– Prespecified Statistical Criterion for the Primary Endpoint in ACTIVATE-KidsT Was Not Met; Results Were Clinically Meaningful, with Observed Response Rates Higher for Mitapivat than Placebo for the ...

– ACTIVATE-Kids is the First Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency Who Are Not Regularly Transfused – – Safety Results Consistent with Safety Profile for ...

Findings showed 28.1% of patients treated with mitapivat achieved a TRR vs 11.8% of patients who received placebo. A phase 3 trial evaluating mitapivat in pediatric patients with pyruvate kinase ...

Mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat. Topline data from ACTIVATE-Kids study of mitapivat in children who are not ...