Surprising Results in New Fatty Liver Drug Trial

Surprising results in a study of a new drug treatment for fatty liver disease caused the stock of a small biotech company — Intercept Pharmaceuticals — to quadruple this week. The company has only 45 employees and no products yet, but its stock value jumped to $1.4 billion when a phase II trial of obeticholic acid for fatty liver disease was stopped because it worked so well. To have continued the placebo-controlled trial would have been unethical.

Intercept Chief Executive and co-founder Mark Pruzanski said:

My reaction is “Wow.”  We didn’t expect this. The bar for stopping the study for efficacy was so high from a statistical significance standpoint that on an interim analysis like this, it seemed out of sight. We couldn’t be happier with the result.

An interim analysis of efficacy by a data safety monitoring board found that the statistical significance of this drug’s superiority (p=0.0024) had surpassed the predefined threshold of p<0.0031. The FLINT trial, sponsored and conducted by the National Institute of Diabetes and Digestive and Kidney Diseases, evaluated the efficacy of a 25 mg oral dose of obeticholic acid in 283 patients over a period of 72 weeks. Patients with biopsy-confirmed non-alcoholic steatohepatitis (NASH or fatty liver disease)  were randomized to receive either the drug or a matching placebo.

Fatty liver disease is an increasingly important consequence of the growing prevalence of severe obesity. No drugs are currently approved to treat this condition. Approximately six million Americans have fatty liver disease that is sufficiently advanced to put them at risk for requiring a liver transplant.

As the complications of obesity — like fatty liver disease — continue to mount, new technology for treating them will continue to emerge. That’s great, but the cost of treating all these complications will grow ever larger. New, innovative treatments are costly for good reasons.

Meanwhile, payers continue to nickle and dime the few providers of evidence-based treatment for obesity itself. It’s just not right.

Click here to read more in the New York Times, here to read more in the Wall Street Journal, and here to read more from Clinical Endocrinology News.

Andrews Liver Salt, photograph © Garrett Miller / flickr

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