Sarepta Therapeutics (SRPT) is:

• Like Vertex Pharmaceuticals, but focused on Duchenne Muscular Dystrophy.
• Like Novartis Gene Therapies, but specifically for rare muscle diseases.

• EXONDYS 51: Treats Duchenne muscular dystrophy in patients with mutations amenable to exon 51 skipping.
• VYONDYS 53: Treats Duchenne muscular dystrophy in patients with mutations amenable to exon 53 skipping.
• AMONDYS 45: An investigational exon-skipping therapy candidate targeting exon 45 of the dystrophin gene for Duchenne muscular dystrophy.
• SRP-5051: An investigational peptide-conjugated phosphorodiamidate morpholino oligomer (PPMO) targeting exon 51 of dystrophin pre-mRNA for Duchenne muscular dystrophy.
• SRP-9001: An investigational micro-dystrophin gene therapy program for Duchenne muscular dystrophy.
• SRP-9003: An investigational gene therapy program for limb-girdle muscular dystrophies.

• Catalent (CTLT)
• Lonza (LONN.SW)
• Thermo Fisher Scientific (TMO)
• Rentschler Biopharma SE

Douglas S. Ingram was appointed President and Chief Executive Officer of Sarepta Therapeutics in June 2017. Prior to joining Sarepta, Mr. Ingram served as President of Allergan, Inc. from 2013 until its acquisition by Actavis in 2015 for $66 billion. Following Allergan, he was the President, Chief Executive Officer, and a Director of Chase Pharmaceuticals Corporation, a clinical-stage biopharmaceutical company, from December 2015 until its sale in November 2016 (acquired by Allergan). His career in healthcare spans nearly 30 years.

Ryan Wong was appointed Executive Vice President and Chief Financial Officer of Sarepta Therapeutics in July 2025. Before this role, he served as the company's Senior Vice President of Strategic Finance, Treasury, and Investor Relations. Prior to joining Sarepta in July 2021, Mr. Wong held roles of increasing responsibility within the finance function at GW Pharmaceuticals plc and Allergan.

Ian M. Estepan was appointed President and Chief Operating Officer of Sarepta Therapeutics in July 2025. He previously served as Sarepta's Executive Vice President and Chief Financial Officer from December 2020 to July 2025. Mr. Estepan joined Sarepta in 2015 and was a key architect in securing $2.5 billion in capital to support the company's growth. Before Sarepta, he spent over a decade as a portfolio manager for Spectra Financial Group, managing a portfolio of pharmaceutical, biotech, and medtech equities.

Louise R. Rodino-Klapac, PhD, was appointed President, Research & Development and Technical Operations in July 2025. She joined Sarepta in June 2018 and previously served as Executive Vice President, Chief Scientific Officer, and Head of R&D. Dr. Rodino-Klapac was the former head of the Laboratory for Gene Therapy Research at Nationwide Children's Hospital and co-founded Myonexus Therapeutics, which Sarepta acquired in 2019. She is also an inventor of five of Sarepta's limb-girdle muscular dystrophy programs.

Rachael Potter, PhD, was appointed Chief Scientific Officer in July 2025. Prior to this role, she served as Senior Vice President, Head of Research Sciences at Sarepta Therapeutics.

1. Regulatory and Safety Risks with Gene Therapy (Elevidys) / Patient Deaths: Sarepta faces significant regulatory and safety challenges, particularly concerning its gene therapy, ELEVIDYS. There have been reports of patient deaths, at least two linked to ELEVIDYS, and a third to another gene therapy utilizing the same platform, all attributed to acute liver failure. These incidents led to a boxed warning (the most serious safety warning) being added to ELEVIDYS' label by the FDA, along with restrictions on its use for patients with pre-existing liver impairment, recent vaccinations, or active/recent infections. Clinical trials for other gene therapy products were also placed on hold, and the FDA revoked Sarepta's platform technology designation. This directly impacts the commercial viability and future development of their gene therapy pipeline.
2. Clinical Trial Failures and Pipeline Risk: The company's RNA-based exon-skipping therapies, AMONDYS 45 and VYONDYS 53, experienced a significant setback when their ESSENCE confirmatory study failed to meet its primary endpoint with statistical significance in Q3 2025. This failure creates considerable uncertainty regarding the long-term commercial future of these approved drugs, which are major revenue drivers for Sarepta.
3. Intensifying Competition in Duchenne Muscular Dystrophy (DMD) Treatment: Sarepta operates in a highly competitive landscape with several companies developing alternative treatments for DMD. Competitors such as Pfizer, with its investigational DMD gene therapy fordadistrogene movaparvovec, are reporting positive Phase 3 data and are expected to enter the market by 2026, posing a direct threat to ELEVIDYS. Other competitors include NS Pharma, PTC Therapeutics, Solid Biosciences, Regenxbio (aiming for approval of RGX-202 in mid-2026), Dyne Therapeutics, and Italfarmaco, which could erode Sarepta's market share and pricing power.

Sarepta Therapeutics (SRPT) focuses on developing therapies for rare diseases, primarily Duchenne Muscular Dystrophy (DMD) and Limb-Girdle Muscular Dystrophies (LGMD). The addressable markets for their main products and services are as follows:

• EXONDYS 51 (exon 51 skipping): This product targets Duchenne Muscular Dystrophy patients with a confirmed mutation of the dystrophin gene amenable to exon 51 skipping. This patient population represents approximately 13% of all Duchenne Muscular Dystrophy cases globally.
• VYONDYS 53 (exon 53 skipping): This therapy is for Duchenne Muscular Dystrophy patients with a confirmed mutation of the dystrophin gene amenable to exon 53 skipping. This patient population constitutes approximately 8% of Duchenne Muscular Dystrophy patients globally.
• AMONDYS 45 (exon 45 skipping): This product candidate uses exon-skipping technology to skip exon 45 of the dystrophin gene. The patient population amenable to exon 45 skipping accounts for approximately 6% of Duchenne Muscular Dystrophy cases globally.
• SRP-5051 (peptide conjugated PMO that binds exon 51): This is a next-generation therapy specifically designed for individuals with Duchenne Muscular Dystrophy susceptible to exon 51 skipping. The addressable market is the same patient population as EXONDYS 51, approximately 13% of Duchenne Muscular Dystrophy cases globally.
• SRP-9001 (Elevidys - DMD micro-dystrophin gene therapy): Elevidys, Sarepta's gene therapy for Duchenne Muscular Dystrophy, is projected by analysts to achieve peak sales of approximately $3.6 billion, with the company itself suggesting a peak revenue opportunity exceeding $4 billion globally.
• SRP-9003 (Limb-Girdle Muscular Dystrophies gene therapy for LGMD Type 2E/R4): The global Limb-Girdle Muscular Dystrophy treatment market was valued at approximately $1.36 billion in 2024 and is expected to reach $1.96 billion by 2032. North America held the largest revenue share, accounting for 41.3% of the global market in 2024, with the U.S. representing 82.4% of the North American revenue.

Expected Revenue Growth Drivers for Sarepta Therapeutics (SRPT) over the Next 2-3 Years

Sarepta Therapeutics is anticipated to drive future revenue growth through several key initiatives and product developments over the next two to three years:

1. Continued U.S. Commercial Expansion and Label Growth of ELEVIDYS (SRP-9001): ELEVIDYS, Sarepta's gene therapy for Duchenne muscular dystrophy (DMD), received FDA accelerated approval in June 2023, followed by a significant label expansion in June 2024 to include patients aged 4 and older, with traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. The company's near-term growth is heavily reliant on the market expansion of ELEVIDYS, with management actively focusing on activating treatment centers, securing payer contracts, and educating caregivers to accelerate patient uptake. Efforts to scale manufacturing capacity and optimize treatment slots through 2025 are also crucial to reduce time-to-infusion for newly eligible patients. In Q1 2025, ELEVIDYS was a primary revenue driver, generating $375.0 million, representing a 180% year-over-year increase. Analysts project ELEVIDYS sales to be a key driver, potentially reaching around $2 billion in 2025.
2. International Expansion of ELEVIDYS through Strategic Partnerships: Sarepta is expanding the global reach of ELEVIDYS through its partnership with F. Hoffman-La Roche Ltd, which holds ex-U.S. commercialization rights. A European Marketing Authorization Application (MAA) is progressing, with an anticipated EMA decision in late 2025 or early 2026, paving the way for staged country launches across Europe. Additionally, Sarepta and Roche are pursuing parallel submissions and early-access pathways in other international markets, such as the Middle East and Latin America, to establish market presence ahead of broad reimbursement. ELEVIDYS also launched in Japan as of February 2026. These international efforts are expected to contribute to Sarepta's revenue through royalties and collaboration income.
3. Sustained Performance of the PMO (Phosphorodiamidate Morpholino Oligomer) Franchise: The company's existing RNA-based PMO therapies, EXONDYS 51, VYONDYS 53, and AMONDYS 45, continue to be core cash-generating products. These therapies are important for supporting near-term cash flow while the gene therapy portfolio scales. The PMO franchise contributed $236.5 million to net product revenues in Q1 2025, with stable performance in Q4 2025. While the confirmatory ESSENCE trial for AMONDYS 45 and VYONDYS 53 did not achieve statistical significance on its primary endpoint, the study reinforced the favorable safety profile and showed positive trends, prompting Sarepta to discuss a path to full FDA approval leveraging this data and real-world evidence.
4. Advancement of Pipeline Programs for Limb-Girdle Muscular Dystrophies (LGMDs): Sarepta is diversifying its gene therapy pipeline beyond DMD by expanding its AAV portfolio into other neuromuscular indications, specifically Limb-Girdle Muscular Dystrophies (LGMDs). Notably, SRP-9003 is advancing to Phase 3 for LGMD type 2E/R4, which could provide a first-mover advantage in addressing these underserved patient populations. The company anticipates important Phase 3 biomarker data readouts for SRP-9003 in the first half of 2025, which could further solidify its position in the LGMD treatment landscape. Sarepta is also progressing other LGMD programs and leveraging collaborations, such as with Arrowhead Pharmaceuticals, for additional therapeutic modalities.

Share Repurchases

• Sarepta Therapeutics announced a share repurchase program of up to $500 million in November 2024.
• As of June 30, 2025, $475.0 million remained authorized for future share repurchases.
• The company held 650,876 shares in treasury stock at cost as of December 31, 2025, compared to 0 shares at December 31, 2024, indicating share repurchases during 2025.

Share Issuance

• Sarepta proposed to issue and sell approximately 6.17 million shares of common stock in an offering in October 2021.
• The company completed an underwritten public offering of 7,098,766 shares of common stock at $81.00 per share, generating approximately $575 million in gross proceeds.
• The weighted average number of shares of common stock outstanding for diluted earnings per share increased from 88.19 million for the three months ended March 31, 2021, to 99.11 million for the three months ended March 31, 2022.

Inbound Investments

• Collaboration revenue from F. Hoffman-La Roche Ltd amounted to $89.5 million in 2021 and $89.2 million in 2022.
• For the full year 2025, Sarepta recognized $334 million in collaboration, contract manufacturing, and royalty revenue from its partnership with Roche.
• The company received a $63.5 million milestone payment linked to ELEVIDYS approval in Japan, stemming from its collaboration with Roche.

Outbound Investments

• Sarepta is expanding its AAV portfolio into additional neuromuscular indications and advancing Phase 1/2 readouts across 2025–2026, leveraging academic and biotech partnerships, including with Nationwide Children's Hospital, University of Western Australia, and Dyno Therapeutics.
• A key part of the updated corporate strategy, announced July 16, 2025, involves prioritizing small interfering RNA (siRNA) candidates developed in collaboration with Arrowhead Therapeutics.

Capital Expenditures

• Capital expenditures were $38 million in 2021, $31 million in 2022, $76 million in 2023, $137 million in 2024, and $102 million in 2025.
• Sarepta invested $22.9 million in capital expenditures in the third quarter of 2025, primarily funding long-term assets and infrastructure.
• Expected capital expenditures are $70.83 million for 2026 and $67.42 million for 2027. The primary focus of capital allocation includes sustained R&D intensity to support pipeline expansion across antisense, AAV-based gene therapy, and gene editing modalities, as well as funding manufacturing capacity.