Slides
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Second Quarter 2025 Earnings Call AUGUST 6, 2025
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Call Agenda 2 Welcome Al Kildani | Senior Vice President, Investor Relations and Corporate Communications Commercial Update T om Garner | Executive Vice President, Chief Commercial Officer CEO Opening Remarks Catherine Owen Adams | Chief Executive Officer Financial Update Mark Schneyer | Executive Vice President, Chief Financial Officer Q&A Session All Closing Remarks Catherine Owen Adams | Chief Executive Officer R&D Update Elizabeth H.Z. Thompson | Executive Vice President, Head of Research and Development
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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,” “outlook,” “potential,” "milestone," "guidance" and similar expressions (including the negative thereof) intended to identify forward-looking statements. Forward-looking statements contained in this presentation, include, but are not limited to, statements about: (i) our business strategy, objectives and opportunities; (ii) plans for, including timing, development and progress of commercialization or regulatory timelines for our products, includingNUPLAZID and DAYBUE, and our product candidates; (iii) benefits to be derived from and efficacy of our products, including the potential advantages of our products, and for DAYBUE in Canada and trofinetide in jurisdictions outside the U.S.; (iv) estimates regarding the prevalence of the diseases targeted by our products and product candidates; (v) potential markets for any of our commercial products; and (vi) our estimates regarding our future financial performance, cash position, profitability or capital requirements. Forward-looking statements are subject to known and unknown risks, uncertainties, assumptions and other factors that may cause our actual results, performance or achievements to differ materially and adversely from those anticipated or implied by our forward-looking statements. Such risks, uncertainties and other factors include, but are not limited to: our dependency on the continued successful commercialization of our products and our ability to maintain or increase sales of our products; the costs of our commercialization plans and development programs, and the financial impact or revenues from any commercialization we undertake; our ability to obtain necessary regulatory approvals for our product candidates and, if and when approved, market acceptance of our products; our dependence on third- party collaborators, clinical research organizations, manufacturers, suppliers and distributors; the impact of competitive products and therapies; our ability to generate or obtain the necessary capital to fund our operations; our ability to grow, equip and train our specialized sales forces; our ability to manage the growth and complexity of our organization; our ability to maintain, protect and enhance our intellectual property; our ability to meet our financialguidance; and our ability to continue to stay in compliance with applicable laws and regulations. Given the risks and uncertainties, you should not place undue reliance on these forward-looking statements. For a discussion of these and other risks, uncertainties and other factors that may cause our actual results, performance or achievements to differ, please refer to our annual report on Form 10-K for the year ended December 31, 2024 as well as our subsequent filings with the Securities and Exchange Commission from time to time, including our quarterly reports on Form 10-Q. The forward-looking statements contained herein are made as of the date hereof, and we undertake no obligation to update them after this date, except as required by law. Forward-Looking Statements 3
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Opening Remarks 4 CHIEF EXECUTIVE OFFICER Catherine Owen Adams
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DAYBUE (trofinetide) is only approved in the U.S. by the FDA and in Canada by Health Canada for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. NUPLAZID (pimavanserin) is only approved in the U.S. by the FDA for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis. Second Quarter Highlights Q2 2025 sales of $96.1 million, up 14% year-over-year Patient uptake continued to grow for the second successive quarter Q2 2025 sales of $168.5 million, up 7% year-over-year Encouraged by continued growth and recent wins defending our intellectual property 5 R&D Updates Top-line results expected in early Q4 2025 following completion of enrollment for COMPASS PWS Phase 3 study in Q2 Held inaugural R&D Day on June 25, 2025 Q2 Total Revenues of $264.6 million, up 9% year-over-year
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Commercial Update T om Garner CHIEF COMMERCIAL OFFICER 6
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DAYBUE Second Quarter Updates KEY DRIVERS ▷ 987 unique patients in the U.S. received DAYBUE, up from 954 in Q1 ▷ Long term persistency a key strength: more than 50% at 12 months and now reporting above 45% at 18 months ▷ 70% of active patients have now been on therapy 12 months or longer ▷ Completed field force expansion to further accelerate growth into the community ▷ Strengthened leadership with addition of Allyson McMillian-Youngblood as SVP , Rare Disease franchise $96.1M 7 in Q2 Net Product Sales Up 14% vs. Q2 2024
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8 Update on DAYBUE Outside United States ▷ Named patient supply programs are active through Clinigen ▷ Continuing to build Acadia commercialization team EU REST OF THE WORLD ▷ Commenced named patient supply of trofinetide for eligible patients based upon healthcare professional requests in in Europe, Israel, and select rest of the world countries. DAYBUE is approved in the U.S. and Canada
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NUPLAZID Second Quarter Updates KEY DRIVERS ▷ Strong performance across key metrics: ▷ Both referrals and new prescriptions increased sequentially from Q1 to Q2 ▷ Referrals up 17% YoY ▷ Highest number of bottles ever shipped ▷ 17x increase in traffic to NUPLAZID.com YoY, helping to drive more patients to speak with their physicians ▷ Strong execution and continued DTC momentum expected to drive sustained growth $168.5M in Q2 Net Product Sales Up 7% year-over-year 1 Mosholder AD, Ma Y, Akhtar S, et al. Mortality among Parkinson’s disease patients treated with pimavanserin or atypical antipsychotics: an observational study in Medicare beneficiaries. Am J Psychiatry. 2022;179(8):553-561. 2 Layton JB, Forns J, McQuay LJ, et al. Mortality in patients with Parkinson’s disease-related psychosis treated with pimavanserin compared with other atypical antipsychotics: a cohort study. Drug Safety. Published online December 14, 2022. doi:10.1007/s40264-022-01260-6. 3 Layton JB, Forns J, McQuay LJ, et al. Mortality in patients with Parkinson’s disease-related psychosis treated with pimavanserin compared with other atypical antipsychotics: a cohort study. Supplementary material. Online resource. Drug Safety. Published online December 14, 2022. doi:10.1007/s40264-022-01260-6. 9
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R&D Update 10 EXECUTIVE VICE PRESIDENT | HEAD OF RESEARCH AND DEVELOPMENT Elizabeth H.Z. Thompson
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Pipeline Highlights from June 25th R&D Day 11 PROGRAM INDICATION MOLECULE DESCRIPTION NEUROLOGICAL DISEASES NUPLAZID Parkinson's Disease Psychosis 5HT2A inverse agonist ACP-204 Alzheimer's Disease Psychosis New 5HT2A inverse agonist ACP-204 Lewy Body Dementia w/ Psychosis New 5HT2A inverse agonist ACP-711 Essential Tremor Selective GABAA-α3 modulator ACP-211 Major Depressive Disorder Deuterated R-norketamine ACP-271 Tardive Dyskinesia GPR88 agonist DISCOVERY IND ENABLING PHASE 1 PHASE 2 PHASE 3 LAUNCHED RARE DISEASES DAYBUE Rett Syndrome Analogue of GPE ACP-101 Hyperphagia in Prader-Willi Syndrome Intranasal Carbetocin ACP-2591 Rett Syndrome; Fragile X Syndrome cGP analogue ACP-271 Huntington’s Disease GPR88 agonist STOKE ASO SYNGAP1 Antisense oligonucleotide (ASO) DAYBUE (trofinetide) is only approved in the U.S. by the FDA and in Canada by Health Canada for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. NUPLAZID (pimavanserin) is only approved in the U.S. by the FDA for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis.
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Building Momentum Across our Pipeline Anticipated Milestones ▷ 3Q25: Phase 2 initiation of ACP-204 in Lewy Body Dementia Psychosis ▷ 3Q25: Phase 3 initiation of trofinetide in Japan ▷ Early 4Q25: Top-line Results COMPASS PWS Phase 3 of ACP-101 ▷ 4Q25: Phase 2 initiation of ACP-211 in Major Depressive Disorder ▷ 4Q25: Initiate first-in-human study of ACP-271 in healthy volunteers 9 Disclosed and Multiple undisclosed programs 7 Phase 2 or Phase 3 study starts expected in 2025 and 2026 5 Phase 2 or Phase 3 study readouts anticipated 2025-2027 Reflects breadth of pipeline and strength of R&D strategy
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13 Trofinetide (DAYBUE) ▷ Caregiver-reported outcomes ▷ Real-world data including findings from less-studied populations (e.g., males, older patients) ▷ Analysis of clinical trials supporting Real-world experience continues in line with clinical trials ACP-101 ▷ Burden of comorbidities and associated behaviors Reinforces Acadia's commitment to partner with Prader-Willi syndrome community IRSF United in Hope Contributing to Scientific Literature for Marketed & Pipeline Products ACP-204 ▷ Specificity for 5HT2A and supportive PK profile ▷ Supportive safety/tolerability profile; data primarily focused on 60 mg experience in Phase 1 including in healthy elderly Data support potential utility of ACP-204 and key aspects of target profile AAIC
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▷ Top-line results expected in early Q4 2025 ▷ Assuming positive data, anticipate filing NDA in Q1 2026 ▷ Potential PDUFA date in 3Q 2026 1 Causes of Death in Prader-Willi Syndrome: Prader-Willi Syndrome Association (USA) 40-Year Mortality Survey. Genet Med. 2017. June; 19(6): 635–642. 2 Hyperphagia Questionnaire for Clinical Trials (HQ-CT) is an observer-reported outcome measure that has been widely used in interventional studies to assess changes in hyperphagia behaviors individuals with PWS. Q1 Q2Q1 Q2 Q3 Q4 2025 COMPASS PWS L A S T P A T I E N T I N Phase 3 TOP-LINE RESULTS 2026 NDA Filing Prader-Willi Syndrome: ▷ Hyperphagia is a defining characteristic (unrelenting hunger) ▷ Affecting ~8,000 – 10,000 patients in the U.S. ▷ ~30 years average life expectancy1 ACP-101 (Intranasal Carbetocin) ▷ Long-acting analogue of human oxytocin ▷ COMPASS PWS is a randomized global placebo controlled Phase 3 trial ▷ Prospective parallel group 14 Q3 Q4 Potential Approval Upcoming Phase 3 Results of COMPASS PWS Trial
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Financial Update Mark Schneyer CHIEF FINANCIAL OFFICER 15
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Millions, Except EPS 2Q25 2Q24 Y oY Change TOT AL Revenue $264.6 $242.0 9% NUPLAZID $168.5 $157.4 7% DAYBUE $96.1 $84.6 14% R&D $78.0 $76.2 2% SG&A $133.5 $117.1 14% EPS $0.16 $0.20 -20% Cash and Investments Balance $762.0 Q2 2025 Financial Highlights 16
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Guidance NUPLAZID Net Sales $665 to $690 Million (Updated from prior guidance of $650 to $690 Million) NUPLAZID Gross-to-Net 22.5% to 25.5% DA YBUE Net Sales* $380 to $405 Million DA YBUE Gross-to-Net 21.5% to 24.5% T otal Revenue $1.045 to $1.095 Billion (Updated from prior guidance of $1.030 to $1.095 Billion) R&D Expense $330 to $350 Million SG&A Expense $535 to $565 Million FY 2025 Financial Guidance 17 * Represents net sales for U.S. patients
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Concluding Remarks 18 = CHIEF EXECUTIVE OFFICER Catherine Owen Adams
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2025-2026 Completed and Anticipated Milestones Corporate 2026 Q1 Q2 Q3 Q4 2025 Filed MAA with EMA P otential Approval Q1 Q2 Q3 Q4 Anticipated Approval in EMA Anticipating top-line results from pivotal Phase 3 PWS study in early Q4 2025 ACP-204 ACP-101 19 (PWS) (ADP/LBDP) Phase 3 LAST PATIENT IN Phase 3 TOP -LINE RESULTS Phase 2 in LBDP FIRST PATIENT IN Phase 2 in ADP LAST PATIENT IN Phase 2 in ADP TOP -LINE RESULTS R&D Day Annual Sales > $1B NDA Filing Named patient supply available in select countries
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Q&A Session 20