Full Press Release Details
Genetics Corporation
DATA ON USE OF tgAAC94
IN THE TREATMENT OF INFLAMMATORY
AT THE AMERICAN COLLEGE OF RHEUMATOLOGY MEETING
of safety at all dose levels and trend in sustained decrease
signs and symptoms of disease in target joint -
November 13, 2006 - Targeted Genetics Corporation (Nasdaq: TGEN) announced
the principal investigator for its current clinical studies, Philip J. Mease,
M.D., Chief, Rheumatology Clinical Research Division of Swedish Hospital Medical
Center and Head of Seattle Rheumatology Associates, will present additional
interim data today from the ongoing Phase I/II trial of tgAAC94 in patients
inflammatory arthritis. Dr. Mease will present these data in a poster
presentation, titled "A Phase 1/2 Clinical Study of Intra-articular
Administration of a Recombinant Adeno-Associated
Vector Containing a TNF- Antagonist Gene in Inflammatory Arthritis,"
American College of Rheumatology meeting, taking place in Washington
continue to support the safety and tolerability of intra-articular
administration of tgAAC94 to affected joints in doses up to 1x1013
of joint fluid and suggest that tgAAC94 may lead to decrease in signs and
symptoms of arthritis in injected joints. Importantly, these decreases in
injected joint tenderness and swelling were observed in inflammatory arthritis
subjects with or without concurrent systemic TNF-alpha antagonist
patients with inflammatory arthritis fail to achieve sufficient disease control
in one or more joints and continue to experience pain and discomfort. The goal
with targeted localized therapy is to increase the amount and duration of TNF
inhibition in the problem joint to improve response and decrease the possibility
that the inflammation will lead to destruction of individual joints," said Dr.
Mease. "The safety and responses observed in clinical trials to date suggest
that tgAAC94 has the potential to advance the care of patients with inflammatory
arthritis. If tgAAC94 proves to be safe and efficacious, I can envision
individualizing treatment of patients with the combination of systemic therapy
and local therapy that works best for the patient."
is an investigational therapeutic designed to inhibit the activity of tumor
necrosis factor-alpha (TNF-alpha), a key mediator of inflammation. tgAAC94
utilizes an adeno-associated virus (AAV) vector to deliver the gene encoding
soluble form of the receptor for TNF-alpha (TNFR:Fc) directly to affected
joints. TNFR:Fc protein is a potent inhibitor of TNF-alpha.
ongoing Phase I/II study, approximately 120 adults are being randomized into
three dose levels to receive a single intra-articular injection of either
tgAAC94 or placebo, followed by an open-label injection of tgAAC94 after 12
30 weeks, depending on when swelling in the target joint meets criteria for
re-injection. As of October 2006, 61 subjects in the first three cohorts, 34
whom were receiving concurrent TNF-alpha antagonists, received an injection
blinded study drug into the knee (n=30), ankle (n=10), wrist (n=11),
metacarpophalangeal joint (n=6) or elbow (n=4). These patients were
predominantly rheumatoid arthritis patients, although a small number of
psoriatic arthritis and ankylosing spondilitis patients have participated.
the favorable safety profile of the first three dose-escalation cohorts, the
Data Monitoring Committee overseeing the study has given permission for
remaining 60 subjects to be enrolled at the same three dose levels to increase
the power of the study.
Stewart Parker, President and Chief Executive Officer of Targeted Genetics
added, "We are encouraged by the continued positive trends in this additional
data and pleased to have the opportunity to share the results of our tgAAC94
clinical studies with members of the medical community. We believe that our
unique approach for localized delivery of a protein therapeutic holds the
promise of more comprehensive treatment for patients suffering with inflammatory
is being developed as a potential supplement 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 Company's rAAV technology platform is used to deliver genes and
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
commercialization of innovative, targeted molecular therapies for the prevention
and treatment of inflammatory arthritis, HIV/AIDS and other acquired and
inherited diseases with significant unmet medical need. Targeted Genetics uses
its considerable knowledge and capabilities in the development and manufacturing
of gene delivery technologies to advance a diverse product development pipeline.
Its product development efforts target inflammatory arthritis, HIV/AIDS,
congestive heart failure, Huntington's disease, and hyperlipidemia. To learn
more about Targeted Genetics, visit its website at
Harbor Statement under the Private Securities Litigation Reform Act of
release contains forward-looking statements regarding the data to be collected
in this trial, the establishment or determination of efficacy endpoints from
data collected in the trial, the timely and complete accrual of subjects in
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
not historical facts. Inaccurate assumptions and known and 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 and enroll suitable trial
participants, the timing, nature and results of research and clinical trials,
potential development of alternative technologies or more effective processes
competitors, and, our ability to obtain and maintain regulatory or institutional
approvals, as well as other risk factors described in Item 1A. Risk Factors
our report on Form 10-K for the year ended December 31, 2005 and updated in
1A. Risk Factors in our Form 10-Q for the quarter ended March 31, 2006. You
should not rely unduly on these forward-looking statements, which apply only
of the date of this release. We undertake no duty to publicly announce or report