Leber Hereditary Optic Neuropathy - Pipeline Review, 2019
Firstview Insight's Leber Hereditary Optic Neuropathy - Pipeline Review, 2019 provides an overview of the pipeline landscape of Leber Hereditary Optic Neuropathy It provides comprehensive insights of all the clinical and non-clinical therapeutics in development with detailed description about the collaborations; deals; designations; patent information etc.These reports encourage the clients in distinguishing the upcoming and existing competitors in their separate therapeutic spaces. The report provides detailed description of the competitor profiles with key milestones and evidence along with analysis by mechanism of action; route of administration; molecule type; stage of development. Information obtained from multiple sources will be used to triangulate and update the profiles. The report also provides key events in the last year related to the indication. This report provides detailed analysis of all the products along with the companies involved.
Leber Hereditary Optic Neuropathy (LHON) is the most common inherited mitochondrial disorder and typically affects young males. It typically begins as a unilateral progressive optic neuropathy with sequential involvement of the fellow eye months to years later. LHON is classically associated with mitochondrial base pair mutations G11778A (guanine to adenine at position 11778), T14484C (tyrosine to cytosine), and G3460A. These mutations primarily affect respiratory chain complex I genes, mitochondrial genes ND1, ND4 and ND6 among others. The G11778A is the most common, reported to account for 70% of cases in Northern European populations, and 90% in Asian populations. It is associated with more severe disease, with less chance of significant visual recovery. The T14484C mutation is associated with the best overall prognosis, with some visual recovery in 37-58% of cases. The G3460A mutation usually portends a more intermediate course and has the worst prognosis.
Treatment options are limited, but include the use of antioxidant supplements. Gene therapy trials are currently underway.
LIST OF PRODUCTS:
The pipeline section provides descriptive drug profiles for the pipeline products including product description, mechanism of action, route of administration, molecule type, technology involved, chemical information.
Clinical Trial Overview:
This section of the report focuses on the clinical activity of the molecule. It includes both clinical and pre-clinical activity which provides detailed information about the safety, efficacy, tolerability, toxicity of pipeline drugs. A graphical representation of the clinical trial landscape of pipeline therapy which includes information about phase of development, trial design, treatment arms, dosage and frequency, formulation of the drug, primary and secondary completion date, enrolment number, exclusion and inclusion criteria, line of therapy. This section also includes the clinical trial results and analysis based on those results.
Product Development Activity:
This section of the report focuses on detail information about designations, exclusivity details, technology, licensing and collaboration, funding and financing, key milestones and various other development activities.
Company Overview:
Company profile includes the detail about type of company, headquarter, global presence, research focus and key financial
Scope
Leber Hereditary Optic Neuropathy (LHON) is the most common inherited mitochondrial disorder and typically affects young males. It typically begins as a unilateral progressive optic neuropathy with sequential involvement of the fellow eye months to years later. LHON is classically associated with mitochondrial base pair mutations G11778A (guanine to adenine at position 11778), T14484C (tyrosine to cytosine), and G3460A. These mutations primarily affect respiratory chain complex I genes, mitochondrial genes ND1, ND4 and ND6 among others. The G11778A is the most common, reported to account for 70% of cases in Northern European populations, and 90% in Asian populations. It is associated with more severe disease, with less chance of significant visual recovery. The T14484C mutation is associated with the best overall prognosis, with some visual recovery in 37-58% of cases. The G3460A mutation usually portends a more intermediate course and has the worst prognosis.
Treatment options are limited, but include the use of antioxidant supplements. Gene therapy trials are currently underway.
LIST OF PRODUCTS:
- Catena
- Lenadogene nolparvovec
- Elamipretide
- BVA203
- IXC-201
- KH176
- Research Programme - Fortify Therapeutics
- Proprietary Libraray-Khondrion
- SPK-LHON
The pipeline section provides descriptive drug profiles for the pipeline products including product description, mechanism of action, route of administration, molecule type, technology involved, chemical information.
Clinical Trial Overview:
This section of the report focuses on the clinical activity of the molecule. It includes both clinical and pre-clinical activity which provides detailed information about the safety, efficacy, tolerability, toxicity of pipeline drugs. A graphical representation of the clinical trial landscape of pipeline therapy which includes information about phase of development, trial design, treatment arms, dosage and frequency, formulation of the drug, primary and secondary completion date, enrolment number, exclusion and inclusion criteria, line of therapy. This section also includes the clinical trial results and analysis based on those results.
Product Development Activity:
This section of the report focuses on detail information about designations, exclusivity details, technology, licensing and collaboration, funding and financing, key milestones and various other development activities.
Company Overview:
Company profile includes the detail about type of company, headquarter, global presence, research focus and key financial
Scope
- The report provides a competitive landscape
- The report also provides clinical trial landscape of the pipeline drugs including status; trial phase; sponsor type and end-point status
- The report provides the list of companies which are the most active in the pipeline
- The report covers pipeline products based on various stages of development ranging from pre-registration till discovery
- The report provides descriptive drug profiles which includes product description; comprising detailed mechanism of action (MoA); route of administration (RoA); Stage of development; clinical trial status; licensing and collaboration details & other developmental activities
- The report features comparative analysis of product profiles based on molecule type; mechanism of action (MoA); route of administration (RoA)
- The report summarizes all the dormant and discontinued pipeline projects
- The report also provides latest news for the past one year
- To identifying prominent players in the treatment landscape
- To determine the drivers; barriers and unmet need in the treatment space
- Gain strategically significant competitor information; analysis; and insights to formulate effective R&D strategies
- Define in-licensing and out-licensing strategies by identifying prospective partners with the most attractive projects to enhance and expand business potential and scope
- To understand the composition of the pipeline in terms of molecule type; molecular target; mechanism of action and route of administration
1. DISEASE OVERVIEW
1.1. Pathophysiology
1.2. Symptoms
1.3. Etiology
1.4. Diagnosis
1.5. Etiology
1.6. Treatment Algorithm
1.7. Treatment Options
2. FEATURED NEWS AND PRESS RELEASES, 2018-2019
3. LIST OF COMPANIES DEVELOPING LEBER HEREDITARY OPTIC NEUROPATHY
4. TREATMENT ALGORITHM
5. CURRENT UNMET NEEDS
6. MARKET TRENDS
7. PIPELINE THERAPEUTICS
8. THERAPEUTICS UNDER DEVELOPMENT BY COMPANIES9. LATE STAGE PRODUCTS (PHASE III)
9.1 Lenadogene nolparvovec
9.1.1. Product Description
9.1.2. Research and Development
9.1.3. Product Development Activities
9.2. XXX
9.2.1. Product Description
9.2.2. Research and Development
9.2.3. Product Development Activities10. Mid Stage Products (Phase II)
10.1 Elamipretide
10.1.1. Product Description
10.1.2. Research and Development
10.1.3. Product Development Activities
10.2. XXX
10.2.1. Product Description
10.2.2. Research and Development
10.2.3. Product Development Activities
11. EARLY STAGE AND PRECLINICAL PRODUCTS (PHASE II)
11.1 XXX
11.1.1. Product Description
11.1.2. Research and Development
11.1.3. Product Development Activities
11.2. BVA203
11.2.1. Product Description
11.2.2. Research and Development
11.2.3. Product Development Activities
12. DRIVERS OF LEBER HEREDITARY OPTIC NEUROPATHY
13. CONSTRAINTS OF LEBER HEREDITARY OPTIC NEUROPATHY
14. THERAPEUTIC ASSESSMENT
14.1. Assessment by Route of Administration
14.2. Assessment by Phase & Route of Administration
14.3. Assessment by Molecule Type
14.4. Assessment by Phase & Molecule type
14.5. Assessment by Target
14.6. Assessment by Mechanism of Action
15. COLLABORATIONS AND ACQUISITIONS DETAILS
16. DORMANT PRODUCTS
17. DISCONTINUED PRODUCTS
18. MARKET TRENDS
19. MARKET OPPORTUNITY ASSESSMENT
20. UNMET NEEDS
21. SWOT ANALYSIS
22. APPENDIX
23. REPORT METHODOLOGY
24. CONSULTING SERVICES
25. DISCLAIMER
1.1. Pathophysiology
1.2. Symptoms
1.3. Etiology
1.4. Diagnosis
1.5. Etiology
1.6. Treatment Algorithm
1.7. Treatment Options
2. FEATURED NEWS AND PRESS RELEASES, 2018-2019
3. LIST OF COMPANIES DEVELOPING LEBER HEREDITARY OPTIC NEUROPATHY
4. TREATMENT ALGORITHM
5. CURRENT UNMET NEEDS
6. MARKET TRENDS
7. PIPELINE THERAPEUTICS
8. THERAPEUTICS UNDER DEVELOPMENT BY COMPANIES9. LATE STAGE PRODUCTS (PHASE III)
9.1 Lenadogene nolparvovec
9.1.1. Product Description
9.1.2. Research and Development
9.1.3. Product Development Activities
9.2. XXX
9.2.1. Product Description
9.2.2. Research and Development
9.2.3. Product Development Activities10. Mid Stage Products (Phase II)
10.1 Elamipretide
10.1.1. Product Description
10.1.2. Research and Development
10.1.3. Product Development Activities
10.2. XXX
10.2.1. Product Description
10.2.2. Research and Development
10.2.3. Product Development Activities
11. EARLY STAGE AND PRECLINICAL PRODUCTS (PHASE II)
11.1 XXX
11.1.1. Product Description
11.1.2. Research and Development
11.1.3. Product Development Activities
11.2. BVA203
11.2.1. Product Description
11.2.2. Research and Development
11.2.3. Product Development Activities
12. DRIVERS OF LEBER HEREDITARY OPTIC NEUROPATHY
13. CONSTRAINTS OF LEBER HEREDITARY OPTIC NEUROPATHY
14. THERAPEUTIC ASSESSMENT
14.1. Assessment by Route of Administration
14.2. Assessment by Phase & Route of Administration
14.3. Assessment by Molecule Type
14.4. Assessment by Phase & Molecule type
14.5. Assessment by Target
14.6. Assessment by Mechanism of Action
15. COLLABORATIONS AND ACQUISITIONS DETAILS
16. DORMANT PRODUCTS
17. DISCONTINUED PRODUCTS
18. MARKET TRENDS
19. MARKET OPPORTUNITY ASSESSMENT
20. UNMET NEEDS
21. SWOT ANALYSIS
22. APPENDIX
23. REPORT METHODOLOGY
24. CONSULTING SERVICES
25. DISCLAIMER
LIST OF TABLES
Table 1: Marketed Leber Hereditary Optic Neuropathy drugs, 2019
Table 2: Sales of Marketed drugs
Table 3: Patent expiration details – marketed drugs
Table 4: Number of Products Under Development for Leber Hereditary Optic Neuropathy
Table 5: Products under Development by Companies
Table 6: Late Stage Products
Table 7: Mid Stage Products
Table 8: Early Stage Products
Table 9: Pre-Clinical and Discovery Products
Table 10: Assessment by Route of Administration
Table 11: Assessment by Stage and Route of Administration
Table 12: Assessment by Molecule Type
Table 13: Assessment by Stage and Molecule Type
Table 14: Assessment by Target
Table 15: Assessment by Mechanism of Action
Table 16: Dormant Products
Table 17: Discontinued Products
Table 1: Marketed Leber Hereditary Optic Neuropathy drugs, 2019
Table 2: Sales of Marketed drugs
Table 3: Patent expiration details – marketed drugs
Table 4: Number of Products Under Development for Leber Hereditary Optic Neuropathy
Table 5: Products under Development by Companies
Table 6: Late Stage Products
Table 7: Mid Stage Products
Table 8: Early Stage Products
Table 9: Pre-Clinical and Discovery Products
Table 10: Assessment by Route of Administration
Table 11: Assessment by Stage and Route of Administration
Table 12: Assessment by Molecule Type
Table 13: Assessment by Stage and Molecule Type
Table 14: Assessment by Target
Table 15: Assessment by Mechanism of Action
Table 16: Dormant Products
Table 17: Discontinued Products
LIST OF FIGURES
Figure 1: Marketed and PipelineLeber Hereditary Optic Neuropathy - Pipeline Review, 2019drugs, 2019
Figure 2: Sales of Marketed drugs
Figure 3: Patent expiration details – marketed drugs
Figure 4: Number of Products Under Development for Leber Hereditary Optic Neuropathy - Pipeline Review, 2019
Figure 5: Products under Development by Companies
Figure 6: Late Stage Products
Figure 7: Mid Stage Products
Figure 8: Early Stage Products
Figure 9: Pre-Clinical and Discovery Products
Figure 10: Assessment by Route of Administration
Figure 11: Assessment by Stage and Route of Administration
Figure 12: Assessment by Molecule Type
Figure 13: Assessment by Stage and Molecule Type
Figure 14: Assessment by Target
Figure 15: Assessment by Mechanism of Action
Figure 16: Dormant Products
Figure17: Discontinued Products
Figure 1: Marketed and PipelineLeber Hereditary Optic Neuropathy - Pipeline Review, 2019drugs, 2019
Figure 2: Sales of Marketed drugs
Figure 3: Patent expiration details – marketed drugs
Figure 4: Number of Products Under Development for Leber Hereditary Optic Neuropathy - Pipeline Review, 2019
Figure 5: Products under Development by Companies
Figure 6: Late Stage Products
Figure 7: Mid Stage Products
Figure 8: Early Stage Products
Figure 9: Pre-Clinical and Discovery Products
Figure 10: Assessment by Route of Administration
Figure 11: Assessment by Stage and Route of Administration
Figure 12: Assessment by Molecule Type
Figure 13: Assessment by Stage and Molecule Type
Figure 14: Assessment by Target
Figure 15: Assessment by Mechanism of Action
Figure 16: Dormant Products
Figure17: Discontinued Products
