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Rocket Pharmaceuticals Presents Positive Top-line Data from Severe Leukocyte Adhesion Deficiency-I Program at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
— RP-L201 well-tolerated with durable CD18 expression for all patients in Phase 2 pivotal trial — — 100% overall survival in patients at one year after
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n— RP-L201 well-tolerated with durable CD18 expression for all patients in Phase 2 pivotal trial —\n\n— 100% overall survival in patients at one year after RP-L201 infusion based on Kaplan-Meier estimate —\n\n— All patients showed clinical reversal of disease course, including statistically significant reduction in all-cause hospitalizations and incidence of severe infections —\n\n— Initiating discussions with health authorities on filing plans; regulatory filings anticipated in the first half of 2023 —\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage, clinical biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders with high unmet need, today announces positive top-line safety and efficacy data from its Phase 2 pivotal trial for severe Leukocyte Adhesion Deficiency-I (LAD-I) at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).\n\n“We are excited to present positive top-line safety and efficacy data from our Phase 2 pivotal trial for LAD-I today at ASGCT, representing a significant step forward in the development of RP-L201 for the treatment of LAD-I, one of the most aggressive and highly fatal immunodeficiencies ever characterized. Moreover, they point to the great possibilities lentiviral-based gene therapies can offer for patients with devastating diseases,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “At three to 24 months after RP-L201 infusion, all nine patients sustained stable CD18 expression (median: 56%) with no therapy-related serious adverse events.”\n\nDr. Shah continued, “We are also pleased to report 100% overall survival at 12-months post-infusion via Kaplan Meier estimate and a statistically significant reduction in all hospitalizations, infection- and inflammatory-related hospitalizations and prolonged hospitalizations for all nine LAD-I patients with three to 24 months of available follow-up. Data also shows evidence of resolution of LAD-I-related skin rash and restoration of wound repair capabilities.”\n\n“While allogeneic transplant options are available, they continue to be associated with considerable toxicity and today’s top-line safety and efficacy data point to the potential of RP-L201 to change the treatment paradigm for patients...