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Frontier Pharma Parkinsons Disease - Identifying and Commercializing First-in-
Class Innovation Analysis & Forecast
In the United States, almost 1.5 million people are reported to be affected by Parkinson’s
disease (PD), wherein 60,000 patients continue to be diagnosed with the condition every year,
costing the U.S. economy US$14 billion on an annual basis in the direct and indirect expenses.
Browse the full Global Parkinson’s Disease Market Report : http://www.mrrse.com/frontierpharma-parkinsons-disease
At present, 302 products are being actively developed in the therapeutic pipeline of
Parkinson’s disease over a number of stages.
However, the mechanisms of action applied on the existing products compared to the ones in
the pipeline shows severe differences. The existing symptomatic treatments employed for PD
target neuromodulatory receptors; whereas, the current product pipeline features a
widespread array of neuroprotective therapies focusing on dysfunctional disease procedures.
The availability of ninety first-in-class products that holds 37% of the overall therapies in
pipeline is one of the major reason behind the diversity in the MoA for both, existing products
and pipeline products.
Since, the global PD industry is driven by first-in-class products compared to the non-first-inclass
ones; the development of this market is widely impacted by this factor. Although the
first-in-class products and therapies have indicated high abrasion rate in past, they have the
potential to modify the global PD treatment market for good.
As the global market for Parkinson’s disease is rising at a rapid pace, let’s take a look at the
future prospects the market is going to deal with:
· The overall revenue generated from sales in the PD markets of the U.S., Germany, France,
Italy, the U.K., Spain, and Japan is expected to register a slight decline from around US$2.7
billion in 2010 to an estimate of US$2.6 billion by 2020.

· The dominant dopamine agonists, ropinirole and pramipexole, are expected to trigger the
sales significantly. The increasing usage of Agilect, the MAO-B inhibitor, and the resurgence of
UCB’s Leganto/Neupro in the U.S. market with an even wider employment in the European
market are also projected to drive the market growth.
· In spite of the increased uptake of some key therapies currently functional in the market and
the announcement to launch 3 new agents (GlaxoSmithKline’s IPX-066, Newron
Pharmaceuticals’ safinamide, and Kyowa Hakko Kirin’s istradefylline) by 2020, the sales of
current agents and their impact on the market of emerging therapies is likely to be
suppressed by generic competition.
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Table of Contents:
1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3
2 Executive Summary 5
2.1 Highly Innovative and Diverse Pipeline 5
2.2 Alignment of Innovation to Disease Processes and Genetics 5
2.3 Deals Landscape Present Substantial Investment Opportunities 5
3 The Case for Innovation 6
3.1 Growing Opportunities for Biologic Products 7
3.2 Diversification of Molecular Targets 7
3.3 Innovative First-in-Class Product Developments Remain Attractive 7

3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 8
3.5 Sustained Innovation 8
3.6 GBI Research Report Guidance 9
4 Clinical and Commercial Landscape 10
4.1 Epidemiology 10
4.2 Disease Etiology 11
4.2.1 Exposure to Environmental Toxins 11
4.2.2 Genetic Causes of Familial Parkinson’s Disease 11
4.2.3 Susceptibility Genes for Parkinson’s Disease 13
4.3 Disease Pathophysiology 13
4.3.1 Basal Ganglia Anatomy and Physiology 13
4.3.2 Processes Underlying Neurodegeneration in Parkinson’s Disease 16
4.4 Disease Symptoms 20
4.5 Co-morbidities and Complications 20
4.6 Diagnosis 21
4.6.1 Classification 21
4.7 Prognosis and Disease Staging 23
4.8 Treatment Options 24
4.8.1 Pharmacological Treatment 24
4.8.2 Non-pharmacological Treatments 28
4.9 Marketed Product Overview 28
4.10 Overview of Marketed Products for Parkinson’s Disease 29

4.11 Efficacy and Safety of Marketed Products 31
4.12 Treatment Algorithm 33
4.13 Current Unmet Needs 35
5 Assessment of Pipeline Product Innovation 37
5.1 Parkinson’s Disease Pipeline by Molecule Type, Phase and Therapeutic Targets 37
5.2 Comparative Distribution of Programs between the Parkinson’s Disease Market and
Pipeline by Therapeutic Target Family 41
6 Signaling Network, Parkinson’s Disease Genetics and Innovation Alignment 46
6.1 The Complexity of Signaling Network in Central Nervous System 46
6.2 Signaling Pathways, Disease-Causing Mutations and First-in-Class Molecular Target
Integration 47
6.3 First-in-Class Target Matrix Assessment 51
7 First-in-Class Target and Pipeline Program Evaluation 54
7.1 Overview of Pipeline Programs Targeting ?-synuclein 54
7.2 Overview of Pipeline Programs Targeting LRRK2 57
7.3 Overview of Pipeline Programs Targeting Glial Cell-Derived Neurotrophic Factor 60
7.4 Overview of Pipeline Programs Targeting Progranulin 63
7.5 Overview of Pipeline Programs Targeting Cerebral Dopamine Neurotrophic Factor 66
7.6 Overview of Pipeline Programs Targeting Mesencephalic Astrocyte-Derived Neurotrophic
Factor 68
7.7 Overview of Pipeline Programs Targeting Tyrosine Receptor Kinase B 70
7.8 Overview of Pipeline Programs Targeting Metabotropic Glutamate Receptor 4 73

7.9 Overview of Pipeline Programs Targeting Metabotropic Glutamate Receptor 8 75
7.10 Overview of Pipeline Programs Targeting C-jun-N-Terminal Kinases 76
7.11 Overview of Pipeline Programs Targeting DJ-1 79
7.12 Overview of Pipeline Programs Targeting Reactive Oxygen Species 80
7.13 Overview of Pipeline Programs Targeting Heat Shock Transcription Factor 1 82
7.14 Overview of Pipeline Programs Targeting Heat Shock Protein 90 84
7.15 Overview of Pipeline Programs Targeting Growth Hormone Secretagogue Receptor 86
7.16 Overview of Pipeline Programs Targeting Protein Phosphatase 2A 88
7.17 Overview of Pipeline Programs Targeting Cytochrome P450 2D6 90
7.18 Conclusion 91
8 Deals and Strategic Consolidations 93
8.1 Industry-wide First-in-Class Deals 93
8.2 Parkinson’s Disease Deals Landscape 95
8.3 Licensing Deals 95
8.3.1 Molecule Type 97
8.3.2 Mechanism of Action 97
8.4 Co-development Deals 100
8.4.1 Mechanism of Action 101
8.5 First-in-Class Programs Not Involved in Licensing or Co-Development Deals 103
9 Appendix 106
9.1 Abbreviations 106

9.2 References 108
9.3 Contact Us 121
9.4 Disclaimer 121
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