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Huntington's Disease

Journal of Huntington’s Disease Publishes Benefits of PBT2

Melbourne – December 13, 2012; Prana Biotechnology (NASDAQ:PRAN; ASX:PBT) today announced that the Journal of Huntington’s Disease has in its December, 2012 edition, published data showing the benefits of PBT2 in Huntington Disease*. The paper describes PBT2’s ability to inhibit the development of the symptoms and pathological features of Huntington Disease in pre-clinical transgenic animal models.

“PBT2 markedly reduced neurodegeneration, significantly increased lifespan and improved motor function and coordination in an aggressive animal model of the disease”, said lead author and Head of Research at Prana, Associate Professor Robert Cherny.

“It is already well established that PBT2 prevents the aggregation of the Abeta protein outside neurons, in Alzheimer’s Disease. It is also established that the mutant Huntingtin (Htt) protein aggregates inside the neuron in Huntington Disease. There is published evidence that the protein aggregation in both diseases is driven by the interaction with metals. Our work has shown that PBT2 can prevent this protein aggregation caused by interaction with metals”, he added.

At a recent New York Academy of Sciences symposium on targeting metals to treat neurodegenerative diseases, Professor Steven M. Hersch, of Massachusetts General Hospital and Harvard Medical School commented that “transition metals, especially iron and copper, have been implicated in the pathogenesis of Huntington Disease. Copper may directly modulate the toxicity of the Htt protein while iron accumulation in response to neurodegeneration likely potentiates the damage to the central nervous system, making both metals potential therapeutic targets. PBT2 is the first clinical candidate that modulates Htt directly.”

PBT2 is being trialed in Huntington Disease patients in 20 sites across the USA and Australia. Results are expected in the second half of 2013.

Key Data Points:

  • PBT2 reduced the toxicity caused by polyQ overexpression in a C.elegans roundworm (transgenic model);
  • In the R6/2 transgenic mouse model of Huntington Disease, PBT2, significantly reduced brain striatal atrophy (40% reduction in lateral ventricular volume);
  • PBT2 increased median lifespan by 26%;
  • PBT2 improved motor function (Rotorod performance);
  • PBT2 reduced the incidence of the ‘clasping behavior’ associated with striatal damage;
  • PBT2 improved maintenance of body weight.

*Cherny R.A. et al, “PBT2 reduces toxicity in a C.elegans model of polyQ aggregation and extends lifespan, reduces striatal atrophy and improves motor performance in the R6/2 mouse model of Huntington’s disease”, J Hunt Dis (2012) 1: 211-219. DOI: 10.3233/JHD-120029

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