Abstract 279: Completion of IND-enabling studies for NRT-YHD_001, a macrophage checkpoint inhibitor in liver cancer
Abstract
NRT-YHD_001 is a modified antisense microRNA targeting let-7i-5p, developed as a novel therapeutic candidate for hepatocellular carcinoma (HCC). This compound functions as a key regulator of macrophage activation within the tumor microenvironment. In in vitro phagocytosis assays, NRT-YHD_001 (GalNAc-conjugated form) demonstrated stronger macrophage activation than NRT-YHD (GalNAc-unconjugated form). Following a single intravenous injection in mice, NRT-YHD_001 selectively inhibited let-7i-5p without affecting other members of the let-7 family, confirming its high target specificity.To evaluate the in vivo efficacy of NRT-YHD_001 against advanced HCC, Ras-transgenic mice that spontaneously develop hepatic tumors at approximately 15-20 weeks of age, were used. After confirming tumor formation (∼5 mm2) by ultrasonography, NRT-YHD_001 was administered intravenously once weekly at 5 mg/kg. Compared with the sorafenib-treated group, NRT-YHD_001-treated mice exhibited greater tumor suppression, demonstrating superior therapeutic efficacy.The NRT-YHD_001 formulation satisfied all CMC quality control criteria. After lyophilization, the formulation was reconstituted into a clear, colorless solution, with an assay value of 105.1% of the label claim (specification range: 90.0-110.0%). All other parameters, including impurity levels, pH (7.5), osmolality (324 mOsm/kg), endotoxin (<2.0 EU/mg), and sterility, met specification limits, confirming excellent quality attributes.Pharmacokinetic…
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