Celladon announced 12 month data from its Phase 2 study of Mydicar for the treatment of advanced heart failure. 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 study of 39 patients met its primary safety and efficacy endpoints at 6 months for high dose Mydicar versus placebo. Additionally, after 12 months of receiving a single infusion of Mydicar, patients treated with the highest dose versus placebo had an 88% risk reduction (Hazard Ratio = 0.12, P=0.003), of major cardiovascular events such as: death, need for left ventricular assist device (LVAD) or cardiac transplant, episodes of worsening heart failure and the number of heart failure-related hospitalizations. The mean duration of hospitalization in the Mydicar high dose group during the 12-month period was 0.4 days per patient compared with 4.5 days per patient in the placebo group. Additionally, the 12-month CUPID data show that heart failure, which is a progressive disease, became stabilized in high dose Mydicar-treated patients: heart failure symptoms, exercise tolerance, serum biomarkers and cardiac function essentially improved or remained the same while these parameters deteriorated substantially in patients treated with placebo and concurrent optimal drug and device therapy.
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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