Agios' Mitapivat Phase 3 Study Achieves Primary and Secondary Endpoints in NTD Thalassemia Adults

Agios Pharmaceuticals has reported positive results from a Phase 3 clinical trial of mitapivat, a treatment for non-transfusion-dependent (NTD) alpha- or beta-thalassemia. The study, known as ENERGIZE, showed that mitapivat significantly increased hemoglobin levels compared to a placebo, which is a key measure of the disease's severity. Additionally, the drug demonstrated improvements in fatigue scores and overall hemoglobin concentration.

The ENERGIZE study involved 194 participants, with 130 receiving mitapivat and 64 receiving a placebo. The primary endpoint was met, with a 42.3% response rate in the mitapivat group versus 1.6% in the placebo group. The drug's efficacy was supported by statistical significance in secondary endpoints, which included changes in fatigue scores and hemoglobin levels from baseline to the 24-week mark.

Agios' Chief Medical Officer, Sarah Gheuens, highlighted the importance of these findings, stating that they bring the company closer to offering a treatment for all thalassemia patients. The company is also conducting the ENERGIZE-T study for transfusion-dependent thalassemia patients and plans to seek regulatory approval for mitapivat by the end of 2024.

Mitapivat has the potential to be the first oral treatment for NTD thalassemia, a condition that affects a significant portion of thalassemia patients and for which there are currently no approved oral treatments. The drug's success in the trial marks a significant step towards addressing an unmet need in this patient population.

The safety profile of mitapivat was found to be comparable to placebo during the 24-week study period, with adverse events leading to discontinuation occurring in a small percentage of participants in the mitapivat group.

Agios intends to present more detailed findings at an upcoming medical conference and will host an investor webcast to discuss the results and next steps for the ENERGIZE-T study. The company's focus on rare diseases and cellular metabolism positions it at the forefront of developing transformative therapies for conditions with limited treatment options.