Upcoming multiple ascending dose data from SRP-1003 in DM1 and SRP-1001 in FSHD expected by year end; Company is actively preparing for Phase 3 development in both programs
Net product revenues for the third quarter 2026 totaled approximately $323.8 million, consisting of approximately $240.3 million of PMO net product revenues and $83.5 million of ELEVIDYS® net product revenue
Continued disciplined financial execution with positive cash flow and GAAP operating loss of ($9.0) million and non-GAAP operating income of $22.9 million for the third quarter 2026
Updates to 2026 guidance range include raising lower end of total net product revenue by $50.0 million, driven by strong PMO performance, and lowering higher end of expense by $25.0 million, reflecting operating discipline
Growing evidence base across ambulatory population reinforces ELEVIDYS benefit-risk profile, with new clinical, biomarker and real-world data supporting durability of benefit, robust dystrophin expression and approaches to liver safety risk mitigation
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the third quarter of 2026.
“After my first 90 days at Sarepta, I believe the fundamental strengths that attracted me to the company remain firmly intact: a durable Duchenne franchise, a promising pipeline with the potential to deliver differentiated medicines, and the financial strength to invest in our highest-value opportunities,” said Michael Severino, M.D., chief executive officer, Sarepta. “This quarter, we saw continued strength in our commercial execution. Our upcoming DM1 and FSHD datasets are important opportunities to demonstrate the depth of knockdown and downstream biological effects we believe our platform can achieve. We are already preparing both programs for potential late-stage development with the intent of moving efficiently if the results support advancement. We have work to do, and I want us to be measured by execution, but I believe Sarepta has the commercial foundation, pipeline opportunities and financial strength to deliver meaningful long-term value.”
Corporate Highlights:
DM1 and FSHD programs advancing toward MAD data in fourth quarter of 2026 and preparing for late-stage development:
Multiple ascending dose data from SRP-1003 and SRP-1001 are expected in the fourth quarter of 2026, providing important insights including DMPK knockdown and splicing correction in DM1 and DUX4 knockdown in FSHD. Sarepta is actively preparing for Phase 3 development in both programs, including beginning manufacturing of clinical supply. The Company is preparing for both accelerated and traditional approval potential pathways, with the ultimate regulatory strategy to be informed by the data and discussions with FDA.
SRP-1003 demonstrates robust nuclear DMPK knockdown in DM1:
New preclinical data presented at the 2026 Oligonucleotide Therapeutics Society Annual Meeting demonstrated that SRP-1003 reduced pathogenic DMPK mRNA in both the nucleus and cytoplasm in a dose-dependent manner, achieving comparable knockdown in each compartment. Nuclear target engagement was supported by three independent methodologies, with correction of MBNL-dependent mis-splicing providing functional evidence that SRP-1003 is degrading disease-causing RNA within the nucleus.
Financial strength supports late-stage DM1 and FSHD execution:
Delivered another quarter of non-GAAP operating profit and positive cash flow, ending with approximately $998.7 million in cash, cash equivalents, restricted cash and investments. Our balance-sheet strength and commercial cash flows are expected to provide the resources to fund these programs through late-stage development while maintaining disciplined capital allocation.
2026 Guidance:
Company expects ELEVIDYS net product revenue in the fourth quarter to trend in line with the third quarter, consistent with previous guidance and subject to normal variability associated with one-time gene therapy. The Company expects the PMO franchise to remain strong and is updating its full-year guidance ranges, raising the low end of total net product revenue guidance by $50.0 million to a range of $1.25 billion to $1.3 billion. The Company also narrows its full-year combined non-GAAP R&D and SG&A expenses, lowering the high end of expense guidance by $25.0 million to a range of $800 million to $825 million.
Growing evidence base reinforces the ELEVIDYS benefit-risk profile:
At the 2026 World Muscle Society Annual Congress, Company presented ELEVIDYS data in ambulatory patients ages 8 to 12 at the time of treatment demonstrating durable and clinically meaningful benefit across the North Star Ambulatory Assessment and timed function measures over two years compared with a well-matched external control group. Company also presented data in patients treated at ages 2 and 3 demonstrating robust ELEVIDYS-dystrophin expression and sarcolemmal localization 12 weeks after infusion. At the Annual Neuromuscular Study Group Scientific Meeting, initial results from the Phase 4 ENDURE study provided preliminary real-world data evaluating approaches to liver safety risk mitigation.
Conference Call
The event will be webcast live under the investor relations section of Sarepta's website at
and following the event a replay will be archived there for one year. This event can be accessed using
this link
.
Q3 2026 Financial Highlights
1
For the Three Months Ended
September 30
2026
2025
QTD Change
(in millions, except for per share amounts)
$
%
Total revenues
$
381.4
$
399.4
$
(18.0
)
(5
)%
Operating (loss) income:
GAAP
$
(9.0
)
$
(103.4
)
$
94.4
(91
)%
Non-GAAP
$
22.9
$
(35.7
)
$
58.6
*
Net (loss) income:
GAAP
2
$
(28.5
)
$
(50.6
)
$
22.1
(44
)%
Non-GAAP
$
15.6
$
(12.9
)
$
28.5
*
Diluted (loss) earnings per share
GAAP
2
$
(0.27
)
$
(0.50
)
$
0.23
(46
)%
Non-GAAP
$
0.13
$
(0.13
)
$
0.26
*
For the Nine Months Ended
September 30
2026
2025
YTD Change
(in millions, except for per share amounts)
$
%
Total revenues
$
1,513.4
$
1,755.3
$
(241.9
)
(14
)%
Operating income (loss):
GAAP
$
362.7
$
(288.2
)
$
650.9
*
Non-GAAP
$
507.1
$
(122.5
)
$
629.6
*
Net income (loss):
GAAP
2
$
297.6
$
(301.2
)
$
598.8
*
Non-GAAP
$
479.6
$
(130.2
)
$
609.8
*
Diluted earnings (loss) per share
GAAP
2
$
2.78
$
(3.06
)
$
5.84
*
Non-GAAP
$
3.91
$
(1.32
)
$
5.23
*
1
For an explanation of our use of non-GAAP financial measures, please refer to the “Use of Non-GAAP Financial Measures” section later in this press release, and for a reconciliation of each non-GAAP financial measure from the most comparable GAAP measures, see the table at the end of this press release.
2
GAAP net loss and diluted loss per share for the three and nine months ended September 30, 2025 have been revised to reflect the correction of an immaterial error associated with the accounting for the August 2025 Exchange, as well as the adoption of ASU 2025-07. Please refer to the Company's Annual Report on Form 10-K for the year ended December 31, 2025 for additional information.
*Not meaningful
As of
September 30, 2026
As of
December 31, 2025
(in millions)
Cash, cash equivalents, restricted cash and investments
$
998.7
$
953.8
Revenues
Total revenues
were $381.4 million for the three months ended September 30, 2026, as compared to $399.4 million for the same period of 2025, a decrease of $18.0 million. This primarily reflects a lower volume of ELEVIDYS sales due to the continued impact of our updated label that only includes the ambulatory patient population for treatment. The decrease is partially offset by an increase of $31.4 million in contract manufacturing revenues associated with increased commercial ELEVIDYS supply delivered to Roche.
Total revenues were $1,513.4 million for the nine months ended September 30, 2026, as compared to $1,755.3 million for the same period of 2025, a decrease of $241.9 million. This primarily reflects a lower volume of ELEVIDYS sales due to our updated label that only includes the ambulatory patient population for treatment. The decrease is partially offset by an increase of $189.5 million in collaboration revenues related to the $365.0 million of collaboration revenue recognized related to Roche's declined option for certain program rights and the milestone recognized under the Roche collaboration agreement for the first commercial dosing of ELEVIDYS in Japan during the nine months ended September 30, 2026, as compared to $175.5 million of collaboration revenue in 2025 related to Roche’s expiration of an option to acquire a certain program and the Japan Approval Milestone. Furthermore, contract manufacturing revenues increased $72.5 million associated with increased commercial ELEVIDYS supply delivered to Roche.
Cost of sales (excluding amortization of in-licensed rights)
Cost of sales (excluding amortization of in-license rights)
were $188.5 million for the three months ended September 30, 2026, as compared to $150.8 million for the same period of 2025, an increase of $37.7 million. Cost of sales (excluding amortization of in-license rights) were $446.7 million for the nine months ended September 30, 2026, as compared to $440.9 million for the same period of 2025, an increase of $5.8 million. The increases in both periods primarily reflect a take-or-pay charge of $62.0 million related to non-cancelable manufacturing obligations with Catalent, Inc. ("Catalent") for certain inventories that we elected not to manufacture. Additionally, there was an increase in cost of sales related to products sold to Roche, primarily related to increased volume of ELEVIDYS shipments as well as write-offs of certain batches of products not meeting quality specifications under the Roche collaboration agreement. The increases in both periods were partially offset by a lower volume of ELEVIDYS sales in the United States and corresponding royalty payments.
Operating expenses and others
Research and development expenses
were $82.5 million
for the three months ended September 30, 2026, as compared to $218.9 million for the same period of 2025, a decrease of $136.4 million. The decrease primarily reflects a $100.0 million milestone payment made to Arrowhead Pharmaceutical, Inc. ("Arrowhead") during the three months ended September 30, 2025, triggered by Arrowhead's achievement of the first of two predetermined enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 study for the DM1 program, with no similar activity during the same period of 2026. In addition, there were decreases in manufacturing and clinical expenses primarily due to our decision to reprioritize our pipeline and developmental priorities announced in July 2025, partially offset by an increase in compensation and other personnel expenses due to the reversal of previously recognized expense related to certain incentive compensation accruals in connection with our restructuring plan announced in July 2025 (the "Restructuring"). For the three months ended September 30, 2026, non-GAAP research and development expenses were $71.6 million, as compared to $206.5 million for the same period of 2025, a decrease of $134.9 million.
Research and development expenses were $327.8 million for the nine months ended September 30, 2026, as compared to $1,196.7 million for the same period of 2025, a decrease of approximately $868.9 million. The decrease primarily reflects the recognition of up-front and collaboration license fees of $583.6 million associated with the licensing, collaboration and stock purchase agreement with Arrowhead as well as the $100.0 million milestone payment made to Arrowhead during the nine months ended September 30, 2025, with no similar activity for the same period of 2026. In addition, there was a decrease in manufacturing and clinical expenses primarily due to our decision to reprioritize our pipeline and developmental priorities announced in July 2025, as well as a decrease in compensation, other personnel, and stock-based compensation expenses, all as a result of the Restructuring. This decrease was partially offset by the $50.0 million annual collaboration license fee incurred and paid to Arrowhead during the nine months ended September 30, 2026. For the nine months ended September 30, 2026, non-GAAP research and development expenses were $285.8 million, as compared to $1,137.4 million for the same period of 2025, a decrease of $851.6 million.
Selling, general and administrative expenses
were $118.6 million for the three months ended September 30, 2026, as compared to $91.9 million for the same period of 2025, an increase of $26.7 million. The increase primarily reflects the reversal of previously recognized expense related to certain stock compensation awards and incentive compensation accruals in connection with the Restructuring during the three months ended September 30, 2025, with no similar activity during the same period of 2026. For the three months ended September 30, 2026, non-GAAP selling, general and administrative expenses were $97.6 million, as compared to $77.1 million for the same period of 2025, an increase of $20.5 million.
Selling, general and administrative expenses were $335.2 million for the nine months ended September 30, 2026, as compared to $363.4 million for the same period of 2025, a decrease of $28.2 million. The decrease primarily reflects a decrease in compensation, other personnel, and stock-based compensation expenses, all as a result of the Restructuring, as well as a decrease in professional services used related to ELEVIDYS commercialization efforts. For the nine months ended September 30, 2026, non-GAAP selling, general and administrative expenses were $271.7 million, as compared to $297.6 million for the same period of 2025, a decrease of $25.9 million.
Litigation settlement charge
was $39.0 million for the nine months ended September 30, 2026, with no similar activity for the same period of 2025. We recorded a litigation settlement charge of $39.0 million in the second quarter of 2026 related to a settlement agreement that was executed in September 2026 between Sarepta Therapeutics, Inc., Sarepta Therapeutics Three, LLC, REGENXBIO, INC, the Trustees of the University of Pennsylvania and Catalent regarding the resolution of certain patent infringement litigations.
Restructuring charges
were $40.5 million for the three and nine months ended September 30, 2025, with no similar activity for the same periods of 2026.
Other (expense) income, net
for the three months ended September 30, 2026 and 2025 was approximately $(16.7) million and $35.0 million, respectively. Other expense, net for the nine months ended September 30, 2026 and 2025 was approximately $47.0 million and $10.1 million, respectively. The change primarily reflects a decrease in our strategic investments as a result of the sale of our investment in Arrowhead in August 2025, partially offset by an increase in interest expense due to our 2030 Notes carrying a higher interest rate than our 2027 Notes during the three and nine months ended September 30, 2026.
Gain on debt extinguishment
was $3.8 million for the three and nine months ended September 30, 2025, with no similar activity for the same periods of 2026. This gain was the result of a partial refinancing of the 2027 Notes where we exchanged $700.0 million in aggregate principal amount of 2027 Notes for $602.0 million in aggregate principal amount of 2030 Notes and made cash payments of $127.3 million, including $4.0 million of accrued interest of the 2027 Notes and 5.9 million shares of our common stock with a fair market value of approximately $104.9 million, net of issuance costs of $2.4 million.
Income tax expense
for the three and nine months ended September 30, 2026, was approximately $2.7 million and $18.1 million, respectively. Income tax (benefit) expense for the three and nine months ended September 30, 2025, was $(14.1) million and $6.6 million, respectively. Income tax expense for all periods presented primarily relates to state income taxes as a result of taxable profits in certain states requiring the capitalization of research and development costs and states which have suspended or limited the utilization of net operating loss carryforwards.
Use of Non-GAAP Financial Measures
In addition to the GAAP financial measures set forth in this press release, we have included the following non-GAAP measurements:
Non-GAAP net (loss) income is defined by us as GAAP net (loss) income excluding interest expense/income, net, depreciation and amortization expense, stock-based compensation expense, restructuring charges, other items, and the estimated income tax impact of each pre-tax non-GAAP adjustment.
Non-GAAP earnings per share is defined by us as non-GAAP net income, as defined previously, divided by the weighted-average number of shares of common stock and dilutive common stock equivalents outstanding, adjusted for the inclusion of additional shares under both the treasury stock method and the “if-converted” method, if applicable and not anti-dilutive. Non-GAAP net loss per share is defined by us as non-GAAP net loss, as defined above, divided by the weighted-average number of shares of common stock outstanding as the inclusion of dilutive common stock equivalents outstanding is anti-dilutive.
Non-GAAP operating income (loss) is defined by us as GAAP operating (loss) income excluding depreciation and amortization expense, stock-based compensation expense, restructuring charges and litigation settlement charges.
Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense and stock-based compensation expense.
Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense and stock-based compensation expense.
Non-GAAP effective tax rate is defined by us as the GAAP effective tax rate excluding the impact of our GAAP to non-GAAP adjustments.
The following components are used to adjust our GAAP financial measures into the previously defined non-GAAP measurements:
Interest, depreciation and amortization - Interest expense/income, net amounts can vary substantially from period to period due to changes in cash and debt balances and interest rates driven by market conditions outside of our operations. Depreciation expense can vary substantially from period to period as the purchases of property and equipment may vary significantly from period to period and without any direct correlation to our operating performance. Amortization expense primarily associated with patent costs are amortized over a period of several years after acquisition or patent application or renewal.
Stock-based compensation expenses - Stock-based compensation expenses represent non-cash charges related to equity awards we have granted. Although these are recurring charges to operations, we believe the measurement of these amounts can vary substantially from period to period and depend significantly on factors that are not a direct consequence of operating performance that is within our control. Therefore, we believe that excluding these charges facilitates comparisons of our operational performance in different periods.
Restructuring charges do not have a direct correlation to future business operations, nor do the resulting charges recorded reflect the performance of our ongoing operations for the period in which such charges are recorded. In addition, restructuring charges are not considered to be normal operating expenses due to the variability of amounts and lack of predictability as to occurrence and/or timing.
Other items - We evaluate other items of expense and income on an individual basis. We take into consideration quantitative and qualitative characteristics of each item, including (a) nature, (b) whether the items relate to our ongoing business operations, and (c) whether we expect the items to continue or occur on a regular basis. These other items include the loss (gain) on strategic investments, the impairment of strategic investments, litigation settlement charges, and gain on debt extinguishment and may include other items that fit the above characteristics in the future. We exclude from our non-GAAP results:
The loss (gain) on strategic investments as the results of such gains and losses are not representative of our normal business operations, which accordingly would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures.
The impairment of strategic investments as such charges are not indicative of the performance of our core operations, which accordingly would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures.
Litigation settlement charge as such charges are considered to be an infrequent event as it is associated with a distinct, non-recurring litigation matter and is not indicative of the performance of our core operations nor representative of our normal business operations, which accordingly would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures.
The gain on debt extinguishment is considered to be an infrequent event as it is associated with a distinct financing decision and is not indicative of the performance of our core operations, which accordingly would make it difficult to compare our results to peer companies that also provide non-GAAP disclosures.
We use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. We also believe these non-GAAP measures increase comparability of period-to-period results and are useful to investors as they provide a similar basis for evaluating our performance as is applied by management. These non-GAAP measures are not intended to be considered in isolation or to replace the presentation of our financial results in accordance with GAAP. Use of the terms non-GAAP research and development expenses, non-GAAP selling, general and administrative expenses, non-GAAP operating income (loss), non-GAAP net income (loss), and non-GAAP diluted earnings (loss) per share may differ from similar measures reported by other companies, which may limit comparability, and are not based on any comprehensive set of accounting rules or principles. All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”
About EXONDYS 51
EXONDYS 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion, or “skipping”, of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.
EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the
DMD
gene that is amenable to exon 51 skipping. This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51.
Contacts
Investor Contacts:
Ian Estepan, 617-274-4052,
iestepan@sarepta.com
Ryan Wong, 617-800-4112,
rwong@sarepta.com
Tam Thornton, 617-803-3825,
tthornton@sarepta.com
Media Contacts:
Tracy Sorrentino, 617-301-8566,
tsorrentino@sarepta.com
Kara Hoeger, 617-710-3898,
khoeger@sarepta.com
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