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Myotonic Dystrophy Type 1

1
Pipeline Programs
4
Companies
2
Clinical Trials
0
Approved Products

Pipeline by Development Stage

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

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Avidity Biosciences
Avidity BiosciencesCA - San Diego
1 program
1
del-desiranPhase 3
Ionis Pharmaceuticals
1 program
IONIS-DMPKRxPHASE_1_21 trial
Active Trials
NCT02312011Completed48Est. Aug 2016
Insmed
InsmedBRIDGEWATER, NJ
1 program
rhIGF-I/rhIGFBP-3PHASE_21 trial
Active Trials
NCT00577577Completed69Est. Dec 2008

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
InsmedrhIGF-I/rhIGFBP-3
Ionis PharmaceuticalsIONIS-DMPKRx

Clinical Trials (2)

Total enrollment: 117 patients across 2 trials

NCT00577577InsmedrhIGF-I/rhIGFBP-3

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Start: Dec 2007Est. completion: Dec 200869 patients
Phase 2Completed
NCT02312011Ionis PharmaceuticalsIONIS-DMPKRx

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

Start: Dec 2014Est. completion: Aug 201648 patients
Phase 1/2Completed

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 late-stage (Phase 3) programs, potential near-term approvals
4 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.