Agios Pharma Phase 3 ENERGIZE Study Of Mitapivat Meets Primary Goal

(RTTNews) - Agios Pharmaceuticals, Inc. (AGIO) Wednesday said its Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary goal of hemoglobin response.

42.3% of patients treated with mitapivat achieved a hemoglobin response, compared to 1.6% of patients who were given placebo.

Hemoglobin response was defined as an increase of more than or equal to 1 g/dL in average hemoglobin concentrations from Week 12 through Week 24 compared with baseline.

Agios is also advancing Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia and expects to announce topline data in mid-2024.

The company plans to file for regulatory approval of mitapivat as a treatment for thalassemia by the end of 2024.