Rocket Pharma reports positive long-term Phase 1 Danon Disease data; PKD program enrollment delayed to 2025

ROCKET PHARMACEUTICALS, INC.

All evaluable Danon disease patients showed cardiac LAMP2 protein expression at 12 months, sustained up to 60 months, and ≥10% LV mass index reduction sustained up to 54 months.
Median reduction in LV mass index 24%; cardiac biomarkers cTnI down 84%, NTproBNP down 57%; NYHA class improved II to I; KCCQ score improved 27 points.
RP-A501 generally well tolerated with manageable AEs; one patient with baseline LVEF<40% required heart transplant 5 months post-treatment.
Phase 2 pivotal trial for Pyruvate Kinase Deficiency (RP-L301) design agreed with FDA; enrollment not started, anticipates resuming in 2025.

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