Rocket Pharma reports positive safety data from first 3 patients in Phase 2 Danon disease trial

ROCKET PHARMACEUTICALS, INC.

Initial three patients treated safely with RP-A501 at recalibrated dose 3.8×10^13 GC/kg; no TMA or capillary leak syndrome observed.
Company engaging with FDA to align on path to complete pivotal Phase 2 trial; regulatory update expected H2 2026.
Comprehensive Danon disease program update on track for H2 2026.
Phase 2 trial designed as 12-patient single-arm study.

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