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Frontier Pharma: Glioblastoma Multiforme - Cancer Immunotherapies Dominate First-in-Class Product Innovation

Frontier Pharma: Glioblastoma Multiforme - Cancer Immunotherapies Dominate First-in-Class Product Innovation

Summary

Glioblastoma multiforme (GBM) is a grade IV tumor that arises from astrocytes. It is the most common and aggressive human brain tumor, accounting for 15.4% of all primary brain tumors and 60-75% of all astrocytomas. It has a peak incidence between 55 and 84 years of age, with the median age of diagnosis being 64 years. In the US and EU, the annual incidence was estimated to be three to four cases per 100,000 people. GBM has a high degree of intratumoral heterogeneity, which is associated with poor prognosis and the development of drug resistance.

The GBM market is characterized by a small selection of marketed product options, consisting of chemotherapies, cancer immunotherapies and receptor tyrosine kinase inhibitor products. The pipeline is moderately large, with 512 products active across all stages of development. First-in-class products only constitute approximately a quarter of the pipeline, and represent 31% of products with a disclosed target. The most widely studied group of first-in-class targets are the receptor tyrosine kinase and ligand inhibitors, a trend that has been heavily influenced by the success of Avastin, and possibly successful EGFR inhibitors such as Tarceva that are used in the treatment of other oncology indications.

Potential driving factors for the market include a typically poor outcome for treated patients, a growing patient pool if disease prognosis can be improved, a lack of approved options in the market, and a strong understanding of the disease pathophysiology that has developed over the last decade, facilitating the development of novel compounds that may fulfill the unmet needs.

Overall, due to the highly complex and polygenic nature of GBM, which has numerous subtype classifications, it is unlikely that the inhibition of a single target will be sufficient to substantially improve patient prognosis. Instead, it is more likely that the concurrent use of multiple targeted therapies, along with other available modes of therapy, may improve treatment outcomes. First-in-class targets analyzed in this report have shown encouraging efficacy profiles, and some show the ability to chemosensitize cancer cells.

Scope

  • The report analyzes innovation in GBM, in the context of the overall pipeline and current market landscape. In addition, it analyzes the deals landscape surrounding first-in-class products, and pinpoints opportunities for in-licensing.
  • A brief introduction to GBM, including symptoms, pathophysiology, and an overview of pharmacotherapy and treatment algorithms.
  • The changing molecular target landscape between the market and the pipeline, including particular focal points of innovation in the pipeline.
  • A comprehensive review of the pipeline for first-in-class therapies, analyzed on the basis of stage of development, molecule type and molecular target.
  • Identification and assessment of first-in-class molecular targets, with a particular focus on early-stage programs for which clinical utility has yet to be evaluated, as well as literature reviews on novel molecular targets.
  • Assessment of the licensing and co-development deal landscape for GBM therapies.
Reasons to buy
  • Understand the current clinical and commercial landscape, including a comprehensive study of disease pathogenesis, diagnosis, prognosis and the available treatment options.
  • Visualize the composition of the GBM market in terms of dominant molecule types and targets, highlighting what the current unmet needs are and how they can be addressed. This knowledge allows a competitive understanding of the gaps in the current market.
  • Analyze the GBM pipeline, stratified by stage of development, molecule type and molecular target.
  • Assess the therapeutic potential of first-in-class targets. Using a proprietary matrix, first-in-class products have been assessed and ranked according to clinical potential. Promising early-stage targets have been further reviewed in greater detail.
  • Identify commercial opportunities in the GBM deals landscape by analyzing trends in licensing and co-development deals.


Glioblastoma market set for strong innovation, with 120 first-in-class programs, says GBI Research

Glioblastoma, a particularly aggressive form of brain tumor, has a promising treatment pipeline, with 120 first-in-class programs acting on 86 first-in-class molecular targets, according to business intelligence provider GBI Research.

The company’s latest report states that the glioblastoma therapy pipeline, which includes 512 products active across all stages of development, is moderately sized but highly innovative. Potential factors driving the pipeline include vast unmet need, a growing patient pool if disease prognosis can be improved, a lack of approved options in the market landscape, and a recently improved understanding of the disease pathophysiology, facilitating the development of novel compounds.

Adam Bradbury, MSc, Associate Analyst for GBI Research, explains: “Pipeline innovation has far-reaching strategic implications for all market participants as, despite the high attrition rate in glioblastoma, it is highly likely that many of the numerous first-in-class products, a number of which are supported by promising preclinical data, will reach the market over the coming decade, potentially transforming the clinical and commercial landscape.”

For players in the market, the case for investment in innovative products has not weakened as a result of the challenging commercial environments in developed markets and, increasingly, developed nations. On the contrary, despite higher stakes and greater risks, the return on investment for innovative products reaching the market remains attractive and could increase in significance in years to come.

Bradbury continues: “There are many signaling pathways and cellular processes in glioblastoma that remain untargeted by the limited number of associated marketed products. While growth factor signaling, such as by vascular endothelial growth factor, is inhibited in current glioblastoma treatments, evidence is mounting for the importance of other parallel mechanisms, such as cancer stem cell growth, and extracellular matrix remodeling.”

In terms of the glioblastoma deals landscape, activity is moderate and the mean value for co-development deals is below the industry average at $196.2 million, although the mean value of licensing deals is above the industry average at $168 million, according to GBI Research.

Bradbury concludes: “One of the most lucrative recent deals, a licensing agreement between AstraZenenca and Targacept, involved a current first-in-class product, and was valued at $1.2 billion. Despite the risk that can be associated with first-in-class products, they have still been shown to be a highly desirable investment option.”

  • Comments provided by Adam Bradbury, MSc, Associate Analyst for GBI Research.
  • Information based on GBI Research’s report:
Frontier Pharma: Glioblastoma Multiforme - Cancer Immunotherapies Dominate First-in-Class Product Innovation
  • 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 Low Prevalence but a High Unmet Need and a Limited Number of Marketed Options
2.2 Moderately Sized but Innovative Pipeline
2.3 Moderately Active Deals Landscape, Mostly Involving Currently Established Targets
3 The Case for Innovation in the Glioblastoma Multiforme Market
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 Changes in Clinical and Commercial Environment to be More Favorable to Products Targeting Niche Patient Populations and Indications
3.5 Sustained Innovation
3.6 Report Guidance
4 Clinical and Commercial Landscape
4.1 Disease Overview
4.2 Epidemiology
4.3 Disease Symptoms
4.4 Etiology
4.4.1 Age, Rae and Gender
4.4.2 Genetic Factors
4.4.3 Environmental Factors
4.5 Pathophysiology
4.5.1 Tumor Initiation and Aberrant Cell Proliferation and Survival
4.5.2 Tumor Metabolic Shift
4.5.3 Tumor Progression, Micro-environment Alteration and Angiogenesis
4.5.4 Cancer Stem Cells
4.6 Diagnosis
4.7 GBM Prognosis and Prognostic Factors
4.8 Classification
4.8.1 Loss of Heterozygosity of Chromosome 10
4.8.2 Epidermal Growth Factor Receptor Amplification
4.8.3 Phosphatase and Tensin Homolog Mutation
4.8.4 Loss of p53 Function
4.8.5 p16INK4a Alteration and Loss of Normal Retinoblastoma 1 Function
4.8.6 Isocitrate Dehydrogenase 1 or Isocitrate Dehydrogenase 2 Mutation
4.9 Glioblastoma Multiforme Treatment
4.10 Surgery and Radiation Therapy
4.11 Overview of Marketed Products for Glioblastoma Multiforme
4.11.1 Chemotherapy
4.11.2 Targeted Therapies
4.12 Treatment Algorithm
4.13 Current Unmet Need in the GBM Market
5 Assessment of Pipeline Product Innovation
5.1 Glioblastoma Pipeline by Phase, Molecule Type and Molecular Target
5.2 First-in-Class Pipeline Programs
6 Signaling Network, Disease Causation and Innovation Alignment
6.1 The Complexity of Signaling Networks in Oncology
6.2 Signaling Pathways, Disease-Causing Mutations 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 AKT 1 and 2
7.2 Pipeline Programs Targeting CDK1/2
7.3 Pipeline Programs Targeting PIK3CB and PIK3CG
7.4 Pipeline Programs which Target NTRK1
7.5 Pipeline Programs Targeting XIAP
7.6 Pipeline Programs Targeting Myc
7.7 Pipeline Programs Targeting PRKCA
7.8 Pipeline Programs which Target CASP8 and CASP9
7.9 Pipeline Programs which Target NR4A1
7.10 Pipeline Programs Targeting HIF-1α
7.11 Conclusion
8 Deals and Strategic Consolidations
8.1 Industry-Wide First-in-Class Deals
8.2 Glioblastoma Multiforme Deals Landscape
8.3 Licensing Deals
8.3.1 Molecule Type
8.3.2 Molecular Target
8.4 Co-development Deals
8.4.1 Molecule Type
8.4.2 Molecular Target
8.5 List of Pipeline Products without Prior Licensing or Co-Development Deal Involvement
9 Appendix
9.1 Abbreviations
9.2 References
9.3 Research Methodology
9.4 Secondary Research
9.4.1 Market Analysis
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: Symptoms of Glioblastoma, 2016
Table 2: Glioblastoma Multiforme, Histopathological and Genetic Subtypes
Table 3: Glioblastoma Multiforme, Key Features and Pipeline Activity of AKT1 and 2, 2016
Table 4: Glioblastoma Multiforme, Key Features and Pipeline Activity of CDK1/2, 2016
Table 5: Glioblastoma Multiforme, Key Features and Pipeline Activity of PIK3CB and PIK3CG, 2016
Table 6: Glioblastoma Multiforme, Key Features and Pipeline Activity of NTRK1, 2016
Table 7: Glioblastoma Multiforme, Key Features and Pipeline Activity of XIAP, 2016
Table 8: Glioblastoma Multiforme, Key Features and Pipeline Activity of Myc, 2016
Table 9: Glioblastoma Multiforme, Key Features and Pipeline Activity of PRKCA, 2016
Table 10: Glioblastoma Multiforme, Key Features and Pipeline Activity of CASP8 and 9, 2016
Table 11: Glioblastoma Multiforme, Key Features and Pipeline Activity of NR4A1, 2016
Table 12: Glioblastoma Multiforme, Key Features and Pipeline Activity of HIF-1α, 2016
Table 13: Abbreviations
1.2 List of Figures
Figure 1: Pharmaceutical Market, Global, Innovation Trends in Product Approvals, 1987-2012
Figure 2: Pharmaceutical Market, Global, Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013
Figure 3: Glioblastoma Multiforme, Genetic Pathways to Primary and Secondary Glioblastoma Multiforme and the Incidence of Mutations
Figure 4: Glioblastoma Multiforme, Marketed Product Overview, 2016
Figure 5: Glioblastoma Multiforme, Treatment Algorithm Overview
Figure 6: Glioblastoma Multiforme, Developmental Pipeline Overview, 2016
Figure 7: Glioblastoma Multiforme, Developmental Pipeline by Molecular Target, 2016
Figure 8: Glioblastoma Multiforme, Molecular Target Category Comparison, Pipeline and Marketed Products, 2016
Figure 9: Glioblastoma Multiforme, Molecular Target Category Comparison, Pipeline First-in-Class and Established Molecular Targets
Figure 10: Glioblastoma Multiforme, First-in-Class Products in the GBM Pipeline Part 1, 2016
Figure 11: Glioblastoma Multiforme, First-in-Class Products in the GBM Pipeline Part 2, 2016
Figure 12: Glioblastoma Multiforme, First-in-Class Molecular Target Analysis Matrix, 2016
Figure 13: Glioblastoma Multiforme, Pipeline Programs Targeting AKT1 and 2, 2016
Figure 14: Glioblastoma Multiforme, CDK1/2 Targeting Products, 2016
Figure 15: Glioblastoma Multiforme, PIK3CB/PIK3CG Targeting Products, 2016
Figure 16: Glioblastoma Multiforme, NTRK1 Targeting Products, 2016
Figure 17: Glioblastoma Multiforme, XIAP Targeting Products, 2016
Figure 18: Glioblastoma Multiforme, Myc Targeting Products, 2016
Figure 19: Glioblastoma Multiforme, PRKCA Targeting Products, 2016
Figure 20: Glioblastoma Multiforme, CASP8 and 9 Targeting Products, 2016
Figure 21: Glioblastoma Multiforme, NR4A1 Targeting Products, 2016
Figure 22: Glioblastoma Multiforme, HIF-1α Targeting Products, 2016
Figure 23: Pharmaceutical Market, Global, Industry-Wide Deals by Stage of Development, 2006-2014
Figure 24: Pharmaceutical Market, Global, Industry Licensing Deal Values by Stage of Development, 2006-2014
Figure 25: Glioblastoma Multiforme, Global, Licensing Deals by Region and Value, 2006-2015
Figure 26: Glioblastoma Multiforme, Global, Licensing Deals by Stage and Value, 2006-2016
Figure 27: Glioblastoma Multiforme, Licensing Deals by Molecule Type, 2006-2016
Figure 28: Glioblastoma Multiforme, Licensing Deals by Molecular Target, 2006-2016
Figure 29: Glioblastoma Multiforme, Licensing Deals with Disclosed Values, 2006-2016
Figure 30: Glioblastoma Multiforme, Co-development Deals by Region and Value, 2006-2016
Figure 31: Glioblastoma Multiforme, Co-development Deals by Stage and Value, 2006-2016
Figure 32: Glioblastoma Multiforme, Co-development Deals by Molecule Type, 2006-2016
Figure 33: Glioblastoma Multiforme, Co-Development Deals by Molecular Target, 2006-2016
Figure 34: Glioblastoma Multiforme, Co-Development Deals with Disclosed Values, 2006-2016
Figure 35: Glioblastoma Multiforme, List of Pipeline Products With Prior Licensing or Co-Development Deal Involvement, 2016
Figure 36: Glioblastoma Multiforme, List of Pipeline Products Without Prior Licensing or Co-Development Deal Involvement, 2016

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