Targeted Genetics Announces Preliminary Results of Phase I
Clinical Trial of tgAAC94 in Patients With Inflammatory Arthritis
- Results Demonstrate Safety and Support Continued Study of tgAAC94 in
Conjunction With TNF-alpha Antagonists in Inflammatory Arthritis -
SEATTLE, July 26 /PRNewswire-FirstCall/ -- Targeted Genetics Corporation
(NASDAQ:TGEN) today announced that the preliminary results from its Phase I
clinical trial of tgAAC94 in patients with inflammatory arthritis demonstrate
that the experimental drug is well tolerated and can be safely administered
directly into joints of patients with inflammatory arthritis. Preliminary data
suggest improvements in signs and symptoms of arthritis in the treated joints.
The trial conducted in the United States and Canada was a multi-center trial of
tgAAC94 injected locally into the arthritic joint. Fifteen patients who
enrolled in the trial were randomized to receive either one of two escalating
dose levels of tgAAC94 (n=11) or a placebo (n=4). The trial contained a
placebo arm at each dose level, which was included primarily to assess whether
any adverse events that might be seen were attributable to an intra-articular
injection itself, as opposed to an intra-articular injection of tgAAC94.
The primary objective of the study was to evaluate safety of intra-articular
injection of tgAAC94 in patients not on concomitant systemic tumor necrosis
factor alpha (TNF-alpha. antagonist therapy. The data demonstrate that tgAAC94
was well-tolerated at doses up to 1x10(11) DRP per mL of joint volume. Data
was also collected on secondary parameters including improvements in arthritis
signs and symptoms in the injected joint as measured by changes in joint
swelling and tenderness using standardized arthritis index scores. Although
the number of patients was small, in those treated with tgAAC94 and followed
for four weeks, there was an indication of sustained improvement in signs and
symptoms in nine of the eleven subjects. From continued follow up, the
preliminary data also indicate that seven out of nine patients who received
tgAAC94, and who have been evaluated through week eight following treatment,
experienced sustained improvement in signs and symptoms of disease. In those
subjects receiving placebo, improvements in arthritis signs were noted in two
out of four subjects.
The trial has now been closed for enrollment and patients will continue to be
followed for 24 weeks after injection. Additional clinical results will be
reported at the next appropriate scientific venue.
"The preliminary results of this trial represent a key milestone in the
development of our inflammatory arthritis program," said H. Stewart Parker,
president and chief executive officer of Targeted Genetics. "Based on the
results of this trial, we expect to advance the development of tgAAC94 into the
next stage of clinical development; including localized treatment of patients
who are concurrently receiving systemic anti-TNF therapies, but who continue to
suffer from one or more affected joints. We expect to begin the next clinical
trial of tgAAC94 in the third quarter of this year." Over the past decade, advances in inflammatory arthritis drug discovery and
development have identified TNF-alpha as a key mediator of disease-related
inflammation and tissue damage. While anti-TNF-alpha therapies are now widely
used in the treatment of inflammatory arthritis, there are a number of patients
taking systemic anti-TNF-alpha therapies who do not fully respond to those
therapies and still have one or several affected joints. tgAAC94 is an
AAV-based product containing the gene for the soluble TNF-alpha receptor
protein. tgAAC94 is delivered directly to the affected joint where it should
express the soluble TNF-alpha receptor protein locally and on a sustained
basis.
"In spite of advances in treatment options, particularly the use of systemic
anti-TNF-alpha therapies, a significant number of patients with inflammatory
arthritis do not experience an adequate response and have ongoing destructive
inflammation in select joints. These patients are potentially ideal candidates
for a localized, more concentrated delivery of anti-TNF-alpha therapy to the
joint," said Philip Mease, M.D., Chief, Rheumatology Clinical Research Division
of Swedish Hospital Medical Center, Head of Seattle Rheumatology Associates, and
a lead investigator in this clinical trial. "Though the patient numbers are
relatively small and the study is not powered to show efficacy, I am encouraged
that delivery of tgAAC94 directly into affected joints appears to be safe and
has the potential to reduce signs and symptoms of arthritis. I am excited to
begin additional studies using Targeted Genetics' interesting approach to
address this ongoing treatment challenge." In June 2004, the Company reported that in preclinical studies, rAAV-rat
TNFR:Fc was delivered to the joints of rats with experimentally induced
arthritis. The data from these studies demonstrated that a single injection
of rAAV-rat TNFR:Fc vector into the ankles of arthritic rats resulted in a
significant reduction in ankle and hind paw swelling as measured by arthritis
index scores. The data also indicated that animals treated in a single joint
experienced a reduction in swelling in the untreated joint as well as the
treated joint. This was observed in the absence of significant levels of
circulating TNFR:Fc protein. Preclinical evaluation of safety showed that
rAAV-TNFR:Fc was safe and well tolerated.
About tgAAC94 and AAV technology tgAAC94 uses Targeted Genetics Corporation's rAAV (recombinant AAV) vector
technology and contains a gene that encodes the soluble TNF-alpha receptor. The
soluble TNF-alpha receptor is a TNF-alpha inhibitor. tgAAC94 is delivered
directly to affected joints to induce the patient's own joint cells to produce
local concentrations of the TNF-alpha inhibitor to reduce inflammation
associated with disease. tgAAC94 is being developed as a potential supplement
to systemic anti-TNF-alpha protein therapy for use in patients with
inflammatory arthritis where one or several joints do not respond to systemic
protein therapy. Local administration of a DNA sequence encoding a soluble
TNF-alpha receptor potentially may supplement currently used drugs in a number
of inflammatory conditions. In addition, a locally administered anti-TNF-alpha
therapy could also be useful in patients having a limited number of joints
impacted by inflammatory arthritis who are at a risk for progressive joint
damage but who may not require systemic therapy. The characteristics of AAV
vectors make them well suited for delivery of genetic material to joints and
other local environments. 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.
About Targeted Genetics Targeted Genetics Corporation develops molecular medicines for the prevention
and treatment of acquired and inherited diseases. The Company has clinical
product development programs targeting inflammatory arthritis and AIDS
prophylaxis. The Company also has a promising pipeline of preclinical product
development programs focused on congestive heart failure, Huntington's disease,
and hyperlipidemia. For more information about Targeted Genetics, visit its
website at http://www.targetedgenetics.com/.
Safe Harbor Statement under the Private Securities Litigation Reform Act of
1995: This release contains forward-looking statements regarding our intellectual
property, research programs and clinical trials, our product development and
our potential development platforms including tgAAC94 and other statements
about our plans, objectives, intentions and expectations. In particular, the
statements regarding the Company's pipeline, ability to maintain patients in
the trial for follow-up and future clinical trial plans are forward-looking
statements. 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 our actual
results include, but are not limited to, initial trial results not indicative
of results from the completion of the trial, the timing, nature and results of
our clinical trials, potential development of alternative technologies or more
effective products by competitors, our ability to obtain and maintain
regulatory or institutional approvals, our ability to obtain, maintain and
protect our intellectual property and our ability to raise capital when needed,
as well as other risk factors described in the section entitled "Factors
Affecting Our Operating Results, Our Business and Our Stock Price" in our
Quarterly Report on Form 10-Q for the period ended March 31, 2005. 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.
Contact: Stacie D. Byars, Director, Communications of Targeted Genetics
Corporation, +1-206-521-7392. DATASOURCE: Targeted Genetics Corporation CONTACT: Stacie D. Byars, Director, Communications of Targeted Genetics Corporation, +1-206-521-7392 Web site: http://www.targetedgenetics.com/
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