NXP800 Gains FDA Orphan Drug Status in ARID1a-Deficient Ovarian Cancers

NXP800, a novel GCN2 kinase activator, received orphan drug designation from the FDA for ARID1a-deficient ovarian, fallopian tube, and primary peritoneal cancers. It is an oral small molecule inhibitor targeting the HSF1 pathway, showing strong antitumor effects in ARID1a-mutated ovarian carcinoma. A phase 1b clinical trial (NCT05226507) is ongoing, evaluating NXP800 in platinum-resistant, ARID1a-mutated ovarian cancers.