Second thalassaemia study of Agios' oral drug hits main goal

03 Jun 2024
firstwordpharma.com
Phase 3Drug ApprovalClinical Result
A Phase III study of Agios Pharmaceuticals’ Pyrukynd (mitapivat) in adults with transfusion-dependent (TD) alpha- or beta-thalassaemia achieved its primary endpoint of transfusion reduction response, supporting its plans to file for expanded US approval of the pyruvate kinase activator later this year. The company noted Monday that statistical significance was also achieved for all key secondary goals of the ENERGIZE-T trial.
Earlier this year, Agios reported that Pyrukynd met the primary endpoint of haemoglobin (Hb) response in the Phase III ENERGIZE study in adults with non-transfusion-dependent alpha- or beta-thalassaemia, becoming the first oral drug to achieve this goal.
Top-line results from ENERGIZE-T showed that in the Pyrukynd arm, 30.4% of patients achieved a transfusion reduction response, compared to 12.6% of patients in the placebo arm.
Pyrukynd is currently approved for the treatment of haemolytic anaemia in adults with pyruvate kinase (PK) deficiencypyruvate kinase (PK) deficiency in the US, and for the treatment of PK deficiency in adults in the EU.
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