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Product Pipeline

We are driven to develop innovative and novel medicines to help address unmet medical needs and to elevate life through science.

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Our pipeline information may include a reference to an investigational agent or an investigational use of the FDA approved product. Safety and efficacy for investigational uses or agents have not been established or approved by the FDA. This table provides an overview of selected projects in development and can be filtered by both therapeutic area and development phase.

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NUPLAZID® (pimavanserin)
Parkinson’s Disease Psychosis
In Market
2016
FDA Approval

NUPLAZID ® (pimavanserin) was approved by the FDA in 2016 for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis. Click for Important Safety Information, including Boxed WARNING.

NUPLAZID® (pimavanserin)

Parkinson’s Disease Psychosis
In Market
2016
FDA Approval

NUPLAZID® (pimavanserin) is the first and only prescription medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis (PDP).

In 2014, The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to NUPLAZID (pimavanserin) for the treatment of Parkinson’s disease psychosis. Read more.

In September 2018, the FDA completed a postmarketing review and issued a public statement reaffirming the positive benefit-risk profile of NUPLAZID (pimavanserin) for patients with Parkinson’s disease psychosis. Read more. 

In September 2023, the FDA made two changes to the NUPLAZID label that clarify its use in patients with Parkinson’s disease-related hallucinations and delusions, with or without dementia. This update is consistent with the current indication. There are no other findings or changes included as part of the label change. Read more.

Important Safety Information

WARNING: INCREASED MORTALITY IN ELDERLY PATIENTS WITH DEMENTIA-RELATED PSYCHOSIS

  • Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death.
  • NUPLAZID is not approved for the treatment of patients with dementia who experience psychosis unless their hallucinations and delusions are related to Parkinson’s disease.
  • Contraindication: NUPLAZID is contraindicated in patients with a history of a hypersensitivity reaction to pimavanserin or any of its components. Rash, urticaria, and reactions consistent with angioedema (e.g., tongue swelling, circumoral edema, throat tightness, and dyspnea) have been reported.
  • Warnings and Precautions: QT Interval Prolongation
    • NUPLAZID prolongs the QT interval. The use of NUPLAZID should be avoided in patients with known QT prolongation or in combination with other drugs known to prolong QT interval (e.g., Class 1A antiarrhythmics, Class 3 antiarrhythmics, certain antipsychotics or antibiotics).
    • NUPLAZID should also be avoided in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes and/or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and presence of congenital prolongation of the QT interval.
  • Adverse Reactions: The adverse reactions (≥2% for NUPLAZID and greater than placebo) were peripheral edema (7% vs 2%), nausea (7% vs 4%), confusional state (6% vs 3%), hallucination (5% vs 3%), constipation (4% vs 3%), and gait disturbance (2% vs <1%).
  • Drug Interactions:
    • Coadministration with strong CYP3A4 inhibitors increases NUPLAZID exposure. Reduce NUPLAZID dose to 10 mg taken orally as one tablet once daily.
    • Coadministration with strong or moderate CYP3A4 inducers reduces NUPLAZID exposure. Avoid concomitant use of strong or moderate CYP3A4 inducers with NUPLAZID.

Please read the full Prescribing Information, including Boxed WARNING

DAYBUE™ (trofinetide)
Rett Syndrome
In Market
2023
FDA Approval

DAYBUE™ (trofinetide) was approved by the U.S. Food and Drug Administration (FDA) in 2023 for the treatment of Rett syndrome in adults and pediatric patients two years of age and older. Click for Important Safety Information.

DAYBUE™ (trofinetide)

Rett Syndrome
In Market
2023
FDA Approval

DAYBUE™ (trofinetide) is the first and only treatment approved by the U.S. Food and Drug Administration (FDA) specifically indicated for Rett syndrome in adults and pediatric patients two years of age and older. Read more.

Acadia holds an exclusive Global License Agreement with Neuren Pharmaceuticals for the development and commercialization of trofinetide for Rett syndrome and other indications. Read more. 

Important Safety Information

  • Warnings and Precautions
    • Diarrhea: In a 12-week study and in long-term studies, 85% of patients treated with DAYBUE experienced diarrhea. In those treated with DAYBUE, 49% either had persistent diarrhea or recurrence after resolution despite dose interruptions, reductions, or concomitant antidiarrheal therapy. Diarrhea severity was of mild or moderate severity in 96% of cases. In the 12-week study, antidiarrheal medication was used in 51% of patients treated with DAYBUE.
      Patients should stop taking laxatives before starting DAYBUE. If diarrhea occurs, patients should notify their healthcare provider, consider starting antidiarrheal treatment, and monitor hydration status and increase oral fluids, if needed. Interrupt, reduce dose, or discontinue DAYBUE if severe diarrhea occurs or if dehydration is suspected.
    • Weight Loss: In the 12-week study, 12% of patients treated with DAYBUE experienced weight loss of greater than 7% from baseline, compared to 4% of patients who received placebo. In long-term studies, 2.2% of patients discontinued treatment with DAYBUE due to weight loss. Monitor weight and interrupt, reduce dose, or discontinue DAYBUE if significant weight loss occurs.
  • Adverse Reactions: The common adverse reactions (≥5% for DAYBUE-treated patients and at least 2% greater than in placebo) reported in the 12-week study were diarrhea (82% vs 20%), vomiting (29% vs 12%), fever (9% vs 4%), seizure (9% vs 6%), anxiety (8% vs 1%), decreased appetite (8% vs 2%), fatigue (8% vs 2%), and nasopharyngitis (5% vs 1%).
  • Drug Interactions: Effect of DAYBUE on other Drugs
    • DAYBUE is a weak CYP3A4 inhibitor; therefore, plasma concentrations of CYP3A4 substrates may be increased if given concomitantly with DAYBUE. Closely monitor when DAYBUE is used in combination with orally administered CYP3A4 sensitive substrates for which a small change in substrate plasma concentration may lead to serious toxicities.
    • Plasma concentrations of OATP1B1 and OATP1B3 substrates may be increased if given concomitantly with DAYBUE. Avoid the concomitant use of DAYBUE with OATP1B1 and OATP1B3 substrates for which a small change in substrate plasma concentration may lead to serious toxicities.
  • Use in Specific Population: Renal Impairment
    • DAYBUE is not recommended for patients with moderate or severe renal impairment.


DAYBUE is available as an oral solution (200mg/mL).

Please read the accompanying full Prescribing Information, also available at DAYBUEHCP.com

Pimavanserin
Negative Symptoms of Schizophrenia
Phase 3

Results from the Phase 2 ADVANCE study were positive, and a Phase 3 study (ADVANCE 2) is currently being conducted with top-line results anticipated in 2024.

Pimavanserin

Negative Symptoms of Schizophrenia
Phase 3

Acadia announced positive top-line results from its Phase 2 ADVANCE study, a 26-week, randomized, double-blind, placebo-controlled study in 403 patients. ADVANCE evaluated the efficacy and safety of adjunctive pimavanserin treatment in patients with predominantly negative symptoms of schizophrenia who have achieved adequate control of positive symptoms with their existing antipsychotic treatment. Read more.

The Phase 3 ADVANCE-2 study, is expected to complete enrollment by the end of 2022 with results available in 2024. This Phase 3 study will evaluate pimavanserin 34 mg in patients with predominant negative symptoms of schizophrenia. No drug is approved by the FDA for the treatment of the negative symptoms of schizophrenia.

Important Note

Investigational use of NUPLAZID® (pimavanserin) for the treatment of negative symptoms of schizophrenia has not been approved by the FDA. Please see below FDA approved indication for NUPLAZID, including Important Safety Information & Boxed WARNING.

Important Safety Information

WARNING: INCREASED MORTALITY IN ELDERLY PATIENTS WITH DEMENTIA-RELATED PSYCHOSIS

  • Elderly patients with dementia-related psychosis treated with antipsychotic drugs are at an increased risk of death.
  • NUPLAZID is not approved for the treatment of patients with dementia who experience psychosis unless their hallucinations and delusions are related to Parkinson’s disease.
  • Contraindication: NUPLAZID is contraindicated in patients with a history of a hypersensitivity reaction to pimavanserin or any of its components. Rash, urticaria, and reactions consistent with angioedema (e.g., tongue swelling, circumoral edema, throat tightness, and dyspnea) have been reported.
  • Warnings and Precautions: QT Interval Prolongation
    • NUPLAZID prolongs the QT interval. The use of NUPLAZID should be avoided in patients with known QT prolongation or in combination with other drugs known to prolong QT interval (e.g., Class 1A antiarrhythmics, Class 3 antiarrhythmics, certain antipsychotics or antibiotics).
    • NUPLAZID should also be avoided in patients with a history of cardiac arrhythmias, as well as other circumstances that may increase the risk of the occurrence of torsade de pointes and/or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and presence of congenital prolongation of the QT interval.
  • Adverse Reactions: The adverse reactions (≥2% for NUPLAZID and greater than placebo) were peripheral edema (7% vs 2%), nausea (7% vs 4%), confusional state (6% vs 3%), hallucination (5% vs 3%), constipation (4% vs 3%), and gait disturbance (2% vs <1%).
  • Drug Interactions:
    • Coadministration with strong CYP3A4 inhibitors increases NUPLAZID exposure. Reduce NUPLAZID dose to 10 mg taken orally as one tablet once daily.
    • Coadministration with strong or moderate CYP3A4 inducers reduces NUPLAZID exposure. Avoid concomitant use of strong or moderate CYP3A4 inducers with NUPLAZID.

Please read the full Prescribing Information, including Boxed WARNING

ACP-101
Hyperphagia in Prader-Willi Syndrome
Phase 3

Acadia recently completed a meeting with the FDA and plans to initiate a Phase 3 study in the fourth quarter of 2023.

ACP-101

Hyperphagia in Prader-Willi Syndrome
Phase 3

In June 2022, Acadia acquired Levo Therapeutics and worldwide rights to ACP-101 for the treatment of hyperphagia in Prader-Willi syndrome (PWS). The company plans to initiate a Phase 3 study in the fourth quarter of 2023. Read more.

ACP-204
Alzheimer’s Disease Psychosis
Phase 2

Phase 1 evaluation enabling future studies in patient populations is complete. Acadia has met with FDA to discuss the clinical development plan.

ACP-204

Alzheimer’s Disease Psychosis
Phase 2

The Phase 1 data supports ACP-204’s target product profile as a potential treatment for Alzheimer’s disease psychosis. Acadia identified the doses for planned evaluation in Phase 2. Acadia has met with FDA to discuss the clinical development plan.

Antisense Oligonucleotide (ASO) Programs
SYNGAP1; Rett Syndrome; Undisclosed
Preclinical

Acadia entered into a collaboration with Stoke Therapeutics to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases. 

Antisense Oligonucleotide (ASO) Programs

SYNGAP1; Rett Syndrome; Undisclosed
Preclinical

Acadia and Stoke Therapeutics have a collaboration to discover, develop and commercialize novel RNA-based medicines for the potential treatment of severe and rare genetic neurodevelopmental diseases of the central nervous system (CNS). The collaboration includes SYNGAP1, MECP2 (Rett syndrome) and an undisclosed CNS target of mutual interest. Read more.

Other Programs
Neuropsychiatric Symptoms
Preclinical

Other Programs

Neuropsychiatric Symptoms
Preclinical