ISIS-DGAT2Rx specifically inhibits diacylglycerol acyltransferase-2, or DGAT-2, a key component in the synthesis of triglycerides. ISIS-DGAT2Rx is designed to reduce liver fat in patients with nonalcoholic steatohepatitis (NASH), a common and often asymptomatic liver disease that can cause irreversible damage to the liver, and lead to liver cirrhosis and cancer. The incidence of NASH in the United States is estimated to be more than 20 million, and is expected to increase as the rate of obesity rises. There are no effective therapies available for patients with NASH and current treatments consist only of lifestyle changes. In addition, because increase in liver fat is strongly associated with insulin resistance, ISIS-DGAT2Rx could also be beneficial for patients with type 2 diabetes who are insulin resistant.
ISIS-FVIIRx is designed to reduce Factor VII, a key component of the tissue factor coagulation pathway, for the treatment or prevention of thrombotic diseases without causing bleeding, which is a common side effect of currently available anti-thrombotic drugs. Elevated levels of Factor VII activity are indicative of poor prognosis in several thrombotic diseases, such as heart attacks. Furthermore, elevated levels of Factor VII activity are strongly linked to cancer-associated thrombosis, which is the second leading cause of death in cancer patients. In preclinical studies, antisense inhibition of Factor VII rapidly reduced Factor VII activity by more than 90 percent in three days, suggesting that ISIS-FVIIRx? has the potential to be used in acute clinical settings, such as following surgery, in which patients are at high risk for developing blood clots. ISIS-FVIIRx is the second drug to enter development as part of Isis' strategy to create more potent and safer anti-thrombotic drugs that do not increase bleeding.
SOURCE Isis Pharmaceuticals, Inc.