Rhythm is a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity.
Obesity is a disease of increased fat mass resulting from an imbalance in the intake and expenditure of energy. Research has shown that genetic factors can influence this balance. When an individual’s genetic makeup is susceptible to an environment that promotes energy consumption over energy expenditure, this is known as polygenic obesity, or common obesity.1
Rare genetic disorders of obesity are different. Sometimes, a variant in the genes can cause a disorder. More specifically, rare genetic disorders of obesity occur in the melanocortin 4 (MC4) pathway, which regulates weight by increasing energy expenditure and reducing appetite.
MC4 pathway deficiencies can result in the disruption of satiety signals and energy homeostasis in the body, which, in turn, leads to unrelenting hunger, known as hyperphagia, and to severe, early-onset obesity. The MC4 pathway is proving to be a compelling target for treating these rare genetic disorders because of its critical role in regulating appetite and weight.
By leveraging these cutting-edge insights and our expertise in precision medicine, we are dedicated to pioneering groundbreaking therapies for these rare disorders.
Our lead product candidate, setmelanotide, is an investigational, first-in-class melanocortin-4 receptor (MC4R) agonist being studied for the treatment of rare genetic disorders of obesity.
Setmelanotide retains the specificity and functionality of the naturally occurring hormone that activates the MC4R, which enables a targeted approach to treating very severe obesity in patients with deficiencies in genes within the MC4 pathway. By restoring lost activity in this pathway, setmelanotide may serve as replacement therapy for MC4 pathway deficiencies, with the potential to reestablish both weight and appetite control.*
Setmelanotide has been granted orphan drug and breakthrough therapy designation by the US Federal Drug Administration for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway, which includes pro-opiomelanocortin (POMC) deficiency obesity, leptin receptor (LEPR) deficiency obesity, Bardet-Biedl Syndrome and Alström Syndrome.
In addition, setmelanotide has also been granted PRIority MEdicines (PRIME) Designation for the treatment of obesity associated with pro-opiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity by the European Medicines Agency (EMA). The PRIME program was launched by the EMA in 2016 to provide early and enhanced support to optimize the development of eligible medicines, speed up their evaluation, and contribute to timely patient access.
RM-853 is an investigational, orally available ghrelin o-acyltransferase (GOAT) inhibitor currently in preclinical development for Prader-Willi syndrome (PWS).
RM-853 is designed to lower levels of the peptide Ghrelin, a key contributor to the stimulation of appetite and promotion of food intake, by blocking the enzyme responsible for its production known as GOAT. By lowering Ghrelin levels, the potential may be there to increase levels of the ghrelin precursor, des-acyl-ghrelin (DAG), which is also believed to have benefits to appetite control and homeostasis.
In preclinical research, RM-853 prevented body weight gain and reduced fat mass in mice fed a high-fat diet, with a favorable pharmacokinetic, pharmacodynamic, and safety profile.
Learn more about the Rhythm pipeline ▸
