Press Release Source: Avigen, Inc.
http://biz.yahoo.com/prnews/050719/sftu051.html?.v=18

Avigen Announces Encouraging Early Data From Parkinson's Disease Clinical
Trial
Tuesday July 19, 8:30 am ET
Evidence for First Successful Gene Transfer of AADC Gene in Humans

ALAMEDA, Calif., July 19 /PRNewswire-FirstCall/ -- Avigen, Inc. (Nasdaq:
AVGN - News), today announced encouraging results from the first patient
treated in a Phase I/II clinical trial of AV201, the Company's drug
candidate for the treatment of mid- to later-stage Parkinson's disease.
The results from positron emission tomography (PET) brain scans obtained
six months after AV201 infusion indicated an increased activity of the
gene product, aromatic L-amino acid decarboxylase (AADC) in the targeted
area of the brain, compared with the patient's pre-treatment PET scans.
These findings are consistent with increased dopamine production and
transgene expression.
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In Parkinson's disease, brain dopamine concentrations decline, causing
the main symptoms of muscle rigidity, slow movements, difficulty walking,
and poor balance. Levodopa, the first-line drug for treating symptoms of
the disease requires AADC in order to be converted to dopamine. However,
over time AADC production in the brain also declines as Parkinson's
disease progresses, making levodopa less effective. The mid- to
later-stages of Parkinson's disease are often complicated by the toxic
side effects associated with the higher doses of levodopa required to
manage the disease's signs and symptoms. AV201 is designed to restore the
activity of AADC in the brain, thereby extending the therapeutic
usefulness and life of levodopa while avoiding side effects.

The Phase I/II clinical trial of AV201 in Parkinson's disease was
initiated in December, 2004, at the University of California San
Francisco (UCSF) and Lawrence Berkeley National Laboratory (LBNL). The
study's Principal Investigator, Michael Aminoff, M.D., D.Sc., Director of
the University of California-San Francisco Parkinson's Disease Clinic &
Clinical Research Center, said, "This is an exciting beginning and a
first for us: evidence of successful AADC gene transfer into humans. Most
importantly for our patient, the procedure and gene appear to be safe.
We're pleased with how well AV201 is being tolerated." Dr. Phillip Starr,
neurosurgeon for the first patient in the study, added: "Based on the PET
scans, it appears that the procedure and the use of convection-enhanced
delivery to optimize the spread of AV201 worked as well as we could have
hoped. We are enthusiastic about enrolling additional patients."

In another first, by using the dopamine tracer [18F] fluorometatyrosine
(FMT)-based PET scanning rather than conventional fluorodopa PET,
clinicians were able to visualize evidence of AADC gene expression with
greater specificity. The use of FMT-PET technology was pioneered at LBNL
by William Jagust, M.D., a neurologist and the Faculty Senior Scientist
at LBNL and professor of Public Health and Neuroscience at UC Berkeley,
and Jamie Eberling, staff scientist at LBNL and Associate Professor of
Neurology at UC Davis. "The tracer is an excellent measure of gene
expression," commented Eberling. "FMT-PET studies in animal models of
Parkinson's disease have shown sustained AADC gene expression for more
than 5 years after convection-enhanced gene delivery, along with
sustained motor improvement," commented Dr. Jagust. "In this study, we
are evaluating FMT-PET in individuals with Parkinson's disease as a
surrogate marker of AADC activity and gene transfer. We are visualizing
evidence of successful AADC gene transfer in the human brain for the
first time. Both the apparent level and the duration of AADC expression
are greater than any of us had anticipated, given the low dose of AV201
with which we began the study."

"These results did exceed our expectations, and we are encouraged that we
are on the right track," said Dr. Dawn McGuire, a neurologist and
Avigen's Chief Medical Officer. "It is, of course, too early to determine
the therapeutic benefit or duration of gene expression. However, the
value of this research cannot be underestimated, both for the individuals
afflicted with Parkinson's disease and for understanding the potential of
gene therapy in neurologic disease."

On April 5, 2005 Avigen announced that it will focus on the development
of traditional pharmaceutical products, particularly small molecules and
biologics. As part of the decision, the Company indicated it would divest
its proprietary AAV technology but was committed to ensuring the
continuation of the ongoing clinical programs, including the AV201
Program in Parkinson's disease.

Commenting on the impact of the trial and the strategic decision to
reposition the Company, Kenneth G. Chahine, Ph.D., J.D., Avigen's
President and CEO, said, "We chose this approach to treating Parkinson's
disease not only for its therapeutic potential, but also the potential of
visualizing AV201 activity using PET imaging. Therefore, we are very
encouraged by these results which underscore the pioneering work of
Avigen and its collaborators to provide novel therapeutics for
neurological disorders."

Dr. Chahine continued, "This news reinforces the confidence we've always
had in our AAV gene therapy program and its potential, but does not
change our longer term strategic vision to seek external funding for the
AAV technology. At this time, we are in advanced discussions with
multiple parties who have the resources and commitment necessary to
secure the long-term future and success of our AAV technology, including
this very exciting Parkinson's trial. As we move through this process,
foremost in our minds is to evaluate our options based on which one will
deliver the best long-term value to our shareholders."

About Parkinson's Disease and AV201

Parkinson's disease (PD) is the second most common degenerative
neurological disease after Alzheimer's disease, and is characterized by
tremor, stiffness of the limbs and trunk, slowness of movement
(bradykinesia), and poor balance. PD results from the death of
specialized dopamine-producing cells in the brain. More than 2 million
individuals in the United States and Europe are afflicted with
Parkinson's disease. The primary and most effective treatment for this
debilitating movement disorder is oral administration of levodopa, which
is converted in the brain by the enzyme AADC, or dopa decarboxylase, into
dopamine. As Parkinson's disease progresses, however, levodopa typically
becomes less effective, believed due at least in part to the decline in
concentrations of AADC resulting from continued neurodegeneration and
cell death. Using higher levodopa doses in an attempt to compensate for
less efficient conversion to dopamine often leads to intolerable side
effects or toxicity. Avigen's AV201 is designed to restore the
therapeutic effectiveness of levodopa by infusing the gene for AADC into
the brain of patients, thus improving dopamine production. AV201 is an
AAV vector containing the gene for human AADC which is delivered directly
to the striatum, the part of the brain requiring dopamine to control
movement. The patient and physician potentially can regulate the activity
of AADC by raising or lowering the amount of levodopa taken. "This
functional 'on-off' switch potentially enhances the safety of AV201 gene
therapy," commented Dr. McGuire.

Early research conducted in animal models was performed by Krzysztof
Bankiewicz, M.D., Ph.D., and Professor of Neurological Surgery at UCSF.
Dr. Bankiewicz helped pioneer convection-enhanced delivery, and initiated
its use to deliver AAV-AADC in animal models of Parkinson's disease.
Studies have demonstrated AADC expression for more than 5 years after a
single administration of AAV-AADC, along with continued therapeutic
benefit in Parkinsonian nonhuman primates. These results encouraged
Avigen to move "from bench to bedside" with the current Phase I-II
clinical trial, designed to evaluate the safety of increasing doses of
AV201 in individuals with mid-to-later-stage Parkinson's disease.

Participation in This Clinical Trial

Patients or physicians who are interested in learning more about this
clinical study, please contact the UCSF Parkinson's Disease Clinic and
Research Center Study Coordinator at 415-476-0947.

About Avigen

Avigen, Inc., based in the San Francisco Bay Area, is committed to
providing physicians and their patients with innovative therapeutics for
the treatment of neurological conditions. Avigen's strength lies in its
innovative employees, rigorous science and a focused strategy on
treatments for serious and life-threatening neurological disorders.
Guided by a strong management team and supported by sound financials,
Avigen's strategy is to build a robust pipeline through a combination of
internal research, acquisitions and in-licensing with the goal of
becoming a fully integrated pharmaceutical company. Additional
information about Avigen can be found at http://www.avigen.com .

About Berkeley Lab

Berkeley Lab is a U.S. Department of Energy national laboratory located
in Berkeley, California. It conducts unclassified scientific research and
is managed by the University of California. Visit the Lab website at
http://www.lbl.gov .

Investors Please Note

This news release contains "forward-looking" statements within the
meaning of the Private Securities Litigation Reform Act of 1995 regarding
Avigen's expectations for the clinical trial of AV201, the ability of
AV201 to restore the activity of AADC in the brain, further successful
AADC gene transfers into humans, anticipated enrollment of additional
patients in the Phase I/II clinical trial and Avigen's intention to
divest its proprietary AAV technology. These statements are not a
guarantee of future performance and are subject to risks and
uncertainties that could cause actual results to differ materially from
those projected in the forward-looking statements. These risks and
uncertainties include whether the Phase I/II clinical trial results will
validate and support the safety and efficacy of AV201, ability to enroll
additional patients, uncertainties related to the timing and completion,
if at all, of the divestiture of its proprietary AAV technology,
uncertainty in obtaining or maintaining approvals and authorizations
required by regulatory or institutional authorities, unanticipated
responses to the treatment; and results from the first patient treated
with AV201 are not necessarily indicative of results that will be
obtained in the clinical trial. In addition, there are many other risks
and uncertainties inherent in the development of drug products. Actual
results may also differ from those projected in forward-looking
statements due to risks and uncertainties associated with the conduct of
clinical trials and in Avigen's operations and business. These risks and
uncertainties are detailed in reports filed by Avigen with the Securities
and Exchange Commission, including Avigen's Annual Report on Form 10-K
and Quarterly Reports on Form 10-Q. Forward-looking statements contained
in this new release are made as of this date and will not be updated.

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Source: Avigen, Inc.

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