Targacept Inc., a clinical-stage biopharmaceutical company developing novel NNR Therapeutics, announced the initiation of a potential registration study of AZD3480 in mild to moderate Alzheimer’s disease. The study is the subject of a Special Protocol Assessment (SPA) agreement with the US Food and Drug Administration. AZD3480 is licensed to AstraZeneca pursuant to a collaboration agreement focused in cognitive disorders. Under a prior amendment to the collaboration agreement, it was agreed that Targacept will conduct and bear all costs and expenses for the study and that AstraZeneca will pay Targacept for certain events with respect to the study. To date, AstraZeneca has paid Targacept $2.5 million and will pay Targacept an additional $3.7 million upon first dosing in the US and Europe. In addition, Targacept remains eligible for future milestone and royalty payments for AZD3480 in accordance with the terms of the parties’ collaboration agreement.

“We are pleased to get this study underway and further evaluate the promise of AZD3480 to meet the enormous unmet medical need in Alzheimer’s disease,” said J Donald deBethizy, PhD, Targacept’s president and chief executive officer. “AZD3480 has previously been studied in clinical trials totalling over 1,350 subjects and in various cognitive disorders. While the overall outcome of a previous phase II b study of AZD3480 in Alzheimer’s disease with a three-month treatment period was inconclusive because both the active comparator, donepezil, and AZD3480 did not meet the primary outcome measure, there were encouraging signals that, following consultation with thought leaders in the field, we believe merit further study in a trial with a longer duration.”

The phase II b study will evaluate the efficacy of AZD3480 head-to-head with donepezil, the marketed medication most often prescribed for Alzheimer’s disease. The trial design provides for approximately 300 patients to be randomly assigned to receive a fixed 30mg dose of AZD3480 or donepezil daily over a 12-month period. The primary efficacy outcome measure for the study is change from baseline on the Alzheimer’s Disease Assessment Scale-cognitive subscale (ADAS-Cog), an established regulatory endpoint for Alzheimer’s disease. The study is being conducted predominantly in Eastern Europe and also at sites in the United States.

Except for the phase II b study and subject to the terms of the parties’ collaboration agreement, AstraZeneca retains its right to make decisions regarding all future development of AZD3480 in cognitive disorders and would be responsible for all future development costs.

Alzheimer’s disease is a progressive, degenerative disorder that attacks the brain’s nerve cells, or neurons, resulting in loss of memory, thinking and language skills, and behavioural changes. The market research firm Decision Resources estimated that there were approximately 9.7 million people with Alzheimer’s disease in the world’s seven major pharmaceutical markets (US, France, Germany, Italy, Spain, United Kingdom and Japan) in 2009.