- The first patient has been dosed in a Phase 3 clinical trial, ESSENCE, evaluating Sarepta Therapeutics' (NASDAQ:SRPT) SRP-4045 and SRP-4053 for the treatment of patients with Duchenne muscular dystrophy (DMD) amenable to exon 45 and exon 53 skipping.
- The double-blind, placebo-controlled study will randomize 99 subjects to 1:1:1 to receive once-weekly infusions of either 30 mg/kg of SRP-4045, 30 mg/kg of SRP-4053 or placebo for up to 96 weeks. The test period will be followed by an open-label extension phase in which all participants will receive active treatment for up to an additional 96 weeks.
- The primary endpoint is the change from baseline to Week 96 in the 6-minute walk test (6MWT). According to ClinicalTrials.gov, the estimated final data collection date for the primary endpoint is September 2019. The estimated study completion date is June 2021.
- SRP-4045 and SRP-4053, incorporating technology to skip exons 45 and 53, respectively, are designed to address the underlying cause of DMD by restoring the messenger RNA reading frame, thereby enabling the production of a shorter form of the dystrophin protein. Recently approved Exondys 51 (eteplirsen) is targeted to DMD patients amenable to skipping exon 51.
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