Adrenoleukodystrophy (ALD) - Pipeline Analysis, 2019
- UNI4065859
- 77 Pages
- June 2019
- Healthcare
Adrenoleukodystrophy is a fatal progressive neurogenerative disorder affecting brain white matter. The common form of ALD is X-linked (X-ALD) and results from mutation of ABCD1 encoded very long chain fatty acid (VLCFA) transporter. It is clinically heterogeneous with the cerebral form being the most severe. X-linked Adrenoleukodystrophy (X-ALD) is a rare X-linked metabolic disorder caused by variants in the ABCD1 gene which causes a deficiency in adrenoleukodystrophy protein (ALDP) and subsequent accumulation of very long chain fatty acids (VLCFA).
The symptoms of Adrenoleukodystrophy are highly variable. There are mainly three types which are present in approximately 90-95% of the affected people: a childhood cerebral form; an adrenomyeloneuropathy type; and an adrenal insufficiency only type.
The diagnosis of ALD, therefore, can be made with the highly sensitive and specific plasma VLCFA assay. When an abnormal pattern is detected with an identifiable ABCD1 mutation in a presymptomatic patient (or in the setting of classic clinical disease in others), males are determined to have the biochemical defect of ALD. Disease may manifest at any age. Most ALD males will develop primary adrenal insufficiency (AI) in childhood or adolescence, and most surviving males will develop spinal cord disease (adrenomyeloneuropathy [AMN]) during adulthood.
The therapeutic pipeline of Adrenoleukodystrophy consists of approximately 9+ products in different stages of development. Currently, 3+ drugs are in Phase III development and major drugs are in late stage.
Top Company Analysed
Some of the key players include Applied Genetic Technologies Corporation; Bluebird bio; Magenta Therapeutics; MedDay Pharmaceuticals; Minoryx Therapeutics; NeuroVia; Orpheris; ReceptoPharm; SOM Biotech; and Viking Therapeutics.
Scope of the report:
• Provides an overview of therapeutic pipeline activity for Adrenoleukodystrophy across the complete product development cycle including all clinical and non-clinical stages
• The report comprises of detailed profiles of Adrenoleukodystrophy 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 discontinued products have been included in the report
Reasons to buy:
• The Adrenoleukodystrophy 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 discontinuation of the products
Customization Options:
The Adrenoleukodystrophy 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 8
1.2 Secondary Research 8
2. Overview 9
2.1 Introduction 9
2.2 Classification of Adrenoleukodystrophy 10
2.3 Signs and Symptoms 11
2.4 Causes 12
2.5 Pathophysiology 14
2.5.1 Biochemical and Genetic Aspects of X-ALD Pathophysiology 15
2.5.2 Lipidomic aspects of the pathophysiology of X-ALD 16
2.5.3 Functional redundancy of ABC transporters and their role in the pathophysiology of X-ALD 17
2.5.4 Pathophysiology of AMN in males and in heterozygous females 18
2.5.4.1 Pathophysiology of inflammation in cerebral X-ALD 19
2.5.5 Pathophysiology of adrenals, testis and hair in X-ALD 21
2.5.6 Cerebral Adrenoleukodystrophy 21
2.6 Diagnosis 24
2.6.1 Neuroimaging 24
2.6.2 Very long-chain fatty acids 24
2.6.3 Mutation analysis 25
2.6.4 Genetic counseling 25
2.6.5 Heterozygote identification 26
2.6.6 Extended family testing 26
2.6.7 Prenatal testing 26
2.6.8 Newborn screening 27
2.6.9 Differential Diagnosis 27
2.6.10 Biochemical and Molecular Diagnosis 28
2.7 Treatment 29
2.7.1 Boys and adult males with X-ALD 29
2.7.2 Women with X-ALD 31
3. Comparative Analysis by Financing 32
3.1 Overview of Comparative Analysis 32
3.2 Company Consolidations under Partnership and Agreements 32
4. Products Under Development by Designation 33
4.1 Comparative Analysis 33
5. Pipeline Therapeutics at a Glance 34
6. Comparative Analysis 35
6.1 Comparative Analysis of Product under Adrenoleukodystrophy 35
7. Late Phase Products (Phase III) 36
7.1 Comparative Analysis 36
7.2 Hydroxypioglitazone: Minoryx Therapeutics 37
7.2.1 Product Description 37
7.2.2 Research and Development 37
7.2.2.1 Clinical Studies 37
7.2.2.1.1 Phase II/III 37
7.2.2.1.2 Results of the Analysis 37
7.2.3 Product Development Activities 38
7.2.3.1 Funding 38
7.3 Lenti-D: bluebird bio 39
7.3.1 Product Description 39
7.3.2 Research and Development 39
7.3.2.1 Clinical Studies 39
7.3.2.1.1 Phase III 39
7.3.2.1.2 Phase II/III 40
7.3.2.1.3 Results of Analysis 40
7.3.3 Product Development Activities 42
7.3.3.1 Designation 42
7.4 RPI-78M: ReceptoPharm 43
7.4.1 Product Description 43
7.4.2 Product Development Activities 43
7.4.2.1 Designation 43
7.4.2.2 Agreement 43
7.5 MD1003: MedDay Pharmaceuticals 44
7.5.1 Product Description 44
7.5.2 Research and Development 45
7.5.2.1 Pre-clinical Studies 45
7.5.2.2 Clinical Studies 45
7.5.2.2.1 Phase II/III 45
8. Mid Phase Products (Phase II) 47
8.1 Comparative Analysis 47
8.2 Spanlecortemlocel: Magenta Therapeutics 47
8.2.1 Product Description 47
8.2.2 Research and Development 48
8.2.2.1 Preclinical Studies 48
8.2.2.2 Clinical Studies 49
8.2.2.2.1 Phase II 49
8.2.2.2.2 Results of Analysis 49
8.2.3 Product Development Activities 51
8.2.3.1 Licensing 51
8.2.3.2 IND Application 51
8.2.3.3 Financing 52
9. Early Stage Products (Phase I) 54
9.1 Comparative Analysis 54
9.2 OP-101: Orpheris 54
9.2.1 Product Description 54
9.2.2 Research and Development 55
9.2.2.1 Pre-clinical Studies 55
9.2.2.2 Clinical Studies 55
9.2.2.2.1 Phase I 55
9.2.3 Product Development Activities 56
9.2.3.1 Designation 56
9.2.3.2 Type C Meeting 56
9.2.3.3 Technology 56
9.3 SOM1201: SOM Biotech 57
9.3.1 Product Description 57
10. Pre-clinical Stage Products 59
10.1 Comparative Analysis 59
10.2 Pre-clinical Program for ALD: Applied Genetic Technologies Corporation 60
10.2.1 Product Description 60
10.3 SOM1202: SOM Biotech 60
10.3.1 Product Description 60
10.4 VK0214: Viking Therapeutics 61
10.4.1 Product Description 61
10.4.2 Research and Development 62
10.4.2.1 Pre-clinical Studies 62
10.4.3 Product Development Activities 64
10.4.3.1 Designation 64
10.4.3.2 Collaboration 64
11. Therapeutic Assessment 65
11.1 Assessment by Stage and Product Type 65
11.2 Assessment by Route of Administration 66
11.3 Assessment by Stage and Route of Administration 67
11.4 Assessment by Molecule Type 68
11.5 Assessment by Stage and Molecule Type 69
12. Approval Timelines 70
13. Analyst Insights 71
14. Inactive Products 72
14.1 Comparative Analysis 72
14.2 Sobetirome: NeuroVia 72
14.2.1 Product Description 72
14.2.2 Research and Development 73
14.2.2.1 Clinical Studies 73
14.2.3 Product Development Activities 73
14.2.3.1 Designation 73
14.2.3.2 Financing 73
14.2.3.3 Agreement 74
Table 2: Total Number of Products Under Adrenoleukodystrophy 35
Table 3: Late Stage Products (Phase III) 36
Table 4: Clinical Trial Description: MIN-102 38
Table 5: General Description: Hydroxypioglitazone 38
Table 6: Clinical Trial Description: Lenti-D 42
Table 7: General Description: Lenti-D 42
Table 8: General Description: RPI-78M 44
Table 9: Clinical Trial Description: MD1003 45
Table 10: General Description: MD1003 45
Table 11: Mid Stage Products (Phase II) 47
Table 12: Clinical Trial Description: MGTA-456 51
Table 13: General Description: Spanlecortemlocel 52
Table 14: Early Stage Products (Phase I) 54
Table 15: Clinical Trial Description: OP-101 56
Table 16: General Description: OP-101 57
Table 17: General Description: SOM1201 57
Table 18: Pre-clinical Stage Products 59
Table 19: General Description: Pre-clinical Program for ALD 60
Table 20: General Description: SOM1202 60
Table 21: General Description: VK0214 64
Table 22: Assessment by Stage and Product Type 65
Table 23: Assessment by Route of Administration 66
Table 24: Assessment by Stage and Route of Administration 67
Table 25: Assessment by Molecule Type 68
Table 26: Assessment by Stage and Molecule Type 69
Table 27: Approval Timelines for Clinical Products 70
Table 28: Inactive Products 72
Table 29: Clinical Trial Description: Sobetirome 73
Table 30: General Description: Sobetirome 74
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