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US DoD backs potential treatment for Fragile X-linked neurodegenerative disorder
2023-08-01
临床2期上市批准临床结果
Robert Barrie
Estimates suggest that 30%-40% of male carriers, and 8%-16% of premutation carriers will develop FXTAS symptoms. Image credit: Shutterstock/Billion Photos.
The US Department of Defense (DoD) has awarded PureTech Health $11.4m to help target Fragile X-associated tremor/ataxia syndrome (FXTAS), a late-onset neurological condition that is difficult to diagnose and currently has no effective treatments.
Based in Massachusetts, US, Puretech Health will use the funds to advance LYT-300 in a Phase II trial.
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LYT-300 is an oral prodrug oNeuromyelitis Optica Spectrum DisorderreguNeuromyelitis Opticasitive allosteric modulation of gamma-aminobutyric-acid type A (GABAA) receptors. It is already approved by the US Food and Drug Administration (FDA) as an infusion treatment for postpartum depression.
PureTech Health said administration limitations have previously restricted the wider use of the neurosteroid. Developed using the company’s Glyph platform that harnesses natural lipid absorption and transport processes, LYT-300 can be orally administered.
FXTAS occurs in premutation Radiprodilf FrNeurodegenerative Diseasess suggest that 30%-40% of male carriers, and 8%-16% of premutation carriers will develop FXTAS symptoms. Symptoms include intention tremors, ataxia, neuropathy, and cognitive symptoms.
The company also recently began a proof-of-concept study for the drug in the treatment of anxiety. Due to similar presentations, patients with FXTAS are often misdiagnosed with Parkinson’s disease.
LYT-300pany has conducted an eallopregnanolone-label trial in six males with FXTAS, which demonstrated pharmacologic benefgamma-aminobutyric-acid type A (GABAA) receptorslanning to now evaluate the safety,Food and Drug Administration (FDA)300 in patients with FXTAS in postpartum depressiontrial.
PureTech Healthial will be conducted in collaboration with the University of California, Davis.LYT-300
UC Davis MIND Institute medical directorFragile X Syndromevestigator for the LYT-300 trial Dr Randi Hagerman said: “FXTAS is a devastating, late-onset neurodegenerative condition characterisintention tremorsecataxiatrneuropathyhe hands and balance problems. Currently, there are no primary treatments for FXTAS, though IV-administrated allopregnanolone has demonstrated therapeutic potential.”
“I am excited to be working with PureTech to evaluate their oral prodrug of allopregnanoloanxiety-300), and I am optimistic that this award will help accelerate the development Parkinson’s diseaseirst therapy for FXTAS.”
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