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Protagonist Therapeutics Announces Positive Phase 2a Data Supporting the Potential Use of Rusfertide as a First-in-Class Therapy in Hereditary Hemochromatosis
Rusfertide demonstrated favorable safety, tolerability, and therapeutic effect, decreasing and normalizing average TSAT and iron levels and reducing
About this update from Protagonist Therapeutics, Inc.
[{"type":"text","content":"Rusfertide demonstrated favorable safety, tolerability, and therapeutic effect, decreasing and normalizing average TSAT and iron levels and reducing phlebotomy frequency during the 24-week study\n Data from 16 patients were presented during an oral presentation session at The Liver Meeting® of the American Association for the Study of Liver Diseases\n\n\nNEWARK, Calif., Nov. 15, 2021 /PRNewswire/ -- Protagonist Therapeutics (Nasdaq: PTGX) (\"Protagonist\" or \"the Company\") today announced results from a Phase 2a study in which rusfertide, an investigational new drug, is being evaluated for the treatment of hereditary hemochromatosis (HH). HH is a genetic disorder arising from a deficiency or dysregulation of the natural hormone hepcidin, a condition which causes the body to absorb too much iron. The clinical data from the study were presented in an oral presentation at The Liver Meeting® of the American Association for the Study of Liver Diseases (AASLD).\n\n \n \n \n \n \n \n\n \n\"Despite its prevalence, no drug therapies have been developed to date for the treatment of hereditary hemochromatosis,\" said Kris V. Kowdley, M.D., Director of Liver Institute Northwest, Professor at the Elson S. Floyd College of Medicine at Washington State University, an author of the ACG Guidelines for HH, and the study's principal investigator. \"I'm encouraged by the efforts Protagonist has undertaken in this area of iron overload diseases, and specifically by the Phase 2 data that demonstrates rusfertide's therapeutic effect by several different measures, including pharmacodynamic effects, reduction in phlebotomy requirements, stabilization of liver iron content, and possible improvements in quality-of-life measures. Hereditary hemochromatosis is characterized by low levels of hepcidin, with variability of severity in clinical manifestations across different patient sub-populations. Rusfertide has the potential to offer a treatment option for patients in whom phlebotomy may be difficult or contraindicated.\"\n\"Rusfertide has the potential to be the first-in-class drug therapy for an HH indication,\" said Dinesh Patel, Ph.D., President and Chief Executive Officer of Protagonist. \"After polycythemia vera, this marks a second indication where rusfertide has demonstrated a positive clinical proof-of-concept, thereby signaling a broader potenti...