Benzinga - by Vandana Singh, Benzinga Editor.
Agios Pharmaceuticals Inc (NASDAQ: AGIO) says the Phase 3 ENERGIZE study of mitapivat in adults with non-transfusion-dependent (NTD) alpha- or beta-thalassemia achieved its primary endpoint of hemoglobin response.
Statistical significance was also achieved for both key secondary endpoints associated with a change from baseline in FACIT-Fatigue Score and hemoglobin concentration.
A total of 194 patients were enrolled in the study, with 130 randomized to mitapivat 100 mg twice-daily (BID) and 64 randomized to matched placebo.
122 (93.8%) in the mitapivat arm and 62 (96.9%) in the placebo arm completed the 24-week double-blind period of the study.
The study met the primary endpoint of hemoglobin response. Hemoglobin response was defined as an increase of ≥1 g/dL in average hemoglobin concentrations from Week 12 through Week 24 compared with baseline.
- 42.3% of patients in the mitapivat arm achieved a hemoglobin response, compared to 1.6% of patients in the placebo arm.
Also Read: Agios Touts Encouraging Action From Sickle Cell Disease Study With Mitapivat
Agios is also advancing the fully enrolled Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha- or beta-thalassemia and expects to announce topline data from this 48-week study in mid-2024.
Following the read-out of ENERGIZE-T, the company intends to file for regulatory approval of mitapivat as a treatment for thalassemia by the end of 2024, incorporating all available data from both studies.
Price Action: AGIO shares are down 0.92% at $22.62 on the last check Wednesday.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.