Celladon Corp. Announces that MYDICAR(R) Meets Primary Endpoint in Phase 2 Trial for Treatment of Advanced Heart Failure

Study Results Demonstrate Favorable Treatment Effects with Genetically Targeted Enzyme Replacement Therapy

Apr 28, 2010, 07:00 ET from Celladon Corp.

LA JOLLA, Calif., April 28 /PRNewswire/ -- Celladon Corp., a biopharmaceutical company focused on the discovery and development of innovative treatments for cardiovascular diseases, today 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 enrollment 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).  The complete results from the trial will be presented at the upcoming annual meeting of the Heart Failure Association of the European Society of Cardiology, the Heart Failure Congress 2010 in Berlin, at the Late Breaking Trials – Session I at 2:45 p.m. CEST on May 30.

"These results are very encouraging, and MYDICAR® may add to the treatment options for patients with advanced heart failure," said Professor Mariell Jessup, M.D., 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, Ph.D., 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 favorable 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."


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 recombinant adeno-associated viral vector (AAV), a naturally occurring virus that is not associated with any disease in humans. MYDICAR® is delivered in a single dose directly to the heart muscle during a short outpatient procedure that is performed in a cardiac catheterization laboratory. The CUPID trial ClinicalTrials.gov Identifier is NCT00454818.

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 U.S. healthcare system of $39.1 billion. About 5 million people in the U.S. 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 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.

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SOURCE Celladon Corp.