Protagonist (PTGX) Starts Study On PTG-300 For Blood Disorder

 | Jan 06, 2020 09:59PM ET

Protagonist Therapeutics, Inc. (NASDAQ:PTGX) announced that it has initiated a phase II study to evaluate its novel hepcidin mimetic candidate, PTG-300, in patients with hereditary hemochromatosis, a rare blood disorder. The disease results in accumulation of iron in the body's tissues and organs. It can lead to restrictive cardiomyopathy, diastolic dysfunction, heart failure, cirrhosis, and other effects, including an increased risk of hepatocellular carcinoma.

The study will evaluate PTG-300 in patients over 24 weeks for reduction in serum transferrin saturation (“TSAT”), serum iron levels and phlebotomy requirements. The candidate has shown potential to reduce TSAT level in other ongoing clinical studies. Current treatment options for hereditary hemochromatosis include regular phlebotomy (removal of blood) and hormone replacement therapy, which can be a significant burden to patients. Successful development of PTG-300 may reduce the burden.

The company is also developing PTG-300 in mid-stage studies as a treatment for beta-thalassemia and polycythemia vera. The candidate enjoys Orphan Drug designation and Fast Track status in the United States as a treatment for beta-thalassemia.

Shares of Protagonist have declined 6% in the past year compared with the industry ’s decrease of 2.5%.