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FDA Approval to Commence Huntington’s Disease Clinical Trial Using Prana’s PBT2: Huntington Study Group appointed to coordinate the trial and start recruitment

Alterity Therapeutics

Melbourne – 4 January, 2012: Prana Biotechnology (NASDAQ:PRAN; ASX:PBT) today announced that it has received approval from the United States Food and Drug Administration (FDA) to start recruiting patients for the company's first clinical trial using PBT2 in patients with Huntington’s Disease (HD).

Prana’s Investigational New Drug Application (IND) is now open. “The opening of this IND for a Phase 2 study follows an extensive review of PBT2 data by the FDA and reflects a favourable analysis from the FDA to support the study of PBT2 in Huntington’s Disease patients”, commented Geoffrey Kempler, Prana’s Executive Chairman.

Huntington’s Disease is a complex and severely debilitating genetic, neurodegenerative disease, for which there is no cure. The disease often affects young adults and, whilst associated with severe physical movement symptoms, progressively impacts the mind and emotions as well. The disease causes incapacitation and death about 15-25 years after onset.

The Company has appointed the Huntington Study Group (HSG) to coordinate the trial. HSG will commence recruitment of patients for the trial, named “Reach2HD, at clinical sites across USA and in Australia. The randomised, double-blind, placebo-controlled trial will enrol 100 patients with early to mid-stage Huntington’s Disease. The Principal Investigator on the study is Dr. Raymond Dorsey of Johns Hopkins University Medical Center. The protocol synopsis appears below in Appendix 1.

Professor Ira Shoulson, Professor of Neurology, Pharmacology and Human Science at Georgetown University (Washington DC) and the Chair of the Executive Committee of the Huntington Study Group said “PBT2 attracted our attention as an experimental drug with the potential to bring real benefit to Huntington’s Disease patients who suffer from a range of motor, behavioural and cognitive symptoms. The favourable signals from the PBT2 trial in Alzheimer’s Disease are particularly promising”.

The disease affects 30,000 people in the US and about 70,000 worldwide. There are no drugs in development that have established clinical evidence for treating cognitive decline. Prana aims, in this trial, to demonstrate safety, motor benefits and the same cognitive benefits for Huntington’s patients that it has already demonstrated in Alzheimer’s patients treated with PBT2. 

Appendix 1 – Protocol Synopsis

Title

A randomised, double-blind, placebo-controlled study to assess the safety and tolerability, and efficacy of PBT2 in patients with early to mid-stage Huntington’s disease (HD) 

Study Number

PBT2-204 

Study Name/Acronym 

Reach2HD 

Study Design

Randomised, double-blind, placebo-controlled, parallel group, multi-centre, Phase 2a study. 

Objectives

Primary objective:

To evaluate the safety and tolerability of two dose levels of PBT2 when administered orally once daily over 26 weeks in patients with HD.
Secondary objectives:

Determine the effect of PBT2 after 26 weeks in patients with HD on:

  1. Cognition
  2. Motor function
  3. Behaviour
  4. Functional abilities
  5. Global function
  6. Plasma and urine biomarkers
  7. Brain volumes and function (imaging), and
  8. To evaluate the Pharmacokinetics of PBT2 in patients with HD. 
Number of Patients 

It is planned that 100 patients will be randomised in to the study. 

Key Patient Criteria 
  • Men and women with Total Functional Capacity (TFC) 6-13, inclusive, and a CAG repeat number of ≥ 36
  • Montreal Cognitive Assessment (MoCA) score ≥ 12 
Doses

Placebo (0mg PBT2), 100mg PBT2 and 250mg PBT2, once daily capsules. 

Per Patient Duration

34 weeks: Four week Screening period, 6 months (26 weeks) treatment period and Follow-up 4 weeks post treatment. 

Endpoint

Primary

  • Safety and Tolerability assessments.

Secondary

  • Cognition Tests: Cognitive Test Battery (consisting of Category Fluency Test, Trail Making Test parts A and B, Map Search, Symbol Digit Modalities Test and Unified Huntington Disease Rating Scale (UHDRS) Stroop Word Reading). MoCA.

  • Motor Function Tests: UHDRS ‘99 Motor component; Speeded

Tapping Task.

  • Behaviour: UHDRS Behavioural component.
  • Functional Abilities: Total Functional Capacity and
  • Independence Scale from UHDRS ’99; Schwab & England
  • Activities of Daily Living Scale (SEADL).
  • Subject and investigator global assessments: Patient Reported
  • Outcomes; Clinical Global Impression – Severity Scale.
  • Biomarkers: small molecule markers of metabolic and oxidative stress in blood and urine; blood levels of total and mutant huntingtin; gene expression markers of HD progression; plasma selenium.
  • Brain Imaging: volumetric and functional measures.
  • Pharmacokinetics: sparse sampling. 
Trial Locations
  • Australia
  • USA 
Trial Standard

Study will be conducted according to ICH GCP

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