Megabios and Glaxo Wellcome Gene-Based Therapeutic for Cystic Fibrosis Found To Be Safe in Phase I/II Study
08:16 a.m. Oct 16, 1998 Eastern

BURLINGAME, Calif., Oct. 16 /PRNewswire/ -- Megabios Corp. (Nasdaq: MBIO) today announced that GR213487B, a gene-based therapeutic for cystic fibrosis ("CF") that is being developed in a partnership with Glaxo Wellcome (NYSE: GLX), revealed no evidence of inflammation and satisfied all of the safety parameters studied in a Phase I/II clinical trial. The results of the trial were presented yesterday by Dr. Peadar Noone, from the University of North Carolina - Cystic Fibrosis Clinical Research Team at the Twelfth Annual North American Cystic Fibrosis Conference which is taking place this week in Montreal, Canada.

GR213487B utilizes Megabios' aerosol plasmid delivery platform to deliver the cystic fibrosis transmembrane regulator ("CFTR") gene to the nasal passages of cystic fibrosis patients. The product was evaluated in a double blind, dose escalation study in eleven adult CF patients. The clinical research team saw no evidence of adverse blood chemistry parameters and no evidence of inflammation as a result of treatment. They examined a comprehensive panel of biochemical markers of inflammation and found that the treatment did not elicit any significant alterations in these parameters.

Vector-specific plasmid was detected at up to five days post-treatment in several patients, after lavage of the nasal passages. The clinical research team reported that there was a possible trend toward correction of the CFTR -- mediated chloride defect in the treated side of the nose, as compared with the untreated side. In this study, levels of vector specific mRNA were not detectable.

"The results of this test were as we expected," said Rodney Pearlman, Senior Vice President of Research and Development at Megabios Corp., "and we are encouraged that our delivery system continues to exhibit a strong safety profile. This is a promising first step in the development of a gene-based therapeutic as a potential treatment for this disease."

Cystic fibrosis is the most common fatal genetic disease in Caucasians, occurring in about 1 in 3,000 live births. The disease is believed to afflict approximately 55,000 patients in the United States and Europe. There are currently no effective products to treat cystic fibrosis, which is caused by a defect in the CFTR gene. This defect results in production of defective CFTR protein, leading to the build-up of mucus in the lungs that often results in infection, loss of lung function and premature death. The median life expectancy of a patient with cystic fibrosis is approximately 30 years, and patients with CF typically incur annual medical costs ranging from $15,000 to $55,000.

Megabios believes that a gene-based therapeutic which results in increased levels of normal CFTR protein on the surface of ciliated epithelial cells may slow or halt the progression of this disease while reducing the total cost of patient care.

Megabios Corp. develops proprietary gene delivery systems and provides development expertise to create gene-based therapeutics designed for the treatment or prevention of genetic and acquired diseases. Megabios has developed several in vivo, plasmid-based gene delivery systems to address a number of potential therapeutic applications including cystic fibrosis, metastatic melanoma, non-small cell lung cancer, peripheral vascular disease and rheumatoid and osteoarthritis using a variety of therapeutic agents. The Company's strategy is to enter into collaborative research and development agreements and corporate partnerships whereby the Company is responsible for preclinical and early clinical development and its partners are responsible for late-stage clinical development and marketing and sales of commercial products. Megabios has corporate partnerships with Glaxo Wellcome plc to develop a treatment for cystic fibrosis using the CFTR gene and with Eli Lilly & Co. to develop treatments for breast and ovarian cancer using the BRCA1 gene.

In addition, the Company has a manufacturing partnership with DSM Biologics based on Megabios' proprietary methods for the manufacture of DNA plasmids and DNA: lipid complexes, creating the first contract facilities that can produce high-quality, ultrapure material for plasmid-based therapeutics on every scale, from preclinical toxicology studies to commercial products.

Additional information about MEGABIOS can be found at www.MEGABIOS.com.

Statements in this press release that are not strictly historical are "forward-looking" statements as defined in the Private Securities Litigation Reform Act of 1995. There can be no assurance that Megabios will be able to develop a commercially viable gene-based therapeutic, that any of the programs will be partnered with a pharmaceutical partner, that necessary regulatory approvals will be obtained, that any clinical trials will be successful. The actual results may differ from those projected in the forward-looking statement due to risks and uncertainties that exist in the Company's operations and business environment, described more fully in the Company's report on Form 10-K for the period ended June 30, 1998, filed with the Securities and Exchange Commission. SOURCE Megabios Corp.

Copyright 1998, PR Newswire 

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