OZ-001

OZ-001 is an oral small molecule investigational drug developed by ONCOZEN Co., Ltd (formerly known by the research code KCP10043F). It features a dual mechanism of action, acting as an antagonist of T-type calcium (Ca2+) channels and an inhibitor of STAT3 (Signal Transducer and Activator of Transcription 3) phosphorylation and activation. By simultaneously targeting these pathways, OZ-001 is designed to disrupt oncogenic signaling and calcium homeostasis in cancer cells, potentially overcoming resistance mechanisms in aggressive tumours. It is currently being evaluated in a Phase 1, open-label, first-in-human dose-escalation and expansion study to assess its safety, tolerability, and preliminary antitumour activity in adults with advanced solid tumours, with a primary focus on triple-negative breast cancer (TNBC) and pancreatic ductal adenocarcinoma (PDAC).