Aardvark Therapeutics (AARD) Piper Sandler 37th Annual Healthcare Conference summary

Pipeline and research focus

• Focus on rare diseases and metabolic disorders, with initial work in Prader-Willi syndrome and current expansion into obesity treatments.

• Two main programs: ARD-101 for Prader-Willi syndrome and ARD-201 for obesity.

• ARD-101 is a bitter taste receptor agonist targeting gut-brain axis to modulate hunger and appetite.

• ARD-201 combines ARD-101 with sitagliptin to enhance and prolong incretin effects.

• Unique mechanism aims to bypass hypothalamic pathways, potentially reducing side effects like nausea.

Clinical trial updates and design

• Phase 3 trial for ARD-101 in Prader-Willi syndrome is ongoing, with top-line data expected in Q3 2026.

• Phase 2 data showed reduction in hyperphagia and strong safety/tolerability, supporting phase 3 progression.

• Phase 3 includes patients aged 10 and above, expanding from previous older cohorts to capture early disease impact.

• HQCT, a physician-assessed questionnaire, is used as the primary endpoint, with measures to ensure consistency and minimize site variability.

• Interim analysis by a third party will focus on sample size re-estimation, not efficacy or futility.

Patient population and trial operations

• About one-third of trial sites are in the U.S., with the rest in Canada, Australia, South Korea, the EU, and the UK.

• Patients on antipsychotics are included to better reflect real-world populations, with 20-30% prevalence in the U.S.

• Training and operational controls are in place to ensure quality and minimize bias in qualitative assessments.

• Baseline stratification is used to reduce placebo effects in hyperphagia scoring.