Business
Rocket Pharmaceuticals Reports Positive Long-Term Clinical Data from RP-L201 Trial for the Treatment of Leukocyte Adhesion Deficiency-I at the Clinical Immunology Society 2021 Annual Meeting
— New Long-Term Follow-Up Data Demonstrate Durable CD18 Expression, Improved Skin Lesions, and Consistent Peripheral Vector Copy Numbers Up to 18-Months
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n— New Long-Term Follow-Up Data Demonstrate Durable CD18 Expression, Improved Skin Lesions, and Consistent Peripheral Vector Copy Numbers Up to 18-Months Post-Treatment —\n\n— Second Patient Nearing One-Year Primary Endpoint for Phase 2 Portion of Study —\n\n— Two Additional Patients with Shorter Follow-Up Demonstrate Evidence of Engraftment —\n\n— RP-L201 was Well Tolerated in All Patients —\n\n— More Comprehensive Phase 2 Results Expected in Second Half of 2021 —\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces positive interim data from the Company’s Phase 1/2 clinical trial studying RP-L201, its lentiviral-based gene therapy for the treatment of severe Leukocyte Adhesion Deficiency-I (LAD-I). Severe LAD-I is a rare pediatric disease that prevents patients from adequately combating infections. LAD-I leads to recurrent life-threatening bacterial and fungal infections that respond poorly to antibiotics, require frequent hospitalizations and are ultimately fatal. These results were presented in a virtual poster session at the Clinical Immunology Society (CIS) 2021 Annual Meeting.\n\n“Today’s positive updates on our LAD-I program add to the growing body of encouraging evidence that RP-L201 may provide durable clinical benefit for patients with severe LAD-I who face recurrent, life-threatening infections from birth,” said Jonathan Schwartz, M.D., Chief Medical Officer and Senior Vice President of Rocket. “We are very pleased to report that a second patient is nearing survival at one-year post-treatment, the primary outcome measure for the Phase 2 portion of the study. In all patients treated, CD18 expression has substantially exceeded the 4-10% threshold associated with survival into adulthood, with consistent peripheral blood vector copy number levels. Improved disease-related skin lesions, absence of new infections post-treatment, and no further requirements for prophylactic anti-infectives were also observed in both Phase 1 patients with prolonged follow-up. Initial evidence of engraftment and phenotypic correction was observed in two additional patients with shorter follow-up. These updates move us one step closer towards BLA/MAA filings in the US and E...