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Aardvark Announces Rare Disease Designation and Expands Ongoing Phase 2 Trial

The FDA has granted Aardvark Therapeutics a Rare Pediatric Designation for the use of ARD-101 in PWS. Aardvark's Phase 2 trial of oral ARD-101 in young adults with PWS is now open to additional volunteers.

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. 

About ARD-101

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.


Susan Hedstrom


Susan Hedstrom is the Executive Director for the Foundation for Prader-Willi Research. Passionate about finding treatments for PWS, Susan joined FPWR in 2009 shortly after her son, Jayden, was diagnosed with Prader-Willi Syndrome. Rather than accepting PWS as it has been defined, Susan has chosen to work with a team of pro-active and tireless individuals to accelerate PWS research in order to change the future of PWS. Inspired by her first FPWR conference and the team of researchers that were working to find answers for the syndrome, she joined the FPWR team in 2010 and led the development of the One SMALL Step walk program. Under Susan’s leadership, over $15 million has been raised for PWS related research.