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Title: Key Peptide Responsible In Cystic Fibrosis-Associated Lung Infection
URL: http://www.pslgroup.com/dg/1EAD2.htm
Doctor's Guide
February 21, 1997


PLYMOUTH MEETING, Pa., Feb. 21, 1997 -- Researchers report critical evidence that inactivation of a natural antibiotic peptide is responsible for life-threatening lung infections in cystic fibrosis patients.

In a paper published today in the journal Cell, scientists at Magainin Pharmaceuticals Inc. (Nasdaq: MAGN), along with collaborators at the University of Pennsylvania report this important finding regarding the role of a key peptide, human beta-defensin-1 (HBD-1), in mediating the onset of lung infection in cystic fibrosis patients. This finding further supports the concept of developing a therapeutic to replace HBD-1 for the treatment and prevention of lung infection. Early studies that aided in the discovery of HBD-1 were supported by the Cystic Fibrosis Foundation.

The reason for cystic fibrosis patients' high susceptibility to serious infection, resulting in lung destruction, has always been unclear. In today's publication, scientists collaborating at Magainin and Penn have now shown that, in healthy subjects, HBD-1 acts as a natural antibiotic, protecting the lung from infection. In cystic fibrosis, however, an increase in the amount of salt present on the surface of the lung bronchial tubes, inactivates this natural defense system. As reported by Goldman et. al., [Cell 88:1-20 (1997)], inactivation of HBD-1 was recreated in a bronchial xenograft model and shown to promote the lung infections typical of cystic fibrosis.

"This discovery paves the way for a new therapeutic approach to address the serious lung infections associated with cystic fibrosis," said Jay Moorin, Chairman, President and Chief Executive Officer of Magainin Pharmaceuticals Inc. "A number of our magainin peptides and their derivatives have been demonstrated to be salt insensitive and mimic the antibiotic activity of the natural peptide, HBD-1. We are currently conducting pre-clinical studies of lead candidates for replacement of HBD-1 as a therapeutic for cystic fibrosis patients. We believe that an aerosol delivery system will be the best method for delivery of this magainin peptide."

"With the contributions of Dr. Michael Zasloff and his team at Magainin, we have been able to elucidate an important new approach to modulating natural antibiotic defenses in the cystic fibrosis patient," said James M. Wilson, M.D., Ph.D., Director, Institute for Human Gene Therapy, University of Pennsylvania Medical Center. "This research is an important first step in exploring therapeutic opportunities that will enhance or even replace natural immunity."

Magainin Pharmaceuticals Inc. is a biopharmaceutical company involved in isolating and developing compounds from the host-defense systems of animals and uses molecular techniques such as gene identification to understand the pathogenesis of disease. Magainin's development efforts are focused on anti-infectives, oncology and, pulmonary and allergic disorders.

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