PBT434 drug candidate shows potential as “next-generation” disease-modifying treatment in Parkinson’s Disease

Data featured in Plenary Lecture and ‘Blue Ribbon Highlights’ conference presentations at the 17th Movement Disorders Congress of Parkinson’s Disease and Movement Disorders.

Thursday, 20 June, 2013: Melbourne-based Prana Biotechnology (ASX:PBT / NASDAQ:PRAN) today welcomed the release of data which indicates its drug candidate PBT434 shows significant disease-modifying capability in multiple animal models of Parkinson’s Disease (PD) with potential utility in a range of movement disorders.

PD is caused by the death of specialized neurons in the region of the brain called the substantia nigra. This is the only part of the brain where iron, dopamine (a neurotransmitter) and the alpha synuclein protein are all present at high concentrations. In PD, iron binds to dopamine, preventing it from functioning normally, and creating toxic free radicals. Iron also binds to alpha synuclein, causing it to aggregate. The aggregation of this protein is a well-established pathological feature of PD, and a target for new disease-modifying therapies.

PBT434 prevents alpha synuclein from aggregating and also prevents the toxic consequences of iron combining with dopamine,

In a further sign of the potential of PBT434 as an effective treatment, its therapeutic benefits were seen to be dose-dependent. Increasing increments of the drug resulted in increased preservation of neurons and increased improvement in motor function.

“These data are highly positive and support the advancement of PBT434 as a first-in-class drug that could change the course of Parkinson’s Disease and related movement disorders,” said Geoffrey Kempler, Prana’s Chairman and CEO. “This would be a major step forward in therapy as existing treatments are focused on symptomatic relief and offer little in the way of halting neurodegenerative decline once it has begun. The drug is progressing through the development process, with the aim of first clinical trials in 2015”.

These findings are being presented today at the 17th Annual Congress of Parkinson’s Disease and Movement Disorders in Sydney by Professor Colin Masters, Director of The Mental Health Research Institute at the Florey Institute of Neuroscience, in a plenary presentation, and Associate Professor David Finkelstein, Head of the Parkinson’s Disease Laboratory also at the Florey Institute.

“What we have known for some time is that dopamine and iron, together in the brain, form a combustible mix and this drives alpha synuclein aggregation and toxicity,” said Associate Professor Finkelstein.

“What we’ve seen with PBT434 is two beneficial modes of action – it prevents cell death by inhibiting the interaction between dopamine and iron and it also stops this accumulation of alpha synuclein.”

This is the first molecule designed to inhibit the neurotoxic build-up of alpha synuclein in the brain and PBT434 could support the “next generation for PD therapies,” Associate Professor Finkelstein also said.

The full Poster is attached.