Full Press Release Details
Targeted Genetics Corporation
TARGETED GENETICS ANNOUNCES PRELIMINARY SAFETY DATA FROM PHASE I HIV VACCINE TRIAL
Seattle, WA February 22, 2005 Targeted Genetics Corp. (Nasdaq: TGEN) today reported preliminary
results from a Phase I trial of tgAAC09, an investigational recombinant adeno-associated viral
vector (rAAV)-based HIV/AIDS vaccine candidate, enrolling healthy volunteers who are not infected
with HIV. The trial is being conducted in Belgium, Germany and India.
The Phase I trial is the first ever to test a rAAV-based vaccine and is primarily designed to
evaluate safety and tolerability of the vaccine at escalating dose levels. The study is also
designed to evaluate immune responses following vaccination. No safety concerns were identified
and the vaccine was well tolerated. In addition, a single administration of the vaccine at the
doses evaluated in this initial study did not elicit significant immune responses.
The favorable tolerability data from this trial are consistent with our growing clinical
experience with AAV and now position us to continue the clinical development of this vaccine
program. We can begin to evaluate higher dose levels and repeat administration of this vaccine,
said H. Stewart Parker, President and Chief Executive Officer of Targeted Genetics. In addition,
our comprehensive development strategy for this AIDS vaccine includes the development of an
AAV1-based vaccine, as well as a multi-component approach involving multiple HIV antigens. We are
also examining in preclinical studies the use of AAV vaccines in a prime boost approach, to
potentially increase immune responses to the HIV antigens further. In short, we continue to be
optimistic about the potential of AAV-based vaccines against HIV/AIDS.
The Phase I clinical trial is a double-blind, placebo-controlled, dose-escalation safety study that
also monitors immune responses to the product candidate. The portion of the study conducted in
Belgium and Germany enrolled 50 volunteers who were in good general health and not infected with
HIV. Each volunteer received a single intramuscular injection into the upper arm. The portion of
the trial being conducted in India will enroll 30 healthy HIV-negative volunteers. A subset of
volunteers also will receive a second dose of the vaccine to determine if repeat dosing is safe,
and if it increases immune responses
The phase I trial for tgAAC09 is being studied in collaboration with the not-for-profit
International AIDS Vaccine Initiative and researchers at Columbus Children s Research Institute and
The Children s Hospital of Philadelphia.
About Targeted Genetics
Targeted Genetics Corporation develops gene-based products for preventing and treating acquired and
inherited diseases. The Company has three clinical product development programs, targeting cystic
fibrosis, AIDS prophylaxis and inflammatory arthritis. The Company also has a promising pipeline of
product candidates focused on hyperlipidemia, congestive heart failure and Huntington s disease
that are being developed under collaboration agreements with others, and a broad platform of gene
delivery technologies for application in nucleic acid-based drug development. For more information
about Targeted Genetics, visit its website at www.targetedgenetics.com.
Safe Harbor Statement under the Private Securities Litigation Reform Act of 1995:
This release contains forward-looking statements regarding our research programs, clinical trials,
product development and potential related to tgAAC09 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, the risk that future
clinical results could negatively differ from those announce today, the timing, nature and results
of our research and our clinical trials, our preclinical results with animals are not necessarily
indicative of results that will be obtained in humans, our ability to obtain and maintain
regulatory or institutional approvals, our ability to obtain, maintain and protect our intellectual
property related to tgAAC09, and our ability to raise capital when needed, as well as other risk
factors described in the section entitled Factors Affecting Our Operating Results, Our Business
and Our Stock Price in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2004.
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.