New class of small molecule inches closer to approval

28 January 2021

Agios Pharmaceuticals (Nasdaq: AGIO) has announced positive results from the Phase III ACTIVATE trial of mitapivat.

The study is designed to test the experimental allosteric activator in people with pyruvate kinase (PK) deficiency, who do not receive regular transfusions.

Agios said the trial met its primary endpoint, with a statistically-significant, sustained increase in hemoglobin for the test group, compared to placebo. There were also no surprises on the safety side.

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