Drug intelligence / Profile preview

RM-853

Development stage
Discontinued
Lead developer
Rhythm Pharmaceuticals
Modality
Small Molecules
Administration
Oral
01

Overview

RM-853 is a potent, orally available inhibitor of **ghrelin O-acyltransferase (GOAT)**, the key enzyme responsible for producing the active form of ghrelin. Ghrelin is a hormone that stimulates appetite, and its active form is increased in conditions such as Prader-Willi syndrome, leading to hyperphagia (an insatiable hunger) and severe obesity. By inhibiting GOAT, RM-853 is designed to reduce levels of active ghrelin, decrease appetite, and potentially lower weight gain. Preclinical studies in mice demonstrated that RM-853 prevented body weight gain and reduced fat mass in animals on a high-fat diet, with favorable pharmacokinetic and safety profiles. RM-853 is currently in preclinical development and was originally discovered by Takeda and subsequently licensed and further developed by Rhythm Pharmaceuticals for rare genetic disorders of obesity, specifically Prader-Willi syndrome[1][4][7].

02

Targets

GOAT (Ghrelin O-acyltransferase)

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