Agios Pharmaceuticals (NasdaqGS:AGIO) Earnings Call Presentation

RISE UP Phase 3 Trial Results - Mitapivat met the primary endpoint with a 40.6% hemoglobin response rate, defined as ≥1.0 g/dL increase in average Hb from Week 24 through Week 52 compared to baseline, significantly higher than the 2.9% in the placebo arm[38] - There was a trend favoring mitapivat with a 14% reduction in the annualized rate of sickle cell pain crises (SCPCs) compared to placebo, although this did not reach statistical significance[39] - Mitapivat showed a statistically significant improvement in average change from baseline in hemoglobin concentration, with a 7.43 g/L difference compared to placebo[41] - Mitapivat significantly reduced the average change from baseline in indirect bilirubin compared to placebo, with a difference of -16.91 µmol/L[46] - The trial showed a trend in favor of mitapivat with a 14% reduction in the annualized rate of hospitalizations for SCPC compared to placebo[50] - Percent reticulocytes substantially decreased from baseline in the mitapivat arm vs placebo[52] Safety and Discontinuation - The safety profile observed in the RISE UP Phase 3 trial was consistent with previous mitapivat sickle cell disease trials, with no new safety signals identified[57] - The discontinuation rate in the double-blind treatment period was low, with 13% in the mitapivat arm and 18.8% in the placebo arm[34] Next Steps - The company intends to submit a marketing application for mitapivat in the U S for sickle cell disease after a pre-sNDA meeting with the FDA in Q1 2026[61, 68]