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Charcot-Marie-Tooth Disease - Pipeline Insight, 2021

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    Clinical Trials

  • 60 Pages
  • September 2021
  • Region: Global
  • DelveInsight
  • ID: 4989323
This “Charcot-Marie-Tooth disease - Pipeline Insight, 2021,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Charcot-Marie-Tooth 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

Charcot-Marie-Tooth disease Understanding

Charcot-Marie-Tooth disease: Overview


Charcot-Marie-Tooth (CMT) disease is a group of disorders in which the motor and/or sensory peripheral nerves are affected, resulting in muscle weakness and atrophy as well as sensory loss. Symptoms occur first in the distal legs and later in the hands. The nerve cells in individuals with this disorder are not able to send electrical signals properly because of abnormalities in the nerve axon or abnormalities in the insulation (myelin) around the axon. In CMT specific gene mutations are responsible for the abnormal function of the peripheral nerves. In many forms of CMT these genes are known and in others, while the condition is known to be inherited, the specific gene has not yet been identified.

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

Charcot-Marie-Tooth disease Emerging Drugs Chapters


This segment of the Charcot-Marie-Tooth 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.

Charcot-Marie-Tooth disease Emerging Drugs


PXT3003: Pharnext


PXT3003 is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution given twice a day. The three individual components of PXT3003 were selected to downregulate the overexpression of PMP22 protein, leading to improvement of neuronal signaling in dysfunctional peripheral nerves that are an essential part of the pathophysiology of this disease. PXT3003 could also have a positive effect on other cellular types of the motor unit such as the axon (direct protection), neuromuscular junctions or muscle cells. Currently, it is in Phase III stage of clinical trial evaluation.

CKD 510: Sarepta Therapeutics


CKD 510 is an HDAC6 inhibitor being developed by Chong Kun Dang pharmaceutical to treat Charcot-Marie-Tooth disease. Currently, it is in Phase I stage of clinical trial evaluation.

Charcot-Marie-Tooth disease: Therapeutic Assessment


This segment of the report provides insights about the different Charcot-Marie-Tooth disease drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Charcot-Marie-Tooth disease


There are approx. 8+ key companies which are developing the therapies for Charcot-Marie-Tooth disease. The companies which have their Charcot-Marie-Tooth disease drug candidates in the most advanced stage, i.e. phase III include, Pharnext.

Phases


This report covers around 8+ 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

Charcot-Marie-Tooth 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
  • 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

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

Charcot-Marie-Tooth disease: Pipeline Development Activities


The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Charcot-Marie-Tooth 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 Charcot-Marie-Tooth disease drugs.

Charcot-Marie-Tooth disease Report Insights

  • Charcot-Marie-Tooth disease Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Charcot-Marie-Tooth disease Report Assessment

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

Key Questions Answered


Current Treatment Scenario and Emerging Therapies:
  • How many companies are developing Charcot-Marie-Tooth disease drugs?
  • How many Charcot-Marie-Tooth disease drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Charcot-Marie-Tooth disease?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Charcot-Marie-Tooth 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 Charcot-Marie-Tooth disease and their status?
  • What are the key designations that have been granted to the emerging drugs?

Key Players

  • Pharnext
  • Chong Kun Dang Pharmaceutical
  • Neurogene Inc.
  • Regenacy Pharmaceuticals
  • Addex Therapeutics
  • Recursion Pharmaceuticals
  • Grunenthal
  • InFlectis BioScience
  • Cellatoz Therapeutics, Inc.
  • DTx Pharma

Key Products

  • PXT-3003
  • CKD 510
  • CMT4J gene therapy
  • Ricolinostat
  • IFB-088
  • CLZ-2002


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Table of Contents

IntroductionExecutive Summary
Charcot-Marie-Tooth 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

Charcot-Marie-Tooth disease - Analytical Perspective
Late Stage Products (Phase III)
  • Comparative Analysis

PXT3003: Pharnext
  • Product Description
  • Research and Development
  • Product Development Activities

Early Stage Products (Phase I)
  • Comparative Analysis

CKD 510: Chong Kun Dang Pharmaceutical
  • Product Description
  • Research and Development
  • Product Development Activities

Preclinical and Discovery Stage Products
  • Comparative Analysis

CMT4J gene therapy: Neurogene Inc.
  • Product Description
  • Research and Development
  • Product Development Activities

Inactive Products
  • Comparative Analysis

Charcot-Marie-Tooth disease Key CompaniesCharcot-Marie-Tooth disease Key ProductsCharcot-Marie-Tooth disease- Unmet NeedsCharcot-Marie-Tooth disease- Market Drivers and BarriersCharcot-Marie-Tooth disease- Future Perspectives and ConclusionCharcot-Marie-Tooth disease Analyst ViewsCharcot-Marie-Tooth disease Key CompaniesAppendix
List of Tables
Table 1 Total Products for Charcot-Marie-Tooth 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 Charcot-Marie-Tooth 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

Companies Mentioned

A selection of companies mentioned in this report includes:

  • Pharnext
  • Chong Kun Dang Pharmaceutical
  • Neurogene Inc.
  • Regenacy Pharmaceuticals
  • Addex Therapeutics
  • Recursion Pharmaceuticals
  • Grunenthal
  • InFlectis BioScience
  • Cellatoz Therapeutics, Inc.
  • DTx Pharma