Company Profile: Axonyx Inc. (AXYX)

Axonyx Inc., a biopharmaceutical company, is engaged in the business of acquiring and developing central nervous system (CNS) drug candidates, primarily in the areas of memory and cognition. Axonyx acquires patent rights to CNS pharmaceutical compounds it believes may have significant potential market impact and works to advance the compounds through preclinical and clinical development towards regulatory approval. Axonyx has acquired worldwide patent rights to four main classes of therapeutic compounds designed for the treatment of Alzheimer's Disease (AD), Mild Cognitive Impairment, and related diseases of memory impairment. Axonyx has also acquired patent rights to a fifth class of potential therapeutic compounds designed for the treatment of prion related diseases. Axonyx licensed these patent rights from New York University (NYU) and, via a sublicense, from the National Institutes of Health\National Institute on Aging (NIA).

Axonyx's current business strategy is to pursue (1) the development of four different types of pharmaceutical products for the treatment of AD, (2) Gilatide, a potential pharmaceutical compound designed to enhance memory and cognition with potential applications to the treatment of AD, (3) a pharmaceutical compound for prion-related diseases, and (4) an assay method for screening drug candidates for Alzheimer's Disease.

The AD targeted approaches include:

1. Phenserine, an inhibitor of acetylcholinesterase;
2. a butyrylcholinesterase inhibitor which will be chosen from a series of selectively acting compounds;
3. Posiphen, a compound that decreases the formation of beta-amyloid precursor protein;
4. through its sublicense with ARS, a subsidiary of Serono, which is described in greater detail below, compounds called Amyloid Inhibitory Peptides (AIPs) which may prevent and reverse the formation of amyloid plaques in AD.

In January 2003 the company announced its decision to launch two European-based clinical trials with the objective of generating additional data in support of the safety and efficacy of Phenserine for regulatory approval for marketing in AD.