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The Journal of Cardiac Failure Publishes Results From Celladon Corporation's First-in-Human Phase 1/2 Clinical Trial in Advanced Heart Failure
Date:4/8/2009

Calcium Upregulation by Percutaneous Administration of Gene Therapy in Cardiac Disease (CUPID Trial) Demonstrated Safety and Biological Effects of SERCA2a Enzyme Replacement Using Gene Transfer

SAN DIEGO, April 8 /PRNewswire/ -- Celladon Corporation today announced the publication of results from a Phase 1/2 trial of MYDICAR(R) (AAV1/SERCA2a), a genetically targeted enzyme replacement therapy for advanced heart failure, in the April 2009 issue of the Journal of Cardiac Failure. The CUPID clinical trial demonstrated acceptable safety and produced quantitative evidence of biological activity across a number of parameters important for assessing heart failure. MYDICAR is designed to restore levels of an enzyme, SERCA2a, known to play a key role in the progression of heart failure. Repairing this molecular defect in animal studies has been shown to reverse the disease and restore cardiac function.

"Despite important therapeutic advances in pharmacologic and device therapies, the prognosis of patients with chronic heart failure remains extremely poor. Moreover, heart transplantation and the use of implantable assist devices are considered only in the later stages of the disease, and access to such therapies is restricted to a fraction of patients in need," said Brian E. Jaski, M.D., Medical Director of Advanced Heart Failure, Sharp Memorial Hospital, San Diego Cardiac Center, San Diego, a principal investigator on the study, and a lead author of the publication. "The CUPID trial is the first to attempt to rescue a failing heart by replacing an enzyme known to play a critical role in normal cardiac muscle cell activity. Our objective is not only to improve the symptoms of heart failure, but restore physiologic function and reverse the severity of the disease in this chronic patient population."

The results of this study include data from the Phase 1 portion of this ongoing Phase 1/2 study for Cohorts 1 and 2 at 12 months of follow-up and Cohort 3 at 6 months of follow-up. A total of 9 patients with NYHA Class III/IV Heart Failure received a single intracoronary infusion of MYDICAR. The overall review of study safety assessments to date has been unremarkable. No consistent clinically meaningful changes in blood pressure, heart rate, or body temperature were observed. No trends or significant changes have been noted in blood chemistries, electrolytes, or liver and kidney function tests. No clinically significant changes or trends were noted in any components of the electrocardiogram including intervals, rhythm, or QRS morphology. Several of these treated patients demonstrated improvements from baseline to month 6 across efficacy/biological parameters, including symptomatic (5 patients), functional (4 patients), biomarker (2 patients) and left ventricular function/remodeling (5 patients). Of the nine patients treated, two with low levels of pre-existing antibodies to the AAV vector did not show improvement in these parameters.

"This peer reviewed publication of our results further validates our target and approach and we are excited to continue to evaluate the ability of MYDICAR to improve heart function in more patients in the Phase 2 portion of the study," said Krisztina M. Zsebo, Ph.D., Chief Executive Officer of Celladon.

The CUPID study is currently enrolling patients with advanced heart failure at 15 medical centers across the United States. MYDICAR is delivered in a single dose directly to the heart muscle during a short outpatient procedure, performed in a standard cardiac catheterization laboratory by inserting a catheter (thin flexible tube) through a peripheral blood vessel in the upper leg under x-ray guidance.

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 www.celladon.net, 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 www.celladon.net.


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SOURCE Celladon Corporation
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