Enzo Biochem Inc. (NYSE:ENZ) announced today that favorable results of the Company's clinical trial for treatment of non-alcoholic steatohepatitis (NASH) were presented at the annual meeting of the American Association for the Study of Liver Diseases. The study was a randomized double blind placebo controlled format of EGS21, Enzo's orally administered beta glucosylceramide formulation, designed to evaluate safety and efficacy of the study drug in patients with NASH and its associated metabolic syndrome.

Enzo scientists and collaborators presented data showing that "oral administration of EGS21 appears to be safe and biologically active in patients with insulin resistance and NASH, leading to decreased hepatic steatosis, and improved insulin resistance."

Twenty one patients with biopsy proven NASH were enrolled and received an oral dose of EGS21 or placebo daily during the 40-week study. The patients were followed by measuring the effect of treatment on BMI (body mass index), lipid profile, insulin sensitivity, HbA1c (glycated hemoglobin) and glucose tolerance. Quantification of hepatic fat was determined by MRI, and liver damage was assessed by a repeat liver biopsy and measurement of liver enzymes. The immune modulatory effect of EGS21 was determined by a measurement of circulatory immune cells, CD4, CD8 and NKT cells.

No treatment-related adverse events were observed during treatment or follow-up in any of the patients, they added.

Treatment with EGS21 was associated with an improvement in the metabolic syndrome. Of the primary endpoints, HbA1c decreased by 0.1% compared with a 0.09% in the placebo group. Of the secondary endpoints, glucose tolerance improved in 50% of the patients compared with 38% of the placebo group. HDL levels increased in 33% of the treated patients compared with none in the placebo group. Patients treated with EGS21 showed a decrease in the hepatic fat score measured by MRI of 14% compared to 7.7% in the placebo group, indicating a reduction in hepatic steatosis. No significant change in liver enzymes was noted between the two patient groups.

The observed changes were associated with changes in peripheral immune cells. A 52% increase in peripheral NKT lymphocytes was observed compared with a 74% decrease in the placebo group, and a 57% decrease in the CD4/CD8 ratio of treated patients compared with a 138% increase in patients who received placebo. These data support the role of immune cell therapies in patients with insulin resistance and NASH.

EGS21 is a proprietary formulation of beta glucosylceramide, a naturally occurring gylcosphingolipid that was shown to improve hepatic steatosis, steatohepatitis and insulin resistance in animal models of non-alcoholic fatty liver disease (NAFLD) by exerting an NKT regulatory lymphocyte dependent immune modulatory effect.

NASH, or nonalcoholic fatty liver disease, is the most common form of chronic liver disease in the Western world. NASH is one stage in a disease spectrum that ranges from simple fatty liver or steatosis to steatohepatitis, advanced fibrosis, and end-state cirrhosis. NASH affects 2 to 5 percent of Americans and is becoming more common, possibly because of the greater number of Americans with obesity, according to The National Institute of Diabetes and Digestive and Kidney Diseases.

The causes of NASH are not well defined, and currently there is no effective medical therapy available.

"Study data support the role of NKT-based immunotherapies in patients with insulin resistance and NASH, and is consistent with Enzo's pre-clinical and clinical studies involving the development of new therapeutic platforms for immune mediated diseases," said Elazar Rabbani, Ph.D., Enzo Chairman and CEO.

Source
Enzo Biochem Inc.