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Disease

Myositis

Late-stage therapeutic developmentEmerging research
MeSHMyositis
4
Publications
19
Clinical trials
12
Associated genes
2
Related proteins
2024
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

A Phase 1, Multicenter, Open-Label Study Of CC-97540 (BMS-986353), CD19-Targeted Nex-T Chimeric Antigen Receptor (CAR) T Cells, in Participants With Severe, Refractory Autoimmune Diseases: Systemic Lupus Erythematosus, Idiopathic Inflammatory Myopathy, Systemic Sclerosis, or Rheumatoid Arthritis (Breakfree-1)

Clinical trial2026-08-03Status changed to Active, not recruiting · ClinicalTrials.gov

A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group, 2-Arm, Multicenter, Operationally Seamless Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Participants Aged 18 Years and Older With Active Idiopathic Inflammatory Myopathy

Clinical trial2026-07-24Status changed to Completed · ClinicalTrials.gov

Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial.

Research2024-09-07Molecular therapy : the journal of the American Society of Gene Therapy

CD19 CAR T-Cell Therapy in Autoimmune Disease - A Case Series with Follow-up.

Research2024-02-01The New England journal of medicine

Rescue therapy of antisynthetase syndrome with CD19-targeted CAR-T cells after failure of several B-cell depleting antibodies.

Research2024-01-01Rheumatology (Oxford, England)

Immune checkpoint inhibitor-related myositis and myocarditis: diagnostic pitfalls and imaging contribution in a real-world, institutional case series.

Research2023-12-23Journal of neurology

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate
Key developments
  • 4 clinical trials expected to report results, the earliest in Q4 2026.
Upcoming Milestones
Clinical Milestones8View
Results expected 4
Recently completed 3
Other updates 1
Activity timeline8

Clinical trials

16 sponsors · 2 new · 2 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme
19
All trials
6
Active
12
Late-stage
6
Completed
Late-stage studies

A Phase 2-Phase 3, Double-blinded, Randomized, Dose-repeating, Cross-over Study to Assess the Safety and Efficacy of Allogeneic ULSC on Disease Severity and Steroid Tapering in Participants With Dermatomyositis/ Polymyositis (DM/PM)

Phase 2/3 · Recruiting · Restem, LLC.

An Open-label, Multicenter Study to Evaluate the Long-term Safety and Efficacy of Ulviprubart (ABC008) in Subjects Who Have Completed a Trial of Ulviprubart for the Treatment of Inclusion Body Myositis

Phase 2/3 · Active, not recruiting · Abcuro, Inc.

A PHASE 3, MULTICENTER, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF PF-06823859 IN PARTICIPANTS WITH ACTIVE IDIOPATHIC INFLAMMATORY MYOPATHIES (INCLUDING PARTICIPANTS WITH ACTIVE DERMATOMYOSITIS OR POLYMYOSITIS)

Phase 3 · Recruiting · Pfizer

Treatment With add-on IVIg in Myositis Early In the diSease Course May be sUperior to Steroids Alone for Reaching CLinical improvemEnt

Phase 2/3 · Active, not recruiting · Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)

A Double-Blind Randomised Controlled Trial (dbRCT) Phase III Trial Investigating the Effect of Sirolimus on Disease Progression in Patients With Inclusion Body Myositis (IBM) as Measured by the IBM Functional Rating Scale (IBM-FRS)

Phase 3 · Active, not recruiting · University of Kansas Medical Center

Nintedanib Plus Standard of Care Immunosuppression Versus Standard of Care Immunosuppression Alone in Patients With Progressive Fibrotic Myositis Associated - Interstitial Lung Disease: A Randomized, Double-Blind, Exploratory Trial

Phase 4 · Completed · Rohit Aggarwal, MD
Recently completed

A Phase II/III Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of ABC008 in the Treatment of Subjects With Inclusion Body Myositis

Phase 2/3 · Completed · Abcuro, Inc.

Nintedanib Plus Standard of Care Immunosuppression Versus Standard of Care Immunosuppression Alone in Patients With Progressive Fibrotic Myositis Associated - Interstitial Lung Disease: A Randomized, Double-Blind, Exploratory Trial

Phase 4 · Completed · Rohit Aggarwal, MD

Belimumab for Maintenance Therapy in Idiopathic Inflammatory Myositis

Phase 2/3 · Completed · Northwell Health

A Phase 3, Randomized, Double-Blind Clinical Trial to Evaluate the Efficacy and Safety of Abatacept SC With Standard Treatment Compared to Standard Treatment Alone in Improving Disease Activity in Adults With Active Idiopathic Inflammatory Myopathy (IIM)

Phase 3 · Completed · Bristol-Myers Squibb

Étude de l'Effet de la Rapamycine Sur la Force Musculaire et la réponse Immunitaire au Cours de la Myosite à Inclusions: étude RAPAMI"

Phase 2/3 · Completed · Institut National de la Santé Et de la Recherche Médicale, France

Extension of the CBYM338B2203 Phase IIb/III Study to Evaluate the Long-term Efficacy, Safety and Tolerability of Intravenous BYM338 in Patients With Sporadic Inclusion Body Myositis

Phase 3 · Completed · Novartis Pharmaceuticals

Research activity

4 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Major themes8
  • Myositis4
  • Antigens, CD192
  • Immunotherapy, Adoptive2
  • Antineoplastic Agents, Immunological1
  • Lupus Erythematosus, Systemic1
  • Myeloablative Agonists1
  • Myocarditis1
  • Receptors, Antigen, T-Cell1
Leading journals4
  • Journal of neurology1
  • Molecular therapy : the journal of the American Society of Gene Therapy1
  • Rheumatology (Oxford, England)1
  • The New England journal of medicine1
Leading researchers8
  • Aigner M2
  • Knitza J2
  • Kretschmann S2
  • Mackensen A2
  • Müller F2
  • Schett G2
  • Taubmann J2
  • Völkl S2
Affiliations (unnormalised)6
  • and the Institute of Clinical Microbiology1
  • Department of Clinical Neurosciences1
  • Friedrich-Alexander-Universität Erlangen-Nürnberg and Universitätsklinikum Erlangen1
  • Institute of Radiology1
  • Lausanne University Hospital and University of Lausanne1
  • Lundin Family Brain Tumor Center1

Associated genes

12 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

NCF20.33FCGRT0.32DGKQ0.28SDK20.27IFNB10.26YDJC0.19IRF70.14SMC40.14CCDC1160.08CDHR50.07SNRNP700.07UCN20.05

Disease biology

2 matches

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

IgG receptor FcRn large subunit p51via FCGRT0.32Interferon regulatory factor 7via IRF70.14

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Inflammation of a muscle or muscle tissue.

Identifiers
MeSHMyositis
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.