ATH-002

ATH-002 is a potent, selective, and orally bioavailable KAT6A (Lysine Acetyltransferase 6A) degrader developed by Atheron Therapeutics. It utilizes the ubiquitin-proteasome system via E3-ligase binding to induce the degradation of KAT6A, a member of the MYST family of histone acetyltransferases that regulates gene transcription through H3K23 acetylation. Unlike dual KAT6A/6B inhibitors, ATH-002 demonstrates high selectivity for KAT6A over KAT6B, which significantly reduces the risk of hematotoxicity (neutropenia and anemia) associated with simultaneous inhibition of both proteins. In preclinical studies, ATH-002 has shown robust anti-tumor activity in KAT6A-amplified cancer models, particularly in breast cancer, and has demonstrated synergistic effects when combined with selective estrogen receptor degraders (SERDs) or CDK4/6 inhibitors.