Pluristem’s PLX-R18 Advances into Second Cohort of Dose Selection Study for Treatment of Acute Radiation Syndrome
December 06 2016 - 7:00AM
Pluristem Therapeutics Inc. (Nasdaq:PSTI) (TASE:PSTI), a
leading developer of placenta-based cell therapy products, today
announced a milestone in its development program for PLX-R18, which
is being evaluated as a medical countermeasure in the treatment of
the hematologic components of Acute Radiation Syndrome (ARS) by the
National Institute of Allergy and Infectious Diseases (NIAID), a
part of the National Institutes of Health (NIH). ARS is caused by
exposure to very high levels of radiation, such as could occur in a
nuclear catastrophe. The syndrome can cause severe illness or
death.
The U.S. Food and Drug Administration (FDA)
previously advised Pluristem to conduct a pilot study in large
animals to determine the optimal dosage of PLX-R18 as a treatment
for the component of ARS that affects bone marrow function. The
NIAID has now completed the dosing of the first cohort and is
preparing to initiate dosing of the second and final cohort. Data
from the completed study are expected in the first half of 2017.
Based on these data the optimal treatment dose will be chosen for a
pivotal large animal study designed to meet the requirements for a
Biologics License Application (BLA) submission under the FDA’s
Animal Rule regulatory pathway.
The Animal Rule regulatory pathway allows for
approval of treatments for diseases such as ARS in which human
trials are not ethical or feasible. With this pathway, the FDA uses
animal efficacy studies and human safety data as the basis for
product approval.
“We are pleased with the advancement to the
second cohort of the study that will determine the optimal dose of
PLX-R18 in preparation for a pivotal trial. The support and
collaboration of the NIH’s NIAID have been instrumental in
advancing PLX-R18 towards becoming an FDA-approved countermeasure
ready for deployment in the case of a nuclear catastrophe,” stated
Pluristem Chairman and CEO Zami Aberman.
Previous NIH/NIAID studies of PLX-R18 in
ARS
The NIAID has supported and completed two
previous studies of PLX-R18, in which small animal models were used
to evaluate the efficacy and mode of action of PLX-R18 as a
potential treatment for the hematologic disorders associated with
ARS. ARS involves severe, potentially lethal damage to the bone
marrow’s ability to produce blood cells and platelets, as well as
to other systems and organs. Severe damage to bone marrow quickly
makes victims vulnerable to life-threatening hemorrhage, infection
and anemia. The more recent of the studies showed that
intramuscular administration of PLX-R18 resulted in a statistically
significant improvement in the recovery of white blood cell, red
blood cell, and platelet levels in animals exposed to high levels
of radiation, and described the treatment’s mechanism of action.
NIAID’s initial studies of PLX-R18 showed a substantial,
statistically significant improvement in 30-day survival and
overall survival of irradiated rodents given PLX-R18 versus a
control group.
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 ARS, certain cancers or cancer treatments, or
immune-mediated bone marrow failure. Pluristem received FDA
clearance to initiate a U.S. Phase I trial of PLX-R18 in incomplete
bone marrow recovery following hematopoietic cell transplantation.
Preclinical data from trials conducted by the NIH, Hadassah Medical
Center, 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. With its capabilities,
PLX-R18 could potentially treat a broad range of hematologic
indications, which together constitute a substantial global
market.
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. The cell products release
a range of therapeutic proteins in response to inflammation,
ischemia, hematological disorders, and radiation damage. PLX cell
products are grown using the Company's proprietary
three-dimensional expansion technology. They are off-the-shelf,
requiring no tissue matching prior to administration.
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 forward-looking
statements within the meaning of the "safe harbor" provisions of
the Private Securities Litigation Reform Act of 1995 and federal
securities laws. For example, we are using forward-looking
statements when we discuss the initiation of the dosing of the
second cohort by NIAID; when we discuss the timing of receiving the
data from the completed study; when we discuss our plan to conduct
a pivotal large animal study in order to submit a BLA under the
FDA’s Animal Rule regulatory pathway; when we discuss the
possibility of approving PLX-R18 by the FDA as a countermeasure in
the case of a nuclear catastrophe; and when we discuss the
potential of PLX-R18 to treat a broad range of hematologic
indications, which together constitute a substantial global market.
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:
Pluristem Therapeutics Inc.
Karine Kleinhaus, MD, MPH
Divisional VP, North America
1-914-512-4109
karinek@pluristem.com
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