NAX035

NAX035 is a first-in-class, berberine-derived small-molecule anticancer agent being developed by Aromics (Applied Research Using Omic Sciences) as an RNA-targeted therapy for malignant mesothelioma and other solid tumors. It is a chemically modified analogue of the natural isoquinoline alkaloid berberine, engineered to improve bioavailability, pharmacologic potency, and safety while maintaining and enhancing berberine’s cytotoxic and antiproliferative properties in cancer models.[1][2][5] NAX035 acts as a selective inhibitor of thymidylate synthase expression by directly binding to its messenger RNA and blocking translation, thereby reducing levels of this enzyme, which is a clinically validated resistance biomarker for pemetrexed-based chemotherapy in mesothelioma and non-small cell lung cancer.[1][2] In preclinical in vitro and in vivo models, including mesothelioma and other tumor systems, NAX035 has demonstrated robust antitumor activity, induction of apoptosis, and significant tumor growth reduction with acceptable toxicology when administered orally, intraperitoneally, or intravenously.[1][2][3][4][6][8] It has completed extensive preclinical evaluation and is entering first-in-human phase 1 clinical testing in patients with advanced solid tumors, including malignant pleural mesothelioma, colorectal cancer, non-small cell lung cancer, and ovarian cancer.[2][3][6]