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Prana Biotechnology Ltd (NASDAQ PRAN) is pleased to announce it has received ethics committee approval and has commenced recruitment for its Phase I Clinical trial evaluating the safety, tolerability and pharmacokinetics of the Company’s lead drug candidate, PBT434, in healthy volunteers.
PBT434 is the first of a new generation of small molecules designed to inhibit the aggregation of alpha(α)-synuclein and tau, vital intracellular proteins that are implicated in neurodegenerative diseases such as Parkinson’s disease and atypical parkinsonism. PBT434 has been shown to reduce the abnormal accumulation of these proteins in animal models of disease by restoring normal iron balance in the brain.
Multiple System Atrophy (MSA) and Progressive Supranuclear Palsy (PSP) are two forms of atypical parkinsonism with no approved therapies and Prana’s initial targets for PBT434. Sufferers experience especially rapid deterioration compared to Parkinson’s disease and typically have motor symptoms that respond poorly to available treatments. Patients with MSA also have difficulty maintaining their blood pressure along with bowel and bladder dysfunction whereas PSP patients have unsteady gait, frequent falls, visual difficulties and cognitive impairment.
The trial is being conducted by the Nucleus Network in Melbourne, Australia, and will recruit healthy adult and elderly volunteers, with the primary goal of assessing the safety and tolerability of PBT434 after single and multiple oral dose administration. Secondary endpoints include a range of pharmacokinetics measures to understand how PBT434 is absorbed and metabolised in the body.
The volunteers in the single ascending dose portion of the study will receive one single oral dose of PBT434 and will be monitored for 72 hours for safety and blood levels of drug. In the repeated dose portion of the study, subjects will receive eight days dosing with PBT434 with safety and testing for drug disposition over this time.
Prana’s Chief Medical Officer and Senior Vice President, Clinical Development Dr David Stamler, MD, said: “We are excited to begin this important phase of clinical evaluation of PBT434. Following successful completion of this study, we aim to evaluate PBT434 in MSA and PSP, which are devastating neurodegenerative diseases with no approved therapies.”
Parkinsonian movement disorders affect around 10 million people worldwide, and are best known for the impairment of motor function, gait, balance and cognition. Both MSA and PSP are orphan diseases in the US and Europe, two of the largest potential markets for PBT434.
Nucleus Network is a highly regarded clinical trial site with a strong network of volunteers to support recruitment of the study.
Prana will update the market on the progress of the clinical trial at significant events including first patient dosing and trial completion.
|Study title||A Phase 1 Single and Multiple Ascending Dose Study of PBT434 in|
|Primary endpoint||To assess the safety and tolerability of PBT434 after single and|
multiple oral dose administration
To determine the pharmacokinetics (PK) of PBT434 and metabolites after single and multiple oral dose administration
To evaluate the pharmacokinetics of PBT434 and metabolites after multiple oral dose administration in healthy elderly subjects
To evaluate the preliminary effect of food on the pharmacokinetics of PBT434
|Study design||This is a Phase 1, single-centre study consisting of two parts, both of which are randomised, double-blind, and placebo-controlled: The single ascending dose (SAD) and the multiple ascending dose (MAD) parts of the study will be conducted in sequential stages in healthy volunteers. Up to six single dose cohorts and four repeated dose cohorts will be evaluated.|
|Populations||Healthy adult volunteers age 18-55 years.|
Healthy elderly volunteers age ≥65 years.
|Trial locations||Nucleus Network, Australia|