Rocket Pharmaceuticals (NASDAQ:RCKT) reported data from several gene therapy programs at the 26th Annual Meeting of the American Society of Gene and Cell Therapy in Los Angeles.
Pyruvate Kinase Deficiency (PKD)
The company reported data from two two adult patients followed up to 30 months and early data from the first pediatric patient treated with ex vivo lentiviral gene therapy RP-L301 for PKD — a genetic blood disorder in which there are low levels of an enzyme called pyruvate kinase, which results in red blood cells breaking down easily.
Rocket said robust and sustained efficacy in both adults showed normalized hemoglobin (from baseline levels in the 7.0-7.5 g/dL range), improved hemolysis parameters, and red blood cell transfusion independence. Improved quality of life was also seen.
The safety profile appeared to be favorable, with no RP-L301-related serious adverse events, the company noted.
In addition, Rocket said the first pediatric results suggest similar efficacy as seen in the adult group.
The first pediatric patient infusion of RP-L301 was well tolerated, and engraftment was achieved at day +15. Hemoglobin normalized six weeks after-infusion and measured 13.4 g/dL at 8 weeks (from median baseline of 7.9 g/dL).
There were no red blood cell transfusion requirements after engraftment, according to the company.
Fanconi Anemia (FA)
The company reported ongoing data from a phase 2 trial of its ex vivo lentiviral gene therapy RP-L102 for FA.
Rocket said RP-L102, provided sustained genetic correction in 8 of 12 evaluable patients and comprehensive phenotypic correction in 7 of 12 evaluable patients with ≥12 months of follow up as seen by increased resistance to mitomycin-C (MMC) in bone marrow (BM)-derived colony forming cells and hematologic stabilization.
The company added that the safety profile of RP-L102 was highly favorable and no signs of bone marrow dysplasia, clonal dominance or insertional mutagenesis related to RP-L102 were signs.
Rocket expects to file an application to FDA in Q4 2023 seeking approval of the therapy.
Leukocyte Adhesion Deficiency-I (LAD-I)
Rocket presented data from an ongoing Phase 1/2 study of ex vivo lentiviral gene therapy RP-L201 in nine patients with LAD-I — an immunodeficiency disorder affecting the immune system due to inability of leukocytes to migrate to the site of infection to kill bacteria and other harmful entities. This leads to recurrent infections.
The company said 100% overall survival was seen at 12 months post-infusion for all 9 patients with 12 to 24 months of follow-up. The data also showed resolution of LAD-I-related skin rash and restoration of wound repair capabilities.
The safety profile of RP-L201 was favorable and there were no RP-L201-related serious adverse events.
Adverse events related to other trial procedures, including busulfan conditioning, were previously disclosed and were consistent with the safety profiles of the procedures, the company added.
Rocket expects to submit as biologics license application (BLA) to the FDA in Q2 2023.
Danon Disease (DD)
The company also reported previously disclosed data (cut-off July 11, 2022) ) in patients followed for up to 3 years in an ongoing phase 1 trial of AAV gene therapy RP-A501 for DD — a rare inherited disorder which affects the heart, skeletal muscles, and retina, with overlying cognitive dysfunction.
Rocket said that as previously disclosed, RP-A501 was had favorable safety at the low dose with an appropriate immunomodulatory regimen.
Efficacy results continue to show sustained improvement or stabilization in all patients with preserved left ventricular systolic function at time of therapy (n=6, across all groups), the company added.
PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM)
In addition, Rocket said robust preclinical proof of concept studies showed RP-A601 for PKP2-ACM decreased arrhythmias and increased survival.
RCKT +2.40% to $21.80 premarket May 19