Copernicus Receives Milestone Payment from Cystic Fibrosis Foundation; Milestone Part of Grant to Support Innovative Gene Delivery Approach to Treat Cystic Fibrosis.

Business Editors & Health/Medical Writers

CLEVELAND--(BW HealthWire)--Nov. 1, 2000

Copernicus Therapeutics, Inc. has received a milestone payment as part of a $1.5 million Phase II grant from the Cystic Fibrosis Foundation to advance the development of therapeutic treatments for Cystic Fibrosis (CF).

Copernicus demonstrated in vitro the development of a non-viral gene delivery system (i) that contains a functional CFTR gene, and; (ii) that targets a receptor protein found on airway cells. The grant supports preclinical and early clinical development of Copernicus' targeted PLASmin(TM) Complexes that should enable the delivery of therapeutic genes to the cells lining the airways and lungs of CF patients. Copernicus' unique, non-viral approach to deliver the Cystic Fibrosis Transmembrane Regulator gene (CFTR) to CF patients' affected cells may eventually provide a long-term treatment for the disease.

"Our partnership with the Cystic Fibrosis Foundation reflects the critical need for an effective CF therapy and underscores the clinical potential of Copernicus' gene delivery and targeting technologies," said Robert C. Moen, M.D., Ph.D., President and Chief Operating Officer of Copernicus. "Copernicus' systemic approach is designed to bypass the difficulties encountered by other CF therapies, which attempt to administer pharmaceuticals and other gene therapies via inhalation to the surface of defective lung cells. We expect that when used together, our PLASmin(TM) compaction technology and our cell specific receptor-targeting approach will enable the delivery and efficient uptake of functional CFTR genes into the epithelial cells of the lung."

"Reaching this milestone reflects the continuing advances being made to efficiently deliver healthy genes into cells," said Robert J. Beall, Ph.D., President and Chief Executive Officer of the CF Foundation. "We are encouraged by the progress made by Copernicus in this innovative approach to therapy. Gene-based therapeutics offer hope for a life-saving treatment that tackles the root cause of CF, rather than only treating the symptoms."

CF, a fatal genetic disease afflicting over 60,000 people in the United States and Europe, is caused by a defect in the gene expressing the CFTR protein. The disease produces a thick mucus that obstructs the airway, resulting in a heightened susceptibility to chronic infections and extensive lung damage that can lead to death in early adulthood. One challenge to successful CF treatment has been an inability to concentrate therapeutic compounds at critical sites in the lungs and airways. Because mucus accumulates in the damaged tissues of CF patients, most inhaled therapeutics become trapped and fail to reach their targets at effective doses.

In recent years, medical research has explored new applications of DNA-based therapeutics for the treatment of CF. These drugs are designed to correct defective CFTR transport by providing a functional copy of the CFTR gene to epithelial cells lining a patient's airway passages and lungs. In order for such DNA-based drugs to have a therapeutic effect, however, these compounds must transport DNA into the nucleus of target cells, where it can achieve functional restoration of normal CFTR activity. Copernicus has developed proprietary methods to produce PLASmin(TM) Complexes, which are compacted structures containing only a single nucleic acid molecule. Consequently, PLASmin(TM) Complexes can much more effectively cross the cell membrane and enter cell nuclei than DNA that has not been compacted. In addition, Copernicus has demonstrated that PLASmin(TM) Complexes produce high levels of nucleic acid expression, can uniquely transfer DNA to non-dividing cells, resist physical and enzymatic destruction, and can be adapted for use with a wide range of nucleic acids.

Copernicus' PLASmin(TM) Complexes can be combined with the Company's proprietary targeting technology, which enables the selective uptake of therapeutic compounds by specific cells and tissues. To target airway cells, a novel binding fragment has been developed that binds to the polymeric immunoglobulin receptor (pIgR) located on airway epithelial cells. By linking a PLASmin(TM) Complex encoding the CFTR gene with the pIgR-targeting agent, Copernicus is developing a strategy that may eventually allow efficient delivery of therapeutic DNA intravenously to CF patients and correct the genetic abnormality.

The CF Foundation was established in 1955 to find a cure for CF and to improve the quality of life for the 30,000 children and young adults with the disease in the United States. Additional information can be accessed through the CF Foundation Web site at: http://www.cff.org. In 1997, the CF Foundation launched the Therapeutics Development Program to facilitate a pipeline of new therapies for CF. The program, in part, provides financial and early clinical support to biotechnology companies to stimulate the development of new therapeutics.

Copernicus Therapeutics, Inc., a privately held biotechnology company, is advancing novel targeting and delivery systems with broad applications in human therapeutics, DNA vaccines and functional genomics. Copernicus' technologies include a targeting platform enabling the efficient uptake of drugs by specific cells and tissues and a multi-component delivery platform that can be applied to nucleic acids to develop therapies for a variety of human diseases as well as to enhance transgene expression. The Company's targeting, delivery and expression platforms are complementary and can be combined to enhance the efficacy and safety of existing drugs, to create novel therapeutics, and to speed up the drug discovery process.