VivoSeq
Neurology

Myotonic Dystrophy Type 1

14

Pipeline Programs↗

8

Active Trials↗

12

Companies Involved↗

Myotonic Dystrophy Type 1 is an emerging landscape with 14 programs across 12 companies. The most active players are Expansion Therapeutics, Inc., Viatris Specialty LLC, Menarini International Operations Luxembourg SA. 2 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

44/100

Pipeline Velocity

19/100

Mech. Concentration

20/100

Completion Rate

93/100

Attrition Rate

5/100

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.

E

Expansion Therapeutics, Inc.

1 program

Phase 1V

Viatris Specialty LLC

1 program

Phase 1M

Menarini International Operations Luxembourg SA

1 program

Phase 1A

Avidity Biosciences, Inc.

1 program

Phase 2H

Harmony Biosciences Management, Inc.

1 program

Phase 2Ionis Pharmaceuticals logo

Ionis Pharmaceuticals

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Trial Status

22

clinical trials

Completed13 · 59%
Recruiting6 · 27%
Not yet recruiting1 · 5%
Enrolling by invitation1 · 5%
Withdrawn1 · 5%

Clinical Trials

Trial Activity

22 trials

The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
Recruiting·LLupin Ltd.·Phase 3·NCT06523400·176 enrolled
Evaluation of the Efficacy and Safety of Metformin in the Myotonic Dystrophy Type 1 (Steinert's Disease)
Recruiting·AAssistance Publique - Hôpitaux de Paris·Phase 3·NCT05532813·142 enrolled
An Open-Label Extension Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1 (FREEDOM-OLE)
Recruiting·PPepGen Inc·Phase 2·NCT07220603·48 enrolled
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Recruiting·PPepGen Inc·Phase 2·NCT06667453·24 enrolled
A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 50 Years of Age With Non-congenital Myotonic Dystrophy Type 1
Recruiting·Sanofi logoSanofi·Phase 1/2·NCT06844214·32 enrolled
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
Recruiting·DDyne Therapeutics·Phase 1/2·NCT05481879·116 enrolled
Calcium Channel Blocker in Myotonic Dystrophy Type 1
Not yet recruiting·UUniversity of Rochester·Phase 1·NCT07075965·20 enrolled
An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.
Enrolling by invitation·LLupin Ltd.·Phase 3·NCT06549400·176 enrolled
Safety, Tolerability, PK, and PD Study of PGN-EDODM1 in Participants With Myotonic Dystrophy Type 1
Completed·PPepGen Inc·Phase 1·NCT06204809·24 enrolled
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
Completed·IInsmed Incorporated·Phase 2·NCT00577577·69 enrolled
Methylphenidate in Myotonic Dystrophy Type 1
Completed·LLaval University·Phase 2/3·NCT01421992·28 enrolled
Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1
Completed·EExpansion Therapeutics, Inc.·Phase 1·NCT03959189·12 enrolled
Study of Ranolazine in Myotonia Congenita, Paramyotonia Congenita and Myotonic Dystrophy Type 1
Completed·OOhio State University·Phase 1·NCT02251457·35 enrolled
Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients
Completed·AAvidity Biosciences, Inc.·Phase 2·NCT05479981·37 enrolled
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
Completed·HHarmony Biosciences Management, Inc.·Phase 2·NCT04886518·30 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)