Rhythm is a biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic disorders.
Our lead peptide product candidate, setmelanotide, is a potent, first-in-class melanocortin-4 receptor (MC4R) agonist 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 for the treatment of MC4 pathway deficiencies. MC4 pathway deficiencies result in the disruption of satiety signals and energy homeostasis in the body, which leads to intense feelings of hunger and to obesity. Our development efforts are currently focused on obesity related to six monogenic MC4 pathway deficiencies – pro-opiomelanocortin (POMC), leptin receptor (LepR), Bardet-Biedl syndrome, Alström syndrome, POMC heterozygous, and POMC epigenetic disorders – for which there are currently no effective or approved treatments. We believe the MC4 pathway is a compelling target for treating these genetic disorders because of its critical role in regulating appetite and weight, and that peptide therapeutics are uniquely suited to activating this target.
We have demonstrated proof of concept in Phase 2 clinical trials in both POMC deficiency obesity and LepR deficiency obesity, in which setmelanotide dramatically reduced both weight and hunger. Setmelanotide has been granted Breakthrough Therapy Designation for these indications by the U.S. Food and Drug Administration.
Setmelanotide is currently in Phase 3 clinical trials for POMC deficiency obesity and LepR deficiency obesity. We have also initiated Phase 2 clinical trials in Bardet-Biedl syndrome, Alström syndrome, POMC heterozygous deficiency obesity, and POMC epigenetic disorders.