BCX7353 is in clinical development for routine prevention of angioedema attacks in patients with hereditary angioedema (HAE). HAE is a rare genetic disorder of blood vessels characterised by recurrent episodes of severe swelling (angioedema) below the skin which often affect the face, throat, stomach, genitals, hands or feet, causing discomfort and pain. HAE may be life threatening when the swelling occurs in the throat as it can obstruct the airways and impede breathing. HAE has no known cure and the goal of treatment is to minimise the burden of illness on patients and enable them to lead normal lives. Long-term preventive treatments are used routinely to reduce the need for treatment of acute attacks. Currently available treatments are given by injection.
BCX7353 is an oral treatment that works by blocking the activity the specific pathway that becomes overactive in patients with angioedema. By blocking this pathway, BCX7353 is expected to reduce the number of angioedema attacks. Early studies of BCX7353 has shown that it helped to prevent swelling and inflammation in HAE. In addition, BCX7353 has the distinct advantage over current treatment of being administered orally, making it easier to use for routine prevention of HAE attacks.
Ruxolitinib is in clinical development for chronic graft versus host disease (cGvHD). After a donor stem cell transplant, the donor’s stem cells (the graft) may sometimes react against the host’s own cells. This is called GVHD. cGvHD may happen more than three months after transplant. It can develop from acute GVHD or happen on its …