Agios' Oral Thalassaemia Drug Achieves Primary Goal in Second Study

Agios Pharmaceuticals recently announced that its Phase III study of Pyrukynd (mitapivat) in adults with transfusion-dependent (TD) alpha- or beta-thalassaemia has successfully achieved its primary endpoint. The key objective of the study, known as ENERGIZE-T, was to demonstrate a significant reduction in transfusion requirements among patients. This success bolsters Agios' plans to seek expanded US approval for Pyrukynd, a pyruvate kinase activator, later in the year.

The company revealed on Monday that ENERGIZE-T not only met its primary endpoint but also achieved statistical significance across all key secondary endpoints. This is a significant milestone for Agios as it highlights the potential of Pyrukynd to make a substantial impact on the treatment of thalassaemia, a genetic blood disorder that results in the production of abnormal hemoglobin.

Earlier in the year, Agios had already reported promising results from another Phase III trial called ENERGIZE. This study focused on adults with non-transfusion-dependent alpha- or beta-thalassaemia and successfully met its primary endpoint of hemoglobin (Hb) response. Pyrukynd thereby became the first oral medication to achieve this goal, marking a significant advancement in the treatment landscape for thalassaemia.

In the ENERGIZE-T trial, the top-line results showed a notable difference between the Pyrukynd and placebo groups. Specifically, 30.4% of patients in the Pyrukynd treatment arm achieved a transfusion reduction response, compared to only 12.6% in the placebo arm. This substantial difference underscores the efficacy of Pyrukynd in reducing the need for transfusions among patients with transfusion-dependent thalassaemia.

Pyrukynd is not new to the marketplace. It is already approved in the United States for the treatment of haemolytic anaemia in adults with pyruvate kinase (PK) deficiency. In the European Union, it is approved for the treatment of PK deficiency in adults as well. The successful results from the ENERGIZE-T trial add another layer of potential applications for this versatile drug and pave the way for its expanded approval.

For Agios Pharmaceuticals, these clinical successes are crucial steps toward addressing unmet medical needs in the field of rare genetic disorders. If approved for the new indication, Pyrukynd could offer a much-needed treatment alternative for patients suffering from transfusion-dependent alpha- or beta-thalassaemia, conditions that currently have limited therapeutic options.

In summary, the positive outcomes from the Phase III ENERGIZE-T trial demonstrate the potential of Pyrukynd to significantly reduce transfusion requirements in adults with transfusion-dependent thalassaemia. This accomplishment, along with prior positive results from the ENERGIZE trial, supports Agios Pharmaceuticals' forthcoming application for expanded approval of Pyrukynd in the United States. The broader approval of this drug could mark a significant advancement in the treatment of thalassaemia, offering new hope to patients and healthcare providers alike.