US-based Conatus Pharmaceuticals has started a Phase II clinical study of its lead drug candidate, emricasan, in patients with nonalcoholic fatty liver disease (NAFLD), including the subset of NAFLD patients with inflammatory and/or fibrotic nonalcoholic steatohepatitis (NASH).
Around 40 patients will be enrolled in the double-blind, placebo-controlled Phase II study, which will be carried out at four planned clinical sites in the US.
In the trial, patients will be randomised 1:1 to receive either 25mg of emricasan or placebo orally twice daily for 28 days and will then be followed for another 28 days.
The primary endpoint of the trial is a reduction of elevated levels of key biomarkers implicated in patients with NAFLD/NASH.
The Phase II study will also assess the safety and tolerability of emricasan in the target patient population and its top-line results are expected to be released in the second half of 2014.
Conatus co-founder, president and chief executive officer Steven Mento said: "Our goal for this trial is to expand our safety database into a broader patient population and to explore emricasan's potential activity in the NAFLD/NASH population."
The company said that clinical endpoints suitable to support approval of new treatments for NASH have not yet been fully defined.
The main aim of the company is to accumulate sufficient and relevant clinical data to allow rapid advancement of emricasan once appropriate regulatory pathways are defined.
Emricasan is being developed by Conatus to treat patients with chronic liver disease and acute exacerbations of chronic liver disease, including acute-on-chronic liver failure (ACLF), chronic liver failure (CLF), NAFLD and NASH.
The drug is also intended for Post Orthotopic Liver Transplant (POLT) recipients with reestablished liver fibrosis as a result of recurrent post-transplant hepatitis C virus infection who have successfully achieved a sustained viral response (SVR) following HCV antiviral therapy (POLT-HCV-SVR).
Image: Micrograph of non-alcoholic fatty liver disease, demonstrating marked macrovesicular steatosis. Photo: courtesy of Nephron.