A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

Phase 3 Active not recruiting 194 enrolled Agios Pharmaceuticals, Inc.

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The primary purpose of this study was to compare the effect of mitapivat versus placebo on hemolytic anemia in participants with alpha- or beta-non-transfusion dependent thalassemia (NTDT).

Timeline

Start: 2021-12-20
Primary completion: 2023-11-13
Completion: 2028-12

Outcome

Met primary endpoint

registry analysis (superiority test); Mitapivat vs Placebo; p <0.0001; Percentage difference 40.9 (95% CI 32.0 to 49.8) NCT04770753 ↗

paper 55 (42%) of 130 patients in the mitapivat group had a haemoglobin response versus one (2%) of 64 in the placebo group PMID 40544857 ↗

release “the global Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary endpoint of hemoglobin response.” agios.com ↗

Publications

Taher AT, Al-Samkari H, Aydinok Y, Besser M, Boscoe AN, Dahlin JL, De Luna G, Estepp JH, Gheuens S, Gilroy KS, Glenthoj A, Sim Goh A, Iyer V, Kattamis A, Loggetto SR, Morris S, Musallam KM, Osman K, Ricchi P, Salido-Fierrez E, Sheth S, Tai F, Tevich H, Uhlig K, Urbstonaitis R, Viprakasit V, Cappellini MD, Kuo KHM; ENERGIZE investigators. Mitapivat in adults with non-transfusion-dependent alpha-thalassaemia or beta-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial. Lancet. 2025 Jul 5;406(10498):33-42. doi: 10.1016/S0140-6736(25)00635-X. Epub 2025 Jun 19.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Mitapivat	Small molecule	100 mg	Oral