Agios Pharmaceuticals (NASDAQ: AGIO) stocks rose ~33% premarket on Wednesday after a late-stage global trial of mitapivat in adults with non-transfusion-dependent alpha or beta thalassemia achieved its primary endpoint of hemoglobin response.
Results from the phase 3 ENERGIZE study showed that 42.3% of patients in mitapivat dosing regimen achieved a hemoglobin response, compared with 1.6% of patients in the placebo group (2-sided P < 0.0001).
Treatment with mitapivat also demonstrated statistically significant improvements compared to placebo in both key secondary endpoints. Overall, during the 24-week double-blind period, the incidence of adverse events was similar in the mitapivat and placebo arms.
Agios (AGIO) is advancing the fully enrolled Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent alpha or beta thalassemia and expects to announce data from this 48-week study in mid-2024.
It intends to apply for regulatory approval of mitapivat as a treatment for thalassemia in late 2024, incorporating all available data from both studies.
