PEPTIDE THERAPEUTICS

We are focused on the development and commercialization of peptide therapeutics for the treatment of GI diseases and obesity, including obesity caused by genetic deficiencies that result in metabolic disorders. We believe our product candidates have the potential to treat these diseases for which there are currently limited therapeutic options. We believe that ghrelin and MC4 are compelling targets because of their critical role in regulating GI function and metabolism, and that peptide therapeutics are well suited for activating these targets.

Based on the promising clinical results to date of relamorelin in diabetic gastroparesis, and the initial, favorable clinical profile of RM-493 for the treatment of obesity, including obesity caused by certain genetic deficiencies in the MC4 pathway, we believe these product candidates are well positioned to significantly improve the treatment of these indications.

Relamorelin is a potent, best-in-class, Phase 2 ghrelin agonist in development for the treatment of diabetic gastroparesis and GI functional disorders. Relamorelin targets the receptor for ghrelin, which is a naturally occurring hormone that plays a critical role in GI motility, digestion, and the absorption of nutrients. Prior drugs targeted the same GI motility disorders, primarily through either the serotonin or dopamine receptors, but had significant safety issues. First-generation ghrelin agonists were small molecules with limited potency and efficacy. In contrast, the relamorelin peptide retains the specificity and functionality of the naturally occurring ghrelin peptide and is designed to increase GI motility, with markedly greater potency than both naturally occurring ghrelin and the first-generation small molecule agonists of ghrelin.

RM-493 is a potent, first-in-class, Phase 2 MC4 agonist that modulates a key pathway in humans that regulates energy homeostasis and food intake. MC4’s critical role in weight regulation was validated with the discovery that a heterozygous mutation of the MC4 receptor gene—a mutation in just one of the two copies of the MC4 receptor gene—results in early onset obesity and severe obesity. RM-493 targets the receptor for MC4, which is a key pathway that regulates energy, homeostasis, and food intake. The first generation of MC4 agonists were generally small molecules that failed in clinical trials due to safety issues, particularly increases in blood pressure, and had limited efficacy. In contrast, the RM-493 peptide retains the specificity and functionality of the naturally occurring hormone that activates MC4, and our Phase 1 and Phase 2 clinical trials have shown promising evidence of weight loss without adversely increasing blood pressure.