ENYO Pharma has reported that its drug candidate, Vonafexor (EYP001), met the primary and multiple secondary goals in the Phase IIa LIVIFY clinical trial in patients with F2-F3 non-alcoholic steatohepatitis (NASH).
A synthetic non-steroidal, non-bile acid, farnesoid X receptor (FXR) agonist, Vonafexor can offer preferential delivery to the liver and lasting target engagement.
The orally bioavailable drug is also being analysed for the treatment of chronic Hepatitis B (cHBV).
The Phase IIa trial enrolled 96 NASH patients, who were randomised in a 1:1:1 ratio to be given once-daily doses of Vonafexor 200mg or 100mg or placebo.
Data showed that Vonafexor reduced liver fat content, thereby meeting the trial’s primary goal. A 6.3% absolute decrease in liver fat content was observed in the 100mg arm and 5.4% in the 200mg arm versus 2.3% in the placebo arm.
Furthermore, in the 100mg and 200mg cohort, Vonafexor offered 30.5% and 25.3% mean relative reductions, respectively, as against 10.6% in the placebo arm.
No substantial differences in efficacy goals were noted between the 100mg and 200mg treatment arms, ENYO said.
The company added that the drug was safe and well-tolerated in the trial and is the first FXR agonist to improve renal function with 12 weeks of therapy.
In addition, treatment with Vonafexor demonstrated a significant effect on various parameters linked to metabolic as well as cardio-vascular morbidity benefits.
The rise in low-density lipoprotein cholesterol associated with Vonafexor usage was in line with other FXR agonists.
In the trial, generally mild, transient and localised pruritus was reported in the treatment groups, the company said.
ENYO Pharma chief medical officer Pietro Scalfaro said: “We were very pleased to obtain these promising results with Vonafexor, which indicate the potential of the product to offer rapid improvements in liver function and liver fat for NASH patients for whom there is no approved therapy.”
Apart from the significant effects on liver parameters, the improvement in kidney function with Vonafexor treatment is expected to benefit those NASH patients who are commonly affected by co-morbidities such as diabetes, which substantially impact kidney function.