Sarepta Therapeutics Announces First Quarter 2025 Financial Results and Recent Corporate Developments

• Net product revenues for the first quarter 2025 totaled $611.5 million, a 70% increase over the same quarter of the prior year
• ELEVIDYS net product revenue for the first quarter totaled $375.0 million; Royalty revenue from the sales of ELEVIDYS by Roche for the quarter totaled $4.0 million
• Revised 2025 total net product revenues guidance to $2.3 to $2.6 billion
• Meaningfully advanced multiple clinical candidates in limb-girdle muscular dystrophy portfolio

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the first quarter 2025.

“In the first quarter, we achieved net product revenue of $611.5 million, a 70% increase over the same quarter prior year; our PMO franchise performed well at $236.5 million; and ELEVIDYS achieved $375.0 million, growing at 180% over the same quarter prior year. However, we also faced headwinds in the quarter. While we are taking a variety of actions to address and resolve these challenges, we have adjusted our guidance for 2025 to $2.3 billion to $2.6 billion,” said Doug Ingram, president and chief executive officer, Sarepta Therapeutics. “The broader biotech market has also faced significant pressure in the first quarter of 2025. Fortunately, as an organization with four life-changing therapies, significant revenue, and a deep pipeline, Sarepta is well-positioned to navigate these chaotic times. To that end, in addition to driving the launch of ELEVIDYS and the continued performance of our three approved PMO therapies, we continue to invest in our pipeline and look forward to a significant number of LGMD gene therapy approvals in the coming few years. We are also advancing our siRNA platform and look forward to proof of concept and proof of biology readouts later this year in both our DM1 and FSHD1 programs.”

Corporate Highlights:

• In the first quarter Sarepta closed its global licensing and collaboration agreement with Arrowhead Pharmaceuticals, Inc. (“Arrowhead”). The agreement includes global rights to four clinical-stage and three preclinical-stage programs in muscle, central nervous system, and rare pulmonary disorders, including potential best-in-class siRNA-based treatments for DM1 and FSHD1. The agreement adds meaningfully to Sarepta’s mid- and early-stage pipeline, complementing the Company’s existing leadership in Duchenne muscular dystrophy and limb-girdle muscular dystrophies and gene therapy, while adding new indications and expanding into adjacent therapeutic areas. Later this year the Company plans to share data from the candidates in development for FSHD1 and DM1.
• Pipeline progress for multiple of Sarepta's limb-girdle muscular dystrophy programs:
  • SRP-9005 for LGMD type 2C/R5: Following input from the FDA’s Office of Therapeutic Products (OTP), the Company is cleared to proceed with dosing in Study SRP-9005-101 (COMPASS) in the U.S. COMPASS is Sarepta’s first-in-human clinical study of SRP-9005, an investigational gene therapy for LGMD type 2C/R5, or gamma-sarcoglycanopathy.
  • SRP-9004 for LGMD type 2D/R3: Enrollment and dosing is complete in Study SRP-9004-102 (DISCOVERY). DISCOVERY is a phase 1, proof-of-concept study evaluating safety and expression of the alpha-sarcoglycan protein after treatment with SRP-9004, an investigational gene therapy for the treatment of LGMD type 2D/R3, or alpha-sarcoglycanopathy.
  • SRP-9003 for LGMD type 2E/R4: Enrollment and dosing is complete in Study SRP-9003-301 (EMERGENE). EMERGENE is a phase 3 clinical trial of SRP-9003 (bidridistrogene xeboparvovec) for the treatment of LGMD type 2E/R4, or beta-sarcoglycanopathy. EMERGENE is a global study and the primary endpoint is the biomarker expression of beta-sarcoglycan protein. A pre-Biologics License Application (BLA) meeting has occurred and the OTP has confirmed eligibility for the accelerated approval pathway for the program. Sarepta remains on track to submit a BLA to the U.S. FDA in the second half of 2025.
• 2025 MDA Clinical and Scientific Conference: Sarepta showcased progress across its portfolio of approved and pipeline therapies for Duchenne and limb-girdle muscular dystrophy with 11 abstracts and two oral presentations. The oral presentations included:
  • Results from the 2-year EMBARK and 3-year pooled analyses indicating stabilization or slowing of disease progression compared with well-matched external control assessed by functional outcomes predictive for delaying loss of ambulation.
  • Two-year muscle MRI data, from a subset of patients in EMBARK Part 1, showing stabilization or minimal progression of underlying muscle pathology which remains consistent with functional benefits.

All of the posters and presentations are available on the Company’s website, here.

• Sarepta remains on track to achieve robust revenue growth in 2025 and maintain a strong balance sheet.

Conference Call

The event will be webcast live under the investor relations section of Sarepta's website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form. After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone.

Q1 2025 Financial Highlights¹

Revenues
Total revenues were $744.9 million for the three months ended March 31, 2025, as compared to $413.5 million for the same period of 2024, an increase of $331.4 million. The increase primarily reflects an increase of $241.0 million in net product revenue of ELEVIDYS as a result of its expanded label approval in June 2024. In addition, collaboration revenues increased $64.0 million related to the $112.0 million of collaboration revenue recognized related to F. Hoffman-La Roche Ltd.'s (“Roche”) expiration of an option for a program during the first quarter of 2025, as compared to $48.0 million of collaboration revenue in 2024 related to Roche’s declined option to acquire the ex-US rights to an external, early-stage Duchenne development program. Furthermore, contract manufacturing revenues and royalty revenues increased $11.6 million and $3.8 million, respectively, associated with an increase in commercial ELEVIDYS supply delivered to Roche as well as royalty revenue from sales of ELEVIDYS by Roche, respectively.

Cost of sales (excluding amortization of in-licensed rights)
Cost of sales (excluding amortization of in-license rights) were $137.6 million for the three months ended March 31, 2025, as compared to $50.6 million for the same period of 2024, an increase of $87.0 million. This increase primarily reflects an increase in cost of sales related to ELEVIDYS as we consume previously expensed inventory, an increased demand for ELEVIDYS following the expanded label approval in June 2024, as well as an increase in cost of sales related to products sold to Roche under the Roche collaboration agreement, partially offset by a decrease in the write-offs of certain batches of products not meeting quality specifications for the three months ended March 31, 2025, as compared to the same period of 2024.

Operating expenses and others
Research and development expenses were $773.4 million for the three months ended March 31, 2025, as compared to $200.4 million for the same period of 2024, an increase of $573.0 million. The increase in research and development expenses primarily reflects an increase in up-front and milestone expense associated with the licensing and collaboration agreement and stock purchase agreement with Arrowhead, partially offset by a decrease in clinical trial expenses primarily due to the discontinuation of the PPMO programs in 2024. For the three months ended March 31, 2025, non-GAAP research and development expenses were $749.2 million, as compared to $178.1 million for the same period of 2024, an increase of $571.1 million.

Selling, general and administrative expenses were $133.6 million for the three months ended March 31, 2025, as compared to $127.0 million for the same period of 2024, an increase of $6.6 million. The increase is primarily driven by an increase in compensation and other personnel expenses primarily due to changes in headcount as well as an increase in professional services used for the continuing efforts to commercialize ELEVIDYS. For the three months ended March 31, 2025, non-GAAP selling, general and administrative expenses were $107.1 million, as compared to $100.5 million for the same period of 2024, an increase of $6.6 million.

Other (expense) income, net for the three months ended March 31, 2025 and 2024, was approximately $(83.1) million and $6.5 million, respectively. The change primarily reflects the loss on strategic investments, which includes the recurring fair value adjustments of our investments in publicly-traded companies, including Arrowhead, during the three months ended March 31, 2025.

Income tax expense for the three months ended March 31, 2025 and 2024, was approximately $64.0 million and $5.3 million, respectively. Income tax expense for all periods presented primarily relates to state, federal and foreign income taxes for which available tax losses or credits were not available to offset.

2025 Financial Guidance

Use of Non-GAAP Measures
In addition to the GAAP financial measures set forth in this press release, we have included the following non-GAAP measurements:

1. Non-GAAP net (loss) income is defined by us as GAAP net (loss) income excluding interest income (expense), net, depreciation and amortization expense, stock-based compensation expense, loss (gain) on strategic investments, the estimated income tax impact of each pre-tax non-GAAP adjustment and other items.
2. 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 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 the “if-converted” method, if applicable and not anti-dilutive.
3. Non-GAAP operating (loss) income is defined by us as GAAP operating (loss) income excluding depreciation and amortization expense, stock-based compensation expense and other items.
4. Non-GAAP research and development expenses are defined by us as GAAP research and development expenses excluding depreciation and amortization expense, stock-based compensation expense and other items.
5. Non-GAAP selling, general and administrative expenses are defined by us as GAAP selling, general and administrative expenses excluding depreciation expense, stock-based compensation expense and other items.

The following components are used to adjust our GAAP financial measures into the previously defined non-GAAP measurements:

1. Interest, depreciation and amortization - Interest income (expense), 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.
2. 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.
3. 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 and changes in the fair value of derivatives and may include other items that fit the above characteristics in the future. We exclude from our non-GAAP results:

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 other income and loss adjustments, non-GAAP operating (loss) income, non-GAAP net (loss) income, and non-GAAP diluted (loss) earnings 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. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

EXONDYS 51 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.

Important Safety Information About EXONDYS 51
Hypersensitivity reactions, including bronchospasm, chest pain, cough, tachycardia, and urticaria have occurred in patients who were treated with EXONDYS 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the EXONDYS 51 therapy.

Adverse reactions in DMD patients (N=8) treated with EXONDYS 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (EXONDYS 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of EXONDYS 51 is not recommended.

The most common adverse reactions from observational clinical studies (N=163) seen in greater than 10% of patients were headache, cough, rash, and vomiting.

Other adverse events may occur.

To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For further information, please see the full Prescribing Information.

About VYONDYS 53
VYONDYS 53 (golodirsen) uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 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 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

VYONDYS 53 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 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with VYONDYS 53. Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials.

VYONDYS 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.

Important Safety Information for VYONDYS 53
CONTRAINDICATIONS: VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53.

WARNINGS AND PRECAUTIONS

Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy and monitor until the condition resolves. VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53.

Kidney Toxicity: Kidney toxicity was observed in animals who received golodirsen. Although kidney toxicity was not observed in the clinical studies with VYONDYS 53, the clinical experience with VYONDYS 53 is limited, and kidney toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Kidney function should be monitored in patients taking VYONDYS 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of kidney function in DMD patients. Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider also measuring glomerular filtration rate using an exogenous filtration marker before starting VYONDYS 53. During treatment, monitor urine dipstick every month, and serum cystatin C and urine protein-to-creatinine ratio every three months. Only urine expected to be free of excreted VYONDYS 53 should be used for monitoring of urine protein. Urine obtained on the day of VYONDYS 53 infusion prior to the infusion, or urine obtained at least 48 hours after the most recent infusion, may be used. Alternatively, use a laboratory test that does not use the reagent pyrogallol red, as this reagent has the potential to cross react with any VYONDYS 53 that is excreted in the urine and thus lead to a false positive result for urine protein.

If a persistent increase in serum cystatin C or proteinuria is detected, refer to a pediatric nephrologist for further evaluation.

ADVERSE REACTIONS: Adverse reactions observed in at least 20% of treated patients and greater than placebo were (VYONDYS 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%).

Other adverse reactions that occurred at a frequency greater than 5% of VYONDYS 53-treated patients and at a greater frequency than placebo were: administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture.

Other adverse events may occur.

To report SUSPECTED ADVERSE REACTIONS, contact Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-7

Investor Contact:
Ian Estepan, 617-274-4052
iestepan@sarepta.com

Media Contact:
Tracy Sorrentino, 617-301-8566
tsorrentino@sarepta.com