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Elafibranor

Phase 2

Primary Biliary Cholangitis (PBC) | Small molecule | Other |Genfit SA Sponsored ADR|Last Updated: Sep 24, 2019

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 Enrollment45
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03124108Study to Evaluate the Efficacy and Safety of Elafibranor in Patients With Primary Biliary Cholangitis (PBC) and Inadequate Response to Ursodeoxycholic AcidPHASE2 COMPLETED 45Apr 5, 2017Oct 31, 2018Sep 24, 201924 United States, France +3
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Study Endpoints
Primary Endpoints
Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) Levels at Week 12 (Endpoint)
Baseline, Week 12 (Endpoint)

Relative change from baseline is in serum ALP levels at Week 12 (endpoint) were reported. Relative change from baseline is defined as percentage (%) change from baseline to endpoint.

Secondary Endpoints
Percentage of Participants With Response Defined by Composite Risk Scores (ALP< 1.67 * Upper Limit of Normal [ULN] at Endpoint, Total Bilirubin [BIL] Within Normal Limits at Endpoint, and Greater Than [>] 15% ALP Reduction From Baseline to Endpoint)
Up to Week 12 (Endpoint)
Percentage of Participants With Response Defined by Composite Risk Scores (ALP < 2 * Upper Limit of Normal at Endpoint, Total Bilirubin Within Normal Limits at Endpoint, and > 40% ALP Reduction From Baseline to Endpoint)
Up to Week 12 (Endpoint)
Percentage of Participants With Response Based on PARIS I Risk Score at Endpoint
At Week 12 (Endpoint)
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
PlaceboPLACEBO_COMPARATORStudy subjects will take two tablets per day orally before breakfast with a glass of water each morning
Elafibranor 80 mgACTIVE_COMPARATORStudy subjects will take two tablets per day orally before breakfast with a glass of water each morning
Elafibranor 120 mgACTIVE_COMPARATORStudy subjects will take two tablets per day orally before breakfast with a glass of water each morning
Interventions
NameTypeDescription
Elafibranor 80 mgDRUGTwo coated tablets daily for 12 weeks
Elafibranor 120 mgDRUGTwo coated tablets daily for 12 weeks
PlaceboDRUGTwo coated tablets daily for 12 weeks
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites24

Inclusion Criteria: 1. Must have provided written informed consent 2. Definite or probable PBC diagnosis as demonstrated by the presence of at least 2 of the following 3 diagnostic factors: * History of elevated ALP levels for at least 6 months prior to Day 0 (randomization visit) * Positive...

Countries:United StatesFranceGermanySpainUnited Kingdom
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