Prana is a world leader in addressing the role of biological metals in human disease.
Abundant scientific evidence suggests that many age-related conditions result from pathological interactions between selected metals and target proteins. Prana Biotechnology has observed that Metal-Protein Attenuating Compounds (MPACs) improve Alzheimer's-like changes in the brain by preventing a build up of beta-amyloid deposits, which destroy cognitive function.
Prana Biotechnology has developed a proprietary library of MPACs which target these interactions, preventing target protein aggregation and consequent toxic gain of function.
Our focus has been to build a pipeline of compounds targeting indications with unmet medical needs in neurodegeneration – from blockbuster indications such as Alzheimer's disease to specialist orphan central nervous system (CNS) indications.
So far the Company has advanced several of these novel MPAC-based therapies to clinical trials or preclinical toxicology testing. The most advanced of these – PBT2 – is being advanced as a novel therapy for Alzheimer’s and Huntington’s disease. Phase 2 trials of PBT2 in Huntington disease and Alzheimer’s disease were reported in February and March respectively.
PBT434 is also being advanced as a treatment for Parkinson’s disease and other movement disorders, and in the past has received funding from the Michael J Fox Foundation. PBT519 is also being advanced as a treatment for brain cancer. Novel MPACs are under screening and assessment for therapeutic applications in neurodegenerative diseases.
A Short History of Prana Biotechnology
The earliest beginnings of Prana Biotechnology began in the laboratory of Professor Rudolph Tanzi at Massachusetts General Hospital in the 1980s when he was investigating the molecular and genetic basis of neurological disease. Dr Tanzi has investigated the genetic causes of Alzheimer's disease since 1982, and co-discovered the three genes which cause early-onset familial Alzheimer’s disease.
In 1994, Dr Tanzi's laboratory discovered the crucial link between zinc and copper, and the formation of beta-amyloid, findings that were published in the journal Science.
Dr Tanzi's research formed the scientific basis for founding Prana Biotechnology, which was incorporated in Melbourne, Australia in 1997. Prana's first and proof-of-concept Metal-Protein Attenuating Compound, or MPAC, was based on a compund named clioquinol or PBT1, originally marketed as an anti-amoebic agent. After investigating PBT1's mechanism of action, Prana scientists showed that it was capable of reducing plaque formation in AD mice.
Subsequent work in 2003 demonstrated a significant reduction in cognitive decline in a pilot Phase 2a study in Alzheimer's patients receiving PBT1. Due to a non-rectifiable manufacturing impurity, PBT1 was retired in 2004 and immediately succeeded by an improved PBT2 version which shares a similar mechanism of action but has superior technical and therapeutic properties.
More than 1000 MPACs have since been developed, although PBT2 remains Prana’s leading drug candidate and the subject of its most advanced clinical trials. Numerous papers from independent scientists show the potential for therapeutic applications of MPACs in neurodegenerative diseases, notably Parkinson's and Huntington's diseases, and other amyloid-based diseases.
Past and Present Supporters
Prana has developed its unique platform technology in association with internationally recognized scientists and academic institutions, including:
- The University of Melbourne
- Massachusetts General Hospital, Boston MA
- The Mental Health Research Institute of Victoria
The company's research collaborators also extend to:
- The Buck Institute for Age Research, Novato, CA
- University of California, San Francisco, CA
- University College, London
- Michael J. Fox Foundation