Core Insights - ACADIA Pharmaceuticals aims to become a leading company in neurological and rare diseases, focusing on delivering meaningful innovations to underserved patients [2][3] Neurological Franchise - The momentum of NUPLAZID is expected to continue driving growth this year and beyond [3] - The company is advancing several pipeline candidates, including a new 5-HT2A agonist, remlifanserin (formerly ACP-204) [3] - Ongoing Phase II programs include ACP-211, a deuterated R-ketamine for major depressive disorder, and potential expansions into essential tremor with ACP-711 and tardive dyskinesia with ACP-271 [3]

Summary of ACADIA Pharmaceuticals FY Conference Call Company Overview - **Company**: ACADIA Pharmaceuticals (NasdaqGS:ACAD) - **Industry**: Neurological and Rare Diseases - **Conference**: 44th Annual J.P. Morgan Healthcare Conference - **Date**: January 13, 2026 Core Points and Arguments Company Mission and Strategy - ACADIA aims to deliver meaningful innovation to underserved patients with neurological and rare diseases, focusing on first-in-class or best-in-class products [2][4] - The company is advancing its pipeline with several candidates, including Remlifansirin (5-HT2A agonist) and ACP-211 (deuterated ketamine) for major depressive disorder [3][4] Pipeline Developments - **Neurological Franchise**: - NUPLAZID is expected to continue its momentum, with sales projected to reach $1 billion by 2028 [6][11] - Remlifansirin is anticipated to have a peak sales potential of $4 billion across indications [6] - Upcoming milestones include the initiation of phase two studies for ACP-271 and results for Remlifansirin expected between August and October 2026 [5][25] - **Rare Disease Franchise**: - DAYBUE is projected to achieve $700 million in global sales by 2028, driven by the launch of DAYBUE Sticks and expansion into the EU [14][30] - The company has treated over 2,000 patients with DAYBUE since its launch in 2023, with a 55% persistency rate at 12 months [13] Market Opportunities - There are approximately 1 million Parkinson's patients in the U.S., with 50% expected to develop hallucinations or delusions, presenting a significant market opportunity for NUPLAZID [10] - The company sees a potential growth opportunity in treating essential tremor, which affects about 7 million patients in the U.S. [23] Commercial Strategy - ACADIA plans to increase its commercial field force for NUPLAZID by 30% and leverage AI for better targeting and engagement [12][8] - The company is focused on expanding its presence in international markets, with DAYBUE already approved in three markets and anticipated approval in Europe [9][27] Financial Projections - Combined net sales for NUPLAZID and DAYBUE are projected to reach $1.7 billion by 2028 [6][30] - The company expects to see an uptick in operating expenses due to investments in commercial expansion and pipeline development [32][33] Additional Important Content - The company is committed to utilizing AI across various functions, including R&D and regulatory processes, to enhance efficiency and decision-making [7][9] - ACADIA is exploring business development opportunities in rare diseases beyond neurology, with a focus on late-stage products [34][35] - The company emphasizes the importance of patient-centric approaches in its clinical trials and product development [18][21] This summary encapsulates the key points discussed during the conference call, highlighting ACADIA Pharmaceuticals' strategic direction, pipeline developments, market opportunities, and financial outlook.

J.P. Morgan Healthcare Conference January 13, 2026 Catherine Owen Adams Chief Executive Officer 1 Forward-Looking Statements This presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include all statements other than statements of historical fact and can be identified by terms such as "may," "will," "should," "could," "would," "expects," "plans," "anticipates," "believes," "estimates," "projects," "predicts," ...

Company Overview - ACADIA Pharmaceuticals is a mid-cap biotechnology company focused on developing and commercializing therapies for central nervous system disorders, leveraging a focused pipeline and established commercial infrastructure to address significant unmet needs in neurology and psychiatry [6] - The company generates revenue primarily through the commercialization of NUPLAZID for Parkinson's disease psychosis, with an advancing pipeline that includes late-stage candidates for Alzheimer's disease psychosis and Rett syndrome [9] - As of the latest report, ACADIA's market capitalization is $4.4 billion, with a trailing twelve-month revenue of $1 billion and net income of $261.2 million [4] Recent Developments - Braidwell, a Connecticut-based fund, has established a new position in ACADIA Pharmaceuticals, acquiring nearly 2.4 million shares valued at approximately $50.8 million as of September 30, which represents 2.1% of the fund's total reportable U.S. equity assets for the quarter [2] - The stock price of ACADIA as of Wednesday is $25.88, reflecting a 59% increase over the past year, significantly outperforming the S&P 500, which has risen 13% in the same period [3] Financial Performance - ACADIA has recently delivered double-digit revenue growth, with expanding sales for both NUPLAZID and DAYBUE, and has more than doubled its net income in the third quarter [7] - The company has raised its full-year guidance and has nearly $850 million in cash on the balance sheet, positioning it well for continued growth [10] Market Position and Outlook - Despite the stock being more than 50% below its pandemic peak, the fundamentals of ACADIA appear stronger than in previous years, suggesting potential for a more durable recovery [7][12] - The confidence shown by a major biotech-focused fund stepping in indicates a positive trajectory for the company, emphasizing the importance of execution in converting late-stage assets into commercial successes [11]

Financial Data and Key Metrics Changes - The company expects over $1 billion in sales from its two commercial products, NUPLAZID and DAYBUE, for the current year [5] - NUPLAZID's sales guidance for the year has been narrowed to a range of $685 million to $695 million, indicating an upward shift from previous estimates [23][24] - The company maintains a strong balance sheet with over $800 million in cash and no debt, allowing for continued investment in business development [6][105] Business Line Data and Key Metrics Changes - NUPLAZID has seen a 20% increase in both referrals and new patient starts, marking the strongest quarter in new patient starts in a long time [10] - DAYBUE has experienced significant increases in new referrals, the largest since Q3 2024, indicating early success from increased investment in U.S. infrastructure [12] - The company plans to expand its customer-facing field force to further drive growth and profitability for both NUPLAZID and DAYBUE [11] Market Data and Key Metrics Changes - The company is preparing for European approval of DAYBUE next year, aiming to expand its market presence outside the U.S. [12][55] - The market for Alzheimer's disease psychosis (ADP) is significantly larger than that for Parkinson's disease psychosis (PDP), presenting a substantial growth opportunity [70] Company Strategy and Development Direction - The company is focused on expanding its commercial footprint and increasing investments in marketing and sales to enhance product awareness and penetration [11][12] - A robust pipeline is in place, with ACP-204 being the most advanced program targeting Alzheimer's disease psychosis and Lewy body dementia psychosis [6][61] - The company is committed to maintaining profitability while navigating potential price decreases due to future negotiations under the IRA [34] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in continued meaningful growth for both franchises, with plans to provide guidance on peak sales for NUPLAZID next year [28] - The company is mindful of potential legislative impacts but does not anticipate significant disruptions to its growth strategy in the near term [34] Other Important Information - The company has a stable patient base for DAYBUE, with over 70% of patients on therapy for 12 months or longer, indicating strong treatment adherence [40] - The company is actively building a team in Europe to prepare for the launch of DAYBUE, with a focus on establishing scientific conversations and market access [55] Q&A Session Summary Question: Can you provide an overview of the commercial performance for NUPLAZID and DAYBUE? - Management highlighted a strong quarter for NUPLAZID with a 20% increase in new patient starts and significant growth for DAYBUE due to increased referrals [10][12] Question: What is the sales guidance for NUPLAZID this year? - The sales guidance has been narrowed to a range of $685 million to $695 million, reflecting an upward adjustment [23][24] Question: How does the IRA impact the company? - Management indicated that while there may be price decreases in the future, the company expects to remain profitable and continue driving growth [34] Question: What is the strategy for expanding DAYBUE in Europe? - The company plans to market DAYBUE independently in Europe, starting with Germany, and has begun building a team to support this effort [55] Question: What are the expectations for the upcoming phase II readout for ACP-204? - Management expressed excitement about the phase II readout for ACP-204, viewing it as a higher probability of success due to prior learnings from pimavanserin [61][62]

Summary of ACADIA Pharmaceuticals Conference Call Company Overview - **Company**: ACADIA Pharmaceuticals (NasdaqGS:ACAD) - **Focus**: Building a leading neurological and rare disease company with aspirations to become a global biotech powerhouse [2][3] Core Industry Insights - **Market Position**: ACADIA is focused on long-term growth in neurological and rare diseases, leveraging its existing product NUPLAZID for Parkinson's disease psychosis and expanding its pipeline [2][3] - **Pipeline Development**: The company is advancing several promising candidates, including ACP-204 for Alzheimer's disease psychosis and DAYBUE for Rett syndrome, with plans for global expansion [3][4] Financial Highlights - **Revenue Projections**: Expected revenues for 2025 are over $1 billion, with peak potential for NUPLAZID and DAYBUE estimated between $1.5 billion and $2 billion [4][5] - **Sales Growth**: NUPLAZID reported record sales of $177.5 million in Q3, reflecting a 12% year-over-year growth, with significant increases in prescriptions and referrals [9][10] - **DAYBUE Performance**: DAYBUE achieved over $100 million in sales in Q3, with over 1,000 patients treated globally since its launch [11][12] Pipeline and Product Development - **Key Products**: - **NUPLAZID**: First FDA-approved treatment for hallucinations and delusions associated with Parkinson's disease, with a market share of approximately 25% [8][9] - **DAYBUE**: First treatment for Rett syndrome, with a growing diagnosis rate and strong efficacy data [10][11] - **Upcoming Milestones**: - Phase two study initiation for ACP-211 in major depressive disorder and phase one study for ACP-271 in Q1 2026 [6][20] - Anticipated CHMP opinion for DAYBUE in Q1 2026 [11][24] Strategic Focus Areas - **Precision Medicine**: Emphasis on using biomarkers in trials and integrating AI and machine learning into development processes [4] - **Global Expansion**: Strong focus on expanding operations beyond the U.S. into Europe and Japan [4] - **Patient Empowerment**: Centering patient journeys in the development and delivery of treatments [4] Unmet Medical Needs - **Alzheimer's Disease Psychosis**: Approximately 800,000-850,000 patients in the U.S. experience hallucinations or delusions, with no approved treatments available [14][17] - **Lewy Body Dementia**: A significant portion of patients (50%-70%) experience psychosis, highlighting the need for effective therapies [17][18] - **Major Depressive Disorder**: 21 million adults diagnosed in the U.S., with only 9 million currently treated, indicating a large market opportunity [19][20] Conclusion - **Future Outlook**: ACADIA is positioned for growth with a strong pipeline and financial health, aiming to address significant unmet needs in neurological and rare diseases while expanding its market presence [24]

Summary of Acadia Pharmaceuticals Conference Call Company Overview - Acadia Pharmaceuticals is a neurological and rare disease company with both commercial and development-stage products, specifically NUPLAZID for Parkinson's disease psychosis and DABU for Rett syndrome [3][4] Financial Performance - The company expects to exceed $1 billion in revenue for the first time this year [4] - Acadia is cash flow positive with over $800 million in cash and no debt [4] Product Insights NUPLAZID - NUPLAZID has been on the market for about 8-9 years and is in its mid-life cycle [6] - The company is focusing on increasing awareness and expanding its commercial footprint, including a 30% increase in its sales force planned for January [10][9] - The product experienced double-digit year-over-year revenue growth and 9% volume growth in the third quarter [8] - An intellectual property (IP) win earlier this year extends the product's patent protection through February 2038 [10] DABU - DABU has 38 territories with representatives supporting its franchise, indicating a need for increased outreach to physicians [18][19] - The company is seeing an increase in referrals, marking the highest count since 2024 [20] Market Dynamics - The company is focusing on educating both physicians and caregivers about the diseases treated by its products, particularly in the context of rare diseases like Rett syndrome [22][25] - The persistency rates for DABU are over 50% at 12 months and 45% at 18 months, indicating a stable patient base [27][29] Pipeline and R&D - Acadia's pipeline includes ACP-204, targeting Alzheimer's disease psychosis and Lewy body dementia psychosis, with a Phase II readout expected mid-next year [4][39] - The company estimates a potential revenue opportunity of $11 billion from its pipeline, despite setbacks with one of its assets [37][38] - ACP-211, a deuterated norketamine, is set to enter Phase II studies for major depressive disorder [41] Competitive Landscape - The company is aware of competitors in the market but emphasizes its unique product profiles and the unmet needs in the neurological space [48][49] - Acadia is focusing on a robust target product profile for its pipeline assets, particularly in terms of safety and dosing convenience [59] International Market Considerations - The company is exploring opportunities in Europe, noting differences in care structures compared to the U.S., particularly in Germany and France [32][33] Conclusion - Acadia Pharmaceuticals is positioned for growth with a strong financial foundation, expanding product lines, and a robust pipeline aimed at addressing significant unmet medical needs in neurological disorders [4][37]

Financial Performance - Total revenue for Q3 2025 reached $278.6 million, an 11% increase year-over-year compared to $250.4 million in Q3 2024[31] - NUPLAZID net sales in Q3 2025 were $177.5 million, a 12% increase year-over-year from $159.2 million in Q3 2024[31] - DAYBUE net sales in Q3 2025 hit a record $101.1 million, an 11% increase year-over-year compared to $91.2 million in Q3 2024[31, 13] - The company's EPS (diluted) increased by 110% to $0.42 in Q3 2025, compared to $0.20 in Q3 2024[31] - The company updated its full-year 2025 revenue guidance to $1.07 to $1.095 billion[32] Commercial Updates - NUPLAZID referrals grew by 21% year-over-year[17] - New prescriptions for NUPLAZID increased by 23% year-over-year[19] - 74% of new prescriptions (NBRx) for DAYBUE came from community physicians in Q3[16, 15] - Over 1,006 patients have been treated with DAYBUE globally[14] Research and Development - A Phase 2 study of ACP-204 for Lewy Body Dementia Psychosis was initiated[10, 27] - A Phase 3 trial of trofinetide for Rett syndrome in Japan was initiated[10, 27]

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]