VivoSeq
Rare Disease

Duchenne Muscular Dystrophy

112

Pipeline Programs↗

40

Active Trials↗

50

Companies Involved↗

Duchenne Muscular Dystrophy is a highly competitive landscape with 112 programs across 50 companies. The most active players are Astellas, Validus Pharmaceuticals, EMD Serono. 5 therapies are approved with 28 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

73/100

Pipeline Velocity

30/100

Mech. Concentration

7/100

Completion Rate

66/100

Attrition Rate

23/100

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.

Astellas logo

Astellas

1 program

Phase 1V

Validus Pharmaceuticals

1 program

Phase 1EMD Serono logo

EMD Serono

1 program

Phase 1H

Hansa Biopharma AB

1 program

Phase 1T

Translational Biosciences

1 program

Phase 1Mario Negri Institute for Pharmacological Research logo

Mario Negri Institute for Pharmacological Research

1 program

Phase 1

Pipeline

Programs by Phase

Preclinical13

Delandistrogene Moxeparvovec

Sarepta Therapeutics

Ataluren

PTC Therapeutics

Testosterone

Auxilium

Dexmedetomidine

McNeil Consumer & Specialty Pharmaceuticals, a Division of McNeil-PPC, Inc.

Ketamine

Aultman Health Foundation

Phase 117

Canakinumab

Novartis

RG6026

Roche

Pentoxifylline

Validus Pharmaceuticals

Metformin

EMD Serono

Imlifidase

Hansa Biopharma AB

Phase 249

Glutamine

Sanofi

SAR444245

Sanofi

Abbvie-Gp1692

AbbVie

VX-264

Vertex Pharmaceuticals

Prednisolone

Pfizer's Upjohn has merged with Mylan to form Viatris Inc.

Phase 328

Eteplirsen

Sarepta Therapeutics

Viltolarsen

Nippon Shinyaku Co., Ltd.

Givinostat

Italfarmaco

Casimersen

Sarepta Therapeutics

Idebenone

Santhera Pharmaceuticals

Approved5

Golodirsen

Sarepta Therapeutics

Gentamicin

Schering-Plough

Isosorbide Dinitrate

AstraZeneca

Ibuprofen

Kenvue Brands

Carvedilol

Akero Therapeutics

Analysis

Modality Breakdown

81%

Small molecule

88 of 109 classified programs — the dominant modality here.

19%ALL OTHERSRNA therapy66%Gene therapy66%Cell therapy55%Monoclonal antibody33%Protein / enzyme1<1%

Small molecule leads; the ring shows all 109 classified programs, each share of the total. Modality classified for 97% of 126 pipeline programs.

Analysis

Trial Status

168

clinical trials

Completed77 · 46%
Terminated32 · 19%
Active, not recruiting19 · 11%
Recruiting17 · 10%
Unknown status8 · 5%
Withdrawn7 · 4%
Other8 · 5%

Clinical Trials

Trial Activity

168 trials

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
Recruiting·EEntrada Therapeutics, Inc.·Phase 1/2·NCT07037862·24 enrolled
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD)
Recruiting·NS Pharma logoNS Pharma·Phase 2·NCT05996003·20 enrolled
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
Recruiting·Roche logoRoche·Phase 2·NCT06450639·50 enrolled
Evaluation of the Safety, Tolerability, and Efficacy of LE051 in Patients With Duchenne Muscular Dystrophy
Recruiting·SShanghai Jiao Tong University School of Medicine·Phase 1·NCT06900049·12 enrolled
Registry Study to Observe Long-term Safety of Vamorolone (AGAMREE®) in Patients With Duchenne Muscular Dystrophy-SUMMIT
Recruiting·CCatalyst Pharmaceuticals, Inc.·N/A·NCT06564974·250 enrolled
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE)
Recruiting·SSolid Biosciences Inc.·Phase 3·NCT07160634·80 enrolled
Efficacy, Safety and Tolerability of Givinostat in Non-ambulant Patients With Duchenne Muscular Dystrophy
Recruiting·Italfarmaco logoItalfarmaco·Phase 3·NCT05933057·138 enrolled
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
Recruiting·EEntrada Therapeutics, Inc.·Phase 1/2·NCT07038824·24 enrolled
Open-label Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy (FORWARD-53)
Recruiting·WWave Life Sciences Ltd.·Phase 1/2·NCT04906460·26 enrolled
Phase 1/2a for Safety, PK and PD of SQY51 in Paediatric and Adult Patients Duchenne Muscular Dystrophy
Recruiting·SSqy Therapeutics·Phase 1/2·NCT05753462·12 enrolled
A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Dose Comparison and Exploratory Efficacy Study of Orally Administered SAT-3247 in Ambulatory DMD Patients
Recruiting·SSatellos Bioscience, Inc.·Phase 2·NCT07287189·51 enrolled
A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Males With Duchenne Muscular Dystrophy (INSPIRE DUCHENNE)
Recruiting·SSolid Biosciences Inc.·Preclinical·NCT06138639·60 enrolled
A Phase 1/2/3 Open-label Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacodynamics, and Pharmacokinetics of Intravenous RGX-202 Gene Therapy in Males With Duchenne Muscular Dystrophy (DMD)
Recruiting·RREGENXBIO Inc.·Preclinical·NCT05693142·65 enrolled
Multicenter, Single-blind, Randomized, Placebo-controlled Study of a Single Intravenous Infusion of a Gene Therapy Product GNR-097 in Pediatric Patients With Duchenne Muscular Dystrophy
Recruiting·AAO GENERIUM·Preclinical·NCT07673809·32 enrolled
An Open-Label, Systemic Gene Delivery Study Using Commercial Process Material to Evaluate the Safety of and Expression From SRP-9001 in Subjects With Duchenne Muscular Dystrophy (ENDEAVOR)
Recruiting·Sarepta Therapeutics logoSarepta Therapeutics·Phase 1·NCT04626674·83 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)