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New Drug Application Filed for AV201 for the Treatment of Advanced Parkinson’s Disease

Alameda, CA, November 5, 2003 -- 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.

Source: Avigen, Inc.

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