Intercept Pharmaceuticals Announces Positive Phase II Results for INT-747 as a Treatment for Primary Biliary Cirrhosis
NEW YORK, Oct. 27 /PRNewswire/ -- 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."
About INT-747 (first-in-class FXR agonist)
INT-747 is a potent, first-in-class farnesoid X receptor (FXR)
agonist derived from the primary human bile acid chenodeoxycholic
acid (CDCA), the natural endogenous FXR agonist. INT-747 is
formulated as an orally bioavailable drug given once daily. Bile
acid signaling through FXR has been shown to regulate the
regenerative properties of the liver and, in numerous animal models
of liver disease, INT-747 treatment prevents, and even reverses,
liver damage caused by progressive fibrosis (scarring). FXR is also
expressed in the intestine and kidney and INT-747 exhibits similar
protective antifibrotic effects in models of inflammatory bowel
disease and diabetic nephropathy.
About Intercept Pharmaceuticals
Intercept is a clinical stage biopharmaceutical company focused
on discovering and developing small molecule drugs for the
treatment of chronic fibrotic and metabolic diseases. The company's
most advanced programs are focused on the development of modified
bile acids that are selective for FXR, a nuclear receptor, and
TGR5, a G protein-coupled receptor. Intercept's lead drug
candidate, the FXR agonist INT-747, is currently being advanced for
chronic liver disease indications and the company has been granted
orphan status in the US for the PBC indication. The company's next
candidate in the pipeline, INT-777, is a selective TGR5 agonist
being advanced to an IND, projected for the second quarter of
2010.
For more information about Intercept, please go to
www.interceptpharma.com, and for information about Intercept's lead
investor, Genextra S.p.A., please go to www.genextra.it.
Source: Intercept Pharmaceuticals, Inc.
CONTACT: Mark Pruzanski, M.D., or Barbara Duncan, both of
Intercept
Pharmaceuticals, +1-646-747-1000
Web Site: http://www.interceptpharma.com/
Posted: October 2009
