Agios Announces Clinical Proof-of-Concept in Phase 2a Trial of AG-946 for the Treatment of Anemia in Lower-Risk Myelodysplastic Syndromes

– 40 Percent of Low Transfusion Burden Cohort Achieved Transfusion Independence;
One Study Patient Achieved Hemoglobin Response Endpoint –

– Safety Profile Consistent with Data Reported in Healthy Volunteers Study –

– Company Expects to Initiate Phase 2b Study in Mid-2024 –

CAMBRIDGE, Mass., Nov. 20, 2023 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), 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.

“We are pleased with the results of the Phase 2a study, which underscore the potential of AG-946 to be a first-in-class, oral, safe and effective option for the treatment of anemia in adults with LR-MDS by improving red blood cell health through its unique mechanism of action. Regular blood transfusions are burdensome for patients. A meaningful reduction in transfusions allows patients to potentially decrease visits to the clinic and experience improved quality of life. We are grateful to all of the patients who participated in this trial, our collaborators, study investigators and advisors in the patient and clinical communities for their partnership in achieving this milestone, and we look forward to advancing the clinical program to Phase 2b,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “Agios is proud to be the leader in PK activation with two distinct PK activators – AG-946 and mitapivat – that have the potential to be meaningful treatment options for a wide range of blood disorders and positively impact many patients’ lives.”