AXYX Axonyx

Axonyx Inc. (NASDAQ: AXYX) announced today completion of the ascending single dose Phase I trial with Posiphen(TM), in clinical development for the treatment of Alzheimer's disease progression. This double-blind, placebo controlled study of Posiphen in healthy men and women sought to establish well tolerated doses. Posiphen appears to be well tolerated at single doses up to and including 80mg. Blood levels of Posiphen associated with this study were higher than those associated with beneficial effects on beta-amyloid metabolism in animal models. The build-up of beta-amyloid (A beta) is generally believed to be causative of the dementia of Alzheimer's disease. No serious adverse events were reported at any dose level. During each dosing period in the study, a single dose level of Posiphen was evaluated. Double-blind Posiphen was administered to groups of five healthy men and five healthy women, and double-blind placebo was administered to one man and one woman, for each dosage level of 10, 20, 40, 80 and 160mg. Blood samples were collected for pharmacokinetic analyses. Safety assessments, including physical examinations, clinical laboratory tests, vital signs, ECGs, and the monitoring of adverse events, were done throughout. Current treatment of Alzheimer's disease focuses primarily on acetylcholinesterase inhibition. Posiphen appears to modify the metabolism of beta-amyloid precursor protein (beta-APP). A major pathological hallmark of AD is the appearance of senile plaques that are primarily composed of aggregated forms of beta-amyloid (A beta) derived from beta-APP. Soluble forms of A beta have been shown to cause significant toxicity in vitro and in vivo and hence represent a target for drug development in AD treatment. The acetylcholinesterase (AChE) inhibitor Phenserine, which is currently in development by the Company mainly for the symptomatic treatment of mild to moderate AD, and its positive isomer, Posiphen, have both been found to significantly reduce beta-APP and A beta in cell culture systems and animals. As a consequence of its apparent lack of AChE inhibitory activity, Posiphen may be administered in relatively high doses. Such high doses may result in potentially meaningful reductions in A beta in Alzheimer's disease patients. The Company anticipates initiating a Phase I ascending multiple dose study in the near future. About Axonyx Axonyx Inc. is a U.S.-based biopharmaceutical company engaged in the acquisition and development of proprietary pharmaceutical compounds for the treatment of Central Nervous System disorders. The Company currently has three compounds in development for Alzheimer's disease, namely Phenserine - a potential symptomatic and disease progression treatment of mild to moderate Alzheimer's Disease (AD), Posiphen(TM) - a potential disease progression treatment for AD now in Phase I, and BisNorCymserine (BNC) - a potential symptomatic treatment of severe AD now in pre-Investigational New Drug (IND) stage. The Company is looking to identify licensing partners to further the development of Phenserine. This press release may contain forward-looking statements or predictions. These statements represent our judgment to date, and are subject to risks and uncertainties that could materially affect the Company, including those risks and uncertainties described in the documents Axonyx files from time to time with the SEC, specifically Axonyx's annual report on Form 10-K. Specifically, with respect to our drug candidates Phenserine, Posiphen(TM) and BisNorCymserine, Axonyx cannot assure that: any preclinical studies or clinical trials, whether ongoing or conducted in the future, will prove successful, and if successful, that the results can be replicated; safety and efficacy profiles of any of its drug candidates will be established, or if established, will remain the same, be better or worse in future clinical trials, if any; pre-clinical results related to cognition and the regulation of beta-APP will be substantiated by ongoing or future clinical trials, if any, or that any of its drug candidates will be able to improve the signs or symptoms of their respective clinical indication or slow the progression of Alzheimer's disease; any of its drug candidates will support an NDA filing, will be approved by the FDA or its equivalent, or if approved, will prove competitive in the market; or that Axonyx will have or obtain the necessary financing to support its drug development programs. Axonyx undertakes no obligation to publicly release the result of any revisions to such forward-looking statements that may be made to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events.