VivoSeq
Neurology

Myotonic Dystrophy

4

Pipeline Programs↗

3

Active Trials↗

4

Companies Involved↗

Myotonic Dystrophy is an early-stage landscape with 4 programs across 4 companies. The most active players are Vertex Pharmaceuticals, Arrowhead Pharmaceuticals, AMO Pharma Limited. 1 therapy is approved with 2 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

28/100

Pipeline Velocity

0/100

Mech. Concentration

100/100

Completion Rate

100/100

Attrition Rate

0/100

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.

Vertex Pharmaceuticals logo

Vertex Pharmaceuticals

1 program

Phase 2Arrowhead Pharmaceuticals logo

Arrowhead Pharmaceuticals

1 program

Phase 2A

AMO Pharma Limited

1 program

Phase 3Boehringer Ingelheim logo

Boehringer Ingelheim

1 program

Approved

Pipeline

Programs by Phase

Analysis

Trial Status

8

clinical trials

Completed4 · 50%
Recruiting3 · 38%
Available1 · 13%

Clinical Trials

Trial Activity

8 trials

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
Recruiting·Vertex Pharmaceuticals logoVertex Pharmaceuticals·Phase 1/2·NCT06185764·44 enrolled
Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy
Recruiting·Arrowhead Pharmaceuticals logoArrowhead Pharmaceuticals·Phase 1/2·NCT06138743·78 enrolled
Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
Recruiting·AAMO Pharma Limited·Phase 2/3·NCT05004129·76 enrolled
Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy
Completed·AAMO Pharma Limited·Phase 2·NCT02858908·16 enrolled
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Completed·AAMO Pharma Limited·Phase 2/3·NCT03692312·56 enrolled
Efficacy and Safety of DHEA for Myotonic Dystrophy
Completed·UUniversity of Versailles·Phase 2/3·NCT00167609·75 enrolled
An Observational Study to Describe the Long-term Safety and Effectiveness of Namuscla in the Symptomatic Management of Myotonia in Adult Patients With Non-dystrophic Myotonic Disorders
Completed·LLupin Ltd.·N/A·NCT04616807·53 enrolled
Tideglusib: Expanded Access Use in Congenital Myotonic Dystrophy
Available·AAMO Pharma Limited·N/A·NCT07119775

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