{"id":61265,"date":"2025-05-15T22:02:16","date_gmt":"2025-05-15T20:02:16","guid":{"rendered":"https:\/\/pharma-trend.com\/en\/rocket-pharmaceuticals-presents-preliminary-data-from-phase-1-clinical-trial-of-rp-a601-for-pkp2-arrhythmogenic-cardiomyopathy-at-28th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\/"},"modified":"2025-05-15T22:02:16","modified_gmt":"2025-05-15T20:02:16","slug":"rocket-pharmaceuticals-presents-preliminary-data-from-phase-1-clinical-trial-of-rp-a601-for-pkp2-arrhythmogenic-cardiomyopathy-at-28th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy","status":"publish","type":"post","link":"https:\/\/pharma-trend.com\/en\/rocket-pharmaceuticals-presents-preliminary-data-from-phase-1-clinical-trial-of-rp-a601-for-pkp2-arrhythmogenic-cardiomyopathy-at-28th-annual-meeting-of-the-american-society-of-gene-and-cell-therapy\/","title":{"rendered":"Rocket Pharmaceuticals Presents Preliminary Data from Phase 1 Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy at 28th Annual Meeting of the American Society of Gene and Cell Therapy"},"content":{"rendered":"
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\nRP-A601 was generally well-tolerated at a dose of 8.0E13 GC\/kg with no dose-limiting toxicities in all three patients with up to 12 months follow-up<\/i>\n<\/p>\n

\nRP-A601 promoted increased protein expression and desmosomal localization of Plakophilin-2 (PKP2), Desmocollin-2, and Cadherin-2 in all three patients<\/i>\n<\/p>\n

\nImprovement or stabilization observed in arrhythmia burden, heart function, and quality of life in all patients<\/i>\n<\/p>\n

\nNo further dose escalation planned<\/i>\n<\/p>\n

\nInvestor webinar to be held later today at 4:30 p.m. ET<\/i>\n<\/p>\n

CRANBURY, N.J.–(BUSINESS WIRE)–Rocket Pharmaceuticals, Inc.<\/a> (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced preliminary data from the Phase 1 clinical trial of RP-A601 for the treatment of plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM). RP-A601 showed a well-tolerated safety profile with no dose-limiting toxicities, increased PKP2 protein expression, and preliminary indications of improvement or stabilization in arrhythmia burden, heart function, and quality of life in all three patients followed for up to 12 months. Results were presented today as a late-breaking oral presentation at the Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) and will be discussed on a company webinar today at 4:30 p.m. ET.\n<\/p>\n

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\n\u201cPreliminary data from the Phase 1 study of RP-A601 for PKP2-ACM are highly encouraging, signaling potential clinical benefit along with a generally well-tolerated safety profile,\u201d said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharma. \u201cThese initial results represent the second gene therapy from our AAV cardiovascular portfolio to show positive clinical data, propelling us one step closer towards our mission of delivering potentially curative treatments to patients with rare and devastating heart conditions.\u201d\n<\/p>\n

\nThe preliminary safety and efficacy of RP-A601 were evaluated in a single-arm, open-label, multi-center Phase 1 study in three patients with PKP2-ACM who received a dose of 8×1013<\/sup> GC\/kg. Initial data from the Phase 1 study (safety cut-off May 6, 2025; efficacy cut-off April 2025) showed that RP-A601 was generally well-tolerated with no dose-limiting toxicities observed in all patients followed for up to 12 months. Most treatment emergent adverse events were mild\/moderate in severity and self-limited with only one patient experiencing severe adverse events which resolved without clinical sequelae within two months post-treatment, believed to be associated with the immunomodulatory regimen.\n<\/p>\n

\nCardiac biopsies showed RP-A601 increased PKP2 protein expression in all three patients. In the patients with low baseline PKP2 expression (N=2), improvements in PKP2 protein expression relative to total cell protein were approximately 110% and 398%, respectively, from baseline to six months follow-up. In addition, RP-A601 increased protein expression and promoted desmosomal localization of PKP2, Desmocollin-2, and Cadherin-2 in all three patients.\n<\/p>\n

\nPreliminary indications of improvement or stabilization were observed in arrhythmia burden, heart function, and quality of life. Patients in the Phase 1 trial also demonstrated:\n<\/p>\n