08 Apr 2013, BioSpectrum Bureau , BioSpectrum
Singapore: Prana Biotechnology reported on the progress of its lead development asset PBT2, which is in phase II trials for Huntington disease and Alzheimer's disease. Across the two trials, approximately 37 percent have been dosed to six months or longer, which is twice the duration of the previous phase II Alzheimer's trial. PBT2, and has been well tolerated and are demonstrating pleasing retention rates and compliance. Each Independent Data Safety Monitoring Board for the two trials met at the end of March and recommended that each of the trials continue as planned without any protocol changes.
Prana's PBT2 represents a novel and differentiated therapeutic action in the treatment of neurodegenerative diseases based on its specialized ability to prevent the toxic relationship between disease proteins and biological metals in the brain, which otherwise can lead to protein amyloid formation. Moreover, the redistribution of such metals by PBT2 promotes neurotransmission and neuronal function.
The Alzheimer's disease trial, which is named Imagine, is a randomized, double-blind, placebo controlled trial that enrolled 41 patients with prodromal or mild Alzheimer's disease, in five sites in Melbourne, Australia. Brain imaging is being used to measure PBT2's effect on amyloid deposits in the brain (using PiB-PET scanning) and effects on increasing brain activity (FDG PET). Cognition effects are being measured by the Neuropsychological Test Battery (NTB). The trial received funding from the Alzheimer's Drug Discovery Foundation (ADDF).
Dr Howard Fillit, ADDF's executive director commented, "PBT2 stands out as one of the few orally available agents with clinical trial evidence of cognitive benefit for Alzheimer's patients. Success in this trial will demonstrate target engagement by PBT2 in the brain of people with Alzheimer's disease, and accelerate the clinical development of PBT2 to patients."
The Huntington Disease trial, which is named Reach2HD, is a six month trial in 109 patients with early to mid-stage Huntington disease. The trial is being conducted across sites in the US and Australia. Only one drug is marketed for Huntington disease and that is only for the relief of the severe motor or chorea symptoms. There are no approved treatments for the significant cognitive and behavioural components of the disease, which typically manifest before motor problems.