Myotonic Dystrophy Type 1 (DM1)

3
Pipeline Programs
2
Companies
3
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

2 companies ranked by most advanced pipeline stage

VP
2 programs
1
1
VX-670Phase 21 trial
VX-670Phase 1/21 trial
Active Trials
NCT06185764Recruiting44Est. Dec 2026
NCT06926621Enrolling By Invitation44Est. Jan 2029
Dyne Therapeutics
1 program
1
DYNE-101Phase 1/21 trial
Active Trials
NCT05481879Recruiting116Est. Jul 2029

Trial Timeline

Clinical trial activity over time

2022
2023
2024
2025
2026
2027
2028
2029
Vertex PharmaceuticalsVX-670
Vertex PharmaceuticalsVX-670
Dyne TherapeuticsDYNE-101

Clinical Trials (3)

Total enrollment: 204 patients across 3 trials

NCT06926621Vertex PharmaceuticalsVX-670

A Study of Long-term Safety and Efficacy of VX-670 in Participants With Myotonic Dystrophy Type I

Start: Apr 2025Est. completion: Jan 202944 patients
Phase 2Enrolling By Invitation
NCT06185764Vertex PharmaceuticalsVX-670

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)

Start: Feb 2024Est. completion: Dec 202644 patients
Phase 1/2Recruiting
NCT05481879Dyne TherapeuticsDYNE-101

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1

Start: Sep 2022Est. completion: Jul 2029116 patients
Phase 1/2Recruiting

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

2 actively recruiting trials targeting 204 patients
2 companies competing in this space