Randomized, double-blind, placebo-controlled, dose-ranging, efficacy and safety study of oral ELND005

Study Design

• Approximately 340 participants aged 50-85 years with mild to moderate AD
• 250, 1000, 2000 mg bid ELND005 vs. placebo
• 62 clinical sites across North America
• Protocol modified in December 2009
   - Discontinued 1000 and 2000 mg bid
   - 250 mg and placebo to continue
   - Last patient last visit in April 2010
• Status: COMPLETED (Aug 2010) - Link to press release

Primary Outcome Measures

• Safety and tolerability
• Cognitive and functional measures

Key Findings

• Reduction in cognitive and functional decline in mild AD population
• Reduction in emergence of new neuropsychiatric symptoms (including depression, anxiety)
• Evidence of target engagement (CSF) and neuronal protection
• Acceptable safety and tolerability profile in humans
• Oral drug that crosses the blood-brain-barrier
• Supports Phase III development in AD