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Rocket Pharmaceuticals Announces First Patient Treated In Higher Dose Cohort In Phase 1 Clinical Trial Of RP-A501 For Danon Disease

—Safety Data from Low Dose Cohort Supports FDA Clearance for Higher Dose Patient Treatment— —Phase 1 Preliminary Safety and Efficacy Data Expected Fourth Quarter 2020— NEW YORK--(BUSINESS

· 09/02/2020 07:00

—Safety Data from Low Dose Cohort Supports FDA Clearance for Higher Dose Patient Treatment—

—Phase 1 Preliminary Safety and Efficacy Data Expected Fourth Quarter 2020—

NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) (“Rocket”), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare pediatric disorders, today announces it has treated the first patient in the higher dose cohort in its 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. Treatment of the higher dose cohort comes after successful completion of the low dose cohort and clearance from the U.S. Food and Drug Administration (FDA) and Independent Data Safety Monitoring Committee (IDSMC). The second cohort of the Phase 1 study evaluates RP-A501 at a higher dose level of 1.1×1014 genome copies/kilogram in male patients 15 years of age and older. The Phase 1 study will assess the safety, tolerability and preliminary efficacy of RP-A501.

“Today marks an important milestone in the development of RP-A501 for Danon Disease, the first investigational gene therapy in development for the treatment of inherited heart failure,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “RP-A501 represents a potentially holistic and transformative approach to Danon Disease, a devastating disease often resulting in end-stage heart failure and early mortality. The only definitive treatment option available for this multisystemic disorder is heart transplantation, which is associated with a 50% mortality rate during the decade following transplant and does not address the full spectrum of the disease course. Our gene therapy approach, utilizing RP-A501, aims to address all manifestations associated with Danon Disease including cardiomyopathy, skeletal myopathy and intellectual disability. We look forward to progressing this trial and enabling a potentially curative treatment option for this devastating disease that affects so many lives.”

The non-randomized, open-label Phase 1 trial aims to enroll both pediatric and young adult male patients in escalating dose cohorts. Following the review of safety data from the first cohort, all subsequent cohorts will include 2-4 patients per cohort, adjusted down from 3-6 patients in the original protocol. The study is designed to assess the safety and tolerability of a single intravenous (IV) 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 cardiac imaging and functional cardiopulmonary testing.