Agios’ Sickle Cell Trial Falls Short as Mitapivat Fails to Significantly Reduce Pain Crises
Agios' Phase 3 Rise Up trial for mitapivat (Pyrukynd) in sickle cell disease missed its primary endpoint of significantly reducing the annualized rate of sickle cell pain crises compared to placebo.
The annualized rate of pain crises was 2.62 in the mitapivat group versus 3.05 in the placebo group, which did not reach statistical significance.
Mitapivat did meet the secondary endpoint of improving hemoglobin response, with 41% of treated patients achieving at least a 1 g/dL increase versus 3% in placebo.
Patients who responded with improved hemoglobin also experienced clinically meaningful reductions in pain crises and hospital visits.
Agios' share price dropped sharply following the announcement due to the mixed results.
The FDA is reviewing mitapivat for sickle cell disease, but its decision may be influenced by the trial's mixed efficacy and emerging safety concerns.