Rocket Pharma To Test Lower Dose Of Gene Therapy In Genetic Disorder Trial

Rocket Pharmaceuticals Inc RCKT has announced updated data from the Phase 1 trial evaluating a single intravenous infusion of RP-A501, an investigational gene therapy, for Danon Disease.
Danon disease is characterized by weakening of the heart muscle, weakening of skeletal muscles, and intellectual disability.
The update includes interim safety and efficacy data from patients in the low-dose (6.7e13 vg/kg; n=3) and high-dose (1.1e14 vg/kg; n=2) adult and adolescent cohorts.
Data demonstrated that RP-A501 was generally well tolerated at the low dose and conferred sustained clinical benefit.
Across both dose levels, the most common serious adverse event observed was steroid-induced myopathy in three patients (two low-dose and one high-dose), which resolved after corticosteroid discontinuation.
Based on the observed safety and efficacy, the Company will focus on the low-dose cohort moving forward and will no longer administer the high-dose to study patients.
Related Link: FDA Lifts Clinical Hold On Rocket Pharma's X-Linked Inherited Disorder Trial.
Among the three low-dose patients, BNP (a key marker of heart failure) decreased from a pretreatment baseline by 57% at 24 months, 79% at 18 months, and 75% at 15 months, respectively.
In the high-dose patient, BNP decreased from a pretreatment baseline by 67% at 12 months.
Price Action: RCKT shares are down 20.20 at $27.09 during the market session on the last check Monday.