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Tue, Sep 1, 2026 · 5:00 – 6:00 AM Bangkok
Understanding Your Care Journey: From Testing to Treatment
Brian L. Miller II
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Agios halts development of oral sickle cell drug following trial

Source
Sickle Cell Anemia News

Agios Pharmaceuticals is discontinuing development of its oral therapy candidate, tebapivat, for people with sickle cell disease (SCD).

The decision follows results from a small Phase 2 clinical trial (NCT06924970) showing that while tebapivat — an activator of the pyruvate kinase (PK) enzyme — reduced measures of red blood cell destruction in SCD patients, it did not offer enough advantages over other drugs in the same class to justify further development.

“These Phase 2 data further reinforce PK activation as a clinically validated mechanism in sickle cell disease, with tebapivat demonstrating [blood-related] activity consistent with this class of medicine,” Sarah Gheuens, MD, PhD, Agios’ chief medical officer and head of research and development, said in a company press release. “However, the results did not establish the level of differentiation we believe is necessary to support continued development.”