Targacept Initiates a Phase 2b Study of AZD3480 in Alzheimer’s Disease

WINSTON-SALEM, N.C.--(BUSINESS WIRE)-- Targacept, Inc. (NASDAQ: TRGT), a clinical-stage biopharmaceutical company developing novel NNR Therapeutics™, today 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 U.S. 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 U.S. 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, Ph.D., 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 2b 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 2b 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 2b 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.

About Alzheimer’s Disease

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 behavioral 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 (U.S., France, Germany, Italy, Spain, United Kingdom and Japan) in 2009.

About Targacept

Targacept is developing a diverse pipeline of innovative NNR Therapeutics™ for difficult-to-treat diseases and disorders of the nervous system. NNR Therapeutics selectively modulate the activity of specific neuronal nicotinic receptors, a unique class of proteins that regulate vital biological functions that are impaired in various disease states. Targacept’s lead program, TC-5214, is being co-developed with AstraZeneca and is in Phase 3 clinical trials as an adjunct treatment for major depressive disorder. Targacept leverages its scientific leadership and proprietary drug discovery platform Pentad™ to generate novel small molecule product candidates to fuel its pipeline and attract significant collaborations with global pharmaceutical companies. For more information, please visit www.targacept.com.

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Forward-Looking Statements

This press release includes “forward-looking statements” made under the provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements other than statements of historical fact regarding, without limitation: the progress or scope of development of AZD3480, such as the target indication(s) for development; the medical benefits that may be derived from AZD3480; the competitive position of AZD3480 or the commercial opportunity in Alzheimer’s disease; any payments that AstraZeneca may make to Targacept; or Targacept’s plans, expectations or future operations, financial position, revenues, costs or expenses. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various important factors, including without limitation risks and uncertainties relating to: the conduct and results of the ongoing clinical trial of AZD3480 in mild to moderate Alzheimer’s disease, including the performance of third parties engaged to execute the trial, delays resulting from any changes to the protocol for the trial and difficulties or delays in the completion of subject enrollment or data analysis; the control that AstraZeneca has over the development of AZD3480, including as to the timing, scope and design of any future clinical trials of AZD3480 and as to the conduct at all of further development of AZD3480, beyond the ongoing trial, in Alzheimer’s disease or ADHD; and the timing and success of submission, acceptance and approval of any regulatory filings for AZD3480, including the impact of Targacept’s special protocol assessment agreement with the FDA on the consideration of any new drug application that may be filed for AZD3480. Risks and uncertainties that Targacept faces are described in greater detail under the heading “Risk Factors” in Targacept's most recent Annual Report on Form 10-K and in other filings that it makes with the Securities and Exchange Commission. As a result of the risks and uncertainties, the results or events indicated by the forward-looking statements may not occur. Targacept cautions you not to place undue reliance on any forward-looking statement.

In addition, any forward-looking statement in this press release represents Targacept’s views only as of the date of this press release and should not be relied upon as representing its views as of any subsequent date. Targacept disclaims any obligation to update any forward-looking statement, except as required by applicable law.

NNR Therapeutics™ and Building Health, Restoring IndependenceSM are trademarks or service marks of Targacept, Inc. Any other service marks, trademarks and trade names appearing in this press release are the properties of their respective owners.


Targacept, Inc.
Alan Musso, SVP and CFO, 336-480-2186
[email protected]
Linnden Communications
Michelle Linn, 508-362-3087
[email protected]

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