Charcot Marie Tooth Disease - Pipeline Insight, 2024

This “Charcot Marie Tooth Disease- Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 10+ 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 disease, a hereditary neuropathy is a group of disorders that affects the motor and/or sensory peripheral nerves resulting in the muscle weakness and atrophy as well as sensory loss. The individual’s nerve cells are not able to send electrical signals properly from the brain to the muscles due to the abnormalities in the nerve axon or abnormalities in the insulation (myelin) around the axon. Charcot Marie tooth disease is known to be a genetic condition caused by gene defect that is responsible for the development of the peripheral nerves. Therefore, individuals having family history of this disease are at higher risk of disease development. Most types of CMT are caused by defects in genes that affect two specific parts of nerves: axons and myelin. Although, the disease is inherited from the family, there are some cases of gene mutation that occur spontaneously. The symptoms may vary from person to person, though the symptoms gradually begin in adolescence affecting the feet and legs at first and later the hands and arms. However, some of the common symptoms are: Weakness of your foot and lower leg muscles, Foot deformities, including a high arch and bent toes (hammer toes), Difficulty lifting your foot while walking (foot drop), Numbness, tingling, or loss of sensation in the lower legs/feet and hands, Contractures (stiffened joints due to abnormal tightening of muscles and associated tissues), Abnormal curvature of the spine (scoliosis), Awkward or higher than normal step (gait) and Loss of muscle around hands and feet. Charcot Marie tooth disease is diagnosis starts with a detailed medical history, family history, and neurological examination. Various diagnosis test such as blood test for genetic problems, nerve conduction studies/electromyography can also be done to measure nerve communication and electrical signals. Lastly, nerve biopsy in which a small piece of peripheral nerve is removed and analyzed under the microscope for abnormalities. There is no cure to the disease. However, the recommended treatments for Charcot Marie tooth disease is symptomatic and supportive which include physical therapy to strengthen/stretch the muscles and occupational therapy to maintain fine finger movements. Although CMT causes loss of sensation, some patients might face pain due to nerve damage or muscle cramping and thus, pain reliving medicines are recommended in such cases. Apart from these non-surgical treatments, the doctor may advice for orthopedic surgery to treat severe foot and joint deformities.

Charcot Marie Tooth Disease- Pipeline Insight, 2024 report 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 SAPXT3003, 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. It is currently in Phase III stage of development for the treatment of Charcot Marie tooth disease type 1A.

Engensis: HELIXMITHEngensis (VM202) is a plasmid DNA designed to simultaneously express two isoforms of hepatocyte growth factor (HGF), HGF 728 and HGF 723. Engensis is being studied in diabetic foot ulcers (DFU), amyotrophic lateral sclerosis (ALS), coronary artery disease (CAD), claudication, and Charcot-Marie-Tooth disease (CMT).

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. 10+ 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 SA.

Phases

This 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

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

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Ophthalmic
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

Molecule Type

Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

Product Type

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

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 SA
• HELIXMITH
• ENCell
• Chong Kun Dang pharmaceutical
• In FlectisBioScience
• Addex Therapeutics
• Augustine therapeutics
• DTx Pharma

Key Products

• PXT3003
• Engensis
• EN001
• CKD-510
• IFB-088

Research programme: GABAB PAM- HDAC6i

• DTx-1252

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