Agios Announces Clinical Proof-of-Concept in Phase 2a Trial of AG-946 for the Treatment of Anemia in Lower-Risk Myelodysplastic Syndromes
Agios Pharmaceuticals, Inc., a leader in the field of cellular metabolism pioneering therapies for rare diseases, today announced that clinical proof-of-concept has been achieved in the Phase 2a portion of a study of investigational pyruvate kinase (PK) activator AG-946 as a potential treatment for anemia in adults with lower-risk myelodysplastic syndromes (LR-MDS). Four of the 10 patients with low transfusion burden (LTB) achieved the transfusion independence endpoint, and one of the 22 patients treated in the study achieved the hemoglobin response endpoint in the 16-week treatment (core) period. The safety profile observed was consistent with data reported in the healthy volunteer study. Based on the favorable efficacy data and positive benefit-risk profile in the Phase 2a core period, Agios intends to advance its clinical program evaluating AG-946 in LR-MDS by initiating the placebo-controlled Phase 2b portion of the study in mid-2024.