Protagonist Initiates Phase 2 Trial for Treatment of Beta Thalassemia

Protagonist Therapeutics, Inc. (Nasdaq:PTGX) today announced that the first patient has been dosed in the Phase 2 study of PTG-300, an injectable hepcidin mimetic peptide in development for the treatment of patients with beta thalassemia, a rare disease characterized by chronic anemia and iron overload. The study is designed to evaluate the safety and preliminary efficacy of PTG-300 in patients with transfusion-dependent or non-transfusion-dependent beta thalassemia. In a completed Phase 1 study in healthy volunteers, administration of PTG-300 was well tolerated and demonstrated a dose-related and sustained reduction in serum iron levels.

“We are encouraged to move forward with clinical development of PTG-300 in patients with beta thalassemia,” commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. “In addition to beta thalassemia, PTG-300 has broad potential in the treatment of other disorders, including hereditary hemochromatosis and the myeloproliferative neoplasms polycythemia vera and myelodysplastic syndrome. The Phase 2 trial incorporates an open label trial design and we expect to report initial results in the second half of 2019. We are actively evaluating additional disease indications for development of PTG-300 and plan to commence on a second indication in the second half of 2019.”

The global Phase 2 study is a single-arm, open label, multiple-ascending dose design which will evaluate safety, proof-of-concept and dose finding in approximately 84 adolescent and adult patients with anemia associated with non-transfusion-dependent or transfusion-dependent beta thalassemia. Non-transfusion-dependent patients will receive 12 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in non-transfusion-dependent patients will be change in hemoglobin from baseline. Transfusion-dependent patients will receive 16 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in transfusion-dependent patients will be a change in transfusion burden from baseline.

https://www.biospace.com/article/releases/protagonist-therapeutics-initiates-phase-2-trial-of-novel-hepcidin-mimetic-ptg-300-for-the-treatment-of-patients-with-beta-thalassemia/