Fragile X Syndrome Clinical Trials Pipeline Offers Promising Novel Treatment Options | DelveInsight
Fragile X Syndrome is a life-long disorder that causes a range of developmental and behavioural challenges including learning disabilities and cognitive impairment. Zynerba Pharmaceuticals Zygel, currently being investigated in the Phase III stage of development, will be the first to market, with a date for its phase-III trial expected in September 2023. Followed by Zynerba, Tetra Therapeutics and Neuren Pharmaceuticals are evaluating the drugs in the mid-stage of development.
New York, USA, March 30, 2022 (GLOBE NEWSWIRE) -- Fragile X Syndrome Clinical Trials Pipeline Offers Promising Novel Treatment Options | DelveInsight
Fragile X Syndrome is a life-long disorder that causes a range of developmental and behavioural challenges including learning disabilities and cognitive impairment. Zynerba Pharmaceuticals Zygel, currently being investigated in the Phase III stage of development, will be the first to market, with a date for its phase-III trial expected in September 2023. Followed by Zynerba, Tetra Therapeutics and Neuren Pharmaceuticals are evaluating the drugs in the mid-stage of development.
DelveInsight’s 'Fragile X Syndrome Pipeline Insight 2022' report provides comprehensive global coverage of available, marketed, and pipeline Fragile X Syndrome therapies in various stages of clinical development, major pharmaceutical companies are working to advance the pipeline space and future growth potential of the Fragile X Syndrome pipeline domain.
Key Takeaways from the Fragile X Syndrome Pipeline Report
- DelveInsight’s Fragile X Syndrome Pipeline analysis depicts a robust space with 15+ active players working to develop 15+ pipeline therapies for Fragile X Syndrome treatment.
- Some of the key 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.
- Key Fragile X Syndrome pipeline therapies in various stages of development include Zygel, Acamprosate, BPN14770, Trofinetide, Sulindac (HLX-0201), and others.
- 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
Request a sample and discover the recent breakthroughs happening Fragile X Syndrome pipeline landscape in @ Fragile X Syndrome Pipeline Outlook
The Fragile X Syndrome pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage Fragile X Syndrome products, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the Fragile X Syndrome pipeline landscape.
Fragile X Syndrome Overview
Fragile X Syndrome is a genetic disorder. Fragile X Syndrome is caused by a mutation in the FMR1 gene located on the X chromosome at Xq27.3. Fragile X Syndrome is characterized by moderate intellectual impairment in afflicted males and mild intellectual disability in affected females. The physical characteristics of affected boys vary and may not be seen until adolescence. The Fragile X Syndrome symptoms might include a huge head, a long face, a prominent forehead and chin, protruding ears, loose joints, and enormous testes. Other Fragile X Syndrome symptoms include flat feet, recurrent ear infections, poor muscular tone, a long narrow face, a high arched palate, dental difficulties, crossed eyes (strabismus), and heart problems such as mitral valve prolapse.
There are various treatment for Fragile X Syndrome that can help afflicted people and their families. Special education, speech, occupational, and sensory integration training, as well as behaviour management programs, are among the Fragile X Syndrome treatment.
Find out more facts about the Fragile X Syndrome @ Fragile X Syndrome
A snapshot of the Fragile X Syndrome Pipeline Drugs mentioned in the report:
| Drugs | Company | Phase | MoA | RoA |
| Zygel | Zynerba Pharmaceuticals | Phase III | Cannabinoid receptor CB1 inverse agonists; Serotonin 1 receptor modulators | Transdermal |
| BPN14770 | Tetra Therapeutics | Phase II/III | Type 4 cyclic nucleotide phosphodiesterase inhibitors | Oral |
| Trofinetide | Neuren Pharmaceuticals | Phase II | Cytokine inhibitors; Glial cell modulators; Neuropeptide receptor modulators | NA |
| AUT00206 | Autifony Therapeutics | Phase I | Shaw potassium channel modulators | Oral |
| Bryostatin 1 | Synaptogenix | Preclinical | Protein kinase C stimulants | NA |
| FXS01 | Lysogene | Discovery | Gene transference | NA |
Learn more about the novel and emerging Fragile X Syndrome pipeline therapies @ Fragile X Syndrome Clinical Trials
Fragile X Syndrome Therapeutics Assessment
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.
Scope of the Fragile X Syndrome Pipeline Report
- Coverage: Global
- Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination
- Therapeutic Assessment By Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III
- Therapeutics Assessment By Route of Administration: Infusion, Intradermal, Intramuscular, Intranasal, Intravenous, Oral, Parenteral, Subcutaneous, Topical
- Therapeutics Assessment By Molecule Type: Gene therapies, Small molecule, Vaccines, Polymers, Peptides, Monoclonal antibodies
- 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.
Dive deep into rich insights for Fragile X Syndrome drug in pipeline, visit @ Fragile X Syndrome Emerging Therapies
Table of Contents
| 1. | Introduction |
| 2. | Executive Summary |
| 3. | Fragile X Syndrome Pipeline: Overview |
| 4. | Analytical Perspective In-depth Commercial Assessment |
| 5. | Fragile X Syndrome Pipeline Therapeutics |
| 6. | Fragile X Syndrome Pipeline: Late Stage Products (Phase III) |
| 6.1 | Zygel: Zynerba Pharmaceuticals |
| 7. | Fragile X Syndrome Pipeline: Late Stage Products (Phase III) |
| 8. | Fragile X Syndrome Pipeline: Mid Stage Products (Phase II) |
| 8.1 | Trofinetide: Neuren Pharmaceuticals |
| 9. | Fragile X Syndrome Pipeline: Early Stage Products (Phase I) |
| 9.1 | AUT00206: Autifony Therapeutics |
| 10. | Therapeutic Assessment |
| 11. | Inactive Products |
| 12. | Company-University Collaborations (Licensing/Partnering) Analysis |
| 13. | Key Companies |
| 14. | Key Products |
| 15. | Unmet Needs |
| 16. | Market Drivers and Barriers |
| 17. | Future Perspectives and Conclusion |
| 18. | Analyst Views |
| 19. | Appendix |
For further information on the Fragile X Syndrome pipeline therapeutics, reach out@ Fragile X Syndrome Therapy
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