Ann: Plans for Phase 3 trial in Rett syndrome, page-61

Study to probe Rett syndrome treatment

By DEREK ROSEAUSTRALIAN ASSOCIATED PRESS11:53AM FEBRUARY 4, 2019FacebookTwitterEmail

A Melbourne company has begun investigations into a possible treatment for a debilitating brain disorder.The three-month clinical trial will involve 180 girls and young women who suffer from Rett syndrome, which affects about 1 in 9000 female live births.The genetic disorder causes problems with language and coordination and those with it require lifelong care.There is no cure or approved medicine to treat the condition.Melbourne-based biopharmaceutical company Neuren Pharmaceuticals has developed a possible treatment called trofinetide.The oral medication aims to mimic a chemical in the brain required for normal functioning.Neuren last year licensed North American rights to the drug to San Diego-based ACADIA Pharmaceuticals, which will conduct the Phase 3 clinical trial in the second half of this year.The patients' caregivers will assess how well the drug has worked.Smaller trials have been promising.Positive results could see ACADIA submit the drug to the US Food and Drug Administration for approval in 2021, the company said.Trofinetide is also being investigated as a treatment for traumatic brain injury and Fragile X syndrome, a genetic condition that causes intellectual disability.