Agios Pharmaceuticals’ mitapivat shows significant promise in Phase 3 ENERGIZE study

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a pioneer in cellular metabolism and pyruvate kinase (PK) activation therapies, unveiled detailed results from the Phase 3 ENERGIZE study of mitapivat for non-transfusion-dependent (NTD) alpha- or beta-thalassemia. The findings, presented at the European Hematology Association 2024 (EHA2024) Hybrid Congress in Madrid, Spain, demonstrated significant improvements in hemoglobin levels and health-related quality of life among patients.

Study Achievements and Primary Outcomes

The ENERGIZE study met its primary endpoint, showing a statistically significant increase in hemoglobin response rate for patients treated with mitapivat compared to those receiving a placebo. Additionally, significant results were achieved for key secondary endpoints related to changes in the FACIT-Fatigue Score and hemoglobin concentration from baseline, confirming the effectiveness of mitapivat across various patient subgroups.

Improvements in Patient Health and Quality of Life

Patients in the mitapivat arm reported clinically meaningful enhancements in health-related quality of life measures, including fatigue and walking capacity, as well as overall patient-reported outcomes. These improvements were highlighted in a poster presentation at the congress, underlining the potential of mitapivat as a transformative treatment for thalassemia.

Agios Pharmaceuticals’ ENERGIZE study, showcases mitapivat’s potential in transforming thalassemia treatment.

Expert Insights and Future Prospects

Dr. Sarah Gheuens, Chief Medical Officer and Head of R&D at Agios, commented on the results, noting, “The data from the ENERGIZE study are compelling, with mitapivat-treated patients achieving meaningful improvements in anemia and key measures of how patients feel and function.” The company anticipates filing for U.S. approval by the end of the year, aiming to make mitapivat an essential therapy for all subtypes of thalassemia.

Dr. Ali Taher, an investigator in the study, also expressed optimism about mitapivat’s impact, stating, “Based on the data collected in the ENERGIZE study, mitapivat has the potential to become a foundational treatment for non-transfusion-dependent thalassemia.”

Detailed Study Data and Observations

The study enrolled 194 patients, with 130 randomized to receive mitapivat. Notably, 42.3% of patients in the mitapivat arm achieved a hemoglobin response, compared to only 1.6% in the placebo arm. Improvements were consistent across all prespecified subgroups, including thalassemia genotype and baseline hemoglobin concentration.

Regulatory Pathway and Broader Impact

Agios Pharmaceuticals plans to incorporate these results, along with data from the ENERGIZE-T study for transfusion-dependent thalassemia, in its upcoming regulatory submissions. The broader implications for treatment options in thalassemia are significant, especially considering the current lack of approved oral therapies for the condition.

The ENERGIZE study marks a significant step forward in the treatment of thalassemia, with mitapivat showing potential to significantly improve patient outcomes and quality of life. As Agios Pharmaceuticals moves towards regulatory approval, the thalassemia community watches with hopeful anticipation for a new era of treatment options.