Business
Rocket Pharmaceuticals Reports Fourth Quarter and Full Year 2022 Financial and Operational Results
Advanced pipeline of four clinical programs across AAV cardiology and LV hematology portfolios delivering strong results for Danon Disease, Fanconi Anemia,
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\nAdvanced pipeline of four clinical programs across AAV cardiology and LV hematology portfolios delivering strong results for Danon Disease, Fanconi Anemia, LAD-I and PKD\n\nExpanded leadership position in AAV cardiac gene therapy, with significant opportunities for value creation across Danon Disease, PKP2-ACM and BAG3-DCM programs\n\nEstablished in-house cGMP manufacturing for AAV, poised to support Danon pivotal trial in Q2’23 and subsequent commercialization\n\nCash, cash equivalents and investments of approximately $400M; expected operational runway through 2024\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 and operational results for the fourth quarter and year ended December 31, 2022.\n\n“Rocket closed 2022 with positive results across four gene therapy programs spanning both our AAV cardiology and LV hematology therapeutic areas in an unprecedented fashion for a gene therapy company, validating our approach to strong science and R&D, carefully selected assets and smart execution. We are thrilled to begin 2023 with the FDA recently granting RMAT designation to RP-A501 for Danon Disease, validating the strength of our results and underscoring RP-A501's potential as a transformative therapy for Danon patients,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma.\n\nDr. Shah continued, “At the same time, we expanded our leadership position in AAV cardiac gene therapy with the recent unveiling of compelling preclinical proof of concept for RP-A601 for the treatment of PKP2 arrhythmogenic cardiomyopathy (PKP2-ACM) and addition of the BAG3-associated dilated cardiomyopathy (DCM) asset following the acquisition of Renovacor. Taken together, these two diseases along with Danon Disease affect more than 100,000 patients in the U.S. and EU. Further, our LV hematology portfolio delivered stellar results as we now advance towards our first regulatory filings for Leukocyte Adhesion Deficiency (LAD-I) in the second quarter of 2023 and Fanconi Anemia (FA) in the fourth quarter of 2023, and subsequent commercialization.”\n\n“I am also pleased that we brought in additional funds of $197.7 million in 2022. We beg...