The stock popped only briefly on this news, but then dropped back to last Friday's level. An IND for the Parkinson's vector has been anticipated by the end of the year. This press release has a lot of spin on it, but it looks like Avigen is still on schedule to achieve that objective. The RAC is requiring some "minor protocol amendments." The company says that it expects to make those changes and "submit our IND to the FDA shortly, and would hope to begin our trial shortly after receiving FDA clearance." So we don't have the IND yet. On the other hand, the news looks promising, particularly the following claim: "During the public meeting, several RAC members commented on the strengths of the proposed protocol and supporting preclinical data, and the committee as a whole stated that they would recommend that the human clinical trial for AV201 be initiated."
Here's the complete press release:
<<NIH RAC Committee Recommends Avigen's AV201 for Advanced Parkinson's Disease Proceed into Human Clinical Testing
Monday October 20, 8:05 am ET
Avigen Expects to File IND With FDA for AV201 Shortly
ALAMEDA, Calif., Oct. 20 /PRNewswire-FirstCall/ -- Avigen, Inc. (Nasdaq: AVGN - News) presented its proposed clinical protocol for AV201 for the treatment of advanced Parkinson's disease at the Friday, October 17, meeting of the National Institutes of Health (NIH) Recombinant DNA Advisory Committee (RAC). The RAC is charged with reviewing all human gene transfer clinical trials to be performed at US clinical sites in which NIH funding is involved directly or indirectly, and all protocols that contain unique or novel issues are discussed in a public forum. During the public meeting, several RAC members commented on the strengths of the proposed protocol and supporting preclinical data, and the committee as a whole stated that they would recommend that the human clinical trial for AV201 be initiated. The committee suggested minor protocol amendments to strengthen the methodology and expressed satisfaction with Avigen's preclinical work supporting the safety and rationale for human clinical testing for subjects with advanced Parkinson's disease.
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"Completing our protocol presentation to the RAC committee is a key step in getting our approach into the clinic," said John Monahan, Ph.D., Avigen's President and CEO. "We benefit by having our ideas vetted by experts in the field in an open forum so that we can incorporate their feedback into our protocol early in the process. Given this positive reception, we expect to submit our IND to the FDA shortly, and would hope to begin our trial shortly after receiving FDA clearance."
Avigen's proposed clinical protocol is for a Phase 1 open-label dose escalation safety study of AV201, a gene therapy for advanced Parkinson's disease. The protocol was presented by Michael Aminoff, M.D., D. Sci., the Director of the Parkinson's Disease Clinic and Research Center at the University of California Medical School in San Francisco who is the Primary Investigator for the study, Philip Starr M.D., Ph.D., Assistant Professor of Neurological Surgery at UCSF who will perform the neurosurgery, and Avigen's long-time collaborator Krys Bankiewicz, M.D., Ph.D., Professor of Neurological Surgery at UCSF, who performed the groundbreaking preclinical research on which the protocol is based. A webcast of the public presentation will be posted at webconferences.com .
Parkinson's disease results from the death of dopamine producing cells in the substantia nigra region of the brain. Dopamine is required to control movement and low levels of dopamine result in slow, jerky or uncontrolled movement, the typical symptoms of Parkinson's. As the disease gets worse, patients suffer from tremors, a shuffling gate, loss of balance, slurred speech and muscle rigidity, ultimately becoming prisoners in their own bodies.
Current therapy is the administration of an oral medicine, levodopa, which is converted in the brain by the enzyme AADC into dopamine. Early in the disease, levodopa works very well to alleviate almost all of the symptoms. However, as Parkinson's progresses, the level of AADC declines and progressively larger doses of levodopa are required. But, increasing doses of levodopa cause increasing side effects including flailing (dyskinesias), psychosis and hallucinations. Most patients begin to experience negative side effects within five years of beginning levodopa therapy and must discontinue levodopa therapy within 10 years.
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 will permit the level of dopamine in the brain to continue to be regulated by the dose of the oral medication consumed. The study's primary objective is to determine the safety and tolerability of administration to the brain. Secondary objectives include determining the dose that most effectively restores normal levels of AADC activity as determined by a non-invasive PET scan, and to assess the effects on patients' symptoms as recorded on clinical rating scales and levodopa intake.
Avigen has conducted extensive preclinical testing to assess the safety and efficacy of this approach to treating Parkinson's disease. In more than 30 non-human primates, treatment with AV201 has restored AADC activity levels to normal, producing an effective and long-lived response to previously non-therapeutic doses of levodopa, relieving Parkinson's symptoms without any side effects. The first animals treated more than 3.5 years ago continue to show stable expression of AADC and significant behavioral response to low doses of levodopa and 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 gene therapy products, based on its AAV (adeno-associated virus) delivery platform technology. 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®, is being conducted at the Children's Hospital of Philadelphia, Stanford University Medical Center and the University of Pittsburgh. Additional information on Avigen's proprietary gene delivery products can be found at www.avigen.com>>
Marc |
