Biotech Values

[DewDiligence]

TGEN’s Cystic Fibrosis trial produces statistically-significant improvement in lung function:

[Although this “proof of concept” trial had only 37 patients and evaluated patients for only one month, the trial nevertheless demonstrated statistically significant efficacy relative to placebo –something that GENR’s Lomucin was unable to do in its own phase-2 trial, even though it contained 50% more patients than TGEN’s did. These results would seem to refute the assertion of “ridge_runner” (#29526 on Yahoo) that one cannot reasonably expect to achieve statistical significance in this kind of trial.]

http://biz.yahoo.com/prnews/040210/nytu052_1.html

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Targeted Genetics Announces Publication Of Cystic Fibrosis Clinical Results In Chest

Tuesday February 10, 9:03 am ET

Improvements in Lung Function and Strong Safety Profile Support Larger-Scale Clinical Trial Currently Underway

SEATTLE, Feb. 10 /PRNewswire-FirstCall/ -- Targeted Genetics Corporation (Nasdaq: TGEN - News) today announced publication of positive results from a Phase II clinical trial of the Company's lead gene delivery-based cystic fibrosis (CF) product candidate, tgAAVCF, in the February 9 issue of Chest. The trial demonstrated that use of tgAAVCF in patients with CF resulted in statistically significant improvements in lung function, as well as a clean safety profile. The article is titled "Repeated AAV2 Aerosol-Mediated CFTR Gene Transfer to the Lungs of Patients with Cystic Fibrosis: A Multicenter, Double-Blind, Placebo-Controlled Study." Chest is the official publication of the American College of Chest Physicians.

"I am very encouraged by initial improvements in lung function after treatment with Targeted Genetics' tgAAVCF in patients with CF. No other gene-based treatment has demonstrated this success to date," said Richard B. Moss, M.D., Pediatric Pulmonary Medicine, Stanford University Medical Center and lead author of the paper. "These results provide support for further investigation of this product candidate in treating the underlying cause of CF, and a larger clinical trial in 100 patients is currently underway. The goal of this study is to confirm previous observations of improvement in lung function, and to monitor other measurements associated with disease improvement. Safety also will continue to be monitored."

Published data was collected from a Phase II, randomized, double-blind, placebo-controlled clinical trial in 37 patients with mild CF. Patients received product candidate tgAAVCF or placebo and were monitored for approximately five months. The study met its primary endpoint demonstrating a clean safety profile throughout the duration of the study. Additionally, statistically significant improvements in lung function were observed after 30 days. Statistically significant decreases in IL-8 levels (a cytokine associated with inflammation) also were observed after 14 days.

Positive trends in lung function also were observed at later time points during the study. After 90 days, 22 percent of patients treated with tgAAVCF sustained a five percent or greater improvement in lung function, while no patients receiving placebo achieved this response. After 90 days, 17 percent of patients treated with tgAAVCF sustained a 10 percent or greater improvement in lung function, while no patients receiving placebo achieved this response.

"Currently available therapies manage only the symptoms of CF. Targeted Genetics' tgAAVCF seeks to treat the underlying cause of disease with the hope of preventing the progression of CF, instead of just managing the symptoms. This therapeutic approach represents significant hope for those suffering from the chronic and debilitating effects of CF," said Barrie J. Carter, Ph.D., chief scientific officer of Targeted Genetics. "We continue to make progress in our current clinical trial, and we look forward to completing the dosing of all participants by the end of 2004."

Targeted Genetics' tgAAVCF uses an adeno-associated virus (AAV) vector to deliver functional copies of the CFTR gene directly into the lungs. tgAAVCF has advanced further than any other CF gene therapy program to date and has the potential to change the course of CF treatment, whereby the underlying cause of the disease is addressed and not just the signs and symptoms.

The current Phase IIb, double-blind, randomized, placebo-controlled study includes bi-monthly evaluation of changes in lung function after repeat dosing of tgAAVCF. Researchers also are assessing the impact of tgAAVCF on inflammation and biologic markers over time when compared to placebo. The study will continue to monitor the safety and tolerability profile of the product candidate. A total of 100 patients, 12 years of age and older, will be evaluated, 50 in the treatment group and 50 in the placebo group. Study participants will receive two doses of 1x1013 DNAse resistant particles (DRP) of tgAAVCF delivered via nebulizer at day 0 and day 30 of the study and will be evaluated for efficacy for a total of 90 days. Study participants will be monitored for safety for seven months.
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