Frontier Pharma: Neurodegenerative Diseases - Protein Misfolding Targets and Neuromodulators Dominate the First-in-Class Pipeline and Lead the Way as Potential Disease-Modifying Therapies in AD and PD

Frontier Pharma: Neurodegenerative Diseases - Protein Misfolding Targets and Neuromodulators Dominate the First-in-Class Pipeline and Lead the Way as Potential Disease-Modifying Therapies in AD and PD

Summary


Neurodegenerative diseases are becoming increasingly prevalent due to an aging population, but this diverse therapy area remains largely untreatable with current therapies. Neurodegenerative diseases are characterized by neuronal death within the brain and/or central nervous system (CNS), leading to progressive decline in functional neurological capacities. It has a devastating effect on quality of life and independence, often requiring full-time care during the later disease stages. Conditions within the therapy area are diverse, and exhibit specific pathophysiologies and etiologies, while affecting people of all ages. This report examines the entire neurodegenerative disease therapy area, with a particular focus on the three most prevalent neurodegenerative disorders: Alzheimer’s disease (AD), Parkinson’s disease (PD) and multiple sclerosis (MS). AD and PD represent the most pressing challenges within the disease cluster, due to rapidly increasing prevalence driven by aging populations. Both AD and PD remain ineffectively treated despite substantial investment into R&D by pharmaceutical companies, due to high clinical trial failure rates. As a result there are no disease-modifying therapies for these two indications, with available treatments able to provide only marginal symptomatic relief. MS, the autoimmune disease of the CNS, contrasts with the rest of this cluster, as it affects a different population demographic, and has a lucrative pharmacological market following breakthrough success in the past decade.

Scope

Unmet need is extremely high in AD and PD, with MS showing continued promise in the development of effective therapies

  • What are the most important etiological risk factors and pathophysiological processes implicated in AD, PD and MS?
  • What is the current treatment algorithm?
  • How effective are current therapies for these indications, and how does this impact prognosis?
The AD pipeline is large and contains a very high proportion of first-in-class product innovation.
  • Which molecule types and molecular targets are most prominent across AD, PD and MS?
  • What are the connections, in terms of first-in-class innovation, between AD, PD and MS?
  • Which first-in-class targets are most promising?
  • How does the level of first-in-class innovation change within different target classes?
  • How do identified first-in-class molecular targets apply to AD, PD, MS and the wider therapeutic area?
  • How does first-in-class target diversity differ by stage of development and molecular target class?
The deals landscape is active and dominated by immunomodulator products
  • Which indications attract the highest deal values?
  • How has deal activity fluctuated over the past decade?
  • Which first-in-class pipeline products have no prior involvement in licensing or co-development deals?
Reasons to buy
  • Appreciate the current clinical and commercial landscapes by considering disease symptoms, pathogenesis, etiology, co-morbidities and complications, epidemiology, diagnosis, prognosis and treatment options.
  • Identify leading products and key unmet needs within the market.
  • Recognize innovative pipeline trends by analyzing therapies by stage of development, molecule type and molecular target.
  • Assess the therapeutic potential of first-in-class targets. Using proprietary matrix assessments, first-in-class targets in the pipeline have been assessed and ranked according to clinical potential. Individual matrix assessments are provided for targets categorized into four major classes: protein misfolding, neuromodulators, immunomodulators and neuroprotectants. The most promising first-in-class targets are reviewed in greater detail.
  • Consider first-in-class pipeline products with no prior involvement in licensing and co-development deals, which may represent potential investment opportunities.


1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Executive Summary
2.1 Robust Pipeline with Attempts to Meet Unmet Needs
2.2 Drugs Targeting Aβ and Elements of Protein Misfolding Pathways Offer Potential New Therapies for the Treatment of AD and PD
2.3 Neurodegenerative Disease Pipeline Emphasizes Move Away from Conventional Areas towards Targets Related to Protein Misfolding and Neuroprotection
3 The Case for Innovation
3.1 Growing Opportunities for Biologic Products
3.2 Diversification of Molecular Targets
3.3 Innovative First-in-Class Product Development Remains Attractive
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation
3.5 Sustained Innovation in Neurodegenerative Diseases
3.6 GBI Research Report Guidance
4 Clinical and Commercial Landscape
4.1 Therapy Area Overview
4.1.1 Alzheimer’s Disease
4.1.2 Parkinson’s Disease
4.1.3 Multiple Sclerosis
4.2 Disease Symptoms
4.3 Epidemiology
4.3.1 Alzheimer’s Disease
4.3.2 Parkinson’s Disease
4.3.3 Multiple Sclerosis
4.4 Etiology
4.4.1 Alzheimer’s Disease
4.4.2 Parkinson’s Disease
4.4.3 Multiple Sclerosis
4.5 Pathophysiology
4.5.1 Alzheimer’s Disease
4.5.2 Parkinson’s Disease
4.5.3 Multiple Sclerosis
4.5.4 Neurodegenerative Disease Area
4.6 Diagnosis
4.6.1 Alzheimer’s Disease
4.6.2 Parkinson’s Disease
4.6.3 Multiple Sclerosis
4.7 Comorbidities and Complications
4.8 Prognosis
4.8.1 Alzheimer’s Disease
4.8.2 Parkinson’s Disease
4.8.3 Multiple Sclerosis
4.9 Treatment Options
4.9.1 Alzheimer’s Disease
4.9.2 Parkinson’s Disease
4.9.3 Multiple Sclerosis
4.10 Overview of Marketed Products in Neurodegeneration
5 Assessment of Pipeline Product Innovation
5.1 Overview
5.2 Pipeline by Stage of Development and Molecule Type
5.2.1 Neurodegenerative Disease Overall
5.2.2 Key Neurodegenerative Disease Indications
5.3 Pipeline by Molecular Target
5.3.1 Neurodegenerative Disease Overall
5.3.2 Key Neurodegenerative Disease Indications
5.4 Comparative Distribution of Programs between the Market and Pipeline by Molecular Target Class
5.5 First-in-Class Programs Targeting Novel Molecular Targets
6 Signaling Network and Innovation Alignment within Neurodegenerative Disease
6.1 Complexity of Signaling Networks in Neurodegenerative Disease
6.2 Signaling Pathways and First-in-Class Molecular Target Integration
6.3 First-in-Class Matrix Assessment
7 First-in-Class Target and Pipeline Program Evaluation
7.1 Pipeline Programs Targeting Protein Misfolding and Aggregation
7.1.1 Pipeline Programs Targeting α-synuclein
7.1.2 Pipeline Programs Targeting Glucosylceramidase
7.1.3 Pipeline Programs Targeting Amyloid Precursor Protein, Amyloid Protein and Peptide, Amyloid Beta Peptide, Amyloid Beta Protein, Beta-amyloid Protein 40 and Beta-amyloid Protein 42.
7.2 Pipeline Programs Targeting Neuromodulators
7.2.1 Pipeline Programs Targeting GTP cyclohydrolase I
7.2.2 Pipeline Programs Targeting Acetylcholine Receptor Subunit Nicotinic Alpha-6
7.2.3 Pipeline Programs Targeting Metabotropic glutamate receptor 4
7.3 Pipeline Programs Targeting Immunomodulators
7.3.1 Pipeline Programs Targeting Integrin alpha m
7.3.2 Pipeline Programs Targeting Leukocyte immunoglobulin like receptors B1 and B2
7.3.3 Pipeline Program Targeting Tumor Necrosis Factor Superfamily Member 10
7.4 Pipeline Programs Targeting Neuroprotectants
7.4.1 Pipeline Programs Targeting Rho-Associated Protein Kinase 2
7.4.2 Pipeline Programs Targeting Angiotensin receptor type 2
7.5 Conclusion
8 Strategic Consolidations
8.1 Industry-Wide First-in-Class Deals
8.2 Licensing Deals
8.2.1 Deals by Region, Value and Year
8.2.2 Deals by Stage of Development and Value
8.2.3 Deals by Molecule Type, Molecular Target and Value
8.2.4 List of Deals with Disclosed Deal Values
8.3 Co-development Deals
8.3.1 Deals by Region, Value and Year
8.3.2 Deals by Stage of Development and Value
8.3.3 Deals by Molecule Type, Molecular Target and Value
8.3.4 List of Deals with Disclosed Deal Values
8.4 First-in-Class Programs with and without Prior Involvement in Licensing and Co-development Deals
9 Appendix
9.1 References
9.2 Abbreviations
9.3 Disease List
9.3.1 Alzheimer’s Disease
9.3.2 Parkinson’s Disease
9.3.3 Multiple Sclerosis
9.3.4 Other
9.4 Top 10 First-in-Class Targets for Alzheimer’s Disease, Parkinson’s Disease and Multiple Sclerosis
9.5 Research Methodology
9.5.1 Data integrity
9.5.2 Innovative and meaningful analytical techniques and frameworks:
9.5.3 Evidence based analysis and insight:
9.6 Secondary Research
9.6.1 Market Analysis
9.6.2 Pipeline Analysis
9.6.3 First-in-Class Matrix Assessment
9.6.4 First-in-Class Target Profiles
9.6.5 Licensing and Co-Development Deals
9.7 Contact Us
9.8 Disclaimer
1.1 List of Tables
Table 1: Neurodegenerative Disease, Global, Symptoms of AD, PD and MS, 2017
Table 2: Neurodegenerative Disease, Global, Epidemiology of Common Neurodegenerative Diseases, 2017
Table 3: Neurodegenerative Disease, Global, Common Comorbidities Associated with AD, PD and MS, 2017
Table 4: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Synuclein and α-synuclein, 2017
Table 5: Neurodegenerative Disease, Global, Pipeline Programs Targeting Synuclein and α-synuclein, 2017
Table 6: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Glucosylceramidase, 2017
Table 7: Neurodegenerative Disease, Global, Pipeline Programs Targeting Glucosylceramidase, 2017
Table 8: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Amyloid Precursor Protein and Amyloid Protein and Peptide, 2017
Table 9: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Amyloid Precursor Protein and Amyloid Protein and Peptide, 2017
Table 10: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of GTP Cyclohydrolase I, 2017
Table 11: Neurodegenerative Disease, Global, Pipeline Programs Targeting GTP Cyclohydrolase I, 2017
Table 12: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Acetylcholine receptor subunit nicotinic alpha-6, 2017
Table 13: Neurodegenerative Disease, Global, Pipeline Programs Targeting Acetylcholine receptor subunit nicotinic alpha-6, 2017
Table 14: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Metabotropic glutamate receptor 4, 2017
Table 15: Neurodegenerative Disease, Global, Pipeline Programs Targeting Metabotropic glutamate receptor 4, 2017
Table 16: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Integrin alpha m, 2017
Table 17: Neurodegenerative Disease, Global, Pipeline Programs Targeting Integrin alpha m, 2017
Table 18: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Leukocyte immunoglobulin receptor B1/2, 2017
Table 19: Neurodegenerative Disease, Global, Pipeline Programs Targeting Leukocyte immunoglobulin receptor B1/2, 2017
Table 20: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Tumor necrosis superfamily member 10, 2017
Table 21: Neurodegenerative Disease, Global, Pipeline Programs Targeting Tumor necrosis superfamily member 10, 2017
Table 22: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Rho-associated protein kinase 2, 2017
Table 23: Neurodegenerative Disease, Global, Pipeline Programs Targeting Rho-associated protein kinase 2, 2017
Table 24: Neurodegenerative Disease, Global, Key Features and Pipeline Activity of Angiotensin receptor type 2, 2017
Table 25: Neurodegenerative Disease, Global, Pipeline Programs Targeting Angiotensin receptor type 2,
1.2 List of Figures
Figure 1: Neurodegenerative Disease, US, Innovation Trends in Product Approvals, 1987-2015
Figure 2: Neurodegenerative Disease, US, Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013
Figure 3: Neurodegenerative Disease, Global, Overview of Marketed Products, 2017
Figure 4: Neurodegenerative Disease, Global, Pipeline by Therapy Area, 2017
Figure 5: Neurodegenerative Disease, Global, Pipeline by Stage of Development and Molecule Type, 2017
Figure 6: Neurodegenerative Disease, Global, Pipeline by Key Indications and Stage of Development, 2017
Figure 7: Neurodegenerative Disease, Global, Pipeline by Key Indications and Molecule Type, 2017
Figure 8: Neurodegenerative Disease, Global, Pipeline by Molecular Target and Stage of Development, 2017
Figure 9: Neurodegenerative Disease, Global, Pipeline Breakdown of Key Molecular Target Classes, 2017
Figure 10: Neurodegenerative Disease, Global, Pipeline Breakdown of Key Indications by Molecular Target Class, 2017
Figure 11: Neurodegenerative Disease, Global, Pipeline and Marketed Products by Molecular Target Class, 2017
Figure 12: Neurodegenerative Disease, Global, Pipeline by Molecular Target Class, First-in-Class Status and Stage of Development, 2017
Figure 13: Neurodegenerative Disease, Global, Pipeline by Molecular Target Class, First-in-Class Status and Key Indication, 2017
Figure 14: Neurodegenerative Disease, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Products by Stage of Development and Molecular Target Class (%), 2017
Figure 15: Neurodegenerative Disease, Global, Ratio of First-in-Class Products to First-in-Class Targets by Stage of Development and Molecular Target Class, 2017
Figure 16: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 1
Figure 17: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 2
Figure 18: Neurodegenerative Disease , Global, List of All Pipeline Programs, 2017 Part 3
Figure 19: Neurodegenerative Disease , Global, List of All Pipeline Programs, 2017 Part 4
Figure 20: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 5
Figure 21: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 6
Figure 22: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 7
Figure 23: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 8
Figure 24: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 9
Figure 25: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 10
Figure 26: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 11
Figure 27: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 12
Figure 28: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 13
Figure 29: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 14
Figure 30: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 15
Figure 31: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 16
Figure 32: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 17
Figure 33: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 18
Figure 34: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 19
Figure 35: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 20
Figure 36: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 21
Figure 37: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 22
Figure 38: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 23
Figure 39: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 24
Figure 40: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 25
Figure 41: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 26
Figure 42: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 27
Figure 43: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 28
Figure 44: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 29
Figure 45: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 30
Figure 46: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 31
Figure 47: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 32
Figure 48: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 33
Figure 49: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 34
Figure 50: Neurodegenerative Disease, Global, List of All Pipeline Programs, 2017 Part 35
Figure 51: Neurodegenerative Disease, Global, First-in-Class Molecular Target Matrix Assessment for Protein Misfolding, 2017
Figure 52: Neurodegenerative Disease Therapeutics Market, Global, First-in-Class Molecular Target Matrix Assessment for Neuromodulators, 2017
Figure 53: Neurodegenerative Disease Therapeutics Market, Global, First-in-Class Molecular Target Matrix Assessment for Immunomodulators, 2017
Figure 54: Neurodegenerative Disease Therapeutics Market, Global, First-in-Class Molecular Target Matrix Assessment for Neuroprotectants, 2017
Figure 55: Neurodegenerative Disease, Global, Industry-Wide Licensing Deals by Stage of Development and First-in-Class Status, 2006-2014
Figure 56: Neurodegenerative Disease, Global, Industry-Wide Licensing Deals by Deal Value, Upfront Payment Value, Stage of Development and First-in-Class Status, 2006-2014
Figure 57: Neurodegenerative Disease, Global, Licensing Deals by Region, Value and Year, 2006-2017
Figure 58: Neurodegenerative Disease, Global, Licensing Deals by Deal Value, Upfront Payment Value and Stage of Development, 2006-2017
Figure 59: Neurodegenerative Disease, Global, Number and Aggregate Deal Value of Licensing Deals by Molecule Type and Molecular Target, 2006-2017
Figure 60: Neurodegenerative Disease, Global, Licensing Deals with Disclosed Deal Values, 2006-2017
Figure 61: Neurodegenerative Disease, Global, Licensing Deals with Disclosed Deal Values, 2006-2017
Figure 62: Neurodegenerative Disease, Global, Co-development Deals by Region, Value and Year, 2006-2017
Figure 63: Neurodegenerative Disease, Global, Co-development Deals by Deal Value, Upfront Payment Value and Stage of Development, 2006-2017
Figure 64: Neurodegenerative Disease, Global, Number and Aggregate Deal Value of Co-development Deals by Molecule Type and Molecular Target, 2006-2017
Figure 65: Neurodegenerative Disease, Global, Co-development Deals with Disclosed Deal Values, 2006-2017
Figure 66: Neurodegenerative Disease, Global, Co-development Deals with Disclosed Deal Values, 2006-2017
Figure 67: Neurodegenerative Disease, Global, First-in-Class Pipeline Products With and Without Prior Involvement in Licensing or Co-development Deals, 2017, Part 1
Figure 68: Neurodegenerative Disease, Global, First-in-Class Pipeline Products With and Without Prior Involvement in Licensing or Co-development Deals, 2017, Part 2
Figure 69: Neurodegenerative Disease, Global, First-in-Class Pipeline Products With and Without Prior Involvement in Licensing or Co-development Deals, 2017, Part 3
Figure 70: Neurodegenerative Disease, Global, First-in-Class Pipeline Products With and Without Prior Involvement in Licensing or Co-development Deals, 2017, Part 4
Figure 71: Neurodegenerative Disease, Global, First-in-Class Pipeline Products With and Without Prior Involvement in Licensing or Co-development Deals, 2017, Part 5
Figure 72: Neurodegenerative Disease, Global, First-in-Class Pipeline Products with and without Prior Involvement in Licensing or Co-development Deals, 2017, Part 6
Figure 73: Neurodegenerative Disease, Global, Top 10 Ranked Matrix Targets in Alzheimer’s Disease, 2017
Figure 74: Neurodegenerative Disease, Global, Top 10 Ranked Matrix Targets in Parkinson’s Disease, 2017
Figure 75: Neurodegenerative Disease, Global, Top 10 Ranked Matrix Targets in Multiple Sclerosis,

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