Leber hereditary optic neuropathy - Pipeline Insight, 2021

September 2021 | 60 pages | ID: LB0332CC8B5EN
DelveInsight

Abstracts

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DelveInsight’s, “Leber hereditary optic neuropathy - Pipeline Insight, 2021,” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in Leber hereditary optic neuropathy 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

Leber hereditary optic neuropathy Understanding

Leber hereditary optic neuropathy: Overview

Leber hereditary optic neuropathy (LHON) is often characterized by bilateral, painless subacute loss of central vision during young adult life. In most cases, symptoms begin with one eye first, followed a few weeks later by visual failure in the other eye. Extremely rarely there may be neurologic abnormalities, such as peripheral neuropathy, postural tremor, nonspecific myopathy, and movement disorders. LHON is caused by mutations in mitochondrial DNA and it is strictly transmitted by maternal inheritance. The prevalence of visual loss from LHON is approximately 1:50,000 people. Many carriers never suffer significant visual loss; males are about four to five times more likely than females to lose vision and be affected. Individuals with LHON typically display symptoms in their young adult years. If vision is lost, then it usually occurs before 40 years of age. The acute phase of LHON is characterized by a loss of central vision, including blurring and reduced perception of color. Individuals usually lose vision in one eye first and then lose vision in the other eye after two to three months. The atrophic phase is characterized by bilateral optic atrophy, resulting in lifelong blindness.

'Leber hereditary optic neuropathy - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Leber hereditary optic neuropathy pipeline landscape is provided which includes the disease overview and Leber hereditary optic neuropathy treatment guidelines. The assessment part of the report embraces, in depth Leber hereditary optic neuropathy 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, Leber hereditary optic neuropathy 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 Leber hereditary optic neuropathy R&D. The therapies under development are focused on novel approaches to treat/improve Leber hereditary optic neuropathy.

Leber hereditary optic neuropathy Emerging Drugs Chapters

This segment of the Leber hereditary optic neuropathy 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.

Leber hereditary optic neuropathy Emerging Drugs

• GS010: GenSight Biologics

GS010 is an AAV2 gene therapy vector that encodes the human wild-type ND4 protein, which we are developing as a treatment of LHON caused by mutation of the ND4 gene. The ND4 gene is normally located in the mitochondria where ND4 proteins are synthesized. GS010 allows efficient allotopic expression of the mitochondrial gene ND4 in the nucleus thanks to a proprietary Mitochondrial Targeting Sequence that shuttles the messenger RNA from the nucleus directly to the outer membrane of the mitochondria. There, the ND4 proteins are synthesized and incorporated into the mitochondria. Wild-type ND4 proteins then integrate into Complex I of the respiratory chain and rescue the deficiency.

• Elamipretide: Stealth BioTherapeutics Inc.

Elamipretide, is a peptide compound that readily penetrates cell membranes, and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin. In preclinical or clinical studies, the company have observed that elamipretide increases mitochondrial respiration, improves electron transport chain function and ATP production and reduces formation of pathogenic ROS levels. This elamipretide-cardiolipin association has been shown to normalize the structure of the inner mitochondrial membrane, thereby improve ng mitochondrial function. Functional benefit is achieved through improvement of ATP production and interruption and potential reversal of damaging oxidative stress. The company is investigating elamipretide in late stage clinical studies in three primary mitochondrial diseases — rare diseases with cardiomyopathy, Barth syndrome and Leber’s hereditary optic neuropathy – as well as a clinical study in dry age-related macular degeneration.

Further product details are provided in the report.

Leber hereditary optic neuropathy: Therapeutic Assessment

This segment of the report provides insights about the different Leber hereditary optic neuropathy drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in Leber hereditary optic neuropathy

There are approx. 4+ key companies which are developing the therapies for Leber hereditary optic neuropathy. The companies which have their Leber hereditary optic neuropathy drug candidates in the most advanced stage, i.e. preregistration include, GenSight Biologics.

• Phases

DelveInsight’s report covers around 4+ 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
• Route of Administration

Leber hereditary optic neuropathy 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
• intravitreal
• Subretinal
• Topical.
• Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal Antibody
• Peptides
• Polymer
• Small molecule
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Leber hereditary optic neuropathy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Leber hereditary optic neuropathy 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 Leber hereditary optic neuropathy drugs.

Leber hereditary optic neuropathy Report Insights

• Leber hereditary optic neuropathy Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Leber hereditary optic neuropathy Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Leber hereditary optic neuropathy drugs?
• How many Leber hereditary optic neuropathy drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Leber hereditary optic neuropathy?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Leber hereditary optic neuropathy 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 Leber hereditary optic neuropathy and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• GenSight Biologics
• Stealth BioTherapeutics
• Neurophth Therapeutics
• Fortify Therapeutics
• Neuroptika
• Mitotech

Key Products

• Lenadogene nolparvovec
• Elamipretide
• NFS 01
• Research programme: Leber's hereditary optic neuropathy therapeutics
• NRO 1

Contents

Introduction
Executive Summary
Leber hereditary optic neuropathy: 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
Leber hereditary optic neuropathy – DelveInsight’s Analytical Perspective
Late Stage Products (Preregistration)
  Comparative Analysis
Lenadogene nolparvovec: GenSight Biologics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report.
Mid Stage Products (Phase II)
  Comparative Analysis
Elamipretide: Stealth BioTherapeutics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report.

Early Stage Products (Phase I)
  Comparative Analysis
Drug name: Company name
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report.
Preclinical and Discovery Stage Products
  Comparative Analysis
Research programme: Leber's hereditary optic neuropathy therapeutics: Fortify Therapeutics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report.

Inactive Products
  Comparative Analysis
Leber hereditary optic neuropathy Key Companies
Leber hereditary optic neuropathy Key Products
Leber hereditary optic neuropathy- Unmet Needs
Leber hereditary optic neuropathy- Market Drivers and Barriers
Leber hereditary optic neuropathy- Future Perspectives and Conclusion
Leber hereditary optic neuropathy Analyst Views
Leber hereditary optic neuropathy Key Companies
Appendix

List Of Tables

LIST OF TABLES

Table 1 Total Products for Leber hereditary optic neuropathy
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

LIST OF FIGURES

Figure 1 Total Products for Leber hereditary optic neuropathy
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