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Additional Relevant Details Sarepta's exon-skipping candidate SRP-4053 for the treatment of patients amenable to exon 53 skipping. The Phase 1/2 study was initiated in 2015 and continues to enroll patients, testing for efficacy and safety. The projected completion of the study is January 2017, expect data from the study in the first half of 2017.

https://www.clinical...

Clinical Data Update on Sept 6 2017: The study comprised two parts. In Part 1, 12 patients were randomized to receive a dose titration of golodirsen (8 patients) or placebo (4 patients). At the end of Part 1 (dose titration), all 12 patients continued on golodirsen and an additional 13 patients started golodirsen (Part 2). In Part 2, all 25 patients were treated for an additional 48 weeks at the time of muscle biopsy. The analysis included biopsies of the bicep muscle at baseline and on-treatment at the Part 2 Week 48 time point. All 25 participants displayed an increase in skipping exon 53 (p < 0.001) over baseline levels, representing a 100 percent response rate as measured by RT-PCR and demonstrating proof of mechanism. Mean dystrophin protein increased to 1.019 percent of normal compared to a mean baseline of 0.095 percent of normal (p < 0.001) as measured by Western blot, the primary biological endpoint in the study, representing a 10.7 fold increase from baseline. The study also showed a statistically significant increase in dystrophin immunofluorescence as measured by immunohistochemistry (IHC), the secondary biological endpoint in the study, confirming sarcolemma-associated protein expression and distribution.

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