Protagonist Therapeutics has dosed the first patient in a Phase II trial of PTG-300 for the treatment of patients with beta thalassemia.

Beta thalassemia is a rare disease caused by chronic anemia and iron overload.

The trial aims to study the safety and preliminary efficacy of PTG-300 in around 84 adolescent and adult patients.

It features a global, single-arm, open label, multiple-ascending dose setting.

The studuy will include patients with transfusion-dependent or non-transfusion-dependent beta thalassemia.

“PTG-300 has broad potential in the treatment of other disorders, including hereditary hemochromatosis.”

Non-transfusion-dependent patients will be treated with PTG-300 in ascending dose over a period of 12 weeks, while the transfusion-dependent patients will receive PTG-300 in an escalating dose for 16 weeks.

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Primary efficacy objective for the non-transfusion-dependent patients and the transfusion-dependent patients include the change in hemoglobin from baseline and change in transfusion burden from baseline, respectively.

Protagonist Therapeutics president and CEO Dinesh Patel said: “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 II 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.”

In a closed Phase I trial, PTG-300 was found to be well tolerated as well as showed a dose-related and sustained reduction in serum iron levels among healthy participants.