Avigen Files Investigational New Drug Application With the FDA For AV201 for the Treatment of Advanced Parkinson's Disease
06 November 2003 - 12:30AM
PR Newswire (US)
Avigen Files Investigational New Drug Application With the FDA For
AV201 for the Treatment of Advanced Parkinson's Disease Team of
Leading Researchers at UCSF to Conduct Clinical Trial ALAMEDA,
Calif., Nov. 5 /PRNewswire/ -- Avigen, Inc., a leader in the
development of DNA-based drugs, announced today that it has filed
an Investigational New Drug (IND) application with the Food and
Drug Administration (FDA) seeking clearance to enter clinical
testing for AV201, its drug for the treatment of advanced
Parkinson's disease. Upon FDA consent, Avigen's next step will be
to present its clinical protocol for the Phase 1 open-label dose
escalation safety study of AV201 to the Institutional Review Board
at the University of California San Francisco (UCSF) where Avigen
intends to conduct its clinical trial. Avigen's clinical trial team
at UCSF is made up of leading doctors and researchers in neurology
and Parkinson's disease including principal investigator Michael
Aminoff, M.D., D.Sc., the Director of the Parkinson's Disease
Clinic and Research Center, and co-investigators Philip Starr M.D.,
Ph.D., Associate Professor of Neurological Surgery, and Chadwick
Christine, M.D., Assistant Professor of Neurology. Avigen's
long-time collaborator Krys Bankiewicz, M.D., Ph.D., Professor of
Neurological Surgery at UCSF, performed the groundbreaking
preclinical research on which the protocol is based. "I am very
excited by the potential of Avigen's approach to treating
Parkinson's disease," said Dr. Aminoff. "I have worked with
Parkinson's patients for more than 30 years and have been involved
in many research studies. While treatments have improved over the
years, I have very high hopes that AV201 may be the next great step
forward in treating this serious medical condition." Parkinson's
disease results from the death of dopamine-producing cells in the
substantia nigra, a small region of the brain. Dopamine is required
to control movement and low levels of dopamine result in the
typical symptoms of Parkinson's. These consist of tremor, slowness
of voluntary movement, muscle stiffness or rigidity, shuffling
gait, loss of balance, slurred speech and increasing dependence on
others. Current therapy is the oral administration of levodopa,
which is converted in the brain by the enzyme Aromatic L-Amino Acid
Decarboxylase (AADC) into dopamine. Early in the disease, levodopa
works very well to alleviate almost all of the symptoms. However,
as Parkinson's disease progresses, the level of AADC declines and
progressively larger doses of levodopa are required. However,
increasing doses of levodopa cause increasing side effects
including a variety of abnormal movements (dyskinesias), psychosis
and hallucinations. Most patients begin to experience negative side
effects within five years of beginning levodopa therapy and
eventually need to limit the dose of levodopa. Further, levodopa
becomes less effective as the disease advances, leading to an
inadequate therapeutic response. Avigen's AV201 is designed to
restore the therapeutic effectiveness of levodopa by putting the
gene for AADC into the striatum of the brain of patients with
advanced Parkinson's disease so that they will respond to a lower
dose of levodopa and not experience debilitating side effects.
Current medical understanding is that AADC's only function is to
convert levodopa to dopamine. Treatment with AV201 is intended to
permit the level of dopamine in the brain to continue to be
regulated by the dose of the oral medication consumed. Primate
studies have shown AV201 to be very effective, long-lasting and
safe. The earliest primates with Parkinsonian symptoms to be
treated with AV201 were treated over three and a half years ago.
Before they were treated, they did not respond to low levels of
levodopa. After a single administration of AV201 to the striatum,
they continue to show stable expression of AADC and significant
behavioral response to low doses of levodopa. They have not
developed dyskinesias or other debilitating side effects. About
Avigen Avigen, Inc., based in the San Francisco Bay Area, is a
leader in the development of DNA-based drugs. Avigen's proposed
gene delivery products are designed for direct administration to
patients in order to achieve expression of therapeutic proteins
within the body. The clinical trial for Avigen's Factor IX gene
therapy product for hemophilia B, Coagulin-B(R), is being conducted
at the Children's Hospital of Philadelphia, Stanford University
Medical Center and the University of Pittsburgh. Avigen is actively
enrolling subjects in the Coagulin-B clinical study. Patients or
physicians who would like more information about enrollment
criteria and the study should contact the Clinical Development
Department at or call at 510-748-7379. Additional information on
Avigen's proprietary gene delivery products can be found at
http://www.avigen.com/. Investors Please Note: The statements in
this news release regarding Avigen's beliefs regarding the
potential of AV201 to treat Parkinson's disease, and to gain FDA
consent to begin clinical trials, are forward-looking statements.
Actual results may differ materially from current expectations due
to a variety of factors, including: results in preclinical trials
with animals are not necessarily indicative of results that will be
obtained in humans; and uncertainty in obtaining or maintaining
approvals required by regulatory or institutional authorities due
to a number of possible reasons including unanticipated responses
to the treatment; and Avigen may experience difficulties in
enrolling suitable trial participants, which would delay the
commencement or continuation of the trials. In addition, there are
many other risks and uncertainties inherent in the development of
gene therapy products. Other risks relating to Avigen are detailed
in Avigen's Quarterly Report on Form 10-Q for the period ended June
30, 2003, under the caption "Risk Factors" in Item 2 of Part 1 of
that report, which was filed with the SEC on August 8, 2003.
DATASOURCE: Avigen, Inc. CONTACT: Lise Needham, Associate Director
of Investor Relations of Avigen, Inc., +1-510-748-7112, or Web
site: http://www.avigen.com/
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