Rocket Pharma Shares Positive LV Hematology Data at ASGCT Annual Meeting

Rocket Pharmaceuticals, Inc., a late-stage biotechnology company, recently presented extended data updates from its lentiviral (LV) vector hematology portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The updates affirmed the persistent safety and efficacy of their Phase 1/2 pivotal studies for KRESLADITM (marnetegragene autotemcel) for severe Leukocyte Adhesion Deficiency-I (LAD-I) and RP-L102 for Fanconi Anemia (FA), along with the Phase 1 study of RP-L301 for Pyruvate Kinase Deficiency (PKD).

Dr. Jonathan Schwartz, Chief Medical & Gene Therapy Officer, emphasized the positive updates, highlighting sustained safety and efficacy across their LV hematology portfolio. KRESLADITM continues to show 100% survival without the need for allogeneic hematopoietic stem cell transplantation (HSCT) and significant reductions in infection-related hospitalizations for patients with severe LAD-I. In the RP-L102 study for FA, genetic and phenotypic corrections have been maintained alongside hematologic stability. For the RP-L301 study in PKD, patients exhibited clinically meaningful hemoglobin improvement, all in a well-tolerated manner with no serious adverse events linked to the drug.

The data presented from the KRESLADITM study (cut-off July 24, 2023) showed sustained efficacy and safety from 18 to 45 months of follow-up in nine patients with severe LAD-I. All patients survived without HSCT for at least 18 months post-infusion, with those enrolled under 12 months of age surpassing 24 months without needing HSCT. The study met all primary and secondary endpoints, demonstrating sustained genetic and phenotypic correction. Significant reductions in infections requiring hospitalization or intravenous antimicrobials were noted, alongside improvements in LAD-I-related skin and periodontal lesions, and enhanced wound healing. No serious adverse events tied to the drug have been reported.

The RP-L102 study for Fanconi Anemia (cut-off September 11, 2023) revealed sustained and progressively increasing genetic correction in eight out of 12 patients with follow-ups beyond 12 months. Genetic correction corresponded with phenotypic correction and hematologic stability. The therapy demonstrated polyclonal insertion patterns indicative of long-term hematopoietic stem cell repopulation and clonal diversity without conditioning. The European Medicines Agency (EMA) has accepted the Marketing Authorization Application (MAA) for review, and Rocket plans to submit a Biologics License Application (BLA) to the FDA in the first half of 2024.

For RP-L301 in PKD, data (cut-off February 5, 2024) from the Phase 1 study included two adult patients followed up to 36 months and two pediatric patients followed up to 12 months. All patients showed sustained and meaningful hemoglobin improvement, with three out of the four patients achieving hemoglobin normalization. None required red blood cell transfusions post-neutrophil engraftment. Observations included improved markers of hemolysis and quality of life. The treatment was well-tolerated, with no serious drug-related adverse events. Insertion site analyses showed highly polyclonal patterns without clonal dominance or mutagenesis in adult patients, with ongoing testing for pediatric patients.

KRESLADITM, RP-L102, and RP-L301, all investigational gene therapies, show promising results for their respective conditions, addressing significant unmet medical needs. Rocket Pharmaceuticals continues to advance their genetic therapy pipeline, aiming for transformative options for patients with rare disorders.

KRESLADITM, developed with autologous hematopoietic stem cells modified to deliver the ITGB2 gene, aims to address severe LAD-I. RP-L102, targeting the FANCA gene for FA, shows potential to prevent bone marrow failure without the need for a donor or the toxicities associated with transplants. RP-L301, containing the PKLR gene, targets energy production in red blood cells to treat PKD, a disorder leading to severe anemia and quality-of-life impacts. Rocket’s work in these areas is supported by various regulatory designations and collaborations, aiming for long-term clinical benefits and safety for patients.