NXP800

NXP800 is a clinical-stage, orally bioavailable small molecule inhibitor that targets the heat shock factor 1 (HSF1) pathway. It was developed as a potent fluorobisamide compound from phenotypic screening and lead optimization efforts to inhibit HSF1-mediated transcription, which supports malignancy in several cancers. NXP800 has demonstrated robust antitumor activity in preclinical models of ARID1a-mutated ovarian carcinoma and other solid tumors by decreasing HSP72 expression, activating the unfolded protein response, and inhibiting androgen receptor (AR)- and E2F-mediated activity. The drug is under development for platinum-resistant, ARID1a-mutated ovarian carcinoma as well as other advanced cancers such as bile duct cancer (cholangiocarcinoma) and treatment-resistant prostate cancer. It has received Fast Track Designation and Orphan Drug Designation from the US FDA for ARID1a-deficient ovarian, fallopian tube, primary peritoneal cancers, and cholangiocarcinoma[2][4][5][6][8].