Rocket Pharma gets FDA alignment on pivotal Danon trial; 12-patient cohort path clear

ROCKET PHARMACEUTICALS, INC.

FDA confirms 12-patient pivotal population for RP-A501; first 3 treated under modified protocol count toward it.
Recalibrated dose 3.8e13 GC/kg; no thrombotic microangiopathy or capillary leak in first 3 patients.
Co-primary endpoints: myocardial LAMP2 expression and 10% reduction in left ventricular mass index at 12 months, supporting potential accelerated approval.
Company expects to complete dosing of remaining 9 patients by mid-2027.
Virtual investor webinar on Danon disease program scheduled for Oct 6, 2026 at 4:30 p.m. ET.

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