Celladon Corporation Announces Publication of Advanced Heart Failure Clinical Trial Results With MYDICAR® Enzyme Replacement Therapy in the Journal Circulation

LA JOLLA, Calif., June 27, 2011 /PRNewswire/ -- Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, announces that the data from its completed Phase 2 CUPID clinical study of MYDICAR® for the treatment of advanced heart failure is being published today online in Circulation, an American Heart Association journal.

"I am very encouraged that our approach of restoring a critical enzyme that is depleted in advanced heart failure may lead to new treatment options for those patients that continue to decline despite the best standard drug and device therapies," said Mariell Jessup, M.D., Professor of Medicine at the University of Pennsylvania's School of Medicine, who was the principal investigator and lead author of the study. "The clinical deterioration seen in the placebo patients receiving ongoing optimal standard therapy emphasizes the tremendous unmet medical need in people with advanced heart failure. Based on these results we are encouraged that MYDICAR may one day fill this need."

The previously announced top line results of 39 patients met its primary safety and efficacy endpoints at 6 months for high dose MYDICAR versus placebo. Additionally, 12 months after receiving a single infusion of MYDICAR, patients treated with the highest dose versus placebo had an 88 percent risk reduction (Hazard Ratio = 0.12, P=0.003) of major cardiovascular events such as:

• Death
• Need for left ventricular assist device (LVAD) or cardiac transplant
• Episodes of worsening of heart failure
• Number of heart failure-related hospitalizations

The mean duration of hospitalization in the MYDICAR high dose group during the 12-month period was 0.4 days per patient compared with 4.5 days per patient in the placebo group. This finding is especially noteworthy because heart failure is the leading cause of hospitalization in Americans 65 years of age and older.

Additionally, the 12-month CUPID data show that heart failure, which is a progressive disease, became stabilized in high dose MYDICAR-treated patients: heart failure symptoms, exercise tolerance, serum biomarkers and cardiac function essentially improved or remained the same while these parameters deteriorated substantially in patients treated with placebo and concurrent optimal drug and device therapy.

The safety profile from this study was very favorable, with no significant side-effects from MYDICAR therapy.

Roger Hajjar, M.D., Director, Cardiovascular Research Center, Mount Sinai School of Medicine, NY, and co-founder of Celladon said: "While conventional treatment modalities have reduced heart failure mortality in the last 20 years, there remains a critical need for new therapeutic approaches in patients with advanced heart failure. The successful and safe completion of the phase 2 CUPID trial may give patients with heart failure a new therapeutic option in the future."

The CUPID Trial

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. Enrolled patients had severe forms of the disease defined by New York Heart Association Class III or IV heart failure, significantly impaired pumping function of their hearts (ejection fraction less than or equal to 35 percent), and less than half the normal ability to transport and utilize oxygen during exercise testing (VO2max less than or equal to 20 mL/kg/min). The CUPID trial ClinicalTrials.gov Identifier is NCT00454818.

Primary outcome measures included safety, worsening of heart failure leading to hospitalization, frequency of and time to cardiac transplantation or LVAD implantation, changes in patients' ability to exercise, echocardiographic assessments, a blood test for NT-proBNP, and symptoms of heart failure. The online publication can be accessed at: http://circ.ahajournals.org/cgi/reprint/CIRCULATIONAHA.111.022889v1

About MYDICAR

MYDICAR is a genetically targeted enzyme replacement therapy intended to restore levels of SERCA2a, a regulator of calcium cycling and contractility. SERCA2a levels decline in all forms of late-stage heart failure resulting in deficient heart function. With MYDICAR, the SERCA2a gene is delivered using a recombinant adeno-associated virus (AAV) as the vector. AAV is a naturally occurring virus not associated with any disease in humans. MYDICAR is delivered in a single dose directly to the heart during a routine outpatient cardiac catheterization procedure, similar to an angiogram. MYDICAR is synergistic and additive across current heart failure treatments such as ACE inhibitors, beta-blockers, sprinolactone/diuretics, and biventricular pacing devices. No treatment substitution decision is required by the treating physician.

About Heart Failure

Chronic heart failure is a leading cause of hospitalization and is expected to result in direct and indirect costs of $39.2 billion to the U.S. healthcare system in 2010. Nearly 6 million people in the U.S. have heart failure, and at least 670,000 new cases will be diagnosed this year. Heart failure leads to 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.

About Celladon

Celladon Corp., based in La Jolla, Calif., was launched in October 2004 as a privately held biotechnology company with the goal of becoming the leader in developing molecular therapies for the treatment of heart failure. The company's products target calcium cycling and contractility deficit in heart muscle cells. In addition to MYDICAR, Celladon is developing traditional small molecule activators of SERCA2a for the treatment of heart failure. To learn more about Celladon, visit Celladon's Web site at www.celladon.net.

SOURCE Celladon Corp.