Avigen's stock has taken a beating recently (who in the gene therapy business hasn't?). But at least the company has a 4-5 year supply of cash. According to Yahoo! the company has $5.65/share in cash. The stock price closed today at $4.57, a new 52-week low. Let's hope they meet their announced goal of filing an IND for their Parkinson's treatment by mid-year. A little positive news flow wouldn't hurt.
<<"Over the last year, Avigen made steady progress in clinical trials and preclinical studies for hemophilia and Parkinson's disease. We believe we are at the forefront of developing effective gene therapies for the treatment of these serious, chronic medical conditions," said John Monahan, Ph.D., president and CEO.
"During the fourth quarter, we took several strategic steps to strengthen our ability to successfully bring products to market. In October, we more tightly focused our organization on our lead product development programs. Based on these actions and our expectations for efficiency and vector production levels, we expect Avigen's cash burn rate to drop to less than $25 million for the full year 2003 down from $29 million in 2002, greatly extending the life of our existing financial resources. With $120 million in the bank, we believe we can support our planned clinical trials and ongoing research and development programs for the next four to five years.
"We realigned our management team to execute this more clinically focused strategy. In November, Glenn Pierce, Ph.D., M.D., joined Avigen as Vice President of Clinical Development bringing extensive experience in biotech research and clinical programs in gene therapy, hemophilia and neurobiology. We also promoted Ken Chahine, Ph.D., J.D., Avigen's Vice President of Business Development and Intellectual Property since 1998, to the new role of Chief Operating Officer, in recognition of his many contributions to Avigen's success and his expanded operating responsibilities."
Dr. Monahan continued, "Now that we have completed the internal refocusing of operations, two of Avigen's vice presidents are leaving the company, Alan McClelland Ph.D., Vice President, Research and Development, and Fred Johnson, Ph.D., Vice President, Operations. I want to thank them both for the many contributions they have made to Avigen."
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Dr. Monahan continued, "We customarily update the scientific community about our progress in hemophilia at the American Society of Hematology Annual Meeting each December. This year, we presented early data from the first six participants, at three progressive dose levels, in our ongoing Phase I clinical trial of Coagulin-B®, our product for the treatment of Hemophilia B. The first participant treated in the third dose group, achieved circulating levels of Factor IX in excess of 10% of normal for approximately four weeks after gene transfer, at which point the level began to decline rapidly. We believe that levels of 3 - 5% of normal can be considered therapeutic.
"This is the first time gene therapy has achieved therapeutic levels of Factor IX in the blood of someone with severe hemophilia. However, we are not yet satisfied with the length of effectiveness. In all of our animal studies, once a stable expression level is reached, it remains stable for the life of the cell that received the vector. In our preclinical studies for the treatment of Hemophilia B, the first dog treated with an AAV vector with the gene for Factor IX continues to express therapeutic levels more than four years after treatment. We are committed to continuing the work necessary to achieve similar results in humans," Monahan said.
"Over the last year, we also made progress in preclinical studies for the treatment of Hemophilia A and for Parkinson's disease," said Monahan. "Earlier in the year, we announced we had achieved long-term expression of Factor VIII in a large animal model. We believe we are the only team to have achieved this important milestone in the development of an effective gene therapy for Hemophilia A. At year end, we continued to see expression more than two years after a single injection of AAV vector.
"Finally, in Parkinson's disease, we are in the process of completing an expanded non-human primate study to evaluate and optimize the AAV vector construct and delivery mechanism to support the filing of an IND for this indication in mid-2003," Monahan concluded.
Ken Chahine, Avigen's Chief Operating Officer added, "Further highlights of the quarter included the granting of an important new patent and a new research collaboration. We received a further patent on our manufacturing process, U.S. Patent No. 6,482,633: Compositions and Methods of Making AAV Free of Helper Virus, strengthening our patent position considerably. This composition of matter patent covers AAV-vector free of helper virus, a key differentiator between Avigen's approach and that of some competitors.
"In October, we announced a collaborative research agreement with Sangamo Biosciences to develop novel therapies for chronic pain that we believe will have significantly fewer side effects than current treatments based on Sangamo's gene targeting and regulation technology combined with our AAV gene delivery system. Intractable neuropathic pain is a significant medical problem affecting 50 million Americans annually," Chahine concluded.
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. Avigen's Factor IX gene therapy product for hemophilia B, Coagulin-B®, is currently in clinical trials at the Children's Hospital of Philadelphia and Stanford University Medical Center. Additional information on Avigen's proprietary gene delivery products can be found at www.avigen.com.>>
Marc |
