Disease Modifying Treatments
Alzheimer’s Disease
Alzheimer’s disease is characterised by the presence of insoluble deposits called senile plaques in regions of the brain that are associated with memory. These plaques are composed of a protein called beta-amyloid, which in high levels is extremely toxic to brain cells. There is now overwhelming evidence that over production and accumulation of beta-amyloid in the brain is central to the pathogenesis of Alzheimer’s disease and leads to the destruction of brain cells resulting in a deterioration of mental function. This has been called the “Amyloid Cascade Hypothesis”
(Source: http://www.ahaf.org/alzdis/about/AmyloidPlaques.htm)
Alzhyme has used its proprietary intellectual property to generate a selection of potentially therapeutic peptides that specifically neutralise the damaging potential of beta-amyloid. The Company has used innovative screening from focused compound libraries and medicinal chemistry strategies to identify novel peptides that target beta-amyloid. A screening method (involving phage display technology) has identified a family of compounds that specifically bind human, but not rat, beta-amyloid.
Two peptides in particular, ANA-1 and ANA-5, were found to prevent beta–amyloid-induced neurotoxicity. In a rat model of beta-amyloid-induced neurotoxicity treatment with ANA-1 was found to enhance the clearance of beta-amyloid from the brain. In a subsequent proof-of-concept study, treatment with ANA-5 significantly reduced brain beta-amyloid plaque levels by 28% in the PS/APP transgenic mouse model of Alzheimer’s disease.
ANA-5 represents a new and promising therapeutic class of drug in development as a treatment for Alzheimer’s disease. The Company is currently developing ANA-5 for the treatment Alzheimer’s disease. Alzhyme has entered into a research collaboration with the Commonwealth Scientific and Industrial Research Organisation (CSIRO), one of the world’s largest scientific research organisations to undertake a lead optimization program to develop an oral available analogue of ANA-5.
Other Indications
The compounds being developed by Alzhyme could also potentially target other amyloid related diseases, such as the orphan disease Inclusion Body Myositis, an age-related muscle disease caused by amyloid toxicity in muscle cells.
