Agios Presented At ASH, Results From Phase 3 ENERGIZE-T Study of Mitapivat; Study Achieved Primary, All Key Secondary Endpoints In Adult Patients With Transfusion-Dependent Alpha- or Beta-Thalassemia
Agios Presented At ASH, Results From Phase 3 ENERGIZE-T Study of Mitapivat; Study Achieved Primary, All Key Secondary Endpoints In Adult Patients With Transfusion-Dependent Alpha- or Beta-Thalassemia
Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today presented positive results from the Phase 3 ENERGIZE-T study investigating mitapivat, an oral, small molecule PK activator, in adults with transfusion-dependent alpha- or beta-thalassemia. These findings were shared in an oral presentation (abstract #409) at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in San Diego, California.
美國Agios Pharmaceuticals公司（納斯達克股票代碼：AGIO）是細胞代謝和丙酮酸激酶（PK）激活領域的領軍者，致力於罕見病治療方面的開創性療法。今日發佈了第3期ENERGIZE-t研究調查mitapivat在依賴輸血的α或β地中海貧血成年患者中的陽性結果。這些發現在加利福尼亞州聖地亞哥舉行的第66屆美國血液學會（ASH）年會及博覽會上以口頭報告（摘要編號＃409）的形式分享。
In the ENERGIZE-T trial, mitapivat demonstrated a statistically significant reduction in transfusion burden compared to placebo in patients with transfusion-dependent alpha- or beta-thalassemia, achieving its primary endpoint. Additionally, the ENERGIZE-T study met all the key secondary endpoints, with mitapivat demonstrating a statistically significant reduction in additional measures of transfusion reduction response compared to placebo. In June 2024, Agios also presented positive results from the Phase 3 ENERGIZE study, which evaluated mitapivat in adults with non-transfusion-dependent alpha- or beta-thalassemia.
在ENERGIZE-t試驗中，米他匹嗪與安慰劑相比顯著減少了依賴輸血的α或β-地中海貧血患者的輸血負擔，實現了主要終點。此外，ENERGIZE-t研究實現了所有關鍵次要終點，米他匹嗪表現出與安慰劑相比，在進一步減少輸血反應措施方面呈現顯著統計學減少。2024年6月，Agios還就在非依賴輸血的α或β-地中海貧血成年患者中評估米他匹嗪的第3期ENERGIZE研究，提出了積極的結果。
