Prana Biotechnology has dosed the first cohort of subjects in its Phase I clinical trial of PBT434 for the treatment of Parkinsonian diseases.
The trial, which is currently enrolling healthy adult subjects, is being conducted in Melbourne, Australia.
Its primary goal is to demonstrate the safety and tolerability of PBT434. Its secondary endpoints are pharmacokinetic measures evaluating and understanding how the drug is absorbed and metabolised in the human body.
PBT434 is a new generation of small molecules capable of inhibiting the aggregation of alpha(α)-synuclein and tau, both of which are critical intracellular proteins that are implicated in neurodegenerative diseases such as Parkinson’s disease and atypical parkinsonism.
The investigational drug has demonstrated its ability to minimise the pathological accumulation of these proteins in animal models of disease and has the potential to treat various forms of atypical parkinsonism, including multiple system atrophy (MSA) and progressive supranuclear palsy (PSP).
Prana Biotechnology Clinical Development senior vice-president and chief medical officer Dr David Stamler said: “MSA and PSP are devastating diseases with no effective treatments and this is an important first step in developing a therapy for individuals with these diseases.”
Parkinson’s disease is a long-term degenerative disorder of the central nervous system that primarily affects the motor system. Symptoms generally appear slowly over time.