The FDA has granted Aardvark Therapeutics a Rare Pediatric Designation for the use of ARD-101 in Prader-Willi Syndrome (PWS), a rare genetic disease characterized by extreme and unabating hunger. This designation means Aardvark is eligible for a Rare Pediatric Disease Priority Review Voucher when ARD-101 receives approval in PWS. The early clinical study results suggest a promising future for a new class of pharmaceuticals that could benefit people with insatiable hunger (hyperphagia) and aggressive food-seeking behaviours, as well as for obesity and metabolic conditions, which are prominent features of the rare genetic condition PWS. Thus far, no drug has been approved by the FDA to treat the hyperphagia associated with PWS.
Aardvark's lead product, ARD-101, is a first-in-class oral composition that has shown promising activity in reducing hunger cravings in clinical studies and promoting weight loss in pre-clinical studies. ARD-101 is substantially gut-restricted and has minimal systemic exposure. It is postulated that ARD-101 conveys its systemic effects by activating secretion of several gut peptide hormones, including glucagon-like peptides-1 and -2(GLP-1, GLP-2), and cholecystokinin (CCK).
Gut CCK is considered a "satiety signal" that acts via the gut-brain axis to control hunger. PWS patients have a normal CCK receptor but their CCK release from gut enteroendocrine I-cells in response to food is impaired, leaving PWS patients with a continuous sense of extreme hunger. Phase I studies demonstrated safety and tolerability in healthy human volunteers. Three Phase II studies have demonstrated an impact on hunger suppression.
Previously, Aardvark had enrolled 12 subjects in a Phase 2 study of oral ARD-101 in PWS led by Dr Diane Stafford at Stanford Children's Health and Dr Shawn McCandless at Colorado Children's Hospital. Based on promising results, Aardvark is enrolling additional subjects and exploring higher drug doses.