Neurogene (NasdaqGM:NGNE) 2026 Conference March 09, 2026 01:40 PM ET Company ParticipantsRachel McMinn - Founder and CEOConference Call ParticipantsMarc Frahm - Managing Director and Senior Equity AnalystMarc FrahmWelcome back to our afternoon session of the first day of the Leerink Partners Global Healthcare Conference here in Miami. I'm still Marc Frahm, Senior Analyst at TD Cowen, and I have with me Rachel McMinn, CEO of Neurogene. Rachel, how are you doing?Rachel McMinnI'm good. I'm good. Thank you for ...

Financial Data and Key Metrics Changes - ACADIA reported adjusted total revenues of $298 million in Q4 2025, a 16% increase from the prior year, and annual revenues exceeded $1 billion for the first time, reaching $1.08 billion, representing a 14% growth from the previous year [6][24] - Adjusted NUPLAZID net sales were $189 million in Q4 and $692 million for the full year, reflecting a 17% and 15% increase year-over-year, respectively [7][26] - DAYBUE achieved net product sales of $110 million in Q4 and $391 million for the full year, representing 13% and 12% year-over-year growth, respectively [8][27] Business Line Data and Key Metrics Changes - NUPLAZID's volume growth was 13% in Q4 and 9% for the full year, with new prescriptions growing 18% year-over-year in Q4 [13][15] - DAYBUE's sales growth was driven by expanded reach into community physician settings and international patient supply programs [8][18] - The launch of DAYBUE STIX, a new powder formulation, is generating significant interest and is expected to unlock additional patient opportunities [17][19] Market Data and Key Metrics Changes - The company expects NUPLAZID net sales of $760 million to $790 million in 2026, indicating a growth of 10% to 14% over 2025 [7][29] - DAYBUE is projected to achieve global net sales between $460 million and $490 million in 2026, representing an 18% to 25% growth over 2025 [9][30] Company Strategy and Development Direction - ACADIA aims to achieve blockbuster status for NUPLAZID with $1 billion in net sales by 2028, supported by strong commercial execution and pipeline advancements [7][32] - The company is committed to advancing access to trofinetide in the EU despite recent regulatory challenges, with plans for reexamination [9][22] - The R&D pipeline includes four unique molecules targeting large addressable markets with a combined peak sales potential of $11 billion [11][23] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the growth trajectory of both NUPLAZID and DAYBUE, with expectations for continued strong performance in 2026 and beyond [6][32] - The company highlighted the importance of upcoming milestones, including phase 2 study results for remlifanserin, which could significantly impact long-term growth [20][32] Other Important Information - The company reported a one-time non-cash income tax benefit of approximately $250 million due to the release of the valuation allowance on deferred tax assets [28] - Cash balance at the end of 2025 was $820 million, providing flexibility for future growth initiatives [28] Q&A Session Summary Question: How should we think about ramp to your 2028 global net sales targets? - Management indicated confidence in achieving low to mid-teens growth for NUPLAZID and low 20% growth for DAYBUE, projecting a CAGR of about 16% through 2028 [36][37] Question: What does good remlifanserin ADP data look like later this year? - Management is looking for evidence of efficacy and a good safety profile, with a focus on compliance and ease of use for patients [44][47] Question: Can you provide insights on DAYBUE's persistency and patient compliance? - Discontinuation rates remain low in the single-digit range, and the company is optimistic about growth driven by the new STIX formulation [52][56] Question: What are the specific concerns regarding the EU opinion for DAYBUE? - Management anticipates questions related to the relevance of endpoints and clinical meaningfulness, and plans to address these in the re-examination process [61][66]

Financial Data and Key Metrics Changes - Acadia reported adjusted total revenues of $298 million in Q4 2025, a 16% increase from the prior year, and annual revenues exceeded $1 billion for the first time, reaching $1.08 billion, representing a 14% growth from the previous year [6][24] - Adjusted NUPLAZID net sales were $189 million in Q4, up 17% year-over-year, and $692 million for the full year, up 15% [7][27] - DAYBUE achieved net product sales of $110 million in Q4, representing a 13% year-over-year increase, and $391 million for the full year, up 12% [8][28] Business Line Data and Key Metrics Changes - NUPLAZID's volume growth was 13% in Q4 and 9% for the full year, with new prescriptions growing 18% year-over-year in Q4 [13][15] - DAYBUE's sales growth was driven by expanded reach into community physician settings and international patient supply programs [8][19] - DAYBUE STIX, a new powder formulation, is generating significant interest and is expected to unlock additional patient opportunities [17][19] Market Data and Key Metrics Changes - The U.S. market for NUPLAZID and DAYBUE continues to show strong demand, with 76% of new DAYBUE prescriptions originating from community-based physicians [16][19] - DAYBUE Liquid is now approved in three international markets, including Israel, expanding Acadia's global footprint [18] Company Strategy and Development Direction - Acadia aims for NUPLAZID to achieve blockbuster status with $1 billion in net sales by 2028, with 2026 guidance for NUPLAZID net sales between $760 million and $790 million [7][30] - The company is focused on expanding its R&D pipeline, with several key studies and potential new product launches planned [20][23] - Acadia is committed to advancing access to trofinetide in the EU despite recent regulatory challenges [9][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's growth trajectory, supported by strong commercial execution and a robust pipeline [6][33] - The anticipated phase II readout of remlifanserin in Alzheimer's disease psychosis is expected to be a significant milestone for the company [11][33] - The company is well-positioned for sustained growth and value creation, with a strong balance sheet allowing for business development opportunities [33] Other Important Information - The company reported a one-time non-cash income tax benefit of approximately $250 million due to the release of a valuation allowance on deferred tax assets [29] - R&D expenses for 2026 are expected to be between $385 million and $410 million, reflecting increased clinical and personnel costs [31][32] Q&A Session Summary Question: How should we think about ramp to your 2028 global net sales targets? - Management expects low to mid-teens growth for NUPLAZID and low 20% growth for DAYBUE, projecting a CAGR of about 16% through 2028 [36][37] Question: What does good remlifanserin ADP data look like later this year? - The focus is on showing efficacy and a good safety profile, with a moderate effect size powered for the primary endpoint [43][47] Question: Can you provide insights on DAYBUE's persistency and patient compliance? - Discontinuation rates remain in the low single-digit range, and the business is stabilizing with a focus on growth through community expansion [51][53] Question: What is the timeline for the upcoming phase III trial in ADP? - The transition from phase II to phase III enrollment is expected later this year, with operationally seamless execution [61] Question: How does remlifanserin differentiate from COBENFY? - Mechanistically, remlifanserin and COBENFY approach psychosis differently, with remlifanserin expected to have a favorable safety profile [67][68]

Financial Data and Key Metrics Changes - The company reported adjusted total revenues of $298 million in Q4 2025, a 16% increase from the prior year, and annual revenues exceeded $1 billion for the first time, reaching $1.08 billion, representing a 14% growth from the previous year [5][24] - Adjusted NUPLAZID net sales were $189 million in Q4 and $692 million for the full year, reflecting a 17% and 15% increase year-over-year, respectively [6][26] - DAYBUE achieved net product sales of $110 million in Q4 and $391 million for the full year, representing 13% and 12% year-over-year growth, respectively [7][27] Business Line Data and Key Metrics Changes - NUPLAZID's volume growth was 13% in Q4 and 9% for the full year, with new prescriptions growing 18% year-over-year in Q4 [11][12] - DAYBUE's sales growth was driven by expanded reach into community physician settings and international patient supply programs [7][17] - The company expects NUPLAZID net sales of $760 million to $790 million in 2026, indicating 10%-14% growth over 2025 [6][29] Market Data and Key Metrics Changes - The company anticipates global net sales for DAYBUE between $460 million and $490 million in 2026, driven by the launch of DAYBUE STIX and continued growth in named patient supply programs [8][30] - The company projects 2028 global net sales for DAYBUE of $700 million, with EU sales representing less than 15% of the total [9] Company Strategy and Development Direction - The company is focused on advancing its R&D pipeline, with several key catalysts expected in 2026, including the phase 2 readout of remlifanserin [10][23] - The company aims to achieve blockbuster status for NUPLAZID with $1 billion in net sales by 2028 [6][14] - The company is committed to expanding access to trofinetide in the EU and has active named patient supply programs [8][22] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's growth trajectory, supported by strong commercial execution and a robust pipeline [33] - The company anticipates continued growth in both NUPLAZID and DAYBUE, with foundational investments in SG&A expected to deliver meaningful top-line growth [29][31] - Management highlighted the importance of regulatory developments and the potential for reexamination of trofinetide in the EU [22][23] Other Important Information - The company reported a one-time non-cash income tax benefit of approximately $250 million in Q4 due to the release of the valuation allowance on deferred tax assets [28] - The cash balance at the end of 2025 was $820 million, providing flexibility for future growth initiatives [28] Q&A Session Summary Question: How should we think about ramp to your 2028 global net sales targets? - Management indicated confidence in achieving low to mid-teens growth for NUPLAZID and low 20% growth for DAYBUE, projecting a CAGR of about 16% through 2028 [35][36] Question: What does good remlifanserin ADP data look like later this year? - Management is looking for evidence of efficacy and a good safety profile, with a focus on not negatively impacting motor or cognitive functions [44][48] Question: Can you provide insight into DAYBUE's persistency and patient compliance? - Discontinuation rates remain in the low single-digit range, and the company is seeing benefits from its community expansion strategy [53][55] Question: What are the specific concerns regarding the EU opinion for DAYBUE? - Management anticipates questions related to the relevance of endpoints and clinical meaningfulness, and plans to address these in the re-examination process [62][66]

Fourth Quarter and Full Year 2025 Earnings Call February 25, 2026 Call Agenda Catherine Owen Adams Chief Executive Officer Welcome Commercial Update Tom Garner Executive Vice President, Chief Commercial Officer Elizabeth H.Z. Thompson, Ph.D. Executive Vice President, Head of Research and Development R&D Update Financial Update Mark Schneyer Executive Vice President, Chief Financial Officer Al Kildani Senior Vice President, IR and Corporate Communications CEO Opening Remarks Closing Remarks Catherine Owen Ad ...

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]