Drug intelligence / Profile preview

[13C6]AG-348

Development stage
Unknown
Lead developer
Agios Pharmaceuticals
Modality
Small Molecules
Administration
Oral, Intravenous
01

Overview

Mitapivat is a novel, first-in-class pyruvate kinase activator. It works as a small-molecule allosteric activator of the red blood cell-specific form of pyruvate kinase (PK-R), enhancing the activity of both wild-type and mutant enzymes associated with hemolytic anemia. By increasing the activity of erythrocyte pyruvate kinase, a key enzyme involved in red blood cell energy production, mitapivat addresses the underlying molecular pathology in pyruvate kinase deficiency, which leads to premature destruction of red blood cells and chronic hemolytic anemia.

Brand names
Pyrukynd
Other names
carbon-13 labeled mitapivatcarbon13 labeled mitapivatcarbon 13 labeled mitapivat[13C6]AG-348
02

Targets

PKLR (Pyruvate Kinase R)

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