Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Rocket 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 announced longer-term data updates from its lentiviral (LV) vector hematology portfolio presented at the 27^th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). Data updates demonstrate the continued safety and efficacy of the Phase 1/2 pivotal studies of KRESLADI (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) and RP-L102 for Fanconi Anemia (FA), in addition to the Phase 1 study of RP-L301 for Pyruvate Kinase Deficiency (PKD).

“The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals. “Ahead of the upcoming PDUFA date, KRESLADI^TM continues to demonstrate 100% HSCT-free survival and significant reductions in infection-related hospitalizations following engraftment in patients with severe LAD-I. In our pivotal studies of RP-L102 for Fanconi Anemia, we continue to see maintained genetic and phenotypic correction combined with hematologic stabilization. Additionally, our Phase 1 study of RP-L301 for PKD shows sustained clinically meaningful hemoglobin improvement in all patients. We are very pleased with the safety profile demonstrated across our LV hematology portfolio, with no drug-related serious adverse events observed to date.”

Autologous Ex-Vivo Lentiviral Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I Provides Sustained Efficacy with a Well-Tolerated Safety Profile

The oral presentation includes positive, updated data (cut-off July 24, 2023) from the global Phase 1/2 pivotal studies demonstrating sustained efficacy and safety of KRESLADI^TM from 18 to 45 months of follow-up for all nine patients with severe LAD-I.

• Observed 100% survival in the absence of allogeneic HSCT at least 18 months post-infusion in all nine patients; all patients enrolled at less than 12 months of age have surpassed 24 months without HSCT. All primary and secondary endpoints were met, including sustained genetic and phenotypic correction.
• When compared with pre-treatment history, data showed substantial decreases in the incidences of significant infections requiring hospitalization or intravenous antimicrobials, combined with evidence of resolution of LAD-I-related skin and periodontal lesions and restoration of wound repair capabilities.
• KRESLADI^TM was well-tolerated in all patients with no drug-related serious adverse events reported to date. Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and are consistent with the safety profiles of those agents and procedures. No cases of graft failure or autologous graft-versus-host-disease (GvHD) were reported.
• Based on the positive efficacy and safety data from the global pivotal studies of KRESLADI^TM for severe LAD-I, the Biologics License Application (BLA) was accepted for review by the U.S. Food and Drug Administration (FDA) who has set the New Prescription Drug User Fee Act (PDUFA) target date of June 30, 2024.

Lentiviral-Mediated Gene Therapy (RP-L102) for Fanconi Anemia [Group A] is Associated with Polyclonal Integration Patterns in the Absence of Conditioning