Acadia Pharmaceuticals Inc. (Acadia) operates as a biopharmaceutical company.
The company focuses on the development and commercialization of medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases.
The company has two core franchises in neuroscience and neuro-rare diseases. The company’s neuroscience franchise is anchored by the commercial product NUPLAZID (pimavanserin), which is the first and only drug approved by the U.S. Food and Drug Adminis...
Acadia Pharmaceuticals Inc. (Acadia) operates as a biopharmaceutical company.
The company focuses on the development and commercialization of medicines that address unmet medical needs in central nervous system (CNS) disorders and rare diseases.
The company has two core franchises in neuroscience and neuro-rare diseases. The company’s neuroscience franchise is anchored by the commercial product NUPLAZID (pimavanserin), which is the first and only drug approved by the U.S. Food and Drug Administration (FDA) for the treatment of hallucinations and delusions associated with PDP. The company’s neuro-rare disease franchise is anchored by the commercial product DAYBUE, which is the first and only drug approved for the treatment of Rett syndrome. In addition to these commercial products, the company has a portfolio of product candidates and research programs that are designed to address significant unmet medical needs in CNS disorders and rare diseases.
The company’s most advanced product candidate is ACP-101 (intranasal carbetocin) for the treatment of hyperphagia in Prader-Willi syndrome (PWS), a neuro rare disease. Hyperphagia is an intense persistent sensation of hunger accompanied by food preoccupations, an extreme drive to consume food, food-related behavior problems, and a lack of normal satiety. In November 2023, the company initiated the Phase 3 COMPASS PWS study evaluating the efficacy and safety of ACP-101 for the treatment of hyperphagia in PWS.
The company’s next most advanced product candidate is ACP-204 for the treatment of Alzheimer’s disease psychosis (ADP). In November 2023, the company initiated a Phase 2 study evaluating the efficacy and safety of ACP-204 for the treatment of hallucinations and delusions associated with ADP.
The company has several product candidates in earlier stages of development for the treatment of CNS disorders and rare diseases, including ACP-711 for the treatment of essential tremor, for which it expects to initiate a Phase 2 study in 2026.
Strategy
The company’s strategy is to build a strong foundation for growth with multiple innovative commercial products and product candidates that address high unmet medical needs and have the potential to be impactful products in its core franchises of neuro-psychiatric disorders and neuro-rare diseases and adjacencies within rare disease. The company plans to execute on this strategy by enhancing the growth of the commercial products in its core franchises while expanding its pipeline of product candidates through business development, partnerships, and collaborations.
The key elements of the company’s strategy are to maximize growth of its successful commercialization of NUPLAZID for Parkinson’s disease psychosis in the United States; drive new patient adoption of DAYBUE for the treatment of patients with Rett syndrome in the United States; expand trofinetide to markets outside the U.S. for the treatment of patients with Rett syndrome; advance its late-stage product candidates to drive further growth; and develop its early-stage product candidates and other business development opportunities.
In addition, the company is investing in the development of four core strategic capabilities to support and accelerate its growth over the long term. These capabilities form the foundation of its operational strategy and are designed to enhance its competitive position, drive innovation, and meet the evolving needs of patients worldwide. The four core capabilities in which the company is investing are:
Precision Medicine. The company is focused on integrating strategies that address the variability in patients’ responses to therapies by identifying and incorporating the use of targeted biomarkers at the early stages of drug development.
Data Innovation. The company plans to leverage advanced technologies, including artificial intelligence (AI) and machine learning (ML) throughout its business from discovery through commercialization. By utilizing these tools, the company intends to generate, analyze, and apply data in innovative ways to enhance the efficiency and effectiveness of its operations in ways that ultimately drive growth and improve patient outcomes.
Globalization. The company is building targeted capabilities beyond the United States, including in the European Union, Japan, and Canada in order to meet patient needs on a global scale.
Patient Empowerment. The company seeks to engage with patients, caregivers and advocacy organizations; and incorporate their feedback into its drug development and commercialization efforts.
Pipeline
NUPLAZID (pimavanserin) as a Treatment for Parkinson’s Disease Psychosis
Pimavanserin is a chemical entity that the company discovered and developed. NUPLAZID is a selective serotonin inverse agonist/antagonist preferentially targeting the 5-HT2A receptor, a key serotonin receptor that plays an important role in psychosis. Through this novel mechanism, NUPLAZID demonstrated significant efficacy in reducing the hallucinations and delusions associated with PDP without negatively impacting motor function in the company’s Phase 3 pivotal trial. NUPLAZID has the potential to avoid many of the debilitating side effects of existing antipsychotics, none of which are approved by the FDA in the treatment of PDP. The company holds worldwide commercialization rights to NUPLAZID for all indications and have established a broad patent portfolio, which includes numerous issued patents in the United States, Europe, and several additional countries. NUPLAZID is available in 34 mg capsule and 10 mg tablet dosage forms.
Other Indications Recently Evaluated with Pimavanserin
In March 2024, the company reported top-line results from a Phase 3 study of pimavanserin for the treatment of the negative symptoms of schizophrenia. Pimavanserin did not demonstrate a statistically significant improvement over placebo on the study’s primary endpoint, the change from baseline to week 26 on the Negative Symptom Assessment-16. The safety and tolerability profile of pimavanserin was consistent with previous clinical trials, showing a low rate of adverse events.
In October 2024, the company completed a Phase 2 trial to evaluate the efficacy and safety of pimavanserin for the treatment of irritability associated with autism spectrum disorder in pediatric populations. The trial did not meet either of its primary or secondary endpoints. With the completion of the trial, the company has completed the FDA’s requirements to qualify for a pediatric exclusivity for pimavanserin. If the pediatric exclusivity is granted, exclusivity of the NUPLAZID franchise would be extended by six months.
The company does not intend to further explore pimavanserin in these or any additional indications.
DAYBUE (trofinetide) as a Treatment for Rett Syndrome
Trofinetide is a novel synthetic analog of the amino-terminal tripeptide of insulin-like growth factor 1 (IGF-1) designed to treat the core symptoms of Rett syndrome by reducing neuroinflammation and supporting synaptic function. The company acquired an exclusive North American license to develop and commercialize trofinetide from Neuren Pharmaceuticals Limited (Neuren) in August 2018. In July 2023, the company expanded the 2018 licensing agreement with Neuren to acquire rights to trofinetide outside of North America, as well as global rights to Neuren’s development candidate NNZ-2591 in Rett syndrome and Fragile X syndrome. Trofinetide has been granted FDA Fast Track Status and Orphan Drug Designation in the U.S. and Orphan Designation in Europe.
In March 2023, the FDA approved DAYBUE for the treatment of Rett syndrome, making it the first and only drug approved for this condition. DAYBUE became available for prescription in the United States in April 2023. The FDA approval of DAYBUE for the treatment of Rett syndrome was based on the positive results from the company’s pivotal Phase 3 LAVENDER study which demonstrated statistically significant and clinically meaning improvement over placebo for both co-primary endpoints in the study as well as the key secondary endpoint of the study. In addition, the company was granted a Rare Pediatric Disease Priority Review Voucher (PRV) following the FDA approval of DAYBUE.
In connection with the FDA approval of DAYBUE, the company is required to conduct post-marketing work, including a nonclinical carcinogenicity studies and nonclinical in vitro studies.
In October 2024, Health Canada granted marketing authorization of DAYBUE (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older under the Priority Review process, making DAYBUE the first and only drug approved in Canada for the treatment of Rett syndrome.
In January 2025, the company announced the submission of a MAA with the EMA. Based on typical review timelines, the company expects to receive approval for this submission in the first quarter of 2026.
ACP-101 as a Treatment for Prader-Willi syndrome
Carbetocin nasal spray (ACP-101) is an investigational drug being developed for the treatment of hyperphagia in PWS. Carbetocin has improved drug qualities relative to oxytocin, including an extended half-life and greater specificity for the oxytocin receptor compared to vasopressin receptors which could provide meaningful efficacy with an attractive safety profile in patients with PWS. For the treatment of PWS specifically, a central nervous system disorder, an intranasal formulation of carbetocin was developed, which provides direct delivery of the drug to the brain, allowing for reduced systemic exposure and the potential for side effects. The company acquired Levo Therapeutics and worldwide rights to carbetocin nasal spray in June 2022. Carbetocin nasal spray has been granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations by the FDA.
In the fourth quarter of 2023, the company initiated the Phase 3 COMPASS PWS study evaluating the efficacy and safety of ACP-101 for the treatment of hyperphagia in PWS. COMPASS PWS is a 12-week, double-blind, randomized, placebo-controlled global Phase 3 trial evaluating the efficacy and safety of carbetocin nasal spray 3.2 mg three times daily (TID) in approximately 170 children and adults aged five to 30 years with PWS. The primary efficacy endpoint of the study is change from baseline to week 12 on the hyperphagia questionnaire for clinical trials (HQ-CT) score, a caregiver assessment for hyperphagia-related behaviors. Participants who complete the Phase 3 study will be eligible to enroll in a long-term, open-label extension study, COMPASS OLE, designed to investigate the safety and tolerability of long-term treatment with ACP-101.
The company expects to complete enrollment in its Phase 3 COMPASS PWS study in in the fourth quarter of 2025 and subsequently report top-line results sometime in the first half of 2026. In the event of positive trial results, ACP-101 has the potential for FDA approval in the fourth quarter of 2026.
ACP-204 as a Treatment for Alzheimer’s Disease Psychosis
The company completed Phase 1 studies of ACP-204 in over 200 patients; data to date support its target product profile as a potential treatment for ADP.
In the fourth quarter of 2023, the company initiated the Phase 2 RADIANT study of ACP-204 for the treatment of hallucinations and delusions associated with ADP. The Phase 2 RADIANT study is part of a Phase 2 / Phase 3 program that includes three studies: a single Phase 2 study and two Phase 3 studies which have almost identical design. The Phase 2 RADIANT study is a global, multi-center, randomized, double-blind, placebo-controlled trial that will enroll approximately 318 patients and evaluate ACP-204 30 mg and 60 mg doses compared to placebo. The primary endpoint is change from baseline to week 6 on the Scale for the Assessment of Positive Symptoms–Hallucinations and Delusions subscales (SAPS-H+D) total score. The clinical trial sites will enroll seamlessly from Phase 2 into Phase 3. Patients who complete the study will have the option of participating in the company’s LUMINOUS long-term open-label extension study.
The company expects to complete enrollment in the Phase 2 RADIANT study in the first quarter of 2026 and subsequently report top-line results around mid-2026.
ACP-204 as a Treatment for Lewy Body Dementia with Psychosis
In January 2025, the company announced plans to evaluate ACP-204 for the treatment of LBDP. LBDP is a progressive brain disorder that affects thinking, movement, mood and behavior. LBDP is associated with abnormal deposits of a protein called alpha-synuclein in the brain.
ACP-711 as a Treatment for Essential Tremor
In November 2024, the company entered into an exclusive worldwide license agreement with Saniona A/S (Saniona) for the development and commercialization of ACP-711 (formerly SAN711). The company intends to study ACP-711 as a potential treatment for essential tremor. ACP-711 is a highly selective GABAA-a3 positive allosteric modulator.
Early-stage Research Programs and Product Candidates
ACP-211
ACP-211 is a NMDA receptor agonist being evaluated for potential use in the treatment of Treatment-resistant depression (TRD), major depressive disorder (MDD) and other potential rare CNS indications and is in Phase 1.
ACP-2591
ACP-2591 is a cGP analogue for which the company obtained global rights to evaluate for use in Rett syndrome and Fragile X syndrome from Neuren as part of its expanded licensing agreement in July 2023. ACP-2591 is in Phase 1 development.
ACP-271
ACP-271 is a GPR88 agonist being evaluated in neurology and is in the IND-enabling stage.
Antisense Oligonucleotide (ASO) Programs
In January 2022, the company entered a license and collaboration with Stoke Therapeutics, Inc. (Stoke) to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the CNS. The collaboration includes three programs: SYNGAP1 syndrome, Rett syndrome (MECP2) and an undisclosed CNS target of mutual interest.
Intellectual Property
The company holds approximately 54 issued U.S. patents and a significant number of related issued foreign patents. The company has also exclusively licensed rights to an additional 23 issued U.S. patents, and a number of related foreign patents.
The company’s strategy is to file patent applications in the United States and any other country that represents an important potential commercial market to the company.
Pimavanserin
The company holds 26 U.S. patents that relate to pimavanserin, NUPLAZID and methods of use of pimavanserin. Ten of these are Orange Book-listed patents that relate to pimavanserin, NUPLAZID and the company’s approved indications, and covers the general formula of the compound, the composition of matter, with claims specifically directed to pimavanserin and salts thereof, the specific polymorph form of pimavanserin, the approved formulations, and the use thereof for treating its approved indication. The composition of matter patent covering pimavanserin and salts thereof has an expiration date in 2030, including a patent term extension approved by the U.S. Patent and Trademark Office. The patents covering the polymorph form and the use of pimavanserin or NUPLAZID for the company’s approved indication are set to expire between 2024 and 2028. These patent terms include adjustments made by the U.S. Patent and Trademark Office, but not extensions.
The remaining 16 U.S. patents relating to pimavanserin that has been issued to the company cover methods of use of pimavanserin, salts and methods of manufacturing pimavanserin. The company also has foreign patents that cover pimavanserin and polymorphs in Europe and Asia, as well as in Australia, Canada, Mexico, and other countries.
The company continues to prosecute patent applications directed to pimavanserin, formulations of pimavanserin, methods of manufacturing, and to methods of treating various diseases using pimavanserin, either alone or in combination with other agents, worldwide. For example, in late 2019 and in 2020, the company obtained and listed in the Orange Book six additional U.S. issued patents, two patents directed to method of use for its 10 mg tablet, expiring in 2037, and four patents directed to its 34 mg capsule formulation, each expiring in 2038.
Trofinetide
The company holds the exclusive licenses to 8 U.S. patents from Neuren that relate to trofinetide, methods of manufacturing and methods of use of trofinetide. The company also holds a U.S. patent to crystalline trofinetide. Three of the U.S. patents are listed in the Orange Book, including a patent claiming the use of trofinetide for treating Rett syndrome. The use patent for treating Rett syndrome has an expiration date in 2032. Subject to a patent term extension request, the expiration date of such patent may be extended to January 2036. The company also holds the exclusive licenses to issued foreign patents that relate to the use of trofinetide in Europe and Asia, as well as in Australia, Canada, Mexico and other countries.
Under the license agreement with Neuren, the company continues to file and prosecute patent applications directed to trofinetide, formulations of trofinetide, methods of manufacturing and methods of treating Rett syndrome using trofinetide.
ACP-101
The company holds one U.S. patent for methods of use of ACP-101 in PWS. The company holds licenses from Ferring International Center SA to one U.S. patent to ACP-101 for use in PWS. Those patents expire in 2035 and 2039, respectively. These patent terms include adjustments made by the U.S. Patent and Trademark Office, but not extensions. The company holds and license a significant number of foreign patents relating to the use of ACP-101 in PWS, including in Europe and Japan.
The company continues to file and prosecute patent applications directed to ACP-101 worldwide.
ACP-204
The company holds two U.S. patents that relate to ACP-204 and methods of use of ACP-204. The patents cover the general formula of the compound, the composition of matter (with claims specifically directed to ACP-204 and salts thereof), and the use thereof for treating certain indications, including ADP and LBDP. The composition of matter patent covering ACP-204 and salts thereof has an expiration date in 2038. These patent terms include adjustments made by the U.S. Patent and Trademark Office, but not extensions. The company also hold issued foreign patents in Australia, China and Japan.
The company continue to file and prosecute patent applications directed to ACP-204 worldwide.
ACP-711
The company holds the exclusive licenses to one U.S. patent that relates to ACP-711 from Saniona A/S, with claims specifically directed to ACP-711 and salts thereof. The composition of matter patent covering ACP-711 and salts thereof has an expiration date in 2039. This parent term includes adjustments made by the U.S. Patent and Trademark Office, but not extensions. The company also hold the exclusive licenses to issued foreign patents in Europe, parts of Asia and Mexico, as well as other countries.
The company continues to file and prosecute patent applications directed to ACP-711 worldwide.
Government Regulations
Before commencing clinical investigations in humans, the company or its collaborators must submit an Investigational New Drug Application (IND), to the FDA. Additionally, the manufacture of the company’s drug product must be done in accordance with good manufacturing practices (cGMPs).
The company and its collaborators and contract manufacturers also are required to comply with the applicable FDA GMP regulations.
If a product is approved, the company must also comply with post-marketing requirements, including, but not limited to, compliance with advertising and promotion laws enforced by various government agencies, including the FDA’s Office of Prescription Drug Promotion, and through such laws as federal and state anti-fraud and abuse laws, including anti-kickback and false claims laws, healthcare information privacy and security laws, post-marketing safety surveillance, and disclosure of payments or other transfers of value to healthcare professionals and entities. In addition, the company is subject to other federal and state regulation including, for example, the implementation of corporate compliance programs.
Manufacturing and Distribution
The company licensed worldwide intellectual property rights related to pimavanserin in certain indications to Acadia Pharmaceuticals GmbH, its wholly owned Swiss subsidiary (Acadia GmbH). The company’s active pharmaceutical ingredient (API) has been manufactured in Switzerland for over 10 years. Acadia GmbH manages the worldwide supply chain of the company’s pimavanserin API and maintains sufficient inventory in its Switzerland contract warehouse. Under the manufacturing agreement, Acadia GmbH has agreed to purchase specified percentages of the company’s commercial requirements of the pimavanserin API at a predefined price.
The company has contracted with Patheon Pharmaceuticals Inc. (Patheon), a subsidiary of Thermo Fisher Scientific Inc., to manufacture NUPLAZID 10 mg tablet and 34 mg capsule drug product for commercial use in the United States. The company has also contracted with a second contract manufacturing organization to manufacture NUPLAZID 34 mg drug product for commercial use in the United States. Under the manufacturing agreement with Patheon, the company has agreed to purchase from Patheon a specified percentage of its commercial requirements of NUPLAZID for the United States.
The company sells NUPLAZID to a limited number of specialty pharmacies (SPs), and specialty distributors (SDs), which it collectively refers to as the company’s customers. SPs subsequently dispense NUPLAZID to patients based on the fulfillment of a prescription and SDs subsequently sell NUPLAZID to government facilities, long-term care pharmacies, and in-patient hospital pharmacies. Four of such customers, each based in the United States, accounted for approximately 73% of the company’s NUPLAZID product revenue and 48% of its total product revenue for the year ended December 31, 2024. The company has retained third-party logistics service providers to perform a variety of functions related to the distribution of NUPLAZID, including warehousing, customer service, order-taking, invoicing, collections, and shipment and returns processing.
The company has contracted with manufacturers to produce supplies of trofinetide to support the development program and for commercial sale. The company has contracted with Corden Pharma Bergamo S.p.A. (Corden), to manufacture the API for trofinetide products. Under the manufacturing agreement with Corden, it has agreed to purchase from Corden the API for trofinetide products at specified price per volume and a specified percentage of the company’s commercial requirements of trofinetide API for the United States and Canada market.
The company also has contracted with F.I.S. Fabbrica Italiana Sintetici S.p.A. (FIS) to manufacture the API for trofinetide products. Under the manufacturing agreement, it has the right to purchase from FIS the API for the trofinetide products at a specified price per volume.
Under the manufacturing agreement with Patheon described above, the company also has the right to purchase trofinetide products for commercial use. In addition, the company has contracted with CoreRx Inc. (CoreRx) to manufacture trofinetide products for commercial use.
The company sells DAYBUE to a single wholesale distributor with specialty pharmacy service, which performs a variety of functions related to the distribution of DAYBUE, including warehousing, customer service, order-taking, shipment and returns processing.
Sales and Marketing
The company has U.S. sales specialists that are focused on promoting NUPLAZID to physicians who treat PDP patients, including neurologists, psychiatrists and long-term care physicians. This sales force is supported by an experienced sales leadership team. The company has experienced commercial team consists of experienced professionals in marketing, key account management, patient access services, commercial operations, and sales force planning and management. In addition, the company’s commercial infrastructure includes capabilities in manufacturing, health outcomes, medical affairs, quality control, and compliance.
The company launched NUPLAZID in May 2016, and its focus is to continue to establish NUPLAZID as the standard of care for patients with PDP.
In addition, the company has U.S. sales specialists that are focused on promoting DAYBUE to physicians who treat Rett syndrome patients, including those at Centers of Excellence, high volume institutions and in the community setting. The sales force is supported by an experienced sales leadership team. The company experienced commercial team consists of rare disease field-based specialists, patient access services, commercial operations, and sales force planning and management. In addition, the company’s commercial infrastructure includes capabilities in manufacturing, health outcomes, medical affairs, quality control, and compliance.
The company launched DAYBUE in April 2023, and its focus is to continue to establish DAYBUE as the standard of care for patients with Rett syndrome. The company also has support services including the Acadia Connect hub for physicians, patients and their families that provide broad resources to help with access, reimbursement and the continual clinical support to help patients start and stay on therapy.
Research and Development Expenses
The company's research and development expenses were $303.2 million in 2024.
History
The company was founded in 1993. It was incorporated in Vermont in 1993 and was reincorporated in Delaware in 1997. The company was formerly known as Receptor Technologies, Inc. and changed its name ACADIA Pharmaceuticals Inc. in 1997.
