Friday, 1 December 2017

Neurodegenerative Diseases - Protein Misfolding Targets and Neuromodulators Dominate the First-in-Class Pipeline | New Study

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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: Alzheimers disease (AD), Parkinsons 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.

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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.

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Table of Contents

1 Table of Contents 2
1.1 List of Tables 4
1.2 List of Figures 4

2 Executive Summary 7
2.1 Robust Pipeline with Attempts to Meet Unmet Needs 7
2.2 Drugs Targeting A and Elements of Protein Misfolding Pathways Offer Potential New Therapies for the Treatment of AD and PD 7
2.3 Neurodegenerative Disease Pipeline Emphasizes Move Away from Conventional Areas towards Targets Related to Protein Misfolding and Neuroprotection 7

3 The Case for Innovation 9
3.1 Growing Opportunities for Biologic Products 10
3.2 Diversification of Molecular Targets 10
3.3 Innovative First-in-Class Product Development Remains Attractive 10
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 11
3.5 Sustained Innovation in Neurodegenerative Diseases 11
3.6 GBI Research Report Guidance 12

4 Clinical and Commercial Landscape 13
4.1 Therapy Area Overview 13
4.1.1 Alzheimers Disease 13
4.1.2 Parkinsons Disease 14
4.1.3 Multiple Sclerosis 14
4.2 Disease Symptoms 14
4.3 Epidemiology 15
4.3.1 Alzheimers Disease 16
4.3.2 Parkinsons Disease 16
4.3.3 Multiple Sclerosis 16
4.4 Etiology 17
4.4.1 Alzheimers Disease 17
4.4.2 Parkinsons Disease 18
4.4.3 Multiple Sclerosis 18
4.5 Pathophysiology 19
4.5.1 Alzheimers Disease 19
4.5.2 Parkinsons Disease 21
4.5.3 Multiple Sclerosis 23
4.5.4 Neurodegenerative Disease Area 24
4.6 Diagnosis 25
4.6.1 Alzheimers Disease 25
4.6.2 Parkinsons Disease 25
4.6.3 Multiple Sclerosis 26
4.7 Comorbidities and Complications 26
4.8 Prognosis 27
4.8.1 Alzheimers Disease 27
4.8.2 Parkinsons Disease 27
4.8.3 Multiple Sclerosis 28
4.9 Treatment Options 28
4.9.1 Alzheimers Disease 28
4.9.2 Parkinsons Disease 29
4.9.3 Multiple Sclerosis 30
4.10 Overview of Marketed Products in Neurodegeneration 31

5 Assessment of Pipeline Product Innovation 33
5.1 Overview 33
5.2 Pipeline by Stage of Development and Molecule Type 33
5.2.1 Neurodegenerative Disease Overall 33
5.2.2 Key Neurodegenerative Disease Indications 34
5.3 Pipeline by Molecular Target 36
5.3.1 Neurodegenerative Disease Overall 36
5.3.2 Key Neurodegenerative Disease Indications 39
5.4 Comparative Distribution of Programs between the Market and Pipeline by Molecular Target Class 40
5.5 First-in-Class Programs Targeting Novel Molecular Targets 41

6 Signaling Network and Innovation Alignment within Neurodegenerative Disease 806.1 Complexity of Signaling Networks in Neurodegenerative Disease 80
6.2 Signaling Pathways and First-in-Class Molecular Target Integration 80
6.3 First-in-Class Matrix Assessment 81

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