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XYA01 is an antibody-drug conjugate (ADC) developed by Xyone Therapeutics for the treatment of cancers that overexpress MUC1, particularly estrogen receptor positive (ER+) breast cancer and triple negative breast cancer. The drug consists of a monoclonal antibody targeting the MUC1 C-terminal subunit—an epitope selectively exposed on carcinoma cells but not normal epithelia—conjugated via a cleavable linker to a cytotoxic payload. This design aims to overcome previous failures in targeting MUC1 by focusing on the non-shedding C-terminal subunit, ensuring delivery of the cytotoxic agent directly into tumor cells. Preclinical studies have shown potent anti-tumor activity in vitro and in vivo with minimal toxicity observed in humanized mouse models[2][3][1].
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