Agios Pharmaceuticals Plans US Filing for Thalassemia Treatment After Unveiling Positive Data ###

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1. Positive Data: Agios Pharmaceuticals announced positive data from its Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent thalassemia, which met its primary endpoint of hemoglobin response and both key secondary endpoints.
2. US Filing Plans: The company plans to file for regulatory approval of mitapivat as a treatment for thalassemia by the end of 2024, incorporating data from both the ENERGIZE and ENERGIZE-T studies.
3. ENERGIZE-T Study: The fully enrolled Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia is expected to announce topline data in mid-2024.
4. Potential FDA Approval: Agios anticipates potential FDA approval for mitapivat in thalassemia in 2025.
5. Agios' Vision: By 2026, the company aims to establish a classical hematology franchise with approvals across pyruvate kinase deficiency, thalassemia, and sickle cell disease, and achieve cash-flow positivity.