The U.S. Food and Drug Administration (FDA) cleared Haifa, Israel-based Pluristem Therapeutics’ Investigational New Drug (IND) application for PLX-R18 to treat acute radiation syndrome (ARS).
The U.S. Food and Drug Administration (FDA) cleared Haifa, Israel-based Pluristem Therapeutics’ Investigational New Drug (IND) application for PLX-R18 to treat acute radiation syndrome (ARS).
The U.S. National Institutes of Health’s National Institute of Allergy and Infectious Diseases (NIAID) sponsored and wrapped a successful Phase II-equivalent trial of PLX-R18 to treat ARS using the FDA’s animal rule pathway. The FDA Animal Efficacy Rule applies to developing and testing of drugs to reduce or prevent serious and life-threatening conditions caused by exposure to lethal or permanently disabling toxic agents where human testing it’s feasible or ethical.
It is also being evaluated by the U.S. Department of Defense (DOD) in supported of the military for treatment before or within the first 24 hours of radiation exposure. The company is currently in talks with the FDA and several U.S. governmental agencies for proposed pivotal study of PLX-R18 in ARS.
The traditional emergency treatment for radiation poisoning is potassium iodide (KI). This compound helps block radioactive iodine from being absorbed by the thyroid gland, which is the part of the body most sensitive to radioactive iodine.
Just last week, Canadian residents in Amherstburg, Ontario, directly across Lake Erie from the Fermi nuclear power plant in Monroe, Michigan, were offered potassium iodide pills as part of a plan to educate people in the “primary zone” of the Enrico Fermi nuclear power plant.
PLX-R18 is a cell therapy designed to treat bone marrow that can’t produce enough blood cells because of ARS, as well as certain cancers, cancer treatments, or immune-mediated bone marrow failure. Early animal studies were conducted in collaboration with the Hadassah Medical Center, and additional preclinical trials were performed with the NIH, Hadassah, the Charite in Berlin, and other research institutions.
“We are proud to have PLX-R18 join the exclusive club of IND approved medical countermeasures for the treatment of ARS,” said Zami Aberman, chairman and co-chief executive officer of Pluristem, in a statement. “This FDA clearance is one of the most significant milestones in the development of PLX-R18 to date and should provide Pluristem with significant support in advancing its off-the-shelf cell therapy into a pivotal trial. The fact that we are now able to treat human casualties in the case of a nuclear event provides us with the ability to protect from severe health consequences, savings lives of population in need.”
The drug is injected into muscle two times, a week apart. It can be used up to 96 hours after exposure. Arik Eisenkraft, the company’s director of homeland defense projects, told Reuters, “We are increasing the survival rate following high-level radiation exposure. We see improvement in all three blood lineages—red cells, white cells and platelets. We think that the everyday use of the compound will be for bone marrow failure of any cause.”
It also has an advantage in that it doesn’t require tissue matching prior to administration. The company is also conducting Phase III trials in the U.S. and Europe of PLX-PAD, to treat patients with the end-stage of peripheral artery disease, to prevent amputation.
The company indicates it will begin to prepare an emergency stock of PLX-R18 for emergency use. Full approval will depend on the Phase III clinical trial results.
Pluristem’s technology uses placental cells and a proprietary 3D technology platform to develop cell therapies for a variety of conditions, including inflammation, ischemia, muscle injuries, hematological disorders and radiation exposure.
