Celladon Corp, a biopharmaceutical company focusing on the discovery and development of innovative treatments for cardiovascular diseases, announced that its first phase-2 trial with Mydicar for the treatment of advanced heart failure has met the prospectively defined safety and efficacy endpoints for the trial as defined in the phase-2 clinical trial protocol.

The CUPID trial (Calcium Up-regulation by Percutaneous administration of gene therapy In cardiac Disease) is a randomized, double-blind, placebo-controlled study to assess the efficacy and safety of Mydicar, a genetically targeted enzyme replacement therapy for advanced heart failure. Patients eligible for enrolment 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).

"These results are very encouraging, and Mydicar may add to the treatment options for patients with advanced heart failure,” said professor Mariell Jessup, of the Hospital of the University of Pennsylvania and principal investigator of the study. “There is an important need for new treatment options in this patient population. A drug such as Mydicar has exciting potential for patients with this debilitating disease and for the physicians who treat them.”

Krisztina Zsebo, president and CEO of Celladon, said, “We are very pleased to announce these positive top-line clinical results. There is a significant unmet medical need for the treatment of patients with advanced heart failure, and today there is no cure for this debilitating disease. The results of the study clearly indicate favourable treatment effects in this severely affected patient population, and the potential benefits of Mydicar to patients with this disease are very encouraging. These results are a significant step in achieving our goal of developing novel and innovative therapies for patients suffering from serious cardiovascular diseases. We are looking forward to presenting the complete results at the upcoming Heart Failure Congress 2010 in Berlin.”