Peptide Therapeutics for Rare Genetic Deficiencies Resulting in Life-Threatening Metabolic Disorders
We are 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 the following genetic disorders of obesity: POMC deficiency obesity, LepR deficiency obesity, Prader-Willi syndrome, Bardet-Biedl syndrome, Alström syndrome, POMC heterozygous deficiency obesity, and POMC epigenetic disorders.
We have initiated a Phase 3 registration study for POMC deficiency obesity and are preparing to initiate a Phase 3 registration study for LepR deficiency obesity. In addition, we have initiated a Phase 2 proof-of-concept clinical study for Bardet-Biedel syndrome and are preparing to initiate Phase 2 proof-of-concept studies for Alström syndrome, POMC heterozygous deficiency obesity, and POMC epigenetic disorders. We are also evaluating setmelanotide for the treatment of Prader-Willi syndrome in a Phase 2 study. We previously presented Phase 2 clinical data for setmelanotide demonstrating substantial efficacy in reducing weight and hyperphagia in both POMC and LepR deficiency obesity.
Setmelanotide: A First-in-Class Phase 2 MC4RAgonist
A new drug class for the treatment of rare genetic disorders of obesity
MC4R modulates a key pathway in humans that regulates energy homeostasis and food intake. The critical role of the MC4 pathway in weight regulation was validated with the discovery that single genetic defects in this pathway result in early-onset and severe obesity.
First-generation MC4R agonists were small molecules that failed primarily due to safety issues—particularly increases in blood pressure—as well as limited efficacy. In contrast, setmelanotide is a peptide that retains the specificity and functionality of the naturally occurring hormone that activates MC4R.