Protagonist Therapeutics Initiates Phase 2 Study of Novel Hepcidin Mimetic PTG-300 in the Treatment of Patients with Hereditary Hemochromatosis
"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
"Current treatments for hereditary hemochromatosis, including periodic phlebotomy, can be a significant burden to patients," commented
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.
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
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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
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