RYTH is a clinical-stage biopharmaceutical company focused on developing and commercializing peptide therapeutics for the treatment of rare genetic deficiencies that result in life-threatening metabolic diseases. Its lead product candidate is called "setmelanotide", a first-in-class melanocortin-4 receptor (MC4R) agonist for the treatment of rare genetic diseases in obesity.
Setmelanotide activates MC4R, which is part of the key pathway that can independently regulate energy homeostasis, which refers to the body's energy balance and appetite. An expanding set of severe obesity genetic defects are now identified that involve genes in the pathway which are either upstream of MC4R—for example POMC deficiency obesity and LepR deficiency obesity—or genes that are downstream of MC4R or affect MC4R itself.
The company's development efforts are initially focused on obesity related to six single gene related pathway deficiencies for which there are currently no effective or approved treatments. It believes that the pathways it is targeting is compelling because of their critical role in regulating appetite and weight by promoting satiety and weight control. Peptide therapeutics are uniquely suited for activating this target.
RYTH has demonstrated proof of concept in Phase 2 clinical trials in POMC deficiency obesity, LepR deficiency obesity, and Bardet-Biedel syndrome, three genetic disorders of extreme and unrelenting appetite and obesity, in which setmalanotide dramatically reduced both weight and hunger. In accordance with the results, the FDA gave setmalanotide breakthrough therapy designation for the treatment of obesity associated with genetic defects upstream of the MC4 receptor in the leptin-melanocortin pathway.
In addition to treating POMC and LepR, RYTH plans to expand setmelanotide development to additional MC4 pathway deficiencies such as Bardet-Biedl syndrome, Alstrom Syndrom, POMC heterozygous deficiency obesity, and POMC epigenetic disorders.