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...