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Frontier Pharma:裘馨氏肌肉萎縮症 (DMD) 、貝克氏肌肉萎縮症 (BMD) - First-in-Class的革新認識和商業化

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

出版商 GBI Research 商品編碼 331416
出版日期 內容資訊 英文 77 Pages
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Frontier Pharma:裘馨氏肌肉萎縮症 (DMD) 、貝克氏肌肉萎縮症 (BMD) - First-in-Class的革新認識和商業化 Frontier Pharma: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy - Identifying and Commercializing First-in-Class Innovation
出版日期: 2015年05月01日 內容資訊: 英文 77 Pages



裘馨氏肌肉萎縮症 (DMD) 和貝克氏肌肉萎縮症 (BMD) 治療藥的開發平台,在全階段中以84分子構成。

本報告提供裘馨氏肌肉萎縮症 (DMD) 、貝克氏肌肉萎縮症 (BMD) 治療藥市場相關調查分析、革新案例、臨床與商業情形、開發中產品的評估、計劃評估、最近的交易等相關的系統性資訊。

第1章 目錄

第2章 摘要整理

第3章 革新案例

  • 生技藥品的機會擴大
  • 分子標的多樣化
  • 創新的First-in-Class產品 (劃時代的醫藥品) 的開發現在也富有魅力
  • 法規、償付政策,對First-in-Class產品的革新有利
  • 持續性的革新
  • 報告指南

第4章 臨床、商業性情形

  • 疾病概要
  • 疾病的免疫學與病因
  • 疾病的病理生理學
  • 疾病的症狀
  • 併發症和共生病症
  • 診斷
  • 疾病的分期和預後
  • 治療的選項
  • 上市產品概要
  • 目前未滿足需求

第5章 開發中產品革新的評估

  • 裘馨氏肌肉萎縮症 (DMD) 、貝克氏肌肉萎縮症 (BMD) 治療藥開發平台:各分子類型、階段、治療標的
  • 裘馨氏肌肉萎縮症 (DMD) 、貝克氏肌肉萎縮症 (BMD) 治療藥市場上計劃比較分佈,開發平台:各分子標的系列

第6章 信號 (信號傳達) 網路、遺傳學、革新的調整

  • First-in-Class標的矩陣的評估
  • 肌肉萎縮症的潛在性重新配置

第7章 First-in-Class目標與開發平台計劃的評估

  • Sarcospan
  • Utrophin
  • bBiglycan
  • Laminin-111
  • Hematopoietic Prostaglandin D Synthase
  • G Protein-Coupled Receptor Mas、等
  • 結論

第8章 交易與策略性整合

  • 產業整體First-in-Class交易
  • 許可證交易
  • 共同開發交易
  • 不參與許可證/共同開發交易的First-in-Class計劃

第9章 附錄


Product Code: GBIHC361MR

Executive Summary

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

Table of Contents

1. Table of Contents

  • 1.1. List of Tables
  • 1.2. List of Figures

2. Executive Summary

  • 2.1. Highly Innovative and Diverse Pipeline
  • 2.2. Alignment of Innovation to Genetics and Disease Processes
  • 2.3. Deals Landscape Present Substantial Investment Opportunities

3. The Case for Innovation

  • 3.1. Growing 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. GBI Research Report Guidance

4. Clinical and Commercial Landscape

  • 4.1. Disease Overview
  • 4.2. Disease Epidemiology and Etiology
    • 4.2.1. Disease Inheritance
  • 4.3. Disease Pathophysiology
    • 4.3.1. Hypothesized Pathophysiological Mechanisms of Duchenne Muscular Dystrophy/ Becker Muscular Dystrophy
    • 4.3.2. Dysfunction in Regeneration and Development of Fibrosis
  • 4.4. Disease Symptoms
  • 4.5. Complications and Co-morbidities
    • 4.5.1. Respiratory Complications
    • 4.5.2. Cardiac Complications
    • 4.5.3. Scoliosis
  • 4.6. Diagnosis
  • 4.7. Disease Staging and Prognosis
  • 4.8. Treatment Options
    • 4.8.1. Pharmacological Treatments
    • 4.8.2. Non-pharmacological Treatments
    • 4.8.3. Treatment Algorithm
  • 4.9. Overview of Marketed Products
    • 4.9.1. Glucocorticoids
    • 4.9.2. Translarna (ataluren)
    • 4.9.3. Molecule Type and Target Analysis
  • 4.10. Current Unmet Needs

5. Assessment of Pipeline Product Innovation

  • 5.1. Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Pipeline by Molecule Type, Phase and Therapeutic Target
  • 5.2. Comparative Distribution of Programs between Duchenne Muscular Dystrophy/Becker Muscular Dystrophy Market, and Pipeline by Therapeutic Target Family

6. Signaling Pathways, Genetics and Innovation Alignment

  • 6.1. First-in-Class Target Matrix Assessment
  • 6.2. Repositioning Potential among Muscular Dystrophies

7. First-in-Class Target Evaluation

  • 7.1. Pipeline Programs Targeting Sarcospan
  • 7.2. Pipeline Programs Targeting Utrophin
  • 7.3. Pipeline Programs Targeting Biglycan
  • 7.4. Pipeline Programs Targeting Laminin-111
  • 7.5. Pipeline Programs Targeting Hematopoietic Prostaglandin D Synthase
  • 7.6. Pipeline Programs which Target G Protein-Coupled Receptor Mas
  • 7.7. Pipeline Programs Targeting Mothers against Decapentaplegic Homolog 3
  • 7.8. Pipeline Programs which Target Myostatin
  • 7.9. Pipeline Programs Targeting Dystrophin
  • 7.10. Conclusion

8. Deals and Strategic Consolidations

  • 8.1. Industry-Wide First-in-Class Deals
  • 8.2. Licensing Deals
  • 8.3. Co-development Deals
  • 8.4. First-in-Class Programs Not Involved in Licensing or Co-Development Deals

9. Appendix

  • 9.1. Abbreviations
  • 9.2. References
  • 9.3. Contact Us
  • 9.4. Disclaimer

List of Tables

  • Table 1: Stages of Duchenne Muscular Dystrophy, 2015

List of Figures

  • Figure 1: Innovation Trends in Product Approvals, 1987-2013
  • Figure 2: Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013
  • Figure 3: Schema for Initiation of Glucocorticoid Treatment in Duchenne Muscular Dystrophy
  • Figure 4: Molecular Targets of Marketed Products, 2015
  • Figure 5: Developmental Pipeline Overview
  • Figure 6: Developmental Pipeline Overview
  • Figure 7: Molecular Target Category Comparison, Pipeline and Marketed Products
  • Figure 8: Molecular Target Category Comparison, Pipeline First-in-Class and Established Molecular Targets
  • Figure 9: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, Global, Pipeline, First-in-class Products in the Pipeline
  • Figure 10: First-in-Class Molecular Target Analysis Matrix
  • Figure 11: Data and Evidence for Sarcospan as a Therapeutic target
  • Figure 12: Pipeline Programs Targeting Sarcospan
  • Figure 13: Data and Evidence for Utrophin as a Therapeutic target
  • Figure 14: Pipeline Programs Targeting Utrophin
  • Figure 15: Data and Evidence for Biglycan as a Therapeutic target
  • Figure 16: Pipeline Programs Targeting Biglycan
  • Figure 17: Data and Evidence for Laminin-111 as a Therapeutic Target
  • Figure 18: Pipeline Programs Targeting Laminin-111
  • Figure 19: Data and Evidence for Hematopoietic Prostaglandin D Synthase as a Therapeutic target
  • Figure 20: Pipeline Programs Targeting Hematopoietic Prostaglandin D Synthase
  • Figure 21: Data and Evidence for G Protein-Coupled Receptor Mas as a Therapeutic target
  • Figure 22: Pipeline Programs Targeting G Protein-Coupled Receptor Mas
  • Figure 23: Data and Evidence for Mothers against decapentaplegic homolog 3 as a Therapeutic target
  • Figure 24: Pipeline Programs Targeting Mothers against decapentaplegic homolog 3
  • Figure 25: Data and Evidence for Myostatin as a Therapeutic target
  • Figure 26: Pipeline Programs Targeting Myostatin
  • Figure 27: Data and Evidence for Dystrophin as a Therapeutic target
  • Figure 28: Pipeline Programs Targeting Dystrophin
  • Figure 29: Industry-Wide Deals by Stage of Development, 2006-2014
  • Figure 30: Industry Licensing Deal Values by Stage of Development, 2006-2014
  • Figure 31: Licensing Deals in Duchenne Muscular Dystrophy, 2006-2015
  • Figure 32: Licensing Deals Global Distribution, 2006-2015
  • Figure 33: Licensing Deals by Molecule Type, 2006-2015
  • Figure 34: Licensing Deals by Molecular Target, 2006-2015
  • Figure 35: Summary of Licensing Deals, 2006-2015
  • Figure 36: Co-development Deals by Year, 2006-2015
  • Figure 37: Co-development Deals Global Distribution, 2006-2015
  • Figure 38: Co-development Deals by Molecule Type, 2006-2015
  • Figure 39: Co-development Deals by Molecular Target, 2006-2015
  • Figure 40: Summary of Co-Development Deals, 2006-2015
  • Figure 41: First-in-Class Programs with no Recorded Prior Deal Involvement, 2006-2015
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