Rhythm is a biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity.
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. We believe that setmelanotide, for which we have exclusive worldwide rights, has the potential to serve as replacement therapy that may restore lost activity in the MC4 pathway, a key biological pathway in humans that regulates weight by increasing energy expenditure and reducing appetite. Variants in genes within the MC4 pathway result in the disruption of satiety signals and energy homeostasis in the body, which leads to unrelenting hunger, known as hyperphagia, and to severe, early-onset obesity.
We are initially focused on 6 rare genetic disorders of obesity for which there are currently no effective or approved treatments:
- Pro-opiomelanocortin (POMC) deficiency obesity
- Leptin receptor (LEPR) deficiency obesity
- Bardet-Biedl syndrome
- Alström syndrome
- POMC heterozygous deficiency obesity
- POMC epigenetic disorders
We have demonstrated proof of concept in Phase 2 clinical trials in POMC deficiency obesity, LEPR deficiency obesity, and Bardet-Biedl syndrome in which setmelanotide demonstrated reductions in both weight and hunger. Setmelanotide has been granted Breakthrough Therapy Designation for these indications by the US Food and Drug Administration.
Setmelanotide is currently in Phase 3 clinical trials for POMC deficiency obesity and LEPR deficiency obesity. We have also initiated a Phase 2 proof of concept basket study in Alström syndrome, POMC heterozygous deficiency obesity, and POMC epigenetic disorders. Additionally, we are evaluating another study for Prader-Willi syndrome.