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Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy-Identifying and Commercializing First-in-Class Innovation

Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy-Identifying and Commercializing First-in-Class Innovation

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Executive Summary

Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy-Identifying and Commercializing First-in-Class Innovation

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


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

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

6 Signaling Pathways, Genetics and Innovation Alignment 34

6.1 First-in-Class Target Matrix Assessment 34

6.2 Repositioning Potential among Muscular Dystrophies 36

7 First-in-Class Target Evaluation 38

7.1 Pipeline Programs Targeting Sarcospan 38

7.2 Pipeline Programs Targeting Utrophin 40

7.3 Pipeline Programs Targeting Biglycan 43

7.4 Pipeline Programs Targeting Laminin-111 45

7.5 Pipeline Programs Targeting Hematopoietic Prostaglandin D Synthase 48

7.6 Pipeline Programs which Target G Protein-Coupled Receptor Mas 50

7.7 Pipeline Programs Targeting Mothers against Decapentaplegic Homolog 3 52

7.8 Pipeline Programs which Target Myostatin 54

7.9 Pipeline Programs Targeting Dystrophin 56

7.10 Conclusion 59

8 Deals and Strategic Consolidations 60

8.1 Industry-Wide First-in-Class Deals 60

8.2 Licensing Deals 61

8.3 Co-development Deals 66

8.4 First-in-Class Programs Not Involved in Licensing or Co-Development Deals 68

9 Appendix 70

9.1 Abbreviations 70

9.2 References 70

9.3 Contact Us 77

9.4 Disclaimer 77

1.1 List of Tables

Table 1: Stages of Duchenne Muscular Dystrophy, 2015 15

1.2 List of Figures

Figure 1: Innovation Trends in Product Approvals, 1987-2013 5

Figure 2: Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013 7

Figure 3: Schema for Initiation of Glucocorticoid Treatment in Duchenne Muscular Dystrophy 20

Figure 4: Molecular Targets of Marketed Products, 2015 24

Figure 5: Developmental Pipeline Overview 26

Figure 6: Developmental Pipeline Overview 28

Figure 7: Molecular Target Category Comparison, Pipeline and Marketed Products 29

Figure 8: Molecular Target Category Comparison, Pipeline First-in-Class and Established Molecular Targets 31

Figure 9: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, Global, Pipeline, First-in-class Products in the Pipeline 32

Figure 10: First-in-Class Molecular Target Analysis Matrix 35

Figure 11: Data and Evidence for Sarcospan as a Therapeutic target 39

Figure 12: Pipeline Programs Targeting Sarcospan 39

Figure 13: Data and Evidence for Utrophin as a Therapeutic target 42

Figure 14: Pipeline Programs Targeting Utrophin 43

Figure 15: Data and Evidence for Biglycan as a Therapeutic target 44

Figure 16: Pipeline Programs Targeting Biglycan 44

Figure 17: Data and Evidence for Laminin-111 as a Therapeutic Target 47

Figure 18: Pipeline Programs Targeting Laminin-111 48

Figure 19: Data and Evidence for Hematopoietic Prostaglandin D Synthase as a Therapeutic target 49

Figure 20: Pipeline Programs Targeting Hematopoietic Prostaglandin D Synthase 49

Figure 21: Data and Evidence for G Protein-Coupled Receptor Mas as a Therapeutic target 51

Figure 22: Pipeline Programs Targeting G Protein-Coupled Receptor Mas 51

Figure 23: Data and Evidence for Mothers against decapentaplegic homolog 3 as a Therapeutic target 53

Figure 24: Pipeline Programs Targeting Mothers against decapentaplegic homolog 3 53

Figure 25: Data and Evidence for Myostatin as a Therapeutic target 55

Figure 26: Pipeline Programs Targeting Myostatin 55

Figure 27: Data and Evidence for Dystrophin as a Therapeutic target 57

Figure 28: Pipeline Programs Targeting Dystrophin 58

Figure 29: Industry-Wide Deals by Stage of Development, 2006-2014 60

Figure 30: Industry Licensing Deal Values by Stage of Development, 2006-2014 61

Figure 31: Licensing Deals in Duchenne Muscular Dystrophy, 2006-2015 63

Figure 32: Licensing Deals Global Distribution, 2006-2015 64

Figure 33: Licensing Deals by Molecule Type, 2006-2015 64

Figure 34: Licensing Deals by Molecular Target, 2006-2015 65

Figure 35: Summary of Licensing Deals, 2006-2015 65

Figure 36: Co-development Deals by Year, 2006-2015 66

Figure 37: Co-development Deals Global Distribution, 2006-2015 66

Figure 38: Co-development Deals by Molecule Type, 2006-2015 67

Figure 39: Co-development Deals by Molecular Target, 2006-2015 67

Figure 40: Summary of Co-Development Deals, 2006-2015 68

Figure 41: First-in-Class Programs with no Recorded Prior Deal Involvement, 2006-2015 69

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Duchenne Muscular Dystrophy, Becker Muscular Dystrophy

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