Fragile X Syndrome - Pipeline Insight, 2022

DelveInsight's "Fragile X Syndrome - Pipeline Insight, 2022," report provides comprehensive insights about 20+ companies and 22+ 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. FXS is caused by changes in a gene that scientists called the fragile X mental retardation 1 (FMR1) gene when it was first discovered. The FMR1 gene usually makes a protein called fragile X mental retardation protein (FMRP). FMRP is needed for normal brain development. People who have FXS do not make this protein. People who have other fragile X-associated disorders have changes in their FMR1 gene but usually make some of the protein. FXS affects both males and females. However, females often have milder symptoms than males. The exact number of people who have FXS is unknown, but a review of research studies estimated that about 1 in 7,000 males about 1 in 11,000 females have been diagnosed with FXS.

Signs that a child might have FXS include:

• Developmental delays (not sitting, walking, or talking at the same time as other children the same age);

• Learning disabilities (trouble learning new skills); and

• Social and behavior problems (such as not making eye contact, anxiety, trouble paying attention, hand flapping, acting and speaking without thinking, and being very active).

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. There is no cure for FXS. However, treatment services can help people learn important skills. Services can include therapy to learn to talk, walk, and interact with others. In addition, medicine can be used to help control some issues, such as behavior problems.

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.

• Zynerba Pharmaceuticals announced that the US Food and Drug Association has granted orphan-drug designation to ZYN002 cannabidiol (CBD) gel, for the treatment of Fragile X syndrome (FXS).

• In March 2022, The European Commission granted orphan drug designation to Zygel, Zynerba Pharmaceuticals' cannabidiol (CBD) transdermal gel candidate to treat for behavioural symptoms in people with fragile X syndrome.

• In February 2022, Ovid entered an option agreement with Healx for an exclusive license to gaboxadol (OV101). Healx secured from Ovid an option to exclusively license rights to develop and commercialize gaboxadol (OV101). Healx has stated that it will investigate the compound as part of a potential combination therapy for Fragile X syndrome, as well as treatment for other indications.

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

Zatolmilast: Tetra Discovery Partners

Tetra's investigational new drug, named BPN14770, can selectively target and inhibit, or suppress, only the PDE4D enzyme. PDE4D plays an important role in cognition. A recent study of over a million healthy human subjects showed connections between genetic variants in PDE4D and years of educational attainment and performance on cognitive tests. Tetra Therapeutics's BPN14770 targets only PDE4D, and only at specific times of cellular activity. BPN14770 is designed to modulate, rather than completely inhibit, PDE4D. The desired result from clinical trial testing is two-fold: that Tetra's BPN14770 will improve cognitive function by prolonging cAMP activity, while safety and tolerability are improved because the enzyme is not completely inhibited. Currently it is being investigated in Phase III stage of development.

Zygel: Zynerba Pharmaceuticals

Zygel is the first and only pharmaceutically-produced CBD, a non-euphoric cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. Zygel is being developed for patients suffering from FXS, ASD in pediatric patients, 22q, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies (DEE). Zynerba Pharmaceuticals is conducting phase III clinical trials for the treatment of Fragile X Syndrome.

Pridopidine: Prilenia Therapeutics

Pridopidine is an orally bioavailable small molecule investigational drug exhibiting potential neuroprotective effect in multiple neurodegenerative diseases with a favorable safety profile. It is the most selective high affinity Sigma-1-receptor (S1R) agonist. The S1R regulates key cellular processes relevant to neurodegenerative diseases, such as calcium homeostasis, cytoskeleton dynamics, restoring mitochondrial health and neurotrophic factor release. S1R is implicated in cellular differentiation, neuroplasticity, neuroprotection, and cognitive functioning of the brain. It is in preclinical stage of development.

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

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. 20+ 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 and others

Phases

DelveInsight's report covers around 22+ products under different phases of clinical development like:

• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage products (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:

• Infusion
• Intradermal
• Intramuscular
• Intranasal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as:

• Gene therapies
• Small molecule
• Vaccines
• Polymers
• Peptides
• Monoclonal antibodies
• 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
• Tetra Therapeutics
• Autifony Therapeutics
• Prilenia Therapeutics
• Alcobra Ltd.
• Seaside Therapeutics, Inc.
• Marinus Pharmaceuticals
• Novartis Pharmaceuticals
• Neuren Pharmaceuticals
• Hoffmann-La Roche
• Healx
• Nova Mentis
• Lysogene
• Actinogen
• Prilenia Therapeutics
• Confluence Pharmaceuticals
• Sentinel Oncology

Key Products

• Zygel
• BPN14770
• AUT00206
• Pridopidine
• MG01CI
• Arbaclofen
• Ganaxolone
• AFQ056
• NNZ-2566
• RO4917523