Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) announced today that researchers presented new longer-term data from an ongoing, open-label extension of the pivotal Phase 3 ARISE trial of Kanuma ® (sebelipase alfa) in children and adults with lysosomal acid lipase deficiency (LAL-D), a genetic and progressive ultra-rare metabolic disease. Two-thirds of patients (8/12) treated with Kanuma for 52 weeks had a reduction in liver fibrosis stage from baseline, as measured by Ishak score. Moreover, half of patients (6/12) achieved at least a 2-stage reduction, including five patients who had fibrosis at baseline and one who had cirrhosis at baseline. Reduction of liver fibrosis stage was accompanied by sustained improvements in alanine aminotransferase (ALT), LDL cholesterol (LDL-C), and liver fat content through 52 weeks of Kanuma treatment. 1 These data were reported in a poster presentation at The Liver Meeting 2016, the annual meeting of the American Association for the Study of Liver Diseases (AASLD), in Boston. Researchers at AASLD also presented new long-term data from the ARISE study showing rapid and sustained improvements in important markers of liver injury and lipid abnormalities in children and adults with LAL-D treated with Kanuma. With 76 weeks of Kanuma treatment, nearly all patients (98 percent) had a sustained reduction in ALT levels, with a mean reduction from baseline of 56 percent. 2 "LAL-D is a devastating and life-threatening disease in which half of children and adults will progress to fibrosis, cirrhosis, or liver transplant within just three years. We are very encouraged that the data presented at AASLD show, for the first time, that Kanuma may have the potential to halt and even reverse the progression of liver damage for patients receiving ongoing treatment," said Martin Mackay, Ph.D., Executive Vice President and Global Head of R&D at Alexion. "Importantly, the greatest impact was observed in those patients who had more significant liver fibrosis at baseline. Findings from this study support the value of early and long-term treatment with Kanuma."