ROCKET PHARMACEUTICALS, INC. — Form 8-K

Rocket Pharmaceuticals announced FDA alignment on its Phase 2 pivotal trial of RP-A501 for Danon disease. The FDA confirmed a 12-patient pivotal efficacy population (with first 3 already enrolled at recalibrated dose of 3.8×10^13 GC/kg), cleared continued enrollment under modified protocol with optimized immunomodulatory regimen, and established 12-month co-primary endpoints (myocardial LAMP2 protein expression and 10% reduction in left ventricular mass index) intended to support accelerated approval. Company expects to complete remaining 9 patient dosing by mid-2027.