PBT2 has exhibited superior performance in a variety of laboratory and animal screening tests designed to assess the ability of the compound to interfere with the toxic mechanisms of AD. Particularly, in mouse models, PBT2:
These findings lead to clinical development of the compound in humans.
| Phase |
Population |
Treatment |
Status & Key Points |
| I |
Healthy males aged 18-50 years. |
Single Dose 8-arm Study: Up to 800 mg PBT2 versus placebo (‘sugar pill’). |
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.
|
| I |
Healthy males/females aged 45-75 years. |
Multiple Dose 5-arm study: Up to 800 mg PBT2 versus placebo once daily for 7 days. |
| IIa |
Mild Alzheimer’s Disease in males/females aged over 55 years. |
3-arm study: 50 mg and 250 mg PBT2 versus placebo once daily for 12 weeks. |
Status:
Primary Objective:
- To assess the safety and tolerability of PBT2 in patients suffering from early 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.
Outcome:
- PBT2 was safe and well tolerated in patients with early AD. PBT2 reduced Abeta biomarkers and showed evidence of improved executive function (cognition).
|
| IIb |
Mild-Moderate Alzheimer’s Disease in males/females aged 55-90 years. |
PBT2 (at doses to be confirmed) versus placebo once daily. |
Status: Planned.
Study Objectives include:
- To determine the impact of PBT2 on progression of cognitive and functional decline in patients suffering from mild to moderate Alzheimer's Disease (AD).
|
