Here we discuss Osiris Therapeutics (OSIR) and Genzyme (GENZ).

This morning, Osiris Therapeutics (OSIR) release two positive press releases on the company’s leading pipeline candidate, Prochymal. As a result, the shares are bucking the negative overall market trend.

The first announced a successful pre-Biologics License Application (pre-BLA) meeting with the U.S. FDA for Prochymal regarding use in the treatment of graft-versus-host disease (GvHD). The BLA filing, which is expected to be the first of its kind for a stem-cell product, will include data from two ongoing phase III programs in GvHD — one in steroid refractory patients and another in acute disease.

Osiris and the FDA have agreed upon the proposed content and structure for the BLA, which will be filed in a electronic Common Technical Document (eCTD) format, allowing for parallel submissions in areas outside the U.S. The agency also approved the primary end point for the two ongoing phase III trials, along with clearing a path for other potential hurdles such as the statistical analysis plan, the safety database, the Chemistry, Manufacturing and Controls (CMC) data, and product stability.

We expect Osiris to be in position to file for approval of Prochymal in GvHD later in 2009. Osiris has indicated they will seek a priority review, potentially allowing for approval during the first half of 2010.

In the meantime, news from Osiris’ second press release, allows for a clearance to broaden its expanded access program (EAP) for Prochymal, now making the investigational stem-cell product available to adults with life-threatening GvHD, prior to final FDA approval. In May of 2008, FDA approved the first Prochymal EAP for the treatment of pediatric GvHD patients. The EAP is designed to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins.

This is clearly a good sign, and bodes well for Prochymal final approval assuming the two phase III trials meet the primary end point later in 2009.

We continue to be very positive on Osiris. The company’s recent billion-dollar licensing deal with Genzyme (GENZ) was a transformational event. Prochymal has several hundred million dollar potential for GvHD, and blockbuster potential with an expanded label to include other indications for which it is being studied, including: Crohn’s disease, myocardial infarction, COPD, type-1 diabetes, and acute radiation syndrome.  

Read the full analyst report on OSIR.

“OSIR” Free Stock Analysis: Buy? Sell? Hold?
“GENZ” Free Stock Analysis: Buy? Sell? Hold?
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