Efficacy UncertaintyA dose-dependent effect was not clearly visible in most of the functional endpoints including Stride Velocity 95th Centile, timed rise from floor, and timed 10-meter walk/run test at the 6 month mark.
Management InstabilityThe company announced the departure of 3 senior executives, the CMO, COO, and CBO.
Safety ConcernsThe tradeoff of magnitude of increased dystrophin expression vs the potential safety risk now appears less favorable.