Rocket Pharmaceuticals reports Q3 EPS (75c), consensus (81c)

As of September 30, 2023, Rocket had cash, cash equivalents and investments of $437.2 million. Rocket expects such resources will be sufficient to fund its operations through 2025, including producing AAV cGMP batches at the Company’s Cranbury, N.J. R&D and manufacturing facility and continued development of its six clinical and/or preclinical programs. “I’m very pleased with Rocket’s results this quarter, as we realized significant regulatory milestones across our pipeline, including reaching FDA alignment on the study design for the 10-patient Phase 2 pivotal trial of RP-L301 for the treatment of severe PKD and securing the FDA’s acceptance of Rocket’s BLA for RP-L201, now named KRESLADITM, for the treatment of severe LAD-I,” said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “At the same time, we initiated the Phase 2 pivotal trial of RP-A501 for Danon Disease following FDA alignment and the Phase 1 study of RP-A601 for PKP2-ACM, which move us substantially closer to delivering potential one-time, curative therapies to patients facing these two devastating cardiac diseases. As we bring 2023 to a close, we have made remarkable progress across the breadth of our AAV cardiovascular and LV hematology portfolios. Four programs have now reached pivotal studies, one of which is under review for approval, thanks to strong collaboration with health authorities and scientific partners, as we collectively seek to fulfill the high unmet needs of patients with rare and life-threatening diseases.”