VivoSeq
Neurology

Niemann-Pick Disease

7

Pipeline Programs↗

0

Active Trials↗

6

Companies Involved↗

Niemann-Pick disease is an early-stage landscape with 7 programs across 6 companies. The most active players are Sanofi, Cidara Therapeutics Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA), Medexus Pharma, Inc.. 2 therapies are approved.

Intelligence

Pipeline Metrics

Competitive Density

31/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

57/100

Attrition Rate

43/100

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.

Sanofi logo

Sanofi

1 program

Phase 1C

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

1 program

Phase 1M

Medexus Pharma, Inc.

1 program

Phase 2V

Vtesse, LLC, a Mallinckrodt Pharmaceuticals Company

1 program

Phase 2Akero Therapeutics logo

Akero Therapeutics

1 program

ApprovedGSK logo

GSK

1 program

Preclinical

Pipeline

Programs by Phase

Analysis

Trial Status

7

clinical trials

Completed4 · 57%
Terminated3 · 43%

Clinical Trials

Trial Activity

7 trials

N-Acetyl-L-Leucine for Niemann-Pick Disease, Type C (NPC)
Completed·IIntraBio Inc·Phase 2·NCT03759639·33 enrolled
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
Completed·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·Phase 1/2·NCT00975689·35 enrolled
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Completed·WWashington University School of Medicine·Phase 1/2·NCT03471143·4 enrolled
Phase 1/2 Study of Vorinostat Therapy in Niemann-Pick Disease, Type C1
Completed·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·Phase 1/2·NCT02124083·12 enrolled
Safety Study of rhASM Enzyme Replacement Therapy in Adults With Acid Sphingomyelinase Deficiency (Niemann-Pick Disease)
Terminated·Sanofi logoSanofi·Phase 1·NCT00410566·11 enrolled
Combined Intrathecal and Intravenous VTS-270 Therapy for Liver and Neurological Disease Associated With Niemann-Pick Disease, Type C1
Terminated·Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)·Phase 1/2·NCT03887533·2 enrolled
Screening of Niemann-Pick Disease, Type C in a Psychiatric Population
Terminated·UUniversity Hospital, Grenoble·N/A·NCT02841358·22 enrolled

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