FDA Grants Pluristem Orphan Drug Designation for Its PLX-R18 Cell Therapy as Treatment for Acute Radiation Syndrome
19 October 2017 - 10:30PM
Pluristem Therapeutics Inc. (NASDAQ:PSTI) (TASE:PSTI), 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.
Safe Harbor Statement
This press release contains express or implied
forward-looking statements within the Private Securities Litigation
Reform Act of 1995 and other U.S. Federal securities laws. For
example, we are using forward-looking statements when we discuss
developing our PLX-R18 therapy as a treatment for ARS, which can
potentially save many lives, or when we discuss the benefits of
receiving Orphan Drug Designation for our PLX-R18 therapy and that
it brings us one step closer to providing a next-generation medical
countermeasure against ARS. These forward-looking statements and
their implications are based on the current expectations of the
management of Pluristem only, and are subject to a number of
factors and uncertainties that could cause actual results to differ
materially from those described in the forward-looking statements.
The following factors, among others, could cause actual results to
differ materially from those described in the forward-looking
statements: changes in technology and market requirements; we may
encounter delays or obstacles in launching and/or successfully
completing our clinical trials; our products may not be approved by
regulatory agencies, our technology may not be validated as we
progress further and our methods may not be accepted by the
scientific community; we may be unable to retain or attract
key employees whose knowledge is essential to the development of
our products; unforeseen scientific difficulties may develop with
our process; our products may wind up being more expensive than we
anticipate; results in the laboratory may not translate to equally
good results in real clinical settings; results of preclinical
studies may not correlate with the results of human clinical
trials; our patents may not be sufficient; our products may harm
recipients; changes in legislation; inability to timely develop and
introduce new technologies, products and applications; loss of
market share and pressure on pricing resulting from competition,
which could cause the actual results or performance of Pluristem to
differ materially from those contemplated in such forward-looking
statements. Except as otherwise required by law, Pluristem
undertakes no obligation to publicly release any revisions to these
forward-looking statements to reflect events or circumstances after
the date hereof or to reflect the occurrence of unanticipated
events. For a more detailed description of the risks and
uncertainties affecting Pluristem, reference is made to Pluristem's
reports filed from time to time with the Securities and Exchange
Commission
Contact:
Karine Kleinhaus, MD, MPH Divisional VP, North
America 1-914-512-4109
karinek@pluristem.com
Efrat Kaduri Head of Investor and Public
Relations 972-74-7108600 efratk@pluristem.com
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