EPI-001 is primarily investigated for its potential in treating prostate cancer, especially in cases that are resistant to conventional therapies. It functions by targeting the androgen receptor, specifically interfering with the N-terminal domain, which plays a key role in the activation of genes involved in prostate cancer progression. This mechanism allows EPI-001 to inhibit the transcriptional activity of both full-length and splice variant forms of the androgen receptor, making it a promising candidate for castration-resistant prostate cancer where other treatments fail. Its ability to block the receptor’s function without binding to the ligand-binding domain offers a unique advantage in overcoming resistance seen with traditional anti-androgens. Research is ongoing to optimize its efficacy and pharmacokinetic profile for clinical use.