As they say, timing is everything! In the last week, while there wasn’t an acquisition, what happened was the surprise announcement of the clinical trial results I was expecting, albeit earlier than anyone expected.
In fact, the multi-center, double-blind, placebo-controlled clinical (FLINT) trial was actually stopped by the Data Safety Monitoring Board (DSMB) because of such positive results after reviewing the study patients’ liver biopsy data. The company announced that the trial for patients with nonalcoholic steatohepatitis (NASH), a serious chronic liver disease, was stopped early due to “highly statistically significant improvement” in measures of liver health. The trial was sponsored and conducted by the National Institute of Diabetes & Digestive & Kidney Diseases (NIDDK), a part of the National Institutes of Health, at eight leading US academic hepatology centers comprising the NIDDK’s NASH clinical research network (CRN).
The NIDDK confirmed that “OCA has a significant beneficial effect on liver damage due to NASH.” Intercept’s CEO highlighted the importance of such treatment as “NASH has grown to epidemic proportions worldwide, having become a leading cause of cirrhosis and liver failure. On its current trajectory, the disease is projected to become the leading indication for liver transplant.”
There’s a huge opportunity to reach those suffering from this disease with no drugs currently approved to treat NASH. Studies have shown that over a 10-year period, at least 10% of NASH patients will develop cirrhosis. Liver-related mortality due to this disease is ten-fold that of the general population.
Quantifying the size of the market, recent epidemiological studies estimate that approximately 12% of the U.S. adult population has NASH, while 2.7% (potentially more than six million patients) are believed to have advanced liver fibrosis or cirrhosis due to progression of the disease.
The proportion of liver transplants attributable to NASH has increased rapidly in past years and over the next decade, the disease is projected to become the leading indication for liver transplant – and that’s ahead of chronic hepatitis C and alcoholic liver disease.