US stock market daily report (January 13, 2014, Monday)
January 14, 2014, Tuesday, 05:43 GMT | 00:43 EST | 10:13 IST | 12:43 SGT
Shooting star Intercept Pharmaceuticals, Inc. (ICPT) was center of attention on Wall Street again on Monday. At the closing bell, ICPT shares were off by 18% or lower by over $81. ICPT went public in 2012 at $17 a share on its IPO. In the span of just two trading days, ICPT saw its market value surge from just $1.4 billion to $8.6 billion! Average trading volume for Intercept was 200,000 however on the heals of the news on OCA, volume surged to 7 million.
The Pharmaceutical company has received orphan drug designation for OCA for the treatment of PBC in the United States and Europe. Orphan drug designation is given to drugs being developed for a rare disease, can gain easier marketing approval and receive financial incentives. Intercept owns worldwide rights to OCA outside of Japan and China where it has out-licensed the product candidate to Dainippon Sumitomo Pharma. Patent rights cover OCA’s key components through 2022 before any potential extensions and adjustments.
Currently, there are no drugs approved to treat NASH thus, OCA provides a huge market for ICPT. Over a 10-year period, studies show nearly 10% of NASH patients will develop cirrhosis. Mortality rate for liver disease is ten-fold that of the general population. Recent epidemiological studies estimate that nearly 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. Over the next decade, NASH is projected to become the leading indication for liver transplant - ahead of chronic hepatitis C and alcoholic liver disease.
ICPT reported the multi-center, double-blind, placebo-controlled clinical (FLINT) trial for patients with nonalcoholic steatohepatitis (NASH), a serious chronic liver disease, was stopped by the Data Safety Monitoring Board due to “highly statistically significant improvement” after reviewing patients’ liver biopsy data. Intercept’s CEO highlighted the importance of treatment as “NASH has grown to epidemic proportions worldwide, having become a leading cause of cirrhosis and liver failure". Intercept said that “a vast majority of the patients completing the 12 months have opted to cross over to the five year long term safety extension open-label phase of the trial.” In five different indications, ICPT obtained positive clinical data in all six Phase 2 clinical trials. Dainippon Sumitomo Pharma is also conducting NASH trials of OCA in Japan during 2014, with top line results expected by the end of 2015.
The National Institute of Diabetes & Digestive & Kidney Diseases, a part of the National Institutes of Health, sponsored and conducted the trial at eight leading U.S. academic hepatology centers comprising the NIDDK’s NASH clinical research network (CRN) and released a statement after markets closed on January 10, 2014:
- Its decision to halt the 72-week therapeutic phase of FLINT was based on interim efficacy results showing that "OCA has a significant beneficial effect on liver damage due to NASH."
- As specified in the protocol, a 24-week follow up period is now commencing and all FLINT patients will stop taking OCA or placebo no later than January 20, 2014.
- Both the FLINT investigators and patients enrolled in the trial will remain blinded until completion of the follow up phase. All the trial data will then be thoroughly analyzed and the NIDDK anticipates presenting the results in 4Q 2014.
- Lipid abnormalities involving increased total cholesterol and LDL as well as decreased HDL were seen in OCA-treated patients compared to placebo.
- Since lipid abnormalities are common in NASH, all of the patients will be followed for the 24-week period to help determine whether lipids return to pre-treatment levels.
NIDDK confirmed that “OCA has a significant beneficial effect on liver damage due to NASH.”
The National Institutes of Health said that patients who received OCA had “disproportionate lipid abnormalities,” including higher cholesterol levels compared with those who used a placebo. Intercept notes that lipid abnormalities are common in NASH patients and will review its data to determine whether or not these lipids return to pre-treatment levels. Intercept said that OCA assists in the removal of excess lipids from the liver, which causes the disease.