Business
Rocket Pharmaceuticals Reports Second Quarter 2024 Financial Results and Highlights Recent Progress
Enrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase 1 study of RP-A601 to treat PKP2
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\nEnrollment of patients ongoing in the pivotal Phase 2 study of RP-A501 for the treatment of Danon disease and the Phase 1 study of RP-A601 to treat PKP2 arrhythmogenic cardiomyopathy\n\n\nWorking toward FDA-approval of KRESLADI for severe LAD-I; Commercial infrastructure and capabilities in place for launch\n\n\nPresented long-term KRESLADI™ follow-up data from the global Phase 1/2 study for severe LAD-I, results from the global Phase 1/2 study of RP-L102 for Fanconi Anemia, and data from the Phase 1 study of RP-L301 for PKD at ASGCT in May\n\n\nCash, cash equivalents and investments of approximately $278.8M; expected operational runway into 2026\n\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending June 30, 2024.\n\n\n“Over the quarter, Rocket has been advancing its clinical pipeline as we progressed our RP-A501 and RP-A601 cardiac programs, targeting Danon disease and PKP2-ACM, and continued to actively enroll patients,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharmaceuticals. “At ASGCT, we shared follow-up data from across our hematology portfolio including 4-year follow-up data for KRESLADI to treat patients with severe LAD-I, demonstrating a 100% survival rate. In parallel, we have been preparing for the anticipated FDA approval of KRESLADI.”\n\n\nRecent Pipeline and Operational Updates\n\n\n\nContinued advancement of Phase 2 pivotal study of RP-A501 for Danon Disease.\n\n\nEnrollment in the Phase 2 pivotal study of RP-A501 to treat Danon Disease is actively progressing. Details of the Phase 2 study can be found at www.ClinicalTrials.gov under NCT identifier NCT06092034.\n\n\n\n\n\n\n\nGranted orphan medicinal product designation from the European Commission (EC) for RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM).\n\n\nIn May, Rocket announced orphan medicinal product designation from the EC for RP-A601 for the treatment of PKP2-ACM. Enrollment in the Phase 1 study is ongoing. Details of the study can be found at www.ClinicalTrials.gov under NCT identifier NCT05885412.\n\n\n\nOrphan medicinal product designation by the EC is available to novel th...