EDP-305 Targets NASH in Phase 2 Trial Testing Two Doses Against Placebo
Enanta Pharmaceuticals tests its FXR agonist EDP-305 in a completed Phase 2 randomized trial for non-alcoholic steatohepatitis.
Summary
Non-alcoholic steatohepatitis, or NASH, is a progressive liver disease with no approved treatments that can cause cirrhosis and liver failure. Enanta Pharmaceuticals ran a completed Phase 2 randomized, double-blind, placebo-controlled trial of EDP-305, a farnesoid X receptor agonist, in NASH patients. Two doses of EDP-305 were tested against placebo to evaluate safety, tolerability, how the drug behaves in the body, and whether it reduces liver inflammation and scarring. NASH is closely linked to obesity, insulin resistance, and metabolic syndrome, making it highly relevant to longevity and metabolic health. Results from this trial have informed ongoing development of FXR-targeting therapies. The study represents an important step in a therapeutic area with significant unmet need affecting millions worldwide.
Detailed Summary
Non-alcoholic steatohepatitis is one of the most consequential and underappreciated metabolic diseases of our time. It affects an estimated 3–5% of the global population, causes progressive liver fibrosis, and is a leading driver of liver transplantation. Despite its prevalence, no broadly approved pharmacological treatment existed at the time this trial was initiated, making clinical development efforts critically important.
This Phase 2 study, sponsored by Enanta Pharmaceuticals, evaluated EDP-305, a potent and selective agonist of the farnesoid X receptor, a nuclear receptor that regulates bile acid metabolism, lipid homeostasis, and hepatic inflammation. The farnesoid X receptor pathway has emerged as a compelling target in NASH because its activation suppresses liver fat accumulation, reduces inflammatory signaling, and may slow fibrosis progression.
The trial was designed as a randomized, double-blind, placebo-controlled study comparing two doses of EDP-305 against placebo in subjects with confirmed NASH. Primary endpoints included safety and tolerability, with pharmacokinetic profiling and efficacy signals also assessed. The double-blind design and inclusion of two active doses allowed the investigators to evaluate dose-response relationships alongside safety margins.
The study has been completed, positioning its results as a foundation for potential Phase 3 development or dose selection decisions. FXR agonism as a class has shown mixed results in NASH trials, with some compounds limited by side effects such as pruritus, making EDP-305's tolerability profile a key point of interest.
For clinicians managing patients with metabolic syndrome, obesity, or type 2 diabetes, NASH represents a serious downstream complication. Novel pharmacological tools targeting its root molecular pathways could meaningfully extend healthspan. Caveats include limited public data available from this abstract alone, and the field awaits full publication of results.
Key Findings
- EDP-305 is an FXR agonist tested at two doses versus placebo in confirmed NASH patients.
- Phase 2 design evaluated safety, tolerability, pharmacokinetics, and early efficacy signals.
- NASH has no broadly approved treatment, making this trial clinically significant.
- FXR pathway activation targets liver fat, inflammation, and fibrosis simultaneously.
- Trial is completed, with results relevant to future Phase 3 dose selection.
Methodology
This was a randomized, double-blind, placebo-controlled Phase 2 trial conducted by Enanta Pharmaceuticals in subjects with confirmed non-alcoholic steatohepatitis. Two active doses of EDP-305 were compared against placebo, enabling dose-response assessment alongside safety and pharmacokinetic profiling. The completed status indicates all participants have finished the protocol.
Study Limitations
This summary is based on the abstract and ClinicalTrials.gov registration only, as the full study data are not publicly available. Key efficacy and safety outcomes, including histological endpoints and adverse event rates, cannot be assessed without access to the full results. The trial was registered in 2018, and publication status of full results is unknown from available information.
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