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Valbenazine

Phase 3

Chorea, Huntington | Small molecule | Rare Disease |Neurocrine Biosciences, Inc.|Last Updated: Apr 9, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment289
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06312189Long-term Study to Evaluate Safety and Tolerability of Valbenazine in Participants With Chorea Associated With Huntington Disease in CanadaPHASE3 ENROLLING BY_INVITATION 7Apr 24, 2024Apr 1, 2026Apr 9, 20263 Canada
NCT04400331Open-Label Rollover Study for Continuing Valbenazine Administration for the Treatment of Chorea Associated With Huntington DiseasePHASE3 ACTIVE NOT_RECRUITING 154Sep 18, 2020Mar 1, 2026Dec 24, 202434 United States, Canada
NCT04102579Efficacy, Safety, and Tolerability of Valbenazine for the Treatment of Chorea Associated With Huntington DiseasePHASE3 COMPLETED 128Nov 13, 2019Oct 26, 2021Oct 11, 202346 United States, Canada
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Study Endpoints
Primary Endpoints
Number of Participants with Treatment-Emergent Adverse Events (TEAEs)
Up to Week 106
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.

Secondary Endpoints
Change from Baseline in the Unified Huntington's Disease Rating Scale (UHDRS) Total Maximal Chorea (TMC) Score
Up to 262 weeks
Percent of Clinical Global Impression of Change (CGI-C) Responders at Week 12
Week 12
Percent of Patient Global Impression of Change (PGI-C) Responders at Week 12
Week 12
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ValbenazineEXPERIMENTALCapsule, administered orally once daily.
PlaceboPLACEBO_COMPARATORCapsule, administered orally once daily for 12 weeks.
Interventions
NameTypeDescription
ValbenazineDRUGvesicular monoamine transporter 2 (VMAT2) inhibitor
PlaceboDRUGnon-active dosage form
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Eligibility Criteria
Age Range18 Years — 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 States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06312189primaryCompletionDate: changed
LOWMay 26, 2026NCT04400331primaryCompletionDate: changed
LOWMay 24, 2026NCT06312189studyFirstPostDate: changed
LOWMay 24, 2026NCT04400331studyFirstPostDate: changed