SEATTLE, Nov. 28, 2017 /PRNewswire/ -- PhaseRx, Inc.
(NASDAQ: PZRX), a biopharmaceutical company developing mRNA
treatments for life-threatening inherited liver diseases in
children, today announced that the European Medicines Agency's
(EMA) Committee for Orphan Medicinal Products (COMP) has issued a
positive opinion recommending orphan medicinal product (orphan
drug) designation for PRX-ASL, for the treatment of
argininosuccinate lyase deficiency (ASLD), PhaseRx's second drug
candidate to treat a urea cycle disorder.
ASLD is a rare liver disorder caused by an inherited single-gene
deficiency that results in hyperammonemia (elevated ammonia in the
blood), and can lead to irreversible neurological impairment, coma
and death. PRX-ASL is an intracellular enzyme replacement therapy
(i-ERT) designed to replace the missing or defective enzyme in
patients with ASLD, thereby correcting the disease. PRX-ASL has
shown therapeutic potential in a preclinical model of ASLD,
including reduction in the levels of compounds whose elevation are
the hallmark of ASLD such as plasma citrulline, argininosuccinic
acid (ASA) and blood ammonia.
"This positive opinion recommending orphan drug designation for
PRX-ASL in the European Union is a significant step in the
development of this product candidate, and follows a similar
decision in September by the U.S. Food and Drug Administration,"
said Robert W. Overell, Ph.D.,
president and chief executive officer of PhaseRx. "We are pleased
that the Committee for Orphan Medical Products has affirmed our
belief in the need for a new therapeutic for this rare liver
disorder."
Applications for orphan designation are initially
reviewed by the COMP. Positive opinions are then forwarded to
the European Commission, which is responsible for formally granting
the orphan designation. Orphan medicinal product designation
in the European Union is given to products that are intended for
the treatment, prevention or diagnosis of a disease that
is life-threatening or chronically debilitating;
where prevalence of the condition in the EU is less than 5 in
10,000; and where the product represents a significant benefit over
existing treatments.
About ASLD
ASLD is a rare liver disorder caused by an inherited single-gene
deficiency that results in hyperammonemia (elevated ammonia in the
blood), and can lead to devastating consequences, including
cumulative and irreversible neurological impairment, coma and
death. The only known cure for ASLD is a liver transplant.
Currently available drug treatments do not correct the disease, and
do not eliminate the risk of life-threatening crises.
About PhaseRx
PhaseRx is a biopharmaceutical company dedicated to developing
mRNA products for the treatment of children with inherited enzyme
deficiencies in the liver using intracellular enzyme replacement
therapy (i-ERT). PhaseRx's initial product development focus is on
urea cycle disorders, a group of rare genetic diseases that
generally present before the age of twelve and are characterized by
the body's inability to remove ammonia from the blood with
potentially devastating consequences for patients. The company's
i-ERT approach is enabled by its proprietary Hybrid mRNA
Technology™ platform. PhaseRx is headquartered in Seattle. For more information, please visit
www.phaserx.com.
Safe Harbor Statement
This press release contains "forward-looking statements." Such
statements may be preceded by the words "intends," "may," "will,"
"plans," "expects," "anticipates," "projects," "predicts,"
"estimates," "aims," "believes," "hopes," "potential" or similar
words. Forward-looking statements are not guarantees of future
performance, are based on certain assumptions and are subject to
various known and unknown risks and uncertainties, many of which
are beyond the company's control, and cannot be predicted or
quantified and consequently, actual results may differ materially
from those expressed or implied by such forward-looking statements.
Such risks and uncertainties include, without limitation, risks and
uncertainties associated with (i) the fact that the company has
incurred significant losses since its inception and anticipates
that it will continue to incur significant losses for the
foreseeable future, (ii) the fact that Nasdaq is seeking to delist
our common stock from the Nasdaq stock market (although the Company
is appealing this decision), (iii) the Company's recent decision to
delay the development of its product candidates, reduce its
workforce and seek strategic alternatives, (iv) the company being
dependent on technologies it has licensed and that it may need to
license in the future, (v) the fact that substantial additional
funding will be required to develop the company's planned products,
(vi) the fact that the company's Hybrid mRNA Technology has not
previously been tested beyond company preclinical studies, and that
mRNA-based drug development is unproven, (vii) the fact that all of
the company's programs are in preclinical studies or early-stage
research and it is uncertain that any company product candidates
will receive regulatory approval or be commercialized, (viii)
the company's ability to adequately protect its proprietary
technology from legal challenges, infringement or alternative
technologies and (ix the biotechnology and pharmaceutical
industries being intensely competitive. More detailed information
about the company and the risk factors that may affect the
realization of forward-looking statements is set forth in the
company's filings with the Securities and Exchange Commission
(SEC), including the most recent annual report on Form 10-K and
its' quarterly reports on Form 10-Q. Investors and security holders
are urged to read these documents free of charge on the SEC's
website at http://www.sec.gov. The company assumes no obligation to
publicly update or revise its forward-looking statements as a
result of new information, future events or otherwise.
Contacts:
Corporate Communications Contact:
Jason Spark
Canale Communications
Senior Vice President
jason@canalecomm.com
619.849.6005
Investor Contact:
Robert H. Uhl
Westwicke Partners, LLC
Managing Director
robert.uhl@westwicke.com
858.356.5932
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SOURCE PhaseRx, Inc.