Prana Biotech’s new drug PBT2 to be tested in Alzheimer’s patients

Prana Biotechnology is initiating a clinical study to test the efficacy of its investigational compound PBT2 in Alzheimer’s disease patients.

Prana is finalising plans to commence a definitive phase IIb trial of its lead Alzheimer’s disease drug, PBT2, before the end of this year.

The phase IIb trial will involve 525 patients with mild to moderate Alzheimer’s disease. Treatment will be over a period of 12 months, with the key performance measure being cognition (including ADAS Cog and Executive Function tests from the NTB).

ADAS–Cog (Alzheimer’s Disease Assessment Scale – cognitive subset) and NTB (Neuropsychological Test Battery) are tests given to Alzheimer’s disease patients to measure changes in memory and Executive Function.

The trial, a double blind placebo controlled study, will test the efficacy of 2 doses of PBT2 (250mg and 100mg).

Over recent years most drug companies have made late stage drug development decisions based on secondary biomarkers and imaging. Many have seen amyloid signals, but not necessarily robust cognition outcomes. PBT2 has shown both positive cognitive and biomarker changes, which is why we are so optimistic in the ability of PBT2 to really help sufferers of Alzheimer’s disease, Prana officials said.

Prana expects that the benefit will be even stronger and more pronounced over a 12 month trial.

PBT2 works in the brains of patients with Alzheimer’s disease. PBT2 is uniquely different to other approaches for treating the disease.

The landscape has changed. Patients need and deserve a therapeutic solution based on strong science as well as clinical trials that translate the science into real benefits for patients.

Discussions with potential sources of finance for the upcoming trial, both with investors and pharmaceutical partners, are progressing well and we will keep our shareholders updated.

Prana Biotechnology was established to commercialise research into Alzheimer’s Disease and other major age-related neurodegenerative disorders.

The Company was incorporated in 1997 and listed on the Australian Stock Exchange in March 2000 and listed on NASDAQ in September 2002.

Researchers at prominent international institutions including The University of Melbourne, The Mental Health Research Institute (Melbourne) and Massachusetts General Hospital, a teaching hospital of Harvard Medical School, contributed to the discovery of Prana’s technology.

Prana has developed a unique platform technology in association with internationally recognized scientists and academic institutions.

The development of Prana’s library of Metal-Protein Attenuating Compounds (MPACs) commenced with the observation that Prana’s first proof-of-concept MPAC – clioquinol (PBT1), abolished Alzheimer’s-like changes in the brains of genetically engineered mice.

Subsequent work demonstrated a significant reduction in cognitive decline in a pilot phase IIa study in Alzheimer’s patients receiving PBT1.

Due to an unrectifiable manufacturing impurity, PBT1 was retired and immediately succeeded by an improved and novel MPAC – PBT2 , the leading candidate to arise out of Prana’s unique drug discovery platform. PBT2, while sharing a similar mechanism of action to PBT1, demonstrates superior technical and medicinal properties.

During the course of the development of MPACs, numerous papers from independent laboratories appeared in the scientific press, demonstrating the potential benefits of PBT1 over a broad range of human diseases, notably Parkinson’s, Huntington’s and certain cancers.

Prana scientists have demonstrated how subtle defects in the metabolism of biological metals lie at the core of common neurodegenerative conditions. For Alzheimer’s Disease (AD) in particular, this theory provides a consistent explanation for many of the otherwise disparate pathological phenomena associated with this condition.