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

  • ID: 3892430
  • Report
  • Region: Global
  • 73 Pages
  • GBI Research
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Glioblastoma Market Set for Strong Innovation, With 120 First-In-Class Programs
Glioblastoma multiforme (GBM) is the most common and aggressive type of human brain tumor, accounting for 15.4% of all primary brain tumors. GBM constitutes 60-75% of all astrocytomas - a cancer of the brain originating in a particular type of glial cell in the cerebrum called astrocytes (NCI, 2013a; ABTA, 2016a). It is most common in the elderly, with a peak incidence rate between the ages of 55 and 84, and a median age of diagnosis of 64 (Nelson et al., 2012; Thakkar et al., 2014).

Glioblastoma multiforme (GBM) has a low incidence and prevalence population in the US and EU, with the annual incidence estimated to be three to four cases per 100,000 people. There is a poor prognosis associated with the current standard of care, which is surgical resection followed by radiation therapy and temozolomide chemotherapy, with an associated median survival duration of 12-14 months (Johnson and O’Neill, 2011). There is a limited number of approved drugs for GBM, and these are subject to regional variations. Gliadel and temozolomide are the most widely approved globally, and are cytotoxic chemotherapies that are associated with more toxicity than targeted treatments.

The GBM cancer pipeline is moderately sized, with 512 products active across all stages of development. The range of mechanisms of action employed by these compounds is also highly diverse, especially in comparison with the existing market landscape. More pertinently, there is a large proportion of breakthrough innovations in this pipeline.
It is identified that 120 first-in-class programs in the GBM pipeline, acting on 86 first-in-class molecular targets. This accounts for 31% of all products with a disclosed molecular target, and reflects the high degree of innovation in this indication.

The report “Frontier Pharma: Glioblastoma Multiforme - Cancer Immunotherapies Dominate First-in-Class Product Innovation” 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.

In particular, this report includes analysis of the following:
  • Provides a brief introduction to GBM, including symptoms, pathophysiology, and an overview of pharmacotherapy and treatment algorithms
  • Provides comprehensive analysis of the pipeline for first-in-class therapies, analyzed on the basis of stage of development, molecule type and molecular target.
  • This report help to Identify 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.
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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-1a
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-1a, 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-1a 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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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 the publisher.

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.

One of our analysts 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.

Our analyst 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 the publisher.

Our analyst 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.”
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