AVGN Submits New Drug App for Parkinson's Drug
Ridgeland, MS, NOV 05, 2003 (EventX/Knobias.com via COMTEX) -- Avigen, Inc. (AVGN) announced that it has filed an
Investigational New Drug application with the 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 where Avigen intends to conduct its clinical trial. Primate studies have shown
AV201 to be very effective, long-lasting and safe.
SOURCE: MacroWorld Investot
http://www.mworld.com/m/m.w?lp=GetStory&id=77680731
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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 05, 2003 /PRNewswire via COMTEX/ -- 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 [log in to unmask] 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.
SOURCE Avigen, Inc.
CONTACT: Lise Needham, Associate Director of Investor Relations of
Avigen, Inc., +1-510-748-7112, or [log in to unmask]
URL: http://www.prnewswire.com
http://www.avigen.com
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SOURCE: PR Newswire / MacroWorld Investor
http://www.mworld.com/m/m.w?lp=GetStory&id=77678671
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