Intercept Pharmaceuticals Inc (NASDAQ: ICPT) — which is a frontrunner along with Gilead Sciences, Inc. (NASDAQ: GILD) in the non-alcoholic steatohepatitis drug development race — reported positive top-line results Tuesday for a late-stage study of its pipeline asset Ocaliva, chemically obeticholic acid.
Gilead previously reported disappointing results for a Phase 3 study that evaluated its NASH candidate selonsertib.
Intercept said Ocaliva 25 mg, which is being evaluated in a late-stage study dubbed REGENERATE, achieved the primary endpoint of demonstrating statistically significant improvement in liver fibrosis without worsening of NASH at 18 months.
Primary efficacy analysis also revealed a numerically greater proportion of patients in the treatment arm met the primary endpoint of NASH resolution with no worsening of liver fibrosis relative to the control arm, although it did not achieve statistical significance.
"As agreed with the U.S. Food and Drug Administration, in order for the primary objective to be met, the study was required to achieve one of the two primary endpoints," Intercept said.
The primary efficacy analysis assessed efficacy at 18 months in 931 patients with stage 2 or stage 3 liver fibrosis due to NASH. The patients were randomized to receive a placebo, Ocaliva 10 mg or Ocaliva 25mg once daily.
Why It's Important
NASH affects about 30 million people in the U.S., or about 12 percent of adults, according to National Institutes of Health estimates, and there is no FDA-approved therapy for the condition. It presents a $20-billion to $35-billion opportunity, according to Biopharmadrive, which cited analyst estimates.
"We are thrilled to report the first positive registrational Phase 3 study results in patients with NASH, a devastating disease that is on track to become a leading cause of liver transplant in coming years," Intercept CEO Mark Pruzanski said in a statement.
"The topline REGENERATE data we are reporting today support our belief that OCA will become the first approved medicine for those living with liver fibrosis due to NASH."
Following Gilead's failed STELLAR-4 study and the likelihood of STELLAR-3 study also failing, Ocaliva is likely to launch in NASH unencumbered by competition, Raymond James analyst Steven Seedhouse said in a note last week.
"Therefore, safety/tolerability questions that we know will be apparent (lipid changes and pruritus) likely won't overshadow an efficacy win, especially in top-line data," the analyst said.
Raymond James has a Strong Buy rating on Intercept.
Analysts also believe Gilead might be interested in buying out Intercept as it searches for a NASH candidate.
Intercept said it intends to file for regulatory approvals in the U.S. and Europe in the second half of 2019. The company also said it will present the results of the REGENERATE study at the European Association for the Study of the Liver: The International Liver Congress 2019.
Intercept shasres hit a five-month high Tuesday and were trading up 11.37 percent at $123.42 at the time of publication.
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