Frontier Pharma: Hematological Disorders - Iron Regulators and Immune Response Targeted Programs Within Anemias and other Red Blood Cell Disorders Hold Potential to Transform Therapy Area with Significant Unmet Need

  • ID: 4390041
  • Report
  • Region: Global
  • 79 Pages
  • GBI Research
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Summary

Hematological disorders market is small and represents a significant investment opportunity. Due to few treatment options, many patients have a poor quality of life and poor prognosis, especially those with more severe disease states. Severe disease phenotype in cases of genetic disease correlates with a homozygous phenotype, as opposed to a heterozygous phenotype. For example, sickle cell anemia is a homozygous disorder, and sickle cell disease is a heterozygous disorder. The lack of pharmacotherapy treatment options means that alternative treatment approaches such as chronic blood transfusions and bone marrow transplants are used.

Although blood transfusions can successfully manage certain red blood cell disorders, this approach itself can lead to iron overload (hemochromatosis), another hematological disorder that itself affects patients’ quality of life and prognosis due to an increase in the risk of organ failure. Similarly, bone marrow transplant, which can treat a range of hematological disorders, carries many risks, ranging from anemia (another hematological disorder) to some more severe and potentially fatal disorders.

Globally prevalent disorders such as anemias, of which the most common form - iron deficiency anemia - is estimated to affect over 13% of the world’s population, represent significant investment opportunities due to the number of patients eligible for treatment. Although some forms of iron deficiency anemia are treatable with iron supplementation, other forms are hereditary in their etiology, such as iron malabsorption, and therefore require an alternative treatment approach to improve patient care. The therapy area also contains rare disorders of much lower prevalence, such as paroxysmal nocturnal hemoglobinuria, which is classified as an orphan disease and has no currently marketed treatment options.

Historically the hematological disorders therapy area has suffered from a lack of funding for research, leading to a low number of treatment options. However, first-in-class pipeline innovation is promising and 92 of 327 pipeline programs (28%) with a disclosed molecular target are first-in-class. Investment in first-in-class development is particularly important within hematological disorders as it holds the potential of achieving clinical breakthroughs through radical pharmaceutical innovation. A successful marketed product has the potential to gain a large market share due to the significant level of unmet need with regard to effective marketed treatment options.

The report has been designed to identify innovative pipeline programs across a therapy area, with a focus on indications with high R&D activity, strong innovation, a competitive commercial landscape and high levels of deal activity. Monitoring innovative new product developments is becoming increasingly important, not just for companies looking to acquire technologies, products or smaller companies that are seeking strategic partnerships, but also as a vital element of competitive intelligence for all market participants.

Scope

  • With 488 products in active development, the pipeline for hematological disorders is modestly sized. Does current pipeline innovation hold the potential to affect the future hematological disorders market?
  • There are 92 first-in-class products in the hematological disorders pipeline, which act on a novel molecular target which is not present in an approved product across any indication the pharmaceutical industry. Which of these hold the greatest potential to improve future disease treatment with regard to their molecular target?
  • Analysis of the history of strategic consolidations revealed a modest level of deal activity in recent years and a large number of first-in-class products not yet involved in any deals. How do deal frequency and value compare between target families and molecule types, and which first-in-class programs which have not yet been involved in a licensing or co-development deal appear to be particularly promising?

Reasons to buy

  • Understand the current clinical and commercial landscape. This includes a comprehensive study of disease pathogenesis, diagnosis and prognosis, and the treatment options available.
  • Visualize the composition of the hematological disorders 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 gaps in the current market.
  • Analyze the hematological disorders pipeline and stratify by stage of development, molecule type and molecular target. There are strong signs in the pipeline that the industry is seeking novel approaches to treating hematological disorders to overcome the overwhelming level of unmet need.
  • 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 reviewed in greater detail.
  • Identify commercial opportunities in the hematological disorders deals landscape by analyzing trends in licensing and co-development deals and analyzing a curated list of hematological disorder therapies that have not yet been involved in deals, and may offer potential investment opportunities.
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1 Table of Contents

2 Executive Summary
2.1 Small Market with Limited Treatment Options
2.2 Multiple Reasons for Investment
2.3 Significant Level of First-in-Class Innovation
2.4 Deal Activity Represents Investment Opportunity

3 The Case for Innovation in the Hematological Disorders Market
3.1 Growing Number of 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 Research Report Guidance

4 Clinical and Commercial Landscape
4.1 Disease Overview
4.2 Signs and Symptoms
4.2.1 Sickle Cell Disease
4.2.2 Anemia
4.2.3 Paroxysmal Nocturnal Hemoglobinuria
4.2.4 Other Disorders
4.3 Diagnosis
4.3.1 Sickle Cell Disease
4.3.2 Anemia
4.3.3 Paroxysmal Nocturnal Hemoglobinuria
4.3.4 Other Disorders
4.4 Etiology and Pathophysiology
4.4.1 Overview of Normal Iron Circulation
4.4.2 Disease State
4.5 Epidemiology
4.5.1 Sickle Cell Disease
4.5.2 Anemia
4.5.3 Paroxysmal Nocturnal Hemoglobinuria
4.5.4 Other Disorders
4.6 Treatment
4.6.1 Sickle Cell Disease
4.6.2 Anemia
4.6.3 Paroxysmal Nocturnal Hemoglobinuria
4.6.4 Other Disorders
4.7 Overview of Marketed Products
4.8 Current Unmet Need in the Hematological disorders Market

5 Pipeline Landscape Assessment
5.1 Hematological Disorders Pipeline Overview
5.2 Pipeline Development Landscape
5.2.1 Pipeline by Indication
5.2.2 Pipeline by Stage of Development and Molecule Type
5.2.3 Molecular Targets in the Pipeline
5.3 Comparative Distribution of Programs between the Hematological Disorders Pipeline and Market by Molecular Target
5.4 First-in-Class Programs Acting Upon Novel Molecular Targets
5.5 Ratio of First-in-Class Programs to First-in-Class Targets
5.6 List of all First-in-Class Pipeline Programs

6 Hematological Disorders Signaling Network and Innovation Alignment
6.1 Complexity of Signaling Networks in Hematological Disorders
6.2 Signaling Pathways and First-in-Class Molecular Target Integration
6.3 First-in-Class Matrix Assessment

7 First-in-Class Target Evaluation
7.1 Sickle Cell Disease
7.1.1 Pipeline Programs Targeting Hemopexin
7.1.2 Pipeline Programs Targeting RAC-Alpha Serine/threonine-Protein Kinase (AKT1)
7.1.3 Pipeline Programs Targeting P-Selectin
7.1.4 Pipeline Programs Targeting Ferroportin (Solute Carrier Family 40 Member 1)
7.2 Anemias
7.2.1 Pipeline Programs Targeting Transferrin Receptor Protein 1
7.2.2 Pipeline Programs Targeting Hepcidin
7.2.3 Pipeline Programs Targeting Natural Resistance Associated Macrophage Protein-2
7.3 Paroxysmal Nocturnal Hemoglobinuria
7.3.1 Pipeline Programs Targeting Complement factor-D (Cfd)
7.3.2 Pipeline Programs Targeting Mannan Binding Lectin Serine Protease-1 (Masp2)
7.3.3 Pipeline Programs Targeting Complement Component C6
7.4 Bleeding Disorders
7.5 Immune Cell Disorders
7.6 Conclusion

8 Deals and Strategic Consolidations
8.1 Industry-Wide First-in-Class Deals
8.2 Hematological Disorders Deals Landscape
8.3 Licensing Deals
8.3.1 Deals by Region, Year and Value
8.3.2 Deals by Stage of Development and Value
8.3.3 Deal Number and Value by Molecule Type and Molecular Target
8.4 Co-development Deals
8.4.1 Deals by Region, Year and Value
8.4.2 Deals by Stage of Development and Value
8.4.3 Molecule Type and Value
8.4.4 Molecular Target and Value
8.5 List of First-in-Class Pipeline Programs with and without Prior Deal Involvement

9 Appendix
9.1 Abbreviations
9.2 References
9.3 Research Methodology
9.3.1 Data integrity:
9.3.2 Innovative and meaningful analytical techniques and frameworks:
9.3.3 Evidence based analysis and insight:
9.4 Secondary Research
9.4.1 Market Analysis
9.4.2 Pipeline Analysis
9.4.3 Overall Pipeline
9.4.4 First-in-Class Analysis
9.4.5 First-in-Class Matrix Assessment
9.4.6 First-in-Class Target Profiles
9.4.7 Licensing and Co-development Deals

List of Tables
Table 1: Hematological Disorders Therapeutics Market, Global, Summary of Signs and Symptoms of Key Hematological Disorders, 2017
Table 2: Hematological Disorders Therapeutics Market, Global and US, Estimates of Prevalent Cases of Key Disorders, 2017
Table 3: Hematological Disorders Therapeutics Market, Global, Data for Hemopexin as a Molecular Target, 2017
Table 4: Hematological Disorders Therapeutics Market, Global, Data for RAC-Alpha Serine/threonine-Protein Kinase as a Molecular Target, 2017
Table 5: Hematological Disorders Therapeutics Market, Global, Data for P-Selectin as a Molecular Target, 2017
Table 6: Hematological Disorders Therapeutics Market, Global, Data for Ferroportin (Solute Carrier Family 40 Member 1) as a Molecular Target, 2017
Table 7: Hematological Disorders Therapeutics Market, Global, Data for Transferrin Receptor Protein 1 as a Molecular Target, 2017
Table 8: Hematological Disorders Therapeutics Market, Global, Data for Hepcidin as a Molecular Target, 2017
Table 9: Hematological Disorders Therapeutics Market, Global, Data for Natural Resistance Associated Macrophage Protein-2 as a Molecular Target, 2017
Table 10: Hematological Disorders Therapeutics Market, Global, Data for Complement factor-D as a Molecular Target, 2017
Table 11: Hematological Disorders Therapeutics Market, Global, Data for Mannan Binding Lectin Serine Protease-2 as a Molecular Target, 2017
Table 12: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Complement Component C6, 2017

List of Figures
Figure 1: Overall Pharmaceutical Industry, Innovation Trends in Product Approvals, Number of Product Approvals by FDA and Five-Year Moving Average of Products Approvals (%), 1987-2016
Figure 2: Hematological Disorders Therapeutics Market, First-in-Class and Non-First-in-Class Products, Sales Performance After Marketing Approval, 2006-2013 ($m)
Figure 3: Hematological Disorders Therapeutics Market, Global, Complement Pathway Overview, 2017
Figure 4: Hematological Disorders Therapeutics Market, Global, Marketed Product Overview, 2017
Figure 5: Overall Pharmaceutical Market, Global, Developmental Pipeline Overview, 2017
Figure 6: Hematological Disorders Therapeutics Market, Global, Pipeline by Indication, 2017
Figure 7: Hematological Disorders Therapeutics Market, Global, Pipeline by Stage of Development and Molecule Type, 2017
Figure 8: Hematological Disorders Therapeutics Market, Global, Pipeline by Molecular Target, 2017
Figure 9: Hematological Disorders Therapeutics Market, Global, Overall Pharmaceutical Industry First-in-Class Pipeline by Therapy Area, 2017
Figure 10: Hematological Disorders Therapeutics Market, Global, Overall Pharmaceutical Industry First-in-Class Pipeline by Therapy Area, 2017
Figure 11: Hematological Disorders Therapeutics Market, Global, First-in-Class Pipeline for Hematological disorders by Stage of Development and Molecule Type, 2017
Figure 12: Hematological Disorders Therapeutics Market, Global, First-in-Class Pipeline for Hematological disorders by Stage of Development and Molecule Type, 2017
Figure 13: Hematological Disorders Therapeutics Market, Global, Molecular Target Family Comparison, Pipeline First-in-Class and Established Molecular Targets, 2017
Figure 14: Hematological Disorders Therapeutics Market, Global, Molecular Target Family Comparison, Pipeline First-in-Class and Established Molecular Targets, 2017
Figure 15: Hematological Disorders Therapeutics Market, Global, Molecular Target Family Comparison, Pipeline First-in-Class and Established Molecular Targets, 2017
Figure 16: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Sickle Cell Disease Pipeline, 2017
Figure 17: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Anemias Pipeline, 2017
Figure 18: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Paroxysmal Nocturnal Hemoglobinuria Pipeline, 2017
Figure 19: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Bleeding Disorders Pipeline, 2017
Figure 20: Hematological Disorders Therapeutics Market, Global, First-in-Class Developmental Programs in Immune Cell Disorders Pipeline, 2017
Figure 21: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Sickle Cell Disease, 2017
Figure 22: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Anemias, 2017
Figure 23: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Paroxysmal Nocturnal Hemoglobinuria, 2017
Figure 24: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Bleeding Disorders, 2017
Figure 25: Hematological Disorders Therapeutics Market, Global, First-in-Class Molecular Target Analysis Matrix for Immune Cell Disorders, 2017
Figure 26: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Hemopexin, 2017
Figure 27: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting RAC Alpha Serine/Threonine Protein Kinase, 2017
Figure 28: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting P-Selectin, 2017
Figure 29: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Ferroportin (Solute Carrier Family 40 Member 1), 2017
Figure 30: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Transferrin Receptor Protein 1, 2017
Figure 31: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Hepcidin, 2017
Figure 32: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Natural Resistance Associated Macrophage Protein-2, 2017
Figure 33: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Complement factor-D, 2017
Figure 34: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Mannan Binding Lectin Serine Protease-2, 2017
Figure 35: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Complement Component C6, 2017
Figure 36: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting Tissue Factor, 2017
Figure 37: Hematological Disorders Therapeutics Market, Global, Pipeline Programs Targeting IL5RA, 2017
Figure 38: Pharmaceutical Market, Global, Industry-Wide Deals by Stage of Development, 2006-2014
Figure 39: Pharmaceutical Market, Global, Industry Licensing Deal Values by Stage of Development, 2006-2014
Figure 40: Hematological Disorders Therapeutics Market, Global, Licensing Deals by Region and Value, 2006-2017
Figure 41: Hematological Disorders Therapeutics Market, Global, Licensing Deals by Stage of Development, 2006-2017
Figure 42: Hematological Disorders Therapeutics Market, Global, Licensing Deals Valued Above $25m, 2006-2017
Figure 43: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Region and Value, 2006-2017
Figure 44: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Stage of Development, 2006-2017
Figure 45: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Molecule Type, 2006-2017
Figure 46: Hematological Disorders Therapeutics Market, Global, Co-Development Deals by Molecular Target, 2006-2017
Figure 47: Hematological Disorders Therapeutics Market, Global, Co-Development Deals Valued Above $25m, 2006-2017
Figure 48: Hematological Disorders Therapeutics Market, Global, First-in-class Programs in Active Development Involved in Previous Deals, 2017
Figure 49: Hematological Disorders Therapeutics Market, Global, First-in-class Programs in Active Development Without Recorded Prior Deal Involvement, 2017

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