News | April 08, 2009

CUPID Trial Demonstrates Safety of Genetically Targeted Enzyme Replacement Therapy for Advanced Heart Failure

April 8, 2009 - The CUPID clinical trial demonstrated acceptable safety and produced quantitative evidence of biological activity across a number of parameters important for assessing heart failure, Celladon Corp. announced today.

Results from the Phase 1/2 trial of MYDICAR (AAV1/SERCA2a), a genetically targeted enzyme replacement therapy for advanced heart failure, appear in the April 2009 issue of the Journal of Cardiac 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.

The CUPID study is currently enrolling patients with advanced heart failure at 15 medical centers in the U.S. 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.

For more information: www.celladon.net

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