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

• DelveInsight’s Fragile X Syndrome Pipeline analysis depicts a robust space with 20+ active players working to develop 22+ pipeline therapies for Fragile X Syndrome treatment.
• The leading Fragile X Syndrome companies developing novel drug candidates to improve the Fragile X Syndrome treatment landscape include Zynerba Pharmaceuticals, Tetra Therapeutics, Autifony Therapeutics, Prilenia Therapeutics, Alcobra Ltd., Seaside Therapeutics, Inc., Marinus Pharmaceuticals, Novartis, Neuren Pharmaceuticals, Roche, Confluence Pharmaceuticals, Tetra Therapeutics, Healx, and others.
• Promising Fragile X Syndrome pipeline therapies in various stages of development include Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), and others.
• The Fragile X Syndrome 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).

 

Recent Developmental Activities in the Fragile X Syndrome Pipeline

• 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.
• In December 2021, Nova Mentis Life Science and Mycrodose Therapeutics announced the successful milestone of merging NOVA’s psilocybin-based drug development program with Mycrodose’s transdermal technology for fragile X syndrome.
• In September 2021, The McQuade Center for Strategic Research and Development, a member of the global Otsuka family of pharmaceutical companies, entered an agreement with Sentinel Oncology (Sentinel), a drug discovery company. The McQuade Center for Strategic Research and Development at Otsuka in the U.S. will support the clinical development of a first-in-class therapeutic with the potential to treat Fragile X syndrome.
• In October 2021, psilocybin drug being developed by Nova Mentis received orphan drug designation from the European Medicines Agency for its propriety for the treatment of fragile X syndrome.

 

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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. 

 

Dive deep into rich insights for Fragile X Syndrome drug in the pipeline, visit @ Fragile X Syndrome Treatment Landscape

 

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.

 

Find out more facts about the Fragile X Syndrome Pipeline Report @ Fragile X Syndrome Clinical Trials

 

Scope of the Fragile X Syndrome Pipeline Report

• Coverage: Global
• Fragile X Syndrome Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
• Fragile X Syndrome Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
• Fragile X Syndrome Therapeutics Assessment By Route of Administration: Infusion, Intradermal, Intramuscular, Intranasal, Intravenous, Oral, Parenteral, Subcutaneous, Topical
• Fragile X Syndrome Therapeutics Assessment By Molecule Type: Gene therapies, Small molecule, Vaccines, Polymers, Peptides, Monoclonal antibodies
• Fragile X Syndrome Therapeutics Assessment By Mechanism of Action: Sigma-1 receptor agonists, Gene transference, Cytokine inhibitors, Glial cell modulators, Neuropeptide receptor modulators, Cannabinoid receptor CB1 inverse agonists, Serotonin 1 receptor modulators
• Key Fragile X Syndrome Companies: Zynerba Pharmaceuticals, Tetra Therapeutics, Autifony Therapeutics, Prilenia Therapeutics, Alcobra Ltd., Seaside Therapeutics, Inc., Marinus Pharmaceuticals, Novartis, Neuren Pharmaceuticals, Roche, Lysogene, Healx,Confluence Pharmaceuticals, and others.
• Key Fragile X Syndrome Pipeline Therapies: HBI-8000, AK104, SHC014748M, F520, Duvelisib, GB226, SP-02L, YY-20394, AZD4205, Brentuximab vedotin, and others.

 

Table of Content

1. Introduction
2. Fragile X Syndrome Executive Summary
3. Fragile X Syndrome: Overview
4. Fragile X Syndrome Pipeline Therapeutics
5. Fragile X Syndrome Pipeline Therapeutic Assessment
6. Fragile X Syndrome– DelveInsight’s Analytical Perspective
7. In-depth Commercial Assessment
8. Fragile X Syndrome Collaboration Deals
9. Late Stage Products (Phase III)
10. Zatolmilast: Tetra Discovery Partners
11. Drug profiles in the detailed report…..
12. Mid Stage Products (Phase II)
13. HLX-0201: Healx
14. Drug profiles in the detailed report…..
15. Preclinical Stage Products
16. Pridopidine: Prilenia Therapeutics
17. Drug profiles in the detailed report…..
18. Inactive Products
19. Fragile X Syndrome Key Companies
20. Fragile X Syndrome Key Products
21. Fragile X Syndrome- Unmet Needs
22. Fragile X Syndrome- Market Drivers and Barriers
23. Fragile X Syndrome- Future Perspectives and Conclusion
24. Fragile X Syndrome Analyst Views
25. Fragile X Syndrome Key Companies
26. Appendix

 

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?

 

For further information on the Fragile X Syndrome pipeline therapeutics, reach out @ Fragile X Syndrome Emerging Therapies

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