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

  • Executive Summary
    • Highly Innovative and Diverse Pipeline
    • Alignment of Innovation to Genetics and Disease Processes
    • Deals Landscape Present Substantial Investment Opportunities
  • The Case for Innovation
    • Table Figure 1: Innovation Trends in Product Approvals, 1987-2013
    • Growing Opportunities for Biologic Products
    • Diversification of Molecular Targets
    • Innovative First-in-Class Product Developments Remain Attractive
      • Table Figure 2: Sales Performance of First-in-Class and Non-First-in-Class Products Post Marketing Approval, 2006-2013
    • Regulatory and Reimbursement Policy Shifts Favor First-in-Class Product Innovation
    • Sustained Innovation
    • GBI Research Report Guidance
  • Clinical and Commercial Landscape
    • Disease Overview
    • Disease Epidemiology and Etiology
      • Disease Inheritance
    • Disease Pathophysiology
      • Hypothesized Pathophysiological Mechanisms of Duchenne Muscular Dystrophy/ Becker Muscular Dystrophy
      • Dysfunction in Regeneration and Development of Fibrosis
    • Disease Symptoms
    • Complications and Co-morbidities
      • Respiratory Complications
      • Cardiac Complications
      • Scoliosis
    • Diagnosis
    • Disease Staging and Prognosis
      • Table Stages of Duchenne Muscular Dystrophy, 2015
    • Treatment Options
      • Pharmacological Treatments
      • Non-pharmacological Treatments
      • Treatment Algorithm
        • Table Figure 3: Schema for Initiation of Glucocorticoid Treatment in Duchenne Muscular Dystrophy
    • Overview of Marketed Products
      • Glucocorticoids
      • Translarna (ataluren)
      • Molecule Type and Target Analysis
        • Table Figure 4: Molecular Targets of Marketed Products, 2015
    • Current Unmet Needs
  • Assessment of Pipeline Product Innovation
    • Duchenne Muscular Dystrophy and Becker Muscular Dystrophy Pipeline by Molecule Type, Phase and Therapeutic Target
      • Table Figure 5: Developmental Pipeline Overview
      • Table Figure 6: Developmental Pipeline Overview
    • Comparative Distribution of Programs between Duchenne Muscular Dystrophy/Becker Muscular Dystrophy Market, and Pipeline by Therapeutic Target Family
      • Table Figure 7: Molecular Target Category Comparison, Pipeline and Marketed Products
      • Table Figure 8: Molecular Target Category Comparison, Pipeline First-in-Class and Established Molecular Targets
      • Table Figure 9: Duchenne Muscular Dystrophy and Becker Muscular Dystrophy, Global, Pipeline, First-in-class Products in the Pipeline
  • Signaling Pathways, Genetics and Innovation Alignment
    • First-in-Class Target Matrix Assessment
      • Table Figure 10: First-in-Class Molecular Target Analysis Matrix
    • Repositioning Potential among Muscular Dystrophies
  • First-in-Class Target Evaluation
    • Pipeline Programs Targeting Sarcospan
      • Table Figure 11: Data and Evidence for Sarcospan as a Therapeutic target
      • Table Figure 12: Pipeline Programs Targeting Sarcospan
    • Pipeline Programs Targeting Utrophin
      • Table Figure 13: Data and Evidence for Utrophin as a Therapeutic target
      • Table Figure 14: Pipeline Programs Targeting Utrophin
    • Pipeline Programs Targeting Biglycan
      • Table Figure 15: Data and Evidence for Biglycan as a Therapeutic target
      • Table Figure 16: Pipeline Programs Targeting Biglycan
    • Pipeline Programs Targeting Laminin-111
      • Table Figure 17: Data and Evidence for Laminin-111 as a Therapeutic Target
      • Table Figure 18: Pipeline Programs Targeting Laminin-111
    • Pipeline Programs Targeting Hematopoietic Prostaglandin D Synthase
      • Table Figure 19: Data and Evidence for Hematopoietic Prostaglandin D Synthase as a Therapeutic target
      • Table Figure 20: Pipeline Programs Targeting Hematopoietic Prostaglandin D Synthase
    • Pipeline Programs which Target G Protein-Coupled Receptor Mas
      • Table Figure 21: Data and Evidence for G Protein-Coupled Receptor Mas as a Therapeutic target
      • Table Figure 22: Pipeline Programs Targeting G Protein-Coupled Receptor Mas
    • Pipeline Programs Targeting Mothers against Decapentaplegic Homolog 3
      • Table Figure 23: Data and Evidence for Mothers against decapentaplegic homolog 3 as a Therapeutic target
      • Table Figure 24: Pipeline Programs Targeting Mothers against decapentaplegic homolog 3
    • Pipeline Programs which Target Myostatin
      • Table Figure 25: Data and Evidence for Myostatin as a Therapeutic target
      • Table Figure 26: Pipeline Programs Targeting Myostatin
    • Pipeline Programs Targeting Dystrophin
      • Table Figure 27: Data and Evidence for Dystrophin as a Therapeutic target
      • Table Figure 28: Pipeline Programs Targeting Dystrophin
    • Conclusion
  • Deals and Strategic Consolidations
    • Industry-Wide First-in-Class Deals
      • Table Figure 29: Industry-Wide Deals by Stage of Development, 2006-2014
      • Table Figure 30: Industry Licensing Deal Values by Stage of Development, 2006-2014
    • Licensing Deals
      • Table Figure 31: Licensing Deals in Duchenne Muscular Dystrophy, 2006-2015
      • Table Figure 32: Licensing Deals Global Distribution, 2006-2015
      • Table Figure 33: Licensing Deals by Molecule Type, 2006-2015
      • Table Figure 34: Licensing Deals by Molecular Target, 2006-2015
      • Table Figure 35: Summary of Licensing Deals, 2006-2015
    • Co-development Deals
      • Table Figure 36: Co-development Deals by Year, 2006-2015
      • Table Figure 37: Co-development Deals Global Distribution, 2006-2015
      • Table Figure 38: Co-development Deals by Molecule Type, 2006-2015
      • Table Figure 39: Co-development Deals by Molecular Target, 2006-2015
      • Table Figure 40: Summary of Co-Development Deals, 2006-2015
    • First-in-Class Programs Not Involved in Licensing or Co-Development Deals
      • Table Figure 41: First-in-Class Programs with no Recorded Prior Deal Involvement, 2006-2015
  • Appendix
    • Abbreviations
    • References
    • Contact Us
    • Disclaimer

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

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

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


Muscular Dystrophy Treatment Pipeline Offers Hope for Improved Life Expectancy, says GBI Research

The highly diverse and innovative pipeline for Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) treatment has the potential to produce therapies that will help repair the mutated gene, halt muscle degeneration, and improve patients’ life expectancy, says business intelligence provider GBI Research.

The company’s latest report* states that 70% of all pipeline programs with disclosed molecular targets are first-in-class. This exceptional level of innovation is largely due to the high number of first-in-class products that solely target the dystrophin gene, which is the primary genetic cause of DMD and BMD.

According to Angel Wong, Senior Analyst for GBI Research, the pipeline offers hope of improved treatment options for a condition that has no cure available at present, as the current DMD and BMD treatment space is extremely sparse and largely symptomatic, with limited long-term benefits.

Wong explains: “Over the last few decades, the market landscape has relied on the use of glucocorticoids, which offer symptomatic benefits in increasing muscle strength and a delay in the loss of ambulation in DMD/BMD patients, without altering the genetic cause and dystrophic pathology.

“Translarna (ataluren), despite being the first approved therapy for treating the underlying genetic cause in the EU, is only applicable in 10–15% of all DMD cases, leaving other patient segments with high unmet needs.”

However, the DMD and BMD pipeline consists of 84 molecules across all stages of development. GBI Research’s analysis has identified 46 first-in-class programs in active development, acting on 13 first-in-class targets.

Wong continues: “Overall, the DMD/BMD pipeline is very diverse in terms of molecular targets. Apart from the technologies that directly target the dystrophin gene, the pipeline also contains novel targets that alleviate the dystrophic pathology regardless of gene mutations. These targets include some dystrophin-associated proteins, which have the potential to functionally compensate for its loss and are therefore intended to treat the whole patient population.

“The diversity of molecular targets across the developmental pipeline presents a dramatic contrast to the current DMD therapeutics market landscape, and is expected to transform the market by fragmenting the current treatment algorithm,” the analyst concludes.

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

This report provides analysis of innovation in Duchenne Muscular Dystrophy (DMD) and Becker Muscular Dystrophy (BMD) therapeutics in the context of the overall pipeline and current market landscape. It includes an evaluation of the deals landscape surrounding first-in-class products in DMD/BMD treatment and pinpoints opportunities for in-licensing.

This report was built using data and information sourced from proprietary databases, primary and secondary research, and in-house analysis conducted by GBI Research’s team of industry experts.

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