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AXT-1003 is a novel, oral, small molecule epigenetic inhibitor targeting enhancer of zeste homolog 2 (EZH2), a histone methyltransferase implicated in cancer progression. It is being developed primarily for the treatment of various cancers, including non-Hodgkin's lymphoma and advanced solid tumors. The drug was originally discovered by Novartis and is now being developed by Axter Therapeutics under a licensing agreement with Novartis. Preclinical activities are ongoing in China and the US, with phase I clinical trials underway for relapsed/refractory non-Hodgkin's lymphoma and advanced solid tumors[1][2][6]. The mechanism of action involves inhibition of EZH2, which plays a role in gene silencing through histone methylation[1][2][3].
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