VivoSeq
Other

Myopathy

37

Pipeline Programs↗

9

Active Trials↗

25

Companies Involved↗

Myopathy is a moderately active landscape with 37 programs across 25 companies. The most active players are Leadiant Biosciences, Inc., CSL Behring, Roche. 8 therapies are approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

56/100

Pipeline Velocity

22/100

Mech. Concentration

22/100

Completion Rate

57/100

Attrition Rate

30/100

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.

L

Leadiant Biosciences, Inc.

1 program

Phase 1CSL Behring logo

CSL Behring

1 program

Phase 1Roche logo

Roche

1 program

Phase 1E

Eastern Cooperative Oncology Group

1 program

Phase 1P

Prism Pharmaceuticals

1 program

Phase 1Pfizer logo

Pfizer

2 programs

Phase 1

Pipeline

Programs by Phase

Preclinical4

Thioctic Acid

MEDA Pharma GmbH & Co. KG

Individual-Specific Statin Causing Myopathy

London Health Sciences Centre Research Institute OR Lawson Research Institute of St. Joseph's

Coenzyme Q10

Mclean Hospital

Salbutamol

GSK

Phase 18

Immune Globulin

CSL Behring

Rituximab

Roche

Filgrastim

Eastern Cooperative Oncology Group

Methylprednisolone

Pfizer

Mannac

Leadiant Biosciences, Inc.

Phase 216

N-Acetylcysteine

Novartis

Albumin

Laboratoire français de Fractionnement et de Biotechnologies

Omaveloxolone

Biogen

Bezafibrate

Intercept Pharmaceuticals

Zilucoplan

UCB

Phase 31

Dantrolene

Aultman Health Foundation

Approved8

Acetylcysteine

Arbor Pharmaceuticals, Inc.

Cyclophosphamide

Baxter Healthcare Corporation

Mesna

Baxter Healthcare Corporation

Prednisolone

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

Simvastatin

Organon and Co

Analysis

Modality Breakdown

85%

Small molecule

33 of 39 classified programs — the dominant modality here.

15%ALL OTHERSProtein / enzyme38%Monoclonal antibody25%Peptide13%

Small molecule leads; the ring shows all 39 classified programs, each share of the total. Modality classified for 100% of 41 pipeline programs.

Analysis

Trial Status

43

clinical trials

Completed17 · 40%
Terminated10 · 23%
Unknown status4 · 9%
Recruiting4 · 9%
Active, not recruiting3 · 7%
Withdrawn3 · 7%
Other2 · 5%

Clinical Trials

Trial Activity

43 trials

Ublituximab in Autoantibody Positive Immune Mediated Necrotizing Myopathy
Recruiting·National Institute of Allergy and Infectious Diseases (NIAID)·Phase 2·NCT07103746·30 enrolled
The MIGHT Trial - An Exploratory Clinical Trial of IVIG in Anti-HMGCR Immune Mediated Necrotizing Myopathy
Recruiting·UUniversity of Alabama at Birmingham·Phase 2·NCT06599697·12 enrolled
Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators
Recruiting·Astellas logoAstellas·Phase 1/2·NCT07052929·9 enrolled
WiTNNess: An International Natural History Study of Autosomal Recessive TNNT1 Myopathy
Recruiting·CClinic for Special Children·N/A·NCT06374719·40 enrolled
A Study to Evaluate Vitamin B3 Derivative to Treat Mitochondrial Myopathy
Active, not recruiting·—·Phase 2·NCT05590468·34 enrolled
Multi-Center Study of ManNAc for GNE Myopathy
Active, not recruiting·LLeadiant Biosciences, Inc.·Phase 2·NCT04231266·54 enrolled
Investigating Muscle Failure in Diabetic Myopathy
Active, not recruiting·UUniversity of Aarhus·N/A·NCT05685927·108 enrolled
A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.
Not yet recruiting·CCure ADSSL1·Phase 1·NCT07412821·2 enrolled
Dantrolene in Statin-induced Myopathy
Not yet recruiting·TTanta University·Phase 2/3·NCT06966843·23 enrolled
A Phase 2 Study to Evaluate the Dose and Pharmacodynamic Efficacy of Sialic Acid-Extended Release (SA-ER) Tablets in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy
Completed·Ultragenyx logoUltragenyx·Phase 2·NCT01517880·46 enrolled
An Open Label Phase 2 Study of ManNAc in Subjects With GNE Myopathy
Completed·NNational Human Genome Research Institute (NHGRI)·Phase 2·NCT02346461·12 enrolled
An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients
Completed·RReneo Pharma Ltd·Phase 2·NCT04535609·213 enrolled
An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy
Completed·Ultragenyx logoUltragenyx·Phase 2·NCT01830972·59 enrolled
Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
Completed·Ultragenyx logoUltragenyx·Phase 3·NCT02377921·89 enrolled
Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy
Completed·NNational Human Genome Research Institute (NHGRI)·Phase 1·NCT00195637·4 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)