Protagonist Therapeutics Initiates Phase 2 Study of Novel Hepcidin Mimetic PTG-300 in the Treatment of Patients with Hereditary Hemochromatosis
- Category: Proteins and Peptides
- Published on Monday, 06 January 2020 18:52
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NEWARK, CA, USA I January 6, 2020 I 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.
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.
SOURCE: Protagonist Therapeutics