During our first-quarter earnings call last month, I was pleased to report that we began treating patients in the ixCELL-DCM Phase 2b trial, which is evaluating ixmyelocel-T for the treatment of advanced heart failure due to ischemic dilated cardiomyopathy (ischemic DCM). This clinical trial offers a potentially historic advancement in the treatment of patients with advanced heart failure due to ischemic DCM, an indication for which we have an orphan drug designation in the United States.
DCM is a condition in which the heart becomes weakened and enlarged and cannot sufficiently pump blood throughout the body. DCM is the third most common cause of heart failure and the most frequent cause of heart transplantation. A majority of the advanced heart failure patients who no longer respond to medical therapy — more than a quarter of a million patients in the U.S. — have DCM, and approximately 60% of these cases are of ischemic origin due to prior heart attacks or atherosclerosis. These patients typically have maximized their use of prescription and device therapies, and are no longer candidates for procedures to restore blood flow such as angioplasty and bypass surgery. At this stage of the disease, they have very limited treatment options, which generally include placement of left ventricular assist devices and heart transplantation.
What makes advanced heart failure due to ischemic DCM so challenging for patients is its impact on daily life. The condition significantly reduces exercise capacity and quality of life, because for many patients, walking even a short distance causes severe shortness of breath and fatigue. There is a strong rationale for developing ixmyelocel-T for the treatment of ischemic DCM. In preclinical studies, ixmyelocel-T was found to significantly reduce cardiac tissue damage and demonstrated additional cardioprotective effects in relevant disease models. In Phase 2a clinical trials, ixmyelocel-T was found to be well-tolerated and demonstrated positive efficacy trends, including improved symptoms and improved walking distance in treated patients compared to placebo.
The ixCELL-DCM Phase 2b trial is designed to enroll 108 patients at about 30 sites in the U.S. and Canada. This is a double-blind, placebo-controlled trial that will primarily measure mortality, hospitalizations, and heart failure emergency room visits in trial participants. The trial also will examine a range of clinical, functional, and symptomatic measures. The study is on track to complete enrollment by the end of Q1 2014 and show top-line results in Q2 2015.
Aastrom is one of the few companies working to develop a treatment for this major unmet medical need, and our clinical research program for ixmyelocel-T is one of the most advanced global research efforts in regenerative medicine. As we continue to make progress, we look forward to the opportunity to share our results and insights with patients, physicians, and researchers around the world.