Charcot Marie Tooth Disease- Pipeline Insight, 2025

This “Charcot Marie Tooth Disease - Pipeline Insight, 2025” report provides comprehensive insights about 10+ companies and 15+ 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.

Charcot Marie Tooth Disease: Understanding

Charcot Marie Tooth Disease: Overview

Charcot-Marie-Tooth disease (CMT) is a group of inherited neurological disorders that affect the peripheral nerves, which are responsible for movement and sensation in the limbs. Named after the three physicians who first described it in 1886, CMT is also known as hereditary motor and sensory neuropathy (HMSN). It is one of the most common inherited neurological disorders, affecting about 1 in 2,500 people. CMT is characterized by progressive muscle weakness and atrophy, primarily in the legs and feet, and, in later stages, in the hands and arms. Although the severity and progression of symptoms can vary widely among individuals, CMT typically manifests in adolescence or early adulthood. The signs and symptoms of CMT often begin with weakness in the foot and lower leg muscles, leading to foot drop, a high-stepping gait, and frequent tripping or falls. Over time, muscle weakness and atrophy can spread to the hands and arms, making fine motor skills difficult. Other common symptoms include loss of sensation in the affected limbs, muscle cramps, and foot deformities such as high arches and hammertoes. Some individuals may also experience scoliosis, or curvature of the spine, and hip dysplasia. The severity of symptoms can vary significantly, with some individuals experiencing only mild impairment while others may become severely disabled.

CMT is caused by mutations in genes that affect the structure and function of the peripheral nerves. These mutations lead to either the demyelination of nerves (CMT type 1), where the protective myelin sheath is damaged, or axonal degeneration (CMT type 2), where the nerve axon itself is affected. CMT can be inherited in an autosomal dominant, autosomal recessive, or X-linked manner, depending on the specific gene involved. More than 80 different genes have been associated with CMT, reflecting its genetic complexity.

The pathophysiology of CMT involves the progressive degeneration of peripheral nerves, which disrupts the transmission of nerve signals between the brain and muscles. In demyelinating forms of CMT, the myelin sheath deteriorates, slowing down nerve conduction and leading to muscle weakness and sensory loss. In axonal forms of CMT, the nerve axons degenerate, resulting in the loss of nerve fibers necessary for muscle control and sensation. This ongoing nerve damage leads to the characteristic muscle wasting and sensory deficits seen in CMT. Diagnosis of CMT is based on clinical evaluation, family history, and a range of diagnostic tests. These tests may include nerve conduction studies (NCS) to assess the speed and strength of electrical signals in the peripheral nerves, electromyography (EMG) to evaluate muscle response to nerve stimulation, and genetic testing to identify specific gene mutations. In some cases, a nerve biopsy may be performed to examine the nerve structure under a microscope. Currently, there is no cure for CMT, but treatment focuses on managing symptoms and improving quality of life. Physical therapy and occupational therapy can help maintain muscle strength, flexibility, and function, while orthopedic devices like braces and custom-made shoes can assist with mobility and prevent deformities. Pain management may involve medications, and in some cases, surgery may be required to correct severe foot and joint deformities. Genetic counseling is recommended for individuals with CMT and their families to understand the inheritance patterns and the risks of passing the condition to future generations. Advances in genetic research and therapies hold promise for more targeted treatments in the future.

'Charcot Marie Tooth Disease- Pipeline Insight, 2025' 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 III, II, II/III 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 SA

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. It is currently in Phase III stage of development for the treatment of Charcot Marie tooth disease type 1A.

IFB-088: InFlectis BioScience

IFB-088 is an orally available small molecule capable of crossing the blood-brain barrier, a cell layer that protects the brain from disease-causing agents and toxins that may be present in the blood. IFB-088 also serves as an inhibitor of certain glutamate receptors. These receptors are involved in a process called excitotoxicity, in which neurons are over-stimulated and fire much more than needed, leading to nerve damage. IFB 088 has the potential to treat several demyelinating subtypes including CMT 1A, CMT 1B and CMT 1E, which affects 150,000 people in the U.S. and Europe. The Company is now planning a Phase II clinical trial in the United States and Europe. The drug is currently in Phase I stage of development for the treatment of Charcot Marie tooth disease.

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

The report covers around 15+ 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

• Intravenous
• Subcutaneous
• Oral
• Intramuscular

Molecule Type

Products have been categorized under various Molecule types such as

• Monoclonal antibody
• Small molecule
• Peptide

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
• InFlectis BioScience
• ENCell
• HELIXMITH
• Chong Kun Dang pharmaceutical
• Addex Therapeutics
• Augustine therapeutics
• Novartis

Key Products

• PXT3003
• IFB-088
• EN001
• Engensis
• CKD-510
• Research programme: GABAB PAM
• HDAC6i
• DTx-1252

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