AV513 is a botanical drug comprising a particular type of non-anticoagulant sulfated polysaccharides, fucoidan, which is extracted from brown seaweed. 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 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. In spite of being structurally similar to heparin, AV513 has a high index of pro-coagulant to anti-coagulant properties and is 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 Factor VIII or Factor IX, boosting clotting efficiency of these 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 disease. As a botanical drug candidate, AV513 may be subject to more expedited regulatory and clinical development under a recent FDA guidance.
Importantly, AV513 is expected to be a relatively low-cost therapy as a single agent and has already demonstrated improved hemostasis in combination with reduced levels of factor replacement in animal models. Accordingly, AV513 may have a global impact on bleeding disorders. Concept and preliminary safety validations have been achieved with AV513 in stringent large and small animal models of hemophilia with clinically relevant end points. Data from our research on AV513 were published in the January 2006 edition of “Thrombosis and Haemostasis” and highlighted by an editorial in the same journal. (Liu et al-2006)(Tuddenham editorial-Jan 2006)(D. Lillicrap, abstract, 2006).
