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.
Key takeaways from the Fragile X Syndrome Pipeline Insight Report
Recent Developmental Activities in the Fragile X Syndrome Pipeline
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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.
Fragile X Syndrome Emerging Therapies
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.
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Fragile X Syndrome Pipeline Therapeutics Analysis
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.
Fragile X Syndrome Therapeutics Assessment Analysis
The Fragile X Syndrome Pipeline report proffers an integral view of the Fragile X Syndrome emerging novel therapies segmented by Stage, Product Type, Molecule Type, Mechanism of Action, and Route of Administration.
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Scope of the Fragile X Syndrome Pipeline Report
Table of Content
Key Questions
Current Treatment Scenario and Emerging Therapies:
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