AC-1204 Long-term Efficacy Response Trial (ALERT Protocol)

A Phase 2-3, 6-12 Month, Double-blind, Randomized, Placebo-controlled, Parallel-group Study to Investigate the Effects of Daily Administration of AC-1204 in Subjects With Mild to Moderate Alzheimer's Disease Who Are APOE4(-)

The study will evaluate the safety & efficacy of AC-1204, a ketogenic compound, administered orally on a daily basis for 6 months. Following the 6 month double-blind phase of the study, subjects may enroll in an optional 6 month open-label extension phase. Efficacy will be evaluated by standard tests of memory and cognition, along with other measurements of activities of daily living and quality of life. Safety will be assessed by frequency of adverse events and changes in laboratory test results. Subjects will be stratified and outcomes will be separately analyzed based on apolipoprotein E4 genotype (APOE4).

Two primary outcome measures will be assessed in APOE4(-) patients:

1. differences from baseline between AC-1204 and placebo groups' ADAS-cog scores at 6 months
2. differences from baseline between AC-1204 and placebo groups' CIBIC+ scores at 6 months Secondary endpoints will include differences between AC-1204 & placebo groups' scores for these same instruments at 3 months.

Inclusion Criteria:

• Males/females between age of 55 -85 years
• MMSE scores between 16-26
• Probable mild to moderate AD

Exclusion Criteria:

• Presence of other CNS disorders as alternative causes of dementia
• Type 1 or Type 2 diabetes
• Significant renal/hepatic disease