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Part 1: LJN452

Phase 2

Primary Biliary Cholangitis | Small molecule | Other |Novartis AG|Last Updated: Jan 5, 2021

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 Trials1
Total Enrollment61
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02516605A Multi-part, Double Blind Study to Assess Safety, Tolerability and Efficacy of Tropifexor (LJN452) in PBC PatientsPHASE2 COMPLETED 61Sep 9, 2015Aug 2, 2018Jan 5, 202128 United States, Canada +4
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Study Endpoints
Primary Endpoints
Fold Change in Serum Gamma-glutamyl Transferase (GGT)
Baseline to Day 28

Fold change in serum gamma-glutamyl transferase (GGT) from baseline to Day 28

Blood Pressure
Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84

Vital signs - Systolic Blood pressure

Pulse Rate
Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84

Vital signs

Body Temperature
Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84

Vital signs

ECG - Heart Rate
Screening, Baseline, day 1, day 28

Electrocardiogram (ECG)

ECG Intervals - PR Interval
Screening, Baseline, day 1, day 28

Electrocardiogram (ECG)

Haemoglobin
Screening, Baseline, day 1, day 7, day 14, day 21, day 28, day 56, day 84

Hematology panel for safety laboratory assessments.

Secondary Endpoints
Plasma PK Parameter - AUC 0-8h
Day 1, Day 28
Plasma PK Parameter - Cmax
Day 1, Day 28
Plasma PK Parameter - Tmax
Day 1, Day 28
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LJN452EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
Part 1: LJN452DRUGLJN452 capsules administered once daily for 28 days
Part 1: PlaceboDRUGMatching placebo capsules administered once daily for 28 days
Part 2: LJN452 Dose level 1DRUGLJN452 capsules administered once a day for 12 weeks
Part 2: PlaceboDRUGMatching placebo to LJN452 administered once a day for 12 weeks
Part 2: LJN452 Dose level 2DRUGLJN452
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites28

Inclusion Criteria: * Age ≥ 18 years * Diagnosis of PBC as demonstrated by the presence of at least 2 of the following 3 diagnostic criteria: * History of alkaline phosphatase (ALP) elevated above upper limit of normal (ULN) for at least 6 months * Positive antimitochondrial antibodies (AMA) t...

Countries:United StatesCanadaGermanyPolandRussiaUnited Kingdom
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