ACADIA PHARMACEUTICALS 2014 ANNUAL REPORT > ABOUT US We are a biopharmaceutical company focused on the development and commercialization of innovative medicines to address unmet medical needs in neurological and related central nervous system disorders. We have a pipeline of product candidates led by NUPLAZID™ (pimavanserin), which has successfully completed Phase III development and has the potential to be the first drug approved in the United States for Parkinson’s disease psychosis. We also are developing pimavanserin in additional areas with severe unmet medical needs, including Alzheimer’s disease psychosis and schizophrenia. Q&A STEPHEN R. DAVIS INTERIM CHIEF EXECUTIVE OFFICER What is the biggest opportunity What is ACADIA’s strategic vision? for ACADIA Pharmaceuticals? At ACADIA, our strategy remains Our pipeline of product candidates is led by unchanged. Our vision is to become a NUPLAZID™ (pimavanserin), which offers a leading biopharmaceutical company new and distinctly different pharma dedicated to developing and cological approach to treating psychosis. commercializing innovative therapies for We believe this nextgeneration approach neurological and related central nervous holds the potential to dramatically change system (CNS) disorders. We are excited the way we think about treating psychosis about the opportunity to build a U.S. — offering relief without many of the specialty CNS franchise using pimavanserin compromises inherent in existing as the foundation. We see a broad therapies. We have completed our Phase III opportunity for pimavanserin to expand program to develop NUPLAZID for into a range of neurological and psychiatric Parkinson’s disease psychosis (PDP) and indications and the potential to transform expect to submit our New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) in the second half of 2015. NUPLAZID has the potential to be the first drug approved in the United States for the treatment of psychosis associated with Parkinson’s disease. We also are developing pimavanserin for Alzheimer’s disease psychosis and schizophrenia — other areas with severe unmet medical needs. In addition, we have collaborative clinicalstage programs for chronic pain and glaucoma. the treatment paradigm in large For ACADIA, what are the most underserved disease states. important priorities going forward? Our highest priority is the planned What progress did ACADIA submission of our NUPLAZID NDA. To that make in 2014? end, we are focused on preparing the Last year we made important progress in organization for the submission and review advancing NUPLAZID toward registration. of the NDA and the planned U.S. launch of We successfully completed our drugdrug NUPLAZID. In addition, we continue interaction program and our stability expanding our medical and development program for registration batches and held capabilities to further leverage successful preNDA meetings with the FDA. pimavanserin and its lifecycle opportunities. We also continued to make significant strides in our commercial preparations for What are the next steps in the NDA the planned launch of NUPLAZID in the submission of NUPLAZID for PDP? United States. Moving from a clinicalstage company to a In addition, the FDA designated commercialstage company requires NUPLAZID as a Breakthrough Therapy, which building infrastructure to accommodate reinforces the serious unmet medical need commercialscale operations. As we in PDP and the importance of our NUPLAZID announced in March 2015, additional time program. We are pleased NUPLAZID met the was needed to complete the preparation of criteria for the Breakthrough Therapy manufacturing quality systems to support designation, which the FDA created to commercial manufacturing and supply. We expedite the development and review of are focused on ensuring that we have drugs that are intended to treat serious or established, tested and evaluated the lifethreatening conditions. appropriate manufacturing and supply BY THE NUMBERS PARKINSON’S DISEASE PSYCHOSIS (PDP) 40% PDP afflicts about 40% of the 1 million Parkinson’s patients in the United States. 70% 90% Over 70% suffer comorbid sleep disturbances. Almost 90% have caregiver support with 74% requiring daily care. ALZHEIMER’S DISEASE PSYCHOSIS (ADP) 25-50% ADP afflicts about 25% to 50% of the 5.2 million Alzheimer’s disease patients in the United States. Zero Currently there is no drug approved by the FDA for ADP. SCHIZOPHRENIA 3 Million There are approximately 3 million people in the United States suffering from schizophrenia. 74% In a landmark government study, about 74% of patients with schizophrenia discontinued their medications because of lack of efficacy or intolerable side effects. systems to prepare for FDA review and NUPLAZID, if approved. Our teams are commercial launch. energized by the potential of NUPLAZID to make a meaningful impact on the lives of How are you preparing for patients with PDP and their caregivers. commercialization? Over the last year, our commercial team has How do you plan to market focused on thoroughly understanding the NUPLAZID for PDP? PDP market landscape and preparing the ACADIA owns the worldwide product, the market, and the organization commercialization rights to NUPLAZID. for commercial launch. We conducted In the United States, our senior commercial extensive market research with team has been building the commercial neurologists, psychiatrists and longterm organization and advancing our commercial care clinicians as well as PDP patients and readiness per plan. In connection with this their caregivers. We conducted scientific plan we expect to hire approximately 135 advisory boards with leading movement sales representatives at or around the time disorder specialists and PDPtreating of approval. Our specialty sales force will physicians in order to gain insight regarding be dedicated to covering the estimated how to position NUPLAZID to best address 11,000 PDPtreating physicians, with the the needs of patients, caregivers and largest segment being neurologists. physicians. We completed foundational In parallel with the advancement of access and reimbursement research with our PDP program in the United States, we key decisionmakers for payors covering also have outlined the path to registration 168 million lives. We also completed for NUPLAZID in Europe. During 2015, we important salesforce sizing assessments will be working on our Marketing and preparation of commercial Authorization Application (MAA) for infrastructure and systems for launch NUPLAZID for submission to the European remains on track. Medicines Agency (EMA) approximately six In January 2015 we launched a PDP to nine months following the submission disease awareness campaign in the of our NDA. physician community and are conducting an extensive speaker training program to What is the impact of PDP? educate healthcare professionals on PDP. Parkinson’s disease affects about one We also continue to build out a medical million people in the United States and affairs organization of highly experienced between four to six million people globally professionals to support the introduction of and is the second most common PARKINSON’S DISEASE PSYCHOSIS (PDP) LARGE UNMET NEED “Among Parkinson’s patients, psychosis causes great distress for patients and caregivers and is the leading cause of institutionalization. Neurologists have limited options to treat this serious disorder, and off-label use of current antipsychotics is linked to increased risk of death and serious adverse events, as well as loss of motor control.” - Jeffrey Cummings, M.D., Sc.D. Director of Cleveland Clinic , Lou Ruvo Center for Brain Health “When we recently examined the topics that were most searched for on our National Parkinson Foundation’s website, we were not surprised to discover that treatment of psychosis, comprising hallucinations and delusions, topped the list. There is a critical unmet need for development of better drugs to address psychosis in the setting of Parkinson’s disease.” - Michael S. Okun, M.D. National Medical Director, National Parkinson Foundation neurological disorder after Alzheimer’s The condition is often treated off label with disease. Research indicates that at any atypical antipsychotics, which can worsen point in time approximately 40 percent of the symptoms of Parkinson’s disease and patients with Parkinson’s disease also carry a black box warning for use in elderly suffer from PDP and that over a lifetime, a patients with dementiarelated psychosis majority of patients with Parkinson’s due to increased risk of mortality and disease eventually develop PDP. PDP is a morbidity. Physicians treating patients debilitating disorder characterized by with PDP face a “dopamine dilemma,” hallucinations and delusions that worsens because drugs used to treat the psychosis over time and severely impacts daily living. can exacerbate the motor symptoms Research has shown that delusions often associated with Parkinson’s disease. involve suspicions of spousal infidelity or At ACADIA, we are developing other paranoid themes and are often NUPLAZID as a potential firstinclass disturbing and debilitating to patients. PDP treatment for PDP. Pimavanserin — a small substantially contributes to the burden of molecule that was discovered in our Parkinson’s disease and deeply affects a laboratories — has a unique mechanism of patient’s quality of life. PDP is also action as a selective serotonin inverse associated with increased caregiver distress and burden, nursing home placement, and agonist preferentially targeting 5HT2A receptors. Outcomes from NUPLAZID’s increased mortality and morbidity. successful Phase III clinical trial, referred to as the 020 Study, indicate that NUPLAZID What is the current treatment for PDP? has the potential to provide a safe and Currently, no drug is approved to treat PDP effective treatment of PDP without in the United States and the problem is compromising motor control or causing expected to grow as our population ages. daytime sedation. OUR PIPELINE COMPOUND/ PROGRAM INDICATION IND-TRACK PHASE I PHASE II PHASE III REGULATORY APPROVAL COMMERCIALIZATION RIGHTS Parkinson’s Disease Psychosis NUPLAZID™ (pimavanserin) Alzheimer’s Disease Psychosis Schizophrenia Adrenergic Chronic Pain Muscarinic Glaucoma ACADIA Allergan Allergan What is the opportunity to develop pimavanserin for According to the Alzheimer’s Association, an estimated 5.2 million Alzheimer’s disease psychosis? people in the United States have We believe pimavanserin has the potential Alzheimer’s disease. Studies have to establish a new paradigm in the way suggested that approximately 25 percent psychosis is treated and we plan to to 50 percent of Alzheimer’s patients may explore pimavanserin in multiple develop psychosis, commonly consisting indications. Currently, in addition to our of hallucinations and delusions. The plans to seek approval of pimavanserin in psychosis in Alzheimer’s patients is PDP, we are conducting a Phase II study of associated with even more rapid cognitive pimavanserin in Alzheimer’s disease and functional decline and increased psychosis (ADP) and expect to complete institutionalization relative to Alzheimer’s enrollment by the end of 2015. patients without psychosis. Like PDP, no drug is currently approved in the United States for the treatment of What other indications are currently ADP. Atypical antipsychotics are frequently planned for the development of used off label to treat ADP despite the black pimavanserin? box warnings of increased risk of We are planning a Phase II clinical study mortality and morbidity in this patient with pimavanserin as a monotherapy population. In addition, research treatment for the maintenance phase of indicates that the use of atypical schizophrenia — that is, the period of time antipsychotics in ADP patients worsens between acute psychotic episodes. We their cognitive deficits in a manner believe the pimavanserin profile may allow equivalent to approximately one year’s for an improved schizophrenia therapy disease progression. resulting in better compliance and safety compared to existing antipsychotic drugs. our public offering of common stock and Schizophrenia is a debilitating lifelong closing the year with $322.5 million in cash disease afflicting approximately 1 percent of and cash equivalents. We believe our cash the population globally, and current runway positions us to continue making the therapies are suboptimal. kinds of investments that we believe will We are also planning to initiate a leverage the full potential of pimavanserin. Phase II study to further explore the potential sleep benefits of pimavanserin in How can ACADIA help improve the lives Parkinson’s disease patients. In clinical of patients and caregivers? studies we observed nonsedating sleep At ACADIA, we are developing innovative related benefits of pimavanserin, including therapies that can improve the lives of a significant improvement in both nighttime patients and the family members who care sleep and daytime wakefulness compared for them every day. This passion is what to placebo. Sleep disorders are a major and drives everything we do at ACADIA. I want to frequent problem for patients with acknowledge our team for their hard work, neurological disorders, and studies commitment and expertise. In the last year, suggest that nighttime sleep disturbances we have brought in highly qualified occur in around 70 percent of Parkinson’s individuals with extensive experience in disease patients. their functional domain and in CNS products. I look forward to updating you on What is ACADIA’s financial position? our progress as we build ACADIA into a We remain in a strong financial position to leading biopharmaceutical company with launch NUPLAZID for PDP and also to significant promise in the CNS area. explore other areas where pimavanserin may have a profound impact in the treatment of other disorders. We strengthened our balance sheet in 2014, raising $197 million in net proceeds from Stephen R. Davis Interim Chief Executive Officer April 2015 Uli Hacksell, Ph.D. After 16 years of service, Uli Hacksell, Ph.D. retired in March 2015. Under Uli’s leadership as CEO, ACADIA grew from a small startup to a fast-growing biopharmaceutical company with innovative drug candidates. We thank him for his contributions to the Company. His dedication, tenacity, deep knowledge of the CNS space, and life-long passion to deliver new drugs that can improve the lives of patients with CNS disorders have benefited ACADIA greatly. We wish Uli all the best in his retirement. EXECUTIVE OFFICERS Stephen R. Davis Interim Chief Executive Officer Roger G. Mills, M.D. Executive Vice President, Development and Chief Medical Officer CORPORATE HEADQUARTERS 3611 Valley Centre Drive, Suite 300 San Diego, CA 92130 Telephone: (858) 558-2871 Fax: (858) 558-2872 www.acadia-pharm.com COMMON STOCK LISTING Ticker Symbol: ACAD, The NASDAQ Global Select Market ANNUAL STOCKHOLDERS’ MEETING ACADIA Pharmaceuticals’ Annual Stock- holders’ Meeting will be held on Monday, June 15, 2015, at the offices of Cooley LLP, 4401 Eastgate Mall, San Diego, CA 92121. STOCK TRANSFER AGENT AND REGISTRAR Computershare Trust Company, N.A. 250 Royall St. Canton, MA 02021 Telephone: (800) 851-3061 www.computershare.com/us INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM Ernst & Young LLP STOCKHOLDERS’ INQUIRIES Stockholders may obtain copies of our news releases, Securities and Exchange Commission filings, including Forms 10-K, 10-Q, and 8-K, and other company information by accessing our website at www.acadia-pharm.com. Stockholders may also contact Investor Relations at (858) 558-2871. Terrence O. Moore Executive Vice President and Chief Commercial Officer Glenn F. Baity Executive Vice President, General Counsel and Secretary BOARD OF DIRECTORS Leslie L. Iversen, Ph.D. Chairman of the Board Professor of Pharmacology University of Oxford, England Stephen R. Biggar, M.D., Ph.D. Partner Baker Brothers Investments Michael T. Borer Former Chief Executive Officer and President, Xcel Pharmaceuticals, Inc. Laura A. Brege Chief Executive Officer and President Nodality, Inc. Mary Ann Gray, Ph.D. President Gray Strategic Advisors, LLC Lester J. Kaplan, Ph.D. Former Executive Vice President and President, Research and Development, Allergan, Inc. Torsten Rasmussen Chief Executive Officer and President Morgan Management ApS Daniel B. Soland Former Senior Vice President and Chief Operating Officer, ViroPharma William (Bill) M. Wells Founder and Chairman, Evizone Limited Former Chief Executive Officer Biovail Corporation FORWARD-LOOKING STATEMENTS Statements in this report that are not strictly historical in nature are forward-look- ing statements. These statements include but are not limited to statements related to the progress and timing of our drug development programs and related trials and regulatory filings, the utility, safety, efficacy and benefits of our product candidates, the future development and commercialization of pimavanserin in a variety of indica- tions, potential approval of NUPLAZID™ (pimavanserin), opportunities and potential for pimavanserin, and future growth for ACADIA or its stockholders. These statements are only predictions representing ACADIA’s expectations and beliefs as of the date this report was prepared based on then-current information. Actual events or results may differ materially from those projected in any of such statements due to various factors, including the risks and uncertainties inherent in drug discovery, development and com- mercialization, risks associated with regulatory review and approval, and the risk that past results of clinical trials may not be indicative of future trial results. For a discussion of these and other factors, please refer to ACADIA’s Annual Report on Form 10-K for the year ended December 31, 2014, as well as other subsequent filings with the Securities and Exchange Commission. You are cautioned not to place undue reliance on these forward-looking statements. This caution is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All forward-looking statements are qualified in their entirety by this cautionary statement and ACADIA undertakes no obligation to revise or update this report to reflect future events or circumstances after the date hereof, except as required by law. ACADIA Pharmaceuticals Inc. 3611 Valley Centre Drive, Suite 300 San Diego, CA 92130 www.acadia-pharm.com