Agios to highlight PK activation portfolio with data in blood disorders at EHA

Agios Pharmaceuticals (AGIO) announced that new data on the company’s PK activators, mitapivat and tebapivat, will be featured in oral and poster presentations during the 30th European Hematology Association Congress in Milan, Italy, June 12-15, 2025. “The clinical results and scientific insights being presented at EHA add to the robust body of efficacy and safety data demonstrating the promise of PK activation in treating both adults and children with rare blood disorders,” said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. Select presentations and publications at EHA 2025 will include: An oral presentation of results from the ACTIVATE-KidsT Phase 3 study of mitapivat in children aged 1 to less than18 years with PK deficiency who are regularly transfused. The study showed a clinically meaningful reduction in transfusion burden with mitapivat, with a higher proportion of patients achieving the primary endpoint of transfusion reduction response compared to placebo, though the prespecified statistical criterion was not met. Safety results were consistent with the safety profile for mitapivat previously observed in adults with PK deficiency who are regularly transfused. The ACTIVATE-KidsT study, along with the positive ACTIVATE-Kids Phase 3 trial, represents the successful execution of Agios’ first pediatric clinical program. An oral presentation of long-term data from the investigator-led ESTIMATE Phase 2 trial, an open-label study investigating mitapivat in patients with sickle cell disease. In the study, mitapivat showed sustained efficacy and tolerability over three years, including improvements in anemia, hemolysis, painful vaso-occlusive crises, and markers of kidney damage. A poster presentation with preclinical data demonstrating that tebapivat reduced red blood cell sickling and adhesion in blood samples from sickle cell disease patients, highlighting its therapeutic potential in this patient population. A preclinical publication examining expression patterns of PKM2, an isoform of the PK enzyme. The study found that, compared with healthy controls, patients with myelodysplastic syndromes have significantly reduced PKM2 expression in CD34+ hematopoietic stem cells, which may be implicated in the development of MDS. These findings further support the ongoing investigation of tebapivat in lower-risk MDS, as it activates PKM2 in addition to PKR.