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]

Acadia Pharmaceuticals Conference Call Summary Company Overview - **Company**: Acadia Pharmaceuticals (ACAD) - **Event**: BofA Healthcare Conference - **Date**: May 13, 2025 Key Industry Insights - **Most Favored Nation (MFN) Executive Order**: - No immediate impact on Acadia as it currently does not sell products outside the US [3] - Plans to commercialize Debut in Europe, with regulatory approval expected in Q1 2026 [4] - Initial launch in Germany with free pricing for the first six months [4] - **Tariffs**: - Acadia does not foresee meaningful exposure to tariffs due to existing inventory in the US that can last until the mid to late 2030s [7][9] - No manufacturing facilities owned, allowing flexibility in response to future tariff changes [8] - **FDA Interactions**: - No immediate negative impacts reported; routine interactions with the FDA are proceeding as expected [17][18] Company-Specific Developments - **Debut Launch**: - Initial strong demand followed by stagnation; new Chief Commercial Officer hired to enhance commercial execution [21][22] - Field force increased by 30% to improve outreach to physicians, particularly in local pediatric neurology centers [23] - Focus on efficacy messaging and one-to-one patient interactions to improve patient retention [24][25] - New patient starts have shown a significant increase in Q1 2025, with expectations for continued growth [26][36] - **NUPLAZID**: - Awareness campaigns have led to the best quarter in five years for new patient starts [49] - Focus on educating patients and families about Parkinson's disease psychosis, with a strong emphasis on early diagnosis [50][52] - Steady growth expected as awareness increases, with a larger patient population compared to Debut [55] Pipeline Updates - **ACP-101**: - Phase 3 trial expected to complete enrollment in Q2 2025, with top-line data anticipated in early Q4 2025 [58][60] - Primary endpoint focuses on the hyperphagia questionnaire, with a safety profile similar to previous trials [61][62] - **ECP-204**: - New inverse agonist targeting 5-HT2A receptors, currently in phase 2/3 trials for Alzheimer's disease psychosis [73] - Aiming for faster onset of action and reduced QT prolongation compared to NUPLAZID [74] Additional Considerations - **Market Dynamics**: - The rare disease market is seen as having room for multiple therapies, with Acadia's products positioned to meet diverse patient needs [66][70] - The company is focused on leveraging its expertise in rare diseases and neurology for successful product launches [71] - **Future Events**: - R&D Day scheduled for June 25, 2025, to provide further updates on pipeline developments [79]

Financial Data and Key Metrics Changes - The company reported total revenues of $244.3 million for Q1 2025, representing a 19% increase year-over-year [7][28] - Debut net product sales were $84.6 million, up 11% from the previous year, while NUPLAZID sales reached $159.7 million, reflecting a 23% increase year-over-year [8][30] - R&D expenses increased to $78.3 million from $59.7 million in Q1 2024, and SG&A expenses rose to $126.4 million from $108 million in the same period [31] Business Line Data and Key Metrics Changes - Debut sales showed a significant increase in unique patients receiving shipments, reaching an all-time high of 954, up from 920 in Q4 2024 [11][12] - NUPLAZID's growth included a 6% increase in volume, with the remainder attributed to one-time changes in gross to net [8][30] - The company expects continued growth in both brands, with a focus on expanding patient engagement and community reach [7][9] Market Data and Key Metrics Changes - The company is preparing for a strong launch of Debut in Europe, anticipating EMA approval in Q1 2026, with a substantial market opportunity for Rett syndrome patients [14][15] - The U.S. market for NUPLAZID continues to show strong momentum, with a nearly 30% increase in awareness of Parkinson's-related hallucinations and delusions since the launch of the More to Parkinson's campaign [17][18] Company Strategy and Development Direction - The company is focused on executing strategic priorities, including accelerating Debut's commercial trajectory, capitalizing on NUPLAZID's momentum, and advancing its pipeline [33][34] - The upcoming R&D Day is expected to provide insights into the company's development strategy and showcase innovations across its pipeline [24][34] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the sustainability of Debut's growth and reiterated full-year sales guidance for both Debut and NUPLAZID [8][9] - The company is optimistic about the upcoming results from the COMPASS PWS Phase III study, with top-line results expected by Q4 2025 [19][33] Other Important Information - The company has a strong cash balance of $681.6 million as of March 31, 2025, despite a sequential decline due to significant payments made [31] - The company is actively looking for strategic transactions to expand its portfolio, particularly in areas of high unmet medical need [84] Q&A Session Summary Question: What does good 101 data look like and how does it relate to FDA discussions? - Management indicated that statistically significant results demonstrating a clinically meaningful impact on hyperphagia would be considered good data [39] Question: How should we model a European price for Debut? - Management expressed confidence in obtaining a strong EU price that reflects the value provided to the Rett community, while monitoring potential pricing risks [40][41] Question: What are the key growth drivers for NUPLAZID and Debut from Q1 to Q2? - Management highlighted that volume will drive performance, with expectations for continued patient engagement and operational improvements [48][49] Question: What is the confidence level for EMA approval for trofinetide? - Management expressed confidence in the EMA approval process, noting that they are on track with the timeline and have received no indications of issues [82] Question: How does the company view its positioning against competitors in the Prader Willi market? - Management believes there is room for multiple therapies in the Prader Willi space, emphasizing the complex needs of patients and the potential for combination therapies [95]

Financial Data and Key Metrics Changes - Acadia Pharmaceuticals reported total revenues of $244.3 million for Q1 2025, representing a 19% increase year-over-year [6][29] - R&D expenses increased to $78.3 million from $59.7 million in Q1 2024, driven by higher spending on clinical stage programs [32] - SG&A expenses rose to $126.4 million from $108 million in Q1 2024, primarily due to ongoing consumer activation campaigns for NUPLAZID and expansion of the Debut team [32] Business Line Data and Key Metrics Changes - Debut sales reached $84.6 million in Q1 2025, an 11% increase from the previous year, with a record number of unique patients receiving shipments [7][29] - NUPLAZID generated $159.7 million in revenue for the quarter, up 23% year-over-year, with 6% of this growth attributed to volume [7][31] Market Data and Key Metrics Changes - The number of unique patients receiving Debut shipments was 954, marking an all-time high for the brand [7][11] - NUPLAZID's gross price increased by over 2% year-over-year, with a gross-to-net adjustment of 24.1% for the quarter [31] Company Strategy and Development Direction - Acadia is focused on accelerating the commercial trajectory of Debut and capitalizing on the momentum of NUPLAZID while expanding its global presence [35] - The company plans to host its first R&D Day to provide insights into its development strategy and pipeline innovations [9][35] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the sustainability of Debut's growth and reiterated full-year sales guidance for both Debut and NUPLAZID [7][35] - The timeline for the COMPASS PWS Phase III study results is now expected by the end of 2025, indicating progress in pipeline development [9][35] Other Important Information - Acadia anticipates EMA approval for Debut in Q1 2026, with a substantial market opportunity in Europe for Rett syndrome patients [14][28] - The company has received orphan drug designation for trofinetide in Japan, which may lead to priority review [28] Q&A Session Summary Question: What does good 101 data look like and how does it relate to FDA discussions? - Management indicated that statistically significant results demonstrating a clinically meaningful impact on hyperphagia would be considered good data [40] Question: How should we model a European price for Debut? - Management expressed confidence in obtaining a strong EU price that reflects the value provided to patients in the Rett community [41] Question: What are the key growth drivers for NUPLAZID and Debut from Q1 to Q2? - Management highlighted patient demand and the impact of expanded customer models as key growth drivers for both brands [44][46] Question: What are the biggest risks to the approval process for trofinetide in the EU? - Management stated confidence in the EMA approval process, noting that they are on track with the regulatory timeline [80] Question: How does Acadia view its positioning against Celeno's product for Prader Willi? - Management believes there is room for multiple therapies in the Prader Willi space, emphasizing the complexity of patient needs [95]