—Follow-up of 12 to 36 Months for FA RP-L102 “Process A” Demonstrates Prolonged Engraftment and Continued Hematologic Correction—
—Sustained Efficacy in LAD-I Provides Initial Proof of Concept for Lentiviral Platform and “Process B” Manufacturing—
—Longer-term “Process B” Data for FA and LAD-I on Track for End of Year—
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 disorders, today presents new clinical data supporting longer-term efficacy and durability of gene therapy for Fanconi Anemia (FA) and Leukocyte Adhesion Deficiency-I (LAD-I) at the 23rd Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) being held virtually May 12-15, 2020. Two oral presentations highlight updates from the company’s Phase 1/2 study of RP-L201 for the treatment of severe LAD-I and the Phase 1/2 study of RP-L102 “Process A” for the treatment of FA.
“The latest additional data from both our LAD-I and FA programs demonstrate sustained engraftment and durable clinical impact,” said Jonathan D. Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “These results further support the viability of gene therapy in LAD-I and FA, disorders in which bone marrow transplant is the primary curative option and is associated with high rates of toxicity.”
“Patients with severe LAD-I have neutrophil CD18 expression of less than 2% of normal, with extremely high mortality in early childhood,” said Dr. Schwartz. “In this first patient treated with RP-L201 using ‘Process B’, an increase from less than 1% to 47% CD18 expression sustained over six months demonstrates that RP-L201 has the potential to correct the neutrophil deficiency that is the hallmark of LAD-I. We are also pleased with the continued visible improvement of multiple disease-related skin lesions. These results lend further support to the applicability of ‘Process B’ across the lentiviral portfolio. The second patient has also recently been treated, and we look forward to completing the Phase 1 portion of the registrational trial for this program.”
Dr. Schwartz continued, “In our FA program, patients followed for a year or more after treatment with RP-L102 ‘Process A’ continue to demonstrate durable engraftment and hematologic correction, without the use of pre-treatment conditioning regimens. All six patients who received minimally adequate drug product and were followed for more than one year display sustained and progressive engraftment. Notably, hemoglobin levels have normalized to baseline in two patients treated. Today’s update not only gives us confidence as we transition to our improved ‘Process B’ drug product, but also supports the potential of gene therapy in the absence of any conditioning regimen as a definitive hematologic treatment for FA. The ability to treat patients without the side effects associated with allogeneic transplant or the use of genotoxic conditioning, and to restore blood cell counts is a major milestone for the FA scientific community.”
Details on Rocket’s oral presentations at ASGCT:
Title: A Phase 1/2 Study of Lentiviral-mediated Ex-vivo Gene Therapy for Pediatric Patients with Severe Leukocyte Adhesion Deficiency-I (LAD-I): Initial Results from the First Treated Patient Session: HSPC Gene Therapies for Blood and Immune Disorders Presenter: Donald B. Kohn, M.D., Professor of Microbiology, Immunology and Molecular Genetics, Pediatrics (Hematology/Oncology), Molecular and Medical Pharmacology, member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and principal investigator of the Phase 1 trial Date: Tuesday May 12, 2020 Time: 4:30 p.m. - 4:45 p.m. EDT
Additional results from the first patient treated with RP-L201 for LAD-I continue to demonstrate evidence of safety and potential efficacy. Analyses of peripheral vector copy number (VCN) and CD18-expressing neutrophils were performed six months post treatment with RP-L201 to evaluate engraftment and phenotypic correction. The patient demonstrated peripheral blood VCN levels of 1.3 and CD18-expression of 47%, which is sustained from the 45% expression observed three months post treatment; pretreatment CD18 expression was