Aardvark Therapeutics Receives FDA Orphan Drug Designation for ARD-101 in Prader-Willi Syndrome
Aardvark Therapeutics, Inc., a clinical stage biopharmaceutical company, has announced that its lead program, ARD-101, has received Orphan Drug Designation from the FDA. ARD-101 is an oral small molecule bitter taste receptor (TAS2R) agonist that shows potential in addressing the challenges associated with Prader-Willi Syndrome (PWS), a rare genetic condition characterized by hyperphagia (insatiable hunger) and obesity-related metabolic conditions.
PWS affects various organ systems and is caused by the loss of function of specific genes on chromosome 15. It results in neurodevelopmental dysfunction, musculoskeletal malformations, and developmental delays. A significant characteristic of PWS is early childhood obesity driven by hyperphagia, which is further complicated by strict food control measures. ARD-101, as a gut-restricted drug, has shown safety and efficacy by activating the secretion of gut peptide hormones like GLP-1, GLP-2, and cholecystokinin (CCK). Impaired CCK release in PWS patients leads to a constant sense of extreme hunger.
ARD-101, the lead product of Aardvark Therapeutics, is an innovative oral composition that has demonstrated positive outcomes in pre-clinical studies, including hunger reduction and weight loss promotion. Phase I studies conducted on healthy volunteers have confirmed its safety and tolerability, while three Phase II studies have exhibited hunger suppression and metabolic benefits. Notably, ARD-101 has minimal systemic exposure but still elicits systemic effects by stimulating the secretion of gut peptide hormones, including CCK, which plays a crucial role in regulating hunger.
