Rocket Pharmaceuticals Announces Preliminary Data from Phase 1/2 Trial of RP-L201 for Leukocyte Adhesion Deficiency-I

- Initial Data Demonstrated Robust CD18 Expression and Resolution of Disease-Mediated Lesions in Months After Gene Therapy Treatment -

NEW YORK--(BUSINESS WIRE)-- 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 preliminary data from its Phase 1/2 clinical trial of RP-L201, the Company’s lentiviral vector (LVV)-based gene therapy for the treatment of Leukocyte Adhesion Deficiency-I (LAD-I). LAD-I is a rare immune disorder characterized by low or absent neutrophil CD18 expression, predisposing affected individuals to recurrent and fatal infections in childhood. Data from the first patient treated with RP-L201 demonstrated early evidence of efficacy.

“We believe the preliminary data from this first patient are impressive and demonstrate that RP-L201 has the potential to correct deficient CD18 expression that is the hallmark of LAD-I,” said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “In LAD-I, stem cells are not believed to be compromised by the underlying disorder. Based on this, and an established RP-L201 treatment process that includes a tailored conditioning regimen, we intend to create a standardized and predictable platform for reversing LAD-I. We believe this treatment approach will enable us to reliably correlate drug product to both early and long-term patient outcomes. We look forward to continued evaluation of this first-in-class gene therapy in the ongoing trial and believe that RP-L201 has the potential to be a favorable therapeutic option for this difficult and frequently fatal disease.”

Initial results from the first pediatric patient treated with RP-L201 demonstrate early evidence of safety and potential efficacy. Analyses of peripheral vector copy number (VCN) and CD18-expressing neutrophils were performed through three months after infusion of RP-L201 to evaluate engraftment and phenotypic correction. The patient exhibited early signs of engraftment with myeloid-lineage VCN levels of 1.5 at three months and CD18 expression of 45%, compared to pre-treatment CD18 expressions of