Frontier Pharma: Parkinson’s Disease - Identifying and Commercializing First-in-Class Innovation

GBI Research
95 Pages - GBI10540


Parkinson’s Disease (PD) is the second most common neurodegenerative disease after Alzheimer’s disease, affecting around 7.4 million people worldwide. It is caused by the progressive loss of dopamine-producing neurons in the substantia nigra within the basal ganglia. The main motor symptoms are tremors, bradykinesia and rigidity, although symptoms vary between individuals. As the disease progresses to more advanced stages, increasing symptoms and complications develop, which can cause severe disability in patients.

The current market offers a number of anti-PD treatment options which provide symptomatic relief. Levodopa is currently the gold standard therapy, with other drug classes including dopamine agonists and Monoamine Oxidase B (MAO-B) inhibitors also used to treat early and advanced cases. With no current treatment showing effectiveness in delaying the course of the disease, PD remains incurable. The high unmet need for a disease-modifying therapy is reflected in the pipeline, where a high proportion of early-stage first-in-class programs target the potential pathogenic mechanisms underlying neurodegeneration.


PD is a crowded market with many neuromodulatory drug classes available. However, a disease-modifying therapy with neuroprotective effects is yet to be developed.
 - What are the primary mechanisms that are thought to contribute to neuronal death?
 - What are the major barriers facing the development of investigational neuroprotective candidates?
Analysis reveals a high level of innovation and diversity in the pipeline, with 121 first-in-class programs acting on 57 unique molecular targets.
Neuromodulatory targets remain the dominant target family, particularly in the late-stage pipeline.
 - What are the first-in-class families with a significant presence?
 - How well do they align with the underlying pathways governing neuronal death in PD?
Some of the first-in-class targets have a potentially stronger chance of being translated into novel treatments for PD.
 - What is the scientific rationale behind these targets? How do they perform in Preclinical studies?
 - What are the commonly used disease models and the parameters measuring neuroprotective effects in PD animal studies?
Deals involving first-in-class PD products are more likely to be made in earlier stages of development than non-first-in-class deals.
 - What is the dominant molecular target in the PD deals landscape?
 - What are the promising first-in-class products still available for future licensing?

Reasons to buy

This report will allow you to -
- Understand the current clinical and commercial landscape by considering the proposed pathogenic processes underlying PD neurodegeneration, diagnosis, prognosis, and the available treatment options and their usage in early and advanced PD.
- Visualize the composition of the PD market to highlight the current unmet needs in order to gain a competitive understanding of the key opportunities.
- Analyze the PD pipeline and stratify by stage of development, molecule type, and molecular target - the diversity of molecular targets in the pipeline is extremely encouraging due to the multifaceted nature of PD.
- Assess the therapeutic potential of first-in-class targets using a proprietary matrix that assesses and ranks first-in-class products according to clinical potential.
- Target the most promising and innovative PD products for early-stage investment by analyzing trends in licensing and co-development deals and accessing a curated list of first-in-class therapies potentially open to deal-making opportunities.


1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3
2 Executive Summary 4
2.1 High Unmet Needs Remain in Parkinson’s Disease Market 4
2.2 Diverse and Innovative Pipeline to Shift Focus to Disease Modification 4
2.3 Deals Landscape Presents Substantial Investment Opportunities 4
3 The Case for Innovation 5
3.1 Growing Opportunities for Biologic Products 6
3.2 Diversification of Molecular Targets 6
3.3 Innovative First-in-Class Product Developments Remain Attractive 6
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 7
3.5 Sustained Innovation 7
3.6 GBI Research Report Guidance 8
4 Clinical and Commercial Landscape 9
4.1 Disease Overview 9
4.2 Epidemiology 9
4.3 Disease Etiology 10
4.3.1 Exposure to Environmental Toxins 10
4.3.2 Genetic Causes of Familial Parkinson’s Disease 10
4.3.3 Susceptibility Genes for Parkinson’s Disease 12
4.4 Disease Pathophysiology 12
4.4.1 Basal Ganglia Anatomy and Physiology 12
4.4.2 Process Underlying Neurodegeneration 15
4.5 Disease Symptoms 19
4.6 Co-morbidities and Complications 19
4.7 Diagnosis 19
4.8 Classification of Disease Stages 20
4.8.1 Hoehn and Yahr Scale 20
4.8.2 Unified Parkinson’s Disease Rating Scale 20
4.8.3 Scale for the Assessment of Positive Symptoms 22
4.8.4 Mini Mental State Examination 22
4.9 Prognosis and Disease Staging 22
4.10 Treatment Options 23
4.10.1 Pharmacotherapy 23
4.10.2 Non-pharmacological Treatment 25
4.11 Overview of Marketed Products 25
4.11.1 Molecule Type and Target Analysis 26
4.11.2 Treatment Algorithm 27
4.12 Current Unmet Needs 33
5 Assessment of Pipeline Product Innovation 35
5.1 Parkinson’s Disease Pipeline by Molecule Type, Phase and Therapeutic Target 35
5.2 Comparative Distribution of Programs between Parkinson’s Disease Market and Pipeline by Therapeutic Target Family 40
5.3 First-in-Class Pipeline Programs Targeting Novel Molecular Targets 40
6 Signaling Pathways, Genetics and Innovation Alignment 45
6.1 The Complexity of Signaling Networks in the Central Nervous System 45
6.2 Signaling Pathways and First-in-Class Molecular Target Integration 46
6.3 First-in-Class Target Matrix Assessment 46
7 First-in-Class Target Evaluation 49
7.1 Pipeline Programs Targeting α-synuclein 49
7.2 Pipeline Programs Targeting DJ-1 52
7.3 Pipeline Programs Targeting Parkin 54
7.4 Pipeline Programs Targeting High Affinity Nerve Growth Factor Receptor 56
7.5 Pipeline Programs Targeting C-jun N-Terminal Kinase 58
7.6 Pipeline Programs Targeting Leucine-Rich Repeat Kinase 2 60
7.7 Pipeline Programs Targeting Growth Hormone Secretagogue Receptor Type 1 62
7.8 Pipeline Programs Targeting Metabotropic Glutamate Receptor 4 64
7.9 Pipeline Programs Targeting NAD-dependent Protein Deacetylase Sirtuin-2 67
7.10 Overview of Pipeline Programs Targeting Progranulin 68
7.11 Conclusion 71
8 Deals and Strategic Consolidations 72
8.1 Industry-Wide First-in-Class Deals 72
8.2 Licensing Deals 73
8.3 Co-development Deals 77
8.4 First-in-Class Programs not Involved in Licensing or Co-development Deals 80
9 Appendix 82
9.1 Abbreviations 82
9.2 Bibliography 84
9.3 Research Methodology 93
9.4 Secondary Research 94
9.4.1 Marketed Product Heatmaps and Treatment Algorithm 94
9.4.2 Pipeline Analysis 94
9.4.3 First-in-Class Matrix Assessment 94
9.4.4 First-in-Class Target Profiles 95
9.4.5 Licensing and Co-development Deals 95
9.5 Contact Us 95
9.6 Disclaimer 95

1.1 List of Tables
Table 1: Definition of the Stages of Disability in Hoehn and Yahr Scale, 1967 20
Table 2: Evaluation of Disability by Unified Parkinson Disease Rating Scale, 2013 21

1.2 List of Figures
Figure 1: Innovation Trends in Product Approvals, 1987-2013 5
Figure 2: Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013 7
Figure 3: Indirect and Direct Pathways in Basal Ganglia 14
Figure 4: Molecular Targets of Marketed Products 27
Figure 5: Treatment Algorithm of Parkinson’s Disease 28
Figure 6: Efficacy and Safety of Treatments for Early Parkinson’s Disease 30
Figure 7: Efficacy and Safety of Treatments for Advanced Parkinson’s Disease 32
Figure 8: Efficacy and Safety of Treatment for Non-Motor Symptoms in Parkinson’s Disease 33
Figure 9: Developmental Pipeline Overview 36
Figure 10: Parkinson’s Disease Pipeline by Molecular Target 37
Figure 11: Parkinson’s Disease Pipeline by Molecular Target 39
Figure 12: Molecular Target Category Comparison, Pipeline and Marketed Products 40
Figure 13: Molecular Target Category Comparison, Pipeline First-in-Class and Established Molecular Targets 42
Figure 14: Parkinson’s Disease, Global, First-in-Class Pipeline Products 43
Figure 15: First-in-Class Molecular Target Analysis Matrix 48
Figure 16: Data and Evidence for α-synuclein as a Therapeutic Target 51
Figure 17: Pipeline Programs Targeting α-synuclein 52
Figure 18: Data and Evidence for DJ-1 as a Therapeutic Target 54
Figure 19: Pipeline Programs Targeting DJ-1 54
Figure 20: Data and Evidence for Parkin as a Therapeutic Target 55
Figure 21: Pipeline Programs Targeting Parkin 55
Figure 22: Data and Evidence for High Affinity Nerve Growth Factor Receptor as a Therapeutic Target 57
Figure 23: Pipeline Programs Targeting High Affinity Nerve Growth Factor Receptor 57
Figure 24: Data and Evidence for C-Jun N-Terminal Kinases as a Therapeutic Target 59
Figure 25: Pipeline Programs Targeting C-Jun N-Terminal Kinase 60
Figure 26: Data and Evidence for Leucine-Rich Repeat Kinase 2 as a Therapeutic Target 61
Figure 27: Pipeline Programs Targeting Leucine-Rich Repeat Kinase 2 62
Figure 28: Data and Evidence for Growth Hormone Secretagogue Receptor as a Therapeutic Target 64
Figure 29: Pipeline Programs Targeting Growth Hormone Secretagogue Receptor Type 1 64
Figure 30: Data and Evidence for Metabotropic Glutamate Receptor 4 as a Therapeutic Target 66
Figure 31: Pipeline Programs Targeting Metabotropic Glutamate Receptor 4 67
Figure 32: Data and Evidence for NAD-dependent Protein Deacetylase Sirtuin-2 as a Therapeutic Target 68
Figure 33: Pipeline Programs Targeting NAD-Dependent Protein Deacetylase Sirtuin-2 68
Figure 34: Data and Evidence for Progranulin as a Therapeutic Target 70
Figure 35: Pipeline Programs Targeting Progranulin 70
Figure 36: Industry-Wide Deals by Stage of Development, 2006-2014 72
Figure 37: Industry-Wide Deals by Stage of Development, 2006-2014 73
Figure 38: Licensing Deals, 2006-2015 74
Figure 39: Licensing Deals by Molecule Type, 2006-2015 75
Figure 40: Licensing Deals by Molecular Target, 2006-2015 75
Figure 41: Summary of Licensing Deals, 2006-2015 76
Figure 42: Co-development Deals, 2006-2015 77
Figure 43: Co-development Deals by Phase and Molecule Type, 2006-2015 78
Figure 44: Co-development Deals by Molecular Target, 2006-2015 78
Figure 45: Summary of Co-development Deals, 2006-2015 79
Figure 46: First-in-Class Programs with no Recorded Prior Deal Involvement, 2006-2015 81


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