Archimedes PM'ed me to ask if I still follow Avigen. I'm still long the stock, but I missed the last stockholders meeting and I hadn't been keeping a very close eye on it. Prompted by Archimedes' interest and inquiry about what went wrong with the Coagulin-B studies, I looked through the most recent 10Q for some answers. I found the following excerpts there:
<<In May 2004, we announced the suspension of patient enrollment in our phase I clinical trial involving the administration of Coagulin-B to the liver. [However} we feel there is considerable value in our years of clinical experience and manufacturing, and intellectual property development within the program, and we hope that these efforts could lead to additional hemophilia trials in the future.>>
On 12/13/04, subsequent to the filing of the 10Q, Avigen reported that Bayer had terminated the agreement to fund development of Coagulin-B:
<<As a result of the termination, Bayer no longer has any financial obligations to finance Phase II/III development of Coagulin-B and all patent and know-how rights licensed to Bayer under the agreement completely revert back to Avigen. Since Avigen was solely responsible for funding the Phase I development, the termination of the agreement has no financial impact on Avigen. More importantly, Avigen now has the right to develop a product for the treatment of hemophilia-B independently or in collaboration with another corporate partner in the future. It is Avigen's intention to preserve the large investment it has made in gene therapy for hemophilia and is exploring avenues to realize value from the asset that has been created over the years in terms of know-how, manufacturing, intellectual property and clinical expertise.
On December 10, 2004, Avigen, Inc. notified BTG International Inc. of its intent to terminate Avigen's exclusive license with BTG to intellectual property rights covering the Factor IX gene in the field of in vivo viral vector human gene therapy products. As Avigen is required to provide BTG with three month's notice to terminate, termination will be effective March 10, 2005. Since this patent will expire on February 19, 2008, it is no longer deemed a critical value-added component if Avigen should decide to move its Coagulin-B program forward.>>
About the reasons for suspending development of Coagulin-B last May, the 10Q says this:
<<[W]hile the results of our gene therapy treatment for hemophilia B were favorable and demonstrated sustained long-term expression in both dogs and mice for multiple years, the one human subject who demonstrated therapeutic levels of circulating factor IX when given a comparable dose size to that used in the successful animal studies was not able to sustain steady factor IX expression beyond five weeks. In addition, this human subject experienced a mild, temporary elevation of two liver enzymes, which was not seen in any of the animal models. Further, we have observed an immune system response in subjects treated with Coagulin-B that was not observed in the animal models, which we have not yet been able to, and may not be able to, adequately address. In May 2004, we suspended patient enrollment in our phase I clinical trial of Coagulin-B. Consequently, you should not rely on the results in any of our animal models as being predictive of the results that we will see in our clinical trials with humans.>>
It is logical to speculate that the immune system responses (a) were responsible for the poor results in terms of sustained factor IX expression and (b) may only be a problem when the AAV vector is injected directly into the liver. If true, then the use of the AAV vector in the current PD trials, which call for the vector to be injected directly into the brain, may not encounter the problems that sank the Coagulin-B trials. So there are reasons to hope that Avigen may yet prosper.
Marc |
