Research & Development
Protagonist Therapeutics Receives Orphan Drug Designation from FDA for PTG-300 for Treatment of Beta-Thalassemia
9 March 2018 - - The US Food and Drug Administration has granted Newark, California-based peptide-based drugs developer Protagonist Therapeutics, Inc. (NASDAQ: PTGX) orphan drug designation for PTG-300, the company's sub-cutaneous injectable hepcidin mimetic, for the treatment of beta-thalassemia, a rare disease characterized by chronic anemia and iron overload, the company said.
Protagonist recently completed a Phase 1 study of PTG-300 that established pharmacodynamic-based clinical proof-of-concept by achieving dose-related and sustained reductions in serum iron levels in normal healthy volunteers.
It was well tolerated with no serious adverse events or dose-limiting toxicities.
PTG-300 is currently in clinical development for the potential treatment of beta-thalassemia, and may also be potentially beneficial in other diseases such as myelodysplastic syndrome, hereditary hemochromatosis, polycythemia vera, siderophilic infections, and liver fibrosis.
Hepcidin is a natural peptide hormone that is the main regulatory hormone governing iron absorption, recycling and utilization by the body. 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.
Protagonist Therapeutics' primary focus is on developing potential first-in-class oral targeted therapy-based peptide drugs that work by blocking biological pathways that are currently targeted by marketed injectable antibody drugs.
The company is headquartered in Newark, California with its pre-clinical and clinical staff in California, and discovery operations both in California and in Brisbane, Queensland, Australia.
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