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

“The Report 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 provides information on pricing, market analysis, shares, forecast, and company profiles for key industry participants. –”

About Hematological Disorders Market

The hematological disorders therapy area encompasses non-malignant disorders of the blood such as anemia, sickle cell disease, neutropenia and hemophilia. These diseases can be grouped into the three categories- red blood cell disorders, bleeding disorders and immune cell disorders – of which red blood cell disorders are characterized by significant unmet need related to few effective treatment options and poor prognosis. As such, they are the primary focus of the report.

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Historically, the hematological disorders therapy area has suffered from a lack of funding for research, which has contributed to the level of unmet need, although there have been instances in which funding for an indication has resulted in a strong return on investment. A notable example is the approval of Soliris in 2007, which is used to treat the orphan disease paroxysmal nocturnal hemoglobinuria. Soliris is one of only two marketed treatment options for PNH and is forecast to generate annual revenue of over $3 billion by the end of 2017, rising to $5.5 billion in 2023.

The report assesses first-in-class innovation in the hematological disorders pipeline, highlighting key trends in the pipeline, and emerging treatment classes. There are 92 first-in-class products in development in this therapy area; which account for 28% of products in the overall pipeline for which there is a disclosed molecular target, and act upon 71 unique individual molecular targets.

These first-in-class targets in the pipeline are numerous and varied, with many of them having been shown to be promising in early stage studies. Immune components are the most common, accounting for a total of 21 first-in-class developmental programs, closely followed by DNA regulators. These target categories combined account for the majority of red blood cell disorder pipeline therapies, although iron regulator programs alone also appear frequently.

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– 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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Table of Contents

1 Table of Contents 2

1.1 List of Tables 4

1.2 List of Figures 4

2 Executive Summary 6

2.1 Small Market with Limited Treatment Options 6

2.2 Multiple Reasons for Investment 6

2.3 Significant Level of First-in-Class Innovation 6

2.4 Deal Activity Represents Investment Opportunity 6

3 The Case for Innovation in the Hematological Disorders Market 7

3.1 Growing Number of Opportunities for Biologic Products 8

3.2 Diversification of Molecular Targets 8

3.3 Innovative First-in-Class Product Developments Remain Attractive 8

3.4 Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation 9

3.5 Sustained Innovation 9

3.6 GBI Research Report Guidance 9

4 Clinical and Commercial Landscape 11

4.1 Disease Overview 11

4.2 Signs and Symptoms 12

4.2.1 Sickle Cell Disease 12

4.2.2 Anemia 13

4.2.3 Paroxysmal Nocturnal Hemoglobinuria 13

4.2.4 Other Disorders 13

4.3 Diagnosis 14

4.3.1 Sickle Cell Disease 14

4.3.2 Anemia 15

4.3.3 Paroxysmal Nocturnal Hemoglobinuria 15

4.3.4 Other Disorders 15

4.4 Etiology and Pathophysiology 16

4.4.1 Overview of Normal Iron Circulation 16

4.4.2 Disease State 17

4.5 Epidemiology 21

4.5.1 Sickle Cell Disease 21

4.5.2 Anemia 21

4.5.3 Paroxysmal Nocturnal Hemoglobinuria 21

4.5.4 Other Disorders 22

4.6 Treatment 23

4.6.1 Sickle Cell Disease 23

4.6.2 Anemia 23

4.6.3 Paroxysmal Nocturnal Hemoglobinuria 24

4.6.4 Other Disorders 24

4.7 Overview of Marketed Products 24

4.8 Current Unmet Need in the Hematological disorders Market 26

5 Pipeline Landscape Assessment 27

5.1 Hematological Disorders Pipeline Overview 27

5.2 Pipeline Development Landscape 27

5.2.1 Pipeline by Indication 27

5.2.2 Pipeline by Stage of Development and Molecule Type 28

5.2.3 Molecular Targets in the Pipeline 30

5.3 Comparative Distribution of Programs between the Hematological Disorders Pipeline and Market by Molecular Target 33

5.4 First-in-Class Programs Acting Upon Novel Molecular Targets 33

5.5 Ratio of First-in-Class Programs to First-in-Class Targets 37

5.6 List of all First-in-Class Pipeline Programs 39

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