23 May 2023 - - US-based biotechnology company Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) has released positive data from the Pyruvate Kinase Deficiency, Fanconi Anemia, Severe Leukocyte Adhesion Deficiency-I (LAD-I) and Danon Disease clinical programs and PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM) preclinical program presented at the 26th annual meeting of the American Society of Gene and Cell Therapy (ASGCT) in Los Angeles, California, the company said.

Global Phase 1 Study Results of Lentiviral Mediated Gene Therapy for Severe Pyruvate Kinase Deficiency

The oral presentation includes positive updated data (cut-off May 3, 2023) from two adult patients followed up to 30 months and encouraging early data from the first pediatric patient treated with RP-L301, Rocket's ex vivo lentiviral gene therapy candidate for Pyruvate Kinase Deficiency.

Robust and sustained efficacy in both adult patients up to 30 months post-infusion demonstrated by normalized hemoglobin (from baseline levels in the 7.0-7.5 g/dL range), improved hemolysis parameters, and red blood cell transfusion independence.

Both adult patients reported improved quality of life with documented improvements via formal quality of life assessments.

The safety profile appears highly favorable, with no RP-L301-related serious adverse events in either of the adult patients. Previously reported transient transaminase elevation seen in both adult subjects post conditioning and infusion with no clinical stigmata of liver injury have fully resolved.

Insertion site analyses in peripheral blood and bone marrow in both adult patients through 24 months post-RP-L301 demonstrated highly polyclonal patterns and there has been no evidence of insertional mutagenesis.

First pediatric results suggest similar efficacy as observed in long-term efficacy data in the adult cohort.

The first pediatric patient infusion of RP-L301 was well tolerated, with engraftment achieved at day +15, hospital discharge less than one month following infusion, and no RP-L301-related serious adverse events.

Hemoglobin normalized six weeks post-infusion and measured 13.4 g/dL at eight weeks (from median baseline of 7.9 g/dL). There were no red blood cell transfusion requirements following engraftment.

Adult and pediatric enrollment is completed in the Phase 1 study. Phase 2 pivotal trial initiation is anticipated in the fourth quarter of 2023.

Lentiviral-Mediated Gene Therapy for Fanconi Anemia [Group A]: Results from Global RP-L102 Clinical Trials

The oral presentation includes positive, updated data (cut-off April 17, 2023) from the ongoing Phase 2 pivotal trial of RP-L102, Rocket's ex vivo lentiviral gene therapy candidate for Fanconi Anemia.

RP-L102 conferred sustained genetic correction in eight of 12 evaluable patients and comprehensive phenotypic correction in seven of 12 evaluable patients with ≥12 months of follow up as demonstrated by increased resistance to mitomycin-C in bone marrow -derived colony forming cells and hematologic stabilization.

The safety profile of RP-L102 remains highly favorable with no significant safety signals, and the treatment, administered without any cytotoxic conditioning, continues to be well tolerated. No signs of bone marrow dysplasia, clonal dominance or insertional mutagenesis related to RP-L102 have been observed.

Polyclonal integration patterns have been observed in each of the seven patients with phenotypic, genetic, and hematologic evidence of engraftment.

Pivotal trial enrollment and treatment have been completed, and the final two patients have been treated with commercial drug product in preparation for launch.

Based on the positive efficacy and safety data from the Phase 2 pivotal FA trial, Rocket anticipates filing the Biologics License Application with the FDA in the fourth quarter of 2023.