Osiris Therapeutics, Inc. (NASDAQ:OSIR) announced that data from a 53-patient double-blind, placebo controlled trial studying the treatment of heart attack patients with Prochymal, a proprietary formulation of mesenchymal stem cells (MSCs), as well as research on the underlying mechanism of action of MSCs for cardiac repair, will be presented at the 2010 BMT Tandem Meeting. Joshua M. Hare, M.D., Louis Lemberg Professor of Medicine (Cardiology) and Director of the Interdisciplinary Stem Cell Institute at the University of Miami Miller School of Medicine, will present the data during a session entitled "Cardiac Stem Cell Therapy" on Thursday, February 25. Results from the study were recently published in the Journal of the American College of Cardiology (JACC).

"Patients receiving Prochymal did better than those receiving only standard-of-care," said Dr. Hare, lead author of the JACC publication. "They had fewer side effects and significant improvements in heart, lung and global function compared to the placebo group. The efficacy seen in this well-controlled trial represents an important step forward in the search for stem cell treatments for heart disease. With a strong safety profile that now includes more than 1,000 patients studied in clinical trials, mesenchymal stem cells are in a position to make significant advances in the field of cardiology."

The study demonstrates that treatment with Prochymal was safe in cardiac patients with recent heart attacks. Additionally, Prochymal significantly improved cardiac function and patient global symptom score, and reduced cardiac arrhythmias (irregular heartbeat), compared to placebo. The abstract and full-length manuscript are available online at the JACC's website. Based on these positive findings, a larger Phase II study in patients who have recently experienced severe acute myocardial infarction is underway.

About the Ongoing Acute Myocardial Infarction Trial

The Phase II double-blind, placebo-controlled trial will evaluate the safety and efficacy of Prochymal in conjunction with standard of care to improve heart function in patients who experienced a first heart attack. The trial will be conducted at leading institutions and academic research centers in the United States and Canada. This trial involves patients who have suffered a severe myocardial infarction, defined as left ventricular ejection fraction, or LVEF, between 20% and 45% at baseline. LVEF, which reflects the fraction of blood pumped out of a ventricle with each heart beat, is a common measurement of overall heart function and typically declines after a heart attack. The target enrollment is 220 patients, and patients will be randomized to Prochymal or placebo at 1:1. Efficacy endpoints determined from cardiac MRI include end systolic volume, LVEF and the ability of Prochymal to preserve functional heart tissue, or limit scar formation following a heart attack. In addition, functional and quality-of-life assessments will be performed.

About Prochymal

Prochymal is a preparation of mesenchymal stem cells (MSCs) formulated for intravenous infusion. The MSCs utilized in Prochymal are isolated from the bone marrow of healthy young adult donors, avoiding the controversy surrounding embryonic and fetal cell sources. They are grown in culture, permitting large-scale production. Because the cells can be expanded, thousands of doses can be produced from a single donation. Studies suggest MSCs are able to safely facilitate tissue repair through a number of mechanisms. Specifically, these studies indicate that MSCs are able to down-regulate severe inflammation and work at the cellular level to rebuild damaged tissue through the coordinated release of tissue-specific growth factors.

Prochymal is being evaluated in Phase III programs for steroid-refractory graft vs. host disease (GvHD), acute GvHD and Crohn's disease. Prochymal has been granted Fast Track status by the FDA for GvHD and Crohn's disease, and is the first stem cell product to receive FDA expanded access approval, making the product available now to patients with life-threatening GvHD. Prochymal obtained Orphan Drug status for GvHD from the FDA and the European Medicines Agency. This stem cell therapy is also being studied in Phase II trials for the treatment of acute myocardial infarction, pulmonary disease and type 1 diabetes.

Source
Osiris Therapeutics