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New Data Shows Breakthrough microRNA-Targeted Therapy Developed Using Santaris Pharma A/S Proprietary LNA Technology Holds Promise as New Treatment for Hepatitis C
Date:12/3/2009

ompany focused on the discovery and development of RNA-targeted therapies for a range of diseases including metabolic disorders, infectious and inflammatory diseases, cancer and rare genetic disorders.

In this preclinical study, SPC3649 successfully inhibited miR-122, a liver-expressed microRNA important for Hepatitis C viral replication. By inhibiting miR-122, SPC3649 dramatically reduced Hepatitis C virus in the liver and in the bloodstream in chimpanzees chronically infected with the Hepatitis C virus[4]. Four HCV chronically infected chimpanzees were treated weekly with 5 or 1 mg/kg of SPC3649 for 12 weeks followed by a treatment free period of 17 weeks. The two animals that received the 5 mg/kg dose had a significant decline in viral levels in the blood and liver of approximately 2.5 orders of magnitude or approximately 350 fold[5].

"In collaborating with Santaris Pharma, we proved that the drug worked exceptionally well in treating HCV infections in chimpanzees," said Robert Lanford, Ph.D., a scientist at the Southwest Foundation for Biomedical Research and one of the lead authors on the study. "The current standard anti-HCV treatment, which combines pegylated interferon-alpha with ribavirin, is effective in only about 50% of patients and is often associated with severe side effects. Because of the unique mechanism of action of SPC3649 and its tolerability profile, this new therapy could have the potential to replace interferon to treat disease progression or be combined with current treatments."

SPC3649 provided continued efficacy in the animals up to several months after the treatment period with no adverse events and no evidence of viral rebound or resistance, an important factor that distinguishes SPC3649 from direct antiviral HCV therapeutics.

Current antiviral therapies that target the virus directly are challenged as the HCV continually mutates to develop resistance to treatment. Because SP
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SOURCE Santaris Pharma A/S
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