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Frontier Pharma: Liver Cancer - Identifying and Commercializing First-in-Class Innovation

出版商 GBI Research 商品編碼 326290
出版日期 內容資訊 英文 80 Pages
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肝臟癌症:First-in-Class創新的識別、商業化 Frontier Pharma: Liver Cancer - Identifying and Commercializing First-in-Class Innovation
出版日期: 2014年12月31日 內容資訊: 英文 80 Pages


第1章 目錄

第2章 摘要整理

第3章 創新案例

  • 生物製藥的機會擴大
  • 分子標的多樣化
  • 創新的First-in-Class產品的開發依然富有魅力
  • 法規、醫療費償付政策變化為First-in-Class產品創新的有利作用
  • 創新的持續
  • GBI Research 調查指導

第4章 臨床、商業形勢

  • 疾病概要
  • 症狀
  • 流行病學、病因
  • 病理學
    • 肝細胞癌症
    • 膽管癌症
    • 結論
  • 診斷
    • 臨床表現
    • 胎兒球蛋白 (AFP)
    • 診斷影像、掃描
    • 篩檢
    • 切片檢查
    • 分類
  • 治療選項
  • 治療流程
    • 最初線
  • 肝臟癌症市售產品概要
    • 分子類型、標的分析
    • 肝臟癌症市場上創新的產品
    • 未滿足需求

第5章 開發中產品創新的評估

  • 肝臟癌症開發平台:各分子類型、階段,及治療標的
  • 肝臟癌症和開發平台間的計劃分配比較:各支援治療標的
  • 以新分子標的為目標的First-in-Class開發平台計劃

第6章 肝臟癌症支援專利分析

第7章 信號傳達網路、生病的原因,及創新的調整

  • 癌症的信號傳達網路的複雜
  • 引起突然變異的信號途徑疾病及First-in-Class分子標的整合
  • First-in-Class標的矩陣評估

第8章 First-in-Class標的評估

  • 以β-連環蛋白做為目標的開發平台計劃
  • 以FGFR4 為目標的開發平台計劃
  • 以GPR55 為目標的開發平台計劃
  • 以蛋白質j二硫鍵異構酶(PDI) 為目標的開發平台計劃
  • 以端粒酶為目標的開發平台計劃
  • 以 Delta-狀1同源(DLK1) 為目標的開發平台計劃
  • 以 eIF5A 為目標的開發平台計劃
  • 以 FAK 為目標的開發平台計劃
  • 把TGFβ受體做為目標的開發平台計劃
  • 結論

第9章 契約、策略性整合

  • 產業規模的First-in-Class契約
  • 肝臟癌症的契約形勢
  • 授權契約
  • 競爭開發契約
  • 未包含在授權或共同開發契約的First-in-Class計劃

第10章 附錄



Executive Summary

Large and Diverse Pipeline

The liver cancer pipeline contains 238 products in active development, approximately 47% of which are first-in-class. The percentage of the pipeline devoted to innovative products is considerably larger than both the industry and oncology average, which is a promising sign for novel therapeutics reaching the liver cancer market.

The contrast between the market and pipeline is vast. Analysis showed that the market contains 70 products, the majority of which are generic formulations of chemotherapies that are not frequently used in treatment, particularly in advanced-stage patients. Nexavar (sorafenib) is the dominant therapeutic on the market, and is also the only targeted therapy that is in regular use for advanced-stage liver cancer patients. However, pipeline analysis revealed that targeted therapies aimed at the underlying oncogenic signaling pathways are under much greater focus in the pipeline than in the market. The success of targeted therapies across the oncology market as a whole implies that the diversity and innovation in the pipeline is a promising sign, with products currently in development having the potential to transform and improve the relatively open liver cancer market.

Alignment of First-in-Class Molecular Targets with Disease Causation

The liver cancer pipeline is showing signs of adapting to the increasing understanding of aberrant signaling pathways and causes of liver cancer. A large portion of pipeline products target components of known dysfunctional signaling pathways, such as Wnt/ß-catenin signaling, which is commonly mutated in liver cancer tumor samples. By aligning the treatment with specific disease-causing features, the damaging off-target cytotoxic effects of treatment can be reduced, resulting in safer and more efficacious therapies.

GBI Research's analysis identified substantial variation in the alignment of first-in-class products to underlying dysfunctional signaling at protein and genetic level. The first-in-class products were compared in an in-depth analysis using various parameters to measure the potential of each target, with the most promising targets being further substantiated by published clinical and scientific evidence. Results of the analysis suggested that first-in-class status is not a feature that, in its own right, will create a successful product. However, there are a large number of first-in-class products backed by clinical and Preclinical data that are exciting future prospects for the liver cancer market.

Analysis of Patent Data

The report features an analysis of granted patent applications in the liver cancer market, which was used as an indication of innovation at the earliest stage of product development. Patent analysis provides an insight into the pre-developmental landscape, and identifies long-term future trends within a disease market. In liver cancer, the trend in patent applications in terms of predominant molecular targets reflects the pipeline landscape, suggesting that liver cancer therapeutics will continue to target key oncogenic signaling pathways in the long term.

The frequency at which companies apply for patents within the market helps to identify companies that are trying to establish themselves or increase their liver cancer market share. This information identifies not only potential competitors, but also companies that may seek strategic partnerships to enter drug development.

First-in-Class Products in Licensing and Co-Development Deals

The deals landscape for liver cancer has been relatively active in recent years, with 62 licensing deals and 23 co-development deals between 2006 and 2014. However, the number pertaining to first-in-class products is very low.

A comparative analysis of the deals revealed that first-in-class products have the potential to command substantially higher deal values than non-first-in-class products, which is a reflection of their market potential and importance. A total of 68 first-in-class products that are currently in development have not yet been entered into a licensing or co-development deal. In a transforming market that will favor innovative, targeted therapies with a strong clinical record, there are numerous opportunities for strategic alliances to bolster a first-in-class portfolio or fund clinical development. Although not all are aligned to disease-causing signaling pathways, many are supported by robust scientific and clinical data, making them attractive prospects as both therapeutics and investment opportunities.


The report includes -

  • A brief introduction to liver cancer, including symptoms, pathophysiology, and an overview of pharmacotherapy and treatment algorithms
  • Coverage of the changing molecular target landscape and particular points of innovation in the pipeline
  • A comprehensive review of the pipeline for first-in-class therapies, analyzed by stage of development, molecule type and molecular target
  • Analysis of patent trends and patent families in liver cancer
  • Identification and assessment of first-in-class molecular targets, with a particular focus on early-stage programs of which clinical utility has yet to be evaluated, as well as literature reviews of novel molecular targets
  • Industry-wide analysis of first-in-class deals compared to non-first-in-class deals
  • An assessment of the licensing and co-development deal landscape for liver cancer therapies, and benchmarking of deals comparing first-in-class and non-first-in-class-products

Reasons to buy

  • The report will enable business development and enable marketing executives to strategize their product launches by allowing them to -
  • Understand the focal shifts in molecular targets in the liver cancer pipeline
  • Understand the distribution of pipeline programs by phase of development, molecule type and molecular target
  • Understand the patent trends in liver cancer and what this means for long-term innovation
  • Access a scientific and clinical analysis of first-in-class developmental programs for liver cancer, benchmarked against non-first-in-class targets
  • Assess the valuations of licensed and co-developed liver cancer treatments
  • Access a list of the first-in-class therapies potentially open to deal-making opportunities

Table of Contents

1. Table of Contents

  • 1.1. List of Tables
  • 1.2. List of Figures

2. Executive Summary

  • 2.1. Unmet Needs and Limited Market Drive an Innovative Pipeline
  • 2.2. Targeted Therapies Segment to Expand over the Coming Decade
  • 2.3. Active Deals Landscape Reflects the Dynamic Pipeline

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. Symptoms
  • 4.3. Epidemiology and Etiology
  • 4.4. Pathophysiology
    • 4.4.1. Hepatocellular Carcinoma
    • 4.4.2. Cholangiocarcinoma
    • 4.4.3. Conclusion
  • 4.5. Diagnosis
    • 4.5.1. Clinical Presentation
    • 4.5.2. Alpha-Fetoprotein
    • 4.5.3. Diagnostic Imaging and Scans
    • 4.5.4. Screening
    • 4.5.5. Biopsy
    • 4.5.6. Classification
  • 4.6. Treatment Options
  • 4.7. Treatment Algorithm
    • 4.7.1. First Line
  • 4.8. Overview of Marketed Products for Liver Cancer
    • 4.8.1. Molecular Type and Target Analysis
    • 4.8.2. Innovative Products in the Liver Cancer Market
    • 4.8.3. Unmet Needs

5. Assessment of Pipeline Product Innovation

  • 5.1. Liver Cancer Pipeline by Molecule Type, Phase and Therapeutic Target
  • 5.2. Comparative Distribution of Programs between the Liver Cancer Market and Pipeline by Therapeutic Target Family
  • 5.3. First-in-Class Pipeline Programs Targeting Novel Molecular Targets

6. Liver Cancer Patent Family Analysis

7. Signaling Network, Disease Causation and Innovation Alignment

  • 7.1. The Complexity of Signaling Networks in Oncology
  • 7.2. Signaling Pathways Disease-Causing Mutations and First-in-Class Molecular Target Integration
  • 7.3. First-in-Class Target Matrix Assessment

8. First-in-Class Target Evaluation

  • 8.1. Pipeline Programs Targeting β-Catenin
  • 8.2. Pipeline Programs Targeting FGFR4
  • 8.3. Pipeline Programs Targeting GPR55
  • 8.4. Pipeline Programs Targeting Protein Disulfide Isomerase
  • 8.5. Pipeline Programs Targeting Telomerase
  • 8.6. Pipeline Programs Targeting Delta-Like 1 Homolog
  • 8.7. Pipeline Programs Targeting eIF5A
  • 8.8. Pipeline Programs Targeting FAK
  • 8.9. Pipeline Programs Targeting TGFβ Receptor
  • 8.10. Conclusion

9. Deals and Strategic Consolidations

  • 9.1. Industry-wide First-in-Class Deals
  • 9.2. Liver Cancer Deals Landscape
  • 9.3. Licensing Deals
    • 9.3.1. Molecule Types
    • 9.3.2. Mechanism of Action
  • 9.4. Co-Development Deals
    • 9.4.1. Molecule Types
    • 9.4.2. Mechanism of Action
  • 9.5. First-In-Class Programs Not Involved in Licensing or Co-Development Deals

10. Appendix

  • 10.1. References
  • 10.2. Abbreviations
  • 10.3. Contact Us
  • 10.4. Disclaimer

List of Tables

  • Table 1: Liver Cancer, Organizations Frequently Applying for Chemical Entity Patent Families, (2008-2012)

List of Figures

  • Figure 1: Innovation Trends in Product Approvals
  • Figure 2: Sales Performance of First-in-Class and Non-First-in-Class Product Post Marketing Approval
  • Figure 3: Overview of Marketed Products
  • Figure 4: Overview of Pipeline Products
  • Figure 5: Breakdown of Pipeline Molecular Targets
  • Figure 6: Pipeline Molecular Targets by Stage of Development
  • Figure 7: Molecular Target Category Comparison, Pipeline and Marketed Products
  • Figure 8: Molecular Target Category Comparison, Pipeline First-in-Class and Established Molecular Targets
  • Figure 9: List of First-in-Class Pipeline Products (Part 1)
  • Figure 10: List of First-in-Class Pipeline Products (Part 2)
  • Figure 11: Liver Cancer, US and EU, Patent Families Filed and Granted by Year, 2008-2012
  • Figure 12: Liver Cancer, US and EU, Granted Patent Families by Molecular Target, 2008-2012
  • Figure 13: Liver Cancer, US and EU, Granted Patent Families by Molecular Target Family, 2008-2012
  • Figure 14: Liver Cancer, US and EU, Molecular Targets Identified in Patents (Part 1), 2008-2012
  • Figure 15: Liver Cancer, US and EU, Molecular Targets Identified in Patents (Part 2), 2008-2012
  • Figure 16: Liver Cancer, US and EU, Molecular Targets Identified in Patents (Part 3), 2008-2012
  • Figure 17: Signaling Networks of Functional Families in Liver Cancer
  • Figure 18: Target Matrix Assessment (Part 1)
  • Figure 19: Target Matrix Assessment (Part 2)
  • Figure 20: Target Matrix Assessment (Part 3)
  • Figure 21: Target Matrix Assessment (Part 4)
  • Figure 22: Target Matrix Assessment (Part 5)
  • Figure 23: Data and Evidence for β-Catenin as a Therapeutic Target
  • Figure 24: Pipeline Products Targeting β-Catenin
  • Figure 25: Data and Evidence for FGFR4 as a Therapeutic Target
  • Figure 26: Pipeline Products Targeting FGFR4
  • Figure 27: Data and Evidence for GPR55 as a Therapeutic Target
  • Figure 28: Pipeline Products Targeting GPR55
  • Figure 29: Data and Evidence for PDI as a Therapeutic Target
  • Figure 30: Pipeline Products Targeting PDI
  • Figure 31: Data and Evidence for Telomerase as a Therapeutic Target
  • Figure 32: Pipeline Products Targeting Telomerase
  • Figure 33: Data and Evidence for DLK1 as a Therapeutic Target
  • Figure 34: Pipeline Products Targeting DLK1
  • Figure 35: Data and Evidence for eIF5A as a Therapeutic Target
  • Figure 36: Pipeline Products Targeting eIF5A
  • Figure 37: Data and Evidence for FAK as a Therapeutic Target
  • Figure 38: Pipeline Products Targeting FAK
  • Figure 39: Data and Evidence for TGFβR1 as a Therapeutic Target
  • Figure 40: Pipeline Products Targeting TGFβR1
  • Figure 41: Industry Wide Deals by Stage of Development, 2006-2014
  • Figure 42: Industry Licensing Deal Values by Stage of Development, 2006-2014
  • Figure 43: Licensing Agreements, 2006-2014
  • Figure 44: Licensing Agreements by Phase, 2006-2014
  • Figure 45: Licensing Agreements by Territory, 2006-2014
  • Figure 46: Licensing Agreements by Molecule Type, 2006-2014
  • Figure 47: Licensing Agreements by Molecular Target, 2006-2014
  • Figure 48: Co-development Agreements, 2006-2014
  • Figure 49: Co-development Agreements by Territory, 2006-2014
  • Figure 50: Co-development Agreements by Molecule Type, 2006-2014
  • Figure 51: Co-development Agreements by Molecular Target, 2006-2014
  • Figure 52: List of First-in-Class Pipeline Products Not Involved in Deals (Part 1)
  • Figure 53: List of First-in-Class Pipeline Products Not Involved in Deals (Part 2)
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