Global Niemann Pick Type C Disease - Pipeline Analysis 2019

Global Niemann Pick Type C Disease - Pipeline Analysis 2019

Global Niemann Pick Type C Disease - Pipeline Analysis 2019

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$2,058

Published: Jun-2019

Pages:79

Table:32

Figure:13

Report ID:UMHE19157

Niemann Pick Type-C Disease (NPC) is a progressive autosomal recessive genetic disorder of lysosome storage due to an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. The accumulation of these substances damages the affected areas. It can range from a fatal disorder within the first few months after birth (neonatal period) to a late onset, chronic progressive disorder that remains undiagnosed well into adulthood. It affects both males and females equally. NPC is estimated to occur in 1 in 100,000-120,000 live births.

The onset of symptoms may begin at any age, most commonly in school age children; however, infants, adolescents and adults can all be affected by the disease.  With the progression of disease, children may experience developmental delays, challenges with motor skills (e.g. increased clumsiness and abnormal posturing) and difficulty in making vertical eye movements. Dystonia and seizures are common in the patients suffering from NPC.

Split of Therapeutic Products

Several tests are available for the diagnosis of NPC. These tests include biochemical testing, molecular genetic testing, NPC screening using biomarkers among others. No marketed drugs are currently present for NPC in the U.S. The drug development companies are involved in quest to develop an effective drug and better treatment strategies. There are several companies utilizing their proprietary technologies for the development of effective and better drugs.

The therapeutic pipeline of NPC Disease consists of approximately 9+ products in different stages of development. Currently 2+ drugs are in Phase III development and major drugs are in pre-clinical and discovery stage.

Top Company Analysed

Some of the key players include Orphazyme, CTD Holdings, Mallinckrodt Pharmaceuticals, IntraBio, Perlara, Neurotrope BioScience, Evox Therapeutics, Okklo Life Sciences, Amicus Therapeutics.

Scope of the study:

  • Provides an overview of therapeutic pipeline activity for Niemann Pick Type-C Disease across the complete product development cycle including all clinical and non-clinical stages
  • The report comprises of detailed description of Niemann Pick Type-C Disease 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

Reasons to buy:

  • The Niemann Pick Type-C Disease (NPC) 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

Customization Options:

The Niemann Pick Type-C Disease 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 Niemann-Pick Disease
      2.2.1 Niemann-Pick disease type A
      2.2.2 Niemann-Pick disease type B
      2.2.3 Niemann-Pick disease type C
    2.3 Classification of NPC
    2.4 Signs and Symptoms
    2.5 Causes    
      2.5.1 NPC1 and NPC2 proteins
    2.6 Pathogenesis  
      2.6.1 Molecular Pathogenesis
      2.6.2 NPC1  
      2.6.3 NPC2  
    2.7 Pathophysiology
      2.7.1 Cell Death and Oxidative Damage in NPC
      2.7.2 Increase in Mitochondrial Cholesterol and Dysfunction in NPC Disease
      2.7.3 Peroxisomes Alterations in NPC Disease
      2.7.4 Intracellular Accumulation of Vitamin E in NPC Disease
      2.7.5 Glycosphingolipid Accumulation in NPC Disease
      2.7.6 Other Sources of Reactive Species in Oxidative Damage in NPC
    2.8 Diagnosis    
      2.8.1 Biochemical Testing:
      2.8.2 Histology:  
      2.8.3 Molecular Genetic Testing:
      2.8.4 Testing Strategy
      2.8.5 Biomarkers:
        2.8.5.1 Oxysterols (cholesterol oxidation products)
        2.8.5.2 Lyso-sphingolipids
        2.8.5.3 Bile acids
      2.8.6 The Filipin test
      2.8.7 Brain imaging
      2.8.8 Differential Diagnosis
        2.8.8.1 Neonatal and infantile presentations
        2.8.8.2 Childhood presentations
        2.8.8.3 Adolescent and adult presentations
    2.9 Diagnosis Algorithm
    2.10 Treatment  
      2.10.1 Symptomatic therapy
      2.10.2 Evaluations Following Initial Diagnosis
      2.10.3 Treatment of Manifestations
      2.10.4 Prevention of Secondary Complications
      2.10.5 Surveillance
      2.10.6 Agents/Circumstances to Avoid
      2.10.7 Evaluation of Relatives at Risk
      2.10.8 Therapies Under Investigation
      2.10.9 Other  
    2.11 Genetic Counselling
    2.12 Treatment Algorithm
  3 PIPELINE THERAPEUTICS AT A GLANCE
    3.1 Products Under Development by Technology
  4 COMPARATIVE ANALYSIS
    4.1 Number of Products in NPC
  5 LATE PHASE PRODUCTS (PHASE III)
    5.1 Comparative Analysis
    5.2 Arimoclomol: Orphazyme
      5.2.1 Product Description
      5.2.2 Research and Development
        5.2.2.1 Clinical Studies
          5.2.2.1.1 Phase II/III
          5.2.2.1.2 Results of Analysis
        5.2.2.2 Observational Study
        5.2.2.3 Results of Analysis
      5.2.3 Product Development Activities
        5.2.3.1 Agreement
        5.2.3.2 Designation
    5.3 Trappsol Cyclo: CTD Holdings
      5.3.1 Product Description
      5.3.2 Research and Development
        5.3.2.1 Clinical Studies
          5.3.2.1.1 Phase II/III
          5.3.2.1.2 Results of Analysis
      5.3.3 Product Development Activities
        5.3.3.1 Designation
        5.3.3.2 Clinical Trial Application Approval
        5.3.3.3 Compassionate Use
    5.4 Adrabetadex: Mallinckrodt Pharmaceuticals
      5.4.1 Product Description
      5.4.2 Research and Development
        5.4.2.1 Clinical Studies
          5.4.2.1.1 Phase IIb/III
          5.4.2.1.2 Results of Analysis
      5.4.3 Product Development Activities
        5.4.3.1 FDA Type A Meeting
        5.4.3.2 Agreement
        5.4.3.3 Funding
        5.4.3.4 Designation
        5.4.3.5 Patent
  6 MID PHASE PRODUCTS (PHASE II)
    6.1 Comparative Analysis
    6.2 IB1001: IntraBio  
      6.2.1 Product Description
      6.2.2 Research and Development
        6.2.2.1 Clinical Studies
          6.2.2.1.1 Phase II
      6.2.3 Product Development Activities
        6.2.3.1 Designation
  7 PRE-CLINICAL AND DISCOVERY STAGE PRODUCTS
    7.1 Comparative Analysis
    7.2 Ursodeoxycholic acid: IntraBio
      7.2.1 Product Description
      7.2.2 Research and Development
        7.2.2.1 Observational Study
      7.2.3 Product Development Activities
        7.2.3.1 Designation
    7.3 PERL101: Perlara
      7.3.1 Product Description
      7.3.2 Research and Development
        7.3.2.1 Preclinical Studies
      7.3.3 Product Development Activities
        7.3.3.1 Technology
        7.3.3.2 Collaboration
    7.4 Bryostatin-1: Neurotrope BioScience
      7.4.1 Product Description
        7.4.1.1 Agreement
    7.5 Exosomes: Evox Therapeutics
      7.5.1 Product Description
      7.5.2 Product Development Activities
        7.5.2.1 Funding
        7.5.2.2 Financing
        7.5.2.3 Technology
        7.5.2.4 Patent
    7.6 Gene therapy for NPC: Amicus Therapeutics
      7.6.1 Product Description
      7.6.2 Product Development Activities
        7.6.2.1 Agreement
    7.7 Cyclodextrins: Okklo Life Sciences
      7.7.1 Product Description
      7.7.2 Product Development Activities
        7.7.2.1 Technology
        7.7.2.2 Agreement
  8 THERAPEUTIC ASSESSMENT
    8.1 Assessment by Stage and Product Type
    8.2 Assessment by Route of Administration
    8.3 Assessment by Stage and Route of Administration
    8.4 Assessment by Molecule Type
    8.5 Assessment by Stage and Molecule Type
  9 APPROVAL TIMELINES
    9.1 Approval Timelines for Clinical Products
  10 ANALYST INSIGHTS  

Overview of pipeline development activities for Niemann Pick Type-C Disease Pipeline Analysis of therapeutic drugs is not limited to drug description and development activities but also focuses on clinical and pre-clinical results. It also includes designations, company consolidations & licensing deals, grants, technology and patent details. Therapeutic segmentation of products for Niemann Pick Type-C Disease is done based on phase of development. The report comprises of comparative pipeline therapeutics assessment by development stage, product 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.

Secondary Research

Detail secondary study was conducted to obtain the Niemann Pick Type-C  Disease 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 Niemann Pick Type-C Disease - Pipeline Analysis 2019

The current pipeline trends of the Niemann Pick Type-C Disease 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 Niemann Pick Type-C Disease across the complete product development cycle including all clinical and non-clinical stages
  • Analyse the detailed profiles of Niemann Pick Type-C Disease 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

Overview of pipeline development activities for Niemann Pick Type-C Disease Pipeline Analysis of therapeutic drugs is not limited to drug description and development activities but also focuses on clinical and pre-clinical results. It also includes designations, company consolidations & licensing deals, grants, technology and patent details. Therapeutic segmentation of products for Niemann Pick Type-C Disease is done based on phase of development. The report comprises of comparative pipeline therapeutics assessment by development stage, product 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.

Secondary Research

Detail secondary study was conducted to obtain the Niemann Pick Type-C  Disease 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 Niemann Pick Type-C Disease - Pipeline Analysis 2019

The current pipeline trends of the Niemann Pick Type-C Disease 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 Niemann Pick Type-C Disease across the complete product development cycle including all clinical and non-clinical stages
  • Analyse the detailed profiles of Niemann Pick Type-C Disease 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

Global Niemann Pick Type C Disease - Pipeline Analysis 2019