Benzinga | Sep 14, 2020 07:04AM EDT

Enanta Pharmaceuticals Initiates Phase 1 Clinical Study Of EDP-297 Targeted Follow-On Farnesoid X Receptor Agonist For Non-Alcoholic Steatohepatitis

-- Initial clinical data including safety, tolerability and pharmacokinetics expected in 2Q 2021 --

Enanta Pharmaceuticals, Inc., (NASDAQ:ENTA), a clinical stage biotechnology company dedicated to creating small molecule drugs for viral infections and liver diseases, today announced that it has dosed the first subjects in its Phase 1 clinical trial of EDP-297, a highly potent and targeted follow-on farnesoid X receptor (FXR) agonist, being developed for the treatment of non-alcoholic steatohepatitis (NASH).

"We are excited to advance our efforts in NASH and progress EDP-297, our follow-on FXR agonist, into clinical development," commented Jay R. Luly, Ph.D., President and Chief Executive Officer of Enanta Pharmaceuticals. "In preclinical studies, EDP-297 demonstrated a compelling product profile, with a potency greater than that published on any FXR agonist in clinical development and high target-tissue distribution in the liver and intestine. Based on these data, we believe we may be able to effectively dose EDP-297 at lower doses and with reduced drug levels in non-targeted tissues, potentially improving tolerability by reducing pruritis. We look forward to reporting clinical data in the second quarter of 2021."

The Phase 1, randomized, double-blind, placebo-controlled, first-in-human study is designed to assess the safety, tolerability, and pharmacokinetics, including the effect of food intake, of orally administered EDP-297 in approximately 74 healthy adult subjects. Two phases are planned: a single ascending dose phase enrolling six cohorts, including a two-part food effect cohort, and a multiple ascending dose phase enrolling three cohorts.

In two recent poster presentations at the European Association for the Study of the Liver (EASL) Digital International Liver Congress(tm) 2020, treatment with EDP-297 demonstrated significantly reduced fibrosis progression and improved liver function in a rat model of NASH. Additionally, in 3D NASH microtissues, EDP-297 modulated multiple pathways associated with the pathogenesis of NASH, including decreased expression of genes encoding multiple lipogenic and inflammatory proteins, and significantly reduced expression of inflammatory and fibrotic genes and normalized circulating markers of liver injury.