Agios Blood Disorder Drug Achieves Phase III Success

Agios Pharmaceuticals revealed on Monday that its oral drug Pyrukynd (mitapivat), a pyruvate kinase activator, has successfully met its primary target in a Phase III clinical trial focused on adults with transfusion-dependent alpha- or beta-thalassemia.

The Phase III trial, titled ENERGIZE-T, aimed to achieve a significant reduction in transfusions. Specifically, a key goal was to lower the number of transfused red blood cell units by at least 50%. The results were promising, as 30.4% of patients treated with Pyrukynd showed this level of reduction, compared to only 12.6% in the placebo group, marked by a statistically significant p-value of 0.0003.

Further, patients in the Pyrukynd group also demonstrated significant success in attaining the secondary goal—transfusion independence, defined as being free from transfusions for eight consecutive weeks or more. Additionally, Pyrukynd showed a consistent statistical benefit in other measures of transfusion reduction. Both Pyrukynd and the placebo groups reported similar adverse events.

Agios intends to present a comprehensive analysis of the ENERGIZE-T trial's data at a future medical conference.

These new findings come after Pyrukynd achieved its primary goal in another Phase III trial focused on patients with non-transfusion-dependent alpha- or beta-thalassemia in January 2024. In that study, a notable 42.3% of patients treated with Pyrukynd responded, compared to just 1.6% in the placebo group.

Sarah Gheuens, Chief Medical Officer and Head of R&D at Agios, commented on the significance of the trial findings. According to her, the data from both Phase III ENERGIZE trials emphasize mitapivat’s potential to improve red blood cell health, offering a significant oral treatment option for thalassemia patients regardless of their transfusion needs.

Agios plans to file a marketing application in the United States by the end of the year, incorporating data from both Phase III trials. Pyrukynd had already received FDA approval in 2022 for treating hemolytic anemia in adults with pyruvate kinase (PK) deficiency.

Moreover, Agios is preparing to submit marketing applications for Pyrukynd in Europe and Gulf Cooperation Council countries, which include Saudi Arabia and the United Arab Emirates.

Maria Domenica Cappellini, a professor of internal medicine at the University of Milan, Italy, highlighted the importance of these findings. She noted that mitapivat, due to its ability to significantly reduce the transfusion burden in alpha- and beta-thalassemia patients and its convenient oral formulation, has the potential to become a groundbreaking treatment for thalassemia.

However, Agios may face competition in the beta-thalassemia market. The FDA recently approved Casgevy, a gene-edited cell therapy developed by Vertex Pharmaceuticals and CRISPR Therapeutics, in January.

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