Agios Pharmaceuticals has released an update from its phase 2 trial of tebapivat, announcing that the sickle cell disease drug failed to establish a differentiated profile.
Tebapivat is an oral pyruvate kinase (PK) activator. In the phase 2 trial, patients received one of three doses or a placebo. The study showed that the hemoglobin response rates were consistent with the class and that the safety and tolerability of the drug were consistent with previous sickle cell disease trials. However, its profile did not achieve a level of differentiation compared to other PK activators necessary to continue the drug’s development.
Agios has another PK activator, mitapivat, under FDA Priority Review for sickle cell disease, with a PDUFA goal date of November 1.