Targeted Genetics Completes Phase II Clinical Study in Cystic Fibrosis
07:44 a.m. Oct 16, 1998 Eastern

SEATTLE, Oct. 16 /PRNewswire/ -- Targeted Genetics Corporation (Nasdaq: TGEN) announced today that it has completed a Phase II clinical trial of tgAAV-CF, the company's proprietary gene therapy for cystic fibrosis (CF). The preliminary data from the trial was presented yesterday at the 12th Annual North American Cystic Fibrosis Conference, in Montreal, Canada by John Wagner, M.D., Ph.D., a fellow in clinical pharmacology and clinical associate physician in the General Clinical Research Center at Stanford University and co-investigator of the trial. The study, titled "A Phase II, double-blind, placebo-controlled clinical trial of tgAAV-CF in the maxillary sinus of cystic fibrosis patients," was presented in the "Advances in Gene Therapy" session of the meeting on October 15.

The Phase II trial, conducted at Stanford University, was designed to assess the safety and the effect of tgAAV-CF in preventing the recurrence of sinusitis in CF patients. Targeted Genetics' proprietary tgAAV-CF uses adeno-associated virus (AAV) to deliver a normal copy of the CFTR gene that is non-functional in CF patients. A total of 23 patients were treated with tgAAV-CF in one sinus and with placebo in the other. Patients were monitored for changes in a variety of serological markers, including detection of circulating vector and production of antibodies that would inhibit the viral vector (neutralizing antibodies). No serious adverse events related to the vector have been reported in more than three months of post-treatment follow-up.

"As predicted, sinusitis recurred unilaterally in more than 60 percent of patients in the study, enabling us to assess the impact of tgAAV-CF treatment on episodes of sinusitis in this patient population," said Dr. Wagner. "Our next step will be to determine if sinusitis recurred in the treated sinuses or in the sinuses that received placebo, and we expect to have our analyses completed by the end of this year. We believe that maxillary sinusitis may be a useful surrogate model for CF lung disease and look forward to sharing our findings with our colleagues and the CF community. The final data package will be submitted for publication during the first quarter of 1999."

The trial was initiated based on positive results from a previous Phase I trial, which demonstrated that tgAAV-CF was safe and provided both efficient gene transfer and long-term gene persistence (up to 70 days) in the maxillary sinus. Since sinus and lung infections in CF patients both result from the accumulation of thick mucus caused by loss of CFTR function, efficacy of tgAAV-CF in preventing infections of the maxillary sinus may be an appropriate model for assessing the activity of the product in lung disease.

"To date, 46 patients have received tgAAV-CF without any adverse side effects related to the drug," said H. Stewart Parker, president and chief executive officer of Targeted Genetics. "We are very pleased with the safety profile of this product and are working to complete the analysis of data from this trial. Targeted Genetics intends to initiate Phase I trials of an aerosolized formulation of tgAAV-CF prior to the end of 1998. We believe that an aerosolized formulation will provide widespread delivery of tgAAV-CF to the airways of the human lung and has the greatest potential to alleviate the pulmonary symptoms associated with CF. Ultimately, we expect to take this aerosolized formulation of tgAAV-CF for the treatment of CF through the regulatory process for approval as a commercial product."

CF Background

CF results from the loss of function of the CFTR gene. Normally, the protein encoded by this gene is found in the respiratory passageways, pancreas, salivary and sweat glands where it helps to regulate the cellular concentration of chloride ions and water. In CF patients, the loss of this gene function causes the production and build-up in the lungs and sinuses of thick mucus, which is readily colonized by bacteria. The thickness of the mucus prevents clearance of bacteria and reduces the efficacy of antibiotics. Repeated infections produce scarring in the lung, which ultimately results in loss of respiratory function. The accumulation of mucus in the sinus results in a high frequency of infection and sinusitis in CF patients. It is estimated that 55,000 people world-wide have cystic fibrosis including approximately 30,000 people in the U.S. The median life expectancy of patients with CF is approximately 31 years.

Targeted Genetics Corporation develops gene and cell therapy products for the treatment of certain acquired and inherited diseases. The Company has three lead product development programs targeting cystic fibrosis, cancer, and infectious disease, as well as an extensive technology platform.

This news release contains forward-looking statements that involve risks and uncertainties, including the timing and results of clinical trials and other product development and commercialization risks. Risks associated with liquidity, capital resources and other risks are detailed in Targeted Genetics' filings with the U.S. Securities and Exchange Commission (SEC).

This release is also available on the Internet at http://www.noonanrusso.com. SOURCE Targeted Genetics Corporation

Copyright 1998, PR Newswire 

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