Prana Parkinson's drug meets development milestone

30 August 2012

Australia’s Prana Biotechnology (ASX: PBT) ,says that its lead compound in development for Parkinson's disease, 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 Program to support its “Therapeutic Development Initiative.” The MJFF Program 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.

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