Fragile X Syndrome - Pipeline Insight, 2024

This “Fragile X Syndrome - Pipeline Insight, 2024” report provides comprehensive insights about 10+ companies and 12+ pipeline drugs in Fragile X Syndrome 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

Fragile X Syndrome Understanding

Fragile X Syndrome Overview

Fragile X Syndrome (FXS) is a genetic disorder caused by a mutation on the FMR1 gene located on the X chromosome. This mutation involves an expansion of the CGG triplet repeat within the FMR1 gene, leading to the gene being turned off and not producing the essential protein FMRP (Fragile X Mental Retardation Protein). The absence or deficiency of FMRP, which plays a crucial role in neural development, results in the wide range of cognitive, behavioral, and physical symptoms associated with FXS. The condition is the most common inherited cause of intellectual disability and autism spectrum disorders. Individuals with FXS often exhibit varying degrees of intellectual disability, from mild to severe, with challenges in learning, reasoning, and problem-solving. Behavioral characteristics may include attention deficits, hyperactivity, anxiety, and social avoidance, along with common autistic-like behaviors such as hand-flapping and repetitive speech. Additionally, people with FXS can have distinctive physical features such as a long face, large ears, and a prominent jaw, which become more pronounced with age. Seizures, connective tissue abnormalities, and speech and language delays are also prevalent among those affected.

FXS can be diagnosed by testing a person’s DNA from a blood test. A doctor or genetic counselor can order the test. Testing also can be done to find changes in the FMR1 gene that can lead to fragile X-associated disorders. A diagnosis of FXS can be helpful to the family because it can provide a reason for a child’s intellectual disabilities and behavior problems. This allows the family and other caregivers to learn more about the disorder and manage care so that the child can reach his or her full potential. However, the results of DNA tests can affect other family members and raise many issues. So, anyone who is thinking about FXS testing should consider having genetic counseling before getting tested. There is no cure for FXS; therefore, management primarily involves symptomatic treatment, including speech therapy, behavioral therapy, sensory integration, occupational therapy, and special education. Early interventions for most FXS-associated conditions are crucial for better outcomes. For instance, nutritional counseling to mitigate the risk of obesity in patients with FXS is recommended. Psychiatric counseling can be beneficial if someone exhibits mood disorders, self-injurious behavior, depression, or specific phobias, as these issues tend to occur more frequently in individuals with FXS. Medications used for symptom-based treatment aim to minimize some behavioral and mental health challenges associated with FXS. Stimulants may target hyperactivity, impulsivity, and attention issues. Antidepressants (eg, bupropion, buspirone, or selective serotonin and norepinephrine reuptake inhibitors) may treat anxiety, obsessive-compulsive behaviors, and mood disorders. However, these antidepressants are more often used in adult patients than children.

"Fragile X Syndrome - Pipeline Insight, 2024" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Fragile X Syndrome pipeline landscape is provided which includes the disease overview and Fragile X Syndrome treatment guidelines. The assessment part of the report embraces, in depth Fragile X Syndrome 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, Fragile X Syndrome 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 Fragile X Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Fragile X Syndrome

Fragile X Syndrome Emerging Drugs Chapters

This segment of the Fragile X Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, 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.

Fragile X Syndrome Emerging Drugs

ZYN002: Zynerba Pharmaceuticals, Inc.

Zynerba’s ZYN002 CBD Gel is the first and only synthetic CBD formulated as a patent-protected permeation-enhanced gel and is being studied in refractory epilepsy, Fragile X syndrome and osteoarthritis. It is a clear, permeation-enhanced gel that is designed to provide consistent, controlled drug delivery transdermally with convenient once- or twice-daily dosing. Currently, the drug is in Phase III stage of its clinical trial for the treatment of fragile x syndrome.

NNZ-2566: Neurons Pharmaceuticals Limited

NNZ-2566 is a synthetic analogue of a naturally occurring molecule produced by the brain in response to injury. That molecule, a small part of the insulin-like growth factor-1 (IGF-1) molecule, belongs to a group of molecules called neuropeptides. Currently, the drug is in phase II stage of its clinical trial for the treatment of fragile x syndrome.

Fragile X Syndrome Therapeutic Assessment

This segment of the report provides insights about the different Fragile X Syndrome drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Fragile X Syndrome

There are approx. 10+ key companies which are developing the therapies for Fragile X Syndrome. The companies which have their Fragile X Syndrome drug candidates in the most advanced stage, i.e. Phase III include, Zynerba Pharmaceuticals, Inc.

Phases

This report covers around 12+ 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

Fragile X Syndrome 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

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

Fragile X Syndrome Pipeline Development Activities

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

Fragile X Syndrome Report Insights

• Fragile X Syndrome Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Zynerba Pharmaceuticals, Inc.
• Tetra Discovery Partners
• Nova Mentis Life Science Corp
• Neuren Pharmaceuticals Limited
• Tetra Discovery Partners

Key Products

• ZYN002
• Zatolmilast
• Psilocybin
• NNZ-2566
• BPN14770

This product will be delivered within 2 business days.