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PBR06

Phase 1

Multiple System Atrophy | Small molecule | Neurology |Biohaven Ltd.|Last Updated: Aug 19, 2022

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment19
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04616456Effect of Verdiperstat on Microglial Activation in Well-characterized MSA PatientsEARLY_PHASE1 COMPLETED 19Dec 30, 2020Jan 30, 2022Aug 19, 20221 United States
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Study Endpoints
Primary Endpoints
standardized uptake values (SUV)
1 month

PET imaging measurement calculated over whole brain within putamen, pons, cerebellum

standardized uptake ratios (SUVRs)
1 month

PET imaging measurement calculated over whole brain within putamen, pons, cerebellum

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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Multiple System Atrophy (MSA)EXPERIMENTALTwenty to twenty-five subjects with probable MSA diagnosis will be recruited for this study. Each subject will undergo an \[F-18\]PBR06 PET and MRI scan at baseline, and will receive the experimental drug, verdiperstat (BHV-3241) under supervision of clinic staff. A follow-up \[F-18\]PBR06 PET and MRI scan will be performed after 6 months (26 weeks) of taking verdiperstat.
Interventions
NameTypeDescription
[F-18]PBR06DRUGPET radiopharmaceutical
VerdiperstatDRUGverdiperstat targets microglial inflammation in the brain
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

\*\*Subjects must be located in close proximity to Brigham and Women's Hospital, Boston, MA in order to meet monitoring criteria outlined in study protocol.\*\* Inclusion/Exclusion criteria: Subject Inclusion Criteria: 1\. Probable MSA clinical diagnosis Subject Exclusion Criteria: 1. Individua...

Countries:United States
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