AXYX Axonyx

Axonyx Inc. (NASDAQ: AXYX) announced today the completion of its second Phase I study with Posiphen(TM) in clinical development for the treatment of Alzheimer's disease progression. This double-blind, placebo-controlled multiple ascending-dose safety and pharmacokinetic study of Posiphen in healthy volunteers sought to establish well tolerated doses. The initial review of the clinical adverse event data appears to be generally consistent with the results of the earlier single ascending dose Phase I study that suggested that the mean Posiphen blood levels associated with well tolerated doses in humans are higher than those associated with potentially beneficial effects on beta-amyloid metabolism in animal models. The build-up of beta-amyloid (Aa) is generally believed to be causative of the dementia of Alzheimer's disease and its progression. No serious adverse events were reported at any dose level in this second Phase I study. This multiple ascending-dose study examined the effects of Posiphen 20, 40 and 60mg given four times daily, for a period of 7, 7 and 10 days respectively. On the first and last day of each dosing period one single dose of Posiphen was given. Each dose period was completed and evaluated for safety and tolerance before the next higher dose level was initiated. Each cohort was composed of a different set of 16 subjects, comprised of 12 who received Posiphen and 4 who received placebo, with equal numbers of males and females in each. The necessary detailed safety, pharmacokinetic and pharmacodynamic analyses are ongoing. Based on this favourable clinical outcome, Axonyx is evaluating plans regarding the further clinical development steps for Posiphen. Posiphen(TM) Current treatment of Alzheimer's disease focuses primarily on acetylcholinesterase inhibition. 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. Posiphen appears to modify the metabolism of beta-amyloid precursor protein (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. 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. 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; Axonyx will be able to successfully out-license any of its drug candidates; Axonyx will be able to successfully in-license any additional compounds; or that Axonyx will have or obtain the necessary financing to support its drug development programs. Axonyx cannot assume that it will be successful with regard to identifying a (sub-) licensing partner for any of its compounds. 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.