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Rocket Pharmaceuticals Announces Positive Updates from Phase 1 Clinical Trial of RP-A501 in Danon Disease
—Results demonstrate sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up— — NYHA class
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\n—Results demonstrate sustained benefit across clinical, functional and biomarker endpoints in all four patients with long-term follow up—\n\n— NYHA class improvement (from II to I) in all three patients (two low-dose, one high-dose) with closely monitored immunosuppressive regimen— \n\n—Decreased cardiac wall thickness with improved or stabilized ejection fraction on echocardiogram in all three patients with closely monitored immunosuppressive regimen— \n\n— Sustained improvement or stabilization in BNP and 6-minute walk test across four patients—\n\n—Sustained cardiac LAMP2B expression greater than 50% by immunohistochemistry and improved cardiac tissue architecture in all three patients with closely monitored immunosuppressive regimen—\n\n—RP-A501 generally well tolerated at low-dose with manageable safety profile—\n\n —Webcast to be held at 8:30 a.m. ET today—\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 updated data from the Phase 1 clinical trial evaluating a single intravenous infusion of RP-A501, the Company’s investigational gene therapy, for the treatment of Danon Disease. This update, which includes interim safety and efficacy data from patients in the low-dose (6.7e13 vg/kg; n=3) and high-dose (1.1e14 vg/kg; n=2) adult and adolescent cohorts, demonstrates RP-A501 was generally well tolerated at the low-dose and conferred sustained clinical benefit.\n\n“We are excited to announce positive data from our RP-A501 trial for Danon Disease showing clinical, functional and biomarker improvements at one year or beyond and potential early separation from the natural course of disease in adult and adolescent patients,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. “Male patients living with Danon Disease suffer a heavy disease burden and face rapidly progressive heart failure in their teenage years. In this setting, even stabilization is highly beneficial, and clinical improvements as seen in our trial could be transformative and represent a step forward for the treatment of monogenic heart failure and gene therapy for cardiac diseases. We have initiated treatment in the pediatric cohort at the low-dose and anticipate moving as ra...