etavopivat
An orally available, small-molecule allosteric activator of the selective red blood cell (RBC) isoform of pyruvate kinase (PK-R), with potential to improve symptoms in sickly cell disease (SCD) patients. Upon oral administration, etavopivat allosterically binds to and activates PK-R, thereby enhancing the glycolytic pathway activity in RBCs. This improves adenosine triphosphate (ATP) levels and reduces 2,3-diphosphoglycerate (2,3-DPG) levels in RBCs. This results in increased oxygen affinity, improved RBC deformability, decreased sickle RBC hemolysis, increased hemoglobin (Hb) levels and improved RBC membrane function. Mutations in PK-R cause deficiency in PK-R which prevents adequate RBC glycolysis, leading to a buildup of the upstream glycolytic intermediate 2,3-DPG and deficiency in the PK-R product ATP.
| Synonym: | erythrocyte pyruvate kinase activator FT-4202 PK-R activator FT-4202 pyruvate kinase isozyme R agonist FT-4202 pyruvate kinase-R activator FT-4202 RBC isoenzyme PK activator FT-4202 red blood cell isoenzyme pyruvate kinase agonist FT-4202 red cell pyruvate kinase agonist FT-4202 |
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| Code name: | FT 4202 FT-4202 |