Pluristem Therapeutics Inc., a leading developer of placenta-based cell therapy products, announced today that the U.S. Food and Drug Administration (FDA) has granted the company an orphan drug designation for its PLX-R18 cell therapy for the prevention and treatment of acute radiation syndrome (ARS).
ARS results from exposure to high levels of radiation, such as in the case of a nuclear accident or attack, and can lead to severe health consequences including death. The Orphan Drug Act provides for granting special status to a drug or biological product, to treat a rare disease or condition. The benefits of achieving Orphan Drug Designation include close guidance by the FDA, which may accelerate the path to potential marketing approval, orphan drug grants, tax credits, and 7-year market exclusivity upon marketing approval.
Pluristem recently reported positive data from non-human primates (NHPs) studies of PLX-R18 cells as a treatment for ARS conducted by the National Institute of Allergy and Infectious Diseases (NIAID) at the National Institutes of Health (NIH), U.S. Department of Health and Human Services (DHHS). The reported data demonstrated improvement in survival rates of such NHPs and the enhancement of recovery across all three major blood lineages—white blood cells, red blood cells, and platelets. In addition, PLX-R18 cells are also being studied by the U.S. Department of Defense’s (DOD) Armed Forces Radiobiology Research Institute (AFRRI) to examine the effectiveness of the cells as a treatment for ARS prior to, and within the first 24 hours of exposure to radiation. Pluristem PLX-R18 cells are also being studied by Fukushima Medical University in Japan for the treatment of ARS and as an adjunct to radiotherapy in cancer patients.
“Pluristem has a vast and dynamic program developing our PLX-R18 therapy as a treatment for ARS, which can potentially save many lives,” said Zami Aberman, Co-CEO and Chairman of Pluristem. “Receiving Orphan Drug Designation brings us one step closer to providing a next-generation medical countermeasure against ARS, which is especially important given today’s volatile political climate.”
About PLX-R18
PLX-R18 is Pluristem’s second cell therapy product in development. It is designed to treat bone marrow that is unable to produce enough blood cells due to a variety of causes including acute radiation syndrome (ARS), certain cancers or cancer treatments, or immune-mediated bone marrow failure. PLX-R18’s first animal studies in ARS were performed in collaboration with Prof. Gorodetsky at Hadassah Medical Center. Further preclinical data from trials conducted by the U.S. National Institutes of Health, Hadassah, the Charite in Berlin and other prominent research institutions have shown that PLX-R18 cells secrete a range of specific proteins that trigger the regeneration of bone marrow hematopoietic cells, thereby supporting the recovery of blood cell production. Pluristem is currently enrolling patients in a U.S. Phase I trial of PLX-R18 in incomplete bone marrow recovery following hematopoietic cell transplantation (HCT) and is preparing for a pivotal trial in ARS.
About Pluristem Therapeutics
Pluristem Therapeutics Inc. is a leading developer of placenta-based cell therapy products. The Company has reported robust clinical trial data in multiple indications for its patented PLX (PLacental eXpanded) cells, and is entering late-stage trials in several indications. PLX cell products release a range of therapeutic proteins in response to inflammation, ischemia, muscle trauma, hematological disorders, and radiation damage. The cells are grown using the Company’s proprietary three-dimensional expansion technology and can be administered to patients off-the-shelf, without tissue matching. Pluristem has a strong intellectual property position; Company-owned and operated, GMP-certified manufacturing and research facilities; strategic relationships with major research institutions; and a seasoned management team.
Filed Under: Drug Discovery