PKP2-ACM is a devastating inherited heart disease. Credit: Tumisu from Pixabay.

Rocket Pharmaceuticals’ investigational new drug (IND) application for a Phase I clinical trial of RP-A601, an AAV.rh74-based gene therapy candidate to treat arrhythmogenic cardiomyopathy due to plakophilin 2 pathogenic variants (PKP2-ACM), has received clearance from the US Food and Drug Administration (FDA).

The dose escalation, multi-centre trial will enrol at least six adult subjects with PKP2-ACM.

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Patients with implantable cardioverter defibrillators (ICDs) and who are at high risk for arrhythmias will receive 8 x 10¹³ GC/kg single dose of RP-A601 having rh74 serotype.

The study intends to assess the safety and preliminary efficacy of RP-A601 along with its impact on cardiac biomarkers and clinical predictors of ventricular arrhythmias.

Furthermore, the impact of RP-A601 on PKP2 myocardial protein expression and sudden cardiac death will be studied in the trial.

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Rocket Pharma president and chief operating officer Kinnari Patel said: “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 the disease.

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“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.”

An estimated 50,000 people in the US and EU are affected with PKP2-ACM, a devastating inherited heart disease.