The results are clearly encouraging and represent a shot in the arm for Alzheimer's research, which has seen a raft of candidates entering clinical trials but stuttering at the crucial stage.
Data from the study showed PBT2 was well tolerated with little difference in the incidence of adverse events between those receiving PBT2 and those receiving the placebo.
In addition, the pharmacokinetic analysis demonstrated the drug exposure increased predictably in a linear manner, both of which are positive characteristics for a central nervous system (CNS) drug.
Concurrent pre-clinical findings also firmly indicated that PBT2 passes into the brain with more than 20 times greater efficiency than did its predecessor, PBT1.
Scientists from Prana Biotechnology discovered a common interaction of metals and proteins leading to oxidative damage to the brain that is characteristic of patients with Alzheimer's Disease, Parkinson's Disease and other neurodegenerative disorders.
MPAC's (Metal Protein Attenuating Compounds) target abnormal protein-metal interactions preserving normal metal biochemistry.
"Prana MPAC's, of which PBT2 is the lead compound, take advantage of novel insights into the role of metals and oxidation as the cause of Alzheimer's Disease, and may be the first 'plaque busting' drugs," said CEO, Geoffrey Kempler.
The double blind, placebo-controlled single dose escalation study, used 55 healthy, male volunteers between the ages of 18 and 50, evaluating safety, tolerability and pharmacokinetics of PBT2.
"The trials confirm our laboratory studies showing that PBT2 has potential for the treatment of Alzheimer's Disease, which currently affects 4.5 million people in the US and more than 14 million people worldwide," said Ross Murdoch, chief operating officer.
According to the charity Alzheimer's Disease International, this figure is set to increase to 34 million by 2025.
The charity also thinks that finding a treatment that could delay onset by just five years could reduce the number of individuals with Alzheimer's disease by nearly 50 per cent after 50 years.
With these increasing patient numbers and the limitations of available therapies, the Alzheimer's disease (AD) market is an attractive investment with huge unmet need.
Although it occupies a small share of the total CNS market it is currently experiencing massive growth.
Alzheimer's disease market in the seven major markets is worth $4.7 billion (€3.8 billion) and will increase to $6.1 billion by the year 2005 and $ 7.8 billion by the year 2010.