Agios drops a sickle cell drug as another nears approval

Agios Pharmaceuticals said its Phase 2 trial of tebapivat, a drug for sickle cell disease, came back neutral — and the company is stopping its development. Sickle cell disease causes red blood cells to warp into a rigid, curved shape that blocks blood flow and causes severe pain; tebapivat was designed to help red blood cells hold their normal shape for longer.

This isn't Agios giving up on sickle cell disease. The company's other drug for the same disease, mitapivat, is advancing toward a possible FDA accelerated approval on separate, positive Phase 3 data — tebapivat was always the company's second, more experimental drug, not its main one.

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Does this hurt Agios's other drug? Not really. A neutral result means tebapivat didn't do enough good to keep testing — not that it did any harm. That distinction matters for a company still running mitapivat through the same regulatory pathway.