NEWARK, Calif. - Protagonist Therapeutics (Nasdaq: PTGX) ('Protagonist' or 'the Company') 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).
'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.'
'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 potential utility to address unmet needs of patients in these diseases. The study results announced at AASLD will serve as an important foundation for the next steps that we will determine in consultation with key clinical investigators and regulatory agencies. Going forward, our focus will be on proving rusfertide's potential to address unmet medical needs in specific subpopulations of HH.'
Study Design and Summary of Results:
The study was an open-label multicenter Phase 2 study in 16 patients that was designed to evaluate the safety and efficacy of rusfertide as a treatment for patients with HH. The requirement for, and frequency of, therapeutic phlebotomies, a key endpoint of the study, was statistically significant: rusfertide-treated patients had 0.009 phlebotomies per month during the study compared to 0.28 phlebotomies per month pre-study (p
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