Agios Pharmaceuticals’ Pyrukynd met both the primary and secondary endpoints in patients with transfusion-dependent alpha- or beta-thalassemia.
Agios Pharmaceuticals announced Monday that its drug Pyrukynd (mitapivat), an oral pyruvate kinase activator, reached its primary endpoint in a Phase III trial evaluating adults with the blood disorder transfusion-dependent alpha- or beta-thalassemia.
The Phase III ENERGIZE-T trial’s primary endpoint was transfusion reduction, defined as a 50% or lower reduction in transfused red blood cell units. Treatment with the drug was statistically significant compared to placebo, as the Pyrukynd arm showed 30.4% of patients reached a transfusion reduction response compared to just 12.6% in the placebo group, with a p-value of 0.0003.
A higher number of patients in the Pyrukynd arm also reached the secondary endpoint of transfusion independence, defined as being transfusion-free for eight or more consecutive weeks. Patients on the drug also showed a statistically significant reduction in additional measures of transfusion reduction response. Adverse events were similar across both Pyrukynd and placebo groups.
Agios plans to present a more detailed analysis of ENERGIZE-T’s data at an unspecified medical meeting.
The trial’s results come as Pyrukynd reached its primary endpoint of hemoglobin response in a Phase III trial in patients with non-transfusion-dependent alpha- or beta-thalassemia in January 2024. In that trial, 42.3% of patients in the Pyrukynd arm netted a response versus just 1.6% in the placebo arm.
“Building on the compelling data generated in the Phase III ENERGIZE study of mitapivat in adults with non-transfusion-dependent alpha- or beta-thalassemia announced earlier this year, today’s results underscore the potential of mitapivat, with its unique mechanism of action improving red blood cell health, to be a meaningful oral treatment option for all thalassemia patients, regardless of transfusion needs,” Sarah Gheuens, chief medical officer and head of R&D at Agios, said in a statement.
Gheuens said the company plans to submit a marketing application in the U.S. by the end of the year, which will include data from both trials. Pyrukynd was approved by the FDA in 2022 to treat hemolytic anemia in adults with pyruvate kinase (PK) deficiency.
Agios is also looking to submit marketing applications for the drug in Europe and the Gulf Cooperation Council countries, which include Saudi Arabia and the United Arab Emirates.
“Based on these data demonstrating that treatment with mitapivat significantly reduces transfusion burden across alpha- and beta-thalassemia patients, along with its convenient oral formulation, mitapivat has the potential to become a novel advancement in care for thalassemia patients,” Maria Domenica Cappellini, a professor of internal medicine at the University of Milan, Italy, said in a statement.
Agios potentially will face competition in the beta-thalassemia market, as the FDA approved Vertex Pharmaceuticals and CRISPR Therapeutics’ Cas9 gene-edited cell therapy Casgevy in January.
Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.
