PF-04449913 (Glasdegib) is an orally bioavailable Hedgehog pathway inhibitor. PF-04449913 acts by binidng Smoothened (Smo) and blocking signal transduction. PF-04449913 has an IC50 of 5 nM in the Gli-Luciferase assay. In a Phase I clinical trial in patients with advanced solid tumors, PF-04449913 caused disease stabilization in 34% of patients.
PF-04449913 maleate salt is also known as 1-((2
R,4
R)-2-(1
H-benzo[
d]imidazol-2-yl)-1-methylpiperidin-4-yl)-3-(4-cyanophenyl)urea.
[1] It increases the differentiation of blood cells from hematopoietic precursors of the lymph gland medullary zone. This human Smo inhibitor protein is now under clinical trials to treat myeloid malignancies.
[1]