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Press Release
Source: Targeted Genetics Corporation
Targeted Genetics Presents Final Results From Phase II
Cystic Fibrosis Clinical Trial
Thursday June 5, 8:02 am ET
Complete Data Analysis Confirms Strong Safety Profile and Improvement In Lung
Function
WASHINGTON and SEATTLE, June 5 /PRNewswire-FirstCall/ -- Targeted Genetics Corporation (Nasdaq:
TGEN - News) today announced final results of its Phase II clinical trial in patients with cystic fibrosis. The
product candidate, tgAAVCF, met its primary endpoint demonstrating safety and tolerability in this first ever
repeat dosing study. Positive trends in pulmonary function, inflammatory cytokine (IL-8) levels and gene
transfer were also observed.
Data will be presented today at the 6th Annual American Society of Gene Therapy Meeting in Washington,
DC by Richard B. Moss, M.D., Pediatric Pulmonary Medicine, Stanford University Medical Center and lead
clinical investigator in this study. Dr. Moss will present a complete data analysis at a workshop titled,
"Respiratory Tract: Planning and Executing a Gene Therapy Trial for the Respiratory Tract."
tgAAVCF, Targeted Genetics' lead gene therapy product candidate for the treatment of cystic fibrosis, was
tested in a randomized, double-blind, placebo-controlled clinical trial that included 37 patients with mild
cystic fibrosis. Patients were monitored for approximately five months. The complete data analysis
confirmed preliminary results presented at the 16th Annual North American Cystic Fibrosis Conference in
October of 2002. Data highlights are as follows:
-- A clean safety profile was sustained over the entire 150-day study
period. No clinically significant differences in adverse events between
the product candidate and placebo were observed.
-- Statistically significant improvements in lung function (FEV1) were
observed after 30 days. In addition, positive trends were observed in
various measurements of lung function after 60 and 90 days in the
treated population, compared to the placebo group.
-- A statistically significant decrease in IL-8 levels (a cytokine
associated with inflammation) was observed after 14 days compared to
placebo.
-- Excellent gene transfer was observed in all patients tested.
After complete data analysis, additional data highlights included:
-- Chest computerized tomography (CT) scans utilized to measure additional
safety showed no statistically significant changes in CT scores between
days 0 and 90, further supporting a strong safety profile associated
with tgAAVCF.
-- 22 percent of patients treated with tgAAVCF sustained a 5 percent or
greater improvement in lung function at 90 days, while no patients
receiving placebo achieved this response.
-- 17 percent of patients treated with tgAAVCF sustained a 10 percent or
greater improvement in lung function at 90 days, while no patients
receiving placebo achieved this response.
-- No correlation was observed between the levels of AAV neutralizing
antibodies and patients' FEV1 measurements, suggesting that antibodies
to the AAV vector do not appear to impact the therapeutic profile of
tgAAVCF when administered via repeat dosing.
"Targeted Genetics Corporation is the only organization to demonstrate a statistically significant
improvement in lung function in a repeat-dosing gene therapy trial in humans with cystic fibrosis. In addition
to demonstrating a favorable safety profile over a five-month period, the CF clinician community was
encouraged by improvement trends observed in lung function throughout this study," said Dr. Moss. "Based
on these positive findings, we plan to initiate a larger, confirmatory study in patients with cystic fibrosis. This
Phase IIb clinical trial will almost triple the number of study participants and will focus primarily on the
change in lung function -- a well-established measurement of improvement in patients with this debilitating
disease. We look forward to launching this study in the coming months and to further our understanding of
the potential of tgAAVCF to go beyond management of signs and symptoms of cystic fibrosis and treat the
underlying cause of the disease."
This phase II clinical trial involved 37 CF patients with mild lung disease, 17 who received placebo and 20
who received tgAAVCF. The mean age in this study was 24 years. Following approvals from an independent
data safety monitoring board, patients as young as 12 years of age were included in the clinical trial.
Patients were randomized to receive three doses at 30-day intervals of tgAAVCF, 1013 DNAse resistant
particles (DRP) per dose, or placebo. Patients were followed for 90 days after receiving their last dose. The
primary endpoint of this study was the safety and tolerability of repeat dosing with tgAAVCF. Activity
measures included lung function (FEV1 -- a standard measurement of lung function), inflammation and
microbiology. In a subset of six patients undergoing bronchoscopy one to two months after last dosing,
gene expression, measured by determining levels of mRNA, was not demonstrated in cells brushed from the
largest airways within the limit of detection of the assay. Over the course of the clinical trial, AAV
neutralizing antibody response occurred systemically and locally, although no correlation was noted
between the levels of AAV neutralizing antibodies and patients' change in lung function (FEV1).
About tgAAVCF and Cystic Fibrosis
tgAAVCF uses an AAV vector to deliver a functionally correct copy of the CFTR gene to the airways of
patients with CF. Loss of proper function of the CFTR gene is the cause of CF. While current therapies treat
the symptoms of CF, tgAAVCF has the potential to address the underlying cause of the disease. 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's function leads to the production and build-up in the lungs and sinuses of thick sticky
mucus, colonization by bacteria and the invasion of white blood cells, thus causing inflammation. These
events produce scarring in the lung, which ultimately results in loss of respiratory function. Common
symptoms include very salty-tasting skin, persistent coughing, wheezing or pneumonia, excessive appetite
but poor weight gain, and bulky stools. It is estimated that 60,000 people worldwide have cystic fibrosis,
including approximately 30,000 people in the United States. The median age of survival for patients with CF
is approximately 33 years. For more information about CF and Cystic Fibrosis Foundation please visit
cff.org .
Targeted Genetics develops gene therapy products for treating acquired and inherited diseases. The
Company has a lead clinical product development program targeting cystic fibrosis and a promising pipeline
of product candidates focused on arthritis, an AIDS vaccine, hemophilia and cancer. The Company has a
broad platform of gene delivery technologies, as well as a promising body of technology for cellular therapy.
For more information about Targeted Genetics Corporation, please visit the Company's web site at
targetedgenetics.com .
NOTE: This release contains forward-looking statements regarding our regulatory filings, research programs,
product development and clinical trials. These statements, involve current expectations, forecasts of future
events and other statements that are not historical facts. Inaccurate assumptions and known and unknown
risks and uncertainties can affect the accuracy of forward-looking statements. Factors that could affect our
actual results include, but are not limited to, the timing, nature and results of our research and our clinical
trials, our ability to obtain regulatory approvals, our ability to protect our intellectual property, and our ability
to raise capital when needed, as well as other risk factors described in the section entitled "Factors
Affecting Our Operating Results, Our Business and Our Stock Price" in our report on Form 10-Q for the
quarter ended March 31, 2003. You should not rely unduly on these forward-looking statements, which apply
only as of the date of this release. We undertake no duty to publicly announce or report revisions to these
statements as new information becomes available that may change our expectations.
Source: Targeted Genetics Corporation |
