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Volixibat

Phase 2

Primary Sclerosing Cholangitis | Small molecule | Other |Mirum Pharmaceuticals, Inc.|Last Updated: Feb 4, 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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment182
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04663308A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)PHASE2 ACTIVE NOT_RECRUITING 182Dec 18, 2020Apr 1, 2027Feb 4, 2026103 United States, Argentina +12
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Study Endpoints
Primary Endpoints
Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire
Baseline through to Week 28

The Adult ItchRO is an 11-point NRS measurement of itch severity ranging from 0=no itch to 10=worst possible itch.

Secondary Endpoints
Proportion of participants with itch response using the Adult ItchRO
Baseline through to Week 28
The incidence of adverse events
Baseline through to Week 28
Changes in serum bile acid levels
Baseline through to Week 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part 1 Arm 1: Volixibat 20mgEXPERIMENTALParticipants randomized to this arm will receive volixibat 20mg twice daily.
Part 1 Arm 2: Volixibat 80mgEXPERIMENTALParticipants randomized to this arm will receive volixibat 80mg twice daily.
Part 1 Arm 3: PlaceboPLACEBO_COMPARATORParticipants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Part 2 Arm 1: Volixibat Selected Dose 20mgEXPERIMENTALParticipants randomized to this arm will receive volixibat 20mg twice daily.
Part 2 Arm 2: PlaceboPLACEBO_COMPARATORParticipants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
Interventions
NameTypeDescription
VolixibatDRUGOral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
PlaceboDRUGCapsules matched to study drug minus active substance
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites103

Inclusion Criteria: 1. Provide freely signed informed consent and assent (as applicable) and be willing to comply with all study visits and requirements through end of study, including the follow-up period. 2. Subjects aged ≥12 years for eligible regions; otherwise ≥18 years 3. Confirmed diagnosis ...

Countries:United StatesArgentinaAustraliaBelgiumBrazilCanadaFranceGermanyIsraelItalyNetherlandsSpainSwitzerlandUnited Kingdom
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04663308primaryCompletionDate: changed
LOWMay 24, 2026NCT04663308studyFirstPostDate: changed