VivoSeq
Neurology

Charcot-Marie-Tooth Disease

10

Pipeline Programs↗

3

Active Trials↗

7

Companies Involved↗

Charcot-Marie-Tooth disease is an early-stage landscape with 10 programs across 7 companies. The most active players are Merck & Co., Vanda Pharmaceuticals, NMD Pharma A/S. 2 therapies are approved.

Intelligence

Pipeline Metrics

Competitive Density

38/100

Pipeline Velocity

11/100

Mech. Concentration

50/100

Completion Rate

71/100

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.

Merck & Co. logo

Merck & Co.

1 program

Phase 2V

Vanda Pharmaceuticals

1 program

Phase 2N

NMD Pharma A/S

1 program

Phase 2F

Flex Pharma, Inc.

1 program

Phase 2Novartis logo

Novartis

1 program

ApprovedBoehringer Ingelheim logo

Boehringer Ingelheim

1 program

Approved

Pipeline

Programs by Phase

Analysis

Modality Breakdown

90%

Small molecule

9 of 10 classified programs — the dominant modality here.

10%ALL OTHERSCell therapy110%

Small molecule leads; the ring shows all 10 classified programs, each share of the total. Modality classified for 100% of 11 pipeline programs.

Analysis

Trial Status

10

clinical trials

Completed5 · 50%
Terminated2 · 20%
Active, not recruiting1 · 10%
Enrolling by invitation1 · 10%
Recruiting1 · 10%

Clinical Trials

Trial Activity

10 trials

A Phase 1/2 Open-Label Study to Evaluate the Safety, Tolerability and Efficacy of a Single Dose of ELP-02 Delivered Via Lumbar Intrathecal Administration in Charcot-Marie-Tooth-4J (CMT4J)
Recruiting·EElpida Therapeutics SPC·Preclinical·NCT07447557·8 enrolled
Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient
Active, not recruiting·VVanda Pharmaceuticals·Phase 1/2·NCT07223632·1 enrolled
Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)
Enrolling by invitation·Nn-Lorem Foundation·Phase 1/2·NCT07226297·1 enrolled
Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A
Completed·EENCell·Phase 1·NCT05333406·9 enrolled
Evaluate the Safety and Potential Efficacy of Human Wharton's Jelly-derived Mesenchymal Stem Cells With Charcot-Marie-Tooth Disease Type 1E
Completed·SSamsung Medical Center·N/A·NCT06218134·3 enrolled
Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease
Completed·NNMD Pharma A/S·Phase 2·NCT06482437·81 enrolled
Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease
Completed·—·Phase 1/2·NCT00541164·23 enrolled
The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
Completed·HHelixmith Co., Ltd.·Phase 1/2·NCT05361031·12 enrolled
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
Terminated·FFlex Pharma, Inc.·Phase 2·NCT03254199·27 enrolled
Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease
Terminated·Merck & Co. logoMerck & Co.·Phase 2·NCT03124459·63 enrolled

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