• Results did not meet predefined threshold to support further development in LR-MDS
     
  • Tebapivat was well tolerated, with no new safety signals observed

CAMBRIDGE, Mass., May 29, 2026 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that it will not advance tebapivat, a next-generation oral pyruvate kinase (PK) activator, in lower-risk myelodysplastic syndromes (LR-MDS). This decision follows results from its Phase 2b trial that did not meet the company's predefined threshold to support further development in this indication.

The open-label, multicenter, 24-week dose-finding trial evaluated once-daily tebapivat at 10 mg, 15 mg, and 20 mg in 65 patients with LR-MDS and anemia, representing a heavily pretreated, heterogeneous population. The primary endpoint was transfusion independence, defined as eight consecutive weeks without a transfusion during the 24-week treatment period. While tebapivat demonstrated evidence of biological activity, clinical benefit was not observed in a sufficient proportion of patients or subgroup of patients to meet the company's predefined threshold for advancement in LR-MDS. Tebapivat was well tolerated across all dose levels, with no new safety signals identified.