Primary Sclerosing Cholangitis

11
Pipeline Programs
6
Companies
9
Clinical Trials
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
6
0
3
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
1100%
+ 10 programs with unclassified modality

On Market (1)

Approved therapies currently available

Ipsen
IQIRVOApproved
elafibranor
Ipsen
Peroxisome Proliferator-activated Receptor Agonist [EPC]oral2024

Competitive Landscape

6 companies ranked by most advanced pipeline stage

Ipsen
IpsenChina - Tianjin
3 programs
2
1
1
ElafibranorPhase 31 trial
Elafibranor 80 mgPhase 21 trial
RitivixibatPhase 21 trial
Active Trials
NCT05627362Active Not Recruiting68Est. Aug 2026
NCT05642468Completed18Est. Jul 2025
NCT07387549Not Yet Recruiting350Est. May 2031
GS
Gilead SciencesFOSTER CITY, CA
4 programs
2
2
CilofexorPhase 31 trial
CilofexorPhase 3
SeladelparPhase 21 trial
SeladelparPhase 2
Active Trials
NCT04024813Terminated1Est. Jan 2020
NCT03890120Terminated419Est. Dec 2022
Pliant Therapeutics
Pliant TherapeuticsCA - South SF
1 program
1
PLN-74809Phase 21 trial
Active Trials
NCT04480840Completed121Est. Mar 2024
Mirum Pharmaceuticals
Mirum PharmaceuticalsFOSTER CITY, CA
1 program
1
VolixibatPhase 21 trial
Active Trials
NCT04663308Active Not Recruiting182Est. Apr 2027
Genentech
GenentechCA - Oceanside
1 program
1
ErlotinibPhase 1Small Molecule1 trial
Active Trials
NCT00955149Completed6Est. Apr 2013
Mauna Kea Technologies
1 program
probe based confocal laser endomicroscopyN/A1 trial
Active Trials
NCT02736708CompletedEst. May 2020

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
IpsenElafibranor
Gilead SciencesCilofexor
IpsenRitivixibat
IpsenElafibranor 80 mg
Mirum PharmaceuticalsVolixibat
Pliant TherapeuticsPLN-74809
Gilead SciencesSeladelpar
GenentechErlotinib
Mauna Kea Technologiesprobe based confocal laser endomicroscopy

Clinical Trials (9)

Total enrollment: 1,165 patients across 9 trials

NCT07387549IpsenElafibranor

A Study to Assess How Well and Safely Elafibranor Works in Adult Participants With Primary Sclerosing Cholangitis

Start: Apr 2026Est. completion: May 2031350 patients
Phase 3Not Yet Recruiting
NCT03890120Gilead SciencesCilofexor

Study of Cilofexor in Adults With Primary Sclerosing Cholangitis

Start: Mar 2019Est. completion: Dec 2022419 patients
Phase 3Terminated
NCT05642468IpsenRitivixibat

Safety and Tolerability of A3907 in Primary Sclerosing Cholangitis

Start: Jan 2023Est. completion: Jul 202518 patients
Phase 2Completed
NCT05627362IpsenElafibranor 80 mg

A Study to Assess Safety and Effectiveness of Elafibranor in Adult Participants With Primary Sclerosing Cholangitis.

Start: Dec 2022Est. completion: Aug 202668 patients
Phase 2Active Not Recruiting
NCT04663308Mirum PharmaceuticalsVolixibat

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)

Start: Dec 2020Est. completion: Apr 2027182 patients
Phase 2Active Not Recruiting
NCT04480840Pliant TherapeuticsPLN-74809

Phase 2a Evaluation of Safety, Tolerability, and Pharmacokinetics of PLN-74809 in Patients With Primary Sclerosing Cholangitis (PSC)

Start: Jul 2020Est. completion: Mar 2024121 patients
Phase 2Completed
NCT04024813Gilead SciencesSeladelpar

A Study to Evaluate the Safety, and Tolerability, and Efficacy of Seladelpar in Patients With PSC

Start: Nov 2019Est. completion: Jan 20201 patients
Phase 2Terminated
NCT00955149GenentechErlotinib

Erlotinib for Chemoprevention in Trisomy 7 Positive Primary Sclerosing Cholangitis (PSC)

Start: Aug 2009Est. completion: Apr 20136 patients
Phase 1Completed
NCT02736708Mauna Kea Technologiesprobe based confocal laser endomicroscopy

Registry Trial to Determine pCLE Image Interpretation Criteria and Preliminary Accuracy for PSC Biliary Strictures

Start: Jan 2013Est. completion: May 2020
N/ACompleted

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 late-stage (Phase 3) programs — potential near-term approvals
6 companies competing in this space