Isis Reports Positive Phase 2 Data on ISIS-APOCIII Rx in Patients with Familial Chylomicronemia

Isis Pharmaceuticals, Inc.

announced data from a Phase 2 study of ISIS-APOCIIIRx as a monotherapy in patients with familial chylomicronemia syndrome, or FCS.  FCS is a rare orphan disease characterized by severely high triglyceride levels that affects an estimated 3,000 to 5,000 patients worldwide.  These data were presented today by Dr. Daniel Gaudet at the National Lipid Association clinical lipid update meeting in Baltimore, MD.  In this study, three patients with FCS treated with ISIS-APOCIIIRx achieved substantial reductions in triglycerides with all three patients achieving a triglyceride level below 500 mg/dL, which substantially reduces the risk of an acute pancreatic event.  Patients also achieved substantial reductions in apoC-III and apoCIII-associated very low-density lipoprotein-cholesterol (VLDL-C) particles.  "Familial chylomicronemia is a rare and very serious genetic disorder that is often associated with triglyceride levels higher than 2,000 mg/dL.  FCS patients with triglyceride levels this high often experience a significant number of health problems such as abdominal pain, eruptive fatty skin lesions, enlargement of the liver and spleen, and recurrent acute pancreatitis