NEWARK, Calif., June 17, 2020 /PRNewswire/ -- Protagonist
Therapeutics, Inc. (Nasdaq: PTGX) today announced that the U.S.
Food and Drug Administration (FDA) has granted Orphan Drug
Designation for PTG-300 for the treatment of polycythemia vera.
PTG-300 is an injectable synthetic peptide mimetic of the natural
hormone hepcidin currently in clinical development for the
treatment of polycythemia vera and hereditary hemochromatosis.
"Receiving FDA orphan drug designation is another important
milestone for Protagonist and underscores the importance of our
work in polycythemia vera," commented Samuel Saks, M.D., Protagonist Chief Medical
Officer. "Individuals living with polycythemia vera face a high
disease burden. PTG-300 has a non-cytoreductive therapeutic
mechanism in the treatment of polycythemia vera and has shown a
well-tolerated safety profile to date. Because of its properties,
PTG-300 may help provide sustained control of hematocrit and
potentially help address symptoms of polycythemia vera and systemic
iron deficiency in these patients."
Protagonist recently announced initial Phase 2 results in
patients with polycythemia vera that demonstrated robust clinical
response and clinically meaningful dose related control of
hematocrit levels on individual patient basis.
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 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. In addition, current options are intolerable to some
patients and may be associated with serious side effects, such as
exacerbation of iron deficiency with phlebotomy. There are an
estimated 100,000 patients with polycythemia vera in the U.S. and
approximately 100,000 patients in major EU countries. Patients are
classified as either low risk or high risk based on prior
thrombotic events and age. A treatment option that could provide
consistent control of hematocrit over time without fluctuations
above 45 percent could be an important component of care for both
low and high risk polycythemia vera patient populations.
About Orphan Drug Designation
The FDA grants Orphan Drug Designation to novel drugs or
biologics that treat rare diseases or conditions affecting fewer
than 200,000 patients in the U.S. The designation allows the
sponsor of the drug to be eligible for a seven-year period of U.S.
marketing exclusivity on approval of the drug, as well as tax
credits for clinical research costs, the ability to apply for
annual grant funding, clinical trial design assistance, and the
waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
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 hereditary hemochromatosis.
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 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 for our clinical
programs, the potential of PTG-300 as a possible treatment for
polycythemia vera and hereditary hemochromatosis, the safety
profile of PTG-300, the potential for PTG-300 to provide sustained
control of hematocrit in patients, the potential for PTG-300 to
help address symptoms of polycythemia vera and systemic iron
deficiency in patients, the results of the Phase 2 study of PTG-300
in polycythemia vera and the results of future studies for the
treatment of polycythemia vera. In some cases, you can identify
these statements by forward-looking words such as "may,"
"potential," 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, our ability to
earn milestone payments under our collaboration agreement with
Janssen, 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 and risks related to
the global COVID-19 pandemic and actions taken to slow its spread.
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 period ended March 31, 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.