Elan Corp. and Transition Therapeutics, Inc reported results from a Phase II study of ELND005 (Scyllo-inositol) in patients with mild to moderate Alzheimer’s disease. The placebo-controlled study included 351 patients who received ELND005 for up to 18 months (Study AD201). Study subjects with MMSE scores between 16 and 26 received ELND005 oral doses of 250 mg, 1000 mg or 2000 mg twice daily or matching placebo.
The study’s cognitive and functional co–primary endpoints did not achieve statistical significance. However, the 250mg twice-daily dose demonstrated a biological effect on amyloid-beta protein in the cerebrospinal fluid (CSF), in a subgroup of patients. This dose achieved targeted drug levels in the CSF, and showed some effects on clinical endpoints in an exploratory analysis. Safety data shows that the 250mg dose has acceptable safety and tolerability. Based on the evidence from both biomarker and clinical data, Elan and Transition Therapeutics intend to advance ELND005 into Phase III development.
The two high dose groups were previously discontinued due to an observed imbalance of serious adverse events.