(News Bulletin 247) – The Lille biotech is soaring on the Paris Stock Exchange after the publication of positive results from a clinical study, carried out in partnership with Ipsen for its flagship molecule in a rare liver disease.

Ipsen and Genfit announce a major breakthrough for elafibranor, an experimental treatment for primary biliary cholangitis (PBC). The two partners indicated Friday morning positive results of the phase III trial (last step before potential marketing) ELATIVE evaluating this molecule in patients with this rare liver disease.

The trial met the primary endpoint, with 51% of patients treated with elafibranor at an 80mg dose experiencing an improvement in cholestasis in response to treatment, compared to only 4% of patients tested with placebo .

The secondary endpoint, normalization of alkaline phosphatase (ALP) level after one year, was also achieved with a statistically significant improvement in favor of elafibranor, again compared to placebo. Also, Genfit reports that elafibranor was well tolerated with “a consistent safety profile” compared to previous studies.

On the Paris Stock Exchange, Genfit ends this week with a bang with a title that soars by 25% to 4.795 euros when Ipsen gains, for its part, 0.4% to 111 euros around 11:10 a.m.

Recall that primary biliary cholangitis or PBC (formerly called primary biliary cirrhosis) is a chronic autoimmune disease, during which the bile ducts of the liver are gradually destroyed, leading to liver damage and possibly leading to cirrhosis. Common symptoms of PBC include general fatigue and pruritus (itching) which can be particularly debilitating.

If left untreated, PBC can lead to liver failure or, in some cases, death. It is also one of the main causes of liver transplantation. The only treatment has long been ursodeoxycholic acid (in particular Ursolvan, Delursan) but in recent years new therapeutic options have appeared, including the drug candidate of Genfit.

A positive success for elafibranor

Reinforced by these positive results in primary biliary cholangitis, Ipsen has indicated its intention to submit authorization applications for elafibranor following exchanges with the American (Food and Drug Administration) and European (European Medicines Agency ).

For Royal Bank of Canada (RBC), this new milestone is positive for Genfit and Ipsen. “The success of the ELATIVE study is positive for elafibranor and should add a new engine of growth to Ipsen’s portfolio, offsetting the headwind from Somatuline”, the family group’s flagship cancer drug which is now in competition with a generic in Europe and the North American market.

The choice to focus on specialty medicine with the greatest potential should begin to bear fruit for Ipsen. At the end of 2020, the family group had signaled to the market its desire to shed its family health division – including the famous drug Smecta – to focus on the therapeutic areas of oncology (cancer treatments), rare diseases and neurosciences. .

To this end, the pharmaceutical group acquired the rights to elafibranor, Genfit’s flagship molecule, at the end of 2021 as part of a global partnership. Also, Ipsen took 8% of the capital of Genfit and paid it 120 million euros upon signing this agreement. The northern group is eligible for additional payments linked to the achievement of regulatory and commercial milestones and linked to sales of up to 360 million euros (i.e. up to 480 million euros in total).

And if successfully marketed, Genfit will additionally receive tiered royalties with “a double-digit percentage of up to 20% of sales. Royal Bank of Canada is planning a 2025 launch for elafibranor, and tables on “sales reaching nearly 300 million euros by 2029”.