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
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Agios reports Phase III data showing that Pyrukynd can reduce transfusion dependence in both alpha and beta thalassemia patients.
The top-line data showing a statistically significant transfusion-reduction response in the 258-patient ENERGIZE-T trial follows Phase III data reported by...
