Full Press Release Details
Genetics Corporation
GENETICS PRESENTS ENCOURAGING INTERIM DATA
AN ONGOING PHASE I/II TRIAL OF tgAAC94 IN PATIENTS WITH INFLAMMATORY
data show positive trends toward sustained improvement in signs and symptoms
WA and Baltimore, MD
1, 2006 - Targeted Genetics Corporation (Nasdaq: TGEND) will present interim
data from its ongoing Phase I/II trial of tgAAC94 in patients with inflammatory
arthritis today at the American Society of Gene Therapy (ASGT) 2006 Annual
Meeting. Pervin Anklesaria, Ph.D., Vice President, Therapeutic Development
at Targeted Genetics, will present highlights of initial data from the ongoing
Phase I/II and a summary of the recently completed Phase I clinical trials
tgAAC94. The data will be presented in an oral presentation, titled
"Clinical Development of an AAV Vector Expressing the TNF Receptor: Fc Fusion
Gene in Inflammatory Arthritis," as part of a workshop on musculoskeletal gene
expression and Immunology of musculoskeletal gene therapy. The
Company will hold a conference call with investors and analysts at 4:30 PM
Eastern Time today. The call will be broadcast live and can be accessed, along
with replay information, at www.targetedgenetics.com.
utilizes an adeno-associated virus (AAV) vector to deliver the gene encoding
soluble form of the receptor for tumor necrosis factor-alpha (TNFR:Fc) directly
to affected joints. TNFR:Fc protein is a potent inhibitor of tumor necrosis
factor alpha (TNF-alpha), a key mediator of inflammation.
ongoing Phase I/II study, approximately 120 adults are being randomized into
three dose groups 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. Entry criteria for this study are similar to that of the Phase
trial, except that concurrent TNF-alpha antagonist use is permitted in the
I/II trial. To date, approximately 40 subjects have received an injection of
blinded study drug or placebo.
data indicate tgAAC94 is safe and well-tolerated at doses of up to 5x10(12)
particles (DRP) /mL in subjects with and without systemic TNF-alpha antagonists.
Data from the first cohort of subjects treated with doses of 1x10(11) (DRP)/mL
of joint volume demonstrate a trend toward sustained improvement in tenderness
and swelling in treated joints, compared to placebo. Additionally, fewer
patients receiving tgAAC94 had symptoms requiring re-injection at the 12-week
time point, compared with patients in the placebo arm. Although the numbers
small, the data also suggest a trend toward greater responses to tgAAC94 in
patients taking systemic TNF-alpha antagonist therapy compared with patients
on these therapies. Additional details of the trial design and data from this
Phase I/II trial will be presented on Sunday in a separate session at the ASGT
meeting (abstract #1092).
represents a promising new approach in treating inflammatory arthritis and
are very encouraged by the data thus far from this Phase I/II study in patients
with inflammatory arthritis," 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. "We
have observed very favorable safety and toxicity profiles at the first two
levels and have seen trends toward improvement. The data suggest that a single,
intra-articular injection of tgAAC94 resulted in measurable and sustained
reductions in pain and swelling. Given the number of inflammatory arthritis
patients who still experience unresolved symptoms despite treatment with
systemic TNF-alpha antagonist therapy, there is a compelling need to explore
additional approaches to treatment, and we believe that tgAAC94 may have
significant potential in improving the long-term management of this
Stewart Parker, president and chief executive officer of Targeted Genetics
added: "We are encouraged by this interim data and believe that Targeted
Genetics' unique approach for localized delivery of a protein therapeutic may
provide patients suffering with inflammatory arthritis additional treatment
options as part of their disease management regimens. Importantly, this data
helps us to further understand the potential of this lead product opportunity,
in particular as we seek additional product opportunities in therapeutic areas
of interest that are complementary to our program in inflammatory arthritis
as we pursue ways to further leverage our investment in AAV manufacturing and
scale up, through strategic collaborations."
Anklesaria will also present the data from a completed Phase I trial of tgAAC94.
As previously reported, the safety of intra-articular administration of tgAAC94
in inflammatory arthritis was first evaluated in a Phase I study in patients
on TNF-alpha antagonists and with persistent moderate or severe inflammation
a target joint due to inflammatory arthritis. Fifteen patients (14 with
rheumatoid arthritis and 1 with ankylosing spondylitis) received a single
intra-articular injection of either tgAAC94 or placebo in two escalating dose
cohorts and were followed for 24 weeks. A trend in sustained improvement
in a composite tenderness and swelling score was noted in all treatment groups,
particularly among subjects who received the higher dose of tgAAC94. Some
improvement was noted in mean tenderness and swelling scores in subjects
receiving placebo, although the study was not powered to interpret this
call and Webcast information
company will host a conference call providing an update on the development
progress of the Company's lead clinical program in inflammatory today beginning
at 4:30 p.m. Eastern Time
p.m. Pacific Time. You may access the live webcast via the homepage of the
Company's website at www.targetedgenetics.com
telephone at 800.240.5318 (domestic) or 303.262.2138 (international).
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 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 inhibits the immune stimulating activity of TNF-alpha. Direct
injection of tgAAC94 into affected joints leads to the localized production
soluble TNFR within joint cells, reducing the activity of TNF-alpha within
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
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