6 unchanged sentences
• 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;
−Removed: • our expectation that our partnerships with manufacturers will support our clinical and commercial manufacturing capacity for our products and product candidates, including our 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;
−Removed: • the possible impacts of the ELEVIDYS Suspension (as defined herein);
+Added: • our expectation that our partnerships with manufacturers will support our clinical and commercial manufacturing capacity for our products and product candidates, including our phosphorodiamidate morpholino oligomer (“PMO”), gene therapy, SRP-9003 Limb-girdle muscular dystrophy (“LGMD”), and small interfering RNA (“siRNA”) programs, while also acting as a manufacturing platform for potential future programs, and our belief that our current network of manufacturing partners is able to fulfill the requirements of our commercial plan;
• the possible impacts of the clinical hold the U.S.
1 unchanged sentence
• 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;
−Removed: • the estimated and potential impacts of the strategic restructuring plan announced in July 2025 ("the Restructuring");
+Added: • the possible impacts of the ELEVIDYS Suspension (as defined herein);
+Added: • the estimated and potential impacts of the strategic restructuring plan announced in July 2025;
• our expectation that our partnership with Catalent, Inc.
2 unchanged sentences
• the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business, including the addition of a boxed warning for acute liver injury ("ALI") and acute liver failure (“ALF”) and removal of the non-ambulatory population from the Indication and Usage section of the Prescribing Information for ELEVIDYS, as well as the development of our product candidates and our financial and contractual obligations;
−Removed: • 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 the Company's siRNA product candidates, SRP-1001 and SRP-1003;
+Added: • estimated timelines and milestones for the remainder of 2026 and beyond, including discussions with the FDA regarding ELEVIDYS, VYONDYS 53, AMONDYS 45 and SRP-9003, and sharing data for certain of our siRNA product candidates, SRP-1001 and SRP-1003;
• our expectations regarding the ongoing study to evaluate the use of sirolimus as an enhanced immunosuppression regimen as part of treatment with ELEVIDYS for non-ambulant individuals living with Duchenne, including discussions with the FDA and possible impacts on the resumption of dosing in the non-ambulatory population;
1 unchanged sentence
including the European Medicines Agency (the “EMA”);
−Removed: • 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;
+Added: • our ability to maintain, and our plan to continue building out our distribution network for in jurisdictions in which our products are approved or in which we are seeking to make our products available, commercially or through our EAP (as defined herein);
• our plan to expand our pipeline through internal research and development and through strategic transactions;
9 unchanged sentences
• our expectation regarding the impact of environmental laws and regulations on our business;
−Removed: • our expectation regarding the outcomes or impacts of our ongoing litigation that we are currently, or may in the future become, party to;
−Removed: • our expectation regarding our ability to satisfy the conditions to borrow under our Credit Agreement;
+Added: • our expectation regarding the outcomes or impacts of our ongoing litigations that we are currently, or may in the future become, party to;
+Added: • our expectation regarding our ability to satisfy the conditions to borrow under our Credit Agreement (defined below);
• our beliefs and expectations regarding milestone, royalty or other payments that could be due to third parties under existing agreements.
7 unchanged sentences
We are also developing potential therapeutic candidates through our partnered program with Arrowhead Pharmaceuticals, Inc.
−Removed: ("Arrowhead"), including Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia ("SCA"), Idiopathic Pulmonary Fibrosis ("IPF"), and Huntington's disease.
+Added: ("Arrowhead"), including Facioscapulohumeral muscular dystrophy ("FSHD"), myotonic dystrophy type 1 ("DM1"), Spinocerebellar ataxia ("SCA"), and Huntington's disease ("HTT").
To date, we have developed and commercialized four products that have been approved by the FDA:
2 unchanged sentences
EXONDYS 51 uses our phosphorodiamidate morpholino oligomer ("PMO") chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene.
−Removed: o VYONDYS 53 (golodirsen) Injection ("VYONDYS 53"), granted accelerated approval by the FDA in 2019, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the
−Removed: dystrophin gene that is amenable to exon 53 skipping.
+Added: o VYONDYS 53 (golodirsen) Injection ("VYONDYS 53"), granted accelerated approval by the FDA in 2019, is indicated for the treatment of Duchenne in patients who have a confirmed mutation of the dystrophin gene that is amenable to exon 53 skipping.
VYONDYS 53 uses our PMO chemistry and exon-skipping technology to skip exon 53 of the dystrophin gene.
15 unchanged sentences
The topline results did not show statistical significance on the study's primary endpoint.
−Removed: We intend to discuss with FDA the potential pathway forward.
−Removed: We are also in the process of conducting various clinical trials for ELEVIDYS, including Cohort 8 of Study 9001-103, 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.
+Added: 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.
+Added: 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.
2 unchanged sentences
territories, we use the designation of ELEVIDYS, which is recognized as inventory in our unaudited condensed consolidated balance sheets.
−Removed: 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 income (loss).
+Added: Separately, when referring to manufactured product for clinical use, such as clinical trials, we use the designation of SRP-9001, which is recognized as research and development expense in our unaudited condensed consolidated statements of comprehensive (loss) income.
Our pipeline includes programs at various stages of discovery, pre-clinical and clinical development.
11 unchanged sentences
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.
−Removed: 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).
+Added: In December 2024, we announced that we
+Added: 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.
1 unchanged sentence
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.
−Removed: 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.
+Added: In December 2025, the FDA confirmed that we remain on clinical hold and informed us that it requires data from the study of sirolimus as an immunosuppressant before accepting a biologic license application ("BLA") for SRP-9003.We anticipate re-engaging with the agency on next steps for the program after we receive data from Cohort 8 of Study 9001-103 (ENDEAVOR).
• SRP-1003 (DM1) .
13 unchanged sentences
We are in the process of transitioning sponsorship of this study to Sarepta.
+Added: • SRP-1005 ( HTT ).
+Added: HTT is a rare and ultimately fatal inherited neurodegenerative disorder which is passed down from generation to generation within affected families.
+Added: 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.
+Added: 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.
+Added: 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.
+Added: Subcutaneous administration is intended to avoid saturating the transferrin receptor and achieve constant and robust penetration across the blood brain barrier.
+Added: During the second quarter of 2026, we enrolled the first patient in the study.
Manufacturing, Supply and Distribution
9 unchanged sentences
We believe our current network of CMOs is able to fulfill our requirements for quantity, quality and purity of our commercial products and product candidates, and is capable of expanding capacity as needed.
−Removed: Additionally, we have evaluated and will continue to evaluate further relationships with additional suppliers as appropriate for specific products, taking into account production volume, logistics, business continuity and other routine business considerations.
+Added: Additionally, we have evaluated and will continue to
+Added: evaluate further relationships with additional suppliers as appropriate for specific products, taking into account production volume, logistics, business continuity and other routine business considerations.
Our gene therapy manufacturing capabilities continue to benefit from partnerships with Aldevron and Catalent.
5 unchanged sentences
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.
−Removed: With respect to the precommercial distribution of our products to patients outside of the U.S., we have contracted
−Removed: with third-party distributors and service providers to distribute our products in certain countries through our EAPs.
+Added: 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.
5 unchanged sentences
Cash, Cash Equivalents, Restricted Cash and Investments
−Removed: As of March 31, 2026, we had $748.3 million of cash, cash equivalents, restricted cash and investments, consisting of $464.5 million of cash and cash equivalents, $270.7 million of investments and $13.1 million of non-current restricted cash.
+Added: 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.
10 unchanged sentences
There have been no changes to our critical accounting policies and significant estimates as detailed in our Annual Report on Form 10-K for the year ended December 31, 2025.
−Removed: Results of Operations for the Three Months Ended March 31, 2026 and 2025
−Removed: The following table sets forth selected unaudited condensed consolidated statements of income (loss) data for each of the periods indicated:
+Added: Results of Operations for the Three and Six Months Ended June 30, 2026 and 2025
+Added: The following tables set forth selected unaudited condensed consolidated statements of (loss) income data for each of the periods indicated:
For the Three Months Ended
7 unchanged sentences
Selling, general and administrative
+Added: Litigation contingency charge
Amortization of in-licensed rights
Total cost and expenses
+Added: Operating income
+Added: Other (loss) income, net:
+Added: Other (expense) income, net
+Added: (Loss) income before income tax expense (benefit)
+Added: Income tax expense (benefit)
+Added: Net (loss) income
+Added: (Loss) earnings per share
+Added: *Not meaningful
+Added: For the Six Months Ended
+Added: (in thousands, except per share amounts)
+Added: Products, net
+Added: Collaboration and other
+Added: Total revenues
+Added: Cost and expenses:
+Added: Cost of sales (excluding amortization of in-licensed rights)
+Added: Research and development
+Added: Selling, general and administrative
+Added: Litigation contingency charge
+Added: Amortization of in-licensed rights
+Added: Total cost and expenses
Operating income (loss)
10 unchanged sentences
Actual amounts of consideration ultimately received or paid may differ from our estimates.
−Removed: The following table summarizes the components of our net product revenues, by product group, for each of the periods indicated:
+Added: The following tables summarize the components of our net product revenues, by product group, for each of the periods indicated:
For the Three Months Ended
1 unchanged sentence
Products, net
−Removed: Net product revenues for the three months ended March 31, 2026 decreased by $281.0 million, or 46%, compared with the same period in 2025.
−Removed: The decrease was primarily driven by lower ELEVIDYS sales volume, reflecting changes in demand following
−Removed: the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment.
−Removed: The following table summarizes the components of our collaboration and other revenues for the periods indicated:
−Removed: For the Three Months Ended
+Added: For the Six Months Ended
(in thousands)
−Removed: Collaboration revenue
−Removed: Contract manufacturing
−Removed: Royalty revenue
−Removed: Total collaboration and other
−Removed: Collaboration and other revenues primarily relate to our collaboration arrangement (the “Roche Collaboration Agreement”) with F.
−Removed: Hoffman-La Roche Ltd.
−Removed: For the three months ended March 31, 2026 and 2025, we recognized $400.3 million and $133.3 million of collaboration and other revenues, respectively.
−Removed: The increase is primarily due to the $365.0 million of collaboration revenue recognized in the three months ended March 31, 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 $112.0 million of collaboration revenue recognized in the same period of 2025 related to the expiration of an option for a certain program.
−Removed: Please refer to Note 3, License and Collaboration Agreements for further discussion of the Roche Collaboration Agreement.
+Added: Products, net
+Added: 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.
+Added: 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.
+Added: Collaboration and other revenues primarily relate to our collaboration agreement (the “Roche Collaboration Agreement”) with F.
+Added: Hoffmann-La Roche Ltd.
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”).
3 unchanged sentences
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.
−Removed: For the three months ended March 31, 2026 and 2025, we recognized $31.1 million and $17.4 million of contract manufacturing revenue, respectively, which is related to these shipments to Roche.
−Removed: In addition, we recognized $4.2 million and approximately $4.0 million of royalty revenue from sales of ELEVIDYS by Roche for the three months ended March 31, 2026 and 2025, respectively.
+Added: The transaction price of the contract manufacturing revenue is estimated at contract inception and reassessed annually and on an as-needed basis when additional information becomes available.
+Added: The following tables summarize the components of our collaboration and other revenues for the periods indicated:
+Added: For the Three Months Ended
+Added: (in thousands)
+Added: Contract manufacturing
+Added: License revenue
+Added: Royalty revenue
+Added: Collaboration revenue
+Added: Total collaboration and other
+Added: *Not meaningful
+Added: Collaboration and other revenues for the three months ended June 30, 2026 decreased by $25.4 million, or 26%, compared with the three months ended June 30, 2025.
+Added: The decrease was primarily driven by the following:
+Added: • $27.4 million increase in contract manufacturing revenue primarily driven by increased deliveries of ELEVIDYS to Roche and increases in our manufacturing costs driven by greater-than-expected write-offs of batches of our products not meeting our quality specifications;
+Added: • $10.0 million increase in license revenue related to the grant of intellectual property rights under a certain license agreement executed during the three months ended June 30, 2026, with no similar activity during the same period in 2025;
+Added: • $63.5 million decrease in collaboration revenue due to the recognition of milestone payments during the three months ended June 30, 2025 under the Roche Collaboration Agreement for the regulatory approval of ELEVIDYS in Japan (the “Japan Approval Milestone”) with no similar activity during the three months ended June 30, 2026.
+Added: Please refer to Note 3, License and Collaboration Agreements for further discussion of the Japan Approval Milestone.
+Added: For the Six Months Ended
+Added: (in thousands)
+Added: Collaboration revenue
+Added: Contract manufacturing
+Added: Royalty revenue
+Added: License revenue
+Added: Total collaboration and other
+Added: *Not meaningful
+Added: 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.
+Added: The increase was primarily driven by the following:
+Added: • $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
+Added: 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;
+Added: • $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;
+Added: • $10.0 million increase in license revenue related to the grant of intellectual property rights under a certain license agreement executed during the six months ended June 30, 2026, with no similar activity during the same period in 2025.
Cost of sales (excluding amortization of in-licensed rights)
3 unchanged sentences
For the PMO Products, all previously expensed manufacturing costs had been fully consumed by December 2022.
−Removed: For ELEVIDYS sold in the three months ended March 31, 2026 and March 31, 2025, a portion of related manufacturing costs incurred had previously been expensed as research and development expenses.
−Removed: 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 $2.6 million and $13.7 million higher for the three months ended March 31, 2026 and 2025, respectively.
−Removed: The following table summarizes the components of our cost of sales (excluding amortization of in-licensed rights) for each of the periods indicated:
+Added: 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.
+Added: 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.
+Added: The following tables summarize the components of our cost of sales (excluding amortization of in-licensed rights) for each of the periods indicated:
For the Three Months Ended
5 unchanged sentences
**See above for further details regarding product supply sold to Roche via contract manufacturing under the Roche Collaboration Agreement .
−Removed: The cost of sales (excluding amortization of in-licensed rights) for the three months ended March 31, 2026 decreased by $28.8 million, or 21%, compared with the same period in 2025.
−Removed: The decrease was 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.
−Removed: This decrease was partially offset by increases in (1) scrapped and expired materials, (2) the volume of ELEVIDYS shipments under the Roche Collaboration Agreement and (3) the write-offs of certain batches of our products not meeting our quality specifications, a portion of which was allocated to Roche product cost of sales.
+Added: 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.
+Added: The decrease was primarily driven by the following:
+Added: • $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;
+Added: • $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;
+Added: • $3.8 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.
+Added: For the Six Months Ended
+Added: (in thousands)
+Added: Product cost of sales (excluding Roche)
+Added: Roche product cost of sales**
+Added: Royalty payments
+Added: Total cost of sales (excluding amortization of in-licensed rights)
+Added: **See above for further details regarding product supply sold to Roche via contract manufacturing under the Roche Collaboration Agreement .
+Added: 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.
+Added: The decrease was primarily driven by the following:
+Added: • $84.6 million decrease in product cost of sales (excluding Roche) primarily due to decreased ELEVIDYS sales volume, reflecting changes in demand following the safety events that occurred in 2025 and subsequent label update for ELEVIDYS which included only the ambulatory patient population for treatment as well as a lower allocation of costs to us associated with batches of ELEVIDYS not meeting our quality specifications, partially offset by an increase in batches of our PMO Products not meeting our quality specifications;
+Added: • $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;
+Added: • $11.2 million decrease in royalty payments primarily corresponding to the decreased ELEVIDYS sales volume in the U.S.
Research and development expenses
5 unchanged sentences
As a result, a significant portion of our research and development expenses are not tracked on a project-by-project basis, as the costs may benefit multiple projects.
−Removed: The following table summarizes our research and development expenses by project for each of the periods indicated:
+Added: The following tables summarize our research and development expenses by project for each of the periods indicated:
For the Three Months Ended
(in thousands)
−Removed: Up-front and collaboration license fees
siRNA platform
+Added: Eteplirsen (exon 51)
+Added: Other gene therapies
LGMD platform
+Added: Casimersen (exon 45)
+Added: Golodirsen (exon 53)
+Added: Other projects
+Added: Internal research and development expenses
+Added: Roche collaboration reimbursement
+Added: Total research and development expenses
+Added: For the Six Months Ended
+Added: (in thousands)
+Added: Up-front and collaboration license fees
+Added: siRNA platform
Eteplirsen (exon 51)
+Added: LGMD platform
Other gene therapies
5 unchanged sentences
Total research and development expenses
−Removed: *Not meaningful
−Removed: The following table summarizes our research and development expenses by category for each of the periods indicated:
+Added: The following tables summarize our research and development expenses by category for each of the periods indicated:
For the Three Months Ended
(in thousands)
−Removed: Up-front and collaboration license fees
Compensation and other personnel expenses
−Removed: Manufacturing expenses
+Added: Clinical trial expenses
Facility- and technology-related expenses
+Added: Stock-based compensation
+Added: Professional services
+Added: Manufacturing expenses
+Added: Pre-clinical expenses
+Added: Research and other
+Added: Roche collaboration reimbursement
+Added: Total research and development expenses
+Added: Research and development expenses for the three months ended June 30, 2026 decreased by $113.1 million, or 55%, compared with the three months ended June 30, 2025.
+Added: The decrease was primarily driven by the following:
+Added: • $17.8 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
+Added: • $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;
+Added: • $5.9 million decrease in facility- and technology-related expenses primarily as a result of the Restructuring;
+Added: • $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;
+Added: • $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;
+Added: • $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;
+Added: • $8.0 million decrease in research and other expenses primarily due to ongoing cost reduction efforts and the Pipeline Reprioritization;
+Added: • $18.6 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to reduced SRP-9001 clinical supply as well as reduced headcount pursuant to the Restructuring resulting in lower reimbursable costs.
+Added: For the Six Months Ended
+Added: (in thousands)
+Added: Compensation and other personnel expenses
+Added: Up-front and collaboration license fees
Clinical trial expenses
+Added: Facility- and technology-related expenses
+Added: Manufacturing expenses
Stock-based compensation
4 unchanged sentences
Total research and development expenses
−Removed: Research and development expenses for the three months ended March 31, 2026 decreased by $619.5 million, or 80%, compared with the three months ended March 31, 2025.
+Added: Research and development expenses for the six months ended June 30, 2026 decreased by $732.6 million, or 75%, compared with the six months ended June 30, 2025.
The decrease was primarily driven by the following:
−Removed: • $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 Agreement”) with Arrowhead recognized in the three months ended March 31, 2025, partially offset by the $50.0 million annual collaboration license fee incurred and paid to Arrowhead pursuant to the Arrowhead Agreement recognized in the three months ended March 31, 2026;
• $39.0 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
−Removed: • $47.7 million decrease in manufacturing expenses primarily due to fewer clinical batches released for our SRP-9001 and LGMD programs as a result of our decision to reprioritize our pipeline announced in July 2025 ("Pipeline Reprioritization") and development priorities;
−Removed: • $5.5 million decrease in facility- and technology-related expenses primarily as a result of our Restructuring;
−Removed: • $14.8 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, resulting in less clinical trial expense for the three months ended March 31, 2026;
−Removed: • $7.0 million decrease in stock-based compensation primarily related to the fulfillment of remaining service conditions associated with certain PSUs in March 2025 with no similar award activity in 2026 and reduced headcount pursuant to our Restructuring;
−Removed: • $4.5 million decrease in professional services primarily related to fewer third-party contractors used during the three months ended March 31, 2026 due to the Pipeline Reprioritization and the completion of certain SRP-9001 and PMO studies in 2025;
−Removed: • $4.1 million decrease in research and other expenses primarily due to the Pipeline Reprioritization and ongoing cost reduction efforts;
−Removed: • $20.3 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 our Restructuring resulting in lower reimbursable costs.
+Added: • $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;
+Added: • $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;
+Added: • $11.4 million decrease in facility- and technology-related expenses primarily as a result of the Restructuring;
+Added: • $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;
+Added: • $13.8 million decrease in stock-based compensation primarily related to reduced headcount pursuant to the Restructuring and the vesting of certain PSUs in March 2025 and March 2026;
+Added: • $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;
+Added: • $1.6 million decrease in pre-clinical expenses primarily due to the Pipeline Reprioritization;
+Added: • $12.1 million decrease in research and other expenses primarily due to ongoing cost reduction efforts and the Pipeline Reprioritization;
+Added: • $38.9 million decrease in the offset to expense associated with a collaboration reimbursement from Roche primarily due to reduced SRP-9001 clinical supply as well as reduced headcount pursuant to the Restructuring resulting in lower reimbursable costs.
Selling, general and administrative expenses
1 unchanged sentence
Other general and administrative expenses include an allocation of our facility- and technology-related costs and professional fees for legal, consulting and accounting services.
−Removed: The following table summarizes our selling, general and administrative expenses by category for each of the periods indicated:
+Added: The following tables summarize our selling, general and administrative expenses by category for each of the periods indicated:
For the Three Months Ended
(in thousands)
+Added: Professional services
Compensation and other personnel expenses
+Added: Stock-based compensation
+Added: Facility- and technology-related expenses
+Added: Roche collaboration reimbursement
+Added: Total selling, general and administrative expenses
+Added: Selling, general and administrative expenses for the three months ended June 30, 2026 decreased by $30.3 million, or 22% compared with the three months ended June 30, 2025.
+Added: This decrease was primarily driven by the following:
+Added: • $12.3 million decrease in professional service expenses primarily related to reduced ELEVIDYS commercialization spending following the label update for ELEVIDYS which included only the ambulatory patient population for treatment and reduced legal spending driven by the settlement of a certain matter in 2025;
+Added: • $11.3 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
+Added: • $5.8 million decrease in stock-based compensation primarily related to reduced headcount pursuant to the Restructuring and the vesting of certain PSUs in March 2026 with no similar award activity during the three months ended June 30, 2026, partially offset by expense recognized for RSUs granted to our CEO in December 2025;
+Added: • $1.6 million decrease in other expenses primarily related to the timing of charitable donations.
+Added: For the Six Months Ended
+Added: (in thousands)
Professional services
+Added: Compensation and other personnel expenses
Stock-based compensation
2 unchanged sentences
Total selling, general and administrative expenses
−Removed: Selling, general and administrative expenses for the three months ended March 31, 2026 decreased by $24.7 million, or 18%, compared with the three months ended March 31, 2025.
+Added: Selling, general and administrative expenses for the six months ended June 30, 2026 decreased by $55.0 million, or 20%, compared with the six months ended June 30, 2025.
This decrease was primarily driven by the following:
−Removed: • $10.0 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to our Restructuring;
−Removed: • $8.4 million decrease in professional service expenses primarily related to reduced ELEVIDYS commercialization spending following the label update for ELEVIDYS which included only the ambulatory patient population for treatment and reduced medical affairs spending related to our Pipeline Reprioritization;
−Removed: • $5.0 million decrease in stock-based compensation primarily related to the fulfillment of remaining service conditions associated with certain PSUs in March 2025 with no similar award activity in 2026, and reduced headcount, partially offset by three months of expense recognition for RSUs granted to our CEO in December 2025;
+Added: • $20.7 million decrease in professional service expenses primarily related to reduced ELEVIDYS commercialization spending following the label update for ELEVIDYS which included only the ambulatory patient population for treatment, reduced legal spending driven by the settlement of a certain matter in 2025 and reduced medical affairs spending related to our Pipeline Reprioritization;
+Added: • $21.3 million decrease in compensation and other personnel expenses primarily due to reduced headcount pursuant to the Restructuring;
+Added: • $10.8 million decrease in stock-based compensation primarily related to reduced headcount pursuant to the Restructuring and the vesting of certain PSUs in March 2025 and March 2026, partially offset by expense recognized for RSUs granted to our CEO in December 2025;
+Added: • $2.0 million increase in facility- and technology-related expenses primarily related to the utilization of our Bedford, Massachusetts facility, which was placed into service in June 2025;
• $4.1 million decrease in other expenses primarily related to the timing of charitable donations.
+Added: Litigation contingency charge
+Added: During the three and six months ended June 30, 2026, we recorded a litigation contingency charge of $39.0 million related to the potential resolution of certain patent litigations.
+Added: Following the parties' agreement in principle and based on management's assessment of the available information, we determined that a loss was probable and estimable as of June 30, 2026 and recognized our best estimate of the liability.
+Added: The potential settlement remains outstanding subject to further negotiation and execution of definitive documentation as of the issuance of these unaudited condensed consolidated financial statements.
Amortization of in-licensed rights
1 unchanged sentence
("BioMarin") and Parent Project Muscular Dystrophy in April 2013, December 2016, July 2017 and May 2018, respectively.
−Removed: 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.
−Removed: For the three months ended March 31, 2026 and 2025, we recorded amortization of in-licensed rights of approximately $0.7 million and $0.6 million, respectively.
−Removed: Other expense, net
−Removed: Other 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 September 15, 2027 (the “2027 Notes”) and our 4.875% convertible senior notes due September 1, 2030 (the “2030 Notes”), interest income on our cash, cash equivalents and investments and accretion of investment discount.
+Added: Each in-licensed right is being amortized on a straight-line basis over the remaining life of the relevant patent from the
+Added: date the related fee was incurred, either the regulatory approval or the first commercial sale of the applicable product.
+Added: For the three and six months ended June 30, 2026, we recorded amortization of in-licensed rights of approximately $0.7 million and $1.4 million, respectively.
+Added: 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.
+Added: Other (expense) income, net
+Added: Other (expense) income, net primarily consists of the unrealized gain or loss from our investments in our strategic equity investments, interest expense on our 1.25% convertible senior notes due September 15, 2027 (the “2027 Notes”) and our 4.875% convertible senior notes due September 1, 2030 (the “2030 Notes”), interest income on our cash, cash equivalents and investments and accretion of investment discount.
Our cash equivalents and investments consist of money market funds, government and government agency bonds, corporate bonds, commercial paper and certificates of deposit.
−Removed: For the three months ended March 31, 2026, other expense, net decreased by $67.9 million compared with the three months ended March 31, 2025.
−Removed: The change was primarily due to an $89.0 million decrease in the loss on our strategic investments, primarily related to our investment in Arrowhead during the three months ended March 31, 2025, which we sold in August 2025.
−Removed: This was partially offset by a $17.2 million increase in interest expense due to the 2030 Notes carrying a higher interest rate than the 2027 Notes, as well as a $5.4 million decrease in interest income as a result of reduced investment balances during the three months ended March 31, 2026 compared to the three months ended March 31, 2025.
−Removed: Income tax expense
−Removed: Income tax expense for the three months ended March 31, 2026 and 2025 was $12.2 million and $64.0 million, respectively.
−Removed: 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.
−Removed: As of March 31, 2026, we continued to maintain a full valuation allowance against our
−Removed: deferred tax assets, with the exception of deferred tax assets in certain foreign jurisdictions.
+Added: For the three months ended June 30, 2026, other expense, net increased by $53.1 million compared with the three months ended June 30, 2025.
+Added: This was primarily due to a $37.2 million decrease in the gain on strategic investments primarily related to our investment in Arrowhead during the three months ended June 30, 2025, which we sold in August 2025, and a $16.3 million increase in interest expense due to the 2030 Notes carrying a higher interest rate than the 2027 Notes.
+Added: For the six months ended June 30, 2026, other expense, net decreased by $14.8 million compared with the six months ended June 30, 2025.
+Added: The change was primarily due to a $51.8 million decrease in the loss on our strategic investments, primarily related to our investment in Arrowhead during the six months ended June 30, 2025.
+Added: This was partially offset by a $33.5 million increase in interest expense due to the 2030 Notes carrying a higher interest rate than the 2027 Notes as well as a decrease in interest income as a result of reduced investment balances during the six months ended June 30, 2026, as compared to the six months ended June 30, 2025.
+Added: Income tax expense (benefit)
+Added: Income tax expense for the three and six months ended June 30, 2026 was $3.1 million and $15.4 million, respectively.
+Added: Income tax (benefit) expense for the three and six months ended June 30, 2025 was $(43.3) million and $20.7 million, respectively.
+Added: Income tax expense for all periods presented primarily relates to state income taxes as a result of taxable profits in certain states requiring the capitalization of research and development costs and states which have suspended or limited the utilization of net operating loss carryforwards.
+Added: As of June 30, 2026, we continued to maintain a full valuation allowance against our deferred tax assets, with the exception of deferred tax assets in certain foreign jurisdictions.
We continue to monitor the available evidence relative to recovery of our deferred tax assets and whether such evidence would be sufficient to conclude that it is more likely than not that such deferred tax assets may be partially or fully recoverable.
−Removed: 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.
+Added: If we were to remove our valuation allowance in part or full, any such adjustment could have a material impact on our effective tax rate in the applicable period.
Liquidity and Capital Resources
−Removed: There have been no material changes to our obligations under lease or debt arrangements as reported in our Annual Report on Form 10-K for the year ended December 31, 2025, except for the partial draw down and repayment of the five-year $600.0 million senior secured revolving credit facility entered into in February 2025 (the “Revolving Credit Facility”), as discussed in Note 13, Revolving Credit Facility .
+Added: There have been no material changes to our obligations under lease or debt arrangements as reported in our Annual Report on Form 10-K for the year ended December 31, 2025, except for the partial draw downs and repayments of the five-year $600.0 million senior secured revolving credit facility entered into in February 2025 (the “Revolving Credit Facility”), as discussed in Note 13, Revolving Credit Facility .
The following table summarizes our financial condition for each of the periods indicated:
−Removed: March 31, 2026
+Added: June 30, 2026
December 31, 2025
4 unchanged sentences
Non-current investments
−Removed: Restricted cash
−Removed: Total cash, cash equivalents, restricted cash
−Removed: and investments
+Added: Restricted cash and investments
+Added: Total cash, cash equivalents, restricted cash and investments
Convertible debt
5 unchanged sentences
*Not meaningful
−Removed: For the three months ended March 31, 2026, our principal sources of liquidity were primarily derived from the sales of our products, our collaboration arrangement with Roche and borrowings from the Revolving Credit Facility.
+Added: For the six months ended June 30, 2026, our principal sources of liquidity were primarily derived from the sales of our products, our collaboration arrangement with Roche, borrowings from the Revolving Credit Facility and the maturities and sales of investments.
Please refer to Note 13, Revolving Credit Facility , for further discussion of our borrowing activity.
−Removed: Our principal uses of cash for the three months ended March 31, 2026 were our repayment of borrowings from the Revolving Credit Facility, the payment of $200.0 million for the second DM1 Milestone to Arrowhead, the $50.0 million annual license fee payment to Arrowhead, inventory commitments, research and development expenses, manufacturing costs, selling, general and administrative expenses, investments, capital expenditures and other working capital requirements.
−Removed: For the year ended December 31, 2025, our principal sources of liquidity were primarily derived from the sales of our products, our collaboration arrangement with Roche and proceeds from the exercise of stock options.
+Added: Our principal uses of cash for the six months ended June 30, 2026 were our repayments of borrowings from the Revolving Credit Facility, the payment of $200.0 million for the second DM1 Milestone to Arrowhead, the $50.0 million annual license fee payment to Arrowhead, inventory commitments, research and development expenses, manufacturing costs, selling, general and administrative expenses, investments, capital expenditures and other working capital requirements.
+Added: For the year ended December 31, 2025, our principal sources of liquidity were primarily derived from the sales of our products, our collaboration arrangement with Roche, the maturities and sales of investments and proceeds from the exercise of stock options.
Our principal uses of cash for 2025 were our $583.6 million up-front payment to Arrowhead, $241.4 million equity investment in Arrowhead's common stock and the $50.0 million cash component of the $100.0 million of the first DM1 Milestone, costs incurred as a result of the partial refinancings of the 2027 Notes in August and December 2025, inventory commitments, research and development expenses, manufacturing costs, selling, general and administrative expenses, investments, capital expenditures, share repurchases under our $500.0 million share repurchase program approved by the Board of Directors in November 2024 (the “2024 Repurchase Program”) and other working capital requirements.
−Removed: Please refer to our Annual Report on Form 10-K for the year ended December 31, 2025 for further discussion of share repurchases under the 2024 Repurchase Program during the period.
−Removed: Beyond March 31, 2027, our cash requirements will depend extensively on our ability to advance our research, development and commercialization of product candidates.
−Removed: We may seek additional financings primarily from, but not limited to, the sale and issuance of equity and debt securities, the licensing or sale of our technologies, and entering into additional government contracts
−Removed: and/or funded research and development agreements.
+Added: Please refer to our Annual Report on Form 10-K for the year ended December 31, 2025 for further discussion of share repurchases under the 2024 Repurchase Program.
+Added: Beyond June 30, 2027, our cash requirements will depend extensively on our ability to advance our research, development and commercialization of product candidates.
+Added: We may seek additional financings primarily from, but not limited to, the sale and issuance of equity and debt securities, the licensing or sale of our technologies, and entering into additional government contracts and/or funded research and development agreements.
Our future expenditures and long-term capital requirements may be substantial and will depend on many factors, including but not limited to the following:
2 unchanged sentences
• our ability to realize the benefits of the Restructuring;
−Removed: • the risk that our Restructuring and pipeline reprioritization efforts may not generate their intended benefits to the extent or as quickly as anticipated;
+Added: • the risk that the Restructuring and Pipeline Reprioritization may not generate their intended benefits to the extent or as quickly as anticipated;
• the impact of potential regulatory actions from the FDA including changes to our drug labels or revocation of accelerated approvals and directives to remove products from the market relating to the topline results of our ESSENCE trial that failed to meet statistical significance on its primary endpoint;
14 unchanged sentences
Additional information regarding our obligations under manufacturing arrangements is provided in Note 16, Commitments and Contingencies to the unaudited condensed consolidated financial statements contained in Item 1.
−Removed: There have been no material changes to our obligations under debt or leasing arrangements as reported in our Annual Report on Form 10-K for the year ended December 31, 2025, except for the partial draw down and subsequent repayment of our Revolving Credit Facility, as discussed in Note 13, Revolving Credit Facility.
+Added: There have been no material changes to our obligations under debt or leasing arrangements as reported in our Annual Report on Form 10-K for the year ended December 31, 2025, except for the partial draw downs and subsequent repayments of our Revolving Credit Facility, as discussed in Note 13, Revolving Credit Facility.
For products and product candidates that are currently approved or are in various research and development stages, we may be obligated to make up to $12.1 billion of future development, regulatory, up-front royalty and sales milestone payments associated with our license and collaboration agreements.
Payments under these agreements generally become due and payable upon achievement of certain development, regulatory or commercial milestones.
−Removed: Because the achievement of these milestones is not probable, and payment is not required as of March 31, 2026, such contingencies have not been recorded in our unaudited condensed consolidated financial statements.
+Added: Because the achievement of these milestones is not probable, and payment is not required as of June 30, 2026, such contingencies have not been recorded in our unaudited condensed consolidated financial statements.
Amounts related to contingent milestone payments are not yet considered contractual obligations as they are contingent on the successful achievement of certain development, regulatory approval and commercial milestones.
The following table summarizes our cash flow activity for each of the periods indicated:
−Removed: For the Three Months Ended
+Added: For the Six Months Ended
(in thousands)
5 unchanged sentences
Operating Activities
−Removed: Cash used in operating activities, which consists of our net income (loss) adjusted for non-cash items and changes in net operating assets and liabilities, totaled $202.7 million and $583.4 million for the three months ended March 31, 2026 and 2025, respectively.
−Removed: Cash used in operating activities for the three months ended March 31, 2026 was primarily driven by the net income of $331.0 million, adjusted for the following:
+Added: Cash used in operating activities, which consists of our net income (loss) adjusted for non-cash items and changes in net operating assets and liabilities, totaled $5.6 million and $322.1 million for the six months ended June 30, 2026 and 2025, respectively.
+Added: Cash used in operating activities for the six months ended June 30, 2026 was primarily driven by the net income of $326.1 million, adjusted for the following:
• $53.9 million in stock-based compensation expense;
1 unchanged sentence
• $19.0 million in non-cash interest expense;
+Added: • $11.6 million in write-downs for obsolete inventory;
• $7.2 million in other non-cash items.
1 unchanged sentence
The net cash outflow from changes in our operating assets and liabilities was primarily driven by the following:
−Removed: • $274.9 million decrease in deferred revenue primarily related to the recognition of $325.0 million associated with Roche's declined option to acquire certain program rights previously recorded as collaboration revenue, partially offset by new orders placed by Roche;
−Removed: • $210.5 million decrease in accounts payable primarily related to timing of payments, including the payment of the second DM1 Milestone to Arrowhead during the three months ended March 31, 2026;
−Removed: • $69.8 million increase in inventory primarily due to a continued manufacturing of ELEVIDYS to meet contractual commitments with contract manufacturing organizations;
−Removed: • $60.0 million decrease in accrued expenses primarily due to payment of accrued compensation, timing of payments to our CMOs related to manufacturing commitments and bi-annual payments of interest on 2027 and 2030 Notes during the three months ended March 31, 2026;
+Added: • $307.0 million decrease in deferred revenue primarily related to the recognition of $325.0 million associated with Roche's declined option to acquire certain program rights previously recorded as deferred revenue, partially offset by new orders placed by Roche;
+Added: • $229.9 million decrease in accounts payable primarily related to timing of payments, including the payment of the second DM1 Milestone to Arrowhead during the six months ended June 30, 2026;
+Added: • $37.3 million decrease in accrued expenses primarily due to timing of payments to our CMOs related to manufacturing commitments and payment of accrued compensation, partially offset by the litigation contingency charge;
+Added: • $29.8 million increase in inventory primarily due to a continued manufacturing of ELEVIDYS to meet contractual commitments with contract manufacturing organizations, partially offset by sales of our products and batches of our products not meeting our quality specifications;
+Added: • $21.4 million decrease in accounts receivable primarily due to a decrease in demand for our products from year-end;
+Added: • $67.8 million decrease in other assets primarily driven by the timing of payment on receivables from Roche;
• $69.3 million decrease in manufacturing-related deposits and prepaids primarily due to timing of prepaid raw materials with Catalent.
−Removed: • $14.5 million increase in other assets primarily driven by the timing of compensation payments;
−Removed: • $3.4 million decrease in accounts receivable primarily due to the timing of PMO sales and collections, partially offset by the timing of ELEVIDYS shipments.
−Removed: Cash used in operating activities for the three months ended March 31, 2025 was primarily driven by the net loss of $447.5 million, adjusted for the following:
−Removed: • $90.7 million loss on strategic investments;
+Added: Cash used in operating activities for the six months ended June 30, 2025 was primarily driven by the net loss of $250.6 million, adjusted for the following:
• $78.5 million in stock-based compensation expense;
+Added: • $54.0 million loss on strategic investments;
• $20.8 million in depreciation and amortization expense;
2 unchanged sentences
The net cash outflow from changes in our operating assets and liabilities was primarily driven by the following:
−Removed: • $177.4 million increase in inventory primarily due to a ramp-up of manufacturing corresponding to an increase in demand for ELEVIDYS;
−Removed: • $85.4 million decrease in deferred revenue primarily related to our collaboration with Roche;
−Removed: • $71.1 million decrease in manufacturing-related deposits and prepaids primarily due to timing of prepaid raw materials with Catalent;
+Added: • $236.5 million increase in inventory primarily due to a continuing build-up of ELEVIDYS inventory as a result of label expansion of the therapy in June 2024;
• $77.7 million decrease in accounts payable primarily due to the timing of invoices and payments;
−Removed: • $57.4 million increase in accounts receivable primarily due to an increase in demand for ELEVIDYS;
−Removed: • $31.7 million increase in accrued expense primarily due to the timing of payment for the annual fee on our Revolving Credit Facility;
−Removed: • $9.0 million increase in lease liabilities and other liabilities primarily due to reimbursements received for build-out of our Bedford facility.
+Added: • $59.8 million decrease in deferred revenue primarily related to our collaboration with Roche, partially offset by our contract manufacturing activities with Roche;
+Added: • $37.3 million increase in other assets primarily due to an increase in receivables from Roche associated with contract manufacturing revenue;
+Added: • $15.4 million increase in lease liabilities and other liabilities primarily to reimbursements received for build-out of our Bedford facility;
+Added: • $20.2 million increase in accrued expenses primarily due to increased revenue-related accruals related to increased product revenue, partially offset by payments on accrued employee compensation costs and timing of payments to our CMOs related to manufacturing commitments;
+Added: • $68.9 million decrease in manufacturing-related deposits and prepaids primarily due to timing of payments to Catalent;
+Added: • $74.7 million decrease in accounts receivable, net primarily due to a decrease in demand for ELEVIDYS from year-end.
Investing Activities
−Removed: Cash used in investing activities was $134.4 million for the three months ended March 31, 2026, compared to $291.2 million of cash provided in the three months ended March 31, 2025.
−Removed: Cash used in investing activities for the three months ended March 31, 2026 primarily consisted of $170.2 million of purchases of available-for-sale securities and $2.1 million of purchases of property and equipment, partially offset by $38.9 million from the maturity of available-for-sale securities.
−Removed: Cash used in investing activities for the three months ended March 31, 2025 primarily consisted of $241.4 million in the acquisition of strategic investments related to Arrowhead, $44.7 million of purchases of available-for-sale securities and $43.7 million of purchases of property and equipment, partially offset by $40.8 million from the maturity of available-for-sale securities.
+Added: Cash used in investing activities was $227.8 million and $259.0 million for the six months ended June 30, 2026 and 2025, respectively.
+Added: Cash used in investing activities for the six months ended June 30, 2026 primarily consisted of $351.0 million of purchases of investments and $2.6 million of purchases of property and equipment, partially offset by $126.9 million from the maturity of investments.
+Added: Cash used in investing activities for the six months ended June 30, 2025 primarily consisted of $245.8 million in the acquisition of strategic investments primarily related to Arrowhead, $75.4 million of purchases of property and equipment and $45.1 million of purchases of investments, partially offset by $109.4 million from the maturity and sale of investments.
Financing Activities
−Removed: Cash provided by financing activities was $0.2 million and $12.5 million for the three months ended March 31, 2026 and 2025, respectively.
−Removed: Cash provided by financing activities for the three months ended March 31, 2026 consisted of $0.2 million in proceeds from exercise of options.
−Removed: During the three months ended March 31, 2026, we also drew down, and subsequently repaid, $250.0 million on our Revolving Credit Facility.
−Removed: Cash provided by financing activities for the three months ended March 31, 2025 consisted of $15.7 million in proceeds from exercise of options and purchase of stock under our Employee Stock Purchase Program, partially offset by the $3.2 million in arrangement and up-front fees related to the Revolving Credit Facility.
+Added: Cash provided by financing activities was $0.2 million for the six months ended June 30, 2026, compared to $11.3 million of cash used in financing activities for the six months ended June 30, 2025.
+Added: Cash provided by financing activities for the six months ended June 30, 2026 consisted of $0.2 million in proceeds from exercise of options.
+Added: During the six months ended June 30, 2026, we also drew down, and subsequently repaid, $425.0 million on our Revolving Credit Facility.
+Added: Cash used in financing activities for the six months ended June 30, 2025 consisted of $25.0 million of purchases of our common stock under our 2024 Repurchase Program and $3.5 million in arrangement, up-front and commitment fees related to the Revolving Credit Facility, partially offset by $17.2 million in proceeds from exercise of options and purchase of stock under our Employee Stock Purchase Program.
Quantitative and Qualitat ive Disclosures about Market Risk
2 unchanged sentences
Our cash is primarily deposited in and invested through highly rated financial institutions in the U.S.
−Removed: As of March 31, 2026, we had $748.3 million of cash, cash equivalents, restricted cash and investments, comprised of $464.5 million of cash and cash equivalents, $270.7 million of investments and $13.1 million non-current restricted cash.
−Removed: The Company only holds debt securities classified as available-for-sale.
+Added: As of June 30, 2026, we had $945.0 million of cash, cash equivalents, restricted cash and investments, comprised of $571.1 million of cash and cash equivalents, $362.7 million of investments and $11.2 million non-current restricted cash and investments.
+Added: We only hold debt securities classified as available-for-sale.
The fair value of cash equivalents and investments is subject to change as a result of fluctuations in market interest rates.
1 unchanged sentence
The potential change in fair value for interest rate sensitive instruments has been assessed on a hypothetical 10 basis point adverse movement across all maturities.
−Removed: As of March 31, 2026, we estimate that such hypothetical adverse 10 basis point movement would result in a hypothetical loss in fair value of approximately $0.2 million to our interest rate sensitive instruments.
+Added: As of June 30, 2026, we estimate that such hypothetical adverse 10 basis point movement would result in a hypothetical loss in fair value of approximately $0.3 million to our interest rate sensitive instruments.
The $158.6 million aggregate principal amount outstanding of our 2027 Notes has a fixed interest rate of 1.25% per annum, payable semi-annually in cash on each March 15 and September 15.
−Removed: The $893.4 million aggregate principal amount outstanding of our
−Removed: 2030 Notes has a fixed interest rate of 4.875% per annum, payable semi-annually in cash on each March 1 and September 1.
+Added: The $893.4 million aggregate principal amount outstanding of our 2030 Notes has a fixed interest rate of 4.875% per annum, payable semi-annually in cash on each March 1 and September 1.
Therefore, no outstanding debt is subject to fluctuations in market interest rates.
5 unchanged sentences
The potential change in fair value for market-price-sensitive instruments has been assessed on a hypothetical 10.0% adverse movement.
−Removed: As of March 31, 2026, we estimate that such hypothetical adverse 10.0% movement would result in a hypothetical loss in fair value of approximately $0.2 million to our market-price-sensitive financial instruments.
+Added: As of June 30, 2026, we estimate that such hypothetical adverse 10.0% movement would result in a hypothetical loss in fair value of approximately $0.2 million to our market-price-sensitive financial instruments.
Compared sentence by sentence after normalising whitespace, quotation marks, case and digits, so re-formatting and restated figures do not read as changed language. Wording changes appear as one removal and one addition. The current filing and the prior one are authoritative.