Agios Pharmaceuticals, Inc. announced its anticipated 2024 milestones and value-driving catalysts through 2026 that support the company?s mission to transform patient outcomes in rare diseases. Anticipated 2024 Milestones: Thalassemia: Following the announcement of topline data from the Phase 3 ENERGIZE study last week, Agios plans to report topline data from the Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year) and submit a New Drug Application (NDA) for mitapivat in thalassemia (year-end); Sickle Cell Disease: Complete enrollment in the Phase 3 portion of the RISE UP study of mitapivat (year-end); Pediatric PK Deficiency: Complete enrollment in the Phase 3 ACTIVATE-kids study of mitapivat (mid-2024). Report topline data from Phase 3 ACTIVATE kids-T study (year-end); Lower-risk Myelodysplastic Syndromes (LR-MDS): Dose first patient in Phase 2b study of AG-946 (mid-year); and Earlier-stage Pipeline: Dose the first patient in the Phase 1 study of PAH stabilizer for the treatment of PKU (H1 2024).

Four Additional Phase 3 Readouts and Two Potential New Indication Approvals Expected by End of 2026 2024: Data readout from Phase 3 ENERGIZE study of mitapivat in non-transfusion-dependent thalassemia (announced January 3, 2024); Data readout from Phase 3 ENERGIZE-T study of mitapivat in transfusion-dependent thalassemia (mid-year); and Data readout from Phase 3 ACTIVATE kids-T study of mitapivat in pediatric PK deficiency (year-end). 2025: Data readout from Phase 3 portion of the RISE UP study of mitapivat in sickle cell disease; Data readout from Phase 3 ACTIVATE kids study of mitapivat in pediatric PK deficiency; and Potential FDA approval for mitapivat in thalassemia. 2026: Potential FDA approval for mitapivat in sickle cell disease and Potential FDA approval for mitapivat in pediatric PK deficiency.