Published January 4, 2024

In a significant development, Agios Pharmaceuticals (AGIO.O) has reported positive results from its late-stage study of the drug mitapivat, bringing the company closer to obtaining approval for the first-ever oral treatment for less severe forms of thalassemia, an inherited blood disorder. The study demonstrated a statistically significant increase in hemoglobin response compared with a placebo in patients with either alpha- or beta-thalassemia who were not dependent on transfusion.

Mitapivat’s Success in the Late-Stage Study: Mitapivat, the oral drug developed by Agios, met its main goal by showcasing a notable increase in hemoglobin levels in patients with thalassemia who did not require transfusions. Thalassemia is characterized by lower-than-normal levels of hemoglobin, and existing treatments for moderate-to-severe cases involve regular blood transfusions and stem cell transplants.

Positive Secondary Goals Achieved: Additionally, mitapivat successfully achieved both of its secondary goals in the 194-patient study, demonstrating an improvement in scores measuring fatigue in patients. This success marks a significant milestone in the development of an effective treatment for thalassemia patients who do not rely on regular blood transfusions.

Unique Positioning in the Thalassemia Treatment Landscape: While current treatments cater to beta-thalassemia patients requiring regular blood transfusions, non-transfusion dependent alpha- and beta-thalassemia patients lack a U.S.-approved drug. Agios believes that mitapivat has the potential to become the first oral therapy approved for non-transfusion dependent thalassemia, filling a critical gap in available treatments.

Future Prospects and Regulatory Approval: Agios anticipates data from thalassemia patients dependent on transfusions by mid-2024 and is concurrently testing mitapivat in individuals with sickle cell disease. The company aims for potential U.S. approval next year to treat all types of thalassemia, as stated by Chief Medical Officer Sarah Gheuens. The Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent thalassemia is underway, with topline data expected in mid-2024. Agios plans to file for regulatory approval of mitapivat as a thalassemia treatment by the end of 2024.

Mitapivat’s Previous FDA Approval and Expanded Applications: Mitapivat, initially approved by the U.S. Food and Drug Administration in 2022 under the brand Pyrukynd, is currently used for the treatment of hemolytic anemia caused by the deficiency of the enzyme pyruvate kinase. The promising results from the recent study underscore the drug’s potential to address various forms of thalassemia, broadening its applications in the field of blood disorder treatments.

This breakthrough not only signifies a significant advancement in thalassemia treatment but also positions Agios Pharmaceuticals as a key player in the evolving landscape of oral therapies for inherited blood disorders.