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Sarepta Therapeutics, Inc. (SRPT): Business Model Canvas [Dec-2025 Updated] |
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You're looking past the noise to understand how Sarepta Therapeutics, Inc. actually makes money after the recent Elevidys label shifts and major restructuring. Honestly, this is a high-wire act: they are projecting between $2.3 billion and $2.6 billion in net product revenue for 2025 while simultaneously budgeting for operating expenses that could hit $2.18 billion. To see the full architecture behind their proprietary gene engine, their key partnerships like the one with Roche, and how they manage four on-market DMD therapies, check out the nine building blocks detailed below.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Key Partnerships
You're looking at the structure of Sarepta Therapeutics, Inc.'s external relationships as of late 2025, which is crucial for understanding how they scale complex therapies like gene and RNA medicines. These partnerships are where significant upfront cash and future obligations live.
F. Hoffmann-La Roche Ltd. for global Elevidys commercialization and royalties
The relationship with F. Hoffmann-La Roche Ltd. (Roche) is directly tied to the commercial performance and geographic expansion of ELEVIDYS. Sarepta Therapeutics, Inc. recognized specific revenue events from this collaboration in 2025.
For the first nine months of 2025, collaboration revenue recognized related to Roche's expiration of an option for a program totaled approximately $112.0 million.
In the second quarter of 2025, collaboration and other revenues increased by $95.6 million, which included a $63.5 million milestone payment from Roche for the regulatory approval of ELEVIDYS in Japan (the Japan Approval Milestone).
Financial impact from ELEVIDYS supply and royalties with Roche in Q2 2025 included:
- Contract manufacturing revenues increased by $27.0 million.
- Royalty revenues increased by $5.1 million.
For the third quarter of 2025 compared to the third quarter of 2024, contract manufacturing revenues and royalty revenues increased by $29.4 million and $9.9 million, respectively, due to increased commercial ELEVIDYS supply delivered to Roche and associated royalty revenue.
Arrowhead Pharmaceuticals for siRNA platform expansion (DM1, FSHD programs)
The Exclusive License and Collaboration Agreement with Arrowhead Pharmaceuticals closed in February 2025, bringing several siRNA programs, including those for DM1 and FSHD1, under Sarepta Therapeutics, Inc.'s purview.
The initial financial terms upon closing were substantial:
| Payment Component | Amount |
| Upfront Payment (Cash) | $500 million |
| Stock Purchase by Sarepta | $325 million |
| Stock Price per Share | $27.25 |
| Future Installment Payments (Total) | $250 million |
| Annual Installment Amount | $50 million over 5 years |
Sarepta Therapeutics, Inc. is also on track to pay near-term payments associated with ARO-DM1 enrollment, totaling a potential $300 million. Arrowhead believes it is on track to earn the first $100 million soon and the remaining $200 million by the end of 2025.
The potential economics for Arrowhead Pharmaceuticals are significant:
- Development milestone payments per program: between $110 million and $410 million.
- Sales milestone payments per program: between $500 million and $700 million.
- Tiered royalties on commercial sales: up to the low double digits.
- Total potential milestones and royalties: a further $10 billion.
To manage its balance sheet amid ELEVIDYS uncertainty, Sarepta Therapeutics, Inc. planned to sell over 9.2 million Arrowhead shares for $174 million in cash and transfer 2.6 million shares back to Arrowhead to help satisfy $100 million in milestone obligations.
Academic and research institutions (e.g., Nationwide Children's Hospital) for R&D
Sarepta Therapeutics, Inc. maintains foundational research relationships to fuel its pipeline, such as the one with Nationwide Children's Hospital.
The agreement with Nationwide Children's Hospital grants Sarepta Therapeutics, Inc. the exclusive option for the calpain 3 (CAPN-3) gene therapy candidate targeting Limb-girdle muscular dystrophy type 2A (LGMD2A). Earlier support for the microdystrophin gene therapy program involved a commitment of $2.2 million from Parent Project Muscular Dystrophy (PPMD) to the trial, with Sarepta providing separate research support.
Dyno Therapeutics for next-generation AAV gene therapy delivery vehicles
The collaboration with Dyno Therapeutics focuses on designing next-generation Adeno-Associated Virus (AAV) vectors. The initial partnership with Sarepta Therapeutics, Inc. began in 2020.
The potential value of the initial Sarepta-Dyno deal was estimated to be over $40 million in upfront, option, and license payments during the research phase, with Dyno eligible for royalties and additional significant future milestones. Together, the initial collaborations with Novartis, Roche, and Sarepta Therapeutics, Inc. could earn Dyno Therapeutics up to $2 billion in partnership revenue.
Specific financial details from the Dyno ecosystem include:
| Partner/Program | Upfront Payment to Dyno | Total Potential Value |
| Sarepta (Muscle Diseases, Initial) | $18 million | Potentially exceeding $1.6 billion |
| Roche (CNS Partnership Expansion, Oct 2024) | US$50 million | Not specified for this tranche |
Roche exercised a license using a Dyno capsid in early 2025, successfully completing the initial partnership tranche.
Specialized contract manufacturing organizations for complex biologics
Sarepta Therapeutics, Inc. relies on specialized Contract Development and Manufacturing Organizations (CDMOs) to produce its complex biologics, such as ELEVIDYS.
Sarepta Therapeutics, Inc. has partnered with the CDMO Catalent for ELEVIDYS manufacturing, which was recently acquired by Novo Holdings.
As of June 30, 2025, Sarepta Therapeutics, Inc. had total noncancelable contract manufacturing payment commitments of nearly $1.1 billion through 2028. The breakdown of these near-term commitments included:
- $626 million for the second half of 2025.
- Approximately $320 million for 2026.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Key Activities
Research and development (R&D) of gene and RNA-targeted therapies involves significant capital outlay. For the nine months ended September 30, 2025, Sarepta Therapeutics, Inc. reported research and development expenses of $1,196.7 million, up from $604.6 million for the same period in 2024. Non-GAAP R&D expenses for the three months ended September 30, 2025, were $206.5 million.
Sarepta Therapeutics, Inc. is advancing its pipeline candidates through clinical trials. The Phase 3 EMERGENE study for SRP-9003 (bidridistrogene xeboparvovec) for Limb-Girdle Muscular Dystrophy Type 2E (LGMD2E/R4) met its primary endpoint, which was the biomarker expression of $\beta$-sarcoglycan protein.
| SRP-9003 EMERGENE Trial Endpoint Data (Day 60 CFBL) | Patient Cohort | N | Mean $\beta$-SG Expression Change (PPF) |
| Primary Endpoint Met | Ambulatory Patients | 11 | 43.4% |
| Secondary Endpoint Analysis | Non-Ambulatory Patients | 6 | 23.9% |
The company is on track to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for SRP-9003 in the second half of 2025. However, the FDA revoked the platform technology designation for the rAAVrh74 viral vector in July 2025.
Manufacturing and supply chain management is critical for the four on-market Duchenne muscular dystrophy (DMD) therapies. Cost of Sales for the third quarter of 2025 totaled $151 million, or $150.8 million excluding amortization. Following a strategic restructuring announced July 16, 2025, Sarepta Therapeutics, Inc. is evaluating opportunities to enhance operational efficiency and adjust manufacturing commitments based on latest demand.
Commercialization and distribution of the four on-market DMD therapies generated significant revenue. Net product revenues for the third quarter of 2025 totaled $370.0 million. This revenue was split between the PMO (exon-skipping) therapies and the gene therapy, ELEVIDYS.
- Net Product Revenue from PMO therapies (Q3 2025): $238.5 million
- Net Product Revenue from ELEVIDYS (Q3 2025): $131.5 million
- Full Year 2025 Total Net Product Revenue Guidance (as of May 6, 2025): $2.3 billion to $2.6 billion
- Expected Annual Revenue from ELEVIDYS in Ambulant Population (FY 2025): At least $500 million
- Expected 2025 Revenue from Three PMO Therapies: Around $900 million
- Cumulative patients treated worldwide with exon-skipping therapies: Over 1,800
Navigating complex global regulatory approvals involves managing label updates and trial requirements. Sarepta Therapeutics, Inc. is progressing with ELEVIDYS labeling discussions, which are expected to conclude soon, following the addition of a black box warning for acute liver injury/acute liver failure (ALI/ALF). The company is working with the FDA on a risk-mitigation approach to potentially bring ELEVIDYS back to the non-ambulatory community.
The company initiated a strategic restructuring on July 16, 2025, which includes laying off 500 staffers, or 36% of its workforce, aiming for annual cash cost savings of $400 million starting in 2026. The company anticipates realizing $100 million in cost savings by the end of 2025, after accounting for severance costs between $32 and $37 million. The Q3 2025 restructuring charge was $41 million or $40.5 million. The full year guidance for combined non-GAAP R&D and SG&A expenses is approximately $1.86 billion.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Key Resources
You're looking at the core assets Sarepta Therapeutics, Inc. (SRPT) relies on to execute its strategy in the rare disease space as of late 2025. These aren't just line items; they are the engines driving their precision genetic medicine approach.
The foundation is the Proprietary Precision Genetic Medicine Engine. This engine powers their development across multiple modalities, including gene therapy, RNA-targeted therapies, and gene editing. As of early 2025, this platform supported a pipeline of more than 40 programs in various stages of development, targeting Duchenne muscular dystrophy (DMD), limb-girdle muscular dystrophies (LGMDs), and expanding into central nervous system and cardiac diseases.
Intellectual Property (IP) is critical, protecting the core technologies behind their approved products. This includes the chemistry for their PMO (exon-skipping) therapies and the AAV-based vectors for their gene therapy. However, it's important to note a recent regulatory shift: the FDA revoked the platform technology designation for Sarepta Therapeutics' AAVrh74 Platform Technology in July 2025, citing new safety information.
The most tangible resources are the four FDA-approved DMD products. These represent years of R&D and regulatory navigation. You need to track the performance and status of each one:
- Elevidys (delandistrogene moxeparvovec-rokl): AAV-based gene transfer therapy.
- Exondys 51 (eteplirsen): PMO exon-skipping therapy.
- Vyondys 53 (golodirsen): PMO exon-skipping therapy.
- Amondys 45 (casimersen): PMO exon-skipping therapy.
The commercial portfolio is generating significant revenue, but the status of the gene therapy is dynamic. For instance, following regulatory discussions, Sarepta Therapeutics resumed shipments of Elevidys for ambulatory individuals in late July 2025, while continuing discussions for the non-ambulatory population. Furthermore, two of the PMO drugs, Vyondys 53 and Amondys 45, are currently operating under accelerated approval, with the company planning discussions with the FDA for traditional approval based on real-world evidence.
Financially, the balance sheet provides operational runway. As of June 30, 2025, Sarepta Therapeutics held approximately $850.3 million in cash, cash equivalents, and investments. The second quarter of 2025 was cash flow positive, with cash increasing by $202.8 million sequentially.
Here's a quick look mapping the key products to their financial context from Q2 2025:
| Key Product/Metric | Value (Q2 2025) | Context/Note |
| Total Revenues | $611.09 million | Up 68.4% year-over-year |
| Net Product Revenues | $513.12 million | Represents a +42.3% change year-over-year |
| Elevidys Net Product Revenue | $281.85 million | Year-over-year change of +131.6% |
| PMO Products Revenue | $231.27 million | From Exondys 51, Vyondys 53, and Amondys 45 |
| Collaboration Revenue (incl. Roche Milestone) | $97.97 million | Aided by a $63.5 million milestone payment from Roche |
The highly specialized scientific and regulatory talent is a core intangible asset, though it has recently undergone significant change. To secure long-term financial viability, Sarepta Therapeutics initiated a restructuring plan, which included a workforce reduction of approximately 500 employees, representing a 36% reduction in staff. This action is aimed at realizing over $100 million in cost savings through the end of 2025 and achieving approximately $400 million in anticipated annual cost savings starting in 2026. The executive team, led by CEO Douglas Ingram, maintains deep expertise in genetic engineering and regulatory affairs to manage these complex assets.
Finance: draft 13-week cash view by Friday.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Value Propositions
You're focused on delivering transformative options where few, if any, existed before. For Sarepta Therapeutics, Inc., the value proposition centers on being the leader in precision genetic medicine for ultra-rare neuromuscular diseases, primarily Duchenne Muscular Dystrophy (DMD).
Life-saving/altering treatments for ultra-rare neuromuscular diseases.
Sarepta Therapeutics, Inc. offers treatments designed to address the underlying genetic cause of these conditions. The company's commitment to a patient-first approach ensures the patient voice is integrated from early development through to access, as seen with their Patient Affairs team engagement and the Route 79, The Duchenne Scholarship Program, which supports post-secondary educational goals for students living with Duchenne.
Elevidys: First and only FDA-approved gene therapy for Duchenne Muscular Dystrophy (DMD).
Elevidys (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy. It is designed to code for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle. In late November 2025, the FDA approved dosing non-ambulant DMD patients with ELEVIDYS in a new ENDEAVOR study cohort, expanding its reach. The company maintained an annual ELEVIDYS revenue floor projection of $500 million.
Exon-skipping PMO therapies for specific DMD mutations (e.g., Exon 51, 53, 45).
The Phosphorodiamidate Morpholino Oligomer (PMO) therapies use exon-skipping technology to treat DMD patients with confirmed mutations amenable to specific exon skipping. For more than a decade, these therapies have treated over 1,800 amenable patients worldwide, including infants as young as 7 months to adults well into their 30s. The ESSENCE study evaluated AMONDYS 45 and VYONDYS 53 in 225 patients, ages 6-13 years old.
Here's the quick math on the PMO and Gene Therapy product revenue performance for the third quarter of 2025:
| Product Category | Q3 2025 Net Product Revenue |
| PMO Therapies (EXONDYS 51, VYONDYS 53, AMONDYS 45) | $238.5 million |
| ELEVIDYS | $131.5 million |
| Total Net Product Revenue | $370.0 million |
The total revenue for the nine months ended September 30, 2025, reached $1,755.3 million.
Multi-platform approach to address diverse genetic disorders (RNA and Gene Therapy).
Sarepta Therapeutics, Inc. is expanding beyond DMD with its RNA platform. The company is advancing candidates for Limb-Girdle Muscular Dystrophy (LGMD) and other genetic disorders. For instance, the investigational siRNA therapy for type 1 myotonic dystrophy (SRP-1003) is in a Phase 1/2 study, where Cohorts 1 and 2 in the SAD arm are fully enrolled. The company also plans to share data from candidates in development for FSHD1 and DM1 later in 2025.
Patient support programs to facilitate access and treatment initiation.
The company actively works to remove barriers to treatment. To address backlogs and improve overall patient access, Sarepta Therapeutics, Inc. plans to intensify support for secondary infusion centers. Educational outreach efforts are comprehensive, targeting both physicians and patient families to address safety and efficacy data, which management views as critical to restoring confidence and driving new treatment initiations.
Key patient engagement activities include:
- Educational resources through advocacy and educational programs.
- Direct conversation and engagement with advocates.
- Community Letters addressing label updates and safety.
- The Route 79 Scholarship Program for students with Duchenne.
If onboarding takes 14+ days, churn risk rises, so this focus on access is defintely important.
Finance: draft 13-week cash view by Friday.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Customer Relationships
You're managing patient access in a rare disease space, where every interaction is critical; Sarepta Therapeutics, Inc. knows this, which is why their customer relationship model is built around intensive, specialized support for a small, high-need population.
High-touch, specialized support is channeled through a dedicated Patient Affairs team, which includes roles like Senior Vice President, Patient Affairs, Wendy Erler. This team integrates the patient voice from the earliest drug development stages right through to market access. They maintain direct lines of communication, evidenced by issuing multiple Community Letters in late 2025, such as the one on November 16, 2025, answering questions about the label update for their FDA-approved gene therapy.
Direct educational outreach is crucial, especially given the evolving safety profiles of their therapies. For instance, a Community Letter was issued on June 15th, 2025, specifically addressing the safety update regarding ELEVIDYS in non-ambulatory individuals. This ongoing dialogue extends to physicians, as Sarepta Therapeutics presented new data on its portfolio, including real-world evidence on pulmonary function for casimersen-treated patients, at the 2025 World Muscle Society Congress in October 2025.
Collaboration with patient advocacy groups remains fundamental, supported by a structured Grants & Giving program. Sarepta Therapeutics prioritizes support for Duchenne muscular dystrophy and limb-girdle muscular dystrophy communities through grants, donations, and sponsorships. They have a defined review process, stating that grant applications are generally reviewed monthly, with a commitment to contact the requester within 90 days of submission. For 2026 Medical Education Grants, the application window was set from July 15, 2025, to November 15, 2025.
The company's commitment to patient support is formalized through programs like SareptAlly, a global initiative to help patients, families, and physicians identify and match with Sarepta clinical trials or assess potential treatment options. For US residents eligible for an approved product, the SareptAssist patient support program assigns a dedicated case manager to navigate insurance benefits, financial assistance options, and treatment logistics, including options for weekly infusions.
The field-based medical and commercial teams manage the complex sales and patient onboarding process, which saw significant recent turbulence. The scale of commercial activity, which directly correlates with the number of patients requiring support, is reflected in the Q3 2025 net product revenues of $370.0 million. However, the structure supporting these relationships underwent a major shift in mid-2025; Sarepta Therapeutics announced a strategic restructuring in July 2025, which included laying off 500 staffers, representing 36% of the workforce, to achieve $400 million in annual cost savings starting in 2026. This definitely impacts the field force deployment and support capacity.
Regarding managed access for investigational therapies, Sarepta Therapeutics has been cautious. While they support a limited managed access program for eteplirsen & golodirsen in certain jurisdictions where those products aren't approved, they explicitly stated in 2025 that they are currently unable to offer compassionate use or pre-approval access for any of their investigational exon skipping or gene therapies without jeopardizing clinical development. This means reimbursement hurdles for new treatments are managed primarily through the established commercial benefit verification and financial assistance pathways of SareptAssist.
Here's a quick look at the operational scale impacting these customer relationships as of late 2025:
| Metric | Value (Latest Reported Data) | Context |
|---|---|---|
| Q3 2025 Net Product Revenue | $370.0 million | Overall commercial scale supporting patient base |
| ELEVIDYS Q3 2025 Net Product Revenue | $131.5 million | Volume of gene therapy patients requiring infusion/post-infusion support |
| PMO Therapies Q3 2025 Net Product Revenue | $238.5 million | Volume of exon-skipping patients requiring ongoing supply/support |
| Workforce Reduction (July 2025) | 500 staffers (36% of workforce) | Indicates restructuring of field/support teams |
| Annual Cost Savings Target (from 2026) | $400 million | Financial discipline impacting operational support structure |
The Route 79, The Duchenne Scholarship Program, named after the 79 exons in the dystrophin gene, is a specific, high-touch initiative supporting the post-secondary educational goals of students living with Duchenne.
Finance: draft 13-week cash view by Friday.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Channels
You're looking at how Sarepta Therapeutics, Inc. gets its specialized, high-value therapies, like ELEVIDYS, into the hands of the patients who need them. For a company focused on rare genetic diseases, the channel strategy is all about precision and specialized access, not mass market reach.
Direct sales force targeting specialized US neuromuscular treatment centers
Sarepta Therapeutics, Inc. relies on a dedicated internal team to manage relationships with the few, highly specialized centers capable of administering its gene therapies. This direct model ensures the complexity of the treatment-including infusion protocols and post-treatment monitoring-is handled by experts. As of late 2025, the company had a total workforce of 1,372 employees. However, following a strategic restructuring in July 2025, Sarepta Therapeutics laid off 500 staffers, which is 36% of its workforce. This action definitely impacts the size and structure of the commercial team supporting the US launch and ongoing management of its therapies.
The channel is characterized by high-touch service, which is necessary given the nature of the product. For instance, following the FDA recommendation to resume shipments for ambulatory individuals on July 28, 2025, the direct sales force and support teams were critical in quickly restarting patient infusions.
Specialized medical centers and secondary infusion sites for administration
The actual delivery of the therapy is restricted to a select network. This channel is defined by the capability to handle complex, often one-time, intravenous infusions for rare neuromuscular disorders. These sites must be equipped for the specialized care required, especially considering the safety labeling updates, such as the black box warning for acute liver injury/acute liver failure associated with ELEVIDYS.
The revenue generated through this channel shows the volume of product moving through these centers:
| Metric | Value (Q1 2025) | Value (Q2 2025) |
| ELEVIDYS Net Product Revenue | $375.0 million | $513.1 million (Total Net Product Revenue) |
| Total Net Product Revenue | $611.5 million | $513.1 million |
The company's revised 2025 total net product revenue guidance sits between $2.3 billion and $2.6 billion.
Selective international partnership agreements for ex-US distribution (e.g., Roche)
Ex-US distribution is primarily managed through a significant licensing agreement with Roche, established in 2019. This partnership leverages Roche's global footprint. The financial structure of this channel includes direct payments to Sarepta Therapeutics, Inc. based on ex-US performance.
- Upfront payment from Roche (2019): $1.15 billion (cash and equity).
- Potential Regulatory and Sales Milestones from Roche: Up to $1.7 billion.
- Royalty Rate on Net Sales: Anticipated to be in the mid-teens percentage.
Recent activity shows this channel is active, though subject to regulatory alignment. For the second quarter of 2025, Sarepta Therapeutics, Inc. recognized $63.5 million in collaboration revenue from a milestone payment from Roche related to the regulatory approval of ELEVIDYS in Japan. Royalty revenue from Roche sales was also recorded in Q2 2025. However, in July 2025, Roche voluntarily paused shipments in certain countries whose approvals referenced the FDA decision.
Digital patient engagement platforms and telehealth consultation services
While specific usage metrics aren't public, this channel supports the high-value product delivery by providing necessary patient and physician support digitally. This is crucial for managing complex therapies remotely, especially for patients in geographically dispersed areas or those needing ongoing guidance post-infusion. This digital layer helps maintain the connection with the patient population outside of the specialized treatment center visits.
The company's focus on its siRNA platform, with expected readouts later in 2025, suggests that digital tools will be increasingly important for managing trials and patient enrollment across these newer programs, too.
Finance: draft 13-week cash view by Friday.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Customer Segments
You're looking at the core groups Sarepta Therapeutics, Inc. (SRPT) serves with its precision genetic medicines. This is a highly specialized market, focused on rare neuromuscular diseases where the unmet need is significant.
Ambulant Duchenne Muscular Dystrophy (DMD) patients with confirmed mutations.
This segment is served by the approved exon-skipping therapies (PMOs) and the gene therapy ELEVIDYS (delandistrogene moxeparvovec-rokl). The PMO franchise, including AMONDYS 45 and VYONDYS 53, has treated over 1,800 amenable patients worldwide across more than a decade. For the first quarter of 2025, this PMO franchise generated net product revenue of $236.5 million. ELEVIDYS, which is indicated for ambulatory patients aged 4 and up with a confirmed DMD mutation, generated net product revenue of $375.0 million in the first quarter of 2025 alone. Following an expanded label in June 2024, the potential U.S. market for ELEVIDYS opened up to approximately ~13,000 DMD patients, representing about 90% of total prevalence. The per-patient price tag for ELEVIDYS is $3.2 million. By the third quarter of 2025, net product revenues for the PMO therapies were $238.5 million, while ELEVIDYS contributed $131.5 million for that quarter.
The customer base for DMD patients is segmented further based on the specific mutation amenable to the therapy:
- Patients amenable to exon 51 skipping (treated with EXONDYS 51).
- Patients amenable to exon 45 skipping (treated with AMONDYS 45).
- Patients amenable to exon 53 skipping (treated with VYONDYS 53).
- Patients eligible for gene therapy based on micro-dystrophin expression (treated with ELEVIDYS).
Physicians and specialized neuromuscular disease treatment centers.
These centers are the gatekeepers for diagnosis, prescription, and administration of the therapies. They manage the patient journey, from diagnosis to ongoing monitoring, especially for gene therapies like ELEVIDYS which require monitoring for potential adverse events like acute serious liver injury. These centers are also the sites for Sarepta Therapeutics, Inc.'s pipeline trials, such as the ongoing Phase 1/2 study for Type 1 Myotonic Dystrophy (DM1) where enrollment for the multiple ascending dose (MAD) cohort 4 is underway.
Global regulatory bodies and national health systems for market access.
Regulatory bodies, primarily the U.S. Food and Drug Administration (FDA), dictate market access and patient eligibility. The FDA granted ELEVIDYS an expanded label in June 2024. However, the segment faced regulatory scrutiny, with the FDA requesting a pause on shipments for non-ambulatory patients in June 2025, though shipments for ambulatory patients resumed on July 28, 2025. In late November 2025, the FDA approved dosing of non-ambulant DMD patients in a new ENDEAVOR study cohort (Cohort 8) to evaluate an enhanced immunosuppressive regimen. National health systems and payers determine reimbursement, which is critical given ELEVIDYS' $3.2 million price tag. The company is working to meet statutory standards for traditional approval for its PMO therapies following the ESSENCE study results.
Patients with other rare neuromuscular diseases (LGMD, DM1) in clinical trials.
Sarepta Therapeutics, Inc. is actively developing treatments for other rare conditions, making these patient groups key future customer segments. The company has several Limb-Girdle Muscular Dystrophy (LGMD) gene therapy candidates in late-stage development:
| Disease/Target | Therapy Candidate | Trial Status (as of late 2025) |
| LGMD Type 2E/R4 | SRP-9003 | Enrollment and dosing complete in Phase 3 EMERGENE; BLA submission planned for the second half of 2025. |
| LGMD Type 2C/R5 | SRP-9005 | Cleared to proceed with dosing in U.S. Phase 1/2 COMPASS study. |
| LGMD Type 2D/R3 | SRP-9004 | Enrollment and dosing complete in Phase 1/2 DISCOVERY study. |
The siRNA platform targets additional diseases, with clinical trial progress noted:
- Type 1 Myotonic Dystrophy (DM1): Phase 1/2 SAD cohort enrollment complete; MAD cohort 4 ongoing.
- Facioscapulohumeral Muscular Dystrophy (FSHD): Phase 1/2 SAD cohort enrollment complete; MAD cohort 6 ongoing.
- Huntington's Disease (HD): Clinical trial for SRP-1005 on track to initiate by the end of 2025.
Readouts for the FSHD and DM1 Phase 1/2 studies are expected in early 2026.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Cost Structure
You're looking at the expense side of Sarepta Therapeutics, Inc.'s (SRPT) operations as of late 2025, and honestly, it's dominated by the cost of innovation and commercial scale-up. The numbers show a company heavily invested in its pipeline, which means high burn rates are the norm right now.
The Research and Development (R&D) engine is clearly the biggest cost driver. For the first half of 2025, the Non-GAAP R&D expense hit $930.9 million. This reflects the aggressive pursuit of their gene therapy and siRNA platforms. To be fair, a massive chunk of this was tied up in strategic external deals.
The collaboration costs are significant, particularly the deal with Arrowhead Pharmaceuticals. Sarepta Therapeutics made a $500 million upfront payment in cash to secure worldwide licensing rights to seven siRNA programs. This single transaction heavily influenced the Q1 2025 R&D figures, even though it's a multi-year investment in future revenue streams.
Manufacturing and quality control for complex AAV gene therapies are another pressure point. You see this reflected in the cost of sales, but also in specific write-offs. For instance, the first six months of 2025 included increased write-offs of certain product batches that didn't meet quality specifications, which is a direct cost of maintaining high standards for these specialized treatments.
Commercialization efforts are reflected in the Selling, General, and Administrative (SG&A) expenses. For the six months ended June 30, 2025, Non-GAAP SG&A expenses totaled $220.5 million. More recently, the adjusted SG&A for the third quarter of 2025 was reported at $77.1 million, which was lower than the prior year due to a restructuring plan initiated in July 2025.
When you put it all together, the overall spending is substantial. Sarepta Therapeutics projects its full-year 2025 operating expenses to fall between $1.78 billion and $2.18 billion. That range shows the financial commitment required to advance their pipeline while supporting the commercial launch of ELEVIDYS.
Here's a quick look at some of the key reported cost figures from the first half of 2025:
| Cost Category (Non-GAAP) | Period Ending June 30, 2025 (Six Months) | Period Ending June 30, 2024 (Six Months) |
| R&D Expenses | $930.9 million | $332.0 million |
| SG&A Expenses | $220.5 million | $206.5 million |
The collaboration expense is a major one-time hit, but the ongoing operational costs are also rising to support growth and quality assurance. You can see the impact of the Arrowhead deal and commercial scale-up in the year-over-year increases:
- Non-GAAP R&D expenses for H1 2025 were up approximately 180% compared to H1 2024.
- The $500 million upfront payment to Arrowhead in 2025 is a key driver of the R&D spike.
- Non-GAAP SG&A expenses for H1 2025 increased by $14.0 million year-over-year.
- The company is actively managing costs, targeting over $100 million in cost savings through the end of 2025 from its restructuring.
The projected 2025 operating expense range of $1.78 billion to $2.18 billion encapsulates these high R&D and commercialization needs. Finance: draft 13-week cash view by Friday.
Sarepta Therapeutics, Inc. (SRPT) - Canvas Business Model: Revenue Streams
You're looking at the core money-makers for Sarepta Therapeutics, Inc. as of late 2025. The revenue streams are heavily concentrated on their Duchenne Muscular Dystrophy (DMD) portfolio, but they also rely on partnerships for non-product income. It's all about getting these specialized genetic medicines to patients, so the numbers reflect that focus.
The primary driver is the Net product revenue from four commercial DMD therapies. This includes the gene therapy, Elevidys, and the three Phosphorodiamidate Morpholino Oligomer (PMO) treatments: EXONDYS 51, VYONDYS 53, and AMONDYS 45. For the third quarter of 2025, the total net product revenue hit $370.0 million.
Here's how that Q3 2025 product revenue broke down between the gene therapy and the PMOs:
| Product Category | Q3 2025 Net Product Revenue |
| PMO Therapies (EXONDYS 51, VYONDYS 53, AMONDYS 45) | $238.5 million |
| ELEVIDYS (Gene Therapy) | $131.5 million |
Despite some near-term headwinds, including a temporary suspension of shipments to non-ambulatory patients earlier in the year, the company still has significant full-year expectations. Sarepta Therapeutics, Inc. revised its 2025 total net product revenue guidance in May 2025 down to a range of $2.3 billion to $2.6 billion. This was a shift from the earlier projection of $2.9 billion to $3.1 billion.
For the flagship gene therapy, the expectation for the ambulant population remains a key target. Sarepta Therapeutics, Inc. continues to expect at least $500 million in annual revenue from Elevidys infusions in the ambulant population for the full year 2025.
Beyond direct product sales, Sarepta Therapeutics, Inc. captures revenue through external agreements, which is important for pipeline funding. This includes collaboration and royalty revenue from international partners like Roche, as well as other non-product income.
Specific figures related to these other revenue streams for the third quarter of 2025 include:
- Collaboration and other revenues were approximately $29.3 million.
- This figure reflected lower contract manufacturing revenues due to reduced ELEVIDYS shipment volumes to Roche.
- In the first quarter of 2025, royalty revenue from Roche specifically for ELEVIDYS sales totaled $4.0 million.
Finally, the pipeline progress translates directly into cash via milestone payments. For instance, the company incurred a significant milestone payment of $200 million to Arrowhead Pharmaceuticals related to the second DM1 program during 2025.
Finance: draft 13-week cash view by Friday.
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