French biopharmaceutical firm Genfit has received protocol clearance from the US Food and Drug Administration (FDA) to conduct a Phase II clinical trial of elafibranor in children and adolescents suffering from non-alcoholic steatohepatitis (NASH).
Elafibranor is a dual agonist of the PPARα and PPARδ receptors that play a key role in various processes related to the development of NASH and its co-morbidities.
The new randomised, open-label trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of 80mg and 120mg elafibranor over 12 weeks in 20 participants aged eight to 17 years across multiple US sites.
Genfit chief operating officer Dean Hum said: “The initiation of a Phase II trial of elafibranor in paediatric NASH is a landmark study, as to our knowledge there has been no other molecule that has shown clinical evidence in a Phase IIb adult NASH trial and then progressed to clinical evaluation in the paediatric NASH setting.
“Beyond the clinical usage of elafibranor, we believe the scientific data generated throughout the trial will be highly beneficial for physicians tackling this disease and impactful for the still immature field of paediatric NASH.”
The paediatric trial builds on findings from a prior Phase II trial conducted in adults with NASH.
Results showed that the drug achieved efficacy on the FDA-recommended endpoint of NASH resolution without worsening of fibrosis. Elafibranor also demonstrated beneficial cardiovascular profile, without any safety and tolerability concerns.
Based on this data, the company obtained the Pediatric Study Plan (PSP) and the Pediatric Investigation Plan (PIP) agreements from the FDA and the European Medicines Agency (EMA), respectively.
The company is also developing the drug for the treatment of primary biliary cholangitis (PBC).