Pluristem Therapeutics Set to Enter Bone Marrow Disease Market as it is Preparing to Apply for Orphan Drug Status With the FDA for the Treatment of Aplastic Bone Marrow
HAIFA, Israel, Jul 23, 2012 -- Pluristem Therapeutics, Inc. (Nasdaq:PSTI) (TASE:PLTR), a leading developer of placenta-based cell therapies, today announced it is preparing to apply to the U.S. Food and Drug Administration for approval of its PLacental eXpanded (PLX) cells for the treatment of aplastic bone marrow as an Orphan Drug. Gaining Orphan Drug status approval is part of Pluristem's strategy for penetrating the bone marrow recovery market, starting with treatment of aplastic anemia, a disease in which bone marrow greatly decreases or stops production of blood cells and which strikes five to ten people in every million.
Gaining Orphan Drug Status carries multiple potential benefits, including the possibility of an expedited regulatory process, availability of grant money, certain tax credits and seven years of market exclusivity. In August 2011, Pluristem successfully applied for, and received, Orphan Drug Status from the FDA for its PLX cell therapy in the treatment of Buerger's disease.
Pluristem announced earlier this year that its PLX cells had saved the life of a seven year-old girl suffering from aplastic bone marrow and who had undergone two failed bone marrow transplants. With her condition rapidly deteriorating, her doctors injected Pluristem's PLX cells intramuscularly, following which the girl experienced a reversal of her condition with a significant increase in her red blood cells, white blood cells and platelets.
"With her body rejecting all possible treatment -- and with no other options -- we finally turned to Pluristem's PLX cells, which literally saved her life," said Professor Reuven Or, Director of Bone Marrow Transplantation, Cell Therapy and Transplantation Research Center at Hadassah Medical Center, and the child's primary physician. "The results of this unique case indicate that PLX cells may be effective in treating other diseases that affect the bone marrow." added Professor Or.
"We are very encouraged by the therapeutic results we've seen for our PLX cells in the treatment of aplastic anemia and we intend to pursue every avenue to bring it to market for this indication as swiftly as possible and to extend the indication to other clinical situations involving bone marrow deficiencies, " said Zami Aberman, Chairman and CEO of Pluristem. "As with the case of the young girl we successfully treated, PLX cells may potentially save lives when no other alternative is working. Orphan Drug Status in the U.S. would help accelerate our path to full FDA approval and we intend to apply for a similar designation in Europe and global territories."
About Pluristem Therapeutics
Pluristem Therapeutics Inc. (Nasdaq: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. |
