
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
Description
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
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?
- 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?
- 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.
Table of Contents
149 Pages
- 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
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