Although we don't believe in timing the market or panicking over market movements, we do like to keep an eye on big changes -- just in case they're material to our investing thesis.
What: Shares of Intercept Pharmaceuticals (ICPT), a clinical-stage biopharmaceutical company developing therapies to treat chronic liver and intestinal diseases, fell as much as 16% following a myriad of weekend data including its quarterly results, the release of phase 3 results utilizing obeticholic acid, or OCA, to treat primary biliary cirrhosis of the liver, and updated adverse event data from its phase 2 study of OCA in treating nonalcoholic steatohepatitis, or NASH.
So what: First, Intercept delivered its quarterly results which resulted in $405,000 in licensing revenue and a net loss of $12.4 million which was considerably lower than the $30.8 million loss reported in Q4 2012. Intercept announced that it ended the quarter with $144.8 million in cash and cash equivalents and likely has enough cash to fund its operations into the third quarter of 2015.
The bigger movers though were its phase 3 study results and phase 2 update. Its phase 3 POISE trial delivered solid results with OCA hitting its primary endpoint of achieving a serum alkaline phosphatase reduction equal to 15% from baseline as well as normal bilirubin after one year of therapy. Relative to the placebo, 47% of the 10 mg OCA cohort and 46% of the 5-10 mg cohort met the primary endpoint compared to just 10% of the control arm. OCA also offered a similar and favorable safety profile to the placebo. Based on this data Intercept will shortly be filing for a new drug application for OCA in treating primary biliary cirrhosis.
What really has investors' concerned, though, are two paragraphs on page 14 of its 10-K annual filing. According to the National Institute of Diabetes and Digestive and Kidney Diseases which released additional data on the FLINT trial for NASH to Intercept, there were 10 cardiovascular serious adverse events that occurred in seven patients (2.5% of total patients) across both NASH treatments groups. We already know that the independent data safety monitoring board expressed concerns previously about elevated lipid levels in the liver since this can be a factor which can lead to a serious cardiac event, among other issues. However, based on the 10-K, the NIDDK, "has advised us that while the incidence of the serious cardiovascular events was numerically higher in the patients receiving OCA therapy, it was not statistically significantly different as compared to the placebo group." Eight patients were deemed to have adverse events not related or probably not related to OCA while two additional patients in an unblinded group were considered to have their events possibly related to OCA. Full FLINT trial data will be released in July.
Now what: That's an absolute mound of data to take in during a single day and is a reason why I'm not shocked that Intercept's share price was all over the map today. What's really important here is that OCA looks great as a potential new therapy for primary bilary cirrhosis, and the concerns about increased lipid levels in its NASH trial appear to be statistically insignificant relative to the placebo group. While I would urge investors to be cautious given that with Intercept's share price has risen so much already, this update would suggest that OCA remains on track to possibly become a blockbuster therapy.
