Sarepta Therapeutics, Inc.

Previous wording

our belief that our proprietary technology platforms and collaborations can be used to develop potential therapeutic candidates to treat a broad range of diseases;

Current wording

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;

Previous wording

our expectation that our partnerships with manufacturers will support our clinical and commercial manufacturing capacity for our Duchenne muscular dystrophy (“Duchenne”) gene therapy programs and Limb-girdle muscular dystrophy (“LGMD”) programs, while also acting as a manufacturing platform for potential future gene therapy programs, and our belief that our current network of manufacturing partners is able to fulfill the requirements of our commercial plan;

Current wording

our expectation that our partnerships with manufacturers will support our clinical and commercial manufacturing capacity for our products and product candidates, including PMO and gene therapy programs and our SRP-9003 Limb-girdle muscular dystrophy (“LGMD”) program, 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;

Previous wording

our expectation that our partnership with Catalent, Inc. (“Catalent”) will support our clinical and commercial manufacturing demand for our Duchenne gene therapy program and LGMD programs, while also acting as a manufacturing platform for potential future gene therapy programs;

Current wording

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 potential future gene therapy programs;

Previous 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");

Current wording

estimated timelines and milestones for the remainder of 2025 and beyond, including discussions with the FDA regarding ELEVIDYS, VYONDYS, AMONDYS and SRP-9003, and sharing data for certain of the Company's siRNA product candidates, SRP-1001 and SRP-1003, in 2026;

Previous wording

our plan to continue building out our network for commercial distribution in jurisdictions in which our products are approved;

Current wording

our 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;

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;

Current wording

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;

Previous wording

our expectation regarding the impact of environmental laws and regulations on our business; and

Current wording

our expectation regarding the impact of environmental laws and regulations on our business;

Previous 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.

Current wording

We are a commercial-stage biopharmaceutical company focused on helping patients through the discovery and development of unique RNA-targeted therapeutics, small interfering RNA ("siRNA") knockdown therapies, gene therapy and other genetic therapeutic modalities for the treatment of rare diseases. Applying 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 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. 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").

Current wording

ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-("AAV") based gene therapy, was approved by the FDA on June 20, 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene as well as 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 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 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. We remain in ongoing discussions with FDA regarding the labeling supplement process for ELEVIDYS, and have agreed to a boxed warning for acute liver injury ("ALI") and acute liver failure ("ALF") and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information.

Previous wording

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.

Current wording

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. On November 3, 2025, we announced top-line results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of two of our PMO Products: AMONDYS and VYONDYS. The topline results did not show statistical significance on the study's primary endpoint. We intend to discuss with FDA the potential pathway forward.

Previous 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:

Current wording

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"). 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).

Current wording

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 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.

Previous wording

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 wording

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 expect to meet with FDA in 2025 regarding a biologics license application ("BLA") submission for SRP-9003.

Previous wording

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 wording

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 1/2a clinical trial. We expect to share initial data in the first quarter of 2026.

Previous wording

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 wording

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. We expect to share initial data in the first quarter of 2026.

Previous wording

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 which will in part utilize these capabilities to support production of certain of our product candidates and their components. We have also opened facilities over the past several years that significantly enhanced 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. For our current and future manufacturing needs, we have entered into supply agreements with specialized contract manufacturing organizations (each a “CMO”) to produce custom raw materials, the active pharmaceutical ingredients (“APIs”), drug product and finished goods for our products and product candidates for both commercial and clinical use. All of our CMO partners have extensive technical expertise, GMP experience and experience manufacturing our specific technology.

Current wording

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 which will in part utilize these capabilities to support production of certain of our product candidates and their components. We have also opened facilities over the past several years that significantly enhanced 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. For our current and future manufacturing needs, we have entered into supply agreements with specialized contract manufacturing organizations (each a “CMO”) to produce custom raw materials, active pharmaceutical ingredients (“APIs”), drug product and finished goods for our products and product candidates for both commercial and clinical use. All of our CMO partners have extensive technical expertise, GMP experience and experience manufacturing our specific technology.

Previous 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.

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 SRP-9003 LGMD program, 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 SRP-9003 LGMD program and future gene therapy programs.

Previous wording

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.

Current wording

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.

Previous wording

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 wording

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 products.

Previous 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.

Current wording

As of September 30, 2025, we had $865.2 million of cash, cash equivalents, restricted cash and investments, consisting of $613.1 million of cash and cash equivalents, $239.0 million of investments and $13.1 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 and Six Months Ended June 30, 2025 and 2024

Current wording

Results of Operations for the Three and Nine Months Ended September 30, 2025 and 2024

Previous wording

The following tables set forth selected unaudited condensed consolidated statements of income (loss) data for each of the periods indicated:

Current wording

The following tables set forth selected unaudited condensed consolidated statements of (loss) income data for each of the periods indicated:

Previous 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.

Current wording

For the nine months ended September 30, 2025 and 2024, net product revenues for our products increased by $344.9 million. The increase primarily reflects increased net product revenues of ELEVIDYS of $351.7 million in the nine months ended September 30, 2025 as a result of its expanded label approval in June 2024.

Previous 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.

Current wording

For the nine months ended September 30, 2025 and 2024, we recognized $260.6 million and $93.8 million of collaboration and other revenues, respectively. For the nine months ended September 30, 2025, we recorded $112.0 million of collaboration revenue recognized in relation to the expiration of an option for a certain program previously recorded as deferred revenue and $63.5 million 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, as compared to $48.0 million of collaboration revenue recognized in the nine months ended September 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. Contract manufacturing revenue increased by $29.4 million related to increased deliveries of ELEVIDYS to Roche for the nine months ended September 30, 2025. In addition, royalty revenue increased by $9.9 million from increased sales of ELEVIDYS by Roche outside of the U.S. for the nine months ended September 30, 2025.

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 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.

Current wording

For ELEVIDYS sold in the three and nine months ended September 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 $2.0 million and $20.2 million higher for the three and nine months ended September 30, 2025, respectively, as compared to $29.1 million and $68.8 million for the three and nine months ended September 30, 2024, respectively.

Previous wording

** See above for further details regarding product supply sold to Roche via contract manufacturing under the Roche Collaboration Agreement.

Current wording

See revenue section above for further details regarding product supply sold to Roche via contract manufacturing under the Roche Collaboration Agreement.

Previous 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.

Current wording

The cost of sales (excluding amortization of in-licensed rights) for the nine months ended September 30, 2025 increased by $254.1 million, or 136%, compared with the same period in 2024. The change primarily reflects (1) increased demand following expanded label approval of ELEVIDYS in June 2024, (2) depletion of previously expensed ELEVIDYS inventory, (3) an increase in the write-offs of certain batches of our products not meeting our quality specifications, (4) an increase in products sold to Roche under the Roche Collaboration Agreement and (5) the impairment of prepaid manufacturing deposits.

Previous wording

$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 wording

$13.1 million decrease in clinical trial expenses primarily due to our decision to pause certain SRP-9001 studies and discontinue our PPMO programs;

Previous 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.

Current wording

$49.4 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due the termination of the Thermo Agreement in the three months ended September 30, 2024 as well as a decrease in reimbursable research & development spend pursuant to our Restructuring.

Previous 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");

Current wording

$683.8 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"). The remaining increase relates to the $100.0 million milestone payment to Arrowhead, triggered by Arrowhead's achievement of the first of two predetermined enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 study for an individual program during the nine months ended September 30, 2025 with no similar activity for the nine months ended September 30, 2024;

Previous 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;

Current wording

$30.5 million decrease in clinical trial expenses primarily due to our decision to discontinue our PPMO programs and completion of certain SRP-9001 studies, partially offset by increased activity in our LGMD programs;

Previous 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;

Current wording

$17.4 million decrease in stock-based compensation expense primarily due to reversal of previously recognized expense related to unvested awards pursuant to our Restructuring, the fulfillment of remaining service conditions associated with certain PSUs in March 2025 and the ESPP Suspension;

Previous 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;

Current wording

$3.5 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 pre-clinical expenses primarily due to a decrease in capsid and toxicology studies across our gene therapy programs; and

Current wording

$1.5 million decrease in pre-clinical expenses primarily due to a decrease in toxicology studies across our gene therapy programs;

Previous wording

$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 wording

$23.9 million decrease in stock-based compensation primarily due to the achievement of performance conditions related to certain PSUs during the nine months ended September 30, 2024 and reversal of previously recognized expense related to unvested awards and reduced headcount pursuant to our Restructuring and the ESPP Suspension, partially offset by fulfillment of remaining service conditions associated with certain PSUs in March 2025;

Previous wording

$5.6 million decrease in other expenses primarily due to timing of charitable contribution activity.

Current wording

$6.5 million decrease in other expenses primarily due to timing of charitable contribution activity.

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 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.

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 nine months ended September 30, 2025, we recorded amortization of in-licensed rights of approximately $0.7 million and $1.9 million, respectively. For the three and nine months ended September 30, 2024, we recorded amortization of in-licensed rights of approximately $0.6 million and $1.8 million, respectively.

Previous 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.

Current wording

Other income, net primarily consists of the unrealized gain or loss from our investments in our strategic equity investments, interest expense on our 2027 Notes, interest expense on our 2030 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 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.

Current wording

For the three months ended September 30, 2025, other income, net increased by $36.1 million compared with the three months ended September 30, 2024. The change is primarily due to a $39.6 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, and a $9.6 million decrease in the change in fair value of derivatives, related to our contingent consideration liability. These changes were partially offset by a decrease of $10.0 million in interest income due to changes in the investment mix of our investment portfolio.

Previous wording

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 wording

For the nine months ended September 30, 2025, other income, net decreased by $29.7 million compared with the nine months ended September 30, 2024. The change is primarily due to a $29.1 million decrease in interest income due to changes in the investment mix of our investment portfolio and a $15.5 million increase in the loss on our strategic investment in publicly-traded companies, including Arrowhead. These changes were partially offset by a $19.7 million decrease in the fair value of derivatives, related to our contingent consideration liability.