Business
Rocket Pharmaceuticals Presents Positive Clinical Data from Danon Disease, Fanconi Anemia and Pyruvate Kinase Deficiency Programs at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)
— RP-A501 for Danon Disease well-tolerated in two pediatric patients with no complement-mediated toxicities reported; efficacy update remains on track for Q3
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n— RP-A501 for Danon Disease well-tolerated in two pediatric patients with no complement-mediated toxicities reported; efficacy update remains on track for Q3 —\n\n— Sustained genetic correction observed in six of nine evaluable Fanconi Anemia patients at least 12 months after RP-L102 treatment; five reached target MMC resistance at two time points with concomitant hematologic stabilization —\n\n— Robust and sustained efficacy in both Pyruvate Kinase Deficiency patients at 18 months post-RP-L301 infusion demonstrated by normalized hemoglobin and improved hemolysis parameters —\n\n— Top-line readout from Phase 2 pivotal trial of RP-L201 for Leukocyte Adhesion Deficiency-I expected on Thursday, May 19 —\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 clinical updates from its Phase 2 pivotal trial for Fanconi Anemia (FA) and Phase 1 trials for Danon Disease and Pyruvate Kinase Deficiency (PKD) at the 25th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT).\n\n“We are highly encouraged by the initial safety data from the two pediatric patients in the Phase 1 Danon Disease trial that suggest RP-A501 was well-tolerated,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “These two patients had markedly reduced complement activation and no complement-related adverse events. They received preventative therapy with a modified immunosuppressive regimen. We believe today’s results represent an important step forward in optimizing the safety of AAV9-based gene therapy for Danon Disease and the Danon patient community. Updated data are anticipated in the third quarter, and we believe that the totality of data from seven patients treated in Phase 1 will support a rapid advance toward a Phase 2 pivotal study.”\n\n“In our Phase 2 pivotal study in Fanconi Anemia, five of nine patients sustained increasing bone marrow cell resistance to mitomycin-C (MMC) confirmed over two consecutive timepoints,” said Dr. Shah. “In these patients, MMC-resistance increased to 51%-94% at 18 to 21 months, up from 21%-42% at 12 to 18 months. The increase in MMC resistance was accompanied by concom...