Agios Pharmaceuticals reports results from Phase 3 ACTIVATE-Kids study

Agios Pharmaceuticals (AGIO) announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to less than18 years with 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 topline 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 positive results for the ACTIVATE-Kids Phase 3 trial represent a very important step forward for the PK deficiency community, building on the clinical benefits demonstrated by mitapivat in adults with PK deficiency. The ACTIVATE-Kids and ACTIVATE-KidsT Phase 3 studies mark Agios’ first pediatric clinical program for a rare hemolytic anemia, providing valuable insights that will help shape the company’s future clinical programs evaluating mitapivat in pediatric patients with thalassemia and sickle cell disease,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “We are deeply grateful to all who are contributing to the ACTIVATE-Kids and ACTIVATE-KidsT trials, especially the children who are participating in the studies and their caregivers. With data now available from the randomized, placebo-controlled, double-blind period of both Phase 3 pediatric PK deficiency studies, we look forward to sharing more detailed findings with the community and interacting with regulators.”