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Clinical Trial Programs

The following clinical trials have been conducted or are underway.

Phase

I

Population

Healthy males aged 18-50 years.
 

Treatment

PBT2-001
Single Dose 8-arm Study: Up to 800 mg PBT2 versus placebo (‘sugar pill’).

Status & Key Points

Status: Completed.

Objective: To determine the safety, tolerability and pharmacokinetics of single and multiple oral doses in healthy volunteers.

Outcome: PBT2 was well tolerated as a single dose in young male volunteers and up to 7 days of treatment in older healthy volunteers.

Phase

I

Population

Healthy males/females aged 45-75 years.

Treatment

Multiple Dose 5-arm study: Up to 800 mg PBT2 versus placebo once daily for 7 days.

Status & Key Points

Status: Completed.

Objective: To determine the safety, tolerability and pharmacokinetics of single and multiple oral doses in healthy volunteers.

Outcome: PBT2 was well tolerated as a single dose in young male volunteers and up to 7 days of treatment in older healthy volunteers.

Phase

II

Population

Mild Alzheimer’s Disease in males/females aged over 55 years.

Treatment

PBT2-201
3-arm study: 50 mg and 250 mg PBT2 versus placebo once daily for 12 weeks.

Status & Key Points

Status: Completed.

Primary Objective:

  • To assess the safety and tolerability of PBT2 in patients suffering from mild Alzheimer's Disease (AD).

Secondary Objectives:

  • To assess the effect of PBT2 on biomarkers associated with AD.
  • To determine the impact of PBT2 on cognition.

Outcomes:

  • PBT2 was safe and well tolerated in patients with mild AD.
  • PBT2 reduced Abeta levels in the CSF of patients on the 250mg dose.
  • Cognitive Executive Function as measured by the NTB (Neuropsychological Test Battery) was significantly improved for patients on the 250mg dose.

References:
Lannfelt et al. Lancet Neurology (2008) vol.7, pp 779-86
Lannfelt et al. Erratum: Lancet Neurology (2009) vol.8, pp 981

Phase

II

Population

Prodromal or Mild Alzheimer's Disease in males/females over 55 years.
(IMAGINE trial)

Treatment

PBT2-204
2 arm study: 250mg PBT2 versus placebo once daily for 52 weeks.

Status & Key Points

Status: Complete.

Primary Objective:

  • To evaluate brain amyloid levels.

Secondary Objectives:
To evaluate the effect of PBT2 on; 

  • Safety and tolerability.
  • Brain metabolic activity.
  • Brain volumes.
  • Cognition.
  • Functional abilities.   

Outcomes:

  • PBT2 was shown to be safe and very well tolerated over the 52 weeks.
  • PBT2 did not meet its primary endpoint of a statistically significant reduction in the levels of beta-amyloid plaques.
  • No improvement was observed on the secondary endpoints of brain metabolic activity, cognition and function.
  • There was a  trend towards preserving hippocampal brain volume in the PBT2 group.

Phase

II

Population

Early to Mild Stage Huntington's Disease in males/females

(REACH2HD Trial)

Treatment

PBT2-203
3 arm study: 100mg and 250 mg PBT2 versus placebo once daily for 26 weeks.

Status & Key Points

Status: Completed.

Primary Objective:

  • To evaluate safety and tolerability in patients with HD.

Secondary Objectives:
To evaluate the effect of PBT2 on;

  • Cognition.
  • Motor Function.
  • Behaviour.
  • Funcional Abilities.
  • Global Function.
  • Biomarkers.
  • Brain Volume and function.
  • Pharmacokinetics. 

Outcomes:

  • PBT2 was shown to be safe and very well tolerated.
  • There was a statistically significant improvement in performance on the Trail Making Test Part B in the PBT2 250mg group compared to placebo at both 12 (p<0.001) and 26 weeks (p=0.042).