Full Press Release Details
| Stacie D. Byars | Carolyn Wang |
| Director, Communications | WeissComm Partners |
| Targeted Genetics Corporation | 415.225.5050 |
| 206.521.7392 |
ON RECOMBINANT DNA ADVISORY COMMITTEE (RAC) ASSESSMENT OF ITS PHASE I/II
FOR INFLAMMATORY ARTHRITIS
Genetics Corporation (Nasdaq: TGEN) today reported on the National Institutes
Health (NIH) Recombinant DNA Advisory Committee's (RAC) assessment of the
previously reported fatal serious adverse event (SAE) in the Phase I/II trial
tgAAC94 for inflammatory arthritis.
Committee's opinion is that the subject's death was a result of complications
from an opportunistic infection, disseminated histoplasmosis, which caused
study participant to experience a retroperitoneal hematoma and multi-organ
failure. Further, the RAC observed that
apparent risk factor that may have had an impact on the patient's susceptibility
to the infection was the patient's longstanding systemic treatment for
rheumatoid arthritis with a TNF antagonist, adalimumab. The
indicated there was insufficient data available to determine if the patient
have had an immune response to the local injection of tgAAC94 as a part of
clinical trial in which she was participating. The panel also observed, however,
that if there was an immune response to the intra-articular injection of
very unlikely to have been a significant contributor, if at all, to the
subject's clinical course and was not the cause of her death.
October 23, 2007, upon comprehensive review of all available data, which
also presented at the American College of Rheumatology Annual Meeting on
November 10, the study's independent data safety monitoring board concluded that
the SAE was not related to the tgAAC94 product. In addition, as recently
reported, the U.S. Food and Drug Administration (FDA) also released the trial
from clinical hold, permitting the clinical trial to resume.
appreciate the collaborative efforts of the parties involved in the review
the thorough analysis of the available data," said H. Stewart Parker, president
and chief executive officer of Targeted Genetics. "Over
next several weeks, we will work with trial sites to obtain approval from
Institutional Review Boards and reconsent the patients eligible to receive
in this trial are followed for 30 weeks after the second injection and during
this time, to increase the data set, blood will be collected to test for
distribution and immune response, if any, to vector. Similar tests were done
the dose-escalation phase of the study.
ongoing 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
the knee, ankle, wrist, metacarpophalangeal or elbow, and 74 subjects out
total 127 have received a second dose of active drug. Of those 74 subjects,
have received two doses of active drug.
Company recently reported interim data from 66 subjects enrolled in the Phase
portion of the trial at the ACR meeting. A higher percentage of subjects
received tgAAC94 reported improvement in joint symptoms, function and pain
compared to the placebo injected group. These interim data also indicated
tgAAC94 is well-tolerated for doses up to 5x10(13) DRP. The most common adverse
events noted were injection site reactions, seen in 10% of patients treated.
arthritis is a serious disease affecting more than two million people in
United States and can lead to debilitating chronic pain, permanent nerve,
and joint damage, and, in some cases, death. While medications are available
slow and sometimes stop symptoms and progression of the disease, they can
compromise the immune system and leave patients with side effects, such as
chronic infections. The Company's tgAAC94 product candidate, a locally targeted
therapy approach, is designed to reduce inflammation locally in the joints
avoid the potential for systemic side effects.
is being developed for use in patients with inflammatory arthritis who have
or few inflamed joints and are not candidates for systemic TNF-antagonist
therapy, or as a supplemental therapeutic for patients on systemic
anti-TNF-alpha protein therapy who 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
TNF-alpha receptor (TNFR: Fc). Soluble TNFR:Fc inhibits the 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
Genetics Corporation is a biotechnology company committed to the development
innovative targeted molecular therapies for the prevention and treatment
acquired and inherited diseases with significant unmet medical need. Targeted
Genetics' proprietary Adeno-Associated Virus (AAV) technology platform allows
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,
Targeted Genetics' website at www.targetedgenetics.com.
Harbor Statement under the Private Securities Litigation Reform Act of 1995:
release contains forward-looking statements regarding the continuation and
results of Targeted Genetics' clinical trial of tgACC94, the data collected in
the tgAAC94 program, the cause of the serious adverse event, establishment
determination of efficacy endpoints from the data collected in the trial,
the timely and complete accrual of patients in the trial. 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 actual future events or results include, but are
limited to: the timing, nature and results of the Company's clinical trials;
Company's ability to obtain and maintain regulatory or institutional approvals;
and the Company's ability to obtain, maintain and protect its intellectual
property, as well as other risk factors described in "Item 1A. Risk Factors"
the Company's most recent annual report on Form 10-K for the year ended December
31, 2006 filed with the SEC and in its most recently filed quarterly report
Form 10-Q for the quarter ended September 30, 2007. You should not rely unduly
on these forward-looking statements, which apply only as of the date of this
release. The Company undertakes no duty to publicly announce or report revisions
to these statements as new information becomes available that may change
Company's expectations.