Agios (AGIO) Up 5% on Upbeat Data From Thalassemia Study

Core Viewpoint - Agios Pharmaceuticals' shares rose 5.2% following positive results from the phase III ENERGIZE study, which evaluated mitapivat for treating non-transfusion-dependent thalassemia patients, achieving both primary and key secondary endpoints [1][3]. Group 1: Study Results - The ENERGIZE study enrolled 194 adult participants with non-transfusion-dependent alpha- or beta-thalassemia, randomized into two groups: 130 received 100mg of mitapivat and 64 received a placebo over 24 weeks [3]. - Patients treated with mitapivat showed a statistically significant hemoglobin response, with 42.3% achieving this compared to only 1.6% in the placebo group [3]. - Key secondary endpoints also showed significant improvements in average FACIT-Fatigue score and hemoglobin concentration, indicating lower fatigue levels in mitapivat patients [3]. Group 2: Market Potential - Agios estimates that the thalassemia patient population in the U.S. is around 8,000, with more than half being non-transfusion-dependent patients, highlighting a significant market opportunity as there are currently no approved therapies for this group [4]. - Mitapivat was previously approved by the FDA in November 2022 for adults with pyruvate kinase deficiency, marketed as Pyrukynd [4]. Group 3: Future Developments - Agios plans to seek regulatory approval for mitapivat as a treatment for all thalassemia sub-types, regardless of transfusion dependency, following the ENERGIZE-T study results expected in mid-2024 [5]. - If approved, mitapivat could have a competitive advantage over existing treatments, as it is an orally administered drug compared to gene therapy and injectable options [6]. - Agios is also conducting the phase II/III RISE UP study for sickle cell disease, with the phase II portion achieving its primary endpoint, and aims for approval by 2026 [6].