Pluristem Announces Promising Results from Non-Human Primate Pilot Study of PLX-R18 in Acute Radiation Syndrome
May 03 2017 - 7:00AM
Pluristem Therapeutics Inc. (NASDAQ:PSTI), (TASE:PSTI), a leading
developer of placenta-based cell therapy products, announced today
the promising results of its non-human primates (NHP) pilot study
for PLX-R18 as a treatment for Acute Radiation Syndrome (ARS). The
study, conducted and funded by the National Institute of Allergy
and Infectious Diseases (NIAID), part of the National Institutes of
Health (NIH), was designed to assess the safety and efficacy of
PLX-R18 following intramuscular injection into irradiated and
non-irradiated NHPs. Efficacy measures included survival as well as
level of bone marrow function, which is affected by exposure to
high levels of radiation as may occur in a nuclear accident or
attack.
While this pilot study was not powered to
demonstrate statistical significance, all cohorts treated with
PLX-R18 showed improved survival compared to cohorts that received
placebo. The two lower dosages, 4 and 10 million cells per kilogram
body weight, resulted in an 85% survival rate in irradiated NHPs
compared to a 50% survival rate in the placebo treated control
group. This pilot study also demonstrated a trend towards enhanced
neutrophil and lymphocyte recovery.
No serious adverse reactions were observed in
non-irradiated NHPs, suggesting that in scenarios requiring the
rapid treatment of large populations, such as in the case of a
nuclear emergency, no determination of an individual’s level of
exposure would be required prior to treatment.
These data will help inform a pivotal study
designed to meet the requirements for a Biologics License
Application (BLA) submission under the FDA’s Animal Rule regulatory
pathway.
“These findings are in line with what we’ve seen
in previous studies and strengthen our commitment to delivering a
ready-to-use treatment to counteract the devastating effects of
ARS,” said Zami Aberman, Co-CEO and Chairman of Pluristem.
“The transition from small to large animals has
always been a challenge in therapeutic product development, and the
success of this study marks a significant milestone. Our unique,
multifactorial PLX-R18 cell therapy was developed to repair the
body’s ability to produce all three blood lineages in a timely
manner. This therapy would protect patients from severe infection,
anemia and hemorrhage, saving lives in the case of a nuclear event.
We are pleased that these findings support this treatment’s
efficacy and are looking forward to results from further studies,”
added Aberman.
“Following this successful trial, we look
forward to continuing our discussions with U.S. government agencies
regarding continued support for a pivotal trial,” said Yaky Yanay,
Co-CEO and President of Pluristem. “We are confident that PLX-R18
can serve as a powerful tool for governments to protect their
citizens against the devastating health impact of potential
exposure to nuclear radiation. We are proud to have developed a
treatment that could save many lives.”
About ARS
Acute Radiation Syndrome occurs following acute
exposure to very high levels of radiation, and involves severe,
potentially lethal injury to the bone marrow as well as to other
organs and systems within the body. High doses of radiation can
destroy the bone marrow’s ability to produce white cells, red cells
and platelets; without these cells patients are at high risk of
death.
About the pilot study
The objective of the study was to evaluate
survival and hematology parameters as well as safety parameters in
irradiated (target LD30/45) and non-irradiated male and female NHPs
(total 48 animals), following intramuscular treatment with 3 doses
(4.0, 10.0 and 20.0 million cells per Kg) of PLX-R18 as compared to
non-treated controls.
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 and is entering late-stage
trials in several indications. 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 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
Pluristem’s belief that the data from the NHP pilot study of
PLX-R18 will pave the way for a pivotal study designed to meet the
requirements for a BLA submission under the FDA’s Animal Rule
regulatory pathway, Pluristem’s discussions with U.S. government
agencies regarding continued support for a pivotal trial and
Pluristem’s confidence that PLX-R18 can serve as a powerful tool
for governments to protect their citizens against the devastating
health impact of potential exposure to nuclear radiation. 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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