SEATTLE and PARIS, June 12 Targeted GeneticsCorporation (Nasdaq: TGEN) announced today that positive results of a Phase1/2 clinical study demonstrate that tgAAC94, its investigational agentdesigned to inhibit activity of tumor necrosis factor-alpha (TNF-alpha), a keymediator of inflammation, is well tolerated and may improve disease symptomsin inflammatory arthritis patients refractory to other therapies, includingsystemic TNF antagonists. Full results of the study were presented here todayduring an oral presentation at the Annual European Congress of Rheumatology(EULAR).
The Phase 1/2 study evaluated multiple doses of tgAAC94 administereddirectly to affected joints with or without concurrent systemic TNF antagonisttherapies. The study achieved its primary endpoint of safety andtolerability.
"We are encouraged by the patient-reported symptom improvement -- earlysigns of efficacy that indicate further study is warranted," said PhilipMease, M.D., Chief, Rheumatology Clinical Research Division of SwedishHospital Medical Center and Head of Seattle Rheumatology Associates, whopresented the data.
127 patients with inflammatory arthritis received a single intra-articularinjection of either tgAAC94 or placebo, followed by a second injection 12-30weeks later depending on when the injected joint met criteria forre-injection. tgAAC94 was well tolerated at all dose levels. Two SAEs offocused interest were reported -- a culture-negative septic arthritisconsidered probably related to study drug and a fatal disseminatedhistoplasmosis and retroperitoneal hemorrhage considered not related to studyagent. Administration site reactions were observed in 12% of subjects.
Preliminary signs of efficacy were also observed in the study, mostnotably a 30% decrease in patient-reported pain and functional scores asmeasured by the global visual analog scale (VAS) in the tgAAC94-treated armversus the placebo arm. Global VAS is a clinically meaningful and commonlyused assessment tool to help patients describe the intensity of their pain andfunctional capacity.
About Targeted Genetics Corporation
Targeted Genetics Corporation is a biotechnology company committed to thedevelopment of innovative targeted molecular therapies for the prevention andtreatment of acquired and inherited diseases with significant unmet medicalneed. Targeted Genetics' proprietary Adeno-Associated Virus (AAV) technologyplatform allows it to deliver genes that encode proteins to increase genefunction or RNAi to decrease or silence gene function. Targeted Genetics'product development efforts target inflammatory arthritis, AIDS prophylaxis,congestive heart failure and Huntington's disease. To learn more aboutTargeted Genetics, visit Targeted Genetics' website athttp://www.targetedgenetics.com.
Safe Harbor Statement under the Private Securities Litigation Reform Actof 1995:
This release contains forward-looking statements regarding the Company'sbusiness strategy and product development, including statements regarding thedata collected in the inflammatory arthritis study, the potential efficacy oftgAAC94, the potential impact of the study on our results of operations andother statements about the Company's plans, objectives, intentions andexpectations. These statements involve current expectations, forecasts offuture events and other statements that are not historical facts. Inaccurateassumptions and known and unknown risks and uncertainties can affect theaccuracy of forward-looking statements. Factors that could affect actualfuture events or results include, but are not limited to, payments anticipatedby the Company under product development collaborations and contracts, theCompany's actual expenses, the Company's ability to raise capital when needed,the timing, nature and results of the Company's clinical trials, potentialdevelopme