VivoSeq
Metabolic & Endocrine

Cerebrotendinous Xanthomatosis

6

Pipeline Programs↗

0

Active Trials↗

3

Companies Involved↗

Cerebrotendinous Xanthomatosis is an early-stage landscape with 6 programs across 3 companies. The most active players are Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA), Mirum Pharmaceuticals, Inc., Leadiant Biosciences, Inc.. 4 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

29/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

67/100

Attrition Rate

20/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

C

Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

1 program

Phase 2M

Mirum Pharmaceuticals, Inc.

4 programs

Phase 3L

Leadiant Biosciences, Inc.

1 program

Preclinical

Pipeline

Programs by Phase

Analysis

Trial Status

5

clinical trials

Unknown status2 · 40%
Completed2 · 40%
Withdrawn1 · 20%

Clinical Trials

Trial Activity

Evaluation of Carotid IMT and Atherogenic Risk Factors in Patients With Cerebrotendinous Xanthomatosis
Unknown status·SSheba Medical Center·N/A·NCT01613898·17 enrolled
Safety and Efficacy of CDCA in CTX Chenodeoxycholic Acid (CDCA) in Patients Affected by Cerebrotendinous Xanthomatosis (CTX)
Completed·LLeadiant Biosciences, Inc.·N/A·NCT05499026·28 enrolled
A Study of Chenodeoxycholic Acid (CDCA) in Newly Diagnosed Participants With Cerebrotendinous Xanthomatosis (CTX)
Withdrawn·LLeadiant Biosciences, Inc.·Phase 3·NCT06260748
Phase II Study of Cholesterol- and Cholestanol-Free Diet, Lovastatin, and Chenodeoxycholic Acid for Cerebrotendinous Xanthomatosis
Unknown status·NNational Center for Research Resources (NCRR)·Phase 2·NCT00004346·5 enrolled
Study to Evaluate Patients With Cerebrotendinous Xanthomatosis (RESTORE)
Completed·MMirum Pharmaceuticals, Inc.·Phase 3·NCT04270682·19 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)