Pluristem Files for Orphan Drug Status with FDA for Use of PLX Cells in Treatment of Aplastic Anemia

Pluristem Therapeutics, Inc, a leading developer of placenta-based cell therapies, today announced that it has filed the necessary documents requesting that the FDA grant orphan drug status to its PLacental eXpanded (PLX) cells for the treatment of aplastic anemia.

The filing marks Pluristem’s second orphan drug application to the FDA. The company applied once before, and successfully received in orphan drug status from the FDA for its PLX cell therapy in the treatment of Buerger’s disease.

Aplastic anemia is a rare but serious disorder caused by destruction of blood-forming stem cells (Hematopoietic Stem Cells or HSCs) in the bone marrow. While normally HSCs develop into three types of blood cells, red blood cells, white blood cells and platelets, in aplastic anemia all blood types are deficient, a condition also known as pancytopenia. The disease is considered an emergency situation where patients are supported either with blood transfusions in anticipation of a bone marrow transplant (BMT) or with drugs that suppress the immune system. Aplastic anemia patients are included in the bone marrow transplant market estimated at $1.3 billion per year in the United States alone.

In May of this year, Pluristem announced that a seven year-old girl in Israel, whose condition was rapidly deteriorating due to aplastic bone marrow, experienced a reversal of her condition with a significant increase in her red blood cells, white blood cells and platelets following the intramuscular injection of the company’s PLX cells. Prior bone marrow transplants had failed. The patient has subsequently been released from the hospital and returned home.

"The tremendous recovery exhibited by the pediatric patient afflicted with aplastic anemia who was given our PLX cells under compassionate use has prompted Pluristem to aggressively work towards quickly making our cells available for these patients, a process that will be expedited with the granting of orphan drug status," said Zami Aberman, chairman and chief executive officer of Pluristem. “We are now in the process of establishing an advisory board made up of key opinion leaders in the area of bone marrow transplantation from the United States, Europe and Israel to provide us with valuable insight as we expand our activities in the treatment of the bone marrow diseases and transplantations."

About Orphan Drug Status

Orphan drug designation qualifies a company for several benefits under the Orphan Drug Act of 1983 (ODA), as amended. These benefits include a 7-year period of orphan drug exclusivity upon product approval, a tax credit for certain clinical testing expenses for the orphan drug, written guidance on the non-clinical and clinical studies needed to obtain marketing approval of an orphan drug, and orphan drug grants.

About Pluristem Therapeutics

Pluristem Therapeutics Inc. (NASDAQCM: PSTI) (TASE:PLTR) is a leading developer of placenta-based cell therapies. The Company's patented PLX (PLacental eXpanded) cells are a drug delivery platform that releases a cocktail of therapeutic proteins in response to a host of local and systemic inflammatory and ischemic diseases. PLX cells are grown using the company's proprietary 3D micro-environmental technology and are an "off-the-shelf" product that requires no tissue matching prior to administration. Pluristem is focusing on the development of PLX cells administered locally to potentially treat systemic diseases and potentially obviating the need to use the intravenous route.

Data from two phase I studies indicate that Pluristem's first PLX product candidate, PLX-PAD, is safe and potentially effective for the treatment of end stage peripheral artery disease when given locally. Additionally, Pluristem is developing PLX-PAD for cardiac ischemia, PLX-BMP for Acute Radiation Exposure, Bone Marrow Transplant Failure and chemotherapy- induced Bone Marrow Aplasia, PLX-ORTHO for orthopedic indications and PLX-PAH for Pulmonary Hypertension in collaboration with United Therapeutics. Pluristem's pre-clinical animal models have demonstrated PLX cells are also potentially effective in other inflammatory/ischemic indications, including diastolic heart failure, inflammatory bowel disease, neuropathic pain and pulmonary fibrosis.

Pluristem has a strong patent portfolio, company-owned GMP certified manufacturing and research facilities, strategic relationships with major research institutions and a seasoned management team. For more information visit www.pluristem.com, the content of which is not part of this press release. Follow Pluristem on Twitter@Pluristem.

SOURCE: Pluristem