In this 24-week clinical trial, lanifibranor, an orally-available small molecule and the only pan-PPAR agonist5 currently in clinical development for the treatment of NASH, met the primary endpoint in the ITT population at the dose of 1200mg/day with a statistically significant (p = 0.004) decrease of at least two points in the SAF activity score6 (combining hepatocellular inflammation and ballooning), compared to baseline, with no worsening of fibrosis. 49% of patients in the lanifibranor 1200mg/day dose group achieved the primary endpoint compared to 27% in the placebo arm.
Lanifibranor also met multiple key secondary endpoints including:
- Resolution of NASH with no worsening of fibrosis in both dose groups (800mg/day and 1200mg/day)
- Improvement of fibrosis by at least one stage7 with no worsening of NASH at the 1200mg/day dose group
- NASH resolution and improvement of fibrosis in both dose groups (800mg/day and 1200mg/day)
Statistically significant results were also obtained in both dose groups (800mg/day and 1200mg/day) on:
- Decrease of insulin, fasting glucose and glycated haemoglobin (HB1AC) in patients with type 2 diabetes
- Decrease in triglycerides
- Increase in high density lipoprotein cholesterol (HDL)
- Decrease in liver enzymes (ALT, AST and GGT)
With these results, lanifibranor is the first drug candidate to achieve statistically significant results on the two Food and Drug Administration (FDA) and European Medicine Agency (EMA) primary endpoints8 relevant for seeking accelerated approval during Phase III clinical development.