Elan Corporation and Transition Therapeutics have announced the results of a Phase II study for ELND005, a potential treatment for mild to moderate Alzheimer's disease.
Participants received ELND005 doses of 250 mg, 1000 mg, 2000 mg or a placebo twice daily for 18 months. However, the two higher dose trials were discontinued in December 2009 due to serious adverse events.
The results showed that the cognitive and functional co-primary endpoints of the study did not achieve statistical significance.
However, the 250mg dose did demonstrate a biological effect on amyloid-beta protein in the cerebrospinal fluid.
It also achieved targeted drug levels in the cerebrospinal fluid and had some effect on clinical endpoints in an exploratory analysis.
After reviewing the final safety data with the study's independent safety monitoring committee, it was decided that the 250mg dose had acceptable safety and tolerability.
Elan and Transition Therapeutics have decided to advance ELND005 into Phase III development.
The Phase II study involved 351 patients and was conducted across approximately 65 sites in the US. It was placebo-controlled, multi-centre, dose ranged and parallel-armed.