Full Press Release Details
Genetics Corporation
GENETICS RESPONDS TO WASHINGTON POST ARTICLE
6, 2007 - Targeted Genetics Corporation (Nasdaq: TGEN) responded to an article
in today's Washington Post with respect to its development program of tgAAC94,
an investigational therapy for the treatment of inflammatory arthritis. The
Company believes that it is unfortunate that the article seems designed to
agitate as opposed to inform. The incident central to the article currently
being assessed by the Company and the FDA, and the Company believes that it
would be premature as well as irresponsible to draw any conclusions prior to
completion of that investigation.
Company and the FDA stopped the trial in a timely manner due to uncertainty
the cause of a serious adverse event (SAE), but as has been stated previously,
there is currently insufficient information to draw conclusions as to the cause.
This patient's course of illness is not consistent with the Company's previous
clinical experience in the 130 patients who have received tgAAC94 to date;
is it consistent in the more than 300 patients that have been treated in the
Company's AAV clinical trials in total.
safety is of paramount importance and we have designed and conducted all of
trials with that in mind. We are confident in the safety of our product and
platform and look forward to the opportunity to complete the clinical trials,
which is the only way to determine the efficacy and safety profile of this
any other drug candidate," said H. Stewart Parker, president and chief executive
officer of Targeted Genetics.
us at Targeted Genetics are deeply saddened by the unexpected death of this
patient, and while our ability to communicate around this event is limited
both patient privacy and FDA regulations, we are committed to being as
transparent as possible with respect to our activities prior to the patient's
death and to sharing additional information as it becomes available," added
Safety Chronology of Reporting
shared a very detailed chronology of our analyses and reporting activities
regarding this particular study patient with the reporter from the Washington
Post, which demonstrated how we acted in strictest compliance with today's best
clinical trial practices, and which was not included in the article," said
February, 2007, the patient was enrolled into the clinical trial for tgAAC94,
and received the first dose. The patient received a second dose of tgAAC94
clinical investigator reported to Targeted Genetics that two patients had
nausea, fever and vomiting. These are not uncommon events and were recorded
adverse events (AEs). The investigator advised that these adverse events were
not related to study drug. Consistent with FDA regulatory procedures and the
study protocol, these adverse events were noted and will be reported to the
in the study's annual report.
Targeted Genetics was notified that this patient had been hospitalized, which
considered a serious adverse event (SAE). The investigator reported at this
that the SAE was unlikely related to study drug, and the event was noted and
held for submission in the annual report. The second patient's symptoms had
investigator notified us that the patient's condition was deteriorating, and
reiterated that the SAE was unrelated to study drug. Although this information
did not trigger a reporting requirement to the FDA, the Company proactively
notified the chairman of its independent data safety monitoring board, or DSMB,
about the situation on July 18th.
Company notified the full DSMB. After a comprehensive analysis in concert with
the DSMB, due to the temporal relationship between dosing and the onset of
event, the Company decided to report the SAE to the FDA as possibly related
FDA requirements are that SAEs possibly related to study drug must be reported
within a week, the Company held a teleconference on July 20th
FDA and the clinical investigator involved in this case, within 24 hours of
making the determination that the SAE was possibly related to drug. The trial
was officially put on hold and all study sites were notified. The official
safety report was filed on July 23rd.
Safety Reported to Date
Phase I/II study, 127 adults have been randomized into three dose levels to
receive a single intra-articular injection of either tgAAC94 or placebo into
knee, ankle, wrist, metacarpophalangeal or elbow, followed by an open-label
injection of tgAAC94 after 12 to 30 weeks, depending on when arthritis symptoms
in the target joint meet criteria for re-injection.
interim data reported by our investigators at leading scientific meetings in
June 2006, November 2006 and June 2007, support the safety and tolerability
single and repeat intra-articular injections of tgAAC94 to affected joints
doses up to 1x10(13) DNase Resistant Particles per milli-liter (DRP/mL) of
fluid in subjects with and without systemic TNF-alpha antagonists. This interim
data also suggest that treatment with tgAAC94 may lead to improvements in signs
and symptoms of arthritis in injected joints.
is being developed as a supplemental therapeutic to systemic anti-TNF-alpha
protein therapy for use in patients with inflammatory arthritis who have one
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 it
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.
Harbor Statement under the Private Securities Litigation Reform Act of 1995:
release contains forward-looking statements regarding the data to be collected
in this trial, the cause of the SAE and its impact, if any, on the timing,
continuance or results of this 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