NEWARK, Calif., June 18, 2018 /PRNewswire/ -- Protagonist
Therapeutics, Inc. (Nasdaq: PTGX) today announced that results of a
Phase 1 study in healthy volunteers and supportive pre-clinical
data for the Company's novel injectable hepcidin mimetic, PTG-300,
were the subject of oral presentations on June 16, 2018, at the 23rd Congress of
the European Hematology Association (EHA) in Stockholm. Protagonist is developing PTG-300
for the treatment of anemia and iron overload in related rare blood
disorder diseases, including beta thalassemia.
"The Phase 1 clinical results demonstrated the ability of
PTG-300 to achieve a sustained, dose-related reduction in serum
iron," commented Richard Shames,
M.D., Chief Medical Officer at Protagonist. "This observation was
evident in both single ascending and repeat doses -- a clear
demonstration of pharmacodynamic-based proof-of-concept in human
volunteers. In addition, the pre-clinical results with PTG-300 in a
mouse model of beta-thalassemia provide proof-of-mechanism activity
for the treatment of anemia and support for advancing this drug
candidate into patient studies."
"These pre-clinical and clinical results demonstrate the
potential of PTG-300 in the treatment of a broad range of blood
disorders," added Dinesh V. Patel,
Ph.D., Protagonist President and Chief Executive Officer. "We look
forward to pursuing initial development of PTG-300 for the
treatment of patients with beta-thalassemia who are non-transfusion
dependent as well as for severely-affected patients who are
dependent on transfusion. We continue to work toward initiation of
an open label Phase 2 trial in the fourth quarter of 2018."
Details of EHA Presentations
- Hepcidin Mimetic PTG-300 for Treatment of Ineffective
Erythropoiesis and Chronic Anemia in Hemoglobinopathy Diseases
Agents with hepcidin activity may help correct iron
distribution abnormalities and have beneficial effects on
erythropoiesis. In a preclinical mouse model of beta-thalassemia,
PTG-300 demonstrated the ability to reduce iron toxicity for
developing erythrocytes in the bone marrow, addressing a
contributing factor to ineffective erythropoiesis. The ability to
address ineffective erythropoiesis could lead to a potential
treatment for chronic anemia in conditions characterized by low
endogenous hepcidin levels and high serum iron levels, such as
beta-thalassemia and myelodysplastic syndrome.
- Hepcidin Mimetic PTG-300 Induces Dose-Related and Sustained
Reductions in Serum Iron and Transferrin Saturation in Healthy
Subjects
This first-in-human randomized, double-blind,
placebo-controlled study of subcutaneous PTG-300 was conducted to
evaluate safety and tolerability, pharmacokinetics and
pharmacodynamic activity of PTG-300 in 62 healthy volunteers. The
single-dose groups received PTG-300 over the dose range of 1-80 mg
and the repeat dose cohort received 40 mg once weekly for two
doses. PTG-300 demonstrated a rapid and sustained dose-related
reduction in serum iron from baseline. At higher doses, maximal
reduction in serum iron extended for at least 72 hours. The repeat
dose administration demonstrated a comparable iron reduction
following both doses. Treatment with PTG-300 was generally well
tolerated and no dose-limiting toxicities or serious adverse events
were reported.
About PTG-300 and Hepcidin
PTG-300, an injectable hepcidin mimetic, is currently in
clinical development for the potential treatment of anemia and iron
overload related to rare blood disorders. Ultimately PTG-300 also
has the potential to treat secondary iron overload in such diseases
by reducing the need for transfusions and by decreasing excessive
dietary iron absorption. Protagonist plans to initiate a Phase 2
study of PTG-300 in patients with beta-thalassemia in Q4 2018.
PTG-300 therapy may also be potentially beneficial in other
diseases such as myelodysplastic syndrome (MDS), myelofibrosis,
hereditary hemochromatosis (HH), polycythemia vera (PCV),
siderophilic infections, and liver fibrosis which provide
additional opportunities for future development. The U.S. Food
Administration granted Orphan Drug Designation to PTG-300 for
the treatment of beta-thalassemia in March of 2018.
Hepcidin is a natural peptide hormone that is the main
regulatory hormone governing iron absorption, recycling and
utilization by the body. Iron plays an essential role in various
body functions, especially blood formation, but too much iron is
toxic and causes anemia and organ damage over time. Abnormally low
hepcidin levels, caused by genetic mutations or secondary
pathology, can result in the body absorbing and storing more iron
than is needed, leading to iron overload.
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-100 is an oral alpha-4-beta-7 integrin antagonist
peptide that is under evaluation for potential treatment of
inflammatory bowel diseases. The Company's interleukin-23 receptor
antagonist peptide, PTG-200, is currently in a Phase 1 clinical
trial in healthy volunteers to support a Phase 2 study in Crohn's
disease. The IL-12/23 pathway blockade is an approach that has been
validated through an FDA-approved injectable antibody drug. The
company has entered into a worldwide license and collaboration
agreement with Janssen Biotech for the clinical development of
PTG-200. Protagonist has also applied its innovative peptide
platform outside of gastrointestinal disease areas and is
developing an injectable hepcidin mimetic, PTG-300, for the
potential treatment of anemia and iron overload related to rare
blood diseases with an initial focus on beta-thalassemia. The
Company has completed a Phase 1 clinical trial with PTG-300, which
established pharmacodynamic-based clinical proof-of-concept in
normal healthy volunteers. The U.S. Food and Drug Administration
has granted Orphan Drug Designation to PTG-300 for
beta-thalassemia.
Protagonist is headquartered in Newark, California, with pre-clinical and
clinical staff in California and
discovery operations in both California and Brisbane, Queensland, Australia. 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 for our programs, our
collaborations and milestone payments we may receive under them,
the initiation and availability of results of our clinical trials,
our research and development plans, the utility of our intellectual
property, and the adequacy of our capital resources. In some cases,
you can identify these statements by forward-looking words such as
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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, 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. We discuss many of these risks in greater detail
under the heading "Risk Factors" contained in our quarterly report
on Form 10-Q for the three months ended March 31, 2018 filed with the Securities and
Exchange Commission. Forward-looking statements are not guarantees
of future performance, and our actual results of operations,
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SOURCE Protagonist Therapeutics, Inc.