Axonyx Inc.

AXONYX is a biopharmaceutical company engaged in the business of acquiring and developing novel post-discovery central nervous system drug candidates, primarily in areas of memory and cognition. AXONYX has acquired worldwide exclusive patent rights to two main classes of therapeutic compounds that deal with the memory enzymes, acetylcholinesterase and butyrylcholinesterase, which are designed for the treatment of age-related and disease-related dementias, in particular Alzheimer.s disease (AD).

AXONYX.s current business strategy is to concentrate its financial resources primarily on the further development of Phenserine, the company.s lead acetylcholinesterase compound for the treatment of AD. AXONYX recently initiated a Phase IIB beta-amyloid clinical trial, as well as a pivotal Phase III clinical trial that will evaluate the safety and efficacy of Phenserine treatment in mild-to-moderate AD patients. Phenserine is both an acetylcholinesterase- and beta-amyloid precursor protein (beta-APP) inhibitor. This compound has previously been shown to be safe and efficacious in improving memory in a 12-week Phase II study in mild-to-moderate AD patients. Phenserine.s dual action involves two unique mechanisms: (1) it inhibits the degradation of the brain neurotransmitter acetylcholine which results in increased memory, and (2) it inhibits the production of toxic beta-amyloid protein, a substance that is a major causative factor in AD patients that leads to the death of brain cells. Unlike other acetylcholinesterase inhibitors that are currently available, which simply suppress the activity of this enzyme, Phenserine’s dual mechanism of action suggests that it not only has the potential to improve memory and cognition but to also slow disease progression.

The company has also acquired the patent rights to two additional platform technologies, the prion and amyloid inhibiting peptides (PIP.s and AIP.s resp.). These compounds have been licensed to Geneva, Switzerland-based Serono S.A., which is the third largest biotech company in the world. The AIP.s (beta-sheet breakers) have the potential to disrupt and/or breakup the toxic beta-amyloid plaque that surrounds the nerve cells of patients with AD. AXONYX received a $1 million milestone payment when Serono initiated a Phase I clinical trial with a beta-sheet breaker peptide for the potential treatment of AD.