Aardvark Therapeutics FY Conference Summary Company Overview - **Company**: Aardvark Therapeutics (NasdaqGS:AARD) - **Founded**: 2017 by Dr. Tien Lee - **Focus**: Development of treatments for rare diseases, specifically in the metabolic space, including Prader-Willi syndrome and obesity [3][4] Pipeline and Research Focus - **Key Programs**: - **Prader-Willi Syndrome**: Lead compound ARD-101, a bitter taste receptor agonist targeting hunger pathways [4][5] - **Obesity**: ARD-201, a combination of ARD-101 and sitagliptin (Januvia), aimed at enhancing incretin release and prolonging their half-life [4][5] Mechanism of Action - **ARD-101**: Targets bitter taste receptors in the gut to release incretins (GLP-1 and CCK), signaling the brain to reduce hunger and appetite [4][5] - **ARD-201**: Combines the effects of ARD-101 with sitagliptin to enhance the incretin pathway and maintain weight loss [41][42] Clinical Trials - **Phase 3 Trial for Prader-Willi Syndrome**: Ongoing, with top-line data expected in Q3 2026 [10][11] - **Phase 2 Data**: Showed significant reduction in hyperphagia in most patients, with a focus on safety and tolerability [12][13] - **Hyperphagia Questionnaire (HQCT)**: Used to assess treatment efficacy, with a one-point difference considered clinically meaningful [17][18] Patient Demographics and Inclusion Criteria - **Age Range**: Phase 3 trial includes patients aged 10 and above, previously 13 and above, to target younger patients who experience hyperphagia earlier [20][21] - **Antipsychotic Use**: Patients on antipsychotics are included to ensure real-world representation, with 20%-30% of the Prader-Willi population typically using them [33][34] Geographic Distribution - **Trial Sites**: Approximately one-third of patients are from the U.S., with the remainder from countries including Canada, Australia, South Korea, and various EU nations [35] Quality Control Measures - **Assessment Consistency**: Caregivers and physicians remain consistent throughout the trial to minimize variability in HQCT scores [24][26] - **Blinded Checks**: Implemented to ensure quality control of assessments [26] Future Studies - **Weight Maintenance Study**: Anticipated preliminary data by the end of next year, focusing on maintaining weight after GLP-1 receptor agonist treatment [41][43] - **Weight Induction Study**: Aims to evaluate the effectiveness of ARD-201 in driving greater weight loss compared to existing treatments [50] Conclusion - Aardvark Therapeutics is positioned to make significant advancements in the treatment of Prader-Willi syndrome and obesity through innovative drug mechanisms and robust clinical trial designs, with key data expected in the coming years [51][52]