PF-07059013

PF-07059013 is an orally active, potent, noncovalent small molecule modulator of sickled hemoglobin (HbS), developed for the treatment of sickle cell disease (SCD). It specifically binds to hemoglobin with nanomolar affinity and displays strong partitioning into red blood cells. Mechanistically, PF‑07059013 acts as a selective allosteric modulator that stabilizes the oxygenated state of hemoglobin, thereby inhibiting HbS polymerization and reducing red blood cell sickling under hypoxic conditions. Preclinical studies demonstrated significant reductions in RBC sickling and improvements in markers of hemolytic anemia. The drug was advanced to phase 1 clinical trials as a prophylactic therapy aimed at reducing anemia severity and vaso‑occlusive crises in SCD patients[1][3][4][6].

Other names

6-((1S)-1-((2-amino-6-fluoroquinolin-3-yl)oxy)ethyl)-5-(1H-pyrazol-1-yl)pyridin-2(1H)-one