Rocket Pharmaceuticals: FDA lifts clinical hold on Phase 2 trial of RP-A501 for Danon disease

ROCKET PHARMACEUTICALS, INC.

FDA lifted clinical hold in under 3 months; trial resumes with recalibrated dose 3.8x10^13 GC/kg for 3 patients with 4-week intervals.
Revised immunomodulatory regimen: discontinue prophylactic C3 inhibitor, maintain sirolimus/rituximab/steroids, lower eculizumab threshold.
Phase 2 trial is 12-patient single-arm; co-primary endpoint: LAMP2 expression and left ventricular mass reduction; key secondary: troponin change.
Six patients previously treated at 6.7x10^13 GC/kg; three additional at lower dose to be treated sequentially.
Natural history study running concurrently; all patients require 3-month pre-treatment run-in for biomarker assessment.

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