Summary of Acadia Pharmaceuticals FY Conference Call Company Overview - **Company**: Acadia Pharmaceuticals (NasdaqGS:ACAD) - **Event**: FY Conference held on September 18, 2025 Key Points on Prader-Willi Syndrome (PWS) Program - **Upcoming Data**: Top line phase 3 COMPASS data for ACP-101 (carbetocin) expected in early Q4, possibly October [3][4] - **Mechanism of Action**: ACP-101 is a modified version of oxytocin, designed to enhance pharmacokinetics and selectivity for the oxytocin receptor, which is relevant for treating hyperphagia in PWS [4][5] - **Animal Studies**: Evidence suggests that oxytocin replacement can mitigate hyperphagic behaviors in animal models of PWS [5] - **Clinical Trials**: Previous oxytocin studies showed mixed efficacy results but consistent safety and tolerability [7] - **Inverse Dose Response**: Prior phase 3 trial indicated an inverse dose response, with a significant p-value of 0.016 for the lower 3.2 mg dose compared to placebo [12] - **Safety Profile**: No serious adverse events reported in previous studies; some nosebleeds observed at higher doses [16][17] COMPASS Study Design - **Enrollment**: Target of 170 patients, randomized 1:1 between 3.2 mg dose and placebo [20] - **Primary Endpoint**: HQ-CT to measure hyperphagia-related behaviors [20][21] - **Secondary Endpoints**: Include clinician global impressions and caregiver burden assessments [21] ACP-204 Development - **New Compound**: ACP-204 is a next-generation 5HT2A inverse agonist aimed at treating Alzheimer's disease psychosis and Lewy body dementia [27][28] - **Study Design**: Phase 2 study with 318 patients, operationally seamless with phase 3 [29] - **Primary Endpoint**: SAPS-H and D at week six [29] - **Biomarker Confirmation**: Required for Alzheimer's disease psychosis patients, but not for Lewy body dementia [36][37] ACP-211 Overview - **New Compound**: ACP-211 is a noradrenaline compound for major depressive disorder (MDD) [38] - **Phase 2 Study**: Set to start in Q4 with 150 patients, focusing on moderate response [40] - **Safety Profile**: Aims for ketamine-like efficacy without sedation or significant dissociation [39][41] Business Development Strategy - **Focus Areas**: Emphasis on neuropsychiatry and rare diseases, particularly phase 2 assets [42] - **Pipeline Catalysts**: Key upcoming data includes ACP-101 in early Q4 and initiation of several new trials [43] Additional Insights - **Stakeholder Engagement**: Positive feedback from KOLs and caregivers regarding the potential treatment options for PWS [25] - **Regulatory Considerations**: FDA's focus on HQ-CT as a primary efficacy endpoint for approval [24] This summary encapsulates the critical insights and developments discussed during the Acadia Pharmaceuticals FY Conference, highlighting the company's ongoing research and strategic direction in neuropsychiatry.