EPI-001

EPI-001 is a code name associated with two distinct therapeutic candidates developed by different companies. 1) **Epigenic Therapeutics** is developing EPI-001 as an epigenetic modulation therapy for the treatment of **heterozygous familial hypercholesterolemia (HeFH)**. This candidate utilizes the proprietary **EPIREG™ platform**, which employs epigenome regulation mechanisms—specifically DNA methylation and histone modification—to modulate gene expression without inducing DNA cleavage. It is currently being evaluated in Phase 1 clinical trials as an intravenous injection. 2) **ESSA Pharma** historically developed a different EPI-001 as a first-generation **small molecule inhibitor** of the **androgen receptor (AR) N-terminal domain (NTD)**. This compound was designed to treat castration-resistant prostate cancer by blocking the transcriptional activity of the AR, including splice variants that lack the ligand-binding domain. While it served as a foundational compound for ESSA's pipeline, the company subsequently pivoted to next-generation candidates such as EPI-506 and the Aniten series (e.g., EPI-7386).