Acadia Pharmaceuticals Inc. ACAD released topline results on Wednesday from its Phase 3 COMPASS PWS trial evaluating the efficacy and safety of intranasal carbetocin (ACP-101) in patients with hyperphagia in Prader-Willi syndrome (PWS).PWS is a rare genetic disorder characterized by muscle weakness and feeding difficulties in infancy, followed by an insatiable hunger and tendency toward severe obesity starting in childhood. PWS also involves delayed development, intellectual disability, behavioral issues an ...

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.

Core Viewpoint - Soleno Therapeutics, Inc. is actively participating in the 2025 United In Hope: International Prader-Willi Syndrome Conference, showcasing its commitment to advancing research and improving the lives of individuals affected by Prader-Willi syndrome (PWS) through multiple presentations [1][3]. Company Overview - Soleno Therapeutics focuses on developing novel therapeutics for rare diseases, with its first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, recently approved by the FDA on March 26, 2025 [11]. Conference Participation - The conference, anticipated to be the largest focused on PWS, is a collaboration between the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [2]. - Soleno will present one oral and six poster presentations, highlighting the significance of data and advocacy in addressing the needs of the PWS community [1][3]. Presentation Details - Key presentations include: - "Swallowability and Dosing Compliance of Diazoxide Choline Extended-Release (DCCR) Tablets in Patients with Prader-Willi Syndrome" [4]. - "Diazoxide Choline Extended-Release (DCCR) Tablets Significantly Reduce Hyperphagia in Patients with PWS Who Are Managed with Strict Food Controls" [4]. - "Long-term Efficacy Results of Diazoxide Choline Extended-Release (DCCR) Tablets in Participants with Prader-Willi Syndrome" [7]. - Presenters include experts from Queen Mary University of London and the University of Florida, indicating a strong academic collaboration [4][7][8]. About Prader-Willi Syndrome - PWS is a rare genetic neurodevelopmental disorder affecting approximately one in every 15,000 live births, characterized by hyperphagia, which can lead to severe health complications [9][10].

Group 1: Conference Overview - The 2025 United In Hope: International Prader-Willi Syndrome Conference is the first-ever international meeting jointly hosted by the Prader-Willi Syndrome Association | USA, the Foundation for Prader-Willi Research, and the International Prader-Willi Syndrome Organisation [1][2] - The conference is expected to be the largest focused on Prader-Willi syndrome (PWS) ever held, taking place from June 24-28, 2025, in Phoenix, AZ [2] Group 2: Company Involvement - Soleno Therapeutics, Inc. announced one oral and six poster presentations at the conference, highlighting its commitment to advancing research and improving the lives of those impacted by PWS [1][3] - The company's first commercial product, VYKAT™ XR (diazoxide choline) extended-release tablets, was approved by the U.S. FDA on March 26, 2025, for the treatment of hyperphagia in individuals with PWS [8] Group 3: Presentation Details - The oral presentation will cover long-term efficacy results of diazoxide choline extended-release tablets in participants with PWS from completed studies, presented by Evelien Gevers [4] - Multiple poster presentations will address various aspects of diazoxide choline extended-release tablets, including swallowability, dosing compliance, and the emotional impact of hyperphagia [4][5] Group 4: About Prader-Willi Syndrome - Prader-Willi syndrome (PWS) is a rare genetic neurodevelopmental disorder caused by an abnormality in gene expression on chromosome 15, occurring in approximately one in every 15,000 live births [6][7] - The defining symptom of PWS is hyperphagia, which can lead to severe health issues and significantly diminish the quality of life for affected individuals and their families [7]

Core Insights - Soleno Therapeutics, Inc. is presenting data on Prader-Willi syndrome (PWS) and its clinical program for VYKAT™ XR at the ISPOR Meeting 2025 [1][2] - VYKAT XR is approved for treating hyperphagia in patients with PWS aged 4 years and older [4][9] Group 1: Presentation Details - The first presentation focuses on mortality rates from a Phase 3 clinical trial of patients with hyperphagia and PWS treated with VYKAT XR, scheduled for May 14 [2] - The second presentation examines the burden of PWS on patients and the healthcare system, highlighting emergency department visits and inpatient stays, scheduled for May 15 [2] Group 2: About Prader-Willi Syndrome - PWS occurs in approximately 1 in 15,000 live births, characterized by hyperphagia, which can severely impact quality of life [3] - Additional symptoms include behavioral issues, cognitive disabilities, low muscle tone, and increased risk of serious health complications [3] Group 3: About VYKAT XR - VYKAT XR was approved by the FDA on March 26, 2025, and is now available for U.S. patients [4] - It is a once-daily oral treatment specifically for hyperphagia in individuals with PWS [9]

Core Insights - Soleno Therapeutics, Inc. announced the presentation of data from its VYKAT XR clinical development program for treating hyperphagia associated with Prader-Willi syndrome (PWS) at the upcoming Joint Congress of ESPE and ESE Meeting 2025 [1][2] Company Overview - Soleno Therapeutics is focused on developing and commercializing novel therapeutics for rare diseases, with its first commercial product being VYKAT XR, an oral treatment for hyperphagia in patients aged 4 years and older with PWS [10] Product Information - VYKAT XR (diazoxide choline) was approved by the FDA on March 26, 2025, and is now available to U.S. patients [2] - The product is indicated for the treatment of hyperphagia in adults and pediatric patients with PWS [4] Clinical Presentation Details - The company will present a study titled "The Impact of Prader-Willi Syndrome (PWS) on Caregivers and the Healthcare System: A Burden of Illness Study Design" in a poster format on May 11 and May 12, 2025 [2] - An oral presentation titled "Developmental Behaviour Checklist 2 Response and Relationship to Hyperphagia Reductions" is scheduled for May 13, 2025 [6] Understanding Prader-Willi Syndrome - PWS occurs in approximately 1 in every 15,000 live births, characterized by hyperphagia, which can severely impact the quality of life for affected individuals and their families [3] - Additional symptoms include behavioral problems, cognitive disabilities, low muscle tone, and increased risk of co-morbidities such as diabetes and cardiovascular disease [3]