Elusys Therapeutics Inc., a privately-held biopharmaceutical company, announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to Anthim (ETI-204), the company's anthrax therapeutic.

The FDA's US Orphan Drug Act is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Orphan Drug designation is awarded to compounds that offer potential therapeutic value in the treatment of rare diseases, defined as those affecting fewer than 200,000 Americans. This designation provides companies with financial and regulatory benefits during the course of orphan drug development, including tax credits related to clinical trial expenses and a possible exemption from the FDA-user fee.

Elizabeth Posillico, Ph.D., president & CEO of Elusys, commented on the notice, "Receiving Orphan Drug Designation is another important milestone for Anthim. Based on its impressive safety and efficacy data, Anthim is a valuable therapeutic for addition to the U.S. Government's Strategic National Stockpile. Anthim's low therapeutic dose allows for rapid intramuscular (IM) delivery, the most effective mode of delivery to first-responders and civilian personnel in emergency situations."

Anthim is a high-affinity monoclonal antibody that targets the protective antigen component of anthrax, blocking the bacteria's ability to form deadly toxins. In animal efficacy studies, Anthim demonstrated complete protection against an anthrax spore challenge with a single prophylactic dose and has shown significant protection when administered up to two days after a lethal challenge.

Anthim has been granted Fast Track status (May 2005) and Orphan Drug Designation by the FDA and is being developed under the FDA Animal Rule, a regulatory process specifically designed for the development of medical countermeasures to bioterror threats.

According to this rule, marketing approval of Anthim can be granted based on efficacy in relevant animal models with an acceptable risk/benefit profile in humans. In March 2006, Elusys announced the successful completion of a phase I human clinical study for Anthim. This study was designed to determine the safety and tolerability of Anthim in healthy volunteers, when administered with or without the antibiotic Ciprofloxacin. There were no serious adverse events and the drug was well tolerated, showing a favourable safety risk/benefit profile, when used either alone or in combination with Ciprofloxacin.

In 2005, Elusys was awarded over $5 Million from the National Institute of Allergy and Infectious Diseases (NIAID) and the Department of Defense (DoD) for advanced formulation development. To date, the Company has been awarded over $20M from the US Government for the development of novel therapeutics to combat bioterror agents.