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 that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold on the Company’s Phase 1 clinical trial of RP-A501 for the treatment of Danon Disease, allowing patient enrollment to resume. The hold was removed after the Company addressed the FDA’s requests to modify the trial protocol and other supporting documents with revised guidelines for patient selection and management. The Company has initiated steps to resume the program as soon as possible and expects to commence dosing in the low-dose (6.7e13 vg/kg) pediatric patient cohort in the third quarter.
“We are grateful for the collaboration between the FDA and our team in reaching agreement on protocol updates allowing us to resume patient enrollment in our Danon Disease trial. We look forward to progressing this critical work on behalf of all Danon patients,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “We are moving as quickly as possible to resume dosing and commence treatment this quarter. Additionally, given the activity observed among young adults in our low-dose cohort, in agreement with the FDA, we are now proceeding with the pediatric cohort. This is another important step forward as we believe the pediatric Danon population has the potential to realize the maximum benefit from our Danon Disease gene therapy program. We continue to anticipate reporting updated longer-term data from the low-dose (6.7e13 vg/kg) and higher-dose (1.1e14 vg/kg) young adult cohorts in the fourth quarter.”
Rocket’s Danon Disease program was placed on clinical hold by the FDA in May of 2021 to modify the study protocol and other supporting documents with revised guidelines for patient selection and safety management. No new drug-related safety events were observed in the low-dose or higher-dose young adult cohorts as part of the clinical hold or during the hold.
The non-randomized, open-label Phase 1 trial is designed to enroll both pediatric and young adult male patients 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.
