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Targeted Genetics Reports on Recombinant DNA Advisory Committee (RAC) Review of its Phase 1/2 Trial of tgAAC94 for Rheumatoid Arthritis |
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18 Sep 2007 |
Targeted Genetics Corporation today reported on the public hearing conducted by the National Institutes of Health Recombinant DNA Advisory Committee
SEATTLE, WA, USA | September 17, 2007 | Targeted Genetics Corporation (Nasdaq: TGEN) today reported on the public hearing conducted by the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC), which reviewed the serious adverse event reported by Targeted Genetics surrounding the death of a patient participating in the Company's Phase I/II trial of tgAAC94 for inflammatory arthritis.
The clinical case was presented for the first time in a comprehensive manner and showed that histoplasmosis played a significant role in the cause of the patient's death. Initial molecular tests showed there was no replication of vector and only trace amounts of vector DNA in tissues outside the joint. Consequently, these data suggest it is unlikely that tgAAC94 contributed to the conditions that caused the death. Molecular tests are being conducted in remaining tissues and Targeted Genetics will continue to collaboratively work with academic colleagues, RAC, FDA and other involved parties to complete the investigation.
"While additional tests are needed to draw final conclusions, we believe the results to date are consistent with preclinical and clinical findings that indicate the level of vector that is present outside the locally treated area is insufficient to have further exacerbated an infection," said H. Stewart Parker, president and chief executive officer of Targeted Genetics.
Barrie J. Carter, Ph.D., executive vice president and chief scientific officer of Targeted Genetics added, "A gene therapy approach may have enormous potential to improve the treatment of rheumatoid arthritis, a disease that leads to profound morbidity and premature mortality. It is critical that we let the clinical trial and scientific process determine the risks and potential of gene therapy before rushing to judgment and hampering the development of what could one day play a significant role in the treatment of serious diseases."
About Histoplasmosis
Histoplasmosis is a fungal infection resulting from exposure to spores of the microscopic fungus, Histoplasma capsulatum. Clinical manifestations can vary from a mild flu-like illness that may not produce any noticeable symptoms to rapidly progressive, sometimes fatal, disseminated disease. The degree of symptoms experienced from this infection can be highly variable depending on a number of factors including the relative strength of the infected person's immune system. Many of the medications commonly prescribed to patients undergoing treatment for inflammatory arthritis, including those that were being taken by the patient, are recognized to have immunosuppressant effects.
About tgAAC94 and the Phase I/II Study
tgAAC94 is being developed as a supplemental therapeutic to systemic anti-TNF-alpha protein therapy for use in patients with inflammatory arthritis who have one or more joints that do not fully respond to systemic protein therapy. The product candidate uses Targeted Genetics' recombinant AAV (rAAV) vector technology to deliver a DNA sequence that encodes a soluble form of the TNF-alpha receptor (TNFR: Fc). Soluble TNFR:Fc inhibits the immune stimulating activity of TNF-alpha. Direct injection of tgAAC94 into affected joints leads to the localized production of secreted TNFR:Fc within joint cells, reducing the activity of TNF-alpha within the joint and, potentially, leading to a decrease in the signs and symptoms of inflammatory disease and inhibition of joint destruction.
The Phase I/II study is designed to assess the safety and potential efficacy of different doses of tgAAC94 administered directly to affected joints of subjects with inflammatory arthritis. Subjects already enrolled in the study will continue to be followed and monitored. Since the trial began in October 2005, 127 subjects have received an initial dose of active drug or placebo into the knee, ankle, wrist, metacarpophalangeal or elbow, and 74 subjects out of the total 127 have received a second dose of active drug. Of those 74 subjects, 55 have received two doses of active drug.
About Targeted Genetics
Targeted Genetics Corporation is a biotechnology company committed to the development of innovative targeted molecular therapies for the prevention and treatment of acquired and inherited diseases with significant unmet medical need. Targeted Genetics' proprietary Adeno-Associated Virus (AAV) technology platform allows it to deliver genes that encode proteins to increase gene function 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 about Targeted Genetics, visit Targeted Genetics' website at http://www.targetedgenetics.com.
SOURCE: Targeted Genetics Corporation |
