DelveInsight’s, “Fragile X Syndrome Pipeline Insight, 2023,” 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.
For Fragile X Syndrome Emerging drugs, the Fragile X Syndrome pipeline analysis report provides a 360° view of the therapeutics landscape by development point, product type, route of administration, molecule type, and MOA. The Fragile X Syndrome pipeline research covers business opportunities, challenges, future partnerships, strong competitors, and growth strategies.
In the Fragile X Syndrome Pipeline Report, a detailed description of the drug is given which includes the mechanism of action of the drug, Fragile X Syndrome clinical trials studies, Fragile X Syndrome 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.
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.
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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.
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.
For further information, refer to the detailed Fragile X Syndrome Drugs Launch, Fragile X Syndrome Developmental Activities, and Fragile X Syndrome News, click here for Fragile X Syndrome Ongoing Clinical Trial Analysis
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 Pipeline 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
Find out more about the Fragile X Syndrome Pipeline Segmentation, Therapeutics Assessment, and Fragile X Syndrome Emerging Drugs @ Fragile X Syndrome Treatment Landscape
Scope of the Fragile X Syndrome Pipeline Report
- Coverage- Global
- Fragile X Syndrome Companies- 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.
- Fragile X Syndrome Pipeline Therapies- Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), and others.
- Fragile X Syndrome Segmentation: Phases, Molecule Type, Mechanism of Action, Product Type
Dive deep into rich insights for drugs for Fragile X Syndrome Pipeline Companies and Therapies, click here @ Fragile X Syndrome Unmet Needs and Analyst Views
Table of Content
- Introduction
- Executive Summary
- Fragile X Syndrome: Overview
- Pipeline Therapeutics
- 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
Got Queries? Find out the related information on Fragile X Syndrome Mergers and acquisitions, Fragile X Syndrome Licensing Activities @ Fragile X Syndrome Emerging Drugs, and Recent Trends
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