ALAMEDA, Calif., May 24 /PRNewswire-FirstCall/ -- Avigen, Inc. announced today a presentation of data for its orally administered drug candidate, AV513, for the treatment of hemophilia and related bleeding disorders at the 2006 World Federation of Hemophilia Congress being held in Vancouver, Canada. The data presented show that AV513, a particular type of fucoidan, was safe and able to significantly improve the bleeding times in a well-studied naturally occurring animal model of severe hemophilia A. In humans, AV513 has the potential to improve hemophilia therapy by reducing the frequency of factor infusion, resulting in both decreased cost and improved quality of life for patients and caregivers. Hemophilia is an inherited bleeding disorder characterized by a deficiency in one of several proteins, called factors, essential to clotting. Current treatment for hemophilia involves frequent intravenous administration of high-cost clotting factors.
The presentation entitled “A Therapeutic Benefit from Non-Anticoagulant Sulfated Polysaccharide (NASP) Treatment in Hemophilic Dogs” was made today by David P. Lillicrap, M.D., a hemophilia physician and researcher from the Department of Pathology and Molecular Medicine, Queen’s University, Kingston, Canada. The presentation highlights the results of multi-week studies designed to assess the efficacy and safety of fucoidan, the specific NASP used in these studies, in animals with severe hemophilia A. In the studies, fucoidan was administered subcutaneously or orally twice daily in intermittent, weekly escalating doses over several months. All of the study animals showed significant dose-dependent improvements in their cuticle bleeding times and plasma thrombelastogram profiles, two clinically relevant endpoints. Additional clinical pathology and other safety endpoints were incorporated into the study. Fucoidan treatment administered subcutaneously or orally was well tolerated over multi-week periods, with no adverse side effects and no change in Factor VIII activity.
“The pre-clinical evaluation of this agent, either subcutaneously or orally in hemophilic dogs, looks very promising in two independent measures of how blood clotting occurs,” said Dr. Lillicrap. “There is no demonstrable toxicity, and the improvement in hemostasis suggests fucoidan could possibly be an effective adjunctive agent in the management of bleeding conditions such as hemophilia.”
“The implications of these data are far reaching and set the stage for further development of AV513 as a novel pharmaceutical approach to improving hemostasis in patients suffering from life-threatening bleeding disorders,” states Ken Chahine, Ph.D., J.D., president and chief executive officer of Avigen. Dr. Chahine added, “We commend the investigators who have worked with our research team at Avigen, led by Dr. Kirk Johnson, vice president of preclinical development. Our unique approach and its advancement for treating hemophilia have led us down a very promising path. We look forward to fully exploring AV513’s utility in clinical settings where its use as supplemental therapy may help reduce the frequency and therefore the cost of factor replacement therapy.”
About AV513
AV513 is a botanical drug comprising a particular type of non-anticoagulant sulfated polysaccharides, fucoidan, which is extracted from sea algae. Examples of botanical drugs are paclitaxel, a treatment for cancer that is purified from the bark of the yew tree, and digitalis, which is purified from the leaves of the foxglove plant. Sulfated polysaccharides are a class of molecules with a history of favorable tolerability profiles in animals and humans.
It is believed that AV513 actually improves the physiological “spark” for normal clot initiation and reduces natural anti-coagulation at local sites of bleeding and thereby allows more normal hemostasis, or clotting. Clotting is a balance between pro-coagulant and anti-coagulant systems. While structurally similar to heparin, AV513 was selected for its high index of pro-coagulant vs. anti-coagulant properties and was thus denoted a non-anticoagulant sulfated polysaccharide (NASP). In contrast to heparin, AV513 can exert its pro-coagulant effects when administered orally. AV513 may act as an adjunct or supplementary agent to boost clotting efficiency with Factor VIII or Factor IX, the standard treatments for hemophilias A and B, respectively. AV513 may be useful in other bleeding disorders including Factor VII deficiency and severe von Willebrand’s. As a botanical drug candidate, AV513 may be subject to more expedited regulatory and clinical development under a recent FDA guidance.
About Avigen
Avigen is a biopharmaceutical company focused on unique small molecule therapeutics and biologics to treat serious neurological disorders, including neuropathic pain and spasticity and neuromuscular spasm. Avigen’s strategy is to complete the requirements of clinical development for each of the candidates in its product pipeline, and continue to look for opportunities to expand its pipeline through a combination of internal research, acquisitions, and in-licensing. Avigen’s goal is to become a fully integrated commercial biopharmaceutical company committed to its neurology products. In addition to AV513 for hemophilia, the company currently has in development AV650 for spasticity and neuromuscular spasm and AV411 for neuropathic pain. For more information about Avigen, consult the company’s website at http://www.avigen.com.
Statement under the Private Securities Litigation Reform Act
This press release contains forward-looking statements. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected in these forward-looking statements. These risks and uncertainties include, among others, the risks and uncertainties described in reports filed by Avigen with the Securities and Exchange Commission. Drug development is a risky and time-consuming process and there is no guarantee that Avigen’s AV513 product, or any of its other treatments in development, will be able to be successfully developed and marketed. We cannot guarantee that Avigen will be able to find a source of raw material for the production of AV513 or a manufacturer capable of producing the appropriate quality and quantity of this botanical drug, or that sufficient intellectual property protection will be able to be obtained to support the product. In addition, there are many other risks and uncertainties inherent in the development of drug products. Other risks relating to Avigen are detailed in Avigen’s Annual Report on Form 10-K for the period ended December 31, 2005, under the caption “Risk Factors” in Item 1 of Part 1 of that report, which was filed with the SEC on March 16, 2006.
Avigen, Inc.
CONTACT: Terri L. Clevenger, President of Continuum Health,+1-203-227-0209, or fax, +1-203-226-8780, or mobile, +1-203-856-4326, ortclevenger@continuumhealthcare.com; or investors, Michael Coffee, ChiefBusiness Officer of Avigen, Inc., +1-510-748-7372, fax, +1-510-748-7155, ormcoffee@avigen.com
Web site: http://www.avigen.com//
