Skip to main content

Myotonic Dystrophy 1

3
Pipeline Programs
8
Companies
7
Clinical Trials
3 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

7 companies ranked by most advanced pipeline stage

AMO Pharma
AMO PharmaNC - Durham
1 program
1
TideglusibPhase 21 trial
Active Trials
NCT02858908Completed16Est. Jan 2018
Arrowhead Pharmaceuticals
1 program
1
ARO-DM1Phase 1/21 trial
Active Trials
NCT06138743Recruiting78Est. Dec 2026
ARTHEx Biotech
ARTHEx BiotechSpain - Paterna
1 program
1
ATX-01Phase 1/21 trial
Active Trials
NCT06300307Recruiting56Est. Jul 2027
Angeles Therapeutics
Angeles TherapeuticsCA - Los Angeles
1 program
Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)N/A1 trial
Active Trials
NCT03981575Recruiting700Est. Dec 2026
Rare Disease Therapeutics
1 program
Training programN/A1 trial
Active Trials
NCT05400629Completed12Est. Jan 2023
Life & Brain
Life & BrainGermany - Bonn
1 program
medical historyN/A1 trial
Active Trials
NCT02729597Completed49Est. Aug 2015
Harmony Biosciences
Harmony BiosciencesPLYMOUTH MEETING, PA
1 program
Pitolisant Oral TabletPHASE_21 trial
Active Trials
NCT04886518Completed30Est. Oct 2024

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
Harmony BiosciencesPitolisant Oral Tablet
AMO PharmaTideglusib
ARTHEx BiotechATX-01
Arrowhead PharmaceuticalsARO-DM1
Rare Disease TherapeuticsTraining program
Angeles TherapeuticsEstab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)
Life & Brainmedical history

Clinical Trials (7)

Total enrollment: 941 patients across 7 trials

NCT04886518Harmony BiosciencesPitolisant Oral Tablet

Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1

Start: Jun 2021Est. completion: Oct 202430 patients
Phase 2Completed
NCT02858908AMO PharmaTideglusib

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Start: Jul 2016Est. completion: Jan 201816 patients
Phase 2Completed
NCT06300307ARTHEx BiotechATX-01

Study of ATX-01 in Participants With DM1

Start: Oct 2024Est. completion: Jul 202756 patients
Phase 1/2Recruiting
NCT06138743Arrowhead PharmaceuticalsARO-DM1

Study of ARO-DM1 in Subjects With Type 1 Myotonic Dystrophy

Start: Mar 2024Est. completion: Dec 202678 patients
Phase 1/2Recruiting
NCT05400629Rare Disease TherapeuticsTraining program

Effects of a 12-week Strength Training Program in Women With Myotonic Dystrophy Type 1

Start: Jan 2022Est. completion: Jan 202312 patients
N/ACompleted
NCT03981575Angeles TherapeuticsEstab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Estab Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1)

Start: Jan 2019Est. completion: Dec 2026700 patients
N/ARecruiting
NCT02729597Life & Brainmedical history

Tracking the Brain in Myotonic Dystrophies: a 5-year Longitudinal Follow-up Study

Start: May 2007Est. completion: Aug 201549 patients
N/ACompleted

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

3 actively recruiting trials targeting 941 patients
8 companies competing in this space

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.