Prana Biotechnology's lead compound in development for Parkinson’s disease (PD), PBT434, had progressed successfully through a series of preclinical development studies used to assess the suitability of a candidate compound for human clinical studies.

The studies were funded  by The Michael J Fox Foundation (MJFF) 2011 Pipeline Programme to support its ‘Therapeutic Development Initiative’. The MJFF Programme grant is milestone based.

PBT434 achieved all of its milestones in preclinical toxicology studies, genotoxicity and safety pharmacology - allowing the compound to be  positioned for  larger scale animal toxicology studies prior to commencing clinical trials.

PBT434 is a novel, orally available compound that easily crosses into the brain and is able to exert potent neuroprotective properties. Most particularly, it preserves the brain tissue in PD that degenerates over time, the substantia nigra. By preserving this tissue, Prana’s scientists have demonstrated significant restoration of motor coordination and strength in animal models.

PBT434 has been shown that it is able to impede the iron-induced oxidative damage and neurotoxic cascade that kills the substantia nigra. As such, PBT434’s mechanism of action offers a novel disease modifying therapeutic strategy in contrast to the currently marketed symptomatic agents that, at best, serve to reduce the side effects of the disease rather than alter its course.

“Based on these efficacy studies and now the completion of this suite of preclinical development assessments, Prana will look to move PBT434 into longer term toxicology studies in parallel to our scale up manufacturing plans for PBT434. All being well, we could file an IND by the end of next year and commence clinical trials in 2014,” said Geoffrey Kempler, Prana’s executive chairman. “PBT434 also marks another product pipeline milestone for Prana to complement our two ongoing trials with PBT2 in Alzheimer’s disease and Huntington disease.”

Parkinson’s disease is a devastating illness which can result in not only the loss of muscle control, speech, balance and digestive functions; it may also impair a patient’s psychiatric and cognitive function. PD is the second most common neurological indication behind Alzheimer’s disease. The current market size for drugs to treat PD is approximately $4 billion per year; however most currently available drugs primarily treat the symptoms and do not actually prevent the ongoing destruction of the substantia nigra in the brain and progression of the disease.

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

The Michael J Fox Foundation is dedicated to accelerating a cure for Parkinson’s disease and improved therapies for those living with the condition today. The Foundation pursues its goals through an aggressively funded, highly targeted research programme coupled with active global engagement of scientists, Parkinson’s patients, business leaders, clinical trial participants, donors and volunteers.