RP-A501

August 3, 2026: Rocket Pharmaceuticals (NASDAQ: RCKT) reported a positive safety update from the initial three patients treated under a modified protocol in its global pivotal Phase 2 trial of RP-A501 for Danon disease.

Under the revised regimen, patients received RP-A501 at 3.8 × 10¹³ GC/kg plus rituximab, sirolimus, and corticosteroids, with at least four weeks between infusions. Rocket said RP-A501 was well tolerated, with no thrombotic microangiopathy, capillary leak syndrome, or other significant safety concerns observed to date in these three patients.

The pivotal study is designed as a 12-patient, single-arm Phase 2 trial. Rocket said it is actively engaging with the FDA to align on dosing additional patients and completing the trial under the modified protocol. A regulatory pathway update is expected in the second half of 2026.

The company also said it remains on track to provide a comprehensive Danon disease program update in the second half of 2026.