SEATTLE, April 25, 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-OTC for the treatment of ornithine
transcarbamylase deficiency (OTCD). OTCD is a rare genetic disorder
characterized by a complete or partial lack of the enzyme ornithine
transcarbamylase, an essential urea cycle enzyme that facilitates
the break down and removal of ammonia from the body.
"This positive opinion recommending orphan drug designation for
PRX-OTC in the European Union represents another important step
forward towards potentially bringing this novel therapy to patients
that suffer from the devastating effects of OTCD," said
Robert W. Overell, Ph.D., president
and chief executive officer of PhaseRx. "We believe that PRX-OTC,
with its first-in-class mechanism of action that can replace the
missing protein in these patients, may address this significant
unmet medical need."
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 PRX-OTC for Ornithine Transcarbamylase
Deficiency
Ornithine Transcarbamylase (OTC) is an enzyme in the urea cycle,
the body's pathway for metabolizing ammonia which results from
protein breakdown. Ornithine Transcarbamylase Deficiency (OTCD), a
rare liver disorder caused by an inherited single-gene deficiency,
results in hyperammonemia (elevated ammonia in the blood), often
leading to irreversible neurological impairment, coma and death.
PRX-OTC is an intracellular enzyme replacement therapy (i-ERT)
designed to replace the missing or defective enzyme inside the
cells of patients with OTCD. PRX-OTC has shown therapeutic
potential in a preclinical model of OTCD, including lowering of
blood ammonia and survival of 100% of treated mice. PRX-OTC has
received Orphan Drug Designation by the U.S. Food and Drug
Administration.
About PhaseRx
PhaseRx is a biopharmaceutical company developing a portfolio of
mRNA products to correct life-threatening inherited liver diseases
in children. PhaseRx's initial product development focus is on urea
cycle disorders, a group of rare genetic diseases that generally
present before the age of 12 and are characterized by the body's
inability to remove ammonia from the blood. The company's i-ERT
approach is enabled by its proprietary Hybrid mRNA
TechnologyTM platform and is applicable to a significant
number of inherited liver diseases. 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 company being dependent on
technologies it has licensed and that it may need to license in the
future, (iii) the fact that the company will need to raise
substantial additional funding to develop its planned products,
(iv) 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, (v) 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, (vi) the
fact that development of the company's product candidates will be
expensive, time-consuming and subject to regulatory approval, (vii)
the company expecting to continue to incur significant research and
development expenses, (viii) the company becoming dependent on
collaborative arrangements for the development and
commercialization of its products, (ix) the company's ability to
adequately protect its proprietary technology from legal
challenges, infringement or alternative technologies and (x) 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 web site 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:
Company Contact:
Erin Cox
PhaseRx, Inc.
Director of Investor Relations
erin@phaserx.com
206.805.6306
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.