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Isis Reports Phase 2 Data on ISIS-APOCIII Rx Showing Substantial Reductions of Triglycerides and Apoc-III in Patients with Familial Chylomicronemia
Date:9/21/2013

CARLSBAD, Calif., Sept. 21, 2013 /PRNewswire/ -- Isis Pharmaceuticals, Inc. (NASDAQ: ISIS) 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. 

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"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 that often requires hospitalization.  Current treatment approaches are usually ineffective in FCS patients and, as a result, FCS patients are required to severely limit their dietary fat intake.  Despite these very restrictive measures, many FCS patients are still unable to lower their triglycerides enough to eliminate the risk of health
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SOURCE Isis Pharmaceuticals, Inc.
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