February 9, 2006 Vanderbilt University, Brigham Young University and Ceragenix Pharmaceuticals have announced that one of a family of compounds, called Ceragenins (or CSAs) shows potent virucidal activity in in vitro laboratory tests against multiple strains of Human Immunodeficiency Virus (HIV), the virus that causes AIDS. CSAs were invented by Dr. Paul D. Savage of Brigham Young University's Department of Chemistry and Biochemistry and exclusively licensed to Ceragenix. In data previously presented by Dr. Savage and other researchers, CSAs have been shown to have broad spectrum antibacterial activity. Dr. Derya Unutmaz, Associate Professor of Microbiology and Immunology at the Vanderbilt University School of Medicine, tested several CSAs in his laboratory for their ability to kill HIV directly and whilst cautious, acknowledged that CSAs could be the breakthrough technology to combat HIV/AIDS researchers the world has been waiting for.
"We found that CSA-54 potently inhibits HIV infection of primary human CD4+ T cells, the virus's in vivo targets, and was not toxic to epithelial cells at concentrations significantly higher than those required to kill the virus," stated Dr. Unutmaz.
"In addition, CSA-54 killed a wide range of HIV isolates, and completely blocked genetically engineered HIV that enters the cells independent of the cell surface receptor the virus normally uses. This finding indicates that CSA-54 likely attacks the viral membrane and disrupts the virus from interacting with its target cells, similar to some of the known microbicidal peptides. This is particularly important as a compound that targets the viral membrane is likely to be effective against all strains of the virus, regardless of mutations as the viral membrane remains unchanged."
"We are encouraged, based on these early in vitro studies, that CSAs may provide a completely unique family of anti-infectives, potentially active against a wide range of viral, fungal, and bacterial targets, including those resistant to current therapies," stated Steven Porter, CEO of Ceragenix. "Given the potent activity of CSA-54 against all strains of HIV tested, we plan on exploring the use of this compound in both topical and systemic applications for HIV therapy."
Ceragenins, or CSAs, are synthetically produced small molecule chemical compounds comprised of a sterol backbone with amino acids and other chemical groups attached to them. These compounds have a net positive charge that is electrostatically attracted to the negatively charged cell membranes of certain viruses, fungi and bacteria. CSAs have a high binding affinity for such membranes (including Lipid A) and are able to rapidly disrupt the target membranes leading to rapid cell death. While CSAs have a mechanism of action that is also seen in antimicrobial peptides, which form part of the body's innate immune system, they avoid many of the difficulties associated with their use as medicines.
Brigham Young University and Vanderbilt have jointly filed a patent on CSA technology, which has been licensed exclusively to Ceragenix.
About Ceragenix Pharmaceuticals
Ceragenix Pharmaceuticals, Inc. (CGXP) is a biopharmaceutical company that discovers, develops and commercializes novel anti-infective drugs based on its proprietary class of compounds, Ceragenins (or CSAs). Active against a broad range of gram positive and negative bacteria, these agents are being developed as anti-infective medical device coatings and as therapeutics for serious antibiotic-resistant organisms.
Ceragenix further owns exclusive rights to Barrier Repair Technology for the treatment of dermatological disorders including atopic dermatitis, neonatal skin disorders and others. Ceragenix's patented Barrier Repair Technology, invented by Dr. Peter Elias and licensed from the University of California, is the platform for the development of two prescription topical creams--Epiceram(TM) and Neoceram--that form human-identical skin barriers. Defects in the skin's barrier function play critical roles in the pathogenesis of skin diseases such as eczema, irritant contact dermatitis and other common skin disorders and may also be of importance in HIV related skin dermatoses. Ceragenix has submitted a 510K to the FDA seeking marketing clearance to commercialize this technology in 2006.