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Agios’ Mitapivat Shows Improvement In Regularly Transfused PKD Patients

Results Follow ACTIVATE Study Data In December

 
• By Alaric DeArment

While representing a smaller population, the ACTIVATE-T trial targeted patients seen as having greater unmet need.

3-D Rendering Red Blood Cells
Agios' trial of mitapivat shows reduction in transfusion burden among patinets with rare anemia. • Source: Shutterstock

The success of Agios Pharmaceuticals, Inc.'s Phase III ACTIVATE-T trial in regularly transfused pyruvate kinase deficiency (PKD) patients was largely expected given the previously announced success of a study in the non-regularly transfused population. But the news comes around a month after the company announced the sale of its cancer drug portfolio to Les Laboratoires Servier SAS, giving a boost to its pivot into genetically defined diseases.

Cambridge, Massachusetts-based Agios said in an after-hours announcement on 26 January that its 27-patient, single-arm trial showed a statistically significant reduction in transfusion burden compared with participants' individual historical transfusion burden,

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