DUBLIN, October 18, 2017 /PRNewswire/ --

The "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" drug pipelines has been added to Research and Markets' offering.

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.

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.

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?

Key Topics Covered:

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

For more information about this drug pipelines report visit https://www.researchandmarkets.com/research/x4ls2s/frontier_pharma

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