Celladon Provides MYDICAR(R) Program Update of First-In-Human Trial for Advanced Heart Failure at American Society of Gene Therapy Annu...( Phase 1 Data Demonstrate Safety and E...),Health

Phase 1 Data Demonstrate Safety and Evidence of Biological Activity in 9 of 12 Patients;

Phase 2 Study Currently Enrolling in 17 Leading U.S. Medical Centers

SAN DIEGO, May 28 /PRNewswire/ -- Celladon Corporation presented today Phase 1 data from the Calcium Up-Regulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID), a First-in-Human Phase 1/2 Clinical Trial, in a scientific symposium at the 12th Annual American Society of Gene Therapy Meeting.

The Phase 1 data showed that MYDICAR(R) had an acceptable safety profile in twelve patients, as determined by study investigators and an independent safety committee. In addition, improvements from baseline to six months were observed across a number of key efficacy parameters important in assessing heart failure status. Efficacy was defined as the mean improvement in at least 2 of 5 domains without any worsening in the remaining domains, including a functional six-minute walk test, oxygen consumption, quality of life questionnaire, biomarker activity and left ventricular size and function.

"We are encouraged by the meaningful improvements in cardiac function and overall condition of patients, findings that we believe demonstrate the return toward normal intracellular calcium cycling and contractility in some of the heart muscle cells of these very sick patients," said Krisztina M. Zsebo, Ph.D., president and chief executive officer. "Our extensive preclinical and clinical investigation to understand the molecular basis of myocardial dysfunction, together with the evolution of safe and efficient gene transfer technology, has placed gene-based therapy for heart failure within reach and yielded valuable insights for the entire field of study."

The Phase 1 open-label, sequential dose escalation, multi-center phase of the trial was designed to investigate safety and biological effects of restoring SERCA
'/>"/>