Business
Rocket Pharmaceuticals Reports Second Quarter 2023 Financial Results and Highlights Recent Progress
Initiating enrollment of two-patient pediatric safety run-in for pivotal single-arm Phase 2 study of RP-A501 (Danon Disease); finalizing primary composite
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\nInitiating enrollment of two-patient pediatric safety run-in for pivotal single-arm Phase 2 study of RP-A501 (Danon Disease); finalizing primary composite endpoint with FDA to support accelerated approval\n\n\nRP-L201 (LAD-I) BLA submitted to FDA; represents Rocket’s first product filing\n\n\nRP-A601 (PKP2-ACM) advancing towards first patient treatment in Phase 1 study following FDA clearance of IND; received FDA Fast Track and Orphan Drug designations\n\n\nRP-L102 (Fanconi Anemia) U.S. pivotal Phase 2 study completed; product filing on track\n\n\nRP-L301 (PKD) granted EMA PRIME and FDA RMAT designations\n\n\nCash, cash equivalents and investments of approximately $307M; expected operational runway into the first half of 2025\n\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial results for the quarter ending June 30, 2023, and updates from the Company’s key pipeline developments, business operations and upcoming milestones.\n\n\n“The second quarter of 2023 marked a period of strong forward momentum across our cardiovascular AAV and hematology LV portfolios highlighted by the BLA submission for our LAD-I program, Rocket’s first product filing. In addition, we are in advanced discussions with the FDA to finalize the components of the primary composite endpoint for the single-arm, pivotal Phase 2 Danon Disease study. In parallel, we are initiating enrollment in the trial following recent alignment with the FDA on our proposed two-patient pediatric safety run-in,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “We appreciate the strong and positive collaboration with the FDA afforded by our RMAT designation, which we believe will lead to the most optimal global development path forward for the first pivotal gene therapy trial for a cardiac condition. Simultaneously, in our second cardiac program, RP-A601 for the treatment of PKP2-ACM, we are rapidly moving towards first patient treatment following IND clearance and have also received FDA Fast Track and Orphan Drug designations.”\n\n\nDr. Shah continued, “Building on the BLA submission for LAD-I, we remain on track to submit the BLA for our LV-based Fanconi...