Intercept Pharmaceuticals, Inc., today announced positive results from a 165 patient, placebo controlled, double-blind Phase II clinical trial of INT-747 in patients with primary biliary cirrhosis (PBC). The study evaluated the effects of adding one of three doses of INT-747 or placebo to ursodeoxycholic acid (UDCA) therapy in patients who did not respond adequately to UDCA therapy alone. All three doses of INT-747 added to UDCA produced a statistically highly significant reduction in alkaline phosphatase (Alk Phos) levels, the primary endpoint at the end of the 12-week treatment period, as compared to patients receiving placebo and UDCA. Alk Phos is a liver enzyme routinely used to evaluate the clinical status and disease progression of PBC patients. All the INT-747 doses added to UDCA produced 20% or greater reductions in Alk Phos, with similar significant falls in other clinical liver enzymes. With the exception of a higher incidence of pruritus (itching) in the two top INT-747 dose groups, adverse events were generally similar across all groups.
PBC is a chronic autoimmune disease of the liver marked by the slow progressive destruction of the small bile ducts within the liver, which may lead to liver failure and the need for( )liver transplantation. PBC primarily afflicts women with up to 300,000 patients estimated worldwide.
Keith Lindor, MD, Dean of the Medical School at Mayo Clinic and a key investigator in the study, commented, "UDCA is the only drug currently approved for PBC and it does not adequately treat the disease in many patients. There is a real need for novel therapies and the clinically meaningful results from this study are supportive of INT-747's potential as an effective drug for PBC."
Mark Pruzanski, MD, Intercept's President and Chief Executive Officer, added, "The remarkable performance of INT-747 in this study, together with the previously announced positive Phase II study results in type 2 diabetes patients with nonalcoholic fatty liver disease, supports INT-747's potential as a novel therapeutic across a range of chronic liver diseases. We intend to present the complete data set from the study at an appropriate scientific meeting in 2010."
Intercept's Chief Medical Officer, David Shapiro, MD, commented, "This study involved more than 30 centers in eight countries and has given us a better understanding of the needs of PBC patients, as well as a good working relationship with the hepatologists looking after them across North America and Europe. With this PBC network in place, we are well positioned to conduct a Phase III program. We intend to request an End of Phase II Meeting with FDA to review the results and plans for a Phase III program."
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