Sarepta Therapeutics, Inc.

What they do

Sarepta discovers and develops RNA-targeted therapeutics, siRNA knockdown therapies and gene therapies for rare diseases, with a commercial portfolio of four approved Duchenne products: EXONDYS 51 (eteplirsen), VYONDYS 53 (golodirsen), AMONDYS 45 (casimersen) and ELEVIDYS. Its PMO-based exon-skipping platform and AAVrh.74 gene therapy vector underpin these products, and it is also developing SRP-9003 for limb-girdle muscular dystrophy. Through a partnered program with Arrowhead it is developing siRNA candidates including SRP-1003 (DM1), SRP-1001 (FSHD) and SRP-1005 (Huntington's disease), plus programs in myotonic dystrophy, FSHD, spinocerebellar ataxia and idiopathic pulmonary fibrosis.

Revenue drivers

• PMO exon-skipping therapies (EXONDYS 51, VYONDYS 53, AMONDYS 45) — Generated $230.6 million of net product revenue in Q2 2026, making up the majority of the quarter's $328.7 million total.
• ELEVIDYS — The gene therapy for Duchenne contributed $98.1 million of net product revenue in Q2 2026, roughly 30% of quarterly net product revenue.
• Total net product revenue — Annual revenue grew from $612.4 million in 2021 to $1.86 billion in 2025, driven by the combination of PMO and ELEVIDYS sales.
• Pipeline-stage candidates — SRP-9003 (LGMD), SRP-1001, SRP-1003 and SRP-1005 are not yet approved and generate no product revenue.

Recent performance

For the second quarter of 2026, Sarepta reported total net product revenue of $328.7 million, consisting of $230.6 million of PMO net product revenue and $98.1 million of ELEVIDYS net product revenue. GAAP operating income was $13.3 million and non-GAAP operating income was $86.5 million. Quarterly revenue declined from $370.0 million in Q3 2025 and $442.9 million in Q4 2025 to $330.5 million in Q1 2026 and $328.7 million in Q2 2026. The company ended the quarter with approximately $945.0 million of cash, cash equivalents, restricted cash and investments, an increase of roughly $197.0 million in the quarter. Full-year 2025 revenue was $1.86 billion with a net loss of $713.4 million, compared with net income of $235.2 million in 2024.

Strategy

Sarepta says its priorities are executing its commercial strategy, advancing its siRNA pipeline, including SRP-1001 and SRP-1003 with data readouts expected in the second half of 2026, and continuing disciplined capital allocation. It is investing in next-generation precision medicine through internal research, partnerships and collaborations, and advancing its gene therapy pipeline including SRP-9003 LGMD and manufacturing with Catalent and Aldevron. The company appointed Michael Severino, MD, as CEO effective July 28, 2026, and narrowed its full-year 2026 total net product revenue guidance to $1.2–$1.3 billion and combined non-GAAP R&D and SG&A expense guidance to $800.0–$850.0 million. It is also pursuing sNDAs to convert AMONDYS 45 and VYONDYS 53 from accelerated to traditional approval, and is conducting an immunosuppression study of sirolimus with ELEVIDYS for non-ambulatory individuals.

Risks

• ELEVIDYS label changes and suspension — The FDA added a boxed warning for acute liver injury and acute liver failure and removed the non-ambulatory population from the ELEVIDYS Indication and Usage section, and the 10-Q references an 'ELEVIDYS Suspension' that could affect sales and dosing.
• PMO confirmatory trial risk — Results of the ESSENCE confirmatory trial for VYONDYS 53 and AMONDYS 45 could lead to FDA directives to remove these products from the market or alter labels, affecting demand, reimbursement and coverage.
• LGMD clinical hold — The FDA placed a clinical hold on Sarepta's investigational gene therapy clinical trials for LGMD in July 2025 and revoked the platform technology designation for its AAVrh74 platform technology on June 2, 2025.
• Sustained losses and cash use — The company reported a 2025 net loss of $713.4 million and negative operating cash flow of $205.5 million in 2025, and may need additional capital to fund pipeline programs and medium-term liabilities.

Outlook

Management narrowed full-year 2026 total net product revenue guidance to $1.2–$1.3 billion, toward the lower end of its prior $1.2–$1.4 billion range, and narrowed combined non-GAAP R&D and SG&A expense guidance to $800.0–$850.0 million. Readouts from the MAD cohorts of the ongoing Phase 1/2 studies in DM1 and FSHD remain on track for the second half of 2026. Full enrollment of ENDEAVOR Cohort 8 for ELEVIDYS is expected by year-end 2026, with 12-week data from the full cohort expected in Q1 2027. The company says it is well positioned to advance its DM1 and FSHD programs using commercial cash flows while maintaining disciplined capital allocation.