Rocket Pharmaceuticals presents clinical data from LV-based gene therapy program

Rocket Pharmaceuticals announces positive clinical data from its lentiviral (LV)-based gene therapy programs at the 64th American Society of Hematology Annual Meeting, taking place in New Orleans, Louisiana, from December 10-13. Lentiviral-mediated Gene Therapy for Patients with Fanconi Anemia: : Updated Results from Global RP-L102 Clinical Trials The poster presentation includes positive updated data from the ongoing Phase 2 pivotal trial of RP-L102, Rocket’s ex vivo lentiviral gene therapy candidate for Fanconi Anemia. RP-L102 conferred phenotypic correction in at least six of 10 evaluable patients with greater than or equal to12 months of follow-up as demonstrated by increased resistance to mitomycin-C in bone marrow-derived colony forming cells, concomitant genetic correction and hematologic stabilization. A seventh patient has displayed evidence of progressively increasing genetic correction as demonstrated by peripheral blood and BM vector copy numbers, with recent development of MMC resistance and indicators of hematologic stability after 36 months of follow-up. The primary endpoint has been achieved, based on a trial protocol in which statistical and clinical significance requires a minimum of five patients to attain increased MMC resistance at least 10% above baseline at two or more timepoints and concomitant evidence of genetic correction and clinical stabilization. The safety profile of RP-L102 has been highly favorable, and the treatment, administered without any cytotoxic conditioning, has been well tolerated. No signs of bone marrow dysplasia, clonal dominance or insertional mutagenesis related to RP-L102 have been observed. As previously disclosed, one patient experienced a Grade 2 transient infusion-related reaction, which resolved; one patient with confirmed engraftment developed a T-cell lymphoblastic lymphoma that was conclusively determined by the investigator, sponsor and the independent data monitoring committee to be related to FA ) and unrelated to RP-L102 gene therapy. Based on the positive efficacy and safety data from the Phase 2 pivotal FA trial, Rocket anticipates regulatory filing in the second half of 2023. Lentiviral-mediated Gene Therapy for Adults and Children with Severe Pyruvate Kinase Deficiency: Results from an Ongoing Global Phase 1 Study The poster presentation includes positive updated data from two adult patients with significant anemia who were treated with RP-L301, Rocket’s ex vivo lentiviral gene therapy candidate for Pyruvate Kinase Deficiency. At 24 months post-infusion, both patients have robust and sustained efficacy demonstrated by normalized hemoglobin, improved hemolysis parameters, independence from red blood cell transfusions and improved quality of life both reported anecdotally and as documented via formal quality of life assessments. The safety profile appears highly favorable, with no RP-L301-related serious adverse events through 24 months post-infusion in both adult patients. Insertion site analyses in peripheral blood and bone marrow in both adult patients up to 12 months post-RP-L301 demonstrated highly polyclonal patterns and there has been no evidence of insertional mutagenesis. Adult and pediatric enrollment is completed in the Phase 1 study. Phase 2 pivotal trial initiation is anticipated in 2023. Interim Results from an Ongoing Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I: The poster presentation includes previously disclosed top-line data at three to 24 months of follow-up after RP-L201 infusion for all patients and overall survival data for seven patients at 12 months or longer after infusion. RP-L201 is Rocket’s ex vivo lentiviral gene therapy candidate for severe Leukocyte Adhesion Deficiency-I. Observed 100% overall survival at 12 months post-infusion via Kaplan Meier estimate and a statistically significant reduction in all hospitalizations, infection- and inflammatory-related hospitalizations and prolonged hospitalizations for all nine LAD-I patients with three to 24 months of available follow-up. Data also shows evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities. The safety profile of RP-L201 has been highly favorable in all patients with no RP-L201-related serious adverse events to date. Adverse events related to other study procedures, including busulfan conditioning, have been previously disclosed and consistent with the safety profiles of those agents and procedures. Based on the positive efficacy and safety data from the Phase 2 pivotal LAD-I trial, Rocket has initiated discussions with the FDA and anticipates regulatory filing in the first half of 2023.