Phase 2 study of Mydicar for the treatment of advanced heart failure

Celladon announced that its Phase 2 trial with Mydicar for the treatment of advanced heart failure, has met its prospectively defined safety and efficacy endpoints. This trial, also known as the CUPID (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) trial, is a randomized, double-blind, placebo-controlled study to assess the efficacy and safety of Mydicar. Patients eligible for enrollment into the CUPID trial had severe forms of heart failure with New York Heart Association Class III or IV heart failure, significantly impaired pumping function of their hearts, and less than half the normal ability to transport and utilize oxygen during exercise testing.

The primary efficacy endpoint is a composite of outcome measures including worsening of heart failure leading to hospitalization, frequency of and time to cardiac transplantation or left ventricular assist device implantation, changes in patients’ ability to exercise, echocardiographic assessments, symptoms of heart failure, and a blood test for NT-proBNP (an important marker of heart failure).

Mydicar is a genetically targeted enzyme replacement therapy intended to restore levels of SERCA2a, a regulator of calcium cycling and contractility. SERCA2a levels decline in all forms of late-stage heart failure resulting in deficient heart function. Mydicar delivers the SERCA2a gene using a recombinant adeno-associated viral vector (AAV), a naturally occurring virus that is not associated with any disease in humans.

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