Efficacy CredibilityThe earlier Phase 3 EMBARK trial failed to meet its primary movement endpoint, creating doubt about consistent functional benefit and weakening confidence in future efficacy claims.
Placebo-controlled Trial RiskA new placebo-controlled study raises the chance that Elevidys will not demonstrate a clear benefit on the core movement test, which would undercut comparisons to external control groups.
Safety ConcernsSerious adverse events in early trials, including a fatal arrhythmia in a DM1 patient, heighten safety scrutiny and could slow development or prompt more stringent regulatory review.