Full Press Release Details
Genetics Corporation
GENETICS REPORTS ADDITIONAL DATA FROM
ARTHRITIS CLINICAL TRIAL
4, 2007 - Targeted Genetics Corporation (Nasdaq: TGEN) today announced
additional data from the dose escalation arm of the ongoing Phase I/II clinical
study of tgAAC94 in patients with inflammatory arthritis. Philip J. Mease,
professor at the University of Washington School of Medicine, chief of
rheumatology clinical research at the Swedish Medical
Center, practicing physician with Seattle Rheumatology Associates and
investigator on the trial presented the data in an oral presentation yesterday
Meeting of the American Society of Gene Therapy in Seattle (ASGT).
aggregate data on the primary and secondary end-points were presented from
first 61 subjects in the dose escalation portion (cohorts 1 to 3) of the Phase
I/II clinical trial of tgAAC94 in patients with inflammatory arthritis.
Completion of enrollment and initial dosing of all subjects in the second
segment (cohorts 4 to 6) of this trial was announced recently. tgAAC94 is an
investigational therapeutic designed to inhibit the activity of tumor necrosis
factor-alpha (TNF-alpha), a key mediator of inflammation. The Phase I/II study
is designed to assess the safety and potential effect of different doses of
tgAAC94 administered directly to affected joints of subjects with inflammatory
arthritis with or without concurrent use of systemic TNF antagonist therapies.
Stewart Parker, president and chief executive officer of Targeted Genetics
"The rapid enrollment in this study, which was completed last month, underscores
the need for additional treatments for inflammatory arthritis. We believe that
tgAAC94 has significant potential to address disease that remains refractory
approved therapies, and are encouraged by the data suggesting that tgAAC94
reduce signs and symptoms of inflammatory arthritis."
dose escalation portion of the study, three cohorts of 20 subjects were
randomized to receive tgAAC94 (n = 15) or placebo (n = 5). Aggregate data on
primary and secondary end-points from the first 61 subjects support the safety
and tolerability of single and repeat intra-articular injections of tgAAC94
affected joints at doses up to 1x10(13) DNase Resistant Particles per
milli-liter (DRP/mL) of joint volume in subjects with and without systemic
TNF-alpha antagonists. Transient injection administration site reactions
consisting of mild to moderate increases in joint tenderness and swelling were
noted in 10% of the subjects overall. At week 12 after treatment with tgAAC94,
13%, 14% and 33% of subjects receiving low, mid and high dose tgAAC94,
respectively, achieved a two-point reduction in swelling compared to none in
placebo group. A trend in reduction of swelling in tgAAC94-injected joints
compared to placebo was also observed in subjects with or without concurrent
of systemic TNF antagonist.
of the cohorts, all subjects receive a dose of tgAAC94 at 12 weeks or later
after first injection. The criteria for timing of the second injection are:
improvement in swelling 12 weeks after first injection; swelling returns to
baseline levels after an initial improvement 12 or more weeks after first
injection; or more than 30 weeks have passed since first injection. According
these criteria, a similar proportion of subjects did not need the second
injection prior to week 30 in all dose groups and placebo. Following the second
injection, subjects are followed for an additional 30 weeks.
results suggest that tgAAC94 has the potential to improve disease symptoms
are refractory to other therapies, including systemic TNF antagonists," said Dr.
Mease. "Data from the remaining cohorts and longer-term follow up of all study
subjects should provide important insight into the role that tgAAC94 may play
the treatment of inflammatory arthritis. Additional treatment options are
essential for allowing all patients with inflammatory arthritis to achieve
optimal relief of their symptoms."
randomized in the second segment (cohorts 4 to 6) of this Phase I/II study
currently being followed for safety and improvement in swelling of injected
joints as well as additional measures aimed to assess functional improvement
treated joints. MRI will also be performed on treated joints in a subset of
subjects. Data from cohorts in the Phase II segment are anticipated to provide
additional insight into clinical observations and duration of response, as
as correlate clinical observations with image-based assessments of disease
severity and response to tgAAC94. These data will guide further clinical
development and design of the subsequent Phase II study. The company expects
present additional data from the study in the second half of 2007, with complete
results anticipated in mid-2008.
I/II trial is designed to enroll 120 patients with inflammatory arthritis in
cohorts, each with 20 subjects. Within each cohort, 15 patients receive tgAAC94
and five receive placebo, administered by injection to affected joints. The
first three cohorts evaluated doses of 1x1011,
joint volume, respectively. Enrollment in these three cohorts was sequential
(e.g. cohort 1 was filled before subjects were enrolled in cohort 2, etc.).
Cohorts 4 to 6 will evaluate the same three doses, but patients will be randomly
assigned to a cohort so that all three cohorts will enroll subjects
simultaneously. This randomization is designed to ensure that each dose is
evaluated in a patient population with similar demographics, prior therapy
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
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
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.
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