Myasthenia Gravis - Pipeline Analysis 2019
- UNI4065855
- 121 Pages
- June 2019
- Healthcare
Myasthenia Gravis (MG) is the prototype neuromuscular disease with the immunological pathogenesis. It is an acquired autoimmune disease affecting synaptic transmission via the neuromuscular junction mainly due to the presence of autoantibodies against the acetylcholine receptors (AChR) between the synaptic space of the skeletal muscles. It leads to an impairment of the neuromuscular transmission and corresponding clinical symptoms such as fluctuating muscle weakness and fatigability.
Myasthenia gravis affects both men and women and most commonly impacts young adult women (under 40) and older men (over 60), but it can occur at any age, including childhood. The indication is diagnosed based upon a thorough clinical evaluation, detection of characteristic symptoms and physical findings, a detailed patient history and a variety of specialized tests. The diagnosis is suspected based on a characteristic distribution of muscle weakness and fatigue, without impairment of other neurologic function. The several tests to confirm the diagnosis include a physical and neurological examination, an edrophonium test, a blood test, electrodiagnostic, diagnostic imaging and pulmonary function testing.
The recommended treatment for MG may include various measures that may alleviate symptoms, including anticholinesterase drugs (cholinesterase inhibitors) or alter the disease course, such as immunosuppressive drugs or surgery (thymectomy). The initial treatment commonly includes the use of cholinesterase inhibitors, which increase muscle strength by preventing the normal breakdown of the neurotransmitter acetylcholine. Pyridostigmine by mouth (orally) is primarily used.
The therapeutic pipeline of Myasthenia Gravis consists of approximately 15+ products in different stages of development. Currently, 3+ drugs are in Phase III development and major drugs are in mid stage and pre-clinical stage.
Top Company Analysed
Some of the key players include Akari Therapeutics, Alpha Cancer Technologies, AnTolRx, Argenx, Catalyst Pharmaceuticals, CSL Behring, CuraVac, Grifols Therapeutics, GT Biopharma, Hibernaid, Immunovant (Roivant Sciences), Momenta Pharmaceuticals, Novartis, Protalex, Ra Pharmaceuticals, Toleranzia, Tolerion and UCB Pharma.
Scope of the report:
• Provides an overview of therapeutic pipeline activity for Myasthenia Gravis across the complete product development cycle including all clinical and non-clinical stages
• The report comprises of detailed profiles of Myasthenia Gravis therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical information
• Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration
• Detailed profiles of the dormant and discontinued products have been included in the report
Reasons to buy:
• The Myasthenia Gravis pipeline presents the detailed profile of drugs. The analysis offered in the report is a combination of deep dive secondary research and input from Key Opinion Leader of the industry
• The report presents a quick review of the current scenario regarding the drug development of the indication at one glance
• The report covers in-depth analysis of prominent industry peers with a primary focus on company consolidation, designation, technology, agreements and patents regarding the therapy
• Detailed examination on diagnosis, treatment and guidelines prevailing in the industry
• Examination of industry attractiveness with the help of approval timelines
• The study comprehensively covers the market across drugs in different phases of development
• Extensive domain knowledge on therapy areas support the client in decision-making process regarding their therapeutic portfolio by identifying the reason behind the dormancy and discontinuation of the products
Customization Options:
The Myasthenia Gravis pipeline analysis report can be customized to the country level or any other competitive segment. Besides this, UMI understands that you may have your own business need, hence we also provide fully customized solutions to clients.
1.1 Objective of the Study 12
1.2 Secondary Research 12
1.3 Scope of the report: 12
2. DISEASE OVERVIEW 13
2.1 Introduction 13
2.2 Classification of MG 14
2.2.1 Congenital MG 14
2.2.2 Ocular MG 14
2.2.3 Generalized MG 14
2.2.4 Transient Neonatal MG 14
2.2.5 Juvenile MG 15
2.3 Clinical Classification 17
2.4 Signs and Symptoms 18
2.5 Causes 20
2.6 Pathogenesis 22
2.7 Pathophysiology 24
2.7.1 Presynaptic Surface 24
2.7.2 Synaptic Cleft 25
2.7.3 Postsynaptic Surface 26
2.7.4 The Acetylcholine Receptor 27
2.7.5 Neuromuscular Junction Formation and Muscle-Specific Kinase 27
2.7.6 Safety Factor for Neuromuscular Transmission 28
2.7.7 Immunopathology 28
2.7.8 Autoantibodies in MG 29
2.7.9 Cellular Autoimmunity 30
2.7.10 Cytokine Influences 31
2.7.11 The Thymus in MG Pathogenesis 32
2.7.12 The Differential Involvement of Muscle Groups by MG 32
2.8 Diagnosis 34
2.8.1 Differential Diagnosis 35
2.9 Treatment 37
2.9.1 International Consensus guidance for management of Myasthenia Gravis 38
2.9.1.1 Development of guidance treatment statements 38
2.9.2 Myasthenia gravis: Association of British Neurologists’ Management Guidelines 44
2.9.2.1 International Consensus Guidance for Management of Myasthenia Gravis 44
2.9.2.2 Treatment of Generalized Myasthenia Gravis 46
2.9.2.3 Management of side effects: Propantheline or mebeverine can help cholinergic side effects 46
2.9.2.4 Assessment and Management of the relapsing patient with Myasthenia 49
2.9.3 Japanese Clinical Guidelines for Myasthenia Gravis 52
2.9.3.1 Recommendations 53
2.9.3.2 Thymectomy for non-thymomatous patients 54
2.9.3.2.1 Recommendations 54
2.9.3.3 Oral immunosuppressive therapy 55
2.9.3.3.1 Recommendations 55
2.9.3.4 High-dose Intravenous Methylprednisolone treatment 56
2.9.3.4.1 Recommendations 56
2.9.3.5 IVIg treatment 56
2.9.3.5.1 Recommendations 56
2.9.3.6 PE/PP 57
2.9.3.6.1 Recommendations 57
2.9.3.7 Treatment for anti-AChR antibody-negative patients (including MuSK-MG) 57
2.9.3.7.1 Recommendations 57
2.9.3.8 Treatment strategies for ocular MG 57
2.9.3.8.1 Recommendations 57
2.9.3.9 Treatment Strategies for LOMG and elderly MG patients 58
2.9.3.9.1 Recommendations 58
2.10 Treatment Algorithm 59
3. PIPELINE THERAPEUTICS AT A GLANCE 60
3.1 Pipeline Therapeutics at a Glance 60
4. COMPARATIVE ANALYSIS 61
4.1 Number of Products in Myasthenia Gravis 61
5. LATE PHASE PRODUCTS (PHASE III) 62
5.1 Comparative Analysis 62
5.2 Amifampridine Phosphate: Catalyst Pharmaceuticals 62
5.2.1 Product Description 62
5.2.2 Research and Development 63
5.2.2.1 Clinical Studies 63
5.2.2.1.1 Phase III 63
5.2.2.1.2 Phase II 63
5.2.2.1.3 Results of Analysis 64
5.2.3 Product Development Activities 64
5.2.3.1 Agreement 64
5.2.3.2 Designation 65
5.3 Efgartigimod: Argenx 66
5.3.1 Product Description 66
5.3.2 Research and Development 66
5.3.2.1 Clinical Studies 66
5.3.2.1.1 Phase III 66
5.3.2.1.2 Results of Analysis 67
5.3.3 Product Development Activities 68
5.3.3.1 Designation 68
5.3.3.2 Grant 68
5.3.3.3 Technology 68
5.4 Human normal immunoglobulin G (IgG): CSL Behring 69
5.4.1 Product Description 69
5.4.2 Research and Development 70
5.4.2.1 Clinical Studies 70
5.4.2.1.1 Phase III 70
5.4.2.1.2 Phase II 70
5.5 IGIV-C: Grifols Therapeutics 71
5.5.1 Product Description 71
5.5.2 Research and Development 72
5.5.2.1 Clinical Studies 72
5.5.2.1.1 Phase III 72
5.5.2.1.2 Phase II 72
6. MID PHASE PRODUCTS (PHASE II) 74
6.1 Comparative Analysis 74
6.2 Iscalimab: Novartis 74
6.2.1 Product Description 74
6.2.2 Research and Development 75
6.2.2.1 Pre-clinical Studies 75
6.2.2.2 Clinical Studies 75
6.2.2.2.1 Phase II 76
6.2.2.2.2 Results of Analysis 76
6.3 M281: Momenta Pharmaceuticals 77
6.3.1 Product Description 77
6.3.2 Research and Development 77
6.3.2.1 Clinical Studies 77
6.3.2.1.1 Phase II 77
6.3.2.1.2 Phase I 78
6.3.2.1.3 Results of Analysis 78
6.4 Rozanolixizumab: UCB Pharma 79
6.4.1 Product Description 79
6.4.2 Research and Development 80
6.4.2.1 Clinical Studies 80
6.4.2.1.1 Phase II 80
6.4.2.1.2 Results of Analysis 80
6.5 RVT-1401: Immunovant (Roivant Sciences) 82
6.5.1 Product Description 82
6.5.2 Research and Development 82
6.5.2.1 Clinical Studies 82
6.5.2.1.1 Phase II 82
6.5.3 Product Development Activities 82
6.5.3.1 Licensing Agreement 82
6.6 Zilucoplan: Ra Pharmaceuticals 83
6.6.1 Product Description 83
6.6.2 Research and Development 84
6.6.2.1 Clinical Studies 84
6.6.2.1.1 Phase II 84
6.6.2.1.2 Results of Analysis 84
6.6.3 Product Development Activities 85
6.6.3.1 Technology 85
6.6.3.2 Patent 85
6.7 Myasterix: CuraVac 87
6.7.1 Product Description 87
6.7.2 Research and Development 87
6.7.2.1 Clinical Studies 87
6.7.2.1.1 Phase I/II 87
6.7.2.1.2 Results of Analysis 87
6.7.3 Product Development Activities 88
6.7.3.1 Designation 88
6.7.3.2 Grant 88
7. EARLY STAGE PRODUCTS (PHASE I) 89
7.1 Comparative Analysis 89
7.2 GTP-004: GT Biopharma 89
7.2.1 Product Description 89
7.2.2 Research and Development 89
7.2.2.1 Clinical Studies 89
7.2.2.1.1 Phase I 89
7.2.2.1.2 Results of Analysis 90
7.2.3 Product Development Activities 90
7.2.3.1 Patents 90
8. PRE-CLINICAL AND DISCOVERY STAGE PRODUCTS 92
8.1 Comparative Analysis 92
8.2 ACT-101: Alpha Cancer Technologies 92
8.2.1 Product Description 92
8.2.2 Research and Development 93
8.2.2.1 Pre-clinical Studies 93
8.2.3 Product Development Activities 93
8.2.3.1 Designation 93
8.3 HBN-1 (IV): Hibernaid 94
8.3.1 Product Description 94
8.4 Myasthenia Gravis-Tolerogen: Toleranzia 95
8.4.1 Product Description 95
8.5 Pre-clinical Program: AnTolRx 96
8.5.1 Product Description 96
8.5.2 Product Development Activities 96
8.5.2.1 Financing 96
8.5.2.2 Technology 96
8.6 PRTX-100: Protalex 97
8.6.1 Product Description 97
8.6.2 Research and Development 97
8.6.2.1 Pre-clinical Studies 97
8.6.3 Product Development Activities 97
8.6.3.1 Patent 97
8.7 TOL 3034: Tolerion 98
8.7.1 Product Description 98
8.7.2 Product Development Activities 98
8.7.2.1 Technology 98
8.8 Coversin: Akari Therapeutics 99
8.8.1 Product Description 99
8.8.2 Product Development Activities 100
8.8.2.1 Patent 100
8.8.2.2 Financing 100
8.8.2.3 Merger Agreement 100
8.9 HBN-100: Hibernaid 101
8.9.1 Product Description 101
9. THERAPEUTIC ASSESSMENT 103
9.1 Assessment by Product Type 103
9.2 Assessment by Stage and Product Type 103
9.3 Assessment by Route of Administration 104
9.4 Assessment by Stage and Route of Administration 105
9.5 Assessment by Molecule Type 106
9.6 Assessment by Stage and Molecule Type 107
10. APPROVAL TIMELINES 108
10.1 Approval Timelines for Clinical Products 108
11. ANALYST INSIGHTS 110
12. Inactive Products 111
12.1 Comparative Analysis 111
12.2 Belimumab: GlaxoSmithKline 111
12.2.1 Product Description 111
12.2.2 Research and Development 112
12.2.2.1 Clinical Studies 112
12.2.2.1.1 Phase II 112
12.2.2.1.2 Results of Analysis 112
12.3 EN101: Amarin Corporation 114
12.3.1 Product Description 114
12.3.2 Research and Development 114
12.3.2.1 Clinical Studies 114
12.3.2.1.1 Phase IIa 114
12.3.2.1.2 Results of Analysis 114
12.3.3 Product Development Activities 115
12.3.3.1 Designation 115
12.3.3.2 Agreement 115
12.4 Tirasemtiv: Cytokinetics 116
12.4.1 Product Description 116
12.4.2 Research and Development 116
12.4.2.1 Clinical Studies 116
12.4.2.1.1 Phase II 116
12.4.2.1.2 Results of Analysis 116
12.4.3 Product Development Activities 117
12.4.3.1 Collaboration Agreement 117
Table 2: Features of different subtypes of MG 16
Table 3: Osserman und Genkins classification of myasthenia gravis, modified by the MGFA/Task Force 35
Table 4: Definition of QMG score 36
Table 5: MG-MMT scores 37
Table 6: Proposed Diagnostic Criteria for Myasthenia Gravis 52
Table 7: Total Number of Products in Myasthenia Gravis 61
Table 8: Late Stage Products (Phase III) 62
Table 9: Clinical Trial Description: Amifampridine Phosphate 64
Table 10: General Description: Amifampridine Phosphate 65
Table 11: Clinical Trial Description: Efgartigimod 68
Table 12: General Description: Efgartigimod 69
Table 13: Clinical Trial Description: Hizentra 70
Table 14: General Description: Human normal immunoglobulin G (IgG) 71
Table 15: Clinical Trial Description: IGIV-C 72
Table 16: General Description: IGIV-C 72
Table 17: Mid Stage Products (Phase II) 74
Table 18: Clinical Trial Description: Iscalimab 76
Table 19: General Description: Iscalimab 76
Table 20: Clinical Trial Description: M281 79
Table 21: General Description: M281 79
Table 22: Clinical Trial Description: Rozanolixizumab 81
Table 23: General Description: Rozanolixizumab 81
Table 24: Clinical Trial Description: RVT-1401 82
Table 25: General Description: RVT-1401 83
Table 26: Clinical Trial Description: Zilucoplan 85
Table 27: General Description: Zilucoplan 86
Table 28: Clinical Trial Description: Myasterix 87
Table 29: General Description: Myasterix 88
Table 30: Early Stage Products (Phase I) 89
Table 31: Clinical Trial Description: GTP-004 90
Table 32: Patents: GTP-004 90
Table 33: General Description: GTP-004 91
Table 34: Pre-clinical and Discovery Stage Products 92
Table 35: General Description: ACT-101 93
Table 36: General Description: HBN-1 (IV) 94
Table 37: General Description: Myasthenia Gravis-Tolerogen 95
Table 38: General Description: Pre-clinical Program 96
Table 39: General Description: PRTX-100 98
Table 40: General Description: TOL 3034 98
Table 41: General Description: Coversin 100
Table 42: General Description: HBN-100 101
Table 43: Assessment by Product Type 103
Table 44: Assessment by Stage and Product Type 104
Table 45: Assessment by Route of Administration 104
Table 46: Assessment by Stage and Route of Administration 105
Table 47: Assessment by Molecule Type 106
Table 48: Assessment by Stage and Molecule Type 107
Table 49: Approval Timelines for Clinical Products 108
Table 50: Inactive Drugs 111
Table 51: Clinical Trial Description: Belimumab 113
Table 52: General Description: Belimumab 113
Table 53: Clinical Trial Description: EN101 114
Table 54: General Description: EN101 115
Table 55: Clinical Trial Description: Tirasemtiv 117
Table 56: General Description: Tirasemtiv 118
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