NEWARK, Calif., Jan. 6, 2020 /PRNewswire/ -- Protagonist
Therapeutics, Inc. (Nasdaq:PTGX) today announced that a Phase 2
study of its novel hepcidin mimetic PTG-300 in patients with
hereditary hemochromatosis has been initiated. Hereditary
hemochromatosis is a blood disorder that causes the body to absorb
too much iron from the diet, resulting in the accumulation of iron
in the body's tissues and organs, particularly in skin, heart,
liver, pancreas and joint tissues.
"We are pleased to begin this clinical proof-of-concept study in
hereditary hemochromatosis, expanding upon our ongoing studies of
PTG-300 in beta-thalassemia and polycythemia vera," commented
Dinesh V. Patel, Ph.D., Protagonist
President and Chief Executive Officer. "PTG-300 was discovered
through the application of our technology platform and is designed
to have better drug-like properties for use as a therapeutic in
comparison to the natural hormone hepcidin. Hereditary
hemochromatosis is a genetic disorder predominantly due to a
mutation in the HFE gene leading to a deficiency of hepcidin in the
body. Therefore, treatment of this disorder with the hepcidin
mimetic PTG-300 could potentially serve as a hormone replacement
therapy for these patients."
"Current treatments for hereditary hemochromatosis, including
periodic phlebotomy, can be a significant burden to patients,"
commented Samuel Saks, M.D.,
Protagonist Chief Medical Officer. "PTG-300 could potentially
reduce the need for phlebotomy and offer a safer and better
long-term solution to management of the disease. Guidelines for
hereditary hemochromatosis focus on controlling serum transferrin
saturation (TSAT) and ferritin to prevent long-term complications.
Given the TSAT reductions from PTG-300 observed to date in both
healthy volunteers and beta-thalassemia patients, as well as
regulation of organ iron content in a mouse model of hereditary
hemochromatosis, we believe that a significant reduction in
phlebotomy may be possible with PTG-300 treatment in patients with
hereditary hemochromatosis."
This Phase 2 study of PTG-300 in hereditary hemochromatosis is
an open label, multicenter study designed to evaluate the effects
of PTG-300 over 24 weeks of treatment. The endpoints of this
proof-of-concept study include change in TSAT and serum iron
levels, reductions in phlebotomy requirements, and an assessment of
participant-reported outcomes (SF-36 survey). Additional
information on the PTG-300 hereditary hemochromatosis study is
available at https://clinicaltrials.gov/ct2/show/NCT04202965.
About Hereditary Hemochromatosis
Hereditary hemochromatosis is a blood disorder caused by a
deficiency of hepcidin hormone and is characterized by excessive
iron accumulation in body tissues. There are approximately 1.3
million individuals diagnosed in the U.S. Current treatment
involves phlebotomy, or removal of blood, at regular intervals.
Accumulation of excess iron can cause restrictive cardiomyopathy,
diastolic dysfunction, heart failure, cirrhosis, and other effects,
including an increased risk for hepatocellular carcinoma.
About PTG-300
PTG-300 is an injectable hepcidin mimetic in clinical
development for the potential treatment of beta-thalassemia,
polycythemia vera (PV) and hereditary hemochromatosis (HH).
Hepcidin is a natural peptide hormone that regulates iron
absorption and utilization in the body through sequestration and
release from tissue macrophages and intestinal enterocytes. Iron
plays an essential role in various body functions, especially blood
formation. Excess iron in the body is toxic, resulting in bone
marrow, tissue and organ damage over time. In settings of tissue
iron overload and dysregulated erythropoiesis, treatment with
PTG-300 can potentially reduce the need for phlebotomies, such as
in the treatment of PV and HH, and the need for transfusions and
chelation therapies in thalassemia and myelodysplastic
syndrome. PTG-300 has been granted Orphan Drug designation in
the U.S. and EU and has received Fast Track designation from the
U.S. Food and Drug Administration for development in the potential
treatment of beta-thalassemia.
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 drugs to transform existing
treatment paradigms for patients with significant unmet medical
needs. PTG-300 is an injectable hepcidin mimetic in development for
the potential treatment of iron overload anemia and related rare
blood diseases including beta-thalassemia, polycythemia vera and
hereditary hemochromatosis. PTG-200 is an oral, gut-restricted
interleukin-23 receptor specific antagonist peptide in Phase 2
clinical development for the potential 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 for the clinical development of PTG-200. PN-943 is
an oral, gut-restricted alpha-4-beta-7 integrin specific antagonist
peptide in clinical development for the potential treatment of
inflammatory bowel disease, with a Phase 2 ulcerative colitis study
expected to commence in the second quarter of 2020.
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 of PTG-300 as a
possible treatment for hereditary hemochromatosis, the Company's
success at finding appropriate doses of PTG-300 for the treatment
of hereditary hemochromatosis, the results of future studies for
the treatment of hereditary hemochromatosis, the potential utility
of PTG-300 in blood disorders including hereditary
hemochromatosis, beta-thalassemia, and polycythemia vera, the
possibility of treatment of hereditary hemochromatosis with PTG-300
as a hormone replacement therapy, the Company's ability to
fund its clinical trials, the potential of PTG-300 to significantly
reduce the need for phlebotomy for patients with hereditary
hemochromatosis, the potential for PTG-300 to be a safer and better
long-term solution to management of hereditary hemochromatosis, the
initiation of and enrollment of patients in the Company's clinical
trials, the results of clinical trials and the outlook for our
other programs. In some cases, you can identify these statements by
forward-looking words such as "believe," "expect," "potential,"
"could," "possible," 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, and our ability to obtain and
maintain regulatory approval of 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 quarterly
period ended September 30, 2019,
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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