The 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.
Report Highlights
The 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).
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.
Fragile X Syndrome Emerging Drugs Chapters
This segment of the Fragile X Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
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: Therapeutic Assessment
This segment of the report provides insights about the different Fragile X Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
Major Players in Fragile X Syndrome
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
Phases
The report covers around 22+ products under different phases of clinical development like
- Late-stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage products (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Products have been categorized under various ROAs such as
- Infusion
- Intradermal
- Intramuscular
- Intranasal
- Intravenous
- Oral
- Parenteral
- Subcutaneous
- Topical
- Molecule Type
Products have been categorized under various Molecule types such as
- Gene therapies
- Small molecule
- Vaccines
- Polymers
- Peptides
- Monoclonal antibodies
- Product Type
Fragile X Syndrome Report Insights
- Fragile X Syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Fragile X Syndrome Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Players
- Zynerba Pharmaceuticals
- Tetra Therapeutics
- Autifony Therapeutics
- Prilenia Therapeutics
- Alcobra Ltd.
- Seaside Therapeutics, Inc.
- Marinus Pharmaceuticals
- Novartis Pharmaceuticals
- Neuren Pharmaceuticals
- Hoffmann-La Roche
- Healx
- Nova Mentis
- Lysogene
- Actinogen
- Prilenia Therapeutics
- Confluence Pharmaceuticals
- Sentinel Oncology
Key Products
- Zygel
- BPN14770
- AUT00206
- Pridopidine
- MG01CI
- Arbaclofen
- Ganaxolone
- AFQ056
- NNZ-2566
- RO4917523
For more information about this clinical trials report visit https://www.researchandmarkets.com/r/ooxrj0
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