Full Press Release Details
Genetics Corporation
GENETICS ANNOUNCES THAT FDA HAS REMOVED HOLD ON PHASE I/II CLINICAL TRIAL
tgAAC94 FOR INFLAMMATORY ARTHRITIS
II Clinical Trial Protocol Design Underway
Genetics Corporation (Nasdaq: TGEN) today announced that the U.S. Food and
Administration (FDA) removed the hold on the Company's Phase I/II clinical trial
of tgACC94 for inflammatory arthritis. This action follows the agency's review
of the safety data to date on all 127 subjects and all data from a fatal
adverse event (SAE), which led to a clinical trial hold in July of this year.
The Company will host
conference call and webcast discussion today beginning at 10:30 a.m. Eastern
Time / 7:30 a.m. Pacific Time. You may access the live webcast via the "Events"
section of the Company's website at www.targetedgenetics.com or via telephone
800.240.2134 (domestic) or 303.275.2170 (international).
obtained during the investigation indicates that tgAAC94 did not contribute
patient's death, which was due to disseminated histoplasmosis.
molecular test results presented earlier this month at the 71st annual meeting
of the American College of Rheumatology (ACR), supported initial observations
that no amplification of viral vector occurred in the patient's body as a
of the investigational therapy, that only trace amounts of vector DNA were
detected in tissues outside the treated joint, and that the amount of
circulating TNF-alpha antagonist protein was as expected from the background
therapy. The subject was on adalimumab,
methotrexate and prednisone, which are known to be immunosuppressive and
factor for histoplasma infection.
Genetics is revising the Phase I/II trial's informed consent to include
information about the SAE, and will amend the protocol to encompass suggestions
made by the FDA and its independent data safety monitoring board. Over the
several weeks, the Company will work with trial sites to obtain Institutional
Review Board approval of the amended protocol and revised informed consent
to re-consent the approximately 35 patients who have yet to receive the second
injection. Patients in this trial are followed for 30 weeks after the second
working closely with trial site physicians to ensure we resume development
the most efficient manner possible with patient safety, as always, of paramount
importance," said H. Stewart Parker, president and chief executive officer of
Targeted Genetics. "We anticipate having full data from the Phase I/II trial in
the second half of 2008. In the meantime, we believe we have the information
needed from this trial to simultaneously plan initiation of a Phase II trial
tgACC94 in the second-half of 2008."
II clinical trial of tgAAC94 will be designed to evaluate efficacy and duration
of response, as well as further assess safety, in patients with inflammatory
arthritis who have one or few inflamed joints because
they are not candidates for systemic therapy
do not fully respond to systemic anti-TNF protein therapy.
ongoing Phase I/II study is designed to assess the safety and potential efficacy
of different doses of tgAAC94 administered directly to affected joints of
subjects with inflammatory arthritis. Subjects 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 of active drug or placebo
the knee, ankle, wrist, metacarpophalangeal or elbow, and 74 subjects out
total 127 have received a second dose of active drug. Of those 74 subjects,
have received two doses of active drug.
Company recently reported interim data from 66 subjects enrolled in the Phase
portion of the trial at the ACR meeting. A higher percentage of subjects
received tgAAC94 reported improvement in joint symptoms, function and pain
compared to the placebo injected group. These interim data also indicated
tgAAC94 is well-tolerated for doses up to 5x10(13) DRP. The most common adverse
events noted were injection site reactions, seen in 10% of patients treated.
arthritis is a serious disease affecting more than two million people in
United States and can lead to debilitating chronic pain, permanent nerve,
and joint damage, and, in some cases, death. While medications are available
slow and sometimes stop symptoms, they can compromise the immune system and
leave patients with side effects, such as chronic infections. The Company's
tgAAC94 product candidate, a locally targeted therapy approach, is designed
reduce inflammation locally in the joints and avoid the potential for systemic
is being developed as a supplemental therapeutic to systemic anti-TNF-alpha
protein therapy for use in patients with inflammatory arthritis who have
few inflamed joints or who do not fully respond to systemic protein therapy.
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
the localized production of secreted TNFR:Fc within joint cells, reducing
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
replay will be available for approximately 30 days at www.targetedgenetics.com;
telephone replay will be available following today's call at approximately 9:30
p.m. PT through 11:59 p.m. PT, Sunday, December 23, 2007, by calling
800.405.2236 (domestic) or 303.590.3000 (international); pass code
Genetics Corporation is a biotechnology company committed to the development
innovative targeted molecular therapies for the prevention and treatment
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,
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 continuation and
results of its clinical trial of tgACC94, the data collected in the tgAAC94
program, the cause of the serious adverse event, establishment or determination
of efficacy endpoints from the data collected in the trial, and the timely
complete accrual of patients in the trial. 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 actual future events or results include, but are not limited
to, the timing, nature and results of the Company's clinical trials, the
Company's ability to obtain and maintain regulatory or institutional approvals,