Agios’ Pyrukynd Misses Primary Endpoint in First Pediatric Study for Rare Blood Disorder

Study Overview: Agios Pharmaceuticals conducted the Phase 3 ACTIVATE-KidsT study to evaluate the efficacy of mitapivat (Pyrukynd) in pediatric patients with pyruvate kinase (PK) deficiency who require regular blood transfusions.
Primary Endpoint Missed: The study missed its primary endpoint, as the predefined statistical criteria were not met despite employing Bayesian techniques and data borrowing from the adult ACTIVATE-T study.
Efficacy Data: While the primary endpoint was not achieved, 28.1% of participants receiving mitapivat reduced their transfusion needs, compared to 11.8% in the placebo group. Secondary outcomes like transfusion-free status and normal hemoglobin levels were also observed in the mitapivat group.
Safety Profile: The safety profile of mitapivat in pediatric patients was generally favorable, consistent with previous findings in adults.
Future Studies: Agios has completed enrollment for another study, ACTIVATE-Kids, which focuses on children with PK deficiency who do not frequently receive transfusions. Preliminary data from this study are expected in 2025.
Financial Impact: The company reported Pyrukynd sales of $8.6 million, an increase from the previous year but below consensus estimates. This led to a 4.87% drop in AGIO shares.