VivoSeq
Neurology

Muscular Dystrophy

24

Pipeline Programs↗

5

Active Trials↗

15

Companies Involved↗

Muscular Dystrophy is an emerging landscape with 24 programs across 15 companies. The most active players are Santhera Pharmaceuticals, Sanofi, Bioblast Pharma Ltd.. 3 therapies are approved with 7 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

50/100

Pipeline Velocity

11/100

Mech. Concentration

17/100

Completion Rate

64/100

Attrition Rate

27/100

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.

S

Santhera Pharmaceuticals

1 program

Phase 1Sanofi logo

Sanofi

1 program

Phase 1B

Bioblast Pharma Ltd.

1 program

Phase 2A

aTyr Pharma, Inc.

1 program

Phase 2Pfizer logo

Pfizer

1 program

Phase 2S

Seelos Therapeutics, Inc.

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

87%

Small molecule

20 of 23 classified programs — the dominant modality here.

13%ALL OTHERSCell therapy29%Gene therapy14%

Small molecule leads; the ring shows all 23 classified programs, each share of the total. Modality classified for 97% of 29 pipeline programs.

Analysis

Trial Status

30

clinical trials

Completed14 · 47%
Withdrawn5 · 17%
Recruiting3 · 10%
Terminated3 · 10%
Active, not recruiting2 · 7%
Unknown status2 · 7%
Other1 · 3%

Clinical Trials

Trial Activity

30 trials

Givinostat in Duchenne's Muscular Dystrophy Long-term Safety and Tolerability Study
Recruiting·Italfarmaco logoItalfarmaco·Phase 2/3·NCT03373968·206 enrolled
A Prospective, Observational Study Evaluating the Real-World Experience of Givinostat in Patients With Duchenne Muscular Dystrophy
Recruiting·IITF Therapeutics LLC·N/A·NCT07127978·300 enrolled
Multiparametric Ultrafast Ultrasound Evaluation of Skeletal Muscle in Duchenne and Becker Muscular Dystrophies
Recruiting·NNantes University Hospital·N/A·NCT07378553·60 enrolled
Study to Evaluate the Efficacy and Safety of BBP-418 (Ribitol) in Patients With Limb Girdle Muscular Dystrophy 2I (LGMD2I)
Active, not recruiting·MML Bio Solutions, Inc.·Phase 3·NCT05775848·81 enrolled
A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
Active, not recruiting·Sarepta Therapeutics logoSarepta Therapeutics·Phase 3·NCT06246513·17 enrolled
L-citrulline and Metformin in Becker's Muscular Dystrophy
Completed·UUniversity Hospital, Basel, Switzerland·Phase 2·NCT02018731·20 enrolled
Assessing Air Travel Safety in Neuromuscular Dystrophy: Standard Versus Prolonged High Altitude Simulation Tests
Completed·TThe Hospital for Sick Children·N/A·NCT03312361·23 enrolled
Sodium Nitrate for Muscular Dystrophy
Completed·—·Phase 1·NCT02434627·5 enrolled
Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C
Completed·GGenethon·Phase 1·NCT01344798·9 enrolled
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
Completed·BBioblast Pharma Ltd.·Phase 2·NCT02015481·25 enrolled
Gentamicin Treatment of Muscular Dystrophy
Completed·National Institute of Neurological Disorders and Stroke (NINDS)·Phase 1·NCT00005574·4 enrolled
Congenital Muscular Dystrophy Ascending Multiple Dose Cohort Study Analyzing Pharmacokinetics at Three Dose Levels In Children and Adolescents With Assessment of Safety and Tolerability of Omigapil (CALLISTO)
Completed·SSanthera Pharmaceuticals·Phase 1·NCT01805024·20 enrolled
Assessment of Response to Treatment of Osteoporosis With Oral Bisphosphonates in Patients With Muscular Dystrophy
Completed·—·Phase 4·NCT01882400·11 enrolled
Weekly Steroids in Muscular Dystrophy
Completed·NNorthwestern University·Phase 2·NCT04054375·20 enrolled
L-citrulline and Metformin in Duchenne's Muscular Dystrophy
Completed·UUniversity Hospital, Basel, Switzerland·Phase 3·NCT01995032·47 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)