Positive Phase III ACTIVATE-Kids study of Agios’ mitapivat

USA-based Agios Pharmaceuticals (Nasdaq: AGIO) has announced that the ACTIVATE-Kids Phase III study of mitapivat in children aged 1 to <18 years with pyruvate kinase (PK) deficiency who are not regularly transfused achieved its primary endpoint of hemoglobin response.

In the ACTIVATE-Kids 20-week double-blind treatment period, the safety results were consistent with the safety profile for mitapivat previously observed for adult patients with PK deficiency who are not regularly transfused. In August 2024, Agios also reported to-line results from the ACTIVATE-KidsT Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are regularly transfused.

Under the trade name Pyrukynd, the drug has been approved in the USA since 2022 to treat hemolytic anemia associated with PK deficiency in adults based on data from the ACTIVATE program, but Agios is hoping to expand its use to children and potentially other conditions like thalassemia and sickle cell disease (SCD).