Agios Pharma Won't Advance Tebapivat for Lower-Risk Myelodysplastic Syndromes

Dow Jones
05/29
 

By Chris Wack

 

Agios Pharmaceuticals said it won't advance its tebapivat, an oral pyruvate kinase activator, in lower-risk myelodysplastic syndromes, after a Phase 2b trial didn't meet the company's predefined threshold to support further development in this indication.

The commercial-stage biopharmaceutical company said the 24-week dose-finding trial evaluated once-daily tebapivat in 65 patients with LR-MDS and anemia.

The primary endpoint was transfusion independence, defined as eight consecutive weeks without a transfusion during the 24-week treatment period. Agios said tebapivat demonstrated evidence of biological activity, but that it didn't see clinical benefit in a sufficient proportion of patients or subgroup of patients to meet the company's predefined threshold for advancement.

Tebapivat was well tolerated across all dose levels, with no new safety signals identified.

Agios said tebapivat is also being evaluated in a Phase 2 trial for the treatment of sickle cell disease, with top-line data anticipated in the second half of 2026.

Shares were down 5.5% to $28.75 in premarket trading.

 

Write to Chris Wack at chris.wack@wsj.com

 

(END) Dow Jones Newswires

May 29, 2026 07:30 ET (11:30 GMT)

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