Elan Corporation, plc (NYSE: ELN) and Transition Therapeutics, Inc (TSX: TTH, NASDAQ:TTHI) today announced the results of a Phase 2 placebo-controlled study in 351 patients with mild to moderate Alzheimer’s disease who received study drug 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 (NTB) and functional (ADCS-ADL) co – primary endpoints did not achieve statistical significance.
The 250mg twice daily dose demonstrated a biological effect on amyloid-beta protein in the cerebrospinal fluid (CSF), in a subgroup of patients who provided CSF samples. This dose achieved targeted drug levels in the CSF, and showed some effects on clinical endpoints in an exploratory analysis. After reviewing the final safety data with the study’s independent safety monitoring committee (ISMC), we concluded that the 250mg twice daily dose has acceptable safety and tolerability. Elan and Transition Therapeutics intend to share the Phase 2 data in a peer-reviewed publication.
The 2 high dose groups were electively discontinued by the companies in December 2009 due to an observed imbalance of serious adverse events, including deaths; no causal relationship between the drug and the deaths could be determined. After discontinuation of the 2 high dose groups the final analysis was based on subjects who received either 250mg twice daily or placebo for up to 18 months.
Based on the preponderance of evidence from both biomarker and clinical data, and after extensive discussions with experts in the field, Elan and Transition Therapeutics intend to advance ELND005 into Phase 3 development. Specifics of the Phase 3 study design will be finalized after receiving input and concurrence from regulatory authorities.
Elan and Transition Therapeutics have agreed to work together to systematically explore all strategic, operational, and global options for the asset with the intent of maximizing the value of this innovative potential therapeutic. No timetable for completing the exploration has been set. No further disclosure is intended until its completion.