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Rocket Pharmaceuticals Announces FDA Clearance of IND for Clinical Trial of RP-A601 for PKP2 Arrhythmogenic Cardiomyopathy (ACM)
First clinical gene therapy program for PKP2-ACM, a devastating inherited heart disease affecting approximately 50,000 people in the U.S. and EU Robust
About this update from Rocket Pharmaceuticals, Inc.
[{"type":"text","content":"\nFirst clinical gene therapy program for PKP2-ACM, a devastating inherited heart disease affecting approximately 50,000 people in the U.S. and EU\n\n\nRobust preclinical proof of concept studies showed RP-A601 decreased arrhythmias and increased survival in the PKP2 knockout mouse model\n\n\nInitiating Phase 1 trial start-up activities and rapidly working towards first patient treatment\n\n\n CRANBURY, N.J.--(BUSINESS WIRE)--\nRocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that it has received clearance from the U.S. Food and Drug Administration (FDA) for the Company’s Investigational New Drug (IND) application for RP-A601, an AAV.rh74-based gene therapy candidate for the treatment of arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM) a devastating inherited heart disease that can lead to life-threatening arrhythmias, cardiac structural abnormalities, and sudden cardiac death. The current standard of care for patients with PKP2-ACM consists of medical therapy, implantable cardioverter defibrillators (ICDs), and ablations, which are not curative. Even with treatment, life-threatening arrhythmias and progression of disease may still occur. PKP2-ACM affects approximately 50,000 people in the U.S. and Europe.\n\n\n“Today’s news is a significant milestone, as we build on our leading cardiovascular gene therapy expertise to advance a historic second program targeting the heart into clinical development, this time for patients with PKP2-ACM who have an urgent need for improved treatment options,” said Kinnari Patel, PharmD, MBA, President and Chief Operating Officer, Rocket Pharma. “RP-A601 offers the potential of a one-time, curative alternative to medical therapy, ICDs, and ablations which are associated with adverse effects, complications, and recurrence of arrhythmias and do not halt the progression of disease. Robust preclinical proof of concept has demonstrated decreased arrhythmias and increased survival. With IND clearance in hand, we are rapidly advancing the first investigational gene therapy for PKP2-ACM into the clinic.”\n\n\nThe multi-center Phase 1, dose escalation trial will evaluate the safety and preliminary efficacy of RP...