Protagonist Therapeutics Begins Phase 2 Clinical Trial of PTG-300 for Treatment of Patients with Beta Thalassemia
January 10, 2019 – Protagonist Therapeutics has begun its Phase 2 clinical trial to evaluate the safety and preliminary efficacy of PTG-300, the company’s sub-cutaneous injectable hepcidin mimetic, for the treatment of patients with transfusion-dependent or non-transfusion-dependent beta thalassemia. During the now completed Phase 1 study, PTG-300 demonstrated a sustained reduction in serum iron levels in healthy volunteers. Protagonist Therapeutics has now moved forward with the second phase of clinical development of PTG-300 in patients with beta thalassemia.
This global Phase 2 study includes approximately 84 adolescent and adult patients with non-transfusion-dependent or transfusion-dependent beta thalassemia. Non-transfusion-dependent patients will receive 12 weeks of treatment with PTG-300 in escalating dose cohorts, and transfusion-dependent patients will receive 16 weeks of treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in non-transfusion-dependent patients will be change in hemoglobin from baseline after 12 weeks of treatment, while the primary efficacy endpoint in transfusion-dependent patients will be a change in transfusion burden from baseline after 16 weeks of treatment.