Publications

Discover some of the most recent publications from the latest advances in neuroscience at Cerevel.

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Disclaimer: Our scientific content is evidence-based, scientifically balanced, and non-promotional. It undergoes rigorous internal medical review and is updated regularly to reflect new information.


Publications

60 Results

August, 2023

Treatment Patterns and Healthcare Resource Use in Medicare Beneficiaries with Parkinson’s Disease

Song, E JY, Guo, et al. Treatment Patterns and Healthcare Resource Use in Medicare Beneficiaries with Parkinson’s Disease. Clinicoecon Outcomes Res. 2023;15:631-643.

Objective: To assess treatment patterns, healthcare resource utilization (HRU), and costs associated with PD symptoms and treatment-related adverse events (AEs) among Medicare beneficiaries in the United States.

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N/A Parkinson’s disease

July, 2023

Medication Experiences of People with Parkinson’s Disease

Mantri, Richards, Arcona, Sasané. Medication Experiences of People with Parkinson’s Disease. Poster presented at: WPC 2023; July 4-7, 2023; Barcelona, Spain

Objective: To better understand experiences with medications from the perspective of people with PD.

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N/A Parkinson’s disease

July, 2023

Dopamine agonists in Parkinson’s disease: Impact of D1-like or D2-like dopamine receptor subtype selectivity and avenues for future treatment

Isaacson, Hauser, Pahwa, et al. Dopamine agonists in Parkinson's disease: Impact of D1-like or D2-like dopamine receptor subtype selectivity and avenues for future treatment. Clin Park Relat Disord. 2023;9:100212.

Background: Dopamine agonists (DAs) have demonstrated efficacy for the treatment of Parkinson’s disease (PD) but are limited by adverse effects (AEs). DAs can vary considerably in their receptor subtype selectivity and affinity, chemical composition, receptor occupancy, and intrinsic activity on the receptor.

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Tavapadon Parkinson’s disease

June, 2023

Evaluation of Darigabat QTc Prolongation in Healthy Volunteers: a Concentration-QTcF Analysis

Otto, Pour, van Gerven, et al. Evaluation of Darigabat QTc Prolongation in Healthy Volunteers: a Concentration-QTcF Analysis. Poster presented at: PAGE 2023. June 27-30, 2023. A Coruña, Spain

Evaluation of Darigabat QTc Prolongation in Healthy Volunteers: a Concentration-QTcF Analysis

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Darigabat Epilepsy Panic disorder

May, 2023

Quantifying the tolerability of antipsychotic treatment-related side effects in schizophrenia: a survey study of patients and caregiver proxies

Maravic, Birch, Nguyen, et al. Quantifying the Tolerability of Antipsychotic Treatment-Related Side Effects in schizophrenia: A Survey Study of Patients and Caregivers. Abstract accepted for Poster Presentation at: SIRS 2023; May 11-15, 2023. Toronto, Canada

Objective: To quantify tolerance for side effects associated with antipsychotics, as reported directly by patients and by caregiver proxy respondents.

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N/A Parkinson’s disease

May, 2023

Dopamine D1 Agonists: First Potential Treatment for Late-Stage Parkinson’s Disease

Lewis, Van Scoy, De Jesus , et al. The potential effect of dopamine D1 agonists in late-stage Parkinson’s disease: Caregivers’ perspective in a phase I trial. Biomolecules. 2023;13(5):829.

Background: Current pharmacotherapy has limited efficacy and/or intolerable side effects in late-stage Parkinson’s disease (LsPD) patients whose daily life depends primarily on caregivers and palliative care. Clinical metrics inadequately gauge efficacy in LsPD patients. 

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Tavapadon Parkinson’s disease

April, 2023

Darigabat Reduces Acute Panic and Fear Symptoms Induced by CO2 Inhalation in Healthy Participants

Gurrell, Chang, Dandurand, et al. Phase 1 Trial of Darigabat for the Reduction of Acute Psychological and Physiological Panic and Fear Symptoms Following CO2 Inhalation in Healthy Participants. Presented at: AAN 2023. April 22-27, 2022. Boston, MA

Objective: The objective of the current trial was to characterize the anxiolytic effect of darigabat in a CO2 inhalation translational model of panic and fear in healthy participants.

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Darigabat Panic disorder

April, 2023

Impact of the Highly Selective D1-Like Partial Dopamine Agonist Tavapadon on Daytime Sleepiness: Evidence From a Phase 2 Clinical Trial

Leoni, Chang, Combs, et al. Impact of the highly selective D1-like dopamine agonist tavapadon on daytime sleepiness: Evidence from Phase 2 clinical trials. Poster presented at: ADPD 2023; March 28-April 1, 2023; Gothenburg, Sweden

Objective: To report daytime sleepiness data from the phase 2 clinical trial of tavapadon in participants with early early-stage Parkinson’s disease (PD) utilizing the Epworth Sleepiness Scale (ESS).

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Tavapadon Parkinson’s disease

April, 2023

Perspectives on Parkinson’s Disease Medications: A Qualitative Study of People With Parkinson’s Disease

Richards, Mantri, Brown, et al. Perspectives on Parkinson’s Disease Medications: A Qualitative Study of People With Parkinson’s Disease. Poster presented at: AAN 2023; April 22-27, 2023; Boston, MA

Objective: To explore the thoughts, beliefs, and expectations of people with Parkinson’s disease ( PwPD ) regarding their PD medications.

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Tavapadon Parkinson’s disease

April, 2023

Work Loss and Direct and Indirect Costs Associated with Parkinson’s Disease

Jerry, Arcona, McMorrow, et al. Work Loss and Direct and Indirect Costs Associated with Parkinson’s Disease. Clinicoecon Outcomes Res. 2023;15:309-319.

Purpose: To examine work loss and indirect costs during the three-year periods prior to and following initial diagnosis of Parkinson’s disease (PD) in patients and in spouses of PD patients, as well as direct costs of healthcare.

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N/A Parkinson’s disease

March, 2023

Rationale and Development of Tavapadon, a D1/D5-Selective Partial Dopamine Agonist for the Treatment of Parkinson’s Disease

Bezard, Gray, Kozak, et al. Rationale and Development of Tavapadon, a D1/D5-Selective Partial Dopamine Agonist for the Treatment of Parkinson’s Disease. CNS Neuro Disord Drug Targets. 2023. doi: 10.2174/1871527322666230331121028.

Objective: This review summarizes currently available evidence of tavapadon’s therapeutic potential for the treatment of early through advanced PD.

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Tavapadon Parkinson’s disease

December, 2022

CVL-354, a novel, brain penetrant and selective kappa opioid receptor antagonist

Suidan, Missig, Pin, et al. CVL-354, a novel, brain penetrant and selective kappa opioid receptor antagonist. Poster presentation at: ACNP 2023. December 4-7, 2022. Phoenix, AZ

Objective: To characterize in vitro and in vivo binding, selectivity, and functional activity of CVL 354 a novel KOR antagonist.

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CVL-354 Pre-clinical

December, 2022

A novel, short-acting kappa opioid receptor antagonist blocks the analgesic effects of U50,488 and attenuates symptoms of spontaneous oxycodone withdrawal in rats

Suidan, Neal, Driscoll, et al. A novel, short-acting kappa opioid receptor antagonist blocks the analgesic effects of U50,488 and attenuates symptoms of spontaneous oxycodone withdrawal in rats. Poster presentation at: ACNP 2022. December 4-7, 2022. Phoenix, AZ

Objective: Determine if CVL-354 is a short-acting (<24 hours) kappa opioid receptor antagonist.

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CVL-354 Pre-clinical

December, 2022

Evaluation of Kappa and Mu Opioid Receptor Occupancy by CVL-354 Using PET in Nonhuman Primates

Duvvuri, Iredale, Suidan, et al. Evaluation of Kappa and Mu Opioid Receptor Occupancy by CVL-354 Using PET in Nonhuman Primates. Poster presentation at: ACNP 2022. December 4-7, 2022. Phoenix, AZ

Objective: The objective of this study was to measure the RO of varying doses of CVL-354 at kappa and mu receptors in nonhuman primates using radiotracers [11C]LY27950502,3 and [11C]carfentanil ([11C]CFN), respectively

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CVL-354 Pre-clinical

December, 2022

Evaluation of Selective Muscarinic M4 Agonists on Cortical Oscillations: a Sleep-EEG Study in Rats

Leiser, Nguyen, Stolyar, et al. Evaluation of Selective Muscarinic M4 Agonists on Cortical oscillations: A Sleep-EEG Study in Rats. Poster presentation at: ACNP 2022. December 4-7, 2022. Phoenix, AZ

Background: A primary role for the M4 muscarinic acetylcholine receptor (M4 mAChR) is to function as an inhibitory autoreceptor

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M4 Agonist Pre-clinical

December, 2022

Dopamine agonist monotherapy utilization in patients with Parkinson’s disease

Frazer, Arcona, Le, Sasane. Dopamine agonist monotherapy utilization in patients with Parkinson's disease. Clin Park Relat Disord. 2022;8:100173.

Objective: To characterize patients with Parkinson’s disease (PD) who initiated dopamine agonist (DA) monotherapy, describe medication utilization and provider types, and estimate medication adherence and discontinuation rates.

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N/A Parkinson’s disease

December, 2022

Emraclidine, a novel positive allosteric modulator of cholinergic M4 receptors, for the treatment of schizophrenia: a two-part, randomised, double-blind, placebo-controlled, phase 1b trial

Krystal, Kane, Correll, et al. CVL-231, a novel positive allosteric modulator of cholinergic M4 receptors for the treatment of schizophrenia: a two-part, randomized, double-blind, placebo-controlled phase 1b trial. Lancet. 2023;400(10369):2210-2220.

Background: Emraclidine is a novel, brain-penetrant, highly selective M4 receptor positive allosteric modulator in development for the treatment of schizophrenia.

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Emraclidine Schizophrenia

November, 2022

Binding and Signal Profiling of Full and Partial M4 Agonists

Pin, Wonsey-Quinn, Smith, et al. Binding and Signal Profiling of Full and Partial M4 Agonists. Poster presentation at: SfN 2022. November 12-16, 2022. San Diego, CA

Objective: The aim of this study was to understand the agonism and the binding profile of M4 ligands with differentdegrees of intrinsic activities by using multiple probes, evaluating different signaling events/pathways, and by performing experiments at steady state, as well as at multiple time points.

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M4 Agonist Pre-clinical

November, 2022

Pronounced antiseizure activity of the subtype-selective GABAA positive allosteric modulator darigabat in a mouse model of drug-resistant focal epilepsy

Gurrell, Iredale, Evrard, et al. Pronounced antiseizure activity of the subtype-selective GABAA positive allosteric modulator darigabat in a mouse model of drug-resistant focal epilepsy. CNS Neurosci Ther. 2022:28(11):1875-1882.

Aim: Darigabat is an α2/3/5 subunit-selective positive allosteric modulator of GABAAreceptors that has demonstrated broad-spectrum activity in several preclinical models of epilepsy as well as in a clinical photoepilepsy trial. 

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Darigabat Epilepsy

November, 2022

Efficacy of Subtype-Selective, Full vs Partial M4 Muscarinic Receptor Agonists in Modulating Amphetamine-Induced Brain Activity Assessed by Functional MRI (fMRI) in Rats

Bardehle, Suidan, Nguyen, et al. Efficacy of subtype-selective, full vs partial M4 muscarinic receptor agonists in modulating amphetamine-induced brain activity assessed by functional MRI (fMRI) in rat. Poster presentation at: SfN 2022. November 12-16, 2022. San Diego, CA

Objective: To investigate the effects of systemic subcutaneous (SC) administration of 2 different test compounds (TC) – a full (CV-0000042) compared with a partial (CV-0000071) M4 receptor agonist – at 2 doses each on amphetamine-induced brain activity in anesthetized Sprague-Dawley rats (Crl:CD(SD)) using relative cerebral blood volume (rCBV) functional MRI (fMRI) readout

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M4 Agonist Pre-clinical

November, 2022

Efficacy of CV-0000042, a Novel M4 Receptor Full Agonist,and CV-0000071, a Novel M4 Receptor Partial Agonist, in Preclinical In Vivo Models

Stolyar, Suidan, Chakilam, et al. Efficacy of CVL-042, a novel M4 receptor full agonist, and CV-0000071, a novel M4 receptor partial agonist in preclinical in vivo models of psychosis. Poster presentation at: SfN 2022. November 12-16, 2022. San Diego, CA

Objective: To evaluate preclinical antipsychotic properties of M4 agonism while minimizing offtarget side effects using the novel M4 full agonist CV-0000042 and novel M4 partial agonist CV-0000071.

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M4 Agonist Pre-clinical

October, 2022

Assessment of Extrapyramidal Symptoms in a Medicaid Population with Schizophrenia Newly Prescribed Second Generation Antipsychotic Medications

Richards, Johnsrud, Zacker, Sasane R. Assessment of Extrapyramidal Symptoms in a Medicaid Population with Schizophrenia Newly Prescribed Second-Generation Antipsychotic Medications. Poster presented at: ACMP Nexus. October 11-14, 2022; National Harbor, MD

Objective: To describe the 1 year incidence of EPS in patients with schizophrenia newly initiated on an SGA medication within a Medicaid population

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N/A Schizophrenia

October, 2022

Considerations for determining the efficacy of new antiseizure medications in children age 1 month to younger than 2 years

French, Cleary, Dlugos, et al. Considerations for determining the efficacy of new antiseizure medications in children age 1 month to younger than 2 years. Epilepsia. 2022;63(10):2664-2670.

Objective: Drug treatment for children with epilepsy should, ideally, be governed by evidence from adequate and well-controlled clinical studies.

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N/A Epilepsy

July, 2022

Darigabat: REALIZE Trial Progress. 2022 Epilepsy Foundation Pipeline Congress

Frohlich

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Darigabat Epilepsy

June, 2022

PET mapping of receptor occupancy using joint direct parametric reconstruction: in-vivo studies

Marin, Belov, Ouyang, et al. PET mapping of receptor occupancy using joint direct parametric reconstruction: in-vivo studies. Poster presented at: Society of Nuclear Medicine and Molecular Imaging 2022 Annual Meeting; June 11-14, 2022; Vancouver, BC, Canada

Background: Receptor occupancy (RO) studies using PET play a critical role in the development of drugs targeting the central nervous system to quantify target engagement, assess drug performance [1]

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Emraclidine Pre-clinical

May, 2022

Darigabat Reduces Acute Panic and Fear Symptoms Induced by CO2 Inhalation in Healthy Participants

Gurrell, Chang, Dandurand, et al. Darigabat reduces acute panic and fear symptoms induced by CO2 inhalation in healthy participants. Presented at: ASCP 2022. May 31 – June 3. Scottsdale, AZ

The objective of the current study was to characterize the anxiolytic effect of darigabat in a CO2 inhalation model of panic and fear in healthy volunteers

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Darigabat Panic disorder

May, 2022

Darigabat: A Potential Treatment Option for Epilepsy

"Gurrell. Darigabat: A Potential Treatment Option for Epilepsy Targeted GABA α 2/3/5 Receptor Selectivity. Presented at Sixteenth Eilat Conference on New Antiepileptic Drugs and Devices. May 23, 2022. Madrid, Spain"

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Darigabat Epilepsy

May, 2022

Setting the TEMPO: A Phase 3 Program to Investigate Tavapadon, a Selective D1/D5 Partial Agonist, for Parkinson’s Disease

Fernandez, Pfister, Leoni, et al. Setting the TEMPO: a phase 3 program to investigate the efficacy, safety, and tolerability of the selective D1/D5 partial agonist tavapadon in Parkinson’s disease. Poster presented at: IAPRD 2022; May 1-4, 2022; Prague, Czech Republic

Objective: To describe the design of the phase 3 TEMPO program that will evaluate the efficacy, safety, and tolerability of once-daily (QD) tavapadon in early-stage and advanced Parkinson’s disease (PD).

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Tavapadon Parkinson’s disease

April, 2022

Ensuring the Continuity of Data Collection in a Focal Epilepsy Clinical Trial During the COVID-19 Pandemic

Frohlich, Whitmore, Pfister, et al. Ensuring the Continuity of Data Collection in a Focal Epilepsy Clinical Trial During the COVID-19 Pandemic. Presented at: AAN 2022; April 2-7, 2022; Seattle, WA

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Darigabat Epilepsy

April, 2022

CVL-231 as a Novel Positive Allosteric Modulator Targeting M4 Muscarinic Receptors: Results From a Phase 1b Trial in Patients With Schizophrenia

Krystal, Kane, Correll, et al. CVL-231 as a novel positive allosteric modulator targeting M4 muscarinic receptors: results from a phase 1b trial in patients with schizophrenia. Poster presented at: SIRS 2022; April 6-10, 2022; Florence, Italy

Objective: To describe the safety, tolerability, and pharmacodynamics (PD) of emraclidine 30 mg once daily (QD) and emraclidine 20 mg twice daily (BID) in patients with acute schizophrenia.

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Emraclidine Schizophrenia

April, 2022

Levodopa and Dopamine Agonist Medication Utilization among Patients with Parkinson’s Disease Enrolled in Commercial and Medicare Advantage Part D Health Plans

Frazer, Le, Arcona, Sasane. Levodopa and dopamine agonist medication utilization among patients with Parkinson’s disease enrolled in commercial and Medicare Advantage Part D health plans. Poster presented at: AAN 2022; April 2-7, 2022; Seattle, WA

Objective: Perform an updated characterization of medication utilization among PD patients prescribed levodopa or dopamine agonist treatments. Estimate predictors of medication discontinuation among patients prescribed levodopa and dopamine agonist treatments.

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N/A Parkinson’s disease

April, 2022

Darigabat: pronounced antiepileptic activity in the mesial temporal lobe mouse model of drug- resistant focal epilepsy

Gurrell, Iredale, Evrard, et al. Pronounced antiepileptic activity of darigabat, a subtype‐selective GABAA receptor positive allosteric modulator, in the mesial temporal lobe model of drug-resistant focal epilepsy. Presented at: AAN 2022; April 2-7, 2022; Seattle, WA

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Darigabat Epilepsy

April, 2022

CVL‑231 as a novel positive allosteric modulator targeting M4 muscarinic receptors: results from a phase 1b trial in patients with schizophrenia. 2022 Congress of the Schizophrenia International Research Society

Krystal, Kane, Correll, et al.

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Emraclidine Schizophrenia

April, 2022

Pharmacokinetics, Pharmacodynamics, and Safety of the Highly Selective Dopamine D1/D5 Agonist Tavapadon: Summary of Early Phase Clinical Studies

Pastino, Yuan, Duvvuri, et al. Pharmacokinetics, pharmacodynamics, and safety of the highly selective dopamine D1/D5 agonist tavapadon: summary of phase 1 clinical studies. Presented at: AAN 2022; April 2-7, 2022; Seattle, WA

Objective: To describe the pharmacokinetic (PK), pharmacodynamic, and safety data from early phase clinical studies of tavapadon.

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Tavapadon Parkinson’s disease

January, 2022

Levodopa treatment patterns in Parkinson’s disease: A retrospective chart review

Navaratnam, Arcona, Friedman, et al. Levodopa treatment patterns in Parkinson’s disease: A retrospective chart review. Clin Park Relat Disord. 2022;6:100135.

Background: Medication regimens for Parkinson’s disease (PD) may change as the disease progresses, symptoms fluctuate, or medication-related adverse events occur. This study evaluated treatment trends by observation year for patients initially receiving monotherapy with levodopa and a peripheral dopa decarboxylase inhibitor (PDDI).

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N/A Parkinson’s disease

December, 2021

Neuroimaging of M4 Muscarinic Acetylcholine Receptors Using [11c]Mk-6884 In Rhesus Macaques

Belov, Guehl, Duvvuri, et al. Neuroimaging of M4 muscarinic acetylcholine receptors using [11C]MK-6884 in rhesus macaques. Poster presented at: XIII International Symposium of Functional Neuroreceptor Mapping of the Living Brain; December 14-16, 2021; Virtual

Objective: Characterize PET imaging and quantitative performance of [11C]MK-6884 using arterial input function-based and reference tissue-based kinetic modelling methods.

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N/A Pre-clinical

December, 2021

CVL-231 as a Novel Positive Allosteric Modulator of Cholinergic M4 Receptors for the Treatmentof Schizophrenia: Results From an Early Proof-of-Concept Study in Patients With Schizophrenia

Krystal, Kane, Correll, et al. CVL-231 as a novel positive allosteric modulator of cholinergic M4 receptors for the treatment of schizophrenia: results from an early proof-of-concept study in patients with schizophrenia. Abstract presented at: ACNP 2021; December 5-8, 2021; San Juan, PR

Objective: To assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics of CVL-231 30 mg once daily (QD) and 20 mg twice daily (BID) in patients with schizophrenia who are experiencing acute exacerbation of psychosis.

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Emraclidine Schizophrenia

December, 2021

Natural history and patterns of treatment change in Parkinson’s disease: A retrospective chart review

Navaratnam, Arcona, Friedman, et al. Natural history and patterns of treatment change in Parkinson’s disease: A retrospective chart review. Clin Park Relat Disord. 2021;6:100125.

Background: Parkinson’s disease (PD) management seeks to balance the benefits and harms of current medications and evolves as the disease progresses. The natural history of PD and associated patterns of treatment change were analyzed to identify unmet needs in treatment of PD symptoms.

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N/A Parkinson’s disease

December, 2021

Evaluation of M4 Muscarinic Receptor Occupancy by CVL-231 Using [11C]MK-6884 PET in Nonhuman Primates

Duvvuri, Iredale, Leoni, et al. Evaluation of M4 muscarinic receptor occupancy by CVL-231 using [11C]MK-6884 PET in nonhuman primates. Abstract presented at: ACNP 2021; December 5-8, 2021; San Juan, PR

Objective: Using [11C]MK-6884, an M4 positive allosteric modulator radioligand, this study evaluated M4 receptor occupancy (RO) of CVL-231 in the striatum of nonhuman primates as a function of CVL-231 dose and plasma concentration.

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Emraclidine Schizophrenia

December, 2021

CVL‑231 as a novel positive allosteric modulator of cholinergic M4 receptors for the treatment of schizophrenia: results from an early proof‑of‑concept study in patients with schizophrenia. 60th Annual Meeting of American College of Neuropsychopharmacology

Krystal, Kane, Correll, et al.

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Emraclidine Schizophrenia

November, 2021

Establishing Signature Profile of Muscarinic M4 Selective Molecules by Evaluating Their Orthosteric/Allosteric Binding Properties and Biased Signaling

Pin, Toh, Nguyen, Iredale. Establishing signature profile of muscarinic M4 selective molecules by evaluating their orthosteric/allosteric binding properties and biased signaling. Poster presented at: SfN 2021; November 8-11, 2021; Virtual

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Emraclidine Pre-clinical

September, 2021

Dopamine D1 Receptor Agonist PET Tracer Development: Assessment in Nonhuman Primates

Barret, Zhang, Alagille, et al. Dopamine D1 Receptor Agonist PET Tracer Development: Assessment in Nonhuman Primates. J Nucl Med. 2021;62:1307-1313.

Background: Non-catechol-based high-affinity selective dopamine D1 receptor (D1R) agonists were recently described, and candidate PET ligands were selected on the basis of favorable properties. 

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N/A Parkinson’s disease

August, 2021

CVL‑865: The Journey to REALIZE. 2020 Epilepsy Foundation Pipeline Virtual Conference

Gurrell

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Darigabat Epilepsy

July, 2021

Safety, Tolerability, and Pharmacokinetics of Multiple Repeated Oral Doses of the α2/3/5-Subtype Selective GABAA -Positive Allosteric Modulator PF-06372865 in Healthy Volunteers

Gurrell, Whitlock, Wei, et al. Safety, tolerability, and pharmacokinetics of multiple repeated oral doses of the α2/3/5-subtype selective GABAA-positive allosteric modulator PF-06372865 in healthy volunteers. Clin Pharmacol Drug Dev. 2021;10(7):756-764.

Background: Multiple-dose pharmacokinetics (PK) and safety were investigated in this phase 1 study of PF-06372865, a positive allosteric modulator of α2/3/5 subunit-containing γ-aminobutyric acid A receptors (NCT03351751).

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Darigabat Epilepsy

June, 2021

Parkinson disease among patients treated for benign prostatic hyperplasia with α1 adrenergic receptor antagonists

Sasane, Bartels, Field, et al. Parkinson disease among patients treated for benign prostatic hyperplasia with α1 adrenergic receptor antagonists. J Clin Invest. 2021;131(11):e145112

Background: Recently the α1 adrenergic receptor antagonist terazosin was shown to activate PGK1, a possible target for the mitochondrial deficits in Parkinson disease related to its function as the initial enzyme in ATP synthesis during glycolysis. 

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N/A Parkinson’s disease

April, 2021

A Novel Positive Allosteric Modulator of the Cholinergic M4 Receptor for the Treatment of Schizophrenia

Kane, Keefe, Walling, et al. CVL-231: a novel positive allosteric modulator of the cholinergic M4 receptor for the treatment of schizophrenia. Poster presented at: SIRS 2021; April 17-21, 2021; Virtual

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Emraclidine Schizophrenia

November, 2020

Progress report on new antiepileptic drugs: a summary of the Fifteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XV). II. Drugs in more advanced clinical development. Epilepsia

Bialer, Johannessen, Koepp, et al.

The Fifteenth Eilat Conference on New Antiepileptic Drugs and Devices (EILAT XV) was held as a fully virtual conference from July 27 to July 30, 2020 for the sessions on drugs…

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Darigabat Epilepsy

September, 2020

Claims Data Analysis of Parkinson’s Disease Medication Utilization

Frazer, Peterson, Arcona, et al. Claims data analysis of Parkinson’s disease medication utilization. Poster presented at: MDS 2020; September 12-16; Virtual

Objective: Characterize prominent medication utilization patterns among patients with Parkinson’s disease (PD).

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N/A Parkinson’s disease

September, 2020

An analysis of Parkinson’s disease medication treatment patterns among Medicaid patients. 

Johnsrud, Richards, Arcona, et al. An analysis of Parkinson's disease medication treatment patterns among Medicaid patients. Poster presented at: MDS 2020; September 12-16; Virtual

Objective: To describe patient demographic and medication utilization patterns within a state Medicaid program for patients diagnosed with Parkinson’s disease(PD).

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N/A Parkinson’s disease

September, 2020

Claims data analysis of Parkinson’s disease medication utilization. MDS‑International Parkinson and Movement Disorder Society Virtual Congress 2020

Frazer, Blauer-Peterson, Arcona, et al.

Objective: Characterize prominent medication utilization patterns among patients with Parkinson’s disease (PD).

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N/A Parkinson’s disease

April, 2020

Dopaminergic D1 Receptor Stimulation Affects Effort and Risk Preferences

Soutschek, Gvozdanovic, Kozak, et al. Dopaminergic D1 Receptor Stimulation Affects Effort and Risk Preferences. Biol Psychiatry. 2020;87(7):678-685.

Background: Activation of D1 receptors has been related to successful goal-directed behavior, but it remains unclear whether D1 receptor activation causally tips the balance of weighing costs and benefits in humans.

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Tavapadon Parkinson’s disease

March, 2020

PF-06649751 efficacy and safety in early Parkinson’s disease: a randomized, placebo-controlled trial

Riesenberg, Werth, Zhang, et al. PF-06649751 efficacy and safety in early Parkinson's disease: a randomized, placebo-controlled trial. Ther Adv Neurol Disord. 2020;13:1756286420911296.

Background: PF-06649751 is a novel, oral, non-catechol-based, D1/D5 dopamine receptor partial agonist under investigation for the treatment of motor symptoms associated with Parkinson’s disease.

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Tavapadon Parkinson’s disease

February, 2020

D1 Agonist Improved Movement of Parkinsonian Nonhuman Primates with Limited Dyskinesia Side Effects

Young, Popiolek, Trapa, et al. D1 agonist improved movement of parkinsonian nonhuman primates with limited dyskinesia side effects. ACS Chem Neurosci. 2020;11(4):560-566.

Background: Parkinson’s disease is a progressive neurodegenerative disease characterized by striatal dopaminergic loss. L-DOPA treatment replaces lost dopamine and enables motor function; however, eventually, fluctuating efficacy and side effects associated with its use become challenging for many patients.

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Tavapadon Parkinson’s disease

September, 2019

Efficacy, Safety and Tolerability of Tavapadon in Subjects With Early Stage Parkinson’s Disease

Gray, Riesenberg, Werth, et al. Efficacy, safety and tolerability of tavapadon in subjects with early stage Parkinson’s disease [MDS abstract 98]. Mov Disord. 2019;34 (suppl 2)

Objective: To evaluate the efficacy and safety of tavapadon (formerly known as PF-06649751) in subjects with early stage Parkinson’s disease.

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Tavapadon Parkinson’s disease

September, 2019

Utility of the double‑blind crossover design protocol for proof‑of‑principle photosensitivity model assessment of PF‑06372865. 2019 International Society for CNS Clinical Trials and Methodology

Gurrell, Gorman, Whitlock, et al.

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Darigabat Epilepsy

September, 2019

Analgesic potential of PF-06372865, an α2/α3/α5 subtype-selective GABAA partial agonist, in humans

van Amerongen, Siebenga, Gurrell, et al. Analgesic potential of PF-06372865, an α2/α3/α5 subtype-selective GABAA partial agonist, in humans. Br J Anaesth. 2019;123(2):e194-e203.

Background: This study investigated the analgesic effects of two doses (15 and 65 mg) of PF-06372865, a novel α2/α3/α5 gamma-aminobutyric acid A (GABAA) subunit selective partial positive allosteric modulator (PAM), compared with placebo and pregabalin (300 mg) as a positive control.

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Darigabat Pain

April, 2019

Design and Identification of a Novel, Functionally Subtype Selective GABAA Positive Allosteric Modulator (PF-06372865)

Owen, Blakemore, Cao, et al. Design and identification of a novel, functionally subtype selective GABAA positive allosteric modulator (PF-06372865). J Med Chem. 2019;62(12):5773-5796.

Background: The design, optimization, and evaluation of a series of novel imidazopyridazine-based subtype-selective positive allosteric modulators (PAMs) for the GABAA ligand-gated ion channel are described. 

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Darigabat Epilepsy

April, 2019

Photosensitive epilepsy: Robust clinical efficacy of a selective GABA potentiator

Gurrell, Gorman, Whitlock, et al. Photosensitive epilepsy: robust clinical efficacy of a selective GABA potentiator. Neurology. 2019;92(15):e1786-e1795.

Objective: The objective of this phase 2a study was to assess the activity of PF-06372865, a positive allosteric modulator (PAM) of α2/3/5 subunit-containing GABAAreceptors with minimal activity at α1-containing receptors, which are believed to mediate many of the adverse events associated with benzodiazepines, in the epilepsy photosensitivity model as a proof-of-principle of efficacy.

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Darigabat Epilepsy

February, 2019

Pronounced antiepileptic activity of the subtype-selective GABAA -positive allosteric modulator PF-06372865 in the GAERS absence epilepsy model

Duveau, Buhl, Evrard, et al. Pronounced antiepileptic activity of the subtype-selective GABAA-positive allosteric modulator PF-06372865 in the GAERS absence epilepsy model. CNS Neurosci Ther. 2019;25(2):255-260.

Objective: Antiepileptic drugs that modulate GABA have the potential to aggravate or improve the symptoms of absence epilepsy.

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Darigabat Epilepsy

July, 2018

Phase 1 Parkinson’s Disease Studies Show the Dopamine D1/D5 Agonist PF-06649751 is Safe and Well Tolerated

Sohur, Gray, Duvvuri, et al. Phase 1 Parkinson's disease studies show the dopamine D1/D5 agonist PF-06649751 is safe and well tolerated. Neurol Ther. 2018;7(2):307-319.

Background: There is a need for new therapies in Parkinson’s disease that may help to address known limitations of current options.

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Tavapadon Parkinson’s disease