Will move for approval in US and Europe later this year
Eagerly awaited Phase 3 trial results were released early Tuesday morning from Intercept Pharmaceuticals, showing positive results for the study’s primary goal of showing a statistically significant reduction in liver fibrosis compared to placebo. As a result, Intercept will seek approval in the US and Europe to permit the drug, known as Ocaliva, to be used as the first ever treatment for NASH-related fibrosis. The stock price rose as high as 23% on the news.
The trial was unfortunately unsuccessful at showing statistical significance in its secondary goal of actual NASH resolution. And the results were not quite as good as earlier trial phase results with regards to fibrosis improvement. Perhaps as a result, the stock price is backing off earlier highs today.
The results: 23 percent of the patients on the higher OCA dose showed a statistically significant reduction in liver fibrosis, or scarring, compared to 12 percent of the placebo patients. This achieved one of two co-primary endpoints of the clinical trial. There may be some disappointment about the magnitude of the liver fibrosis benefit observed in this clinical trial, which was lower than OCA showed in a previous study.
Twelve percent of the high-dose OCA patients also showed NASH “resolution,” defined as a reductions in liver fat and inflammation compared to 8 percent of placebo patients. This difference trended in favor of OCA but did not reach statistical significance.
With last week’s announcement that competitor Gilead had not had a successful Phase 3 result, this leaves Intercept as the current leader in the treatment race. Two more companies, Genfit and Allergan, are finishing up Phase 3 trials, but Allergan has recently delayed their trial conclusion until 2020.