APT Starts Human Trials of Aerosolized Hydroxychloroquine for Asthma

Tucson, Ariz. – Sep 13, 2004

APT Pharmaceuticals is beginning clinical studies of aerosolized hydroxychloroquine (AHCQ) for treatment of respiratory diseases such as asthma, chronic obstructive pulmonary disease (COPD), rhinitis and severe acute respiratory syndrome (SARS).

Hydroxychloroquine is best known as a treatment for malaria, but the drug is also classified as a slow-onset disease-modifying antirheumatic drug (DMARD) administered in tablet form as a first-line therapy for systemic lupus erythematosus, rheumatoid arthritis and sarcoidosis. APT’s patented technology is based on targeted administration of amino quinolines to inflamed tissues. APT’s proprietary aerosolized dosage forms and routes of administration achieve a faster onset of action and greater therapeutic effect than conventional oral therapy, and at substantially lower systemic doses. The company believes targeted delivery of hydroxychloroquine will be a highly effective and safer alternative to corticosteroid treatments.

APT starts human safety and tolerability studies of AHCQ today in Australia and plans to begin Phase II studies in asthmatics in the first quarter of 2005. These studies will use the advanced AERx® pulmonary delivery system by Aradigm Corporation of Hayward, Calif., which is designed to maximize drug delivery in a patient-friendly format.

“The goal of the first study is to establish safety parameters of this new route of administration and dosage form in order to set the stage for efficacy studies in diseases such as asthma, COPD and SARS,” said APT President Gino Di Sciullo, Ph.D. “AHCQ offers the prospect of achieving antiviral and anti-inflammatory therapeutic effects within hours rather than the weeks to months needed in current oral dosing of hydroxychloroquine.”

APT has collaborated with researchers from leading academic centers in the United States and Canada to investigate the benefit of AHCQ on respiratory viral infections.

“Laboratory studies have demonstrated that hydroxychloroquine inhibits both the transmission and the inflammatory responses of human airway cells to the common cold virus (human rhinovirus),” said B. Lauren Charous, M.D., director of the Allergy and Respiratory Care Center at Advanced Healthcare in Milwaukee, who is scientific adviser to APT. “We are encouraged that the levels needed to block the virus can be achieved by aerosolized delivery. The combined anti-inflammatory and antiviral activities of AHCQ have the potential to create a new product category for treatment of pulmonary inflammation.”

This summer, with funding from the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health, Dale Barnard, Ph.D., at the Institute for Anti-Viral Research at Utah State University in Logan, completed initial laboratory studies showing hydroxychloroquine inhibits SARS-associated corona virus (SARS-CoV) at similar low concentrations. These results with hydroxychloroquine were corroborated recently in findings reported by Marc Van Ranst, Ph.D., a virologist at the Rega Institute for Medical Research in Belgium. He and his colleagues found that chloroquine, a closely related drug, is also effective at inhibiting SARS-CoV in vitro. NIAID is supporting further studies by Barnard of hydroxychloroquine in an animal model. These studies are currently underway.

Research Corporation Technologies (RCT) in Tucson, Ariz., founded APT Pharmaceuticals to advance development of the AHCQ technology. As the primary investor, RCT funded early formulation work, efficacy studies, preclinical safety studies and continues its support of these clinical trials. U.S. Patent No. 6,572,858 and other pending worldwide patent applications protect the APT technology.