Development of a new knock-in mouse model and evaluation of pharmacological activities of lusutrombopag, a novel, nonpeptidyl small-molecule agonist of the human thrombopoietin receptor c-Mpl

- Lusutrombopag is a novel, orally active, small-molecule thrombopoietin (TPO) receptor agonist.
- Lusutrombopag acts specifically acts on human TPO receptors.
- Lusutrombopag exhibited differentiation and proliferation of megakaryocytic cells.
- A novel knock-in mouse was created by replacing mMpl with human-mouse chimera Mpl.
- Oral dosing of lusutrombopag significantly increased platelets in TPOR-Ki/Shi mice.