Agios unveils 2023-2026 catalysts: PYRUKYND approvals, pipeline expansion, cash-flow positivity by 2026

AGIOS PHARMACEUTICALS, INC.

Five ongoing pivotal PYRUKYND trials in thalassemia, sickle cell, and pediatric PK deficiency.
Potential FDA approvals: thalassemia 2025, sickle cell 2026, pediatric PK deficiency 2026.
Expects to complete ENERGIZE/ENERGIZE-T enrollment by mid-2023; RISE UP Phase 2 data and go/no-go by mid-year.
AG-946 Phase 2a enrollment in LR-MDS completed by year-end 2023; IND filing for PAH stabilizer in PKU planned.
Appointed Tsveta Milanova as CCO; EU/Great Britain approval for PYRUKYND in adult PK deficiency already received.

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