1:29AM ISIS Pharm reports Phase 2 data on ISIS-APOCIII Rx Showing substantial reductions of triglycerides and Apoc-III in patients with familial chylomicronemia (ISIS) 36.06 : Co announces 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.
In this study, three patients with FCS treated with ISIS-APOCIIIRxachieved 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 particles.