Niemann Pick C Disease - Pipeline Insight, 2021
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DelveInsight’s, “Niemann Pick C Disease – Pipeline Insight, 2021,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Niemann Pick C Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Niemann Pick C Disease: Overview
Niemann-Pick disease type C (NPC) is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. NPC belongs to a larger group of more than 50 disorders known as lysosomal storage disorders. This leads to the abnormal accumulation of these substances within various tissues of the body, including brain tissue. Individuals with NPC can have onset of symptoms at different ages that have been grouped historically as: perinatal (shortly before and after birth), early infantile (3 months to < 2 years), late infantile (2 to < 6 years), juvenile (6 to < 15 years), and adult (15 years and greater). Individuals with NPC have mutations in one of two genes, NPC1 or NPC2 and are inherited in an autosomal recessive manner. Approximately 95% of affected individuals have mutations in NPC1.
“Niemann Pick C Disease - Pipeline Insight, 2021” report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Niemann Pick C Disease pipeline landscape is provided which includes the disease overview and Niemann Pick C Disease treatment guidelines. The assessment part of the report embraces, in depth Niemann Pick C Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Niemann Pick C Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Niemann Pick C Disease R&D. The therapies under development are focused on novel approaches to treat/improve Niemann Pick C Disease.
Niemann Pick C Disease Emerging Drugs Chapters
This segment of the Niemann Pick C Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Niemann Pick C Disease Emerging Drugs
VTS-270(Adrabetadex): Mallinckrodt
VTS-270 (Mallinckrodt) also known as Kleptose; 2-Hydroxypropyl- beta-cyclodextrin; HP-beta- CD; HPBCD) is under development. The drug has received various designations from the United States Food Drug and Administration (FDA)-Rare Pediatric Disease Designation, Promising Innovative Medicine (PIM) Designation, Breakthrough Therapy Designation and Orphan Drug Designation for the treatment of NPC. VTS-270 is currently in phase III stage of development,an intrathecal preparation that can cross the Blood Brain Barrier (BBB) and as a result, it targets the neurological symptoms of NPC such as dementia and cataplexy.
IB1001: IntraBio
IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. IBI001 is in Phase 2 of clinical study and is being developed by IntraBio. IBI001 acts as calcium channel modulators. In addition to the Fast Track Designation, IB1001 has previously received Orphan Drug Designations in the US (FDA) and EU (European Commission), and granted a Rare Pediatric Disease Designation in the US (FDA) for the treatment of NPC.
Further product details are provided in the report……..
Niemann Pick C Disease: Therapeutic Assessment
This segment of the report provides insights about the different Niemann Pick C Disease drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Niemann Pick C Disease
There are approx. 10+ key companies which are developing the therapies for Niemann Pick C Disease. The companies which have their Niemann Pick C Disease drug candidates in the most advanced stage, i.e. phase III include, Mallinckrodt.
Phases
DelveInsight’s report covers around 10+ products under different phases of clinical development like
Niemann Pick C Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Niemann Pick C Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Niemann Pick C Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Niemann Pick C Disease drugs.
Niemann Pick C Disease Report Insights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Niemann Pick C Disease – Pipeline Insight, 2021,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in the Niemann Pick C Disease pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Niemann Pick C Disease: Overview
Niemann-Pick disease type C (NPC) is a rare progressive genetic disorder characterized by an inability of the body to transport cholesterol and other fatty substances (lipids) inside of cells. NPC belongs to a larger group of more than 50 disorders known as lysosomal storage disorders. This leads to the abnormal accumulation of these substances within various tissues of the body, including brain tissue. Individuals with NPC can have onset of symptoms at different ages that have been grouped historically as: perinatal (shortly before and after birth), early infantile (3 months to < 2 years), late infantile (2 to < 6 years), juvenile (6 to < 15 years), and adult (15 years and greater). Individuals with NPC have mutations in one of two genes, NPC1 or NPC2 and are inherited in an autosomal recessive manner. Approximately 95% of affected individuals have mutations in NPC1.
“Niemann Pick C Disease - Pipeline Insight, 2021” report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Niemann Pick C Disease pipeline landscape is provided which includes the disease overview and Niemann Pick C Disease treatment guidelines. The assessment part of the report embraces, in depth Niemann Pick C Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Niemann Pick C Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
The companies and academics are working to assess challenges and seek opportunities that could influence Niemann Pick C Disease R&D. The therapies under development are focused on novel approaches to treat/improve Niemann Pick C Disease.
Niemann Pick C Disease Emerging Drugs Chapters
This segment of the Niemann Pick C Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Niemann Pick C Disease Emerging Drugs
VTS-270(Adrabetadex): Mallinckrodt
VTS-270 (Mallinckrodt) also known as Kleptose; 2-Hydroxypropyl- beta-cyclodextrin; HP-beta- CD; HPBCD) is under development. The drug has received various designations from the United States Food Drug and Administration (FDA)-Rare Pediatric Disease Designation, Promising Innovative Medicine (PIM) Designation, Breakthrough Therapy Designation and Orphan Drug Designation for the treatment of NPC. VTS-270 is currently in phase III stage of development,an intrathecal preparation that can cross the Blood Brain Barrier (BBB) and as a result, it targets the neurological symptoms of NPC such as dementia and cataplexy.
IB1001: IntraBio
IB1001, N-acetyl-L-leucine, is an orally administered modified amino acid. IBI001 is in Phase 2 of clinical study and is being developed by IntraBio. IBI001 acts as calcium channel modulators. In addition to the Fast Track Designation, IB1001 has previously received Orphan Drug Designations in the US (FDA) and EU (European Commission), and granted a Rare Pediatric Disease Designation in the US (FDA) for the treatment of NPC.
Further product details are provided in the report……..
Niemann Pick C Disease: Therapeutic Assessment
This segment of the report provides insights about the different Niemann Pick C Disease drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Niemann Pick C Disease
There are approx. 10+ key companies which are developing the therapies for Niemann Pick C Disease. The companies which have their Niemann Pick C Disease drug candidates in the most advanced stage, i.e. phase III include, Mallinckrodt.
Phases
DelveInsight’s report covers around 10+ products under different phases of clinical development like
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
Niemann Pick C Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Oral
- Parenteral
- Subcutaneous
- Intravenous
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Niemann Pick C Disease: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Niemann Pick C Disease therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Niemann Pick C Disease drugs.
Niemann Pick C Disease Report Insights
- Niemann Pick C Disease Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Niemann Pick C Disease drugs?
- How many Niemann Pick C Disease drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Niemann Pick C Disease?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Niemann Pick C Disease therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Niemann Pick C Disease and their status?
- What are the key designations that have been granted to the emerging drugs?
- IntraBio
- Mallinckrodt
- Cyclo Therapeutics
- SOM Biotech
- Evox therapeutics
- Orphazyme
- SOM0208
- IB1001
- Trappsol Cyclo
- VTS-270
- Arimoclomol
- Hydroxypropyl-beta-cyclodextrin
Introduction
Executive Summary
Niemann Pick C Disease: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Niemann Pick C Disease – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Niemann Pick C Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Niemann Pick C Disease Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
VTS-270(Adrabetadex): Mallinckrodt
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
Comparative Analysis
IB1001: IntraBio
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I/II)
Comparative Analysis
Hydroxypropyl Betadex: Cyclo Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
Comparative Analysis
Niemann Pick C Disease Key Companies
Niemann Pick C Disease Key Products
Niemann Pick C Disease- Unmet Needs
Niemann Pick C Disease- Market Drivers and Barriers
Niemann Pick C Disease- Future Perspectives and Conclusion
Niemann Pick C Disease Analyst Views
Niemann Pick C Disease Key Companies
Appendix
Executive Summary
Niemann Pick C Disease: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Niemann Pick C Disease – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Niemann Pick C Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Niemann Pick C Disease Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
VTS-270(Adrabetadex): Mallinckrodt
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Mid Stage Products (Phase II)
Comparative Analysis
IB1001: IntraBio
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I/II)
Comparative Analysis
Hydroxypropyl Betadex: Cyclo Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
Comparative Analysis
Niemann Pick C Disease Key Companies
Niemann Pick C Disease Key Products
Niemann Pick C Disease- Unmet Needs
Niemann Pick C Disease- Market Drivers and Barriers
Niemann Pick C Disease- Future Perspectives and Conclusion
Niemann Pick C Disease Analyst Views
Niemann Pick C Disease Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Niemann Pick C Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Table 1 Total Products for Niemann Pick C Disease
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
LIST OF FIGURES
Figure 1 Total Products for Niemann Pick C Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Figure 1 Total Products for Niemann Pick C Disease
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
