Christopher Marai's questions to Protagonist Therapeutics (PTGX) leadership • Q4 2019
Question
Christopher Marai of Nomura Securities Co. Ltd. inquired about the development of PTG-300, specifically asking about its use in non-transfusion-dependent beta-thalassemia patients, the company's capacity to run multiple registration trials in 2021, and how the established dose for beta-thalassemia might inform dosing for polycythemia vera (PV) and hereditary hemochromatosis (HH).
Answer
President and CEO Dinesh Patel confirmed that the non-transfusion-dependent patient population is still being pursued, though enrollment is slow as expected. He clarified that the immediate goal is to select the first, not necessarily the only, indication for a pivotal study. Mr. Patel explained that since transfusion-dependent beta-thalassemia has the highest iron burden, the effective doses for PV and HH are logically expected to be the same or lower. The company has tested up to 80 mg weekly and has the safety coverage to go higher if needed.