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.

Company Overview - ACADIA Pharmaceuticals Inc. is a biopharmaceutical company focusing on therapies for central nervous system disorders and rare diseases [2] - Key approved products include NUPLAZID for Parkinson's disease psychosis and DAYBUE for Rett syndrome, both showing commercial growth [2] Financial Performance - In Q2 2025, NUPLAZID net sales increased by 7% year-over-year to $168.5 million, while DAYBUE sales grew by 14% to $96.1 million, reaching nearly 1,000 unique patients in the U.S. [2] Pipeline Development - A significant catalyst for 2025 is the upcoming Phase 3 readout of the COMPASS trial for ACP-101 in Prader-Willi Syndrome, with top-line results expected in early Q4 [3] - Positive trial outcomes could lead to a New Drug Application filing in Q1 2026 and potential approval by Q3 2026, expanding the company's pipeline into rare neurodevelopmental disorders [3] Research and Development Initiatives - The company is advancing multiple programs targeting neuropsychiatric symptoms and rare neurological diseases, including Alzheimer's disease and psychosis [4] - During its R&D Day in June 2025, ACADIA unveiled nine disclosed programs, with seven planned to enter Phase 2 or 3 stages from 2025 to 2026, and five anticipated readouts through 2027 [4] Market Position and Legal Developments - Recent patent litigation wins have secured market exclusivity for NUPLAZID through 2030 and extended formulations through 2038, providing stability for the commercial franchise [5] - With strong product momentum and a deepening pipeline, ACADIA is positioned for continued growth in CNS and rare disease therapies [5]

Summary of Acadia Pharmaceuticals (ACAD) FY Conference Call - August 13, 2025 Company Overview - Acadia Pharmaceuticals is a neurological and rare disease company with two commercial assets: NUPLAZID for Parkinson's disease psychosis and Debut for Rett syndrome [6][7] - The company anticipates over $1 billion in net sales for both assets combined this year [7] Key Products and Pipeline NUPLAZID - NUPLAZID is the first and only approved treatment for Parkinson's disease psychosis [7] - The company has seen solid growth due to effective field team execution and direct-to-consumer (DTC) marketing efforts, including a partnership with Ryan Reynolds [31][32] - A 17-fold increase in website traffic for NUPLAZID was reported, indicating successful engagement with the patient community [33] - The product is sensitive to legislative changes, particularly the Inflation Reduction Act, which may affect pricing and negotiation starting in 2029 [38][39] Debut (Trofinetide) - Debut is approved for Rett syndrome and has shown strong patient persistency rates: over 50% at 12 months and above 45% at 18 months [11][12] - Approximately 40% of patients treated with Debut are from Centers of Excellence (COEs), with a significant opportunity for growth in the remaining 60% [18][19] - The company is expanding its field team to increase penetration in underrepresented areas [19][22] - Acadia is seeking approval for Debut in the European Union, with expectations for Q1 of next year [27][29] Pipeline Assets - ACP-101 is in Phase 3 trials for Prader-Willi syndrome, with results expected in early Q4 2025 [8] - ACP-204 is being tested for Alzheimer's disease psychosis and Lewy Body dementia psychosis, with Phase 2 results anticipated mid-next year [8] - The company aims to leverage its experience from NUPLAZID to compete effectively in the Alzheimer's market, which has a large patient population [48][49] Business Development and Strategy - Acadia is actively pursuing business development opportunities to enhance its pipeline and sustain growth [9] - The company is focused on bringing in assets that are either first-in-class or best-in-class, with a preference for mid to late clinical-stage products [56][57] - The management emphasizes the importance of data-driven decision-making regarding investment in pipeline assets [52][53] Market Dynamics and Challenges - The company acknowledges the potential impact of legislative changes on its marketing strategies and pricing models [35][38] - Acadia is committed to maintaining compliance with all regulations while seeking innovative ways to engage with healthcare providers and patients [37][40] Conclusion - Acadia Pharmaceuticals is positioned for growth with its unique product offerings and a robust pipeline focused on neurological and rare diseases. The company is actively expanding its market presence while navigating potential regulatory challenges.

Core Insights - Acadia Pharmaceuticals reported Q2 2025 earnings with a slight beat on both GAAP earnings and revenue, driven by growth in NUPLAZID and DAYBUE product lines [1][5] - GAAP EPS reached $0.16, exceeding analyst expectations of $0.14, while revenue stood at $264.6 million, above the consensus of $262.0 million [1][2] - Revenue increased by 9.3% compared to Q2 2024, although GAAP net income declined due to higher tax and selling expenses [1][8] Financial Performance - Q2 2025 EPS (GAAP) was $0.16, down 20.0% from $0.20 in Q2 2024 [2] - Revenue for Q2 2025 was $264.6 million, with NUPLAZID net product sales at $168.5 million (up 7.0% year-over-year) and DAYBUE sales at $96.1 million (up 14% year-over-year) [2][6] - Research & Development expenses rose by 2.4% to $78.0 million, while Selling, General & Administrative expenses increased by 14.0% to $133.5 million [2][7] Business Overview - Acadia Pharmaceuticals focuses on developing treatments for central nervous system disorders, with primary products being NUPLAZID for Parkinson's disease psychosis and DAYBUE for Rett syndrome [3][4] - The company's strategy includes maximizing its drug franchises in the U.S., expanding internationally, and advancing its pipeline of new drug candidates [4] Market Position and Strategy - Acadia's drug pipeline includes nine experimental programs, with seven expected to enter Phase 2 or Phase 3 studies in the next two years [10] - Recent patent litigation victories have strengthened NUPLAZID's market protection, extending product exclusivity until at least 2030 [11] - The company is progressing its international plans for DAYBUE, with regulatory submissions in Europe under review and commercial launch activities ramping up ahead of anticipated approvals in 2026 [12] Guidance and Future Outlook - Management updated its full-year 2025 financial outlook, raising the lower bound of projected revenue and NUPLAZID net product sales guidance [13] - Total U.S. revenue guidance for FY2025 is set at $1.045 billion to $1.095 billion, with R&D expense guidance maintained at $330 million to $350 million [13] - Investors should monitor DAYBUE's patient ramp-up and retention rates, NUPLAZID's market share, and the progress of late-stage development programs [14]

Financial Data and Key Metrics Changes - Total revenue for the second quarter was $264.6 million, representing a 9% year-over-year increase [6][28] - Debut net product sales were $96.1 million, up 14% year-over-year, with a 12% increase in volume [28] - NUPLAZID net product sales were $168.5 million, up 7% year-over-year, with 5% of that growth attributable to volume [29] - R&D expenses increased to $78 million from $76.2 million year-over-year, while SG&A expenses rose to $133.5 million from $117.1 million [29] - Cash balance at the end of the quarter was $762 million, up from $681.6 million at the end of Q1 [30] Business Line Data and Key Metrics Changes - Dayview sales were $96.1 million, with 987 unique patients receiving paid shipments, an increase from 954 in Q1 [9][10] - NUPLAZID saw a 17% year-over-year increase in referrals, with the highest number of new Puzzet bottles shipped since launch [16][17] Market Data and Key Metrics Changes - The proportion of new referrals from non-Center of Excellence (COE) accounts grew to about 75% of overall referrals, up from two-thirds the previous quarter [43] - The number of healthcare providers writing prescriptions for Debut increased to 900, with a significant portion coming from community settings [12][43] Company Strategy and Development Direction - The company is focused on accelerating the commercial trajectory of Debut and sustaining long-term growth for NUPLAZID [32] - Plans to initiate seven Phase II or Phase III studies over 2025 and 2026, with five data readouts expected between 2025 and 2027 [20][21] - The company aims to expand globally and reach more patients in need, with a strong emphasis on community engagement and patient support [7][32] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the business's performance and raised the low end of NUPLAZID's revenue guidance for the year to between $665 million and $690 million [30] - The company anticipates sustained growth through the second half of the year, driven by strong execution and ongoing direct-to-consumer campaigns [16][17] Other Important Information - The company hosted its first R&D Day, showcasing nine programs in development and highlighting the potential of new molecules [8][19] - Management noted recent litigation wins that enforce intellectual property for Neuclazid, reinforcing long-term strategy [7] Q&A Session Summary Question: Can you provide details on ACP101's top-line approach and key risks? - Management indicated that the focus will be on the primary endpoint and key safety and tolerability overview in the top-line announcement, with ongoing monitoring of trial conduct [34][35] Question: What percentage of new patients for Debut came from community settings? - Approximately 75% of referrals came from non-COE accounts, indicating successful penetration into the community [43] Question: How is the growth trajectory for Debut expected to evolve? - The company expects continued acceleration in new patient starts, supported by the new customer model and a stable base of persistent patients [49] Question: What are the key drivers for NUPLAZID's growth? - Growth is driven by increased referrals and ongoing direct-to-consumer campaigns, with strong performance across all channels [68] Question: What is the outlook for 2026 regarding growth for both brands? - The company anticipates continued growth for both Debut and NUPLAZID, leveraging recent momentum and expanding market opportunities [100][102]

Financial Data and Key Metrics Changes - Total revenue for Q2 2025 was $264.6 million, representing a 9% year-over-year increase [5][27] - Debut net product sales were $96.1 million, up 14% year-over-year, with a gross to net adjustment of 23.3% [27] - NUPLAZID net product sales were $168.5 million, up 7% year-over-year, with a gross to net adjustment of 24.6% [27][28] - R&D expenses increased to $78 million from $76.2 million in the previous year, while SG&A expenses rose to $133.5 million from $117.1 million [28] Business Line Data and Key Metrics Changes - Debut saw a patient base increase to 987 unique patients, up from 954 in Q1 2025 and 920 in Q4 2024, indicating strong growth in new patient starts and persistency [9][10] - NUPLAZID referrals increased by 17% year-over-year, with both referrals and new prescriptions rising sequentially from Q1 to Q2 [15][16] Market Data and Key Metrics Changes - The community setting accounted for approximately three-quarters of new referrals for Debut, indicating successful penetration beyond centers of excellence [41] - The company is expanding its reach in the EU, with named patient supply available in multiple countries [13] Company Strategy and Development Direction - The company is focused on accelerating the commercial trajectory of Debut and sustaining long-term growth for NUPLAZID, while advancing a deep pipeline through rigorous clinical development [31] - The company raised the low end of its NUPLAZID guidance for the year, reflecting confidence in business performance [29] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the continued growth of Debut and NUPLAZID, supported by direct-to-consumer campaigns and field execution [5][15] - The company anticipates sustained growth through the second half of the year and beyond, with several key milestones in the pipeline [16][19] Other Important Information - The company hosted its first R&D Day, showcasing nine programs in development and five Phase II or Phase III data readouts expected through 2027 [7][19] - The company is progressing through the review process with EMA for trofinetide, with a decision expected in 2026 [21] Q&A Session Summary Question: About ACP101's top line approach and key risks - Management indicated that the focus will be on the primary endpoint and key safety and tolerability overview in the top line announcement, with ongoing monitoring of assessment variability [33][34] Question: Momentum in Debut and community setting penetration - Management reported that approximately three-quarters of new referrals came from non-center of excellence accounts, indicating successful penetration [41] Question: Discontinuations for Debut and inventory changes - Discontinuations remained below 10%, reflecting a stable patient base, and there were no significant inventory changes reported [73][74] Question: Growth perspective for 2026 - Management expects continued growth for both Debut and NUPLAZID, driven by new field force models and ongoing campaigns [96][100]

Financial Performance - Total revenue for Q2 2025 reached $264.6 million, a 9% increase year-over-year compared to $242.0 million in Q2 2024[10, 41] - NUPLAZID net sales in Q2 2025 were $168.5 million, up 7% year-over-year from $157.4 million in Q2 2024[10, 41] - DAYBUE net sales in Q2 2025 amounted to $96.1 million, reflecting a 14% increase compared to $84.6 million in Q2 2024[10, 15, 41] - The company's cash and investments balance stands at $762.0 million[41] - The company updates the full year 2025 total revenue guidance to $1.045 to $1.095 billion[44] DAYBUE Updates - 987 unique patients in the U S received DAYBUE in Q2, an increase from 954 in Q1[16] - More than 50% of patients persistency at 12 months and above 45% at 18 months[16] - 70% of active patients have been on therapy for 12 months or longer[16] NUPLAZID Updates - Referrals for NUPLAZID increased by 17% year-over-year[23] - Traffic to NUPLAZID com increased 17 times year-over-year[23] Research and Development - Top-line results from the COMPASS PWS Phase 3 study of ACP-101 are expected in early Q4 2025[11, 35] - The company anticipates filing an NDA for ACP-101 in Q1 2026, assuming positive data from the Phase 3 trial[35] - Phase 2 initiation of ACP-204 in Lewy Body Dementia Psychosis is expected in 3Q25[32]

Company Overview - ACADIA Pharmaceuticals (ACAD) is a commercial-stage biopharmaceutical company focused on neurological and rare diseases [1] - The company's portfolio includes two commercial drugs, with NUPLAZID specifically targeting positive symptoms in Parkinson's disease [1] Industry Insights - The healthcare sector is complex, requiring financial professionals and investors to have scientific and clinical expertise to navigate effectively [1] - There is a growing need for bridging the gap between cutting-edge science and financial strategy to uncover hidden value and assess risks accurately [1]

Summary of Acadia Pharmaceuticals (ACAD) R&D Day - June 25, 2025 Company Overview - Acadia Pharmaceuticals focuses on advancing care for patients with underserved neurological and rare diseases, guided by a mission to address high unmet needs in these areas [4][5][11] - The company has two profitable brands, NUPLAZID and Debut, which are expected to generate over $1 billion in combined revenue for the first time in 2025 [5][11] Financial Performance - NUPLAZID is projected to achieve net sales between GBP $650 million and GBP $690 million in 2025, reflecting a 7% to 13% year-over-year growth [6] - Debut is expected to generate net sales between $380 million and $405 million, indicating a 916% growth [7] - The total peak sales potential for NUPLAZID and Debut combined could reach between $1.5 billion and $2 billion [12] Pipeline and Future Growth - Acadia has a robust pipeline with nine disclosed programs, eight of which are expected to be in clinical stages by the end of the year [17] - The company anticipates five study readouts from Phase II or Phase III trials between now and the end of 2027 [17] - The pipeline includes later-stage assets like ACP101 for Prader-Willi syndrome and ACP204 for Alzheimer's disease psychosis, with top-line data expected in 2025 and 2026, respectively [8][17] Key Pipeline Assets 1. **ACP204**: A novel 5-HT2A inverse agonist targeting Alzheimer's and Lewy body dementia psychosis, with a significant unmet need as there are currently no approved therapies for these conditions [24][60] - Approximately 3 million people are affected by psychosis related to these diseases [27] - ACP204 is designed to avoid QT prolongation, allowing for higher dosing and potentially greater efficacy [30][60] 2. **ACP211**: An oral agent with potential ketamine-like efficacy for major depressive disorder (MDD) and treatment-resistant depression (TRD), aiming to minimize sedation and dissociation [62][81] - MDD affects approximately 21 million adults in the U.S., with only 9 million receiving treatment [67] - The drug is designed to provide rapid antidepressant effects without the severe side effects associated with current treatments [81] Strategic Focus - Acadia's growth strategy is anchored in precision medicine, data innovation, globalization, and patient empowerment [4] - The company aims to expand into adjacent rare disease categories, leveraging its scientific approach and internal capabilities [11] Market Opportunity - The potential market for the pipeline assets is significant, with an estimated combined peak sales potential of up to $12 billion if all five pipeline assets succeed [12][13] - Acadia believes that the current market undervalues its clinical programs, and it is committed to delivering data and executing its strategy to unlock this potential [13] Conclusion - Acadia Pharmaceuticals is positioned for transformational growth through its innovative pipeline and strong commercial brands, addressing critical needs in neurological and rare diseases while aiming to enhance patient care and outcomes [2][4][11]

Financial Performance and Expectations - Acadia Pharmaceuticals expects to generate over $1 Billion in total U S revenues in 2025[12] - The company estimates a risk-adjusted peak sales potential of $25 Billion from its pipeline molecules[17] - The full potential peak sales for pipeline molecules is estimated at $12 Billion, assuming successful approval and commercialization[17] - Potential peak sales of current products are projected to be in the range of $15-$2 Billion[19] Pipeline Development and Strategy - Acadia Pharmaceuticals is focused on advancing care for underserved neurological and rare diseases[11] - The company has a late-stage pipeline with ACP-101 (P3-PWS) data expected in early Q4 2025 and ACP-204 (P2-ADP) data expected in mid-2026[12] - Acadia has 2 approved medicines and 9 disclosed and multiple undisclosed programs[30] - The company anticipates 7 Phase 2 or Phase 3 study starts in 2025 and 2026, and 5 Phase 2 or Phase 3 study readouts between 2025 and 2027[30] Key Programs and Indications - ACP-204 is being developed for Alzheimer's Disease Psychosis (ADP) and Lewy Body Dementia with Psychosis (LBDP), targeting approximately 3 million patients with psychosis across PDP, LBDP, and ADP in the US[45] - ACP-211 is in development for Major Depressive Disorder (MDD), a condition affecting approximately 21 million adults in the US[93, 125] - ACP-711 is being investigated for Essential Tremor (ET), which affects an estimated 7 million people or 22% of the US population[136, 171] - DAYBUE, approved for Rett syndrome, has seen >50% of patients remain on treatment at 12 months and 65% on treatment for 12+ months[271]