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Celladon Provides MYDICAR(R) Program Update of First-In-Human Trial for Advanced Heart Failure at American Society of Gene Therapy Annual Meeting

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 SERCA2a enzyme activity in heart muscle cells. The enzyme levels are decreased in late stages of heart failure, and extensive research shows loss of SERCA2a levels represents a common pathway resulting in a defect in the ability of the heart to contract properly. Replacing the enzyme may restore function and reverse heart failure.

The Phase 2 randomized, double-blind, placebo-controlled, parallel-group, dose ranging portion of the study is designed to evaluate the use of MYDICAR at two or three dose levels compared to placebo in 37 patients. CUPID is currently enrolling patients with advanced heart failure at 17 U.S. medical centers.

Zsebo adds, "We anticipate completion of Phase 2 enrollment late this summer and plan to report results in the first half of 2010. In addition, we have adequate MYDICAR product manufactured to complete Phase 3 and recently acquired exclusive license to utilize Adeno-Associated Viral (AAV) vector technology in heart failure, which bodes well for commercial product development of MYDICAR and important to potential strategic partners. "

Celladon scientists, led by company co-founder Roger J. Hajjar, M.D., Director of the Cardiovascular Research Center at Mount Sinai School of Medicine, New York, developed MYDICAR for restoring the SERCA2a calcium transporter in heart failure and validated the overall beneficial effects on cardiac function. MYDICAR is a recombinant adeno-associated viral (rAAV) vector that transfers the SERCA2a gene into heart muscle cells. MYDICAR is delivered in a single dose directly to the heart muscle during a short outpatient procedure, performed in a standard cardiac catheterization laboratory via a small incision in the upper leg.

Of the twelve patients treated, two with low levels of pre-existing antibodies to the AAV vector did not show improvement in these parameters. The data are consistent with safety established for other rAAV vectors, which has been demonstrated in clinical studies of more than 500 patients. AAV vectors are the product of decades of research focused on the safety of gene transfer agents, and are derived from components of a non-replicating, non-pathogenic, commonly occurring human virus. AAV vectors do not integrate into the chromosome and are considered non-mutagenic. In addition, they have not been associated with the types of inflammatory reactions observed in trials involving adenoviral vectors, which are known to induce acute inflammation of tissues due to activation of the body's immune system.

Patients with heart failure may assess eligibility to participate in the CUPID trial and download a patient questionnaire and additional information on MYDICAR to review with your physician by visiting, under the "For Patients" tab.

About Heart Failure

Chronic heart failure is an increasingly important health problem. It is the leading medical cause of hospitalization and is expected to result in an estimated direct and indirect cost to the healthcare system in 2009 of $37.2 billion. About 5 million people in the United States have heart failure, and another 550,000 new cases are diagnosed each year. Heart failure contributes to or causes about 280,000 deaths annually. The most common symptoms of heart failure are shortness of breath, feeling tired, and swelling in the ankles, feet, legs, and sometimes the abdomen. There is no cure for heart failure.

About Celladon

Celladon Corporation, based in La Jolla, California, was launched in October 2004 as a privately held biotechnology company founded with the goal of becoming the leader in developing molecular therapies for the treatment of heart failure. The company's products target the key enzyme deficiency in advanced heart failure, SERCA2a, which regulates calcium cycling and contractility in heart muscle cells. Celladon's first product candidate, MYDICAR, delivers the gene for the SERCA2a enzyme. MYDICAR is currently being tested in Phase 1 and 2 clinical trials. Celladon is also developing traditional small molecule activators of SERCA2a for the treatment of heart failure. To learn more about Celladon, visit Celladon's website at

SOURCE Celladon Corporation
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