November 3, 2025 — Sarepta Therapeutics (NASDAQ:SRPT) reported its third-quarter financial results for 2025 and the completion of ESSENCE, a Phase 3 study that evaluated the efficacy and safety of AMONDYS 45 (casimersen) and VYONDYS 53 (golodirsen) compared to placebo in 225 patients with Duchenne muscular dystrophy.
Topline results from ESSENCE found that numerical trends favored treatment versus placebo; however, the observed difference of 0.05 steps/second in least square means (LSM), did not reach statistical significance (P=0.309) on the primary endpoint, the 4-step ascend velocity at 96 weeks.
The ESSENCE study was conducted over a nine-year period that included the COVID-19 pandemic, which impacted study participants and outcomes. An analysis that excludes data from participants whose double-blind period overlapped with the COVID-19 pandemic (n=57), shows a 30% reduction (LSM 0.11 steps/second, P=0.09) in disease progression over 2 years on the 4-step ascend velocity in non-COVID impacted treated participants versus placebo (n=168). This is a clinically meaningful change.
There were no new safety signals in the ESSENCE study. Adverse events were mostly mild (88%) or moderate (10.3%) and comparable between treatment and placebo groups. The most common treatment-emergent adverse events (≥10%) were vomiting, nasopharyngitis, pyrexia, headache, cough, fall, and upper respiratory infections.
Sarepta intends to schedule a meeting with the FDA to discuss the possibility of converting from accelerated to traditional approval, based on encouraging trends, substantial real-world evidence, and a positive safety profile.
Sarepta continues to analyze the results from ESSENCE and, together with real-world evidence, full results will be submitted to the FDA as part of the planned sNDA filings for these two exon-skipping therapies. Additionally, results from ESSENCE will be shared at future medical meetings, and publication will be pursued in a medical journal.
Net product revenue stood at $370.0 million for the quarter, and positive cash flow was reported. Safety labeling discussions with the FDA for ELEVIDYS are expected to be finalized in the near-term.
Readouts of FSHD and DM1 phase 1/2 studies from both SAD and MAD cohorts expected in early 2026. Huntington’s Disease (HD): On track to initiate clinical trial for SRP-1005 by end of 2025 utilizing subcutaneous route of administration.
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Contact: Sarepta Therapeutics, Inc. at 1-888-SAREPTA (1-888-727-3782) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch
Source: Sarepta Therapeutics, Inc.
