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Rocket Pharmaceuticals Highlights Presentation Of LAD-l Clinical Update And Comprehensive IMO Preclinical Review At European Society For Immunodeficiencies 2020 Meeting

—Longer-Term Data from the Registration-Enabling Phase 1/2 Trial for Leukocyte Adhesion Deficiency-I Demonstrate Safety and Efficacy of RP-L201— —Preclinical Data From RP-L401 for Infantile Malignant

· 10/16/2020 07:04

—Longer-Term Data from the Registration-Enabling Phase 1/2 Trial for Leukocyte Adhesion Deficiency-I Demonstrate Safety and Efficacy of RP-L201—

—Preclinical Data From RP-L401 for Infantile Malignant Osteopetrosis Support Accelerated Clinical Development—

—Results Provide Further Validation for Rocket's "Process B" Lentiviral Platform—

Rocket Pharmaceuticals, Inc. (NASDAQ:RCKT) ("Rocket"), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces clinical data at the European Society for Immunodeficiencies (ESID) 2020 Meeting being held virtually October 14-17, 2020. An oral presentation provides positive longer-term follow-up data from the Phase 1/2 clinical trial of RP-L201 for Leukocyte Adhesion Deficiency-I (LAD-I). An e-poster highlights preclinical study data on RP-L401 for Infantile Malignant Osteopetrosis (IMO) supporting clinical development of the trial.

"Today, Rocket presents positive results from our LAD-I gene therapy trial demonstrating further clinical benefit in this severely affected patient population," said Jonathan Schwartz, M.D. Chief Medical Officer and Senior Vice President of Rocket. "Patients with LAD-I have markedly diminished expression of the integrin CD18 and suffer from life-threatening bacterial and fungal infections. Natural history studies indicate that an increase in CD18 expression to 4-10% is associated with survival into adulthood. The two patients enrolled in our Phase 1 trial demonstrated restored CD18 expression substantially exceeding this threshold. In addition, we continue to observe a durable treatment effect in the patient followed through one year, with improvement of multiple disease-related skin lesions after therapy and no further requirements for prophylactic anti-infectives."

Dr. Schwartz continued, "In addition, preclinical results in Infantile Malignant Osteopetrosis represent an early positive signal of in vivo efficacy that support evaluation of RP-L401 in a Phase 1 trial. IMO is a devastating bone resorption disorder resulting in skeletal deformities, neurologic abnormalities and bone marrow failure. Rocket has developed RP-L401 as a potential treatment option to prevent the devastating morbidity and childhood mortality associated with IMO. Preclinical data indicate that even a modest level of engraftment can correct the disease phenotype, with increased long-term survival, growth, and normalized bone and tooth development."

Preliminary Data Highlights from Rocket's Phase 1/2 Study of RP-L201 in LAD-I

The data presented in the oral presentation are from two pediatric patients with severe LAD-I, as defined by CD18 expression of less than 2%. Both patients were treated with RP-L201, Rocket's ex vivo lentiviral gene therapy candidate. Patient L201-003-1001 was 9-years of age at treatment and has been followed for 12-months and Patient L201-003-1004 was 3-years of age at treatment and has been followed for four months. Key highlights from the presentation include:

  • RP-L201 was well tolerated, and no safety issues were reported with infusion or post-treatment
  • Both subjects achieved hematopoietic reconstitution in less than 4 weeks
  • Peripheral blood vector copy number (VCN) and neutrophil CD18-expression were assessed post-treatment to evaluate engraftment and phenotypic correction:
    • Patient L201-003-1001 demonstrated durable CD18 expression of 40%, peripheral blood VCN levels of 1.0, visible signs of improvement in existing skin lesions and no new infections, as reported 12 months post-treatment
    • Patient L201-003-1004 demonstrated CD18 expression of 28% and early peripheral blood VCN trending similarly to first patient
  • As previously reported, the drug product VCN for patient L201-003-1001 was 3.8 with a CD34+ cell dose of 4.2 x 106 cells/kilogram (kg)
  • Patient L201-003-1004's CD34+ cell dose was 2.8 x 10 6 cells/kg. The drug product VCN was 2.5.

A copy of the oral presentation and e-poster can be accessed by visiting: https://www.rocketpharma.com/ESID/