Pluristem Receives FDA Orphan Drug Status Designation for Treatment of Aplastic Anemia
February 21 2013 - 1:30AM
Pluristem Therapeutics Inc. (Nasdaq:PSTI) (TASE:PLTR), a leading
developer of placenta-based cell therapies, announced today that
the U.S. Food and Drug Administration (FDA) has designated
Pluristem's PLacental eXpanded (PLX) cells orphan drug status for
the treatment of aplastic anemia.
Orphan drug designation qualifies a company for several benefits
under the Orphan Drug Act of 1983 (ODA), as amended. These benefits
include a 7-year period of orphan drug exclusivity upon product
approval, a tax credit for certain clinical testing expenses for
the orphan drug, written guidance on the non-clinical and clinical
studies needed to obtain marketing approval of an orphan drug, and
orphan drug grants.
This is Pluristem's second orphan drug designation from the FDA.
The company also received orphan drug status from the FDA for its
PLX cells for the treatment of Buerger's disease in August of
2011.
Aplastic anemia is a rare but serious disorder with a prevalence
of less than 200,000 in the U.S. The disease is caused by the
failure of hematopoietic stem cells (HSCs) contained within the
bone marrow to produce red blood cells, white blood cells and
platelets. The disease is considered an emergency and patients are
supported with blood products in anticipation of a bone marrow
transplant (BMT) or drugs that suppress the immune system.
"Receiving orphan drug designation for aplastic anemia is an
important event for Pluristem as it open pathways for using our PLX
cells for additional indications in the field of hematology,"
stated Zami Aberman, Chairman and CEO of Pluristem.
Pluristem has established clinical advisory board made up of key
opinion leaders in the area of bone marrow transplantation from the
United States, Europe and Israel to provide the company with
valuable insight towards expanding its activities in the treatment
of the bone marrow diseases and transplantations.
About Pluristem Therapeutics
Pluristem Therapeutics Inc. is a leading developer of
placenta-based cell therapies. The Company's patented PLX
(PLacental eXpanded) cells are a drug delivery platform that
releases a cocktail of therapeutic proteins in response to a host
of local and systemic inflammatory and ischemic diseases. PLX cells
are grown using the company's proprietary 3D micro-environmental
technology and are an "off-the-shelf" product that requires no
tissue matching prior to administration.
Pluristem has a strong patent and patent applications portfolio,
company-owned GMP certified manufacturing and research facilities,
strategic relationships with major research institutions and a
seasoned management team. For more information visit
www.pluristem.com, the content of which is not part of this press
release.
The Pluristem Therapeutics Inc. logo is available at
http://www.globenewswire.com/newsroom/prs/?pkgid=6882
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 that this indication may open pathways for the use of PLX
cells in additional indications in the field of hematology, or when
we discuss the expansion of our activities in the treatment of the
bone marrow diseases and transplantations. 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 surgical 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.:
William Prather R.Ph., M.D. Sr. VP Corporate Development
1-303-883-4954
William.PratherMD@pluristem.com
Daya Lettvin
Investor & Media Relations Director
+972-54-674-5580
daya@pluristem.com