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Agios Reports Phase III Success With Oral Drug For Thalassemia

 
• By Jessica Merrill

The company hopes to position mitapivat as an oral treatment for patients across the thalassemia spectrum. A Phase III trial in non-transfusion-dependent alpha- or beta-thalassemia was positive.

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Agios hit the mark in a Phase III trial in thalassemia. • Source: Shutterstock

Agios Pharmaceuticals, Inc. hopes its pyruvate kinase activator mitapivat will be an oral treatment for patients across the spectrum of thalassemia, including alpha- and beta-thalassemia, and both transfusion-dependent and non-transfusion-dependent (NTD) patients. The company reported positive Phase III data for mitapivat in thalassemia on 3 January, positioning it closer to reaching that goal.

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