Full Press Release Details
Genetics Corporation
GENETICS' INFLAMMATORY ARTHRITIS
I/II TRIAL PLACED ON CLINICAL HOLD
24, 2007 - Targeted Genetics Corporation (Nasdaq: TGEN) announced that after
recent discussions with the United States Food and Drug Administration (FDA),
the development program of tgAAC94, an investigational therapy for the treatment
of inflammatory arthritis, has been placed on clinical hold.
precautionary measure, the FDA stopped further administration of tgAAC94 due
the uncertainty of the cause of a serious adverse event (SAE) that occurred
one subject enrolled in the study. Currently, there is insufficient information
to draw conclusions as to the cause of the SAE, and therefore the study will
remain on clinical hold while additional data is gathered and analyzed. 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
active drug or placebo, and 74 subjects out of the total 127 have received
second dose of active drug.
primary concern remains that of the safety of the patients, and we are working
diligently with the study's Data Safety Monitoring Board and the FDA to
determine the cause of this unexpected event as quickly as possible," said H.
Stewart Parker, president and chief executive officer of Targeted
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. tgAAC94 is an investigational therapy that utilizes
adeno-associated virus (AAV) vector to deliver the gene encoding a soluble
of the receptor for TNF-alpha (TNFR: Fc). The TNFR:Fc protein is a potent
inhibitor of tumor necrosis factor-alpha (TNF-alpha), a key mediator of
inflammation. In March 2006, the Company received approval from the FDA to
its protocol for the tgAAC94 clinical trial to include a higher dose group
increase the number of patients. 127 adults have been randomized into three
levels to receive a single intra-articular injection of either tgAAC94 or
placebo into the knee, ankle, wrist, metacarpophalangeal or elbow, followed
an open-label injection of tgAAC94 after 12 to 30 weeks, depending on when
arthritis symptoms in the target joint meet criteria for re-injection.
interim data reported by our investigators at leading scientific meetings in
June 2006, November 2006 and June 2007, support the safety and tolerability
single and repeat intra-articular injections of tgAAC94 to affected joints
doses up to 1x10(13) DNase Resistant Particles per milli-liter (DRP/mL) of
fluid in subjects with and without systemic TNF-alpha antagonists. This interim
data also suggest that treatment with tgAAC94 may lead to improvements in signs
and symptoms of arthritis in injected joints.
is being developed as a supplemental therapeutic to systemic anti-TNF-alpha
protein therapy for use in patients with inflammatory arthritis who have one
more joints that do not fully respond to systemic protein therapy. The product
candidate uses Targeted Genetics' recombinant AAV (rAAV) vector technology
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 Company's rAAV technology platform is used to deliver genes
is based on AAV, a naturally occurring virus that has not been associated with
any disease in humans.
Genetics Corporation is a biotechnology company committed to the development
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 www.targetedgenetics.com.
Harbor Statement under the Private Securities Litigation Reform Act of 1995:
release contains forward-looking statements regarding the data to be collected
in this trial, the cause of the SAE and the impact, if any, on the timing
continuance or results of this trial, establishment or determination of efficacy
endpoints from the data collected in the trial, the timely and complete accrual
of patients in the trial and our ability to commercialize tgAAC94 and other
statements about our plans, objectives, intentions and expectations. These
statements, involve current expectations, forecasts of future events and other
statements that are not historical facts. Inaccurate assumptions and known
unknown risks and uncertainties can affect the accuracy of forward-looking
statements. Factors that could affect our actual results include, but are not
limited to, our ability to obtain, maintain and protect our intellectual
property, our ability to raise capital when needed, our ability to recruit
enroll suitable trial participants, the timing, nature and results of research
and clinical trials, potential development of alternative technologies or more
effective processes by competitors, and, our ability to obtain and maintain
regulatory or institutional approvals, as well as other risk factors described
in Item 1A. Risk Factors in our report on Form 10-K for the year ended December
31, 2006 and updated in Item 1A. Risk Factors in our Form 10-Q for the quarter
ended March 30, 2007. You should not rely unduly on these forward-looking
statements, which apply only as of the date of this release. We undertake no
duty to publicly announce or report revisions to these statements as new
information becomes available that may change our expectations.