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PBT434 poster presented at the 6th International Multiple System Atrophy Conference

Prana Biotechnology Ltd (ASX PBT: NASDAQ PRAN) has today announced a scientific presentation demonstrating further pre-clinical evidence for PBT434 was presented at the 6th International Multiple System Atrophy Conference in New York.

The poster presents new in-vivo evidence of the efficacy of PBT434 to prevent the loss of neurons and improved function in an animal model of multiple system atrophy (MSA).  MSA is a devastating neurological disease with no established treatments and is one of the potential indications for PBT434. 

PBT434 is the first of a new generation of small molecules from the quinazolinone class of drugs that was specifically designed to block the accumulation and aggregation of alpha-synuclein, an abundant brain protein widely believed to be involved in the pathogenesis of Parkinson’s disease and related disorders.  Prior non-clinical characterization of PBT434, including animal models of Parkinson’s disease, was published last year in Acta Neuropathologica Communications and may be found at

The new experimental data demonstrate that in an animal model of multiple system atrophy, PBT434 prevents α-synuclein accumulation, preserves neurons and decreases the number of glial cell inclusions in the brains of treated animals.  Glial cell inclusions are the key pathological finding in MSA and contain abundant aggregated α-synuclein that is associated with neurodegeneration.  Importantly, these benefits led to improved motor function in treated animals.  Alpha-synuclein is of great interest because aggregated forms of the protein are considered a pathological hallmark of Parkinsonian conditions and are a recognised therapeutic target by basic and clinical neuroscientists. 

“Multiple system atrophy, or MSA, is a devastating disease with limited treatment options.  The data from this animal model of MSA are robust and indicate that PBT434 has excellent potential to treat this progressive neurodegenerative disease.  We expect to begin dosing in healthy volunteers mid-year and are actively planning our first patient study”, said David Stamler, Chief Medical Officer and Senior Vice President of Clinical Development.