Genfit enrolls first patients in its liver disease drug test

Genfit, a biopharmaceutical company focused on developing therapeutic and diagnostic solutions in metabolic and inflammatory diseases, that notably affect the liver or the gastrointestinal system, has started patient testing in the Phase 2a trial evaluating elafibranor in PBC.

The company explained that the trial is designed to be a multicenter, double-blind, randomized, placebo-controlled, Phase 2a study to evaluate the efficacy and safety of elafibranor in adult patients with PBC and inadequate response to ursodeoxycholic acid (UDCA).

The primary objective is to determine the effect of daily oral administration of elafibranor on serum alkaline phosphatase (ALP) in these patients, based on relative change from baseline to end of treatment compared to placebo, the company said.

Dr. Velimir A. Luketic, MD, Division of Gastroenterology, Hepatology and Nutrition Virginia Commonwealth University School of Medicine, Richmond, VA (USA), commented: “PBC is a chronic progressive liver disease characterized by immune mediated destruction of the small intrahepatic bile ducts that if untreated progresses to end-stage liver disease and liver failure. A substantial number of patients do not benefit from the currently available therapies – UDCA or OCA – either because of lack of response or intolerable side effects. This represents a major unmet need for this population. In the literature, targeting PPAR receptors have shown to reduce the synthesis of bile acids and to improve detoxification of bile in the bile duct. In clinical trials, PPAR targeting drugs have shown significant decrease in ALP, and improved biochemical profiles and pruritus in PBC patients.”

Sophie Mégnien, Chief Medical Officer of GENFIT, added: “We are excited to have this first randomized PBC patient in this Phase 2 trial and advance our PBC program. Elafibranor, a dual PPAR alpha & delta agonist, is an attractive candidate for PBC patients due to its impact on lowering alkaline phosphatase levels, as shown in previous studies, on other populations. This attribute, along with what PPARs have consistently demonstrated on ALP reduction, provides a strong rationale for elafibranor in PBC. Starting randomization in such a rare disease is a key milestone, and we hope elafibranor will provide a meaningful benefit to patients, and will ultimately address the unmet need.”