Fragile X Syndrome Pipeline Insight Report | 20+ Companies and 22+ Drugs 1

DelveInsight’s, “Fragile X Syndrome Pipeline Insight 2023,” report provides comprehensive insights about 20+ companies and 22+ pipeline drugs in the Fragile X Syndrome pipeline landscape. It covers the Fragile X Syndrome pipeline drug profiles, including Fragile X Syndrome clinical trials and nonclinical stage products. It also covers the Fragile X Syndrome pipeline 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 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 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 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 Fragile X Syndrome pipeline therapies under development are focused on novel approaches to treat/improve Fragile X Syndrome.


Discover the recent breakthroughs happening Fragile X Syndrome pipeline landscape report @ Fragile X Syndrome Pipeline Outlook


Recent Developmental in the Fragile X Syndrome Treatment Landscape

  • 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.


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.


Find out more facts about the Fragile X Syndrome Companies, Fragile X Syndrome Clinical Trials, and Fragile X Syndrome Drugs @ Fragile X Syndrome Treatment Landscape


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. 


Fragile X Syndrome Therapeutics Assessment

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.


Learn more about the novel and emerging Fragile X Syndrome pipeline therapies and Fragile X Syndrome Key Players @ Fragile X Syndrome Ongoing Clinical Trials Analysis


Scope of the Fragile X Syndrome Pipeline Report

  • Coverage- Global
  • Fragile X Syndrome CompaniesZynerba 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.
  • Fragile X Syndrome Pipeline Therapies- include Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), and others.
  • Fragile X Syndrome Segmentation: Phases, Molecule Type, Mechanism of Action, Product Type


Table of Content

  • Introduction
  • Executive Summary
  • Fragile X Syndrome: Overview
  • Fragile X Syndrome Pipeline Therapeutics
  • Fragile X Syndrome Therapeutic Assessment
  • Fragile X Syndrome– DelveInsight’s Analytical Perspective
  • In-depth Commercial Assessment
  • Fragile X Syndrome Collaboration Deals
  • Late Stage Products (Phase III)
  • Zatolmilast: Tetra Discovery Partners
  • Drug profiles in the detailed report…..
  • Mid Stage Products (Phase II)
  • HLX-0201: Healx
  • Drug profiles in the detailed report…..
  • Preclinical Stage Products
  • Pridopidine: Prilenia Therapeutics
  • Drug profiles in the detailed report…..
  • Inactive Products
  • Fragile X Syndrome Key Companies
  • Fragile X Syndrome Key Products
  • Fragile X Syndrome- Unmet Needs
  • Fragile X Syndrome- Market Drivers and Barriers
  • Fragile X Syndrome- Future Perspectives and Conclusion
  • Fragile X Syndrome Analyst Views
  • Fragile X Syndrome Key Companies
  • 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?


Dive deep into rich insights for Fragile X Syndrome Pipeline Drugs, Visit @ Fragile X Syndrome Key Companies, and Drugs


About Us 

DelveInsight is a Business Consulting and Market research company, providing expert business solutions for the healthcare domain and offering quintessential advisory services in the areas of R&D, Strategy Formulation, Operations, Competitive Intelligence, Competitive Landscaping, and Mergers & Acquisitions.

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