Orphan Drug Designation for Pluristem's Aplastic Anemia Treatment Could Open $1.3 Billion Bone Marrrow Market

8-31-12

With its first Orphan Drug application for Beurger`s disease approved last fall, Pluristem Therapeutics (PSTI) will seek a second one for aplastic anemia (AA), an extremely dangerous condition associated with bone marrow degeneration that the company successfully treated earlier this month. If approved for clinical trials, it would mark Pluristem`s entry into the $1.3 billion bone marrow market that includes conditions stemming from chemotherapy and radiation poisoning. Orphan drugs aimed toward the most life threatening diseases take the shortest time to approval, and AA is considered a medical emergency.

Finding and treating rare disorders is big business; orphan drugs average $200,000 per patient per year. Pharmaceutical firms, recognizing the advantages in developing these `niche-busters` that are expected to comprise a $27 billion market in 2015, are highly focused in their search for them, and investors place more value on orphan drugs for various economic reasons - less review time, higher probability of FDA approval, advocacy organizations that act as free marketing, less price sensitivity - and also because in an environment of patent litigation, orphan market exclusivity cannot be easily challenged.

According to the FDA`s November 2011 issue of Innovative Drug Approvals, of 35 compounds cited for their medical novelty, 28% were orphan drugs approved during the FDA`s `first cycle`, meaning that the agency did not seek additional requests for information. Of these, over 80% targeted cancer and were bestowed with highly flexible clinical trial obligations involving only one study and a very small patient sample. Approval times were remarkably short - Adcetris, made by Seattle Genetics, Inc. (SGEN) for a deadly form of lymphoma, achieved approval in only 5.7 months. Likewise, Xalcori by Pfizer, Inc. (PFE), for patients with lung cancer possessing an abnormal gene that makes their disease more perilous, gained approval in 4.9 months.

On another note, in keeping with our observation that Pluristem is progressing at a rate atypical of biotechs its size, the company announced that it will start Phase II trials in the US next month at Duke University for intermittent claudication, a condition of the legs related to peripheral artery disease, only five months after the FDA gave them the go-ahead in April.

About the Analyst: Sharon di Stefano

Sharon di Stefano has spent 20 years as a healthcare analyst, beginning her career at Smith Barney, Harris Upham & Co. specializing in medical devices, pharmaceuticals, healthcare information technology, and biopharmacology. Ms. di Stefano had also served as Senior Venture Officer for the Edison Innovation Fund, implemented through the New Jersey Economic Development Authority that provided funding for early-stage life sciences companies. Industry experience includes laboratory research for Johns Hopkins Hospital and the Department of Defense.