SEATTLE--(BW HealthWire)--Nov. 17, 2000--Targeted Genetics Corporation (Nasdaq: TGEN - news) today announced that the first patient in a Phase II clinical trial of tgAAV-CF, the Company's lead product for the treatment of cystic fibrosis (CF), received study drug yesterday at Stanford University Medical Center.
The trial is being conducted in collaboration with the Cystic Fibrosis Foundation's Therapeutics Development Network. Under the terms of the Company's product development and commercialization collaboration with Celltech Group plc (NYSE: CLL - news; LSE: CCH), the opening for enrollment of a Phase II study of aerosolized tgAAV-CF triggers a $2 million milestone payment to Targeted Genetics.
``We are pleased to be moving forward in the clinical development of tgAAV-CF,'' said Dr. Thomas C. Reynolds, M.D., Vice President, Clinical Affairs at Targeted Genetics. ``Based on the data generated in our studies of tgAAV-CF in the sinuses of patients with CF and the results of the Phase I study of aerosolized tgAAV-CF delivered to the lung, we believe that the product is well-tolerated and has a good safety profile. In addition, our earlier clinical studies of tgAAV-CF have shown evidence of functional correction of the CFTR defect. This Phase II trial will provide us with our first look at clinical efficacy and will help to evaluate appropriate endpoints for future tgAAV-CF trials. As in the Phase I study of aerosolized tgAAV-CF, we are pleased to be working with the Therapeutics Development Network and expect to work closely with the organization throughout the course of this trial.''
The Phase II, double-blind, randomized, placebo-controlled study will evaluate the safety and tolerability of repeat dosing of tgAAV-CF and will assess the impact of tgAAV-CF on lung function, inflammation and microbiology in CF adolescents and adults with mild lung disease. Immune response to the product also will be evaluated; gene transfer and gene expression will be assessed in a subset of patients. A total of 36 patients will be evaluated, 18 in the treatment group and 18 in the placebo group. At four-week intervals, patients will receive 10(to the 13th power) DNAse resistant particles (DRP) of tgAAV-CF delivered via nebulizer for a total of three doses and will be followed for 90 days after receiving their last dose. People with cystic fibrosis interested in obtaining more information about entry requirements for the trial should contact Patty Pedersen at 206/521-7815.
``I've been encouraged by the tgAAV-CF safety profile and gene transfer efficiency and persistence in our single-dose, direct delivery trials to the sinuses and lungs of patients with cystic fibrosis,'' said Stanford principal investigator Dr. Richard Moss M.D., Professor and Chief of Pediatric Pulmonology. ``The Phase II trial will allow us to assess repetitive dosing and collect pilot data on potential efficacy endpoints such as pulmonary function, chest CT scans, and inflammation.''
``Our clinical, regulatory and manufacturing teams and our partners at Celltech Group plc are doing a superb job of keeping the development of tgAAV-CF on track,'' said H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics. ``Targeted Genetics' expertise in process development and the strength of our quality assurance and quality control groups helped to make tgAAV-CF the first AAV-based product to enter clinical development. We have treated 70 patients to date with this product and data from our previous clinical studies suggest that the product is well-tolerated and has a good safety profile. Our scalable manufacturing process has enabled us to meet the needs of our expanded clinical trial program and we believe that we have the most robust AAV manufacturing process available. We have a number of other AAV-based product candidates in preclinical development and believe we have the best infrastructure available with which to leverage the potential of these programs.'' |
