Palatin Technologies has announced the FDA has granted "orphan drug" designation to PL7737, an oral treatment that activates the melanocortin-4 receptor (MC4R), for leptin receptor (LEPR) deficiency, including obesity caused by this condition.
Obesity caused by LEPR deficiency is a rare genetic condition where mutations in the LEPR gene disrupt MC4R signalling. The Leptin-Melanocortin pathway in the hypothalamus plays a key role in regulating hunger, energy storage, and body weight. People with this condition experience extreme, constant hunger from a young age, leading to severe early-onset obesity. PL7737, an MC4R agonist, is designed to restore impaired signalling caused by these genetic mutations.
Genetic mutations that inhibit signalling through the MC4R pathway lead to hyperphagia, decreased energy expenditure and early-onset obesity; such mutations have been identified as the cause of several rare genetic obesity disorders. MC4R agonism represents an attractive target for potential obesity treatments.
The MCR system has effects on inflammation, immune system responses, metabolism, food intake, and sexual function. There are five melanocortin receptors, MC1R through MC5R. Modulation of these receptors, through use of receptor-specific agonists, which activate receptor function, or receptor-specific antagonists, which block receptor function, can have medically significant pharmacological effects.
"This FDA orphan designation is a key step in developing Palatin's MC4R receptor agonists for rare obesity conditions," said Dr Carl Spana, President and CEO of Palatin. "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."
Supporting the development and evaluation of new treatments for rare diseases is a key priority for the FDA. The FDA has authority to grant orphan drug designation to a drug or biological product to prevent, diagnose or treat a rare disease or condition.
"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 2 clinical study of PL8177 oral formulation for the treatment of ulcerative colitis,” Spana added. “We look forward to releasing topline data results for both of these studies later this month."
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