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Agios Shows Pyrukynd Can Reduce Transfusions In Thalassemia

 
• By Joseph Haas

Having already posted Phase III success in non-transfusion dependent patients, Agios’s Pyrukynd now has shown the ability to reduce transfusions in alpha and beta thalassemia patients.

Red Blood Cells
Agios says Pyrukynd can improve red blood cell health to treat thalassemia • Source: Shutterstock: fusebulb

Agios Pharmaceuticals, Inc. unveiled another set of Phase III data showing efficacy for its pyruvate kinase inhibitor Pyrukynd (mitapivat) in thalassemia patients on 3 June, showing therapeutic benefit for patients with transfusion-dependent alpha or beta thalassemia, which it will file by the end of 2024 to expand its earlier approval in non-transfusion-dependent patients to cover all thalassemia patients.

Key Takeaways

  • Agios reports Phase III data showing that Pyrukynd can reduce transfusion dependence in both alpha and beta thalassemia patients.

  • With a US filing planned...

The top-line data showing a statistically significant transfusion-reduction response in the 258-patient ENERGIZE-T trial follows Phase III data reported by Agios in January demonstrating that Pyrukynd could improve hemoglobin response...

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