Targeted Genetics
Corporation (Nasdaq: TGEN) reported on the public hearing conducted
by the National Institutes of Health (NIH) Recombinant DNA Advisory
Committee (RAC), which reviewed the serious adverse event reported by
Targeted Genetics surrounding the death of a patient participating in the
Company's Phase I/II trial of tgAAC94 for inflammatory arthritis.
The clinical case was presented for the first time in a comprehensive
manner and showed that histoplasmosis played a significant role in the
cause of the patient's death. Initial molecular tests showed there was no
replication of vector and only trace amounts of vector DNA in tissues
outside the joint. Consequently, these data suggest it is unlikely that
tgAAC94 contributed to the conditions that caused the death. Molecular
tests are being conducted in remaining tissues and Targeted Genetics will
continue to collaboratively work with academic colleagues, RAC, FDA and
other involved parties to complete the investigation.
"While additional tests are needed to draw final conclusions, we
believe the results to date are consistent with preclinical and clinical
findings that indicate the level of vector that is present outside the
locally treated area is insufficient to have further exacerbated an
infection," said H. Stewart Parker, president and chief executive officer
of Targeted Genetics.
Barrie J. Carter, Ph.D., executive vice president and chief scientific
officer of Targeted Genetics added, "A gene therapy approach may have
enormous potential to improve the treatment of rheumatoid arthritis, a
disease that leads to profound morbidity and premature mortality. It is
critical that we let the clinical trial and scientific process determine
the risks and potential of gene therapy before rushing to judgment and
hampering the development of what could one day play a significant role in
the treatment of serious diseases."
About Histoplasmosis
Histoplasmosis is a fungal infection resulting from exposure to spores
of the microscopic fungus, Histoplasma capsulatum. Clinical manifestations
can vary from a mild flu-like illness that may not produce any noticeable
symptoms to rapidly progressive, sometimes fatal, disseminated disease. The
degree of symptoms experienced from this infection can be highly variable
depending on a number of factors including the relative strength of the
infected person's immune system. Many of the medications commonly
prescribed to patients undergoing treatment for inflammatory arthritis,
including those that were being taken by the patient, are recognized to
have immunosuppressant effects.
About tgAAC94 and the Phase I/II Study
tgAAC94 is being developed as a supplemental therapeutic 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 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 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 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 into the knee, ankle, wrist, metacarpophalangeal or elbow,
and 74 subjects out of the total 127 have received a second dose of active
drug. Of those 74 subjects, 55 have received two doses of active drug.
About Targeted Genetics
Targeted Genetics Corporation is a biotechnology company committed to
the development of 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 targetedgenetics.
Safe Harbor Statement under the Private Securities Litigation Reform
Act of 1995:
This release contains forward-looking statements regarding the data to
be collected in this trial, the cause of the SAE and the impact, if any, on
the timing, continuance or results of this trial, establishment or
determination of efficacy endpoints from the data collected in the trial,
the timely and complete accrual of patients in the trial and our ability to
commercialize tgAAC94 and other statements about our plans, objectives,
intentions and expectations. 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, our ability to obtain, maintain and protect our intellectual property,
our ability to raise capital when needed, our ability to recruit and enroll
suitable trial participants, the timing, nature and results of research and
clinical trials, potential development of alternative technologies or more
effective processes by competitors, and, our ability to obtain and maintain
regulatory or institutional approvals, as well as other risk factors
described in Item 1A. Risk Factors in our report on Form 10-K for the year
ended December 31, 2006 and updated in Item 1A. Risk Factors in our Form
10-Q for the quarter ended June 30, 2007. 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.
Targeted Genetics Corporation
targetedgenetics
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