Looking to add to the body of evidence collected in pre-clinical studies, Prana Biotechnology Ltd. (NASDAQ:PRAN) reported Thursday morning that the first patients in a phase 1 clinical trial have been enrolled and administered PBT434, the Melbourne, Australia-based company's experimental drug under investigation for the treatment of Parkinsonian diseases.
In animal models, PBT434 has been shown to prevent (alpha) α-synuclein accumulation and neuron loss. Atypical Parkinsonian diseases are a group of progressive conditions often associated with protein buildup in brain cells that present with the signs and symptoms of Parkinson's disease, but typically do not respond very well to treatment with the drug levodopa.
These can include dementia with Lewy bodies, progressive supranuclear palsy, corticobasal syndrome and multiple system atrophy, amongst other diseases and conditions. There are no effective therapies today for these devastating diseases.
Like many other phase 1 trials, Prana's early clinical research is designed to evaluate safety and tolerability of the drug in healthy adult and enderly volunteers while looking to determine the optimal dose of PBT434.
Secondary endpoints will look at the pharmacokinetic properties of the small molecule drug. PBT434 is the first of a new generation of drugs designed to inhibit the aggregation of alpha-synuclein and tau, critical intracellular proteins that are implicated in neurodegenerative diseases such Parkinson’s disease and atypical parkinsonism.
Hosted in Melbourne, Australia, the trial is being spearheaded by Dr. David Stamler and his team from Prana's office in San Francisco. Dr. Stamler is an esteemed scientist that has led two neurological agents through FDA approval, including deutetrabenazine for treating Huntington's disease chorea and Tardive dyskinesia.
Shares of PRAN opened Thursday trading at $1.90 after closing Tuesday ahead of the Independence Day holiday at $1.87. The stock galloped ahead as high as $2.60 shortly after the opening bell before cooling back to $2.31 as of 11:30 a.m. in New York, representing a gain of 22.5% so far on the day.
