Agios Pharmaceuticals’ mitapivat meets primary endpoint in Phase 3 thalassemia trial

Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), renowned for its innovative therapies in cellular metabolism and pyruvate kinase (PK) activation, has announced promising results from its global Phase 3 ENERGIZE-T study. The study focused on mitapivat, a novel treatment for adults with transfusion-dependent alpha- or beta-thalassemia, achieving its primary endpoint of significant transfusion reduction, along with all key secondary endpoints, marking a significant stride in thalassemia care.

Key Outcomes of the ENERGIZE-T Study of Mitapivat

The ENERGIZE-T study enrolled 258 patients, testing the efficacy of mitapivat in reducing the need for transfusions, a common and burdensome requirement for thalassemia patients. Results showed a remarkable 30.4% of patients in the mitapivat group achieving at least a 50% reduction in transfusion requirements, compared to only 12.6% in the placebo group, a statistically significant improvement. The study also noted a commendable safety profile, with similar incidence of adverse events across both study groups.

Expert Opinions and Future Prospects

Dr. Sarah Gheuens, Chief Medical Officer and Head of R&D at Agios, highlighted the potential of mitapivat, stating, “Today’s results underscore the potential of mitapivat to be a meaningful oral treatment option for all thalassemia patients, regardless of transfusion needs.” She also expressed gratitude towards the patients, collaborators, and clinical communities involved in the study.

Professor Maria Domenica Cappellini, from the University of Milan, emphasized the transformative potential of the study’s results for thalassemia patient care, pointing out the significant need for alternative treatment options that reduce the risks associated with frequent transfusions.

Regulatory Plans and Anticipated Impact

With these encouraging results, Agios Pharmaceuticals plans to submit a marketing application for PYRUKYND® (mitapivat) in the U.S. by the end of 2024, aiming to extend its use to thalassemia treatment. Applications in Europe and the Gulf Cooperation Council (GCC) countries are also on the agenda, potentially making mitapivat a global solution for thalassemia patients.

The data from the ENERGIZE and ENERGIZE-T studies collectively suggest that mitapivat could significantly improve the quality of life for patients with thalassemia by reducing their dependence on transfusions and mitigating associated health risks.

Agios Pharmaceuticals’ successful completion of the ENERGIZE-T study is a pivotal moment in the fight against thalassemia. The company is set to present detailed data from this study at upcoming medical meetings, including a plenary session at the European Hematology Association 2024 Hybrid Congress. This ongoing research and development effort underscores Agios’s commitment to pioneering significant advancements in the treatment of rare diseases through innovative therapeutic approaches.