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Mydicar Receives ‘Breakthrough Therapy’ Designation as Heart Failure Treatment

Cardiac catheter delivers gene therapy directly to heart

Mydicar (Celladon Corporation) has been granted a “breakthrough therapy” designation by the FDA for reducing hospitalizations for heart failure in neutralizing antibody (NAb)-negative patients with New York Heart Association (NYHA) class III or IV chronic heart failure.

Mydicar is being developed as a first-in-class therapy for patients with chronic heart failure due to systolic dysfunction. The new treatment uses genetic enzyme-replacement therapy to correct deficiencies in the enzyme SERCA2a, which becomes deficient in heart failure patients and results in inadequate pumping of the heart.

A companion diagnostic test has been developed to identify patients who are adeno-associated virus-1 (AAV-1) NAb negative and therefore eligible for treatment with Mydicar.

Mydicar uses gene therapy to selectively target and restore SERCA2a enzyme levels by transferring the SERCA2a gene directly into cardiac muscle cells, which improves the heart’s ability to pump blood. Mydicar uses a nonpathogenic recombinant AAV and is delivered directly to the heart in a routine outpatient procedure, similar to an angiogram, in a cardiac catheterization laboratory.

Results of the 39-patient phase IIa CUPID 1 trial of a single intracoronary infusion of high-dose Mydicar in patients with advanced heart failure due to systolic dysfunction showed that the therapy was safe and well tolerated. Mydicar reduced heart failure-related hospitalizations and improved patients’ symptoms, quality of life, and key markers of cardiac function that were predictive of survival, such as elevated levels of natriuretic peptides and left ventricular end-systolic volume.

Long-term results from the CUPID 1 trial showed that, in the additional 2-year follow-up period, the durability of reduced cardiovascular and terminal events previously observed in the Mydicar high-dose cohort at 12 months was maintained. The risk of recurrent cardiovascular events in the presence of terminal events over 3 years of follow-up was reduced by 82% in the high-dose group compared with the placebo group (P = 0.048). No safety concerns were noted during the 3-year follow-up period for patients who received Mydicar.

Mydicar is currently being studied in the phase IIb CUPID 2 trial to determine its efficacy in reducing the frequency of and/or delaying heart failure-related hospitalizations. This randomized, double-blind, placebo-controlled, multinational trial is evaluating a single intracoronary infusion of Mydicar compared with placebo added to a maximal, optimized heart failure regimen in patients with NYHA class III or IV symptoms of chronic heart failure due to systolic dysfunction. A total of 250 patients have been enrolled. The results are expected in April 2015.

Source: Celladon Corporation; April 10, 2014.

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