OUR PIPELINE

Our lead product candidate, setmelanotide, is an investigational, first-in-class melanocortin-4 receptor (MC4R) agonist in development for the treatment of rare genetic disorders of obesity. Setmelanotide is thought to activate the MC4R, part of a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite. Variants in genes within the MC4 pathway are associated with unrelenting hunger, known as hyperphagia, and severe, early-onset obesity.

We believe setmelanotide is a potential replacement therapy that may restore lost activity in the MC4 pathway, reestablishing weight and appetite control in patients with these rare genetic disorders.

RM-853 is an investigational, orally available ghrelin o-acyltransferase (GOAT) inhibitor currently in preclinical development for Prader-Willi syndrome (PWS). PWS is a rare genetic disorder that results in hyperphagia and early-onset, life-threatening obesity, for which there are no approved therapeutic options.

Ghrelin is an orexigenic peptide secreted by the stomach and proximal small intestine in response to a negative energy balance. Ghrelin plays a key physiological role in stimulating appetite and promoting food intake, thereby maintaining overall energy balance. In people living with PWS, levels of active ghrelin are elevated, contributing to an unrelenting hunger known as hyperphagia, which leads to severe obesity. RM-853 is designed to block GOAT, the key enzyme involved in the production of the active form of ghrelin, with the expected effect of lowering active ghrelin levels. This blockage increases the levels of des-acyl-ghrelin (DAG), a ghrelin precursor. High levels of DAG are believed to have independent beneficial effects on the control of appetite and tissue homeostasis, which might add to the potential efficacy of RM-853 in PWS.