Rocket advances Danon disease trial
- Rocket Pharmaceuticals received FDA alignment on continued enrollment and dosing for its RP-A501 Phase 2 trial.
- The pivotal study will include 12 male patients treated with a recalibrated dose of RP-A501.
- Rocket expects to complete dosing of the remaining patients by mid-2027.
Rocket Pharmaceuticals (NASDAQ:RCKT) received alignment from the U.S. Food and Drug Administration on continued enrollment, dosing, and key design elements for its global Phase 2 pivotal trial of RP-A501 in patients with Danon disease.
The FDA reviewed data from the first three patients treated under the modified protocol and confirmed continued enrollment and dosing at the recalibrated dose after no clinical or laboratory evidence of thrombotic microangiopathy or capillary leak syndrome was observed.
The pivotal study will include 12 male patients receiving RP-A501 at a recalibrated dose of 3.8 × 10¹³ genome copies per kilogram using commercial-grade product, with the first three treated patients counting toward the study population.
Rocket expects to enroll and dose the remaining nine patients by mid-2027, with the primary 12-month assessment based on co-primary endpoints measuring myocardial LAMP2 protein expression and a 10% reduction in left ventricular mass index.
Rocket Pharmaceuticals develops genetic medicines for rare and life-threatening diseases, with a focus on inherited cardiovascular disorders and other genetic conditions.
The RP-A501 program is designed to treat Danon disease, a rare genetic disorder associated with cardiac complications caused by mutations affecting LAMP2 protein expression.
