Agios Pharmaceuticals has reached a pivotal milestone with its secondary PK activator, easing transfusion requirements for specific anaemia patients and setting the stage for the subsequent phase of a Phase 2 study in mid-2023.
On Monday, the specialized disease company announced the successful achievement of “proof-of-concept” in a Phase 2a trial involving AG-946, a pyruvate kinase (PK) activator aimed at treating anemia in patients suffering from lower-risk myelodysplastic syndromes. The primary objective focused on effectiveness in participants with a low transfusion dependency.
Agios revealed that four out of 10 patients from this subset met the “transfusion independence endpoint.” Additionally, one patient among the enrolled 22 achieved the haemoglobin response endpoint within the 16-week treatment duration.
Sarah Gheuens, M.D., Ph.D., Agios’ Chief Medical Officer and R&D head, expressed her satisfaction with the outcomes in a statement. She mentioned that “[a] significant reduction in transfusions enables patients to possibly reduce clinic visits and enjoy an enhanced quality of life.” The forthcoming placebo-controlled segment will offer a more rigorous evaluation of the medication’s efficacy, with results anticipated around mid-2024.
Both the preliminary research program and AG-946 follow behind Pyrukynd, an FDA-approved treatment for PK deficiency that received approval in February 2022.