NEW YORK, NY, USA I June 18, 2019 I 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. Further information about the clinical program is available here.

About Danon Disease

Danon disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and the European Union. The disease is often fatal in male patients in the second or third decade of life due to rapidly progressive heart failure. Available therapies for Danon disease include cardiac transplantation, which is associated with substantial complications and is not considered curative. There are no specific therapies available for the treatment of Danon disease.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) (“Rocket”) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket’s first two clinical programs are a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer, and an AAV-based gene therapy for Danon disease, a devastating, pediatric heart failure condition. Rocket’s pre-clinical pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). For more information about Rocket, please visit www.rocketpharma.com.

SOURCE: Rocket Pharmaceuticals