DM1
1
Pipeline Programs
3
Companies
3
Clinical Trials
1 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
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
3 companies ranked by most advanced pipeline stage
Vertex PharmaceuticalsBOSTON, MA
1 programEstablishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) ExtensionN/A
Dyne TherapeuticsWALTHAM, MA
1 programEstablishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) ExtensionN/A1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Avidity BiosciencesAOC 1001
Avidity BiosciencesAOC 1001
Dyne TherapeuticsEstablishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Clinical Trials (3)
Total enrollment: 1,269 patients across 3 trials
Global Open-Label Extension Study of Del-desiran for the Treatment of DM1
Start: Jun 2025Est. completion: Oct 2030230 patients
Phase 3Enrolling By Invitation
Study of AOC 1001 in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Start: Oct 2021Est. completion: Feb 202339 patients
Phase 1/2Completed
NCT07700225Dyne TherapeuticsEstablishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Extension
Start: Jul 2026Est. completion: Dec 20321,000 patients
N/ARecruiting
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
1 actively recruiting trials targeting 1,269 patients
3 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.