MYDICAR® Fact Sheet

CUPID Trial Locations

MYDICAR® is a genetically-targeted enzyme replacement therapy for advanced heart failure, one of the most expensive and debilitating conditions facing the health care industry. End stage patients are often hospitalized and experience severe difficulty breathing because their hearts cannot pump blood with enough pressure to keep fluid out of their lungs. Heart failure is expected to cost about $38 billion in direct and indirect costs 2008, primarily because patients are chronically in and out of intensive care units. Candidates for MYDICAR are patients who continue to deteriorate from their disease despite optimal drug therapy and who are eligible for either an implantable mechanical support device or a heart transplant, which are highly invasive procedures reimbursable by Medicare at costs of more than $200,000 each.

MYDICAR treatment involves a one-time outpatient infusion in a cardiac catheterization laboratory, similar to undergoing an angiogram. MYDICAR therapy is designed to restore levels of an enzyme known to play a key role in the progression of heart failure. Repairing this molecular defect in preclinical studies reversed the disease and restored cardiac function. In early stage human clinical trials, MYDICAR has been shown to be safe, and improved cardiac function parameters as well as exercise performance.

MYDICAR’s molecular target is an enzyme found in the sarcoplasmic reticulum (SR) that is critical to the contraction of the cardiac muscle cell. The SR is a specialized part of a cell (cellular organelle) that regulates the contraction and relaxation of cardiac muscle cells by coordinating the outflow (contraction) and inflow (relaxation) of calcium ions (Ca2+). The heart muscle’s ability to contract, and thus to pump blood and maintain oxygenation of the body, is determined by a continual re-loading of the SR with Ca2+ to “stage” for the next cycle of contraction. The key factor that enables the re-loading of the SR with Ca2+ is the enzyme SERCA2a. Numerous human studies have established a clear association between depleted SERCA2a enzyme in cardiac cells and the progression of end-stage heart failure.