Why Is Blood Disorder Focused Agios Pharmaceuticals Stock Trading Higher Today?

Agios Pharmaceuticals Inc 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.

Overall, during the 24-week period, the incidence of adverse events was similar across the mitapivat and placebo arms. 

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.