Sarepta Therapeutics, Inc.

Previous wording

our expectation that our partnerships with manufacturers will support our clinical and commercial manufacturing capacity for our products and product candidates, including our phosphorodiamidate morpholino oligomer (“PMO”), gene therapy, SRP-9003 Limb-girdle muscular dystrophy (“LGMD”), and small interfering RNA (“siRNA”) programs, while also acting as a manufacturing platform for potential future programs, and our belief that our current network of manufacturing partners is able to fulfill the requirements of our commercial plan;

Current wording

our expectation that our partnerships with manufacturers and strategic partners will support our clinical and commercial manufacturing capacity for our products and product candidates, including our phosphorodiamidate morpholino oligomer (“PMO”), gene therapy, SRP-9003 Limb-girdle muscular dystrophy (“LGMD”), and small interfering RNA (“siRNA”) programs, while also acting as a manufacturing platform for potential future programs, and our belief that our current network of manufacturing and strategic partners is able to fulfill the requirements of our commercial plan;

Previous wording

the possible impacts of the results of our ESSENCE confirmatory trial for VYONDYS 53 and AMONDYS 45, including the timing and outcome of additional results, potential regulatory actions from the FDA, including directives to remove these products from the market or alter labels, patient demand for these products and changes to reimbursement and coverage by insurance companies;

Current wording

the possible impacts of the results of our MIS51ON trial of EXONDYS 51 and our ESSENCE confirmatory trial for VYONDYS 53 and AMONDYS 45, including the timing and outcome of additional results, potential regulatory actions from the FDA, including directives to remove these products from the market or alter labels, patient demand for these products and changes to reimbursement and coverage by insurance companies, as well the timing and outcome of FDA review of our supplemental new drug applications ("sNDAs") for VYONDYS 53 and AMONDYS 45, including the anticipated PDUFA date;

Previous wording

estimated timelines and milestones for the remainder of 2026 and beyond, including discussions with the FDA regarding ELEVIDYS, VYONDYS 53, AMONDYS 45 and SRP-9003, and sharing data for certain of our siRNA product candidates, SRP-1001 and SRP-1003;

Current wording

estimated timelines and milestones for the remainder of 2026 and beyond, including discussions with the FDA regarding our products and product candidates, and sharing data for certain of our clinical trials;

Previous wording

our ability to maintain, and our plan to continue building out our distribution network for in jurisdictions in which our products are approved or in which we are seeking to make our products available, commercially or through our EAP (as defined herein);

Current wording

our ability to maintain, and our plan to continue building out our distribution network in jurisdictions in which our products are approved or in which we are seeking to make our products available, commercially or through our early access programs ("EAP");

Previous wording

our ability to further secure long-term supply of our commercial products and our product candidates to satisfy our planned commercial, early access programs (“EAP”) and clinical needs;

Current wording

our ability to further secure long-term supply of our commercial products and our product candidates to satisfy our planned commercial, EAP and clinical needs;

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, differentiated and innovative technologies, and through collaborations with our strategic partners, we have developed multiple approved products for the treatment of Duchenne and are developing potential therapeutic candidates for a broad range of diseases and disorders, including LGMD. We are also developing potential therapeutic candidates through our partnered program with Arrowhead Pharmaceuticals, Inc. ("Arrowhead"), including Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia ("SCA"), and Huntington's disease ("HTT").

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, differentiated and innovative technologies, and through collaborations with our strategic partners, we have developed multiple approved products for the treatment of Duchenne and are developing potential therapeutic candidates for a broad range of diseases and disorders, including LGMD. We are also developing potential therapeutic candidates through our partnered program with Arrowhead Pharmaceuticals, Inc. ("Arrowhead"), including Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia ("SCA"), and Huntington's disease ("HD").

Previous wording

EXONDYS 51 (eteplirsen) Injection ("EXONDYS 51"), granted accelerated approval by the FDA in 2016, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping. EXONDYS 51 uses our phosphorodiamidate morpholino oligomer ("PMO") chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.

Current wording

EXONDYS 51 (eteplirsen) Injection ("EXONDYS 51"), granted accelerated approval by the FDA in 2016, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 51 skipping. EXONDYS 51 uses our PMO chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.

Previous wording

ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-("AAV") based gene therapy, received traditional approval by the FDA in June 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS was also approved for non-ambulatory patients under the accelerated approval pathway in June 2024. ELEVIDYS was previously granted accelerated approval by the FDA in June 2023 for the treatment of ambulatory patients aged four through five years with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene. In response to safety events announced in March and June 2025, we suspended all shipments of ELEVIDYS to non-ambulatory patients in the U.S. in June 2025. In response to a request from the FDA that we voluntarily stop all shipments of ELEVIDYS in the U.S., we temporarily suspended all shipments of ELEVIDYS in the U.S., effective July 22, 2025, to allow us the necessary time to respond to the FDA's requests for information and complete a labeling supplement process (the "ELEVIDYS Suspension"). On July 28, 2025, the FDA informed us that it recommended the removal of the voluntary hold for ambulatory patients. On July 31, 2025, we resumed shipments of ELEVIDYS for ambulatory patients in the U.S. In November 2025, we announced a boxed warning for ALI and ALF and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information for ELEVIDYS.

Current wording

ELEVIDYS (delandistrogene moxeparvovec-rokl), an adeno-associated virus-("AAV") based gene therapy, received traditional approval by the FDA in June 2024 for the treatment of ambulatory patients at least four years old with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS was also approved for non-ambulatory patients under the accelerated approval pathway in June 2024. ELEVIDYS was previously granted accelerated approval by the FDA in June 2023 for the treatment of ambulatory patients aged four through five years with Duchenne with a confirmed mutation in the Duchenne gene. ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the Duchenne gene. In response to safety events announced in March and June 2025, we suspended all shipments of ELEVIDYS to non-ambulatory patients in the U.S. in June 2025. In response to a request from the FDA that we voluntarily stop all shipments of ELEVIDYS in the U.S., we temporarily suspended all shipments of ELEVIDYS in the U.S., effective July 22, 2025 (the "ELEVIDYS Suspension"). On July 28, 2025, the FDA informed us that it recommended the removal of the voluntary hold for ambulatory patients. On July 31, 2025, we resumed shipments of ELEVIDYS for ambulatory patients in the U.S. In November 2025, we announced a boxed warning for ALI and ALF and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information for ELEVIDYS.

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. In November 2025, we announced topline results from our ESSENCE trial, a confirmatory trial intended to verify the clinical benefits of two of our PMO Products: AMONDYS 45 and VYONDYS 53. The topline results did not show statistical significance on the study's primary endpoint. We intend to discuss with FDA the potential pathway forward and submitted supplemental new drug applications ("sNDAs") related to these products in April 2026, which the FDA accepted for filing in June 2026. We are also in the process of conducting various clinical trials for ELEVIDYS, including Cohort 8 of Study 9001-103 (ENDEAVOR), a study to evaluate the use of sirolimus as an enhanced immunosuppression regimen as part of treatment with ELEVIDYS for non-ambulant individuals living with Duchenne. We intend to discuss with the FDA the results of this study and a potential pathway forward to resume commercial dosing in the non-ambulatory population. Resumption of dosing in the non-ambulatory population will depend on the FDA's analysis of whether the sirolimus data positively changes ELEVIDYS' risk/benefit profile and on aligning with the FDA on the process for revising the label, both of which involve risks and uncertainties. Below, when referring to manufactured inventory for commercial sale in the U.S. and ex-U.S. territories, we use the designation of ELEVIDYS, which is recognized as inventory in our unaudited condensed consolidated balance sheets. Separately, when referring to manufactured product for clinical use, such as clinical trials, we use the designation of SRP-9001, which is recognized as research and development expense in our unaudited condensed consolidated statements of comprehensive (loss) income.

Current wording

In the tables referenced in this section, when referring to manufactured inventory for commercial sale in the U.S. and ex-U.S. territories, we use the designation of ELEVIDYS, which is recognized as inventory in our unaudited condensed consolidated balance sheets. Separately, when referring to manufactured product for clinical use, such as clinical trials, we use the designation of SRP-9001, which is recognized as research and development expense in our unaudited condensed consolidated statements of comprehensive (loss) income.

Previous wording

On July 21, 2025, we announced that the FDA placed a clinical hold on our investigational use gene therapy trials for LGMD, including our trials for product candidates SRP-9003 (LGMD2E/R4/bidridistrogene xeboparvovec), SRP-9004 (LGMD2D/patidistrogene bexoparvovec), SRP-6004 (LGMD2B/R2) and SRP-9005 (LGMD2C/R5 g-sarcoglycan), following the death of a patient in our Phase 1 LGMD clinical trial for SRP-9004. We previously announced on July 16, 2025 that we had suspended each of the LGMD programs mentioned above as part of the Restructuring, with the exception of SRP-9003. In December 2025, the FDA confirmed that we remain on clinical hold and informed us that it requires data from the study of sirolimus as an immunosuppressant before accepting a biologic license application ("BLA") for SRP-9003.We anticipate re-engaging with the agency on next steps for the program after we receive data from Cohort 8 of Study 9001-103 (ENDEAVOR).

Current wording

In July 2025, we announced that the FDA placed a clinical hold on our investigational use gene therapy trials for LGMD, including our trials for product candidates SRP-9003 (LGMD2E/R4/bidridistrogene xeboparvovec), SRP-9004 (LGMD2D/patidistrogene bexoparvovec), SRP-6004 (LGMD2B/R2) and SRP-9005 (LGMD2C/R5 g-sarcoglycan), following the death of a patient in our Phase 1 LGMD clinical trial for SRP-9004. We previously announced on July 16, 2025 that we had suspended each of the LGMD programs mentioned above as part of the Restructuring, with the exception of SRP-9003. In December 2025, the FDA confirmed that we remain on clinical hold and informed us that it requires data from the study of sirolimus as an immunosuppressant before accepting a biologic license application ("BLA") for SRP-9003. We anticipate re-engaging with the agency on next steps for the program after we receive data from Cohort 8 of Study 9001-103 (ENDEAVOR).

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 dystrophia myotonica protein kinase ("DMPK") gene. Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often have cardiac conduction abnormalities, and may become physically disabled and have a shortened life span. SRP-1003 is designed to reduce expression of the DMPK gene. There is currently no approved disease-modifying therapy for DM1. We are currently investigating SRP-1003 in a Phase 1/2 clinical trial, which our strategic partner Arrowhead has conducted to date. We shared early results from this trial in March 2026, and expect to share further data in the second half of 2026. We are in the process of transitioning sponsorship of this study to Sarepta.

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 1/2 clinical trial, which our strategic partner Arrowhead has conducted to date. We shared early results from this trial in March 2026, and expect to share multiple ascending dose data in the fourth quarter of 2026. We are in the process of transitioning sponsorship of this study to Sarepta.

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 1/2 clinical trial, which our strategic partner Arrowhead has conducted to date. We shared early results from this trial in March 2026, and expect to share further data in the second half of 2026. We are in the process of transitioning sponsorship of this study to Sarepta.

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 1/2 clinical trial, which our strategic partner Arrowhead has conducted to date. We shared early results from this trial in March 2026 and expect to share multiple ascending dose data in the fourth quarter of 2026. We are in the process of transitioning sponsorship of this study to Sarepta.

Previous wording

SRP-1005 (HTT). HTT is a rare and ultimately fatal inherited neurodegenerative disorder which is passed down from generation to generation within affected families. HTT is caused by a mutation in the gene for a protein called huntingtin, which leads to progressive deterioration of nerve cells in the brain, affecting cognition, movement, and behavior. Study SRP-1005-101 (INSIGHTT) is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants. SRP-1005 leverages an advanced TfR1 (transferrin receptor protein 1) approach that uses monovalent fragment antigen binding (fAb) designed for efficient delivery to the central nervous system. Subcutaneous administration is intended to avoid saturating the transferrin receptor and achieve constant and robust penetration across the blood brain barrier. During the second quarter of 2026, we enrolled the first patient in the study.

Current wording

SRP-1005 (HD). HD is a rare and ultimately fatal inherited neurodegenerative disorder which is passed down from generation to generation within affected families. HD is caused by a mutation in the gene for a protein called huntingtin, which leads to progressive deterioration of nerve cells in the brain, affecting cognition, movement, and behavior. Study SRP-1005-101 (INSIGHTT) is a Phase 1, multi-center, dose escalation study that will evaluate the safety and tolerability of subcutaneous dosing of SRP-1005 in approximately 24 participants. SRP-1005 leverages an advanced transferrin receptor protein 1 approach that uses monovalent fragment antigen binding (fAb) designed for efficient delivery to the central nervous system. Subcutaneous administration is intended to avoid saturating the transferrin receptor and achieve constant and robust penetration across the blood brain barrier. During the second quarter of 2026, we enrolled the first patient in the study.

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 (specialized contract manufacturing organizations, or "CMOs"), which will in part utilize these capabilities to support production of certain of our products and product candidates and their components. Specifically, we have entered into agreements with CMOs to produce custom starting materials, active pharmaceutical ingredients ("APIs"), drug product and finished goods for our products and product candidates for both commercial and clinical use. We also have opened facilities over the past several years to further enhance our internal research and development capabilities. However, we currently do not have internal GMP manufacturing capabilities to produce our products and product candidates for commercial and/or clinical use.

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, or specialized contract manufacturing organizations ("CMOs"), which will in part utilize these capabilities to support production of certain of our products and product candidates and their components. Specifically, we have entered into agreements with CMOs to produce custom starting materials, active pharmaceutical ingredients ("APIs"), drug product and finished goods for our products and product candidates for both commercial and clinical use. We also have opened facilities over the past several years to further enhance our internal research and development capabilities. However, we currently do not have internal GMP manufacturing capabilities to produce our products and product candidates for commercial and/or clinical use.

Previous wording

With respect to the siRNA programs in our portfolio, we collaborate with our partner Arrowhead to maintain continuity of manufacturing and testing services for ongoing and future clinical trials. Arrowhead’s cGMP manufacturing facility has the capability to manufacture drug substance at multiple scales available and can expand capacity as needed to support future clinical and potential commercial-scale needs. We plan to leverage existing contract partners, which have technical expertise and experience working with siRNA therapies, for the manufacture of drug product and finished goods as well as for distribution. As appropriate, we will evaluate additional relationships with supply partners for specific products, taking into account production volume, logistics, business continuity and other routine business considerations.

Current wording

With respect to the siRNA programs in our portfolio, we collaborate with our partner Arrowhead to maintain continuity of manufacturing and testing services for ongoing and future clinical trials. We believe Arrowhead’s cGMP manufacturing facility has the capability to manufacture drug substance at multiple scales available and can expand capacity as needed to support future clinical and potential commercial-scale needs. We plan to leverage existing contract partners, which have technical expertise and experience working with siRNA therapies, for the manufacture of drug product and finished goods as well as for distribution. As appropriate, we will evaluate additional relationships with supply partners for specific products, taking into account production volume, logistics, business continuity and other routine business considerations.

Previous wording

As of June 30, 2026, we had $945.0 million of cash, cash equivalents, restricted cash and investments, consisting of $571.1 million of cash and cash equivalents, $362.7 million of investments and $11.2 million of non-current restricted cash and investments. We believe that our balance of cash, cash equivalents and investments, along with cash inflows from operations and availability under our Revolving Credit Facility (defined below), is sufficient to fund our current operational plan for at least the next twelve months.

Current wording

As of September 30, 2026, we had $998.7 million of cash, cash equivalents, restricted cash and investments, consisting of $472.8 million of cash and cash equivalents, $514.8 million of investments and $11.1 million of non-current restricted cash and investments. 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.

Previous wording

Results of Operations for the Three and Six Months Ended June 30, 2026 and 2025

Current wording

Results of Operations for the Three and Nine Months Ended September 30, 2026 and 2025

Previous wording

Net product revenues for the three and six months ended June 30, 2026 decreased by $184.4 million and $465.4 million compared with the same periods in 2025. The decreases were primarily driven by lower ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment.

Current wording

Net product revenues for the three and nine months ended September 30, 2026 decreased by $46.3 million and $511.7 million compared with the same periods in 2025. The decreases were primarily driven by lower ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment.

Previous wording

Collaboration and other revenues for the six months ended June 30, 2026 increased by $241.5 million, or 104%, compared with the six months ended June 30, 2025. The increase was primarily driven by the following:

Current wording

Collaboration and other revenues for the nine months ended September 30, 2026 increased by $269.8 million, or 104%, compared with the nine months ended September 30, 2025. The increase was primarily driven by the following:

Previous wording

$189.5 million increase in collaboration revenue due to $365.0 million of revenue recognized in the six months ended June 30, 2026 related to (1) $325.0 million for Roche's declined option to acquire certain program rights previously recorded as deferred revenue, and (2) $40.0 million from a milestone recognized under the Roche Collaboration Agreement for the first commercial dosing of ELEVIDYS in Japan, as compared to $175.5 million of collaboration revenue recognized in the same period of 2025 related to the expiration of an option for a certain program and the Japan Approval Milestone payment of $63.5 million under the Roche Collaboration Agreement;

Current wording

$189.5 million increase in collaboration revenue due to $365.0 million of revenue recognized in the nine months ended September 30, 2026 related to (1) $325.0 million for Roche's declined option to acquire certain program rights previously recorded as deferred revenue and (2) $40.0 million from a milestone recognized under the Roche Collaboration Agreement for the first commercial dosing of ELEVIDYS in Japan, as compared to $175.5 million of collaboration revenue recognized in the same period of 2025 related to the expiration of an option for a certain program and $63.5 million for the regulatory approval of ELEVIDYS in Japan under the Roche Collaboration Agreement;

Previous wording

$41.1 million increase in contract manufacturing revenue primarily driven by increased deliveries of ELEVIDYS to Roche and increases in our manufacturing costs driven by greater-than-expected write-offs of batches of our products not meeting our quality specifications; and

Current wording

$72.5 million increase in contract manufacturing revenue primarily driven by increased deliveries of ELEVIDYS to Roche and increases in our manufacturing costs which impact the selling price of ELEVIDYS delivered to Roche; and

Previous wording

$10.0 million increase in license revenue related to the grant of intellectual property rights under a certain license agreement executed during the six months ended June 30, 2026, with no similar activity during the same period in 2025.

Current wording

$10.0 million increase in license revenue related to the grant of intellectual property rights under a certain license agreement executed during the nine months ended September 30, 2026, with no similar activity during the same period in 2025.

Previous wording

For the PMO Products, all previously expensed manufacturing costs had been fully consumed by December 2022. For ELEVIDYS sold in the three and six months ended June 30, 2026 and June 30, 2025, a portion of related manufacturing costs incurred had previously been expensed as research and development expenses. If certain product related costs had not previously been expensed as research and development expenses prior to receiving FDA approval, the incremental inventory costs related to ELEVIDYS sold, including products sold to Roche under the Roche Collaboration Agreement, would have been $2.8 million and $5.4 million higher for the three and six months ended June 30, 2026, respectively, as compared to $4.5 million and $18.2 million for the three and six months ended June 30, 2025, respectively.

Current wording

For the PMO Products, all previously expensed manufacturing costs had been fully consumed by December 2022. For ELEVIDYS sold in the three and nine months ended September 30, 2026 and September 30, 2025, a portion of related manufacturing costs incurred had previously been expensed as research and development expenses. If certain product related costs had not previously been expensed as research and development expenses prior to receiving FDA approval, the incremental inventory costs related to ELEVIDYS sold, including products sold to Roche under the Roche Collaboration Agreement, would have been $2.5 million and $7.9 million higher for the three and nine months ended September 30, 2026, respectively, as compared to $2.0 million and $20.2 million for the three and nine months ended September 30, 2025, respectively.

Previous wording

The cost of sales (excluding amortization of in-licensed rights) for the three months ended June 30, 2026 decreased by $3.2 million, or 2%, compared with the same period in 2025. The decrease was primarily driven by the following:

Current wording

The cost of sales (excluding amortization of in-licensed rights) for the three months ended September 30, 2026 increased by $37.7 million, or 25%, compared with the same period in 2025. The increase was primarily driven by the following:

Previous wording

$36.4 million increase in Roche product cost of sales primarily due to the timing of allocation of costs to Roche associated with write-offs of certain batches of ELEVIDYS not meeting our quality specifications, scrapped and expired materials and the volume of ELEVIDYS shipments under the Roche Collaboration Agreement; and

Current wording

$30.1 million increase in Roche product cost of sales primarily due to increases in manufacturing costs, the volume of ELEVIDYS shipments under the Roche Collaboration Agreement, the timing of allocation of costs to Roche associated with write-offs of certain batches of ELEVIDYS not meeting our quality specifications and scrapped and expired materials; and

Previous wording

$3.8 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.

Current wording

$1.2 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.

Previous wording

The cost of sales (excluding amortization of in-licensed rights) for the six months ended June 30, 2026 decreased by $32.0 million, or 11%, compared with the same period in 2025. The decrease was primarily driven by the following:

Current wording

The cost of sales (excluding amortization of in-licensed rights) for the nine months ended September 30, 2026 increased by $5.8 million, or 1%, compared with the same period in 2025. The increase was primarily driven by the following:

Previous wording

$35.8 million decrease in product cost of sales (excluding Roche) primarily due to decreased ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment as well as a lower allocation of costs to us associated with batches of ELEVIDYS not meeting our quality specifications, partially offset by an increase in batches of our PMO Products not meeting our quality specifications;

Current wording

$75.7 million decrease in product cost of sales (excluding Roche) primarily due to decreased ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment as well as decreased write-offs of certain batches of ELEVIDYS not meeting our quality specifications and an impairment of prepaid manufacturing deposits recorded during the nine months ended September 30, 2025. The decrease was partially offset by a take-or-pay charge of $62.0 million recorded during the nine months ended September 30, 2026 related to non-cancelable manufacturing obligations with Catalent for certain inventories that we elected not to manufacture, as well as increased manufacturing costs for the PMO Products;

Previous wording

$63.9 million increase in Roche product cost of sales primarily due to the timing of allocation of costs to Roche associated with write-offs of certain batches of ELEVIDYS not meeting our quality specifications, scrapped and expired materials and the volume of ELEVIDYS shipments under the Roche Collaboration Agreement; and

Current wording

$93.9 million increase in Roche product cost of sales primarily due to the timing of allocation of costs to Roche associated with write-offs of certain batches of ELEVIDYS not meeting our quality specifications, scrapped and expired materials and increases in the volume and manufacturing costs of ELEVIDYS shipments under the Roche Collaboration Agreement; and

Previous wording

$11.2 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.

Current wording

$12.5 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.

Previous wording

$10.4 million decrease in clinical trial expenses primarily related to the pause of several studies for our LGMD programs as a result of our decision to reprioritize our pipeline announced in July 2025 (the "Pipeline Reprioritization") and the completion of certain SRP-9001 and PMO studies in 2025, partially offset by increased activity in our siRNA programs;

Current wording

$8.1 million decrease in clinical trial expenses primarily related to the completion of certain PMO studies as well as the pause of several studies for our LGMD programs as a result of our decision to reprioritize our pipeline announced in July 2025 (the "Pipeline Reprioritization"), partially offset by increased activity in our siRNA programs;

Previous wording

$3.7 million decrease in professional services primarily related to fewer third-party contractors used during the three months ended June 30, 2026 due to the Pipeline Reprioritization and the completion of certain SRP-9001 studies in 2025;

Current wording

$3.3 million decrease in professional services primarily related to fewer third-party contractors used during the three months ended September 30, 2026 due to the Pipeline Reprioritization and the completion of certain SRP-9001 studies in 2025;

Previous wording

$78.8 million decrease in manufacturing expenses primarily due to fewer clinical batches released for our SRP-9001 and LGMD programs as a result of our Pipeline Reprioritization, as well as our settlement agreement with Brammer Bio MA, LLC (“Brammer”) to resolve outstanding claims related to the termination of the Thermo Agreement (the “Brammer Settlement”) during the three months ended June 30, 2025, with no similar activity in 2026;

Current wording

$31.6 million decrease in manufacturing expenses primarily due to fewer clinical batches released for our SRP-9001 program;

Previous wording

$38.9 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to reduced SRP-9001 clinical supply as well as reduced headcount pursuant to the Restructuring resulting in lower reimbursable costs.

Current wording

$5.0 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to reduced SRP-9001 clinical supply.

Previous wording

$25.3 million decrease in clinical trial expenses primarily related to the pause of several studies for our LGMD programs as a result of our Pipeline Reprioritization and the completion of certain SRP-9001 and PMO studies in 2025, partially offset by increased activity in our siRNA programs;

Current wording

$33.4 million decrease in clinical trial expenses primarily related to the completion of certain PMO studies and the pause of several studies for our SRP-9001 and LGMD programs, the latter of which as a result of the Pipeline Reprioritization, partially offset by increased activity in our siRNA programs;

Previous wording

$5.9 million decrease in facility- and technology-related expenses primarily as a result of the Restructuring;

Current wording

$19.6 million decrease in facility- and technology-related expenses primarily as a result of the Restructuring and lower depreciation expense resulting from certain assets becoming fully depreciated during 2025;

Previous wording

$533.8 million decrease in up-front and collaboration license fees primarily due to the $583.6 million in payments allocated to the up-front license fee associated with our exclusive global licensing and collaboration agreement and stock purchase agreement (collectively, the “Arrowhead Collaboration Agreement”) with Arrowhead recognized during the six months ended June 30, 2025, partially offset by the $50.0 million annual collaboration license fee incurred and paid to Arrowhead pursuant to the Arrowhead Collaboration Agreement recognized during the six months ended June 30, 2026;

Current wording

$633.8 million decrease in up-front and collaboration license fees related to our exclusive global licensing and collaboration agreement and stock purchase agreement (collectively, the “Arrowhead Collaboration Agreement”) with Arrowhead. For the nine months ended September 30, 2025, we recorded (1) $583.6 million in payments allocated to the up-front license fee and (2) a $100.0 million milestone payment to Arrowhead for the First Arrowhead DM1 Milestone, neither of which recurred in the nine months ended September 30, 2026. For the nine months ended September 30, 2026, we recorded $50.0 million in annual collaboration license fee incurred and paid to Arrowhead;

Previous wording

$6.8 million decrease in stock-based compensation primarily due to reduced headcount pursuant to the Restructuring and the completion of vesting for certain restricted stock units with performance conditions ("PSUs") in March 2026 with no similar award activity during the three months ended June 30, 2026;

Current wording

$9.4 million decrease in stock-based compensation primarily related to reduced headcount pursuant to the Restructuring and the vesting of certain restricted stock units with performance conditions ("PSUs") in March 2025 and March 2026, partially offset by the reversal of previously recognized expense related to the forfeiture of certain awards in connection with the Restructuring during the nine months ended September 30, 2025, with no similar activity during the nine months ended September 30, 2026;

Previous wording

$126.6 million decrease in manufacturing expenses primarily due to fewer clinical batches released for our SRP-9001 and LGMD programs as a result of the Pipeline Reprioritization, as well as the Brammer Settlement during the six months ended June 30, 2025, with no similar activity in 2026;

Current wording

$158.2 million decrease in manufacturing expenses primarily due to fewer clinical batches released for our SRP-9001 and LGMD programs, the latter of which as a result of the Pipeline Reprioritization, as well as the Brammer Settlement during the nine months ended September 30, 2025, with no similar activity in 2026;

Previous wording

$8.2 million decrease in professional services primarily related to fewer third-party contractors used during the six months ended June 30, 2026 due to the completion of certain SRP-9001 and PMO studies in 2025 and the Pipeline Reprioritization;

Current wording

$11.5 million decrease in professional services primarily related to fewer third-party contractors used during the nine months ended September 30, 2026 due to the completion of certain SRP-9001 and PMO studies in 2025 and the Pipeline Reprioritization;