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Frontier Pharma: Fatty Liver Disease - High Degree of First-in-Class Innovation, Dominated by Nuclear Receptor-Targeting NASH Products

Published By :

GBI Research

Published Date : Mar 2017

Category :

Diseases & Conditions

No. of Pages : 76 Pages

Fatty liver disease (FLD) comprises a spectrum of chronic liver disorders characterized by excessive lipid accumulation in the liver (steatosis), which may lead to inflammation (steatohepatitis) and fibrosis. It has the potential to progress to end-stage liver diseases such as cirrhosis, liver cancer and liver failure, and is also associated with numerous complications and co-morbidities, including cardiovascular and metabolic diseases. FLD can be divided into non-alcoholic FLD (NAFLD) and alcoholic FLD (AFLD), depending on the patent’s history of alcohol use.

FLD is the most common chronic liver disease in the world, and its global prevalence has increased rapidly in the past several decades. The worldwide prevalence of FLD is estimated at 20–45% in the general population, and up to 90% in obese patients. There is a broad consensus to describe the condition as the hepatic manifestation of metabolic syndrome, and it is closely associated with obesity, diabetes and dyslipidemia. NAFLD has become the main driver of the rapid growth of FLD prevalence, mainly due to the rising prevalence of obesity.

FLD is increasingly recognized as a major global health problem. However, despite this the FLD market is still in its infancy, with no FDA-approved drugs for this indication, and only a limited number of generic drugs approved in non-US markets in recent years.

Due to the increasing health burden of FLD and the lack of therapeutic options, there is a pressing need to develop pharmacological strategies. This is especially the case for patients with steatohepatitis, who are at the greatest risk of developing cirrhosis or liver cancer, which can lead to liver failure. Due to the pathophysiological complexity of FLD and its diverse population, different therapeutic agents are likely to be needed to tackle the lipotoxicity, inflammation and fibrogenesis that drive FLD progression.

FLD has an active pipeline and first-in-class products account for a considerable proportion, which is very promising considering the level of unmet need and lack of approved treatment options. First-in-class innovation is concentrated heavily at the early drug development stages, and prominent first-in-class molecular targets include nuclear receptors, immune mediators, and molecules involved in lipid synthesis. Additionally, the first-in-class targets identified show considerable diversity, reflecting the multifaceted aspects of FLD pathophysiology.

Scope

FLD comprises a diverse patient population with significant unmet needs

  •  What is the pathophysiology of FLD?
  •  What are the common co-morbidities and complications?
  •  What are the most significant unmet needs within the market?

The FLD pipeline is relatively large and has a high degree of first-in-class innovation

  •  Which molecule types and molecular targets are most prominent within the pipeline?
  • Which first-in-class targets are most promising?
  • How does the ratio of first-in-class targets to first-in-class products differ by stage of development and molecular target class?

The FLD deal landscape shows rising deal volumes and considerable investment opportunities

  • Do FLD products attract high deal values?
  • Which molecule types and molecular targets dominate the deals landscape?
  • Which first-in-class pipeline products have no prior involvement in licensing or co-development deals?

Reasons to buy

This report will allow you to - 

  • Appreciate the current clinical and commercial landscapes by considering disease pathogenesis, etiology, epidemiology, symptoms, co-morbidities and complications, and treatment options and algorithms.
  • Visualize the composition of the FLD market in terms of dominant classes of therapies. Key unmet needs are identified to allow a competitive understanding of gaps in 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 a proprietary matrix tailored to FLD, all first-in-class targets in the pipeline have been assessed and ranked according to clinical potential. Promising early-stage targets have been further reviewed in greater detail.
  • Consider first-in-class pipeline products with no prior involvement in licensing and co-development deals that may represent potential investment opportunities.

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 Urgent Unmet Need Driven By Rising Prevalence 5
2.2 High Degree of First-in-Class Innovation 5
2.3 Rising Deal Volumes and Considerable Investment Opportunities in First-in-Class Product Development 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 Development Remains 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 8

4 Clinical and Commercial Landscape 10
4.1 Disease Overview 10
4.2 Disease Symptoms 11
4.3 Epidemiology 11
4.4 Etiology and Risk Factors 11
4.4.1 Environmental Factors 11
4.4.2 Host Factors 12
4.5 Pathophysiology 13
4.5.1 Steatosis 13
4.5.2 Hepatocellular Injury and Inflammation 14
4.5.3 Fibrosis 15
4.5.4 Conclusion 15
4.6 Co-morbidities and Complications 16
4.7 Diagnosis 16
4.7.1 Diagnosis of Fatty Liver Disease 16
4.7.2 Disease Prognosis 17
4.8 Treatment 17
4.8.1 Lifestyle Intervention 17
4.8.2 Pharmacological Treatment 18
4.8.3 Liver Transplantation 19

5 Assessment of Pipeline Product Innovation 20
5.1 Pipeline by Stage of Development, Molecule Type and Molecular Target 20
5.2 First-in-Class Programs Targeting Novel Molecular Targets 27
5.3 ASH 33
5.4 AFLD 33
5.5 Steatohepatitis (unspecified) 34
5.6 FLD (unspecified) 34
5.7 Table of All Pipeline Products 35

6 Signaling Network, Disease Causation and Innovation Alignment 40
6.1 Complexity of Signaling Networks in FLD 40
6.2 Signaling Pathways and First-in-Class Molecular Target Integration 40
6.3 First-in-Class Matrix Assessment 41

7 First-in-Class Target Evaluation 44
7.1 Pipeline Programs Targeting Connective Tissue Growth Factor 44
7.2 Pipeline Programs Targeting Sterol Regulatory Element-Binding Protein 1 and 2 45
7.3 Pipeline Programs Targeting Acetyl-CoA Carboxylase 1 and 2 48
7.4 Pipeline Programs Targeting Galectin 3 50
7.5 Pipeline Programs Targeting Cathepsin B 52
7.6 Pipeline Programs Targeting NACHT, KKR and PYD Domains-Containing Protein 3 53
7.7 Pipeline Programs Targeting P2Y Purinoceptor 13 55
7.8 Pipeline Programs Targeting Serpin H1 56
7.9 Conclusion 58

8 Strategic Consolidations 59
8.1 Industry-Wide First-in-Class Deals 59
8.2 Licensing Deals 60
8.2.1 Deals by Region, Value and Year 60
8.2.2 Deals by Stage of Development and Value 62
8.2.3 Deals by Molecule Type, Molecular Target and Value 62
8.2.4 List of Deals with Disclosed Deal Values 63
8.3 Codevelopment Deals 64
8.3.1 Deals by Region, Value and Year 64
8.3.2 Deals by Stage of Development and Value 65
8.3.3 Deals by Molecule Type, Molecular Target and Value 65
8.3.4 List of Deals with Disclosed Deal Values 66
8.4 First-in-Class Programs with and without Prior Involvement in Licensing and Co-development Deals 66

9 Appendix 69
9.1 References 69
9.2 Abbreviations 73
9.3 Research Methodology 74
9.3.1 Data integrity 75
9.3.2 Innovative and meaningful analytical techniques and frameworks: 75
9.3.3 Evidence based analysis and insight: 75
9.4 Secondary Research 75
9.4.1 Market Analysis 75
9.4.2 Pipeline Analysis 75
9.4.3 First-in-Class Matrix Assessment 75
9.4.4 First-in-Class Target Profiles 76
9.4.5 Licensing and Co-Development Deals 76
9.5 Contact Us 76
9.6 Disclaimer 76

List of Table

Table 1: Fatty Liver Disease Therapeutics Market, Survival Rates of Simple Steatosis and NASH/ASH, 2017 17
Table 2: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Connective Tissue Growth Factor, 2017 45
Table 3: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting Connective Tissue Growth Factor, 2017 45
Table 4: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Sterol Regulatory Element-Binding Protein 1, 2017 46
Table 5: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Sterol Regulatory Element-Binding Protein 2, 2017 47
Table 6: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting Sterol Regulatory Element-Binding Protein 1, 2017 47
Table 7: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting Sterol Regulatory Element-Binding Protein 2, 2017 48
Table 8: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Acetyl-CoA Carboxylase 1, 2017 49
Table 9: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Acetyl-CoA Carboxylase 2, 2017 50
Table 10: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting Acetyl-CoA Carboxylase 1 and 2, 2017 50
Table 11: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Galectin 3, 2017 51
Table 12: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting Galectin 3, 2017 52
Table 13: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Cathepsin B, 2017 53
Table 14: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting Cathepsin B, 2017 53
Table 15: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of NACHT, KKR and PYD Domains-Containing Protein 3 Receptor, 2017 54
Table 16: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting NACHT, KKR and PYD Domains-Containing Protein 3, 2017 55
Table 17: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of P2Y Purinoceptor 13, 2017 56
Table 18: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting P2Y Purinoceptor 13, 2017 56
Table 19: Fatty Liver Disease Therapeutics Market, Global, Key Features and Pipeline Activity of Serpin H1, 2017 57
Table 20: Fatty Liver Disease Therapeutics Market, Global, Pipeline Programs Targeting Serpin H1, 2017 57

List of Chart

Figure 1: Fatty Liver Disease Therapeutics Market, US, Innovation Trends in Product Approvals, Number of Product Approvals by FDA and Five-Year Moving Average of Product Approvals, 1987-2014 6
Figure 2: Fatty Liver Disease Therapeutics Market, Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval ($m), 2006-2013 8
Figure 3: Fatty Liver Disease Therapeutics Market, Global, Overall Pharmaceutical Industry Pipeline by Therapy Area, 2017 20
Figure 4: Fatty Liver Disease Therapeutics Market, Global, Pipeline by Stage of Development and Molecule Type, 2017 21
Figure 5: Fatty Liver Disease Therapeutics Market, Global, Non-Alcoholic Steatohepatitis and Non-Alcoholic Fatty Liver Disease Pipeline by Stage of Development and Molecule Type, 2017 23
Figure 6: Fatty Liver Disease Therapeutics Market, Global, Pipeline by Molecular Target and Stage of Development, 2017 25
Figure 7: Fatty Liver Disease Therapeutics Market, Non-Alcoholic Steatohepatitis and Non-Alcoholic Fatty Liver Disease, Global, Pipeline by Molecular Target and Stage of Development, 2017 26
Figure 8: Fatty Liver Disease Therapeutics Market, Global, Distribution of Pipeline First-in-Class and Non-First-in-Class Products by Stage of Development and Molecular Target, 2017 27
Figure 9: Fatty Liver Disease Therapeutics Market, Global, Non-Alcoholic Steatohepatitis and Non-Alcoholic Fatty Liver Disease, Distribution of Pipeline First-in-Class and Non-First-in-Class Products by Molecular Target, 2017 28
Figure 10: Fatty Liver Disease Therapeutics Market, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Products by Stage of Development (%), 2017 29
Figure 11: Fatty Liver Disease Therapeutics Market, Global, Percentage Distribution of First-in-Class and Non-First-in-Class Pipeline Products by Molecular Target (%), 2017 30
Figure 12: Fatty Liver Disease Therapeutics Market, Global, Ratio of First-in-Class Products to First-in-Class Targets by Stage of Development, 2017 31
Figure 13: Fatty Liver Disease Therapeutics Market, Global, Ratio of First-in-Class Products to First-in-Class Targets by Molecular Target, 2017 32
Figure 14: Fatty Liver Disease Therapeutics Market, Alcoholic Steatohepatitis, Global, Pipeline Products, 2017 33
Figure 15: Fatty Liver Disease Therapeutics Market, Alcoholic Fatty Liver Disease, Global, Pipeline Products, 2017 33
Figure 16: Fatty Liver Disease Therapeutics Market, Steatohepatitis (Unspecified), Global, Pipeline Products, 2017 34
Figure 17: Fatty Liver Disease Therapeutics Market, Fatty Liver Disease (Unspecified), Global, Pipeline Products, 2017 34
Figure 18: Fatty Liver Disease Therapeutics Market, Global, Pipeline Products, 2017 (Part 1) 35
Figure 19: Fatty Liver Disease Therapeutics Market, Global, Pipeline Products, 2017 (Part 2) 36
Figure 20: Fatty Liver Disease Therapeutics Market, Global, Pipeline Products, 2017 (Part 3) 37
Figure 21: Fatty Liver Disease Therapeutics Market, Global, Pipeline Products, 2017 (Part 4) 38
Figure 22: Fatty Liver Disease Therapeutics Market, Global, Pipeline Products, 2017 (Part 5) 39
Figure 23: Fatty Liver Disease Therapeutics Market, Global, First-in-Class Matrix Assessment, 2017 (Part 1) 42
Figure 24: Fatty Liver Disease Therapeutics Market, Global, First-in-Class Matrix Assessment, 2017 (Part 2) 43
Figure 25: Fatty Liver Disease Therapeutics Market, Global, Industry-Wide Deals by Stage of Development and First-in-Class Status, 2006-2014 59
Figure 26: Fatty Liver Disease Therapeutics Market, Global, Industry-Wide Licensing Deals by Deal Value, Upfront Payment Value, Stage of Development and First-in-Class Status, 2006-2014 60
Figure 27: Fatty Liver Disease Therapeutics, Global, Licensing Deals by Region, Value and Year, 2006-2017 61
Figure 28: Fatty Liver Disease Therapeutics, Global, Licensing Deals by Deal Value, Upfront Payment Value and Stage of Development, 2006-2017 62
Figure 29: Fatty Liver Disease Therapeutics, Global, Number and Aggregate Deal Value of Licensing Deals by Molecule Type, 2006-2017 62
Figure 30: Fatty Liver Disease Therapeutics, Global, Number and Aggregate Deal Value of Licensing Deals by Molecular Target, 2006-2017 63
Figure 31: Fatty Liver Disease Therapeutics, Global, Licensing Deals with Disclosed Deal Values, 2006-2017 63
Figure 32: Fatty Liver Disease Therapeutics, Global, Co-development Deals by Region, Value and Year, 2006-2017 64
Figure 33: Fatty Liver Disease Therapeutics, Global, Co-development Deals by Stage of Development, Deal Value and Upfront Payment Value, 2006-2017 65
Figure 34: Fatty Liver Disease Therapeutics, Global, Number and Aggregate Deal Value of Co-development Deals by Molecule Type, 2006-2017 65
Figure 35: Fatty Liver Disease Therapeutics, Global, Number of Co-development Deals by Molecular Target, 2006-2017 66
Figure 36: Fatty Liver Disease Therapeutics, Global, Co-development Deals with Disclosed Deal Values, 2006-2017 66
Figure 37: Fatty Liver Disease Therapeutics Market, Global, First-in-Class Pipeline Products with Prior Involvement in Licensing or Co-development Deals, 2017 67
Figure 38: Fatty Liver Disease Therapeutics Market, Global, First-in-Class Pipeline Products with No Disclosed Prior Involvement in Licensing or Co-development Deals, 2017 68

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