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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

  • August 2017
  • 79 pages
  • GBI Research
Report ID: 5089261

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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

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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