Study of SRP-4045 (Casimersen) and SRP-4053 (Golodirsen) in Participants With Duchenne Muscular Dystrophy (DMD)

Summary

The main objective of this study is to evaluate the efficacy of SRP-4045 (casimersen) and SRP-4053 (golodirsen) compared to placebo in participants with DMD with out-of-frame deletion mutations amenable to skipping exon 45 and exon 53, respectively.

Timeline

Start

2016-09-28

Primary completion

2024-11-12

Completion

2025-10-16

Outcome

Missed primary endpoint

release “While the ESSENCE study did not achieve statistical significance on its primary endpoint, results indicate positive and encouraging trends favoring therapy at 96 weeks” sarepta.com ↗

release “In the Phase 3 ESSENCE study evaluating casimersen and golodirsen versus placebo, the primary endpoint was not met; however, numerical trends favored treatment.” sarepta.com ↗

Publications

• Vandekerckhove I, Hanssen B, Peeters N, Dewit T, De Beukelaer N, Van den Hauwe M, De Waele L, Van Campenhout A, De Groote F, Desloovere K. Anthropometric-related percentile curves for muscle size and strength of lower limb muscles of typically developing children. J Anat. 2025 Aug;247(2):348-362. doi: 10.1111/joa.14241. Epub 2025 Mar 17.
• Wagner KR, Kuntz NL, Koenig E, East L, Upadhyay S, Han B, Shieh PB. Safety, tolerability, and pharmacokinetics of casimersen in patients with Duchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double-blind, placebo-controlled, dose-titration trial. Muscle Nerve. 2021 Sep;64(3):285-292. doi: 10.1002/mus.27347. Epub 2021 Jun 29.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Casimersen	Unknown	30 mg/kg	Intravenous
Subject	Golodirsen	Unknown	30 mg/kg	Intravenous

Indications