RARE Daily

Agios Reports Mitapivat Met Primary Endpoint and Key Secondary Endpoints in NDT Alpha- and Beta-Thalassemia

January 3, 2024

Rare Daily Staff

Agios Pharmaceuticals reported its experimental therapy mitapibat met its primary and key secondary endpoints in its global phase 3 ENERGIZE study in adults with non-transfusion-dependent alpha- or beta-thalassemia, a rare blood disease.

Mitapibat achieved its primary endpoint of hemoglobin response. Statistical significance was also achieved for both key secondary endpoints associated with change from baseline in FACIT-Fatigue Score and hemoglobin concentration.

Mitapivat, marketed as Pyrukynd, is a first-in-class, oral PK activator and the first approved disease-modifying therapy for patients in the EU with this rare, debilitating, lifelong hemolytic anemia. It is indicated for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States, and for the treatment of PK deficiency in adult patients in the European Union.

“The results of the ENERGIZE study support the potential of mitapivat to be the first oral therapy for all NTD thalassemia patients, including those with alpha- or beta-thalassemia,” said Ali Taher, professor of Medicine, Hematology & Oncology and Director of the Naef K. Basile Cancer Institute, American University of Beirut Medical Center in Beirut, Lebanon. “For NTD thalassemia patients across the globe, there are currently no approved oral treatments, and NTD thalassemia has consistently been associated with morbidity and mortality if left untreated. NTD thalassemia represents over half of clinically significant forms of thalassemia, so there is a tremendous unmet need. Based on the data reported to date, mitapivat has the potential to be a foundational treatment option for the thalassemia community.”

Treatment with mitapivat demonstrated a statistically significant increase compared to placebo. Some 42.3 percent of patients in the mitapivat arm achieved a hemoglobin response, compared to 1.6 percent of patients in the placebo arm.

Treatment with mitapivat also demonstrated statistically significant improvements compared to placebo for both key secondary endpoints.

Overall, during the 24-week double-blind period, incidence of adverse events was similar across mitapivat and placebo arms. Four subjects (3.1 percent) in the mitapivat arm experienced adverse events (AEs) leading to discontinuation. There were no AEs leading to discontinuation in the placebo arm.

Agios plans to present a more detailed analysis of the phase 3 ENERGIZE data at an upcoming medical meeting.

“The results of the phase 3 ENERGIZE study underscore the potential of mitapivat to be a meaningful treatment option for adults with non-transfusion dependent alpha- or beta-thalassemia,” said Sarah Gheuens, chief medical officer and head of R&D at Agios. “All subgroup analyses favored the mitapivat treatment arm compared to placebo.”

Photo: Sarah Gheuens, chief medical officer and head of R&D at Agios

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