Aardvark Therapeutics (AARD) investor relations material

Program overview and clinical development

• Lead program ARD-101 is a gut-restricted oral small molecule targeting gut-brain signaling to reduce hunger and improve metabolic outcomes, with primary focus on Prader-Willi syndrome.

• Phase 2 data in Prader-Willi showed rapid and significant reduction in hyperphagia scores, with additional benefits in anxiety and inflammation.

• Phase 3 HERO trial is ongoing, with top-line results expected in Q3 2026; trial design includes measures to reduce placebo response and broaden patient eligibility.

• Enrollment is progressing well, with global sites and strong engagement from advocacy groups.

• ARD-101 is excluded for patients on contemporaneous Vycat, but prior Vycat use is allowed after a washout period.

Mechanism of action and scientific rationale

• ARD-101 activates gut enteroendocrine cells, releasing hormones like CCK, GLP-1, and GIP, which act via vagal signaling to reduce both hunger and appetite.

• The approach is distinct from GLP-1 drugs, which mainly reduce appetite but not hunger; ARD-101 addresses the hunger-driven phenotype of Prader-Willi.

• Clinical and inferential data suggest Prader-Willi patients have deficient gut-brain signaling, supporting the drug's mechanism.

• Dose selection was informed by phase 2 data, with 800 mg b.i.d. chosen to ensure consistent target engagement.

Comparative data and trial design

• Phase 2 open-label data showed a mean reduction of minus eight in HQCT scores over 28 days, exceeding typical placebo and competitor results.

• Secondary endpoints include anxiety, metabolic biomarkers, and body composition; phase 2 showed 1.5% body fat reduction and 2% lean mass gain.

• Phase 3 design includes a second baseline to minimize placebo effect and is powered for robust statistical confidence, with interim sample size re-estimation planned.