P.11.21 Bioanalysis of a double blind, placebo-controlled clinical phase 2 study of drisapersen for the treatment of boys suffering from Duchenne muscular dystrophy and comparison to clinical outcome results

To Skip or Not to Skip: That Is the Question for Duchenne Muscular Dystrophy

“…One is the amount of functional dystrophin protein generated, which was probably marginal for clinical efficacy. 1 This contention is supported by reported drisapersen phase II dystrophin quantification data 4 that failed to show a correlation between 6MWT outcome and a dystrophin-recovery composite score. The other factor is the 6MWT primary outcome measure itself.…”

Dystrophin Analysis in Clinical Trials

“…Like for healthy muscle, dystrophin intensities varied between fibers for BMD and DMD patients (up to three-fold) (unpublished observations, C Beekman and A Lourbakos, Prosensa Therapeutics, Leiden, the Netherlands (2013)). Notably, after treatment of patients in the phase 2 dose regimen study with drisapersen, a shift towards higher dystrophin intensities could be observed in the majority of post-treatment samples compared to pre-treatment fibers [37]. Furthermore, a 30% increase in intensities was observed for post-treatment samples vs pre-treatment samples from patients involved in the phase 2a trial with PRO044 for exon 44 skipping [36](unpublished observations, C Beekman and A Lourbakos, Prosensa Therapeutics, Leiden, the Netherlands (2013)).…”