Prana Provides Huntington Disease Trial Update

in conjunction with the Huntington Study Group Annual Conference

Melbourne – 14 November, 2012; Prana Biotechnology (NASDAQ:PRAN; ASX:PBT)
today reported on its progress in the Reach2HD trial, following a presentation by the Ms
Dianne Angus, Prana’s Chief Operating Officer, at the Huntington Study Group Annual
Conference held in Seattle this past weekend.
The Reach2HD trial is a Phase IIa, 6 month trial in 100 patients with early to mid-stage
Huntington disease that are treated with one of two doses of PBT2 or placebo.
Enrollment commenced in April 2012 following approval from the FDA to conduct the trial
across sites in the USA and Australia. All twenty Reach2HD sites are open and
recruiting. Based on current recruitment activity, it is expected that over 80% of patients
will be in dosing by the end of this month in line with Prana’s recruitment completion
target at the end of the year and reporting of results in 2H13.
During the presentation, Ms Angus noted that PBT2 has a unique therapeutic action
because of its specialized ability to prevent the toxic relationship between disease
proteins and biological metals in the brain. Of special relevance to Huntington disease,
PBT2 has been shown in animal modeling that it can reduce the aggregation of a mutant
form of the Huntingtin protein that is associated with the disease, improve motor
function, preserve neuronal tissue and significantly improve life expectancy*. Moreover
PBT2 has demonstrated a significant ability to improve cognitive Executive Function
(“thinking ability”) in a Phase IIa study in Alzheimer’s disease**. Based on the breadth of
pre-clinical and clinical data to date, Prana’s Reach2HD trial has been designed to
investigate safety and tolerability of PBT2 in Huntington disease patients and to measure
potential cognitive, functional and motor benefits in patients and also explore
mechanistic biomarker readouts.
“There is mounting evidence that compounds that can restore metal homeostasis in the
neuron can stop and even reverse cognitive decline associated with neurodegenerative
diseases. We think Prana’s PBT2 could be such a compound,” said New York based
MLV & Co. Equity Research, Senior Biotech Analyst George Zavoico, Ph.D.
“The first patient has already completed the 6 month treatment period and no patients
have withdrawn from the trial for any safety or other reasons, so we are very pleased
with our progress to date. The Data Safety Monitoring Board will report again next
February” said Mr Kempler, Prana’s Chairman and CEO.
Huntington disease is a complex and severely debilitating genetic, neurodegenerative
disease, for which there is no cure. The disease often affects young adults and, whilst
associated with severe physical movement symptoms, it progressively impacts the mind
and emotions as well. The disease causes incapacitation and death about 15-25 years
after onset. The disease affects over 30,000 people in the US and about 70,000
worldwide. There is only one marketed drug for Huntington disease, with limited utility and notably

there are no drugs either available or in development that have established clinical
evidence for treating the cognitive decline associated with Huntington disease.


Source: Prana Biotechnology

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