Sarepta Shatters Expectations, Redefines Gene Therapy Leadership with Unprecedented Milestones in 2024's First Half

Sarepta Therapeutics achieved remarkable milestones in gene therapy and muscular dystrophy treatments during the first half of 2024, solidifying its leadership in the biotech sector.

A standout achievement was the expanded FDA approval of ELEVIDYS, Sarepta's pioneering gene therapy for Duchenne muscular dystrophy (DMD). Initially approved for ambulatory patients, ELEVIDYS received traditional approval for this group and accelerated approval for non-ambulatory patients aged 4 and above in June 2024. This expansion, contingent on ongoing confirmatory trials, positions ELEVIDYS as a groundbreaking therapy, generating $133.9 million in Q1 2024 alone.

Sarepta's financial performance reflected this success, with Q1 2024 total revenues reaching $359.5 million—a 55% increase compared to the same period in the previous year. The company’s GAAP net income of $36.1 million marked a significant turnaround from the previous year's losses, with non-GAAP earnings of $78.2 million further underscoring its financial strength.

On the research front, Sarepta advanced its gene therapy pipeline with promising data from the Phase 2 MOMENTUM study of SRP-5051, demonstrating significant dystrophin expression for DMD patients amenable to exon 51 skipping. The company also launched the Phase 3 EMERGENE study for SRP-9003, targeting limb-girdle muscular dystrophy Type 2E, aiming to validate the therapy's efficacy.

Sarepta’s commitment to the muscular dystrophy community was further evident through its LGMD 2024 Grant Program, aimed at improving diagnostic processes, and the continuation of its Route 79 Duchenne Scholarship Program, providing educational opportunities to individuals with DMD and their families.

Additionally, Sarepta continued to expand its team, regularly awarding equity grants as part of its ongoing investment in talent under Nasdaq Listing Rule 5635(c)(4).

The first half of 2024 marked significant clinical, financial, and strategic progress for Sarepta Therapeutics, setting a strong foundation for continued growth and innovation in the gene therapy space.