Stage (next event)
Phase 2 (Data)
Catalyst Info & Data Links
TITLE: PTG-300 (Hepcidin Mimetic) for Hereditary Hemochromatosis (HH) - Phase 2 Data
ClinicalTrials.gov (NCT04057040): Hepcidin Mimetic in Patients With Polycythemia Vera
WHAT IS THE CATALYST EVENT?
Phase 2 Data
WHEN WILL THE EVENT (OR DID THE EVENT) OCCUR?
Mechanism of Action
MECHANISM OF ACTION
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.
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.
Updated by HC
Prior Data (click to view full image)
Trial Design / Revenue (click to view full image)
See what the community is saying - click to see full post