Osiris' Prochymal Promising

The following post highlights Osiris Therapeutics, Inc. (OSIR - Analyst Report) and Genzyme Corporation (GENZ - Analyst Report).

Osiris' Stem Cell Product Shows Promise in Heart Attack Patients

This morning, Osiris Therapeutics (OSIR - Analyst Report) announced final analysis from its phase I clinical trial studying Prochymal for the treatment of acute myocardial infarction (heart attack). Data from the 53 patient program designed to evaluate safety and efficacy found that patients with acute MI who received intravenous Prochymal had lower rates of adverse events and significant improved heart function vs. patients on placebo.

Specifically, patients taking Prochymal had notably fewer cases of chest pain (24.2% vs. 63.2%), cardiac arrhythmia (11.8% vs. 474.%), and required significantly lower all-cause hospitalization (38.2% vs. 47.4%) during the two-year analysis.

Left ventricular ejection fraction (LVEF), which reflects the fraction of blood pumped out of a ventricle with each heartbeat, is a common measurement of overall heart function and typically declines after a heart attack. Patients with significantly compromised LVEF often progress to heart failure.

Two-year MRI data demonstrates there was statistically significant improvement in LVEF over baseline (6.6 vs. 3.9) for patients on Prochymal. For patients with more severe myocardial infarction, defined as a baseline LVEF of 45% or less, even greater effects were observed (9.5 vs. 3.1) for the Prochymal group.

Prochymal is a patented, intravenously administered form of mesenchymal adult stem cells (MSC) currently in a host of clinical programs. Management has shown that MSC offer three important benefits that make the development of Prochymal an attractive disease modifying therapeutic agent.

The first is a potent anti-inflammatory characteristic that limits swelling and pain. This is followed by the second characteristic, anti-fibrotic mediation that limits scaring and sores. And finally, the third characteristic is the expected regenerative properties that can be beneficial in re-growing damaged or dying tissue. Data shows MSC treatment has the ability to stimulate the formation of connective tissue or new blood vessels.

With this confirmed positive two-year phase I data, Osiris is now in position to move Prochymal in a phase II program for acute MI. Management is working on sites for the roughly 220 patient program now. This opportunity could be enormous for Osiris, given the some 13 million American's living with coronary artery disease (CAD) in the U.S.

Incidence of MI is about 1.2 million events per year, and accounts for nearly 1 in 5 deaths in the U.S. A viable treatment option that results in an improvement in overall patient condition (safety, hospitalization, LVEF) could be a blockbuster.

Osiris is also developing Prochymal in a host of other potential indications, including Crohn's disease (phase III), chronic obstructive pulmonary disease (COPD) (phase II), type 1 diabetes (phase II) and acute radiation syndrome (phase I).

The most advanced indication is in graft vs. host disease (GvHD), currently in two phase III trials with pivotal data expected around the middle of the year. Both GvHD and Crohn's have been granted FDA "fast track" status.

If successful, management should be in position to file for approval of Prochymal, along with development partner Genzyme (GENZ - Analyst Report), in GvHD before the end of the year. Prochymal is already being used under an FDA pre-approval expanded access program for GvHD patients.

Once approved, we expect that Prochymal will become the world's first blockbuster stem-cell product.

Read the full analyst report on OSIR