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

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


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.

Parkinson’s Disease Treatment Pipeline Holds Significant Potential for Patients and Investors, says GBI Research

The Parkinson’s disease treatment pipeline presents a distinctly more promising landscape than the current market, which is burdened with unmet needs and a lack of disease-modifying drugs, says business intelligence provider GBI Research.

According to the company’s latest report*, the current Parkinson’s disease treatment market relies on neuromodulators, and although levodopa and dopaminergic treatments can be used to relieve symptoms, there is not an agent that has demonstrated neuroprotective properties to prevent or at least significantly slow neuronal cell death.

However, Angel Wong, Senior Analyst for GBI Research, states that the pipeline for the disease is highly active, consisting of 365 programs across all stages of development, and a diverse range of molecular targets.

Wong says: “The Parkinson’s disease treatment pipeline is characterized by a high level of innovation compared to the central nervous system segment and the industry as a whole, with 43% of all pipeline programs with disclosed molecular targets being first-in-class.

“While neuromodulators remain dominant in the pipeline, there are a number of targets which address different pathogenic mechanisms underlying neurodegeneration in Parkinson’s disease. This demonstrates a shift from traditional symptomatic treatments to disease-modifying therapies.”

The analyst adds that as the majority of potentially disease-modifying treatments are in the early stages of development, they are expected to fulfill a huge unmet need in the Parkinson’s disease treatment market in the long term.

GBI Research’s report also states that there are 94 first-in-class products available for strategic consolidations, meaning a vast number of investment opportunities are available for licensing or co-development deals in Parkinson’s disease therapeutics.

Wong explains: “There is substantial focus on early-stage collaboration in Parkinson’s disease treatment, especially for first-in-class products. This is predominantly driven by growing unmet needs, the current lack of disease-modifying therapies and a desire for risk-sharing.

“Several first-in-class products have demonstrated promising preclinical evidence, and have significant potential to become game-changing products, representing high-reward investments.”

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

This report provides analysis of the Parkinson’s disease treatment pipeline, stratified by stage of development, molecule type and molecular target. It includes information on the current clinical and commercial landscape, and the composition of the Parkinson’s disease therapeutics market in terms of dominant molecule types and targets, as well as highlighting current unmet needs.

This report was built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis conducted by GBI Research’s team of industry experts.

1 Table of Contents
1.1 List of Tables
1.2 List of Figures
2 Executive Summary
2.1 High Unmet Needs Remain in Parkinson’s Disease Market
2.2 Diverse and Innovative Pipeline to Shift Focus to Disease Modification
2.3 Deals Landscape Presents Substantial Investment Opportunities
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 Developments Remain Attractive
3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation
3.5 Sustained Innovation
3.6 GBI Research Report Guidance
4 Clinical and Commercial Landscape
4.1 Disease Overview
4.2 Epidemiology
4.3 Disease Etiology
4.3.1 Exposure to Environmental Toxins
4.3.2 Genetic Causes of Familial Parkinson’s Disease
4.3.3 Susceptibility Genes for Parkinson’s Disease
4.4 Disease Pathophysiology
4.4.1 Basal Ganglia Anatomy and Physiology
4.4.2 Process Underlying Neurodegeneration
4.5 Disease Symptoms
4.6 Co-morbidities and Complications
4.7 Diagnosis
4.8 Classification of Disease Stages
4.8.1 Hoehn and Yahr Scale
4.8.2 Unified Parkinson’s Disease Rating Scale
4.8.3 Scale for the Assessment of Positive Symptoms
4.8.4 Mini Mental State Examination
4.9 Prognosis and Disease Staging
4.10 Treatment Options
4.10.1 Pharmacotherapy
4.10.2 Non-pharmacological Treatment
4.11 Overview of Marketed Products
4.11.1 Molecule Type and Target Analysis
4.11.2 Treatment Algorithm
4.12 Current Unmet Needs
5 Assessment of Pipeline Product Innovation
5.1 Parkinson’s Disease Pipeline by Molecule Type, Phase and Therapeutic Target
5.2 Comparative Distribution of Programs between Parkinson’s Disease Market and Pipeline by Therapeutic Target Family
5.3 First-in-Class Pipeline Programs Targeting Novel Molecular Targets
6 Signaling Pathways, Genetics and Innovation Alignment
6.1 The Complexity of Signaling Networks in the Central Nervous System
6.2 Signaling Pathways and First-in-Class Molecular Target Integration
6.3 First-in-Class Target Matrix Assessment
7 First-in-Class Target Evaluation
7.1 Pipeline Programs Targeting α-synuclein
7.2 Pipeline Programs Targeting DJ-1
7.3 Pipeline Programs Targeting Parkin
7.4 Pipeline Programs Targeting High Affinity Nerve Growth Factor Receptor
7.5 Pipeline Programs Targeting C-jun N-Terminal Kinase
7.6 Pipeline Programs Targeting Leucine-Rich Repeat Kinase 2
7.7 Pipeline Programs Targeting Growth Hormone Secretagogue Receptor Type 1
7.8 Pipeline Programs Targeting Metabotropic Glutamate Receptor 4
7.9 Pipeline Programs Targeting NAD-dependent Protein Deacetylase Sirtuin-2
7.10 Overview of Pipeline Programs Targeting Progranulin
7.11 Conclusion
8 Deals and Strategic Consolidations
8.1 Industry-Wide First-in-Class Deals
8.2 Licensing Deals
8.3 Co-development Deals
8.4 First-in-Class Programs not Involved in Licensing or Co-development Deals
9 Appendix
9.1 Abbreviations
9.2 Bibliography
9.3 Research Methodology
9.4 Secondary Research
9.4.1 Marketed Product Heatmaps and Treatment Algorithm
9.4.2 Pipeline Analysis
9.4.3 First-in-Class Matrix Assessment
9.4.4 First-in-Class Target Profiles
9.4.5 Licensing and Co-development Deals
9.5 Contact Us
9.6 Disclaimer
1.1 List of Tables
Table 1: Definition of the Stages of Disability in Hoehn and Yahr Scale, 1967
Table 2: Evaluation of Disability by Unified Parkinson Disease Rating Scale, 2013
1.2 List of Figures
Figure 1: Innovation Trends in Product Approvals, 1987-2013
Figure 2: Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013
Figure 3: Indirect and Direct Pathways in Basal Ganglia
Figure 4: Molecular Targets of Marketed Products
Figure 5: Treatment Algorithm of Parkinson’s Disease
Figure 6: Efficacy and Safety of Treatments for Early Parkinson’s Disease
Figure 7: Efficacy and Safety of Treatments for Advanced Parkinson’s Disease
Figure 8: Efficacy and Safety of Treatment for Non-Motor Symptoms in Parkinson’s Disease
Figure 9: Developmental Pipeline Overview
Figure 10: Parkinson’s Disease Pipeline by Molecular Target
Figure 11: Parkinson’s Disease Pipeline by Molecular Target
Figure 12: Molecular Target Category Comparison, Pipeline and Marketed Products
Figure 13: Molecular Target Category Comparison, Pipeline First-in-Class and Established Molecular Targets
Figure 14: Parkinson’s Disease, Global, First-in-Class Pipeline Products
Figure 15: First-in-Class Molecular Target Analysis Matrix
Figure 16: Data and Evidence for α-synuclein as a Therapeutic Target
Figure 17: Pipeline Programs Targeting α-synuclein
Figure 18: Data and Evidence for DJ-1 as a Therapeutic Target
Figure 19: Pipeline Programs Targeting DJ-1
Figure 20: Data and Evidence for Parkin as a Therapeutic Target
Figure 21: Pipeline Programs Targeting Parkin
Figure 22: Data and Evidence for High Affinity Nerve Growth Factor Receptor as a Therapeutic Target
Figure 23: Pipeline Programs Targeting High Affinity Nerve Growth Factor Receptor
Figure 24: Data and Evidence for C-Jun N-Terminal Kinases as a Therapeutic Target
Figure 25: Pipeline Programs Targeting C-Jun N-Terminal Kinase
Figure 26: Data and Evidence for Leucine-Rich Repeat Kinase 2 as a Therapeutic Target
Figure 27: Pipeline Programs Targeting Leucine-Rich Repeat Kinase 2
Figure 28: Data and Evidence for Growth Hormone Secretagogue Receptor as a Therapeutic Target
Figure 29: Pipeline Programs Targeting Growth Hormone Secretagogue Receptor Type 1
Figure 30: Data and Evidence for Metabotropic Glutamate Receptor 4 as a Therapeutic Target
Figure 31: Pipeline Programs Targeting Metabotropic Glutamate Receptor 4
Figure 32: Data and Evidence for NAD-dependent Protein Deacetylase Sirtuin-2 as a Therapeutic Target
Figure 33: Pipeline Programs Targeting NAD-Dependent Protein Deacetylase Sirtuin-2
Figure 34: Data and Evidence for Progranulin as a Therapeutic Target
Figure 35: Pipeline Programs Targeting Progranulin
Figure 36: Industry-Wide Deals by Stage of Development, 2006-2014
Figure 37: Industry-Wide Deals by Stage of Development, 2006-2014
Figure 38: Licensing Deals, 2006-2015
Figure 39: Licensing Deals by Molecule Type, 2006-2015
Figure 40: Licensing Deals by Molecular Target, 2006-2015
Figure 41: Summary of Licensing Deals, 2006-2015
Figure 42: Co-development Deals, 2006-2015
Figure 43: Co-development Deals by Phase and Molecule Type, 2006-2015
Figure 44: Co-development Deals by Molecular Target, 2006-2015
Figure 45: Summary of Co-development Deals, 2006-2015
Figure 46: First-in-Class Programs with no Recorded Prior Deal Involvement, 2006-2015

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