Sarepta Therapeutics Reports Q2 2024 Financials and Recent Updates

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), a leader in precision genetic medicine for rare diseases, announced its financial results for the second quarter of 2024, marking a significant milestone in advancing treatments for Duchenne Muscular Dystrophy (DMD).

Doug Ingram, President and CEO of Sarepta Therapeutics, emphasized that the second quarter of 2024 represented a pivotal moment since identifying the lack of dystrophin as the underlying cause of Duchenne in 1986. He highlighted the FDA's decision to broaden access to their gene therapy, ELEVIDYS, which now includes all DMD patients aged four and above. The FDA granted traditional approval for ambulatory patients and accelerated approval for non-ambulatory patients. This expansion is backed by extensive clinical evidence, including muscle and cardiac MRI data showcasing improvements in muscle health among treated patients. Sarepta plans to present comprehensive data on ELEVIDYS at the 29th Annual Congress of the World Muscle Society in October.

Sarepta reported robust financial performance, with second-quarter net product revenues reaching $360.5 million, a 51% increase compared to the same period last year. The company's PMO products, EXONDYS 51, VYONDYS 53, and AMONDYS 45, contributed $238.8 million in revenues, while ELEVIDYS added $121.7 million. This strong performance positions Sarepta for significant growth in the latter half of 2024 and beyond, with projected net product revenues in 2025 expected to range between $2.9 and $3.1 billion.

Recent developments include the FDA's expanded approval of ELEVIDYS to treat individuals with DMD who are at least four years old, with both traditional and accelerated approval based on ambulatory status. Continued approval for non-ambulatory patients will depend on verification of clinical benefits in a confirmatory trial. Sarepta has committed to conducting a randomized, controlled trial to confirm ELEVIDYS's clinical benefits for non-ambulatory patients.

Furthermore, Roche, Sarepta's partner, announced that the European Medicines Agency (EMA) has begun reviewing the marketing authorization application for ELEVIDYS, targeting approval for ambulatory patients aged three to seven years by 2025. Roche is responsible for marketing ELEVIDYS outside the United States.

Sarepta plans to present data from its SRP-9001 program at the World Muscle Society Congress in Prague, Czechia, in October. The presentations will cover muscle MRI and cardiac MRI outcomes, micro-dystrophin expression, and safety from various studies, including EMBARK and ENDEAVOR. Additionally, the company is conducting long-term follow-up studies, including ENDURE, a Phase 4 observational study, and EXPEDITION, a Phase 3 study, to monitor the safety and efficacy of ELEVIDYS over extended periods.

The U.S. FDA has granted Fast Track designation to SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy Type 2E (LGMD2E/R4). This designation is aimed at expediting the development and review of drugs that address serious conditions with unmet medical needs. SRP-9003 aims to deliver a full-length beta-sarcoglycan transgene, essential for expressing in the heart, which is crucial for patients with LGMD2E/R4.

Renowned neuromuscular researcher and gene therapy pioneer Jerry R. Mendell, M.D., has been named to the inaugural TIME100 Health list for 2024, recognizing his significant contributions to global health and genetic medicine. Dr. Mendell's work has been instrumental in advancing treatments for genetic diseases, including Duchenne muscular dystrophy.

Sarepta's financial performance for the second quarter of 2024 showed total revenues of $362.9 million, a 39% increase compared to the same period in 2023. The company's GAAP operating loss was reduced from $133.5 million in 2023 to $0.7 million in 2024, while non-GAAP operating income improved from a loss of $75.5 million to a gain of $57.9 million. For the first half of 2024, total revenues increased by 51% to $776.4 million, with GAAP operating income of $34.2 million, compared to a loss of $271.6 million in the same period of 2023. Non-GAAP operating income for the first half of 2024 was $141.6 million, up from a loss of $161.2 million in 2023.

Sarepta's continued dedication to developing and commercializing therapies for rare diseases is reflected in their expanding pipeline and strong financial performance, positioning them for sustained growth in the coming years.