NB003

NB003 is a potent and selective small-molecule tyrosine kinase inhibitor that targets mutated forms of platelet-derived growth factor receptor alpha (PDGFR alpha) and the stem cell factor receptor c-Kit (KIT). It is designed to inhibit a broad spectrum of primary and acquired imatinib-resistant mutations in KIT/PDGFRα, including those found in the ATP-binding site and activation loop of the kinase domain. The drug is administered orally as a tablet. Its primary indication is for advanced gastrointestinal stromal tumor (GIST), particularly in patients who have progressed on or are intolerant to imatinib and other standard treatments. Clinical studies have shown encouraging antitumor activity across various secondary resistance mutations in KIT, with ongoing trials evaluating its safety, tolerability, pharmacokinetics, and efficacy[1][2][4].

Other names

PDGFR alpha/KIT mutant-specific inhibitor NB003