Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”), a leading U.S.-based multi-platform clinical-stage gene therapy company, today announces that patient dosing has commenced in the open-label, Phase 1 clinical trial of RP-A501, the Company’s adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease.
University of California San Diego (UCSD) Health is the initial and lead center for the Phase 1 clinical trial under the leadership of Eric Adler, M.D., Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health and Professor of Medicine at University of California, San Diego School of Medicine and Barry Greenberg, M.D. Dr. Greenberg is the Director of the Advanced Heart Failure Treatment Program at UC San Diego Health and Professor of Medicine at UC San Diego School of Medicine, and is principal investigator of the trial.
“The initiation of patient dosing in our Phase 1 trial is a significant milestone for our RP-A501 program, the first investigational gene therapy for a monogenic heart failure syndrome,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “Danon disease is a rapidly progressive cardiomyopathy where we believe gene therapy can make a meaningful difference in patient outcomes. Current standards of care for Danon disease, including heart transplant, are not curative and are associated with considerable morbidity and mortality. As a result, median survival for male Danon disease patients has been reported at age 19, caused by progressive heart failure. This underscores the urgent need for new treatment options like RP-A501 gene therapy for the patients and families contending with this debilitating, fatal disease.”
“The advancement of RP-A501 into the clinic is a monumental step forward for the treatment of this devastating disease and, more broadly, the treatment of rare cardiac disorders,” said Dr. Adler. “The team at UC San Diego Health is pleased to be the initial and lead center for the Phase 1 clinical trial of RP-A501 and we look forward to rapidly advancing it through the clinic on behalf of patients and families in need.”
The non-randomized, open-label Phase 1 trial is expected to enroll 12-24 pediatric and young adult male patients. Two dose levels will be investigated in four patient cohorts separated by pediatric and adult age groups. The first cohort will receive a low dose level of 6.7×1013 genome copies/kg. Upon completion of patient dosing at the low dose, the Company plans to move to a higher dose. The study is designed to assess the safety and tolerability of a single infusion of RP-A501. Additional outcome measures include cardiomyocyte and skeletal muscle transduction by gene expression, histologic correction via endomyocardial biopsy, and clinical stabilization via cardiopulmonary testing.
Source: Company Press Release