Aardvark Therapeutics, a clinical stage biopharmaceutical company, has reported receipt of a Rare Pediatric Disease Designation in Prader-Willi Syndrome (PWS) from the FDA for its lead program ARD-101. 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 PWS who are challenged by insatiable hunger (hyperphagia) and aggressive food-seeking behaviors. Thus far, no drug has been approved by the FDA to treat the hyperphagia associated with PWS.
In a previous Phase 2 study of ARD-101, Aardvark enrolled 12 volunteers with PWS to take ARD-1o1. Based on promising results from this study, FPWR has provided a second round of financial support to assist Aardvark with enrolling additional volunteers to explore the impact of higher doses on hyperphagia.
ARD-101, is a first-in-class oral medication 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.