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LUM001

Phase 2

Progressive Familial Intrahepatic Cholestasis (PFIC) | Small molecule | Gastrointestinal |Mirum Pharmaceuticals, Inc.|Last Updated: Oct 23, 2023

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 Enrollment33
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02057718Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic CholestasisPHASE2 COMPLETED 33 — — Mar 1, 2014May 8, 2020Oct 23, 202310 United States, France +2
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Study Endpoints
Primary Endpoints
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level
Baseline (Day 0) to Week 13
Secondary Endpoints
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)
Baseline (Day 0) to Week 13
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)
Baseline (Day 0) to Week 13
Change From Baseline to Week 13/ET in ALT
Baseline (Day 0) to Week 13
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
LUM001 (Maralixibat)EXPERIMENTALParticipants will receive LUM001, also known as Maralixibat (MRX) twice a day (BID).
Interventions
NameTypeDescription
LUM001 (Maralixibat)DRUGLUM001 also known as Maralixibat (MRX) oral dose up to twice a day (BID).
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Eligibility Criteria
Age Range12 Months — 18 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria 1\. Male or female subjects between the ages of 12 months and 18 years inclusive. 2\. Diagnosis of PFIC based on: 1. Intrahepatic cholestasis manifest by total serum bile acid \>3x upper limit of normal (ULN) for age and, b or c: 2. Two documented mutant alleles in ATP8B1, or A...

Countries:United StatesFrancePolandUnited Kingdom
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