Full Press Release Details
Genetics Corporation
GENETICS PROVIDES UPDATE ON
ARTHRITIS PHASE I/II TRIAL
26, 2007 - Targeted Genetics Corporation (Nasdaq: TGEN) provided an update
on its development program of tgAAC94, an investigational therapy for the
treatment of inflammatory arthritis. This Phase I/II study is designed to assess
the safety and potential efficacy of different doses of tgAAC94 administered
directly into affected joints of subjects with inflammatory arthritis. As we
previously announced on July 24, the Company and the United States Food and
Administration (FDA) stopped the trial as a precautionary measure after the
occurrence of a Serious Adverse Event (SAE) in one subject. The individual
experienced this SAE has subsequently died.
deeply saddened by the death of an individual enrolled in our clinical
trial. The welfare of the trial participants is always our foremost
consideration and concern," said H. Stewart Parker, president and chief
executive officer of Targeted Genetics.
adds, "The clinical course that this individual experienced has, to our
knowledge, never been seen as a consequence of exposure to adeno-associated
viral (AAV) vectors or naturally occurring AAV. We continue to work
closely and diligently with the FDA and the study's independent Data Safety
Monitoring Board to determine the cause of the SAE as quickly as possible."
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, 74 subjects out of the total 127 have received a second
dose of active drug, and of those 74 subjects, 55 have received two doses of
is an investigational therapy utilizing an adeno-associated virus (AAV) vector
to deliver the gene encoding a soluble form of the receptor for TNF-alpha
(TNFR:Fc). The TNFR:Fc protein is an inhibitor of tumor necrosis factor-alpha
(TNF-alpha), a key mediator of inflammation. In March 2006, the Company received
approval from the FDA to amend its protocol for the tgAAC94 clinical trial
include a higher dose group and increase the number of patients. 127 adults
been randomized into three dose levels to receive a single intra-articular
injection of either tgAAC94 or placebo into the knee, ankle, wrist,
metacarpophalangeal or elbow, followed by 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.
Company's abstract titled, "Local Treatment for Inflammatory Arthritis: A Phase
1/2 Clinical Study of Intra-Articular Administration of a Recombinant
Adeno-Associated Vector Containing a
recently been accepted for presentation at the American College of Rheumatology
Annual Scientific Meeting in Boston, November 2007. The
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. These 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.