"The
Report Frontier Pharma: Duchenne Muscular Dystrophy and Becker
Muscular Dystrophy - Identifying and Commercializing First-in-Class
Innovation provides information on pricing, market analysis, shares,
forecast, and company profiles for key industry participants. -
MarketResearchReports.biz"
Highly Innovative and
Diverse Pipeline
The Duchenne
Muscular Dystrophy (DMD)
and Becker Muscular Dystrophy (BMD) pipeline consists of 84 molecules
across all stages of development. GBI Research’s analysis revealed
a high degree of innovation and diversity in this indication, with
70% of the pipeline being first-in-class products, acting on 13
first-in-class targets. This exceptional first-in-class innovation is
largely due to the high number of first-in-class products solely
targeting the dystrophin gene, which is the primary genetic cause of
DMD and BMD. The strong presence of first-in-class products in the
pipeline therefore creates a distinctly different landscape to the
market landscape, which relies on symptomatic treatment
glucocorticoids. Although Translarna (ataluren) is developed to
correct the genetic defects, significant unmet needs remain in the
market, as the treatment is applicable to only 10–15% of all DMD
cases caused by nonsense mutations.
Despite a strong focus on
personalized treatments that treat the genetic cause of the disease
in the DMD/BMD pipeline, innovation is also concentrated on novel
molecular targets that alleviate the dystrophic pathology regardless
of gene mutations, thereby allowing widespread use in contrast to the
mutation-specific treatments. These therapies are expected to be used
alongside primary treatment to repair the mutated gene, halt muscle
degeneration, and improve life expectancy of patients in the future
market.
Strong Alignment of Innovation
to Genetics and Disease Processes in Early Pipeline
DMD, and BMD, which is the less
severe form, are neuromuscular diseases caused by heritable mutations
in the single dystrophin gene, which ultimately lead to progressive
muscle weakness and degeneration due to destabilization of the
sarcolemma (muscle cell membrane) and the resultant loss of muscle
integrity. However, increasing evidence suggests that multiple
secondary pathological mechanisms, rather than dystrophin deficiency
alone, cause or contribute to the pathological features of DMD/BMD
and drive disease progression. This further substantiates the need
for better understanding of the downstream events of dystrophin
deficiency to enable the identification of more potential molecular
targets that in turn could be translated into disease-modifying
treatments.
Our proprietary analyses show
that the 13 first-in-class targets differ substantially in terms of
clinical and commercial potential based on how well their functional
roles align to the disease pathophysiology and the strength of
evidence in Preclinical studies. Some molecular targets are therefore
considered more promising than others due to a stronger potential to
be translated into novel treatments. The most promising targets
provide a strong scientific rationale to support their therapeutic
development, as indicated by substantial improvement in both muscle
histopathology and function in vivo across different animal model
systems.
Analysis also indicates
opportunities for some of the first-in-class DMD/BMD targets to be
repositioned to other MDs, although this is expected to be
challenging given the currently limited understanding of the common
molecular processes defected across multiple types of MD.
Numerous Investment
Opportunities in Deals Landscape
Strategic consolidation is
relatively uncommon in the DMD/BMD market, with 15 licensing
agreements and 18 co-development deals between 2006 and April 2015.
Supported by findings from the industry-wide analysis, there is a
tendency for first-in-class DMD programs to attract higher deal
values than non-first-in-class programs, thus highlighting their
commercial attractiveness. Despite the high-risk profile of
first-in-class products, they have greater potential to revolutionize
or improve therapeutic options, meaning that identifying promising
first-in-class compounds early in development offers the greatest
potential commercial benefit to pharmaceutical companies.
With 36 first-in-class products
that are currently in development having not yet been involved in a
licensing or co-development deal, there are numerous opportunities
for in-licensing or co-development in this indication
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Scope
The
report analyzes innovation in DMD/BMD in the context of the overall
pipeline and current market landscape. In addition, it analyzes the
deals landscape surrounding first-in-class products in DMD/BMD and
pinpoints opportunities for in-licensing.
The report covers and
includes
- A brief introduction to DMD/BMD, including symptoms, pathophysiology, and an overview of pharmacotherapy and treatment algorithms
- The changing molecular target landscape between market and pipeline and particular focal points of innovation in the pipeline
- A comprehensive review of the pipeline for first-in-class therapies, analyzed on the basis of stage of development, molecule type, and molecular target
- Identification 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 of novel molecular targets
- Assessment of the licensing and co-development deal landscape for DMD/BMD therapies and benchmarking of deals involving first-in-class versus non-first-in-class-products
Reasons to buy
The report will assist
business development and enable marketing executives to strategize
their product launches, by allowing them to
- Understand the focal shifts in molecular targets in the DMD/BMD pipeline
- Understand the distribution of pipeline programs by phase of development, molecule type, and molecular target
- Access scientific and clinical analysis of first-in-class developmental programs for DMD/BMD, benchmarked against non-first-in-class targets
- Access a list of the first-in-class therapies potentially open to deal-making opportunities
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Table of content
1 Table of Contents
1 Table of Contents 2
1.1 List of Tables 3
1.2 List of Figures 3
2 Executive Summary 4
2.1 Highly Innovative and
Diverse Pipeline 4
2.2 Alignment of Innovation to
Genetics and Disease Processes 4
2.3 Deals Landscape Present
Substantial Investment Opportunities 4
3 The Case for Innovation 5
3.1 Growing Opportunities for
Biologic Products 6
3.2 Diversification of
Molecular Targets 6
3.3 Innovative First-in-Class
Product Developments Remain Attractive 6
3.4 Regulatory and
Reimbursement Policy Shifts Favor First-in-Class Product Innovation 7
3.5 Sustained Innovation 7
3.6 GBI Research Report
Guidance 8
4 Clinical and Commercial
Landscape 9
4.1 Disease Overview 9
4.2 Disease Epidemiology and
Etiology 9
4.2.1 Disease Inheritance 10
4.3 Disease Pathophysiology 10
4.3.1 Hypothesized
Pathophysiological Mechanisms of Duchenne Muscular Dystrophy/ Becker
Muscular Dystrophy 11
4.3.2 Dysfunction in
Regeneration and Development of Fibrosis 13
4.4 Disease Symptoms 13
4.5 Complications and
Co-morbidities 14
4.5.1 Respiratory Complications
14
4.5.2 Cardiac Complications 14
4.5.3 Scoliosis 14
4.6 Diagnosis 14
4.7 Disease Staging and
Prognosis 15
4.8 Treatment Options 16
4.8.1 Pharmacological
Treatments 16
4.8.2 Non-pharmacological
Treatments 18
4.8.3 Treatment Algorithm 19
4.9 Overview of Marketed
Products 21
4.9.1 Glucocorticoids 21
4.9.2 Translarna (ataluren) 23
4.9.3 Molecule Type and Target
Analysis 23
4.10 Current Unmet Needs 24
5 Assessment of Pipeline
Product Innovation 25
5.1 Duchenne Muscular Dystrophy
and Becker Muscular Dystrophy Pipeline by Molecule Type, Phase and
Therapeutic Target 25
5.2 Comparative Distribution of
Programs between Duchenne Muscular Dystrophy/Becker Muscular
Dystrophy Market, and Pipeline by Therapeutic Target Family 29
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