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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
Executive 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
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Reasons to buy
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?
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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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
Table of Contents
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 AB 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 Alzheimer's Disease 13
4.1.2 Parkinson's Disease 14
4.1.3 Multiple Sclerosis 14
4.2 Disease Symptoms 14
4.3 Epidemiology 15
4.3.1 Alzheimer's Disease 16
4.3.2 Parkinson's Disease 16
4.3.3 Multiple Sclerosis 16
4.4 Etiology 17
4.4.1 Alzheimer's Disease 17
4.4.2 Parkinson's Disease 18
4.4.3 Multiple Sclerosis 18
4.5 Pathophysiology 19
4.5.1 Alzheimer's Disease 19
4.5.2 Parkinson's Disease 21
4.5.3 Multiple Sclerosis 23
4.5.4 Neurodegenerative Disease Area 24
4.6 Diagnosis 25
4.6.1 Alzheimer's Disease 25
4.6.2 Parkinson's Disease 25
4.6.3 Multiple Sclerosis 26
4.7 Comorbidities and Complications 26
4.8 Prognosis 27
4.8.1 Alzheimer's Disease 27
4.8.2 Parkinson's Disease 27
4.8.3 Multiple Sclerosis 28
4.9 Treatment Options 28