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Valbenazine

Phase 3

Chorea, Huntington | Small molecule | Neurology |Neurocrine Biosciences, Inc.|Last Updated: Jun 18, 2026

Target and mechanism

Molecular targetSLC18A2
Target classInhibitor
ModalitySmall molecule

Also known as Valbenazine Oral Capsule

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials3
Total Enrollment289

FDA Designations

No designations recorded

Clinical trial landscape

Valbenazine · 11 trials · 8 indications

Phase 3 6Phase 2 5
NCT06312189Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in CanadaChorea, Huntington
ENROLLING BY_INVITATION7 Analytics
NCT05206513Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral PalsyDyskinesia
COMPLETED86 Analytics
NCT05110157Journey Study: Evaluate the Efficacy, Safety, and Tolerability of Valbenazine as Adjunctive Treatment for SchizophreniaSchizophrenia
COMPLETED442 Analytics
NCT04400331Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington DiseaseChorea, Huntington
COMPLETED154 Analytics
NCT04102579Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington DiseaseChorea, Huntington
COMPLETED128 Analytics
NCT02736955Rollover Study for Continuing Valbenazine (NBI-98854) Administration for the Treatment of Tardive DyskinesiaTardive Dyskinesia
COMPLETED161 Analytics
PHASE3ENROLLING BY_INVITATION
Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in Canada
Chorea, HuntingtonUnlock trial analytics
PHASE3COMPLETED
Study to Assess the Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Dyskinesia Due to Cerebral Palsy
DyskinesiaUnlock trial analytics
PHASE3COMPLETED
Journey Study: Evaluate the Efficacy, Safety, and Tolerability of Valbenazine as Adjunctive Treatment for Schizophrenia
SchizophreniaUnlock trial analytics
PHASE3COMPLETED
Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington Disease
Chorea, HuntingtonUnlock trial analytics
PHASE3COMPLETED
Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington Disease
Chorea, HuntingtonUnlock trial analytics
PHASE3COMPLETED
Rollover Study for Continuing Valbenazine (NBI-98854) Administration for the Treatment of Tardive Dyskinesia
Tardive DyskinesiaUnlock trial analytics

Study Endpoints

Primary Endpoints

Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Up to Week 210
Change in the Total Maximal Chorea (TMC) Score of the Unified Huntington Disease Rating Scale (UHDRS) from Baseline to the Average of the Week 12 and Week 14 assessments
Baseline, Week 12 and Week 14
Change in Positive and Negative Syndrome Scale (PANSS) total score from baseline to Week 10
Baseline to week 10
Change From Screening Period Baseline to Maintenance Period in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score.
Baseline (average of screening and Day -1), maintenance (average of Weeks 10 and 12)

The TMC is part of the motor assessment of the UHDRS and measures chorea in 7 different body parts including the face, oral-buccal-lingual region, trunk and each limb independently. The TMC score is the sum of the individual scores and ranges from 0 to 28. A decrease in TMC scores indicates improvement in chorea symptoms.

Number of Participants Monitored for Long-term Safety of Valbenazine
60 weeks

Incidence of adverse events and monitoring of vital signs, clinical laboratory values, and electrocardiograms. All AEs were coded into preferred terms according to MedDRA (Medical Dictionary for Regulatory Activities) and classified by system organ class (SOC). Summaries of the incidence of all treatment-emergent AEs, treatment-related AEs, SAEs, and AEs leading to study drug discontinuation were prepared.

Yale-Brown Obsessive-Compulsive Scale (Y-BOCS)
Baseline to week 14

A clinician-administered scale assessing OCD severity that will be used at all 8 visits. Total scores range from 0-50. Higher scores indicate worse outcomes.

Change in Massachusetts General Hospital Hairpulling Scale (MGH-HPS) from baseline to Week 12
baseline to week12

Massachusetts General Hospital Hairpulling Scale (MGH-HPS) scores range from 0-28 with 28 being the most severe. The lower the total score, the lower the severity level. This scale is given and measured once every 2 weeks for a total of 12 weeks.

Change From Baseline to Week 12 in the Yale Global Tic Severity Scale (YGTSS) Total Tic Score (TTS)
Baseline, Week 12

The YGTSS is designed to rate the overall severity of motor and phonic tic symptoms across a range of dimensions: number, frequency, intensity, complexity, and interference. The YGTSS was administered by the investigator (or qualified designee) using a computer-based structured clinical interview. The TTS is the sum of the 5 motor tic items and the 5 phonic (vocal) tic items and ranges from 0 to 50, with higher scores representing greater severity.

Frequency of Treatment-emergent Adverse Events (TEAEs)
Baseline through Week 28

A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.

Secondary Endpoints

Change in the Clinical Global Impression of Severity (CGI-S) Score from Baseline to Week 14
Baseline, Week 14
Change in the Movement Disorders - Childhood Rating Scale (MD-CRS) Part I Score from Baseline to the Average of the Week 12 and Week 14 Assessments
Baseline, Week 12 and Week 14
Change in the Total Maximal Dystonia (TMD) Score of the UHDRS from Baseline to the Average of the Week 12 and Week 14 Assessments
Baseline, Week 12 and Week 14
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ValbenazineEXPERIMENTALCapsule, administered orally once daily.
PlaceboPLACEBO_COMPARATORPlacebo dosed for 14 weeks (double-blind treatment period) followed by open-label valbenazine treatment. Participants may opt to enter an open-label valbenazine treatment extension phase.
Vesicular monoamine transporter 2 (VMAT2) inhibitorEXPERIMENTALValbenazine once daily
Valbenazine followed by placeboOTHERFollowing baseline measures, 50% of subjects will receive valbenazine (initially 40mg per day for 2 weeks and then 80mg per day for 4 weeks). This will be followed by a 2-week washout period and then 6 weeks of being assigned to the other agent. Participants will be assessed at baseline and every 2 weeks during the remainder of the study period.
Placebo followed by valbenazineOTHERFollowing baseline measures, 50% of subjects will receive inactive placebo for 6 weeks. This will be followed by a 2-week washout period and then 6 weeks of being assigned to the other agent (initially 40mg per day for 2 weeks and then 80mg per day for 4 weeks). Participants will be assessed at baseline and every 2 weeks during the remainder of the study period.
Valbenazine (Children)EXPERIMENTALChildren (6 to 11 years of age) received valbenazine 10 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 20 mg or continue with the participant's current dose for the remainder of the treatment period.
Valbenazine (Adolescents)EXPERIMENTALAdolescents (12 to 17 years of age) received valbenazine 20 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 40 mg or continue with the participant's current dose for the remainder of the treatment period.
Valbenazine (Adults)EXPERIMENTALAdults (18 to 64 years of age) received valbenazine 40 mg once daily for 4 weeks. At the end of Week 4, investigators could escalate the dose to 80 mg or continue with the participant's current dose for the remainder of the treatment period.

Interventions

NameTypeDescription
ValbenazineDRUGvesicular monoamine transporter 2 (VMAT2) inhibitor
PlaceboDRUGCapsule, administered once daily orally or via gastrostomy/gastrojejunostomy tube
Valbenazine Oral CapsuleDRUGParticipants randomized to active treatment will be given a starting dose of 40mg of valbenazine , which may be escalated to 80mg to achieve an optimal dose of the medication for each subject.
Placebo Oral capsuleOTHERParticipants randomized to placebo will be given a capsule that is the same shape and color as the active medication and will subject to the same dose escalation requirements as the other arm but will receive placebo during the treatment period.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites3

Key Inclusion Criteria: * Participated in Study NBI-98854-HD3006 and completing dosing through the Week 156 visit; or were actively participating, receiving study treatment, and completed an early termination visit Study NBI-98854-HD3006 at the time of the study closure at sites in Canada. * Partic...

Countries:CanadaUnited StatesArgentinaBelgiumBrazilIsraelItalyMexicoPolandPortugalSpainBulgariaSerbiaPuerto Rico
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Recent Changes (Last 90 Days)

MEDIUMJul 18, 2026NCT04400331TRIAL_REMOVED: changed
MEDIUMJul 18, 2026NCT04400331TRIAL_REMOVED: changed
MEDIUMJul 18, 2026NCT04400331TRIAL_REMOVED: changed
MEDIUMJun 18, 2026NCT06312189primaryCompletionDate: changed
HIGHJun 18, 2026NCT04400331Status: ACTIVE_NOT_RECRUITING → COMPLETED
MEDIUMJun 18, 2026NCT06312189primaryCompletionDate: changed
HIGHJun 18, 2026NCT04400331Status: ACTIVE_NOT_RECRUITING → COMPLETED
MEDIUMJun 18, 2026NCT06312189primaryCompletionDate: changed
HIGHJun 18, 2026NCT04400331Status: ACTIVE_NOT_RECRUITING → COMPLETED
HIGHJun 18, 2026NCT04400331Status: ACTIVE_NOT_RECRUITING → COMPLETED

Frequently asked questions about Valbenazine

What is Valbenazine used for?

Valbenazine is an investigational small molecule in Phase 3 development for several neurological and psychiatric conditions, including tardive dyskinesia, trichotillomania (hair-pulling disorder), dyskinesia, obsessive-compulsive disorder, chorea associated with Huntington disease, and schizophrenia. It is being studied as an adjunctive treatment for schizophrenia.

Who makes Valbenazine?

Valbenazine is being developed by Neurocrine Biosciences, Inc., a biopharmaceutical company traded on NASDAQ under the ticker NBIX. The company is conducting Phase 3 clinical trials to evaluate the drug's efficacy and safety across multiple indications.

What phase is Valbenazine in?

Valbenazine is in Phase 3 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Multiple Phase 3 trials have been completed, and one active trial is ongoing, according to the clinical trial data.

What clinical trials is Valbenazine in?

Valbenazine has been studied in several Phase 3 trials, including NCT02736955 for tardive dyskinesia, NCT04400331 for chorea associated with Huntington disease, NCT05110157 for schizophrenia, and NCT05206513 for dyskinesia due to cerebral palsy. These trials have been completed, with total enrollment of 442 participants across studies.

Is Valbenazine the same as Valbenazine Oral Capsule?

Yes, Valbenazine Oral Capsule is another name for Valbenazine. The drug is being developed in an oral capsule formulation by Neurocrine Biosciences for the treatment of various neurological and psychiatric disorders.