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"The Phase 1 clinical results demonstrated the ability of PTG-300 to achieve a sustained, dose-related reduction in serum iron," commented
"These pre-clinical and clinical results demonstrate the potential of PTG-300 in the treatment of a broad range of blood disorders," added
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.
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