“The clarity of the efficacy and safety from those studies - the strength and consistency of the data - led us to believe we could clearly be best in class,’’ said Bart Henderson, cofounder and president of Rhythm.
Rhythm, which is also backed by New Enterprise Associates and Third Rock Ventures, is now completing Phase 1 human trials of its lead compound, RM-131, in patients with type 2 diabetes. The drug is derived from ghrelin, “the hunger hormone.’’ Ghrelin is produced in the gut and regulates functions such as food consumption, nutrient absorption, and the movement of food through the digestive tract.
Several drug companies have tried targeting ghrelin to treat diseases, with limited success. Rhythm is going after a common but largely untreated complication of both Type 1 and Type 2 diabetes called gastroparesis, a digestive disorder marked by an abnormal emptying of the stomach.
Normally, ghrelin receptors prompt a well-synchronized handoff of food from the stomach to the rest of the digestive tract.
“In diabetes, that process seems to be attenuated, so many patients complain of abdominal pain and bloating,’’ said Elizabeth Stoner, cofounder and chief development officer of Rhythm. “It causes their glucose control to go amiss.’’
According to Rhythm, about a third of the 25.8 million Americans with diabetes have gastroparesis. The primary treatment, a generic drug called metoclopramide, can be taken only for a short time because of the risk of a muscle disorder.
Rhythm plans to start a phase 2 gastroparesis study in early 2012.
Rhythm’s second compound, RM-493, could address an equally broad population: severely obese people who have diabetes or are at risk of developing it. RM-493 targets the melanocortin type 4 receptor, which when mutated, is estimated to cause as much as 6 percent of severe obesity.
The drug is designed to curb food intake.