Genfit’s most advanced liver disease drug elafibranor has today achieved a breakthrough tag from the US FDA following positive data from a phase 2 trial.
The move comes just a few weeks after Genfit launched a $135m IPO to help it complete phase 3 trials of the first-in-class drug in non-alcoholic steatohepatitis (NASH), which is due to finish towards the end of this year.
However, today’s breakthrough designation for elafibranor is in the Primary Billary Chloangitis (PBC) setting, a chronic autoimmune disease where bile ducts located in the liver are gradually destroyed, potentially leading to cirrhosis.
During a phase 2 trial, those dosed with elafibranor showed a significant decrease of alkaline phosphatase (ALP) levels, resulting in significant treatment effects versus placebo on the primary endpoint.
These results were from non-cirrhotic patients with PBC, and with those who had inadequate response to ursodeoxycholic acid (UDCA), the current standard of care.
In addition to significant reductions in ALP, those in both elafibranor-treated groups showed improvements in other PBC markers, including gamma-glutamyl transferase (GGT), lipid markers and anti-inflammatory markers.
“Elafibranor has shown promising anticholestatic effects in a Phase 2 clinical trial,” said Pascal Birman, Deputy Chief Medical Officer, Genfit.
“The FDA’s decision to grant elafibranor the Breakthrough Therapy Designation on the basis of our Phase 2 data, is of course a milestone that will allow us to accelerate elafibranor’s development, and further confirms our strong belief that elafibranor could potentially address this significant unmet medical need.”
Approximately half of patients have an inadequate response to existing therapies, either because they are unresponsive to treatments already on the market or they experience intolerable side effects such as aggravated pruritus (itching).
Genfit’s drug has been shown to reduce this however, operating as a dual agonist of the peroxisome proliferator-activated receptor (PPAR) alpha and delta pathways.
The drug was initially developed to treat those with NASH, which the company believes it has the potential to address multiple facets of that disease, including inflammation, insulin sensitivity, lipid/metabolic profile, and liver markers.
It has a lot riding on the drug, especially in NASH where drug developers are racing to get their candidates on the market first.
Although Genfit has already suffered a setback in the area after elafibranor failed a phase 2 trial in 2015, and Evaluate Pharma analysts have said hopes for the drug’s success in NASH are already low.
Meanwhile, Cymabay has its own NASH PPAR candidate in its pipeline, which is due for a crucial data readout in mid-2019.
The US based biopharma claims its drug can reduce fat associated with NASH by 30%, suggesting a strong competitive profile.