Rocket Pharmaceuticals has made significant strides in gene therapy with its RP-A601, which recently became the first treatment for plakophilin-2 related arrhythmogenic cardiomyopathy (PKP2-ACM) to receive FDA clearance.

This innovative therapy aims to address a critical treatment gap for PKP2-ACM, a condition known for causing life-threatening ventricular arrhythmias and cardiac death.

At the ASGCT conference in New Orleans, early phase 1 data revealed that RP-A601 is well-tolerated and shows potential efficacy in improving heart function for patients with PKP2-ACM.

All three patients in the trial demonstrated normal right ventricular systolic function at their latest follow-up, indicating overall improvements in heart function.

The trial results suggest that RP-A601 may improve or stabilize heart function, with patients experiencing a notable reduction in arrhythmia burden.

Cardiac biopsies from the patients showed increased PKP2 protein expression, with one patient exhibiting a remarkable 398% increase from baseline.

Patients tracked for at least six months reported significant improvements in their Kansas City Cardiomyopathy Questionnaire (KCCQ)-12 scores, along with a shift from New York Heart Association (NYHA) class II to class I.

Notably, one patient reduced episodes of nonsustained ventricular tachycardias from five to zero per day, showcasing the therapy's potential impact.

Despite elevated liver enzyme levels observed in all patients, most adverse events were mild to moderate and resolved without intervention.

However, analysts have advised caution regarding immunosuppression strategies due to concerns raised by a recent patient death linked to a similar gene therapy.

Rocket Pharmaceuticals has opted against future dose escalation, focusing instead on the promising activity observed at the current dose and planning for a pivotal trial.

CEO Gaurav Shah described the preliminary findings as highly encouraging, suggesting that RP-A601 could offer substantial clinical benefits alongside its well-tolerated safety profile.