Market Overview

Prana to Commence Phase 1 Clinical Trial of PBT434 for Treatment of Parkinsonian Diseases

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Highlights:

  • Prana receives ethics committee approval for a clinical trial
    evaluating first in human dosing of PBT434
  • Recruitment has commenced for the first cohort of healthy volunteers
  • Study conducted by leading Australian early phase clinical trial
    facility Nucleus Network, in Melbourne, Australia
  • PBT434 has been shown to prevent α-synuclein accumulation and
    neuron loss in experimental animal models of Parkinsonian diseases

Prana Biotechnology Ltd (ASX PBT: 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.

Clinical Appendix

 
Study title   A Phase 1 Single and Multiple Ascending Dose Study of PBT434 in

Healthy Volunteers

Unique identifier   PBT434-101
Primary endpoint   To assess the safety and tolerability of PBT434 after single and

multiple oral dose administration

Secondary endpoints

     

1)

 

To determine the pharmacokinetics (PK) of PBT434 and metabolites
after single and multiple oral dose administration

2)

To evaluate the pharmacokinetics of PBT434 and metabolites after
multiple oral dose administration in healthy elderly subjects

       

3)

 

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
 

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