared with the three months ended March 31, 2024. The change is primarily due to a $91.7 million increase in the loss on our strategic investments, which includes the recurring fair value adjustments of investments in publicly-traded companies, including Arrowhead.
Current wording
For the three months ended June 30, 2025, other income, net increased by $23.8 million compared with the three months ended June 30, 2024. The change is primarily due to a $36.6 million increase in the fair value of our strategic investments, which includes the recurring fair value adjustments of investments in publicly-traded companies, including Arrowhead, offset by a decrease of $8.9 million in accretion of investment discount, net due to changes in the investment mix of our investment portfolio.
Previous wording
Income tax expense for the three months ended March 31, 2025 and 2024 was $64.0 million and $5.3 million, respectively. Income tax expense for both periods presented primarily relates to state, federal and foreign income taxes for which available tax losses or credits were not available to offset. As of March 31, 2025, 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 and beyond.
Current wording
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 (benefit) expense for all periods presented primarily relates to state, federal and foreign income taxes for which available tax losses or credits were not available to offset. As of June 30, 2025, 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 and beyond.
Showing the first 40 of 66 pairs. Open the source sections for the rest.
Rearranged current paragraphs (6)
Current report — wording carried from elsewhere
our plans and ability to file and progress to issue additional patent applications to enhance and protect our new and existing technologies and programs;
Current report — wording carried from elsewhere
Collaboration and other revenues primarily relate to our collaboration arrangement (the “Roche Collaboration Agreement”) with F. Hoffman-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 regulatory approvals for ELEVIDYS. We are eligible to receive royalties on these sales.
Current report — wording carried from elsewhere
$17.4 million decrease in clinical trial expenses primarily due to our decision to discontinue our PPMO programs, partially offset by increased activity in our LGMD programs;
Current report — wording carried from elsewhere
$4.3 million increase in professional services primarily due to an increase in reliance on third-party research and development contractors for clinical programs;
Current report — wording carried from elsewhere
$14.6 million increase in the offset to expense associated with a collaboration reimbursement from Roche primarily due to an increase in ELEVIDYS clinical supply batch releases.
Current report — wording carried from elsewhere
$91.2 million in stock-based compensation expense;
Rearranged previous paragraphs (2)
Previous report — wording carried elsewhere
$1.1 million increase in facility- and technology-related expenses primarily due to our continuing expansion efforts.
Previous report — wording carried elsewhere
$40.7 million in stock-based compensation expense;