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RG-125 (AZD4076) is a GalNAc-conjugated antisense oligonucleotide therapeutic targeting microRNA-103 and microRNA-107 (miR-103/107). It is designed to inhibit these specific microRNAs, which are implicated in the development of insulin resistance and metabolic dysfunction. By inhibiting miR-103/107, RG-125 aims to improve insulin sensitivity and glucose tolerance. The drug was developed for the treatment of non-alcoholic steatohepatitis (NASH) in patients with type 2 diabetes or pre-diabetes. The compound was selected as a clinical candidate by AstraZeneca from an alliance with Regulus Therapeutics[1][4][7][8]. Clinical trials have focused on evaluating its safety, tolerability, pharmacokinetics, and effects on liver fat content and insulin sensitivity in subjects with type 2 diabetes mellitus and non-alcoholic fatty liver disease[2][5].
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