SEATTLE, Sept. 20, 2017 /PRNewswire/ -- PhaseRx, Inc.
(NASDAQ: PZRX), a biopharmaceutical company developing mRNA
treatments for life-threatening inherited liver diseases in
children, today announced that its second drug development
candidate, PRX-ASL, for the treatment of argininosuccinate lyase
deficiency (ASLD), has received orphan drug designation by the U.S.
Food and Drug Administration (FDA).
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 including plasma citrulline, argininosuccinic
acid (ASA) and blood ammonia.
"The FDA's decision to grant PRX-ASL orphan drug designation for
ASLD represents an important milestone in the development of our
second therapeutic candidate," said Robert
W. Overell, Ph.D., president and chief executive officer.
"PRX-ASL is our second drug to show preclinical proof of concept
using our Hybrid mRNA TechnologyTM. Like our lead
candidate PRX-OTC, we believe PRX-ASL also has the potential to
correct the disease in children, a population that could
particularly benefit from treatment for this rare disease."
The FDA grants orphan drug designation to investigational drugs
and biologics that are intended for the treatment of rare diseases
that affect fewer than 200,000 people in the U.S. Orphan drug
status is intended to facilitate drug development for rare diseases
and may provide several benefits to drug developers, including
assistance with clinical study design and drug development, tax
credits for qualified clinical trials costs, exemptions from
certain FDA application fees, and seven years of market exclusivity
upon regulatory product approval.
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 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
TechnologyTM 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 company being dependent on
technologies it has licensed and that it may need to license in the
future to develop its products, (iii) the fact that the company
will need to raise substantial additional funding to bring its
planned products through clinical trials, regulatory approval,
manufacturing and marketing and to become profitable, (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 and may never lead to marketable products,
(v) the fact that all of the company's programs are in preclinical
studies or early stage research, so the company cannot predict how
these results will translate into results in humans, nor can it be
certain 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 and
time-consuming, and if the development of company product
candidates does not produce favorable results or is delayed, the
company may be unable to commercialize these products, (vii) the
company expecting to continue to incur significant research and
development expenses, which may make it difficult to attain
profitability, (viii) the company becoming dependent on
collaborative arrangements with third parties for a substantial
portion of its revenue, and its development and commercialization
activities being delayed or reduced if it fails to initiate,
negotiate or maintain successful collaborative arrangements, (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, with competition from
existing drugs, new treatment methods and new technologies that may
prove to be more effective or marketable than the company's
products. 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 company's
Annual Report on Form 10-K for the year ended December 31, 2016, and our Quarterly Report on
Form 10-Q for the quarter ended June 30,
2017. 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:
Corporate Communications Contact:
Jason Spark
Canale Communications
Managing Director
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.
