CRANBURY, N.J.– Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces updated data from the Phase 1 clinical trial evaluating a single intravenous infusion of RP-A501, the Company’s investigational gene therapy, for the treatment of Danon Disease. This update, which 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, demonstrates RP-A501 was generally well tolerated at the low-dose and conferred sustained clinical benefit.
“We are excited to announce positive data from our RP-A501 trial for Danon Disease showing clinical, functional and biomarker improvements at one year or beyond and potential early separation from the natural course of disease in adult and adolescent patients,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “Male patients living with Danon Disease suffer a heavy disease burden and face rapidly progressive heart failure in their teenage years. In this setting, even stabilization is highly beneficial, and clinical improvements as seen in our trial could be transformative and represent a step forward for the treatment of monogenic heart failure and gene therapy for cardiac diseases. We have initiated treatment in the pediatric cohort at the low-dose and anticipate moving as rapidly as possible toward Phase 2.”
Dr. Barry Greenberg, Director of the Advanced Heart Failure Treatment Program at UC San Diego Health, Professor of Medicine at UC San Diego School of Medicine, and the principal investigator of the RP-A501 clinical study, is presenting a subset of the data at the American Heart Association (AHA) Scientific Sessions 2021. Dr. Greenberg added, “I am very encouraged by the ability to safely administer RP-A501 and show robust gene expression in the hearts of these young male patients with this devasting monogenic disorder. The clinical data to date also demonstrate encouraging results including clinical stabilization of this rapidly progressive disease.”
