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.