Agios Pharma Says It Will Not Advance Tebapivat, Next-Gen Oral Pyruvate Kinase Activator, In Lower-Risk Myelodysplastic Syndromes As Phase 2b Trial Results Did Not Meet Predefined Threshold To Support Further Development In LR-MDS

Agios Pharmaceuticals, Inc.

Agios Pharmaceuticals, Inc.

AGIO

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  • 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.

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