This condition, caused by genetic mutations in the LEPR gene, leads to disrupted melanocortin-4 receptor (MC4R) signalling, resulting in extreme, constant hunger and severe early-onset obesity.
PL7737, an MC4R agonist, aims to restore the signalling pathway affected by these genetic mutations.
Palatin president and CEO Carl Spana said: “This FDA orphan designation is a key step in developing Palatin’s MC4R receptor agonists for rare obesity conditions.
“Currently, the only FDA-approved treatment for obesity due to leptin receptor deficiency is a daily injection. PL7737’s oral form could provide a more convenient and effective option for these patients and others with rare genetic obesity disorders. We are also exploring PL7737 for hypothalamic obesity and plan to begin a Phase 1 SAD/MAD study in late 2025.”
The melanocortin receptor (MCR) system impacts various physiological processes, including inflammation, immune responses, metabolism, food intake, and sexual function.
Last month, Palatin Technologies completed Phase II BMT-801 clinical study of the co-administration of MC4R bremelanotide + GLP-1/GIP tirzepatide for obesity treatment.
Topline data results from the Phase II BMT-801 study, which enrolled 113 patients with 96 patients randomised, are anticipated later this quarter.
Spana added: “Statistical analysis is now complete for our Phase 2 BMT-801 clinical study of the co-administration of MC4R bremelanotide + GLP-1/GIP tirzepatide for the treatment of obesity, and for our Phase II clinical study of PL8177 oral formulation for the treatment of ulcerative colitis.”