NEWARK, Calif., Oct. 21, 2020 /PRNewswire/ -- Protagonist
Therapeutics, Inc. (NASDAQ:PTGX) today announced that the European
Medicines Agency (EMA) has granted orphan drug designation for
PTG-300 in the treatment of polycythemia vera. PTG-300 is an
injectable synthetic peptide mimetic of the natural hormone
hepcidin and has previously received orphan drug designation for
the treatment of polycythemia vera from the U.S. Food and Drug
Administration (FDA).
"This designation reflects the potential of PTG-300 as a
treatment candidate and the global need for novel treatments for
individuals living with polycythemia vera," commented Samuel Saks, M.D., Protagonist Chief Medical
Officer. "Early clinical results that were reported in May, as well
as the novel non-cytoreductive therapeutic mechanism of PTG-300 in
regulating iron, suggest that PTG-300 may help a broad population
of polycythemia vera patients. We are currently engaged in
discussions with leaders in treatment of polycythemia vera to
design a pivotal study. Completion of enrollment for the ongoing
study of 50 patients is expected in mid-2021. A pivotal study is
expected to begin in the second half of 2021, pending our planned
discussions with regulatory agencies."
A Phase 2 study of PTG-300 in patients with polycythemia vera is
currently enrolling subjects. Additional information is available
at http://ptg300pvstudy.com/.
About Polycythemia Vera
Polycythemia vera is a myeloproliferative neoplasm characterized
primarily by the increased production of red blood cells.
Well-established treatment guidelines focus on maintaining
hematocrit levels continuously below 45 percent to reduce the risk
of thrombotic events. Unfortunately, current treatment options are
unable to maintain hematocrit to below the 45 percent target for
many patients and may be associated with serious side effects.
There are an estimated 100,000 patients with polycythemia vera in
the U.S. and approximately 100,000 patients in major EU
countries.
About the European Medicines Agency (EMA) Orphan Drug
Designation
Orphan drug designation in the European Union (EU) is granted by
the European Commission based on a positive opinion issued by the
EMA Committee for Orphan Medicinal Products. To qualify, a
therapeutic candidate must be intended to treat a serious condition
that affects fewer than five in 10,000 people in the EU, and there
must be sufficient data to suggest the candidate may produce
clinically relevant outcomes. The designation provides companies
with certain benefits and incentives for clinical development and a
period of market exclusivity, if approved.
About Protagonist Therapeutics, Inc.
Protagonist Therapeutics is a clinical stage biopharmaceutical
company that utilizes a proprietary technology platform to discover
and develop novel peptide-based therapeutics to address significant
unmet medical needs and transform existing treatment paradigms for
patients. The Company currently has three clinical-stage assets.
PTG-300 is an injectable hepcidin mimetic in development for the
treatment of polycythemia vera and other blood disorders. PTG-200
is an orally delivered, gut-restricted, interleukin-23 receptor
specific antagonist peptide in development for the treatment of
inflammatory bowel disease, with Crohn's disease as the initial
indication. The Company has a worldwide license and collaboration
agreement with Janssen Biotech, Inc., for the development of
PTG-200. PN-943 is an orally delivered, gut-restricted
alpha-4-beta-7 integrin specific antagonist peptide in development
for the treatment of inflammatory bowel disease, with ulcerative
colitis as the initial targeted indication.
Protagonist is headquartered in Newark, California. For further information,
please visit http://www.protagonist-inc.com.
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements for
purposes of the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995. Forward-looking statements
include statements regarding our intentions or current expectations
concerning, among other things, the potential benefits of PTG-300
in treating polycythemia vera, the timing of enrollment in our
ongoing PTG-300 clinical trial and the commencement of a pivotal
study of PTG-300 in polycythemia vera patients. In some
cases, you can identify these statements by forward-looking words
such as "anticipate," "believe," "may," "will," "expect," or the
negative or plural of these words or similar expressions.
Forward-looking statements are not guarantees of future performance
and are subject to risks and uncertainties that could cause actual
results and events to differ materially from those anticipated,
including, but not limited to, our ability to develop and
commercialize our product candidates, the impact of the current
COVID-19 pandemic on our discovery and development efforts, our
ability to use and expand our programs to build a pipeline of
product candidates, our ability to obtain and maintain regulatory
approval of our product candidates, our ability to operate in a
competitive industry and compete successfully against competitors
that have greater resources than we do, and our ability to obtain
and adequately protect intellectual property rights for our product
candidates. Additional information concerning these and other
risk factors affecting our business can be found in our periodic
filings with the Securities and Exchange Commission, including
under the heading "Risk Factors" contained in our Quarterly Report
on Form 10-Q for the quarter ended June 30,
2020, filed with the Securities and Exchange
Commission. Forward-looking statements are not guarantees of
future performance, and our actual results of operations, financial
condition and liquidity, and the development of the industry in
which we operate, may differ materially from the forward-looking
statements contained in this press release. Any
forward-looking statements that we make in this press release speak
only as of the date of this press release. We assume no obligation
to update our forward-looking statements, whether as a result of
new information, future events or otherwise, after the date of this
press release.
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SOURCE Protagonist Therapeutics, Inc.