Global Adrenoleukodystrophy (ALD) - Pipeline Analysis, 2019

Global Adrenoleukodystrophy (ALD) - Pipeline Analysis, 2019

Global Adrenoleukodystrophy (ALD) - Pipeline Analysis, 2019

$ 1,500
$1,950
$2,550

Published: Jun-2019

Pages:77

Table:30

Figure:20

Report ID:UMHE19159

Adrenoleukodystrophy is a fatal progressive neurogenerative disorder affecting brain white matter. The common form of ALD is X-linked (X-ALD) and results from mutation of ABCD1 encoded very long chain fatty acid (VLCFA) transporter. It is clinically heterogeneous with the cerebral form being the most severe. X-linked Adrenoleukodystrophy (X-ALD) is a rare X-linked metabolic disorder caused by variants in the ABCD1 gene which causes a deficiency in adrenoleukodystrophy protein (ALDP) and subsequent accumulation of very long chain fatty acids (VLCFA).

The symptoms of Adrenoleukodystrophy are highly variable. There are mainly three types which are present in approximately 90-95% of the affected people: a childhood cerebral form; an adrenomyeloneuropathy type; and an adrenal insufficiency only type.

Split of Therapeutic Products

 

The diagnosis of ALD, therefore, can be made with the highly sensitive and specific plasma VLCFA assay. When an abnormal pattern is detected with an identifiable ABCD1 mutation in a presymptomatic patient (or in the setting of classic clinical disease in others), males are determined to have the biochemical defect of ALD. Disease may manifest at any age. Most ALD males will develop primary adrenal insufficiency (AI) in childhood or adolescence, and most surviving males will develop spinal cord disease (adrenomyeloneuropathy [AMN]) during adulthood.

The therapeutic pipeline of Adrenoleukodystrophy consists of approximately 9+ products in different stages of development. Currently, 3+ drugs are in Phase III development and major drugs are in late stage.

Top Company Analysed

Some of the key players include Applied Genetic Technologies Corporation; Bluebird bio; Magenta Therapeutics; MedDay Pharmaceuticals; Minoryx Therapeutics; NeuroVia; Orpheris; ReceptoPharm; SOM Biotech; and Viking Therapeutics.

Scope of the study:

  • Provides an overview of therapeutic pipeline activity for Adrenoleukodystrophy across the complete product development cycle including all clinical and non-clinical stages
  • The report comprises of detailed profiles of Adrenoleukodystrophy therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical information
  • Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration
  • Detailed profiles of the discontinued products have been included in the report

Reasons to buy:

  • The Adrenoleukodystrophy pipeline presents the detailed profile of drugs. The analysis offered in the report is a combination of deep dive secondary research and input from Key Opinion Leader of the industry
  • The report presents a quick review of the current scenario regarding the drug development of the indication at one glance
  • The report covers in-depth analysis of prominent industry peers with a primary focus on company consolidation, designation, technology, agreements and patents regarding the therapy
  • Detailed examination on diagnosis, treatment and guidelines prevailing in the industry
  • Examination of industry attractiveness with the help of approval timelines
  • The study comprehensively covers the market across drugs in different phases of development
  • Extensive domain knowledge on therapy areas support the client in decision-making process regarding their therapeutic portfolio by identifying the reason behind the discontinuation of the products

Customization Options:

The Adrenoleukodystrophy pipeline analysis report can be customized to the country level or any other competitive segment. Besides this, UMI understands that you may have your own business need, hence we also provide fully customized solutions to clients.

             
  1 REPORT INTRODUCTION
    1.1 Objective of the Study
    1.2 Secondary Research
    1.3 Scope of the report:
  2 DISEASE OVERVIEW  
    2.1 Introduction  
    2.2 Classification of Adrenoleukodystrophy
    2.3 Signs and Symptoms
    2.4 Causes    
    2.5 Pathophysiology
      2.5.1 Biochemical and Genetic Aspects of X-ALD Pathophysiology
      2.5.2 Lipidomic aspects of the pathophysiology of X-ALD
      2.5.3 Functional redundancy of ABC transporters and their role in the pathophysiology of X-ALD
      2.5.4 Pathophysiology of AMN in males and in heterozygous females
        2.5.4.1 Pathophysiology of inflammation in cerebral X-ALD
      2.5.5 Pathophysiology of adrenals, testis and hair in X-ALD
      2.5.6 Cerebral Adrenoleukodystrophy
    2.6 Diagnosis    
      2.6.1 Neuroimaging
      2.6.2 Very long-chain fatty acids
      2.6.3 Mutation analysis
      2.6.4 Genetic counseling
      2.6.5 Heterozygote identification
      2.6.6 Extended family testing
      2.6.7 Prenatal testing
      2.6.8 Newborn screening
      2.6.9 Differential Diagnosis
      2.6.10 Biochemical and Molecular Diagnosis
    2.7 Treatment  
      2.7.1 Boys and adult males with X-ALD
      2.7.2 Women with X-ALD
  3 COMPARATIVE ANALYSIS BY FINANCING
    3.1 Overview of Comparative Analysis
    3.2 Company Consolidations under Partnership and Agreements
  4 PRODUCTS UNDER DEVELOPMENT BY DESIGNATION
    4.1 Comparative Analysis
  5 PIPELINE THERAPEUTICS AT A GLANCE
  6 COMPARATIVE ANALYSIS
    6.1 Comparative Analysis of Product under Adrenoleukodystrophy
  7 LATE PHASE PRODUCTS (PHASE III)
    7.1 Comparative Analysis
    7.2 Hydroxypioglitazone: Minoryx Therapeutics
      7.2.1 Product Description
      7.2.2 Research and Development
        7.2.2.1 Clinical Studies
          7.2.2.1.1 Phase II/III
          7.2.2.1.2 Results of the Analysis
      7.2.3 Product Development Activities
        7.2.3.1 Funding
    7.3 Lenti-D: bluebird bio
      7.3.1 Product Description
      7.3.2 Research and Development
        7.3.2.1 Clinical Studies
          7.3.2.1.1 Phase III
          7.3.2.1.2 Phase II/III
          7.3.2.1.3 Results of Analysis
      7.3.3 Product Development Activities
        7.3.3.1 Designation
    7.4 RPI-78M: ReceptoPharm
      7.4.1 Product Description
      7.4.2 Product Development Activities
        7.4.2.1 Designation
        7.4.2.2 Agreement
    7.5 MD1003: MedDay Pharmaceuticals
      7.5.1 Product Description
      7.5.2 Research and Development
        7.5.2.1 Pre-clinical Studies
        7.5.2.2 Clinical Studies
          7.5.2.2.1 Phase II/III
  8 MID PHASE PRODUCTS (PHASE II)
    8.1 Comparative Analysis
    8.2 Spanlecortemlocel: Magenta Therapeutics
      8.2.1 Product Description
      8.2.2 Research and Development
        8.2.2.1 Preclinical Studies
        8.2.2.2 Clinical Studies
          8.2.2.2.1 Phase II
          8.2.2.2.2 Results of Analysis
      8.2.3 Product Development Activities
        8.2.3.1 Licensing
        8.2.3.2 IND Application
        8.2.3.3 Financing
  9 EARLY STAGE PRODUCTS (PHASE I)
    9.1 Comparative Analysis
    9.2 OP-101: Orpheris
      9.2.1 Product Description
      9.2.2 Research and Development
        9.2.2.1 Pre-clinical Studies
        9.2.2.2 Clinical Studies
          9.2.2.2.1 Phase I
      9.2.3 Product Development Activities
        9.2.3.1 Designation
        9.2.3.2 Type C Meeting
        9.2.3.3 Technology
    9.3 SOM1201: SOM Biotech
      9.3.1 Product Description
  10 PRE-CLINICAL STAGE PRODUCTS
    10.1 Comparative Analysis
    10.2 Pre-clinical Program for ALD: Applied Genetic Technologies Corporation
      10.2.1 Product Description
    10.3 SOM1202: SOM Biotech
      10.3.1 Product Description
    10.4 VK0214: Viking Therapeutics
      10.4.1 Product Description
      10.4.2 Research and Development
        10.4.2.1 Pre-clinical Studies
      10.4.3 Product Development Activities
        10.4.3.1 Designation
        10.4.3.2 Collaboration
  11 THERAPEUTIC ASSESSMENT
    11.1 Assessment by Stage and Product Type
    11.2 Assessment by Route of Administration
    11.3 Assessment by Stage and Route of Administration
    11.4 Assessment by Molecule Type
    11.5 ASSESSMENT by Stage and Molecule Type
  12 APPROVAL TIMELINES
  13 ANALYST INSIGHTS  
  14 INACTIVE PRODUCTS  
    14.1 Comparative Analysis
    14.2 Sobetirome: NeuroVia
      14.2.1 Product Description
      14.2.2 Research and Development
        14.2.2.1 Clinical Studies
      14.2.3 Product Development Activities
        14.2.3.1 Designation
        14.2.3.2 Financing
        14.2.3.3 Agreement

Overview of pipeline development activities for Adrenoleukodystrophy Pipeline Analysis of therapeutic drugs is not limited to drug description and development activities but also focuses on clinical and non-clinical results. It also includes designations, company consolidations & licensing deals, grants, technology and patent details. Therapeutic segmentation of products for Adrenoleukodystrophy is done based on phase of development. The report comprises of comparative pipeline therapeutics assessment along with detailed drug profile of pipeline products by development stage, therapy type, molecule type, and administration route across this indication. The report also consists of the approval timelines forecasted for the upcoming pipeline therapies. Additionally, Analyst Insight have also been focused upon to give a summary regarding the current market scenario. The detailed profiles of the dormant products have been elucidated in the complete report with the relevant reasons for their discontinuation.

Secondary Research

Detail secondary study was conducted to obtain the Adrenoleukodystrophy Pipeline Analysis through company internal sources such as annual reports, performance presentations, press releases, inventory records etc., and external sources including trade journals, news & articles, government publications, competitor publications, sector reports, regulatory bodies publications, safety standard organizations, third-party database and other creditable publications.

The secondary research on internal and external sources is being carried out to source qualitative and quantitative information relating to each market.

  • Company websites, annual reports, quarter reports, financial reports, broker reports, investor presentations and SEC filings
  • Industry trade journals and other literature
  • National government documents, statistical databases and market reports
  • News articles, press releases and web-casts specific to the companies operating in the market
  • Several databases for patents and clinical trials

Main objective of the Adrenoleukodystrophy - Pipeline Analysis 2019

The current pipeline trends of the Adrenoleukodystrophy are pinpointed in the study. Investors can gain strategic insights to base their discretion for investments from the qualitative and quantitative analysis performed in the study. Current and future pipeline trends would determine the overall attractiveness of the market, providing a platform for the industrial participant to exploit the untapped market to benefit as first mover advantage. The quantitative objectives for the report include:

  • An overview of therapeutic pipeline activity for Adrenoleukodystrophy across the complete product development cycle including all clinical and non-clinical stages
  • Analyse the detailed profiles of Adrenoleukodystrophy therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical properties information
  • Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration

Overview of pipeline development activities for Adrenoleukodystrophy Pipeline Analysis of therapeutic drugs is not limited to drug description and development activities but also focuses on clinical and non-clinical results. It also includes designations, company consolidations & licensing deals, grants, technology and patent details. Therapeutic segmentation of products for Adrenoleukodystrophy is done based on phase of development. The report comprises of comparative pipeline therapeutics assessment along with detailed drug profile of pipeline products by development stage, therapy type, molecule type, and administration route across this indication. The report also consists of the approval timelines forecasted for the upcoming pipeline therapies. Additionally, Analyst Insight have also been focused upon to give a summary regarding the current market scenario. The detailed profiles of the dormant products have been elucidated in the complete report with the relevant reasons for their discontinuation.

Secondary Research

Detail secondary study was conducted to obtain the Adrenoleukodystrophy Pipeline Analysis through company internal sources such as annual reports, performance presentations, press releases, inventory records etc., and external sources including trade journals, news & articles, government publications, competitor publications, sector reports, regulatory bodies publications, safety standard organizations, third-party database and other creditable publications.

The secondary research on internal and external sources is being carried out to source qualitative and quantitative information relating to each market.

  • Company websites, annual reports, quarter reports, financial reports, broker reports, investor presentations and SEC filings
  • Industry trade journals and other literature
  • National government documents, statistical databases and market reports
  • News articles, press releases and web-casts specific to the companies operating in the market
  • Several databases for patents and clinical trials

Main objective of the Adrenoleukodystrophy - Pipeline Analysis 2019

The current pipeline trends of the Adrenoleukodystrophy are pinpointed in the study. Investors can gain strategic insights to base their discretion for investments from the qualitative and quantitative analysis performed in the study. Current and future pipeline trends would determine the overall attractiveness of the market, providing a platform for the industrial participant to exploit the untapped market to benefit as first mover advantage. The quantitative objectives for the report include:

  • An overview of therapeutic pipeline activity for Adrenoleukodystrophy across the complete product development cycle including all clinical and non-clinical stages
  • Analyse the detailed profiles of Adrenoleukodystrophy therapeutic products with key coverage of developmental activities including licensing & collaboration deals, patents issued, designations, technologies and chemical properties information
  • Therapeutic assessment of the active pipeline products by stage, product type, molecule type, and route of administration

Global Adrenoleukodystrophy (ALD) - Pipeline Analysis, 2019