Agios posts EHA 2026 investor event presentation on rare hematology pipeline

Agios Pharmaceuticals, Inc.

Agios Pharmaceuticals, Inc.

AGIO

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  • Agios Pharmaceuticals published an EHA 2026 investor event presentation highlighting Phase 3 results for mitapivat in sickle cell disease.
  • RISE UP trial: hemoglobin response 40.6% on mitapivat vs 2.9% on placebo (p<0.0001); response maintained through Week 52.
  • Responders on mitapivat showed a 1.6 g/dL mean increase in average hemoglobin from Week 24 to Week 52.
  • Sickle cell pain crises annualized rate fell 14% vs placebo; p-value 0.1213.
  • Pipeline update: tebapivat Phase 2 data expected in H2; cevidoplenib Phase 3 planned for H1 2028; AG-236 posted positive Phase 1 data.


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