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 acts as a bitter taste receptor agonist, stimulating incretin release to reduce hunger and appetite.

• ARD-201 combines ARD-101 with sitagliptin to prolong incretin effects, targeting both hunger and appetite pathways.

• 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 advancement to phase 3.

• Phase 3 includes patients aged 10 and above, expanding from previous age limits to capture dynamic changes in younger patients.

• HQCT, a physician-assessed qualitative questionnaire, is used as the primary endpoint for hyperphagia.

• Quality control measures include consistent caregivers and physicians, training, and blinded operational checks.

Statistical and operational considerations

• Interim unblinded analysis by a third party is for sample size re-estimation, not for efficacy or futility.

• Patient baseline scores are used for stratification to minimize placebo effects.

• Patients on antipsychotics are included to ensure real-world representation, with about 20-30% prevalence in the population.

• About one-third of trial sites are in the U.S., with the rest globally distributed.

• Efforts are made to minimize geographic and site-to-site variability in HQCT assessments.