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 melanocortin 4, or MC4, pathway deficiencies. The MC4 pathway is a compelling target for treating these genetic disorders because of its critical role in regulating appetite and weight, and peptide therapeutics are uniquely suited to activating this target.
We are currently evaluating setmelanotide for the treatment of six genetic disorders of obesity—pro-opiomelanocortin (POMC) and leptin receptor (LEPR) deficiency obesity, Prader-Willi syndrome (PWS), Bardet-Biedl syndrome, Alström syndrome, and POMC heterozygous deficiency obesity.
In 2016, The New England Journal of Medicine reported results from a setmelanotide Phase 2 trial in POMC deficiency obesity that demonstrated substantial weight loss in two adult patients. At ObesityWeek 2016, investigators presented initial data for the first patient enrolled in a Phase 2 non-randomized, open-label clinical trial of setmelanotide for the treatment of LEPR deficiency obesity. Both POMC and LEPR deficiency obesity are rare genetic disorders associated with severe, early-onset obesity and unrelenting hyperphagia. The initial efficacy data with setmelanotide in these disorders demonstrate that setmelanotide has the potential to provide meaningful efficacy in genetic forms of obesity due to MC4 pathway deficiency by restoring absent LEPR-POMC signaling.
Obesity is epidemic in the U.S., and current treatment approaches have demonstrated limited long-term success for most obese patients. We are taking a different approach to obesity drug development by leveraging new understanding of the genetic causes of severe obesity to develop innovative therapies that we believe have the potential for compelling efficacy. Setmelanotide’s unique mechanism of action at MC4R enables a targeted approach to treating very severe obesity in patients with specific, monogenic defects in the MC4 signaling pathway. By restoring impaired function in this pathway, setmelanotide can serve as replacement therapy for genetic deficiencies, with the potential for dramatic improvements in weight and appetite.
We are at the forefront of improving treatment outcomes in subtypes of severe obesity that are caused by genetically defined defects in the MC4 pathway.