An important readout occurred today in the very hot pharmaceutical development area called NASH, a severe fatty liver disease. NASH is likely to create $10s of billions of new pharma revenue over the next 5-10 years, as there are currently no FDA-approved medicines to treat this prevalent disease (https://www.wsj.com/articles/a-big-fatty-opportunity-for-big-pharma-11547807401). Intercept (ICPT) reported out phase 3 interim efficacy and safety data earlier this morning. The results were no too surprising as mixed positive. The results were positive for the primary fibrosis endpoint with high confidence in the high dose patient group, and with less confidence in the low dose subgroup. However, the percent of patients that reached this endpoint was about 11% higher than placebo, which may not be enough for tremendous commercial success. Plus, a second primary endpoint related to NASH resolution was not met. Pruritus (chronic itching) as expected, was a prevalent AE, but higher than seen in the Phase 2 trial, with around 50% of patients experiencing pruritus in the high dose group and around 25% in the low dose group. LDL-Cs were elevated at week 4 but came back to much closer to baseline by 18 months.
It will be exciting to see more detailed results including results for diabetic patients at the upcoming scientific EASL meeting in April. ICPT confirmed that it plans to file for FDA approval in the second half of this year based on these results. The drug seems to have a high chance of being approved by the FDA since there apparently was an agreement by the FDA that it only had to meet one of its primary endpoints and since Ocaliva is already approved in another liver condition where it also shows high pruritus side effects. The market reaction today has been mixed (stock up 5% on 2/19 in PM). ICPT arguably ran up to this data since it's up about 100% in 1 year.