Agios Reports Phase III Success With Oral Drug For Thalassemia

 
• By Jessica Merrill
  
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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.

Key Takeaways

  • The Phase III ENERGIZE trial in adults with NTD thalassemia achieved the primary endpoint of hemoglobin response, as well as two key secondary endpoints.

However, more data will be needed to clarify the drug’s commercial role in a competitive market. The Phase III ENERGIZE...

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