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X-linked adrenoleukodystrophy - Pipeline Insight, 2021

July 2021 | 60 pages | ID: XFADE37D6F56EN
DelveInsight

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DelveInsight’s, “X-linked adrenoleukodystrophy - Pipeline Insight, 2021,” report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in X-linked adrenoleukodystrophy 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
X-linked adrenoleukodystrophy Understanding

X-linked adrenoleukodystrophy: Overview

X-linked adrenoleukodystrophy (ALD) is a rare genetic disorder that affects the white matter of the nervous system and the adrenal cortex. White matter is made up of nerve fibers called axons that relay nerve impulses from one cell to another. These nerve fibers are covered by myelin, an insulating layer or sheath that protects the nerve fibers. Myelin is made up of proteins and fats and gives white matter its white color. Without myelin, the signals between nerve cells cannot be transmitted properly, resulting in neurological symptoms. The adrenal cortex is the outer layer of cells of the adrenal glands. The adrenal glands sit atop the kidneys and produce hormones that are vital to proper health and development including cortisol and the sex hormones. Many of those affected experience serious neurological problems either during childhood or during adulthood with rather different types of disabilities.

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

This segment of the X-linked adrenoleukodystrophy 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.

X-linked adrenoleukodystrophy Emerging Drugs
  • Leriglitazone: Minoryx Therapeutics
Leriglitazone (MIN-102) is a novel, orally bioavailable and selective PPAR gamma agonist with a potential best-in-class profile indicated for CNS diseases. It is one of the several metabolites of pioglitazone and has a demonstrated sufficient brain penetration and favorable safety profile in humans, allowing PPAR gamma engagement in the CNS above the level that can be safely achieved with pioglitazone and other glitazones. It showed robust preclinical proof-of-concept in animal models of multiple diseases by modulating pathways leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. Currently, it is in Phase II/III stage of development to treat X-linked adrenoleukodystrophy.
  • VK0214: Viking Therapeutics
VK0214, a novel, orally available, small molecule selective thyroid hormone receptor beta agonist being developed for the treatment of X-linked adrenoleukodystrophy (X-ALD). Currently, it is in Phase I stage of development.

Further product details are provided in the report……..

X-linked adrenoleukodystrophy: Therapeutic Assessment

This segment of the report provides insights about the different X-linked adrenoleukodystrophy drugs segregated based on following parameters that define the scope of the report, such as:
  • Major Players in X-linked adrenoleukodystrophy
There are approx. 3+ key companies which are developing the therapies for X-linked adrenoleukodystrophy. The companies which have their X-linked adrenoleukodystrophy drug candidates in the most advanced stage, i.e. phase II/III include, Minoryx Therapeutics.
  • Phases
DelveInsight’s report covers around 3+ 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
X-linked adrenoleukodystrophy 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
  • intravenous
  • Subcutaneous
  • 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.

X-linked adrenoleukodystrophy: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses X-linked adrenoleukodystrophy 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 X-linked adrenoleukodystrophy drugs.

X-linked adrenoleukodystrophy Report Insights
  • X-linked adrenoleukodystrophy Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs
X-linked adrenoleukodystrophy 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 X-linked adrenoleukodystrophy drugs?
  • How many X-linked adrenoleukodystrophy drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of X-linked adrenoleukodystrophy?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the X-linked adrenoleukodystrophy 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 X-linked adrenoleukodystrophy and their status?
  • What are the key designations that have been granted to the emerging drugs?
Key Players
  • Minoryx Therapeutics
  • Viking Therapeutics
  • Poxel
  • Orpheris
  • Applied Genetic Technologies Corporation
  • NEURALGENE
Key Products
  • Leriglitazone
  • VK0214
  • OP 101
  • Research programme: Adrenoleukodystrophy gene therapy
  • PRCN-323
Introduction
Executive Summary
X-linked adrenoleukodystrophy: 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
Late Stage Products (Phase II/III)
  Comparative Analysis
Leriglitazone: Minoryx Therapeutics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report…..
Early Stage Products (Phase I)
  Comparative Analysis
VK0214: Viking Therapeutics
  Product Description
  Research and Development
  Product Development Activities
Drug profiles in the detailed report…..
Inactive Products
  Comparative Analysis
X-linked adrenoleukodystrophy Key Companies
X-linked adrenoleukodystrophy Key Products
X-linked adrenoleukodystrophy- Unmet Needs
X-linked adrenoleukodystrophy- Market Drivers and Barriers
X-linked adrenoleukodystrophy- Future Perspectives and Conclusion
X-linked adrenoleukodystrophy Analyst Views
X-linked adrenoleukodystrophy Key Companies
Appendix


LIST OF TABLES

Table 1 Total Products for X-linked adrenoleukodystrophy
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 X-linked adrenoleukodystrophy
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


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